Medicines in Development Cancer

2014

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In recent decades, great progress has been made in the fi ght against cancer. Advances in molecular and genomic research have unveiled complexities and changed the perception of cancer, which we now know is more than 200 unique diseases. Continued research has expanded our knowledge of how cancer develops and how to target medicines for specifi c cancer types, which has resulted in more effective therapies for patients.

A cancer diagnosis once meant almost certain death but with new therapies, better testing, earlier diagnosis and follow-up care, patients are often able to live longer with a better quality of life. According to the American Cancer Society (ACS), the number of cancer survivors living in the United States has increased from 3 million in 1971 to 13.7 million in 2012.

Although great progress has been made and continues to be made against cancer, it remains the second leading cause of death in the United States, behind heart disease, accounting for nearly 1 of every 4 deaths. The ACS estimates that in 2014, more than 1.6 million new cancer cases will be diagnosed and nearly 600,000 Americans will die from cancer, approximately 1,600 people per day.

America’s biopharmaceutical companies are responding to the needs of cancer patients, working to develop more and better treatments. Researchers are now exploring new high-tech weapons to fi ght the disease as well as ways to use existing medicines, either alone or in combination with other therapies, to treat various cancers. The 771 medicines and vaccines are either in clinical trials or awaiting review by the U.S. Food and Drug Administration (FDA) and include:

• A monoclonal antibody for colorectal cancer that targets a cell surface protein that plays a role in tumor growth and metastases;

Nearly 800 Medicines and Vaccines in Clinical Testing For Cancer Offer New Hope to Patients

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Medicines in Development for Cancer

Some medicines are listed in more than one category.

MEDICINES IN DEVELOPMENT

CancerPRESENTED BY AMERICA’S BIOPHARMACEUTICAL RESEARCH COMPANIES

ContentsMedicines and Vaccines in Development to Treat Cancer ...............2

Advances in Cancer Treatment .............3

Value in Cancer Carein the United States ............................5

Research Collaboration: Public-Private Partnerships ...................................... 6

Facts About Cancer in the United States .................................... 8

Medicines and Vaccines in Development Chart ........................... 12

The Drug Discovery, Development and Approval Process ...................... 103

Medicines in Development Cancer 20142

• A monoclonal antibody for non-small-cell lung cancer that blocks a signaling pathway allowing for immune responses that can recognize and destroy cancer cells;

• A medicine that targets and disrupts a signaling pathway associated with tumor growth in stomach cancer.

The development of new therapies would not be possible without the patients who volunteer to participate in clinical trials. Currently, there are 3,137 active clinical trials for cancer in the United States. Of those, 1,824 are now seeking volunteers to participate or have not yet started recruiting patients; the other 1,313 are ongoing but are not currently recruiting new patients.*

The progress made against cancer has resulted in better treatments that help extend lives, improve patients’ quality of life, and increase productivity contributing to economic growth. The hundreds of medicines being developed are our best hope for continuing that progress and lessening the burden of cancer. However, researching and developing new medicines remains a risky investment and a lengthy process. On average, it costs $1.2 billion and takes between 10–15 years to bring a new, effective medicine to patients. Despite those risks, America’s biopharmaceutical research companies are continuing their efforts to develop effective new therapies for the prevention and treatment of cancer.

Medicines and Vaccines in Development to Treat Cancer Many of the medicines in the pipeline today are using novel approaches to attack cancer at the molecular level. Approximately 80 percent of cancer pipeline drugs are potentially fi rst-in-class treatments, according to the Tufts Center for the Study of Drug Development. Among the 771 medicines and vaccines in development are potential treatments for:

Colorectal Cancer—A humanized monoclonal antibody in development for metastatic colorectal cancer targets the cell surface protein “endosialin,” which is expressed on cells that are part of the tumor blood vessel structure. In preclinical studies, blocking the function of endosialin inhibited tumor growth and metastases.

Liver Cancer—A medicine in development for hepatocellular carcinoma (liver cancer) is a small molecule kinase inhibitor designed to selectively block transforming growth factor-beta (TGF-beta) signaling. The overexpression of TGF-beta in cells may enhance tumor growth and intensify metastases.

Key Issues

*Source: www.clinicaltrials.gov, search performed 9/25/2014. Search criteria: cancer, United States, Phase 0, 1, 2, 3; industry only; excluding unknown status.

Medicines in Development Cancer 2014 3

Lung Cancer—A fully human monoclonal antibody in development for non-small-cell lung cancer targets the PD-1 (programmed death-1) checkpoint receptor which is expressed on activated T-cells. Cancer cells may exploit the checkpoint pathway to hide from the immune system and protect the tumor. Blocking this pathway may allow for immune responses that recognize and destroy cancer cells. Another medicine which blocks the PD-1 receptor was recently approved by FDA for the treatment of malignant melanoma.

Pancreatic Cancer—In malignant solid tumors, oxygen levels are often low (called tumor hypoxia) relative to oxygen levels in healthy tissue. Tumor hypoxia is associated with tumor progression, metastases, and resistance to chemotherapy and radiation treatment. A medicine in development is activated when it reaches the hypoxic region of the tumor environment where it eventually kills tumor cells in its vicinity. The medicine is also being tested in patients with soft tissue sarcoma.

Stomach Cancer—A monoclonal antibody in testing for stomach cancer with high MET (mesenchymal-epithelial transition factor) expression is designed to inhibit hepatocyte growth factor (HGF)/scatter factor from binding to the c-MET receptor. The HGF/c-Met signaling pathway is

thought to play a role in tumor growth and metastases in many cancers, including stomach cancer. An exploratory biomarker analysis found that the addition of the medicine to chemotherapy improved median overall survival in patients with tumors that expressed high levels of MET protein.

Advances in Cancer TreatmentAs we continue to advance cancer treatment, progress can be seen in declining death rates from cancer and increases in cancer survivors. According to the ACS, the death rate from cancer has been steadily declining since the 1970s—declining more than 15 percent in the period from 2000-2011 alone. Five-year survival rates have increased 39 percent across all cancer types since 1975, with 2 out of 3 people diagnosed with cancer surviving at least fi ve years.

To sustain this progress in an environment of increasing pressure to contain health care costs, it is important to understand the valuable role new medicines play in improving patient outcomes. According to the ACS, 83 percent of survival gains are attributable to new treatments, including medicines. Some key advances in treatment include:

Personalized Medicines—Personalized medicines are therapeutics that are tailored to individual patients based on certain biologic and physiologic characteristics, such as genetic makeup. Patients can be identifi ed by their susceptibility to a certain disease or how they will likely respond to a specifi c therapy. By targeting therapies to patients most likely to benefi t, personalized medicines represent an important tool, as they may reduce the use of

Key Issues

“ � ere is no question that when we talk about turning the tide against cancer, the most exciting opportunities, the new opportunities in fact, are understanding the biology and applying that biology to new treatments. We are certainly at a turning point.”—J. Leonard Lichtenfeld, M.D., American Cancer Society


unnecessary and often costly treatments or procedures in patients who are unlikely to benefi t from them. Innovative personalized medicines make up 12 to 50 percent of the pipeline for all diseases, according to the Tufts Center for the Study of Drug Development.

Targeted Therapies—Targeted therapies are designed to interfere with specifi c molecular targets involved in cancer cell growth or progression, whereas traditional chemotherapy acts against all rapidly dividing cells, including healthy ones. Several targeted therapies have been approved for use by the FDA. According to IMS Health, the role of targeted therapies in oncology treatment has grown from 11 percent in 2003 to 46 percent in 2013.

Therapeutic Cancer Vaccines—In the late 1990s, scientists began experimenting with new vaccines that could harness the power of the immune system to fi ght cancer rather than to prevent it. One type of therapeutic vaccine, called autologous cellular immunotherapy, takes a patient’s own immune cells and reprograms them to attack the cancer. The fi rst and only therapeutic cancer vaccine was approved by the FDA in 2010 to treat metastatic prostate cancer.

Preventive Vaccines—Preventive vaccines target viruses or other infectious agents that can cause or contribute to the development of cancer. They are intended to prevent people from being infected with those viruses and agents so that they do not develop cancer. For example, nearly all cases of cervical cancer can be attributed to human papillomavirus (HPV), with two HPV subtypes (types 16 and 18) alone causing 70 percent of cervical cancer cases. The FDA has

Key Issues

HOW CANCER COULD ECHO HIV SUCCESS

Innovations in cancer treatment could echo the success we have seen in HIV/AIDS treatment. Advances in medicines for HIV/AIDS have helped lower the death rate by 83 percent since the disease was fi rst recognized in 1981. Prior to 1995, when the fi rst antiretroviral medicine was approved by the FDA, an HIV diagnosis was a death sentence. Now, thanks to medicines developed by biopharmaceutical scientists and their research partners, it is a chronic condition with manageable costs, and patients are able to reach nearly full life expectancy.


approved vaccines to prevent infection by HPV types 16 and 18 and vaccines to prevent hepatitis B virus, which can cause liver cancer.

Value in Cancer Care in the United StatesNew cancer medicines bring tremendous value to cancer patients and the U.S. health care system. Medicines are an important part of cancer treatment but represent just a small portion of all cancer costs. When compared to total

“ We used to think HIV costs would overwhelm us…but we � gured it out and let drug development progress…similarly, cancer care will evolve.”

—Ira Klein, M.D., M.B.A., FACP, Aetna

18Bladder Cancer47Brain Cancer

73Breast Cancer46

19Colorectal Cancer

63Head/Neck Cancer

24Kidney Cancer87Leukemia

37Liver Cancer98Lung Cancer

78Lymphoma33Myeloma

48Ovarian Cancer39Pancreatic Cancer

45Prostate Cancer11Sarcoma

56Skin Cancer213Solid Tumors

24Stomach Cancer36Other Cancers

53Unspecified Cancer

Hematological Malignancies

Application SubmittedPhase III

Phase II

Phase I

Medicines in Development By Disease and PhaseSome medicines are listed in more than one category.


health care spending, cancer care represents an even smaller portion of costs.

• Spending on cancer medicines represents less than 1 percent of all health care spending for all diseases —$25.9 billion out of $2,800 billion.1

• Cancer medicines represent 20 percent of total spending on cancer care.2

• Between 1988 and 2000, 23 million years of life were saved due to cancer treatment advances. During that time, the United States realized $1.9 trillion in value due to improved productivity, extended life, and other factors resulting from improved cancer treatment.3

• On average, privately insured cancer patients pay out-of-pocket more than 20 percent of their total prescription drug costs, compared to 4 percent of inpatient hospital care and 7 percent of outpatient care.4

Sources: 1. IMS Institute for Healthcare Informatics, “Declining Medicine Use and Costs: For Better or Worse? A Review of the Use of Medicines in the United States in 2012.” 2. K. Fitch et al, “Benefi t Designs for High Cost Medical Conditions,” Milliman Research Report. 3. Lakdawalla et al, “An Economic Evaluation of the War on Cancer,” Journal of Health Economics.4. PhRMA analysis based on Medical Expenditure Panel Survey (MEPS).

Research Collaboration: Public-Private PartnershipsAs scientists probe deeper into the causes and signs of cancer, the amount of information has increased exponentially, and making sense of all the data is a colossal undertaking that no single individual or institution can handle alone.

As a result, biopharmaceutical research companies are working together and with academic medical centers, government, and non-profi t organizations to share, organize and make sense of huge volumes of information. Below are examples of collaboration to advance progress against cancer.

The Cancer Genome Atlas (TCGA) is a project to create a catalog (or atlas) of genomic changes associated with specifi c types of tumors to improve the prevention, diagnosis, and treatment of cancer through the collection and analysis of tissue samples. Sponsored by the National Cancer Institute and the National Human Genome Research Institute, the TCGA network includes scientists, bioinformaticians, bioethicists, doctors, nurses, advocates, and others. TCGA is currently focused on 32 tumor types, including nine rare cancers.


Lung-MAP (Lung Cancer Master Protocol) is a multi-drug, multi-arm, biomarker-driven clinical trial focused on developing new treatments for lung cancer. The trial will recruit patients with advanced squamous cell lung cancer, which represents approximately 25 percent of all lung cancers, from more than 500 medical centers in the United States. Unlike traditional clinical trials, Lung-MAP uses advanced molecular screening to match patients to an investigational treatment targeted to the genetic alterations driving their cancer. Lung-MAP is a joint effort by government, biopharmaceutical companies, patient advocacy groups, and research institutions designed to improve clinical trial effi ciency and make new targeted therapies in lung cancer available as quickly and safely as possible.

I-SPY 2 is a research collaboration among the Biomarkers Consortium, the FDA, the U.S. National Institutes of Health, and biopharmaceutical companies. I-SPY 2 uses genetic and biological markers from individual patient tumors and adaptive clinical trial design to develop new breast cancer medicines quicker by identifying which ones are most

effective in which patients and at a reduced cost. I-SPY 2 is focused on women with newly diagnosed, locally-advanced breast cancer and will test whether adding investigational drugs to standard chemotherapy is better than standard chemotherapy alone prior to having surgery.

The CEO Roundtable on Cancer is committed to improving the health of employees and making progress against cancer. The Roundtable is comprised of executives from diverse American corporations, nonprofi t organizations fi ghting cancer, and National Cancer Institute-designated cancer centers. Programs initiated by the Roundtable include the CEO Cancer Gold Standard, a workplace-based wellness accreditation program that provides a framework for employers to lower the risk of cancer, detect it early, and ensure access to high-quality care. The Life Sciences Consortium brings together leading biopharmaceutical research companies focused on cancer in collaboration to effectively deliver new medicines to prevent and treat cancer and continue the emerging understanding of cancer biology.

RESEARCHING CANCER MEDICINES: SETBACKS AND STEPPING STONESWith the many advances made in cancer research, there are also setbacks. Researchers are discovering that cancer is actually a set of more than 200 extremely complex diseases. Finding medicines that effectively treat each one is an immense challenge. The path to new oncology drugs is littered with so-called failures that do not make it through human clinical trials to approval.

Although it is very disappointing to see a promising drug candidate cut from the pipeline, researchers learn from every setback and build upon each one of them to achieve success with future research and get effective therapies to patients.

A new PhRMA report looks at setbacks and approvals in melanoma, brain cancer and lung cancer between 1998 and 2014:

• Researchers have had recent successes in treating melanoma with the approval of seven new medicines, building on 96 medicines that didn’t make it through clinical trials.

• There has been progress against some subsets of lung cancer, but the disease remains deadly. Ten lung cancer medicines were approved, while another 167 potential medicines failed in clinical testing.

• Brain cancer continues to be a diffi cult cancer to treat despite concentrated research. There were three approvals and 75 unsuccessful candidate medicines.

Although these setbacks are valuable to researchers, they are also an indication of the diffi culty in developing medicines to treat cancer. These setbacks serve as a reminder that to make progress, we need a public policy framework that supports the drug development process as well as the promising science so that important innovations can reach patients.


Facts

• Cancer is the second leading cause of death by disease—nearly 1 of every 4 deaths—in the United States, exceeded only by heart disease.1

• Men have slightly less than a 1 in 2 lifetime risk of developing cancer; for women, the risk is a little more than 1 in 3. Approximately 77 percent of all cancers are diagnosed in people ages 55 and older.1

• This year alone, more than 1.6 million new cancer cases are expected to be diagnosed, and an estimated 585,720 people are expected to die of cancer.1

• Approximately 13.7 million Americans with a history of cancer were alive in January 2012.1 The 5-year survival rate for all cancers diagnosed between 2004 and 2010 is 66 percent, up from 49 percent in the mid-70s.2

• Lung cancer is the leading cause of cancer death in both men and women. An estimated 159,260 deaths, accounting for about 27 percent of all cancer deaths, are expected to occur in 2014. From 2006 to 2010, rates decreased 2.9 percent per year in men and 1.4 percent per year in women.1

• Colorectal cancer is the second leading cause of cancer death and accounts for 8.2 percent of all new cancer cases. It is more common in men than in women, and more common in African Americans.2

• Prostate cancer is the second leading cancer cause of death in men with nearly 30,000 deaths estimated this year. Prostate cancer death rates have been decreasing since the early 1990s in men of all races/ethnicities, though they remain more than twice as high in African Americans as in any other ethnicity.1

• An estimated 40,430 breast cancer deaths are expected in 2014, with men accounting for 430 of those. Breast cancer is the second cause of cancer death in women. From 2006 to 2010, rates decreased 3.0 percent per year in women under age 50 and 1.8 percent per year in women 50 and older. The decrease in breast cancer death rates represents improvements in early detection and treatment, and possibly decreased incidence.1

• This year nearly 300,000 new cases of gastrointestinal cancer will be diagnosed and nearly 150,000 deaths will occur in the United States.2

• Pancreatic cancer is the fourth leading cause of cancer death. The average survival rate from pancreatic cancer is low, in part because it is diffi cult to detect early. It is most frequently diagnosed among people aged 75-84.2

• Stomach cancer accounts for 1.3 percent of all new cancer cases and mostly affects older people with an average age of diagnosis at 69. Approximately 60 percent of people diagnosed with stomach cancer each year are age 65 or older.2

• Liver cancer and intrahepatic bile duct cancer is the seventh leading cause of cancer death. It is also more common among Asian/Pacifi c Islander and American Indian/Alaska Native populations.2

• In 2014, an estimated 10,450 new cases of cancer will be diagnosed and 1,350 cancer deaths will occur in children ages 0-14. Among adolescents ages 15-19, 5,330 new cases will be diagnosed and 610 deaths will occur.1

• The most common cancers among children are acute lymphoblastic leukemia (26 percent), brain and central nervous system (CNS) cancers (21 percent), neuroblastoma (7 percent), and non-Hodgkin lymphoma (6 percent).1

• Among adolescents the most common cancers are Hodgkin lymphoma (15 percent), thyroid carcinoma (11 percent), brain and CNS cancers (10 percent), and testicular germ cell tumors (8 percent).1

Overview

• Although extraordinary advances are being made against cancer, the disease remains a major health care challenge and a huge fi nancial burden, both nationally and internationally.3

• The National Institutes of Health (NIH) estimates the overall costs for cancer in 2009 at $216.6 billion: $86.6 billion for direct medical costs (total of all health expenditures) and $130 billion for indirect mortality costs (cost of lost productivity due to premature death).3

• Globally, 12.9 million new cancer cases cost $286 billion in 2009. In 2030, the 21.5 million projected new cancer cases will cost the world economy $458 billion.3

Costs

Facts About Cancer in the United States


Facts

New Cancer Cases/Deaths1

Cancer Type Estimated 2014 New Cases Estimated 2014 Deaths

All Sites ........................................................... 1,665,540 ............................................................. 585,720

Male ....................................................... 855,220 ..............................................................310,010

Female .................................................... 810,320 .............................................................. 275,710

Acute Lymphocytic Leukemia ................................... 6,020 .................................................................1,440

Male ........................................................... 3,140 ................................................................... 810

Female ....................................................... 2,880 ................................................................... 630

Acute Myeloid Leukemia .........................................18,860 ...............................................................10,460

Male ..........................................................11,530 .................................................................6,010

Female ........................................................7,330 ................................................................ 4,450

Bladder Cancer ......................................................74,690 ............................................................... 15,580

Male ........................................................ 56,390 .................................................................11,170

Female ......................................................18,300 .................................................................4,410

Brain Cancer (and other nervous system) ...................23,380 ............................................................... 14,320

Male ......................................................... 12,820 ................................................................ 8,090

Female ...................................................... 10,560 ................................................................ 6,230

Breast Cancer ......................................................235,030 .............................................................. 40,430

Male .......................................................... 2,360 ................................................................... 430

Female .................................................... 232,670 .............................................................. 40,000

Cervical and Uterine Cancers .................................. 64,990 ................................................................12,610

Male ................................................................— ......................................................................—

Female ..................................................... 64,990 ................................................................12,610

Chronic Lymphocytic Leukemia .................................15,720 ................................................................4,600

Male ...........................................................9,100 ................................................................ 2,800

Female ....................................................... 6,620 .................................................................1,800

Chronic Myeloid Leukemia ....................................... 5,980 ................................................................... 810

Male ........................................................... 3,130 ................................................................... 550

Female ....................................................... 2,850 ................................................................... 260

table continued on next page


Facts

New Cancer Cases/Deaths1 (continued)


Colorectal Cancer ................................................. 136,830 ............................................................... 50,310

Male ......................................................... 71,830 ............................................................... 26,270

Female ..................................................... 65,000 .............................................................. 24,040

Esophageal Cancer .................................................. 18,170 ............................................................... 15,450

Male .........................................................14,660 ............................................................... 12,450

Female ........................................................ 3,510 ................................................................ 3,000

Kidney Cancer .......................................................63,920 ............................................................... 13,860

Male ......................................................... 39,140 ................................................................ 8,900

Female ......................................................24,780 ................................................................ 4,960

Leukemia (all types) ................................................52,380 .............................................................. 24,090

Male .........................................................30,100 ...............................................................14,040

Female ......................................................22,280 ............................................................... 10,050

Liver Cancer (and bile duct) ..................................... 33,190 .............................................................. 23,000

Male ........................................................ 24,600 ............................................................... 15,870

Female ....................................................... 8,590 ..................................................................7,130

Lung Cancer (and bronchus) ................................... 224,210 ............................................................. 159,260

Male ....................................................... 116,000 .............................................................. 86,930

Female .....................................................108,210 ...............................................................72,330

Lymphoma (all types) ..............................................79,990 ................................................................20,170

Male ........................................................ 43,340 ................................................................ 11,140

Female ..................................................... 36,650 ................................................................ 9,030

Melanoma-Skin Cancer ........................................... 76,100 ................................................................. 9,710

Male ........................................................ 43,890 .................................................................6,470

Female ...................................................... 32,210 .................................................................3,240

Myeloma ..............................................................24,050 ............................................................... 11,090

Male ......................................................... 13,500 ..................................................................6,110

Female ...................................................... 10,550 ................................................................ 4,980

Non-Hodgkin Lymphoma ....................................... 70,800 ...............................................................18,990

Male .........................................................38,270 ............................................................... 10,470

Female ......................................................32,530 .................................................................8,520

table continued on next page


Facts

Sources:

1. Cancer Facts & Figures 2014, American Cancer Society, www.cancer.org

2. National Cancer Institute, www.cancer.gov

3. AACR Cancer Progress Report 2014, American Association for Cancer Research, www.cancerprogressreport.org

New Cancer Cases/Deaths1 (continued)


Ovarian Cancer ......................................................21,980 ............................................................... 14,270

Male ................................................................— ......................................................................—

Female ......................................................21,980 ............................................................... 14,270

Pancreatic Cancer ................................................. 46,420 ...............................................................39,590

Male .........................................................23,530 ................................................................20,170

Female ......................................................22,890 ............................................................... 19,420

Prostate Cancer ...................................................233,000 .............................................................. 29,480

Male .......................................................233,000 .............................................................. 29,480

Female .............................................................— ......................................................................—

Stomach Cancer .....................................................22,220 ...............................................................10,990

Male ..........................................................13,730 .................................................................6,720

Female ....................................................... 8,490 .................................................................4,270

Thyroid Cancer ..................................................... 62,980 .................................................................1,890

Male ..........................................................15,190 ................................................................... 830

Female ...................................................... 47,790 .................................................................1,060


Medicines and Vaccines in Development for Cancer

*For more information about a specifi c medicine or company in this report, please use the website provided.

Bladder Cancer

Product Name Sponsor Indication Development Phase*

ABI-009(nanoparticle albumin-bound mTOR inhibitor)

AadiPaci� c Palisades, CA

non-muscle invasive bladder cancer Phase I/II

ALT-801(immunotherapy-fusion protein)

Altor BioScienceMiramar, FL

advanced bladder cancer, non-muscle invasive bladder cancer

Phase IIwww.altorbioscience.com

ALT-803(IL-15 superagonist complex)


non-muscle invasive bladder cancer(see also hematological, myeloma, skin)

Phase I/IIwww.altorbioscience.com

apatorsen(Hsp27 inhibitor)

OncoGenex PharmaceuticalsBothell, WA

metastatic bladder cancer(see also lung, pancreatic, prostate)

Phase IIwww.oncogenex.com

apaziquone(DNA synthesis inhibitor)

Spectrum PharmaceuticalsHenderson, NV

non-muscle invasive bladder cancer(Fast Track)

Phase IIIwww.sppirx.com

ASG-15ME(antibody drug conjugate)

AgensysSanta Monica, CASeattle GeneticsBothell, WA

bladder cancer Phase Iwww.agensys.comwww.seattlegenetics.com

CG0070(oncolytic virus therapy)

Cold GenesysNewport Beach, CA

non-muscle invasive bladder cancer Phase II/IIIwww.coldgenesys.net

CV-301(targeted immunotherapy)

Bavarian NordicMountain View, CA

bladder cancer(see also breast, colorectal)

Phase II completedwww.bavarian-nordic.com

Cynviloq™paclitaxel polymeric micellefor injection

Sorrento TherapeuticsSan Diego, CA

bladder cancer(see also breast, lung, ovarian, pancreatic)

Phase IIwww.sorrentotherapeutics.com

Cyramza®

ramucirumabLillyIndianapolis, IN

bladder cancer(see also breast, colorectal, liver, lung, ovarian, solid tumors, stomach)

Phase IIwww.lilly.com

instiladrin(gene therapy)

FKD TherapiesKuopio, Finland

super� cial bladder cancer Phase II

Keytruda®

pembrolizumabMerckWhitehouse Station, NJ

bladder cancer(see also breast, head/neck, hematological, kidney, lung, myeloma, stomach)

Phase Iwww.merck.com



Bladder Cancer

Product Name Sponsor Indication Development Phase*

MAGE-A3 immunotherapeutic(recombinant antigen-speci� ccancer immunotherapeutic)

GlaxoSmithKlineResearch Triangle Park, NC

bladder cancer Phase IIwww.gsk.com

mocetinostat(HDAC inhibitor)

Mirati TherapeuticsSan Diego, CA

bladder cancer(see also hematological, lymphoma)

Phase IIwww.mirati.com

mycobacterium cell wall-DNA complex (MCNA)

Bioniche Life SciencesBelleville, Canada

refractory non-muscle invasivebladder cancer

Phase IIwww.bioniche.com

RG7446(anti-PD-L1 mAb)

GenentechSouth San Francisco, CA

metastatic urothelial bladder cancer(Breakthrough Therapy)(see also kidney, lung, skin)

Phase IIwww.gene.com

TMX-101(TLR7 agonist)

TelormedixBioggio, Switzerland

bladder cancer Phase IIwww.telormedix.com

vesigenurtacel-L(cancer immunotherapy)

Heat BiologicsDurham, NC

bladder cancer Phae I/IIwww.heatbio.com

Brain Cancer

Product Name Sponsor Indication Development Phase

2B3-101(doxorubicin liposomal)ORPHAN DRUG

to-BBB technologiesLeiden, Netherlands

brain metastases, recurrent malignant glioma

Phase IIwww.tobbb.com

ABT-414(antibody drug conjugate)ORPHAN DRUG

AbbVieNorth Chicago, IL

glioblastoma multiforme(see also solid tumors)

Phase Iwww.abbvie.com

ADU-623(bivalent Listeria-based immunotherapy)

Aduro BiotechBerkeley, CA

glioblastoma Phase Iwww.adurobiotech.com

aldoxorubicin(delivery-enhanced doxorubicin)

CytRxLos Angeles, CA

glioblastoma multiforme(see also lung, sarcoma)

Phase IIwww.cytrx.com

AMG 595(tubulin inhibitor)

AmgenThousand Oaks, CA

advanced malignant glioma, anaplastic astrocytomas, glioblastoma multiforme

Phase Iwww.amgen.com

ANG1005(targeted taxane derivative)ORPHAN DRUG

AngiochemMontreal, Canada

brain metastases, malignant glioma(Fast Track)

Phase IIwww.angiochem.com



Brain Cancer


antineoplastonORPHAN DRUG

Burzynski Research InstituteHouston, TX

glioma (combination therapy) Phase IIwww.burzynskiresearch.com

Avastin®

bevacizumabGenentechSouth San Francisco, CA

glioblastoma multiforme (1st-line)(see also lung, ovarian, other)

Phase III completedwww.gene.com

Azedra®

iobenguane I-131ORPHAN DRUG

Progenics PharmaceuticalsTarrytown, NY

paraganglioma, malignant pheochromocytoma(Fast Track)

Phase IIwww.progenics.com

BGJ398(FGF-R kinase inhibitor)

Novartis PharmaceuticalsEast Hanover, NJ

glioblastoma(see also hematological, liver, solid tumors)

Phase IIwww.novartis.com

cancer gene therapy(AdV-tK)

AdvantageneAuburndale, MA

anaplastic astrocytoma, glioblastoma, malignant glioma, recurrent ependymoma(see also pancreatic, prostate)

Phase I/IIwww.advantagene.com

CPP-1X(e� ornithine)

Cancer Prevention PharmaceuticalsTucson, AZ

neuroblastoma(see also colorectal)

Phase IIwww.canprevent.com

crenolanib(PDGFR inhibitor)ORPHAN DRUG

AROG PharmaceuticalsDallas, TX

glioma (pediatric) (see also stomach)

Phase Iwww.arogpharma.com

CTO(multiple oncokinase inhibitor)

Tactical TherapeuticsNew York, NY

glioma (combination therapy)(Fast Track)

Phase IIwww.tacticaltherapeutics.com

DCVax®-Ldendritic cell vaccine

Northwest BiotherapeuticsBethesda, MD

glioblastoma multiforme Phase IIIwww.nwbio.com

DM-CHOC-PEN DEKK-TECNew Orleans, LA

brain tumors Phase IIwww.dekk-tec.com

DNX-2401(cell death stimulant)

DNAtrixHouston, TX

recurrent glioblastoma(Fast Track)

Phase Iwww.dnatrix.com

E7080(multi-targeted kinase inhibitor)

EisaiWoodcli� Lake, NJ

glioma(see also liver, lung, skin, other)

Phase IIwww.eisai.com

EGFRvIII CAR(chimeric antigen receptor)

Kite PharmaSanta Monica, CANational Cancer InstituteBethesda, MD

glioblastoma Phase I/IIwww.kitepharma.com

ERC-1671(cancer vaccine)ORPHAN DRUG

Epitopoietic ResearchLes Isnes, Belgium

glioblastoma, gliosarcoma Phase IIwww.ercbelgium.com



Brain Cancer


G-202(calcium-transporting ATPaseinhibitor)

GenSperaSan Antonio, TX

glioblastoma multiforme(see also liver, prostate)

Phase IIwww.genspera.com

galunisertib(TGF-beta1 inhibitor)

LillyIndianapolis, IN

glioblastoma, glioma(see also liver, pancreatic)


heat shock protein vaccineORPHAN DRUG

AgenusLexington, MA

recurrent glioblastoma multiforme, newly diagnosed glioma(see also skin)

Phase IIwww.agenusbio.com

ICT-107(dendritic cell-based vaccine)ORPHAN DRUG

ImmunoCellular TherapeuticsCalabasas, CA

glioblastoma (1st-line) Phase IIwww.imuc.com

ICT-121(dendritic cell vaccine)

ImmunoCellular TherapeuticsCalabasas, CA

recurrent glioblastoma Phase Iwww.imuc.com

KPT-330(XPO1 inhibitor)

Karyopharm TherapeuticsNewton, MA

glioblastoma multiforme(see also leukemia, prostate, solid tumors, other)

Phase IIwww.karyopharm.com

KX02 Kinex PharmaceuticalsBu� alo, NY

glioma Phase Iwww.kinexpharma.com

LDE225(SMO protein inhibitor)


medulloblastoma(see also skin, solid tumors)


macitentan Actelion PharmaceuticalsSouth San Francisco, CA

glioblastoma Phase Iwww.actelion.com

mibefradil(t-type calcium channel antagonist)ORPHAN DRUG

CavionCharlottesville, VA

recurrent high-grade glioma Phase Iwww.cavion.com

mipsagargin(ATPase inhibitor)


glioblastoma multiforme(see also liver, prostate)


MM-398(irinotecan nanotherapeutic)

Merrimack PharmaceuticalsCambridge, MA

glioma(see also colorectal, pancreatic)

Phase IIIwww.merrimackpharma.com

nivolumab(PD-1 inhibitor)

Bristol-Myers SquibbPrinceton, NJ

glioblastoma(see also colorectal, head/neck, hematological, kidney, liver, lung, lymphoma, skin, solid tumors)

Phase IIwww.bms.com



Brain Cancer


nivolumab + Yervoy®

ipilimumabBristol-Myers SquibbPrinceton, NJ

glioblastoma(see also colorectal, head/neck, hematological, kidney, lung, skin)

Phase IIwww.bms.com

PEG-SN38(angiogenesis inhibitor)ORPHAN DRUG

Belrose PharmaPrinceton, NJ

pediatric neuroblastoma(see also breast, solid tumors)

Phase Iwww.belrosepharma.com

PLX3397(Fms/Kit/Flt3-ITD inhibitor)

Daiichi SankyoParsippany, NJPlexxikonBerkeley, CA

glioblastoma(see also leukemia, skin)

Phase IIwww.dsi.comwww.plexxicon.com

RG7666(PI3K inhibitor)


glioblastoma (2nd-line) Phase Iwww.gene.com

rindopepimut(EGFRvIII antagonist)ORPHAN DRUG

Celldex TherapeuticsHampton, NJ

glioblastoma (1st-line)(Fast Track)-----------------------------------------recurrent glioblastoma(combination therapy) (Fast Track)

Phase IIIwww.celldex.com-----------------------------------------Phase IIwww.celldex.com

SL-701(cancer immunotherapy)

Stemline TherapeuticsNew York, NY

glioblastoma multiforme Phase I/IIwww.stemline.com

Toca 511 & Toca FC(� ucytosine gene therapy)

TocagenSan Diego, CA

high-grade gliomas Phase I/IIwww.tocagen.com

TPI 287(microtubule stabilizing taxoid)

Cortice BiosciencesNew York, NY

recurrent glioblastoma, neuroblastoma-----------------------------------------breast cancer brain metastases, secondary brain metastases

Phase IIwww.corticebio.com-----------------------------------------Phase Iwww.corticebio.com

TRC105(ENG protein inhibitor)

TRACON PharmaceuticalsSan Diego, CA

glioblastoma multiforme(see also kidney, sarcoma)

Phase IIwww.traconpharma.com

TSC(trans sodium crocetinate)ORPHAN DRUG

Di� usion PharmaceuticalsCharlottesville, VA

glioblastoma Phase I/IIwww.di� usionpharma.com

TVI-Brain-1(personalized cancer immunotherapy)

TVAX BiomedicalLenexa, KS

late-stage glioma Phase II completedwww.tvaxbiomedical.com

VAL-083ORPHAN DRUG

DelMar PharmaceuticalsVancouver, Canada

glioblastoma Phase I/IIwww.delmarpharma.com


Brain Cancer


VB-111(vascular targeting system)ORPHAN DRUG

VBL TherapeuticsOr Yehuda, Israel

recurrent glioblastoma multiforme(Fast Track)(see also ovarian, other)

Phase I/IIwww.vblrx.com

veliparib AbbVieNorth Chicago, IL

brain metastases(see also breast, lung)

Phase I/IIwww.abbvie.com

Breast Cancer


abemaciclib(CDK 4/6 inhibitor)


hormone receptor positive, HER2-negative breast cancer(see also lung, lymphoma)-----------------------------------------metastatic breast cancer

Phase IIIwww.lilly.com

-----------------------------------------Phase IIwww.lilly.com

Abraxane®

paclitaxel protein-bound particles for injectable suspension, albumin-bound

CelgeneSummit, NJ

triple-negative breast cancer (1st-line)(see also pancreatic)

Phase IIIwww.celgene.com

ADI-PEG 20(PEG arginine deiminase)

Polaris PharmaceuticalsSan Diego, CA

HER2 negative breast cancer(see also leukemia, liver, lung, lymphoma, skin)

Phase Iwww.polarispharma.com

Ad-RTS-IL-12(interleukin-12 gene therapy)

ZIOPHARM OncologyBoston, MA

breast cancer(see also skin)

Phase IIwww.ziopharm.com

AE37(immunotherapeutic)

Antigen ExpressWorcester, MA

HER2/neu positive breast cancer(see also prostate)

Phase IIwww.antigenexpress.com

A� nitor®

everolimusNovartis PharmaceuticalsEast Hanover, NJ

HER2-positive breast cancer (1st-line), HER2-positive breast cancer (2nd/3rd-line)(see also lung, lymphoma, stomach)

Phase IIIwww.novartis.com

ARN-810(selective estrogen receptor degrader)

Seragon PharmaceuticalsSan Diego, CA

breast cancer Phase Iwww.seragonpharm.com

ARRY-380/ONT-380(HER2 inhibitor)

Array BioPharmaBoulder, COOncothyreonSeattle, WA

breast cancer Phase Iwww.arraybiopharma.comwww.oncothyreon.com



Breast Cancer


AV-203(ERBB-3 receptor antagonist)

AVEO OncologyCambridge, MA

early breast cancer Phase I completedwww.aveooncology.com

AZD5363(AKT kinase inhibitor)

AstraZenecaWilmington, DE

breast cancer Phase IIwww.astrazeneca.com

bavituximab Peregrine PharmaceuticalsTustin, CA

HER2-negative breast cancer(see also liver, lung, skin, other)

Phase Iwww.peregrineinc.com

BBI608(cancer stemness kinase inhibitor)

Boston BiomedicalCambridge, MA

breast cancer(see also colorectal, lung, ovarian, skin, stomach)

Phase I/IIwww.bostonbiomedical.com

BKM120(PI3K inhibitor)


metastatic breast cancer(ER-positive AI resistant/mTOR naïve)(see also solid tumors)-----------------------------------------metastatic breast cancer(ER-positive post AI and mTOR inhibitor)


-----------------------------------------Phase IIwww.novartis.com

BMN 673(PARP inhibitor)

BioMarin PharmaceuticalNovato, CA

metastatic breast cancer Phase IIIwww.bmrn.com

BZL-101(glycolysis inhibitor)

BionovoEmeryville, CA

breast cancer Phase Iwww.bionovo.com

CV-301(targeted immunotherapy)


breast cancer(see also bladder, colorectal)

Phase II completedwww.bavarian-nordic.com

Cynviloq™paclitaxel polymeric micellefor injection


metastatic breast cancer(see also bladder, lung, ovarian, pancreatic)

Phase IIIwww.sorrentotherapeutics.com

Cyramza®


breast cancer (combination therapy)(see also bladder, colorectal, liver, lung, ovarian, solid tumors, stomach)


eniluracil Adherex TechnologiesResearch Triangle Park, NC

breast cancer Phase II completedwww.adherex.com

ENMD-2076(aurora A/angiogenic kinase inhibitor)

CASI PharmaceuticalsRockville, MD

triple-negative breast cancer(see also ovarian, sarcoma)

Phase IIwww.casipharmaceuticals.com

entinostat(HDAC inhibitor)

Syndax PharmaceuticalsWaltham, MA

HR-positive breast cancer(Breakthrough Therapy)

Phase IIIwww.syndax.com



Breast Cancer


etirinotecan pegol(PEGylated irinotecan)

Nektar TherapeuticsSan Francisco, CA

metastatic breast cancer(Fast Track)(see also colorectal, ovarian)

Phase IIIwww.nektar.com

ganetespib(Hsp90 inhibitor)

Synta PharmaceuticalsLexington, MA

breast cancer(see also leukemia, lung, ovarian)

Phase IIwww.syntapharma.com

GDC-0980(mTOR/PI3K inhibitor)


breast cancer (combination therapy) Phase IIwww.gene.com

glembatumumab vedotin(antibody drug conjugate)

CellDex TherapeuticsHampton, NJ

triple negative breast cancer(Fast Track)(see also skin)

Phase IIwww.celldex.com

GTx-024/GTx-027(SARM)

GTxMemphis, TN

androgen receptor-positive breast cancer

Phase IIwww.gtxinc.com

Halaven®

eribulinEisaiWoodcli� Lake, NJ

HER2-negative breast cancer(1st-line, 2nd-line)(see also lung, sarcoma)

Phase IIIwww.eisai.com

HER2 cancer immunotherapy(AVX901)

AlphaVaxResearch Triangle Park, NCDuke UniversityDurham, NC

late-stage breast cancer Phase I/IIwww.alphavax.com

indoximod(IDO pathway inhibitor)

NewLink GeneticsAmes, IA

metastatic HER2-negative breast cancer(see also prostate)

Phase IIwww.newlinkgenetics.com

ipatasertib(GDC-0068)(Akt inhibitor)

Array BioPharmaBoulder, COGenentechSouth San Francisco, CA

(see also prostate, stomach) Phase IIwww.arraybiopharma.comwww.gene.com

Jaka� ®ruxolitinib

Incyte Wilmington, DE

breast cancer(see also colorectal, lung, pancreatic, other)

Phase Iwww.incyte.com

Kadcyla®

ado-trastuzumab emtansineGenentechSouth San Francisco, CA

HER2-positive breast cancer (1st-line), HER2-positive breast cancer (adjuvant), HER2-positive breast cancer (adjuvant combination therapy), HER2-positive breast cancer (neoadjuvant combination therapy)(see also stomach)

Phase IIIwww.gene.com

KD019(TKI inhibitor)

Kadmon PharmaceuticalsWarrendale, PA

breast cancer(see also lung)

Phase I/IIwww.kadmon.com



Breast Cancer


Keytruda®


breast cancer(see also bladder, head/neck, hematological, kidney, lung, myeloma, stomach)


LCL161(IAP inhibitor)


breast cancer Phase IIwww.novartis.com

LEE011(CDK4/6 inhibitor)


breast cancer (1st-line)(see also solid tumors)


LFA102(anti-prolactin receptor mAb)

Novartis PharmaceuticalsEast Hanover, NJXOMABerkeley, CA

prolactin receptor-positive metastaticbreast cancer(see also prostate)

Phase Iwww.novartis.comwww.xoma.com

lucitanib(FGFR/VEGFR/PDGFR inhibitor)

Clovis OncologyBoulder, CO


Phase IIwww.clovisoncology.com

LY2606368(Chk1 inhibitor)


BRCA 1/2 mutation breast cancer, triple-negative breast cancer(see also ovarian, solid tumors)


margetuximab(anti-HER2 mAb)

MacroGenicsRockville, MD

metastatic breast cancer(see also solid tumors, other)

Phase IIwww.macrogenics.com

MEDI-573(anti-IGF mAb)

MedImmuneGaithersburg, MD

metastatic breast cancer Phase IIwww.medimmune.com

mifepristone Corcept TherapeuticsMenlo Park, CA

triple-negative breast cancer (combination therapy)

Phase Iwww.corcept.com

MLN0128(TORC1/TORC2 inhibitor)

Millennium PharmaceuticalsCambridge, MA

metastatic breast cancer(see also hematological, solid tumors)

Phase IIwww.millennium.com

MM-121(ErbB-3 receptor antagonist)


breast cancer(see also lung, ovarian)

Phase IIwww.merrimackpharma.com

MM-302(HER2-targeted nanotherapeutic)


advanced HER2-positive breast cancer Phase Iwww.merrimackpharma.com

MVA-BN-HER2(immunotherapy vaccine)


breast cancer Phase I/IIwww.bavarian-nordic.com

Myocet™ doxorubicin liposomal

Sopherion TherapeuticsEast Brunswick, NJ

metastatic breast cancer (1st-line)(Fast Track)

Phase IIIwww.sopherion.com




Breast Cancer


NeuVax™nelipepimut-S

Galena BiopharmaPortland, OR

node positive, HER2-positive breast cancer(see also stomach)-----------------------------------------node-positive & negative HER2-positive breast cancer (combination therapy)-----------------------------------------node-positive & negative HER2-positive breast cancer, breast cancer (neoadjuvant)

Phase IIIwww.galenabiopharma.com

-----------------------------------------Phase IIwww.galenabiopharma.com

-----------------------------------------Phase I completedwww.galenabiopharma.com

Nexavar®

sorafenibBayer HealthCare PharmaceuticalsWhippany, NJ

breast cancer(see also kidney)

Phase IIIwww.bayerpharma.com

niraparib(PARP inhibitor)

TESAROWaltham, MA

BRCA-positive breast cancer(see also ovarian, sarcoma)

Phase IIIwww.tesarobio.com

olaparib(PARP inhibitor)


breast cancer (adjuvant), metastatic breast cancer(see also ovarian, stomach)

Phase IIIwww.astrazeneca.com

palbociclib(CDK4/6 inhibitor)

P� zerNew York, NY

advanced breast cancer (1st-line)(Breakthrough Therapy)(see also colorectal)-----------------------------------------high risk early breast cancer, recurrent advanced breast cancer

application submittedwww.pfizer.com

-----------------------------------------Phase IIIwww.pfizer.com

patritumab(anti-HER3 antibody)

Daiichi SankyoParsippany, NJ


Phase IIwww.dsi.com

PB272(neratinib)

Puma BiotechnologyLos Angeles, CA

metastatic breast cancer(see also solid tumors)-----------------------------------------metastatic breast cancer with brain metastases, breast cancer (neoadjuvant)

Phase IIIwww.pumabiotechnology.com-----------------------------------------Phase IIwww.pumabiotechnology.com

PEG-SN38(angiogenesis inhibitor)


metastatic breast cancer(see also brain, solid tumors)

Phase IIwww.belrosepharma.com

Perjeta®

pertuzumabGenentechSouth San Francisco, CA

HER2-positive metastatic breast cancer (2nd-line), HER2-positive breast cancer (adjuvant)(see also stomach)


PF-05280014(trastuzumab biosimilar)


metastatic breast cancer Phase IIIwww.pfizer.com



Breast Cancer


pictrelisib(PI3K inhibitor)


estrogen receptor-positive metastatic breast cancer, metastatic HER2-negative/hormone receptor-positive breast cancer(see also lung)


PM01183(marine-derived alkylating agent)

Pharma MarMadrid, Spain

metastatic breast cancer(see also leukemia)

Phase IIwww.pharmamar.com

RAD1901(SERM modulator)

Radius HealthWaltham, MA

brain metastases from breast cancer Phase Iwww.radiuspharm.com

reparixin(IL-8A/B receptor antagonist)

DompeMilan, Italy

early-stage breast cancer

-----------------------------------------meatastatic breast cancer

Phase IIwww.dompe.com-----------------------------------------Phase Iwww.dompe.com

SGN LIV1A(anti-LIV-1 mAb)

Seattle GeneticsBothell, WA

metastatic breast cancer (2nd-line) Phase Iwww.seattlegenetics.com

taselisib(PI3K inhibitor)


HER2-negative metastatic breast cancer, hormone receptor-positive breast cancer(see also solid tumors)

Phase Iwww.gene.com

ThermoDox®

doxorubicin liposomalCelsionLawrenceville, NJ

breast cancer(see also colorectal, liver)

Phase IIwww.celsion.com

topotecan liposomal Spectrum PharmaceuticalsHenderson, NV


Phase Iwww.sppirx.com

TPIV100(cancer immunotherapy)

TapImmuneSeattle, WA

breast cancer Phase Iwww.tapimmune.com

trastuzumab biosimilar ActavisDublin, IrelandAmgenThousand Oaks, CA

breast cancer Phase IIIwww.actavis.comwww.amgen.com

Tykerb®

lapatinibGlaxoSmithKlineResearch Triangle Park, NC

breast cancer (neoadjuvant, adjuvant) Phase IIIwww.gsk.com


BRCA-de� cient breast cancer, triple negative breast cancer (neoadjuvant)(see also brain, lung)

Phase IIIwww.abbvie.com


Medicines and Vaccines in Development for Cancers

Breast Cancer


WT1 immunotherapeutic(antigen-speci� c cancer immunotherapeutic)


breast cancer Phase IIwww.gsk.com

Xgeva®

denosumabAmgenThousand Oaks, CA

delay or prevention of bone metastases in breast cancer(see also lung)

Phase IIIwww.amgen.com

Xtandi®enzalutamide(androgen receptor inhibitor)

Astellas Pharma USNorthbrook, ILMedivationSan Francisco, CA

breast cancer(see also prostate)

Phase IIwww.astellas.comwww.medivation.com

Zytiga®

abiraterone acetateJanssen Research & DevelopmentRaritan, NJ

metastatic estrogen receptor-positive HER2-negative breast cancer in postmenopausal patients

Phase IIwww.janssenrnd.com

Colorectal Cancer


abituzumab(anti-integrin mAb)

EMD SeronoRockland, MA

colorectal cancer(see also prostate)

Phase IIwww.emdserono.com

AD-p53 gene therapy MultiVirSan Diego, CA

colorectal cancer Phase I completedwww.multivir.com



colorectal cancer(see also breast, lung, ovarian, skin, stomach)-----------------------------------------colorectal cancer(combination therapy)

Phase IIIwww.bostonbiomedical.com

-----------------------------------------Phase IIwww.bostonbiomedical.com

birinapant(apoptosis stimulator)

TetraLogic PharmaceuticalsMalvern, PA

colorectal cancer(combination therapy)(see also hematological, ovarian)

Phase I/II completedwww.tetralogicpharm.com

CEA cancer immunotherapy(AVX701)

AlphaVaxResearch Triangle Park, NCDuke UniversityDurham, NC

late-stage colorectal cancer Phase I/IIwww.alphavax.com

CEP-32496(BRAF kinase inhibitor)

Teva PharmaceuticalFrazier, PA

metastatic colorectal cancer (see also skin, solid tumors)

Phase IIwww.tevapharm.com



Colorectal Cancer


CPP-1X/sulindac(e� ornithine/sulindac)

Cancer Prevention PharmaceuticalsTucson, AZ

colorectal cancer(risk reduction)(see also brain)

Phase IIIwww.canprevent.com

CPX-1(irinotecan/� oxuridine liposomal)

Celator PharmaceuticalsEwing, NJ

colorectal cancer Phase IIwww.celatorpharma.com

CRLX101(camptothecin nanopharmaceutical)

Cerulean PharmaCambridge, MA

late-stage rectal cancer Phase I/IIwww.ceruleanrx.com

CV-301(cancer immunotherapy)

BN ImmunoTherapeuticsMountain View, CA

colorectal cancer(see also bladder, breast)

Phase IIwww.bavarian-nordic.com

Cyramza™ramucirumab


colorectal cancer(see also bladder, breast, liver, lung, ovarian, solid tumors, stomach)


E7820(integrin alpha2 inhibitor)


colorectal cancer Phase IIwww.eisai.com

encapsulated cell therapy Rogosin InstituteNew York, NY

colorectal cancer Phase IIwww.rogosin.org

ensituximab(neoplasm antigen inhibitor)

Precision BiologicsDallas, TX

advanced colorectal cancer(see also pancreatic)

Phase IIwww.precision-biologics.com

ETBX-011(vector cancer vaccine)

EtubicsSeattle, WA

colorectal cancer Phase IIwww.etubics.com

etirinotecan pegol(PEGylated irinotecan)


colorectal cancer (2nd-line)(see also breast, ovarian)

Phase II/IIIwww.nektar.com

FANG™ Vaccinegenetically modi� ed autologous tumor cell vaccine

GradalisDallas, TX

colorectal cancer(see also ovarian, skin, solid tumors)

Phase IIwww.gradalisinc.com

IMMU-130(antibody drug conjugate)

ImmunomedicsMorris Plains, NJ

metastatic colorectal cancer Phase IIwww.immunomedics.com

Imprime PGG®

intravenous immunostimulantBiotheraEagan, MN

metastatic colorectal cancer (3rd-line)(see also leukemia, lung, lymphoma, pancreatic)

Phase IIIwww.biothera.com


IncyteWilmington, DE

colorectal cancer(see also breast, lung, pancreatic, other)



Colorectal Cancer


KD018(Chinese botanical formulation)


colorectal cancer(see also liver)


KD032(Ras antagonist)


colorectal cancer(see also lung, pancreatic)

Phase Iwww.kadmon.com

labetuzumab-SN-38(IMMU-130)


metastatic colorectal cancer Phase IIwww.immunomedics.com

LGX818(BRAF inhibitor)


BRAF-mutant metastatic colorectal cancer (combination therapy)(see also skin, solid tumors)

Phase I/IIwww.novartis.com

linaclotide ActavisDublin, IrelandIronwood PharmaceuticalsCambridge, MA

colon cancer prevention Phase Iwww.ironwoodpharma.com

Mekinist® trametinib +Ta� nlar® dabrafenib + panitumumab combination


colorectal cancer Phase IIwww.gsk.com

MM-398(irinotecan nanotherapeutic)


colorectal cancer(see also brain, pancreatic)


MORAb-004(CD248 antigen inhibitor)

EisaiWoodcli� Lake, CA

colorectal cancer(see also lung, sarcoma, skin)




microsatellite instability (MSI)-positive colon cancer(see also brain, head/neck, hematological, kidney, liver, lung, lymphoma, skin, solid tumors)

Phase IIwww.bms.com



MSI-positive colon cancer(see also brain, head/neck, hematological, kidney, lung, skin)

Phase IIwww.bms.com

OncoVAX®

cancer immunotherapyVaccinogenFrederick, MD

stage II colon cancer Phase IIIwww.vaccinogeninc.com

palbociclib(CDK4/6 inhibitor)


colorectal cancer(see also breast)

Phase Iwww.pfizer.com

pelareorep Oncolytics BiotechCalgary, Canada

KRAS mutant-positive metastatic colorectal cancer (see also head/neck, lung, myeloma, ovarian, pancreatic, skin, solid tumors, other)

Phase Iwww.oncolyticsbiotech.com



Colorectal Cancer


pexastimogene devacirepvec(genetically-engineered oncolytic vaccinia virus)

StillaJen BiotherapeuticsSan Francisco, CA

treatment refractory colorectal cancer(see also kidney, liver)

Phase IIwww.sillajen.com

PF-05212384(PI3K-mTOR inhibitor)


colorectal cancer (3rd-line) Phase IIwww.pfizer.com

RG7221(Ang2-VEGF Mab)


colorectal cancer Phase IIwww.gene.com

RG7597(anti-HER3/EGFR DAF mAb)


colorectal cancer (see also head/neck)


RRx-001(free radical stimulant)

RadioRxMountain View, CA

metastatic colorectal cancer(see also lymphoma, solid tumors)

Phase IIwww.radiorx.com

simtuzumab Gilead SciencesFoster City, CA

colorectal cancer(see also hematological)

Phase IIwww.gilead.com

SM04755(Wnt inhibitor)

SamumedSan Diego, CA

advanced colorectal cancer(see also liver, pancreatic, stomach)

Phase Iwww.samumed.com

Stivarga®

regorafenibBayer HealthCare PharmaceuticalsWhippany, NJ

colorectal cancer with resected liver metastases (see also kidney, liver, solid tumors)


Sym004(anti-EGFR mAb)


colorectal cancer(see also head/neck, solid tumors)


TAS-102(tipiracil/tri� uridine)

Taiho OncologyPrinceton, NJ

colorectal cancer Phase IIIwww. taihooncology.com

ThermoDox®

doxorubicin liposomalCelsionLawrenceville, NJ

colorectal metastases(see also breast, liver)

Phase IIwww.celsion.com

vemurafenib(BRAF inhibitor)


colorectal cancer Phase IIwww.dsi.com

Xilonix™monoclonal (IgG1k) antibody

XBiotechAustin, TX

colorectal cancer in patients with cachexia, colorectal cancer(Fast Track)

Phase IIIwww.xbiotech.com



Head/Neck Cancer


Ad-IL-24(interleukin-24 gene therapy)

MultiVirSan Diego, CA

head and neck cancer Phase IIwww.multivir.com

ADXS-HPV(live, attenuated Listeria monocytogenes (Lm)-basedimmunotherapy vaccine)ORPHAN DRUG

AdvaxisPrinceton, NJ

head and neck cancer(see also other)

Phase I/IIwww.advaxis.com

AZD6738(ATR serine/threonine kinase inhibitor)


head and neck cancer(see also leukemia)

Phase Iwww.astrazeneca.com

GC4419(superoxide dismutase modulator)

Galera TherapeuticsMalvern, PA

squamous cell carcinoma of thehead and neck

Phase Iwww.galeratx.com

Gilotrif®

afatinibBoehringer Ingelheim PharmaceuticalsRidge� eld, CT

squamous cell carcinoma of the head and neck

Phase IIIwww.boehringer-ingelheim.com

GL-0817(cancer immunotherapy)ORPHAN DRUG

GliknikBaltimore, MD

squamous cell cancer of the oral cavity

Phase IIwww.gliknik.com

GL-ONC1(oncolytic virus immunomodulator)

GeneluxSan Diego, CA

head and neck cancer (see also lung, solid tumors, other)

Phase Iwww.genelux.com

HF10(oncolytic virus immunomodulator)

Takara BioShiga, Japan

head and neck cancer Phase Iwww.takara-bio.com

INO-3112(cancer therapeutic vaccine)

Inovio PharmaceuticalsPlymouth Meeting, PA

head and neck cancer caused byHPV types 16/18(see also other)

Phase Iwww.inovio.com

IRX-2(immunostimulant)

IRX TherapeuticsNew York, NY

head and neck cancer(Fast Track)

Phase II completedwww.irxtherapeutics.com

Keytruda®


advanced head and neck cancer(see also bladder, breast, hematological, kidney, lung, myeloma, stomach)


MGCD265(MET/Axl/VEGFR kinase inhibitor)


squamous cell carcinoma of the head and neck(see also lung)

Phase Iwww.mirati.com



Head/Neck Cancer


Multikine®

leukocyte interleukin, injectionCEL-SCIVienna, VA

head and neck cancer (1st-line) Phase IIIwww.cel-sci.com



head and neck cancer (2nd-line)(see also brain, colorectal, hematological, kidney, liver, lung, lymphoma, skin, solid tumors)

Phase IIIwww.bms.com



head and neck cancer (2nd-line)(see also brain, colorectal, hematological, kidney, lung, skin)



advanced head and neck cancer(see also colorectal, lung, myeloma, ovarian, pancreatic, skin, solid tumors, other)

Phase III completedwww.oncolyticsbiotech.com

RG7597(HER3/EGFR DAF mAb)


head and neck cancer(see also colorectal)




squamous cell carcinoma of the head and neck(see also colorectal, solid tumors)


VTX-2337(TLR8 agonist immunotherapy)

VentiRx PharmaceuticalsSeattle, WA

head and neck cancer(see also ovarian)

Phase IIwww.ventirx.com



ABT-199/RG7601(Bcl-2 inhibitor)

AbbVieNorth Chicago, ILGenentechSouth San Francisco, CA

hematological malignancies(see also leukemia)

Phase Iwww.abbvie.comwww.gene.com

ACE-536(TGF-ß protein super family inhibitor) ORPHAN DRUG

Acceleron PharmaCambridge, MACelgeneSummit, NJ

myelodysplastic syndromes Phase IIwww.acceleronpharma.comwww.celgene.com





AG-120(IDH1 inhibitor)

Agios PharmaceuticalsCambridge, MACelgeneSummit, NJ

hematological malignancies(see also solid tumors)

Phase Iwww.agios.comwww.celgene.com

AG-221(IDH2 inhibitor)ORPHAN DRUG


hematological malignancies Phase Iwww.agios.comwww.celgene.com



hematological malignancies(see also bladder, myeloma, skin)


AMG 319(PI3K-delta inhibitor)


hematological malignancies Phase Iwww.amgen.com

AMG 900(aurora kinase inhibitor)




anti-LAG3 antibody Bristol-Myers SquibbPrinceton, NJ

hematological malignancies Phase Iwww.bms.com

AR-42(pan-DAC inhibitor)

Arno TherapeuticsFlemington, NJ


Phase Iwww.arnothera.com

AZD1208(PIM kinase inhibitor)


hematological malignancies Phase Iwww.astrazeneca.com

AZD9150(STAT3 inhibitor)

AstraZenecaWilmington, DEIsis PharmaceuticalsCarlsbad, CA

hematological malignancies Phase Iwww.astrazeneca.comwww.isispharm.com



hematological malignancies(see also brain, liver, solid tumors)




myelodysplastic syndromes (see also colorectal, ovarian)

Phase IIwww.tetralogicpharm.com

BP-100-1.01(loposomal Grb 2)

Bio-Path HoldingsHouston, TX

myelodysplastic syndromes(see also leukemia)

Phase Iwww.biopathholdings.com

BPX-501(T cell replacement therapy)

Bellicum PharmaceuticalsHouston, TX

bone marrow transplantation in hematological malignancies

Phase I/IIwww.bellicum.com

CB-839(glutaminase inhibitor)

Calithera BiosciencesSouth San Francisco, CA


Phase Iwww.calithera.com





CC-486 CelgeneSummit, NJ

myelodysplastic syndromes(see also leukemia, solid tumors)


CPI-0610(BET inhibitor)

Constellation PharmaceuticalsCambridge, MA

myelodysplastic syndromes(see also leukemia, lymphoma, myeloma)

Phase Iwww.constellationpharma.com

CPX-351(cytarabine/daunorubicin)



Phase IIwww.celatorpharma.com

EP-100(membrane disrupting peptide)

Esperance PharmaceuticalsSan Francisco, CA


Phase IIwww.esperancepharma.com

FF-10501(cell di� erentiation stimulant)

Boston StrategicsBoston, MAFUJIFILM Pharmaceuticals USABoston, MA

hematological malignancies Phase I/IIwww.bostonstrategics.com

GDC-0853 GenentechSouth San Francisco, CA

hematological malignancies Phase Iwww.gene.com

GS-9973(Syk inhibitor)

Gilead SciencesFoster City, CA

hematological malignancies Phase IIwww.gilead.com

HDM201(tumor suppressor proteinp53 modulator)



Phase Iwww.novartis.com

HSC835(stem cell therapy)


hematological malignancies Phase I/IIwww.novartis.com

imetelstat GeronMenlo Park, CA

hematological malignancies Phase IIwww.geron.com

ixazomib(proteasome inhibitor)


hematological malignancies(see also lymphoma, myeloma, solid tumors)

Phase Iwww.millennium.com

KB004(EphA3 kinase inhibitor)

KaloBios PharmaceuticalsSouth San Francisco, CA

hematological malignancies Phase IIwww.kalobios.com

Keytruda®


hematological malignancies(see also bladder, breast, head/neck, kidney, lung, myeloma, stomach)




relapsed or refractory hematological malignancies(see also brain, leukemia, prostate, solid tumors)

Phase Iwww.karyopharm.com





LGH447(proto oncogene protein cpim-1 inhibitor)


myelodysplastic syndromes(see also leukemia, myeloma)


MEDI-551 + rituximab(anti-CD19 mAb/anti-CD20 mAb)


hematological malignancies Phase Iwww.medimmune.com



hematological malignancies(see also breast, solid tumors)


MLN4924(Nedd8-activating enzyme inhibitor)




mocetinostat(HDAC inhibitor)ORPHAN DRUG


myelodysplastic syndromes(see also bladder, lymphoma)


momelotinib(JAK inhibitor)ORPHAN DRUG


myelo� brosis(see also pancreatic)

Phase IIIwww.gilead.com

MRX34(liposome-encapsulatedmiR-34 mimic)

Mirna TherapeuticsAustin, TX

hematological malignancies(see also liver)

Phase Iwww.mirnarx.com

NiCord®

cord blood stem cell therapyGamida CellJerusalem, Israel

hematological malignancies Phase IIIwww.gamide-cell.com



hematological malignancies(see also brain, colorectal, head/neck, kidney, liver, lung, lymphoma, skin, solid tumors)

Phase Iwww.bms.com



hematological malignancies(see also brain, colorectal, head/neck, kidney, lung, skin)

Phase Iwww.bms.com

OMP-52M51(NOTCH-1 inhibitor)

OncoMed PharmaceuticalsRedwood City, CA


Phase Iwww.oncomed.com

oprozomib(proteasome inhibitor)

Onyx PharmaceuticalsSouth San Francisco, CA

hematological malignancies (monotherapy)(see also solid tumors)-----------------------------------------hematological malignancies(combination therapy)

Phase IIwww.onyx.com

-----------------------------------------Phase Iwww.onyx.com





OXi4503(vascular disrupting agent)

OxiGENESouth San Francisco, CA


Phase Iwww.oxigene.com

pracinostat(HDAC inhibitor)

MEI PharmaSan Diego, CA

myelodysplastic syndromes (1st-line),refractory myelodysplastic syndromes(see also leukemia)

Phase IIwww.meipharma.com

Promacta®

eltrombopagGlaxoSmithKlineResearch Triangle Park, NC


Phase IIwww.gsk.com

Revlimid®

lenalidomideORPHAN DRUG

CelgeneSummit, NJ

myelodysplastic syndromes (non deletion 5q) (see also leukemia, lymphoma, myeloma)


RG7388(dual Raf/MEK inhibitor)



Phase Iwww.gene.com

rigosertib(PI3K/Plk1 inhibitor)ORPHAN DRUG

Onconova TherapeuticsNewtown, PA

high-risk and low-risk myelodysplastic syndromes

Phase IIIwww.onconova.com

sapacitabineORPHAN DRUG

Cyclacel PharmaceuticalsBerkeley Heights, NJ

myelodysplastic syndromes(see also leukemia, lung)

Phase IIwww.cyclacel.com

SGI110(DNMT inhibitor)

Astex PharmaceuticalsDublin, CA

myelodysplastic syndromes(see also leukemia, liver, ovarian)

Phase IIwww.astx.com

simtuzumab Gilead SciencesFoster City, CA

myelo� brosis(see also colorectal)


SL-401(IL-3R inhibitor)


blastic plasmacytoid dendritic cell neoplasm(see also leukemia)

Phase I/IIwww.stemline.com

sotatercept(activin inhibitor)ORPHAN DRUG

Acceleron PharmaCambridge, MACelgeneSummit, NJ

myelodysplastic syndromes Phase IIwww.acceleronpharma.comwww.celgene.com

SP-02(darinaparsin)

Solasia PharmaTokyo, Japan


Phase Iwww.solasia.co.jp

StemEx®

carlecortemcel-LORPHAN DRUG

Gamida Cell-Teva Joint VentureJerusalem, Israel

hematological malignancies(Fast Track)

Phase IIIwww.gamida-cell.com

TAK-659(SYK kinase inhibitor)








TGR-1202(PI3-delta kinase inhibitor)

TG TherapeuticsNew York, NY

hematological malignancies Phase Iwww.tgtherapeutics.com

TH-302(hypoxia-activated prodrug)

EMD SeronoRockland, MAThreshold PharmaceuticalsSouth San Francisco, CA

hematological malignancies (see also lung, pancreatic, sarcoma, skin, solid tumors)

Phase Iwww.emdserono.comwww.thresholdpharm.com

TZ101(human recombinant enzyme)

TargazymeSan Antonio, TX

cord blood stem cell transplantation for hematological malignancies

Phase Iwww.targazyme.com

ulocuplumab Bristol-Myers SquibbPrinceton, NJ

hematological malignancies Phase Iwww.bms.com

urelumab Bristol-Myers SquibbPrinceton, NJ


Phase Iwww.bms.com

varlilumab(CD27 antigen inhibitor)



Phase Iwww.celldex.com

WT2725(cancer immunotherapy)

Sunovion PharmaceuticalsMarlborough, MA


Phase Iwww.sunovion.com

Kidney Cancer


AGS-003(personalized dendritic cell-based vaccine)

Argos TherapeuticsDurham, NC

metastatic renal cell carcinoma(combination therapy) (Fast Track)-----------------------------------------metastatic renal cell carcinoma(monotherapy)

Phase IIIwww.argostherapeutics.com-----------------------------------------Phase IIwww.argostherapeutics.com

AGS-16M8F/AGS-16C3F(antibody drug conjugate)

AgensysSanta Monica, CAAstellas PharmaTokyo, Japan

kidney cancer Phase Iwww.agensys.comwww.astellas.com

AMG 172(antibody drug conjugate)


kidney cancer Phase Iwww.amgen.com

Archexin®

AKT1 inhibitorORPHAN DRUG

RexahnRockville, MD

metastatic kidney cancer Phase I/IIwww.rexahn.com



Kidney Cancer


BNC105(vascular disrupting agent)

BionomicsThebarton, Australia

renal cell carcinoma(see also ovarian)

Phase IIwww.bionomics.com.au

Cometriq®

cabozantinibExelixisSouth San Francisco, CA

metastatic renal cell carcinoma(see also liver)

Phase IIIwww.exelixis.com

dalantercept(ALK1 antagonist)

Acceleron PharmaCambridge, MA

renal cell carcinoma(see also liver)

Phase IIwww.acceleronpharma.com

HyperAcute™ Renalrenal cancer immunotherapyvaccine


metastatic renal cancer Phase Iwww.newlinkgenetics.com

IMA901(multiple tumor-associated peptides)

immatics BiotechnologiesTuebingen, Germany

renal cancer Phase IIIwww.immatics.com

Inlyta®

axitinibP� zerNew York, NY

renal cell carcinoma (adjuvant)(see also liver)

Phase IIIwww.p� zer.com

Keytruda®


kidney cancer(see also bladder, breast, head/neck, hematological, lung, myeloma, stomach)

Phase I/IIwww.merck.com

LY2510924(CXCR4 peptide antagonist)


metastatic clear-cell renal cell carcinoma(see also lung)


Nexavar®

sorafenibBayer HealthCare PharmaceuticalsWhippany, NJ

renal cell carcinoma (adjuvant)(see also breast)




renal cell carcinoma (2nd/3rd-line)(see also brain, colorectal, head/neck, hematological, liver, lung, lymphoma, skin, solid tumors)-----------------------------------------renal cell carcinoma

Phase II/IIIwww.bms.com

-----------------------------------------Phase Iwww.bms.com



renal cell carcinoma (1st-line)(see also brain, colorectal, head/neck, hematological, lung, skin)-----------------------------------------renal cell carcinoma



pexastimogene devacirepvec(genetically-engineered oncolytic vaccinia virus)

SillaJen BiotherapeuticsSan Francisco, CA

treatment refractory kidney cancer(see also colorectal, liver)




Kidney Cancer




renal cell carcinoma(combination therapy)(see also bladder, lung, skin)


SGN-CD70A Seattle GeneticsBothell, WA

renal cell carcinoma(see also leukemia)

Phase Iwww.seattlegenetics.com

sonepcizumab(anti-sphingosine-1-phosphate mAb)

LpathSan Diego, CA

renal cell carcinoma Phase IIwww.lpath.com

Stivarga®


renal cancer(see also colorectal, liver, solid tumors)

Phase IIwww.bayerpharma.com

Sutent®

sunitinibP� zerNew York, NY

renal cell carcinoma (adjuvant) Phase IIIwww.pfizer.com



renal cell carcinoma(see also brain, sarcoma)

Phase I/IIwww.traconpharma.com

volitinib(MET tyrosine kinase inhibitor)


papillary renal cell carcinoma Phase IIwww.astrazeneca.com

Votrient®

pazopanibGlaxoSmithKlineResearch Triangle Park, NC

renal cell cancer (adjuvant)(see also ovarian)-----------------------------------------renal cell cancer (combination with MK-3475)

Phase IIIwww.gsk.com-----------------------------------------Phase Iwww.gsk.com

Leukemia


Å6 peptide Angstrom PharmaceuticalsSolana Beach, CA

chronic lymphocytic leukemia (CLL), small lymphocytic leukemia

Phase IIwww.angstrominc.com

ABL 001(Bcr-abl tyrosine kinase inhibitor)


leukemia Phase Iwww.novartis.com

ABT-199/RG7601(Bcl-2 inhibitor)

AbbVieNorth Chicago, ILGenentechSouth San Francisco, CA

CLL(see also hematological)

-----------------------------------------acute myeloid leukemia (AML)

Phase IIIwww.abbvie.comwww.gene.com-----------------------------------------Phase IIwww.abbvie.comwww.gene.com



Leukemia


ACP-196(Btk inhibitor)

Acerta PharmaSan Carlos, CA

CLL (combination therapy) (see also lymphoma)

Phase Iwww.acerta-pharma.com



CLL (combination therapy) Phase Iwww.acerta-pharma.com

ADI-PEG-20(PEG arginine deaminase)


AML(see also breast, liver, lung, lymphoma, skin)

Phase IIwww.polarispharma.com

alvocidib(angiogenesis inhibitor)

Tolero PharmaceuticalsLehi, UT

AML (1st-line)

-----------------------------------------relapsed/refractory AML

Phase IIIwww.toleropharmaceuticals.com-----------------------------------------Phase IIwww.toleropharmaceuticals.com

AMG 232 AmgenThousand Oaks, CA

AML(see also solid tumors)


Arzerra™ofatumumabORPHAN DRUG


relapsed CLL(see also lymphoma)

Phase IIIwww.gsk.com

ASP2215 Astellas Pharma USNorthbrook, IL

AML Phase I/IIwww.astellas.com

AZD6738(ATR serine/threonine kinase inhibitor)


CLL(see also head/neck)


bendamustine ready-to-diluteliquid formulationORPHAN DRUG

Eagle PharmaceuticalsWoodcli� Lake, NJ

CLL(see also lymphoma, solid tumors)

application submittedwww.eagleus.com

BI 811283(aurora kinase B inhibitor)

Boehringer Ingelheim PharmaceuticalsRidge� eld, CT

AML Phase II completedwww.boehringer-ingelheim.com

BI 836858 Boehringer Ingelheim PharmaceuticalsRidge� eld, CT

AML Phase Iwww.boehringer-ingelheim.com

BL-8040(CXCR4 receptor antagonist)ORPHAN DRUG

BioLineRxJerusalem, Israel

AML (combination therapy) Phase IIwww.biolinerx.com

blinatumomabORPHAN DRUG


Philadelphia-negative relapsed/refractory ß-precursor ALL(Breakthrough Therapy)(see also lymphoma)

application submittedwww.amgen.com



Leukemia


Bosulif®

bosutinibAvillionLondon, United KingdomP� zerNew York, NY

Philadelphia-chromosome positive chronic myeloid leukemia (CML) (1st-line)

Phase IIIwww.avillionllp.comwww.pfizer.com

BP-100-1.01(liposomal Grb 2)ORPHAN DRUG

Bio-Path HoldingsHouston, TX

ALL, AML, CML(see also hematological)

Phase Iwww.biopathholdings.com

CC-292(Btk inhibitor)

CelgeneSummit, NJ

CLL(see also lymphoma)

Phase Iwww.celgene.com


post-induction AML maintenance(see also hematological, solid tumors)


cerdulatinib(dual Syk-JAK inhibitor)

Portola PharmaceuticalsSouth San Francisco, CA


Phase I/IIwww.portola.com

CNDO-109(natural killer cell stimulant)ORPHAN DRUG

Coronado BiosciencesBurlington, MA

AML Phase I/IIwww.coronadobiosciences.com



acute leukemia(see also hematological, lymphoma, myeloma)


CPX-351(cytarabine/daunorubicin)


ALL, AML(see also hematological)

Phase IIwww.celatorpharma.com

CSL-362(anti-CD123 mAb)

CSLVictoria, AustraliaJanssen Research & DevelopmentRaritan, NJ

AML Phase Iwww.csl.com.auwww.janssenrnd.com

CTL019(chimeric antigen receptor T-cell therapy)


ALL(Breakthrough Therapy)


CWP232291 JW PharmaceuticalSeoul, Korea

AML Phase I

DFP-10917(cell cycle inhibitor)

Delta-Fly PharmaTokushima, Japan

ALL, AML Phase I/II

EPZ-5676(DOT1L inhibitor)ORPHAN DRUG

CelgeneSummit, NJEpizymeCambridge, MA

ALL and AML with altered MLL gene Phase Iwww.celgene.comwww.epizyme.com



Leukemia


Erivedge®

vismodegibGenentechSouth San Francisco, CA

AML(see also skin)


Erwinaze®

asparaginase erwinia chrysanthemi intravenousORPHAN DRUG

Jazz PharmaceuticalsPalo Alto, CA

ALL

-----------------------------------------ALL in patients who have developed a hypersensitivity to 1st-lineasparaginase therapy

application submittedwww.jazzpharma.com-----------------------------------------Phase IIwww.jazzpharma.com

ERY-ASP(asparagine modulator)ORPHAN DRUG

ERYTECH PharmaceuticalsLyon, France

ALL (adults) Phase Iwww.erytech.com

EZN-2285(calaspargase pegol)

Sigma-Tau PharmaceuticalsGaithersburg, MD

ALL Phase III completedwww.sigmatau.com

FPI-01(multi-peptide immunotherapeutic)

Formula PharmaceuticalsBerwyn, PA

ALL, AML(see also myeloma)

Phase IIwww.formulapharma.com



AML(see also breast, lung, ovarian)


GMI-1271(E-selectin antagonist)

GlycoMimeticsGaithersburg, MD

AML Phase Iwww.glycomimetics.com

HSV-Tk(thymidine kinase cell therapy)ORPHAN DRUG

MolMedMilan, Italy

high risk acute leukemia Phase IIIwww.molmed.com

Iclusig®

ponatinibARIAD PharmaceuticalsCambridge, MA

AML(see also stomach)

Phase IIwww.ariad.com

IGN523(antibody drug conjugate)

IgenicaBurlingame, CA

AML Phase Iwww.igenica.com

Imbruvica®

ibrutinibJanssen Research & DevelopmentRaritan, NJ

relapsed/refractory CLL(see also lymphoma)-----------------------------------------relapsed/refractory CLL (combination therapy), treatment naïve CLL

application submittedwww.janssenrnd.com-----------------------------------------Phase IIIwww.janssenrnd.com

IMMU-114(humanized anti-HLA-DR)



Phase Iwww.immunomedics.com

Imprime PGG®


high-risk CLL (1st-line)(see also colorectal, lung, lymphoma, pancreatic)

Phase IIwww.biothera.com



Leukemia


inotuzumab ozogamicin(CD22-targeted cytotoxic agent)ORPHAN DRUG


ALL Phase IIIwww.pfizer.com

Iomab™ BBC8-I-131 construct

Actinium PharmaceuticalsNew York, NY

hematopoietic stem cell transplantation in ALL

Phase IIwww.actiniumpharma.com

IPI-145(PI3K inhibitor)

In� nity PharmaceuticalsCambridge, MD

CLL(see also lymphoma)-----------------------------------------high-risk CLL (treatment naïve)

Phase IIIwww.in� .com-----------------------------------------Phase Iwww.in� .com

JCAR014(T lymphocyte immunotherapy)

Juno TherapeuticsSeattle, WA

acute ALL, refractory CLL(see also lymphoma)

Phase I/IIwww.junotherapeutics.com



B-cell ALL Phase Iwww.junotherapeutics.com



leukemia Phase I/IIwww.junotherapeutics.com



elderly AML (2nd-line)(see also brain, hematological, prostate, solid tumors)


KX01(non-ATP competitive Sre kinase inhibitor)

Kinex PharmaceuticalsBu� alo, NY

AML Phase I completedwww.kinexpharma.com



AML(see also hematological, myeloma)


lintuzumab Ac-226 Actinium PharmaceuticalsNew York, NY

AML (1st-line) Phase I/IIwww.actiniumpharma.com

Marqibo®

vinCRIStine sulfate LIPOSOME injectionORPHAN DRUG


elderly Ph-negative ALL (1st-line)(Fast Track)(see also lymphoma, other)


MEDI-551(anti-CD19 mAb)



Phase IIwww.medimmune.com

MGD006(CD123/CD3 bispeci� c antibody)

MacrogenicsRockville, MD

AML Phase Iwww.macrogenics.com

milatuzumab-doxorubicin (antibody drug conjugate)



Phase I/IIwww.immunomedics.com



Leukemia


mogamulizumab(anti-CCR4 humanized antibody)ORPHAN DRUG

Kyowa Hakko PharmaPrinceton, NJ

adult T-cell leukemia (see also lymphoma)

Phase IIwww.kyowa-kirin.com

MOR208(CD19 antigen inhibitor)ORPHAN DRUG

MorphoSysMartinsried, Germany

ALL, CLL(see also lymphoma)

Phase IIwww.morphosys.com

moxetumomab pasudotox(anti-CD22 recombinantimmunotoxin)


hairy cell leukemia

-----------------------------------------pediatric ALL

Phase IIIwww.medimmune.com-----------------------------------------Phase Iwww.medimmune.com

nivolumab + Sprycel®dasatinib


CML Phase Iwww.bms.com

Oncoquest™-CLLpersonalized cancer vaccine

XEME BiopharmaMonmouth Junction, NJ

CLL (1st-line) Phase Iwww.xemebiopharma.com

otlertuzumab(CD37 protein inhibitor)ORPHAN DRUG

Emergent BioSolutionsRockville, MD

CLL Phase IIwww.emergentbiosolutions.com

OXi4503(vascular disrupting agent)ORPHAN DRUG

OxiGENESouth San Francisco, CA

refractory AML(see also hematological)

Phase Iwww.oxigene.com

PF-04449913(SMO [smoothened] antagonist)


AML Phase IIwww.pfizer.com

PKC412(signal transduction inhibitor)ORPHAN DRUG


AML Phase IIIwww.novartis.com



AML(see also brain, skin)

Phase IIwww.dsi.comwww.plexxikon.com

PLX8394(BRAF inhibitor)


hairy cell leukemia(see also solid tumors)

Phase Iwww.dsi.comwww.plexxikon.com

PM01183(marine-derived alkylating agent)

Pharma MarMadrid, Spain

acute leukemia(see also breast)

Phase IIwww.pharmamar.com

pracinostat(HDAC inhibitor)


AML (1st-line) (see also hematological)

Phase IIwww.meipharma.com



Leukemia


PRI-724(CBP/β-catenin inhibitor)

PRISM PharmaTokyo, Japan

AML, CML(see also pancreatic, solid tumors)

Phase I/IIwww.prismbiolab.com

Promacta®

eltrombopagGlaxoSmithKlineResearch Triangle Park, NC

AML (see also hematological)

Phase IIwww.gsk.com

Qinprezzo™vosaroxinORPHAN DRUG

Sunesis PharmaceuticalsSouth San Francisco, CA

relapsed/refractory AML(Fast Track)

Phase IIIwww.sunesis.com

quizartinib(FLT3 inhibitor)

Ambit BiosciencesSan Diego, CA

relapsed refractory AML(Fast Track)

Phase IIIwww.ambitbio.com

Revlimid®


CelgeneSummit, NJ

CLL maintenance (2nd-line)(see also hematological, lymphoma, myeloma)


RG7775(MDM2 IN prodrug)


AML Phase Iwww.gene.com



AML(see also hematological, lung)

Phase IIIwww.cyclacel.com

SG2000(DNA cross linking agent)

SpirogenLondon, United Kingdom

AML, CLL Phase I/II



AML (see also hematological, liver, ovarian)


SGN-CD19A(antibody drug conjugate)


AML(see also lymphoma)




AML Phase Iwww.seattlegenetics.com

SL-401(IL-3R inhibitor)ORPHAN DRUG


advanced AML(see also hematological)

Phase IIwww.stemline.com

Tasigna®

nilotinibNovartis PharmaceuticalsEast Hanover, NJ

CML treatment free remission Phase IIIwww.novartis.com

tetradecanoylphorbol acetate Rich PharmaceuticalsBeverly Hills, CA

AML Phase IIwww.richpharmaceuticals.com



Leukemia


TG02(CDK inhibitor)

Tragara PharmaceuticalsCarlsbad, CA

refractory AML, relapsed CLL(see also myeloma)

Phase Iwww.tragarapharma.com

TG-1101(anti-CD20 mAb)



Phase Iwww.tgtherapeutics.com

volasertib(PLK-1 antagonist)ORPHAN DRUG


AML(Breakthrough Therapy)


Zydelig®

idelalisibGilead SciencesFoster City, CA

CLL (1st-line)(see also lymphoma)


Liver Cancer




hepatocellular carcinoma(see also breast, leukemia, lung, lymphoma, skin)

Phase IIIwww.polarispharma.com

BA-003(doxorubicin nanoparticles)ORPHAN DRUG

OnexoParis, France

hepatocellular carcinoma (2nd-line)(Fast Track)

Phase IIIwww.onexo.com


liver cancer(see also breast, lung, skin, other)

Phase IIwww.peregrineinc.com



advanced cholangiocarcinoma(see also brain, hematological, solid tumors)


CBL0102(SSRP1 protein inhibitor)ORPHAN DRUG

Cleveland BioLabsBu� alo, NYIncuronMoscow, Russia

hepatocellular carcinoma Phase I/IIwww.cbiolabs.comwww.incuron.ru

CF102(A3 adenosine receptor agonist)

Can-Fite BioPharmaPetach Tikva, Israel

hepatocellular carcinoma Phase IIwww.canfite.com

Cometriq®

cabozantinibExelixisSouth San Francisco, CA

late-stage hepatocellular carcinoma(see also kidney)

Phase IIIwww.exelixis.com



Liver Cancer


CX-4945(casein kinase II inhibitor)

Senhwa BiosciencesSan Diego, CA

cholangiocarcinoma Phase IIwww.senhwabiosciences.com

Cyramza®

ramucirumabORPHAN DRUG


hepatocellular carcinoma (combination therapy)(see also bladder, breast, colorectal, lung, ovarian, solid tumors, stomach)


dalantercept(ALK1 antagonist)

Acceleron PharmaCambridge, MA

hepatocellular carcinoma(see also kidney)

Phase IIwww.acceleronpharma.com

DCR-MYC(c-myc inhibitor)

Dicerna PharmaceuticalsWatertown, MA

hepatocellular carcinoma Phase Iwww.dicerna.com

doxorubicin nanoparticlesORPHAN DRUG

BioAlliance PharmaParis, France

liver cancer (2nd-line) Phase IIIwww.bioalliancepharma.com



hepatocellular carcinoma (see also brain, lung, skin, other)


G-202(calcium-transporting ATPase inhibitor)ORPHAN DRUG


liver cancer(see also brain)




liver cancer(see also brain, pancreatic)


GC33/RG7686(anti-GPC3 antibody)

Chugai PharmaceuticalTokyo, JapanRocheNew York, NY

metastatic liver cancer Phase IIwww.chugai.comwww.roche.com

INC280(c-Met inhibitor)

IncyteWilmington, DENovartis PharmaceuticalsEast Hanover, NJ

advanced liver cancer with c-Met dysregulation

Phase IIwww.incyte.comwww.novartis.com

Inlyta®

axitinibP� zerNew York, NY

liver cancer(see also kidney)

Phase IIwww.pfizer.com

ipafricept(Wnt protein inhibitor)

Bayer HealthCare PharmaceuticalsWhippany, NJOncoMed PharmaceuticalsRedwood City, CA

hepatocellular cancer(see also ovarian, pancreatic)

Phase Iwww.bayerpharma.comwww.oncomed.com

KD018(Chinese botanical extract)


liver cancer(see also colorectal)




Liver Cancer


melphalan drug delivery system(drug-device combination)ORPHAN DRUG

Delcath SystemsNew York, NY

secondary liver cancer arising from metastatic ocular melanoma(Fast Track)

application submittedwww.delcath.com

mipsagargin(ATPase inhibitor)ORPHAN DRUG


liver cancer(see also brain, prostate)


MRX34(liposome-encapsulated miR-34 mimic)

Mirna TherapeuticsAustin, TX

unresectable primary liver cancer, liver metastatases from other solid tumors(see also hematological)

Phase Iwww.mirnarx.com



hepatocellular carcinoma(see also brain, colorectal, head/neck, hematological, kidney, lung, lymphoma, skin, solid tumors)

Phase Iwww.bms.com

PDT with Photofrin®

por� mer sodiumORPHAN DRUG

Pinnacle BiologicsBannockburn, IL

unresectable advanced perihilar cholangiocarcinoma Bismuth type III/IV (see also lung)

Phase IIIwww.pinnaclebiologics.com

pexastimogene devacirepvec(genetically-engineered oncolytic vaccinia virus)ORPHAN DRUG

StillaJen BiotherapeuticsSan Francisco, CA

advanced primary liver cancer(see also colorectal, kidney)


PF-03446962(ALK1 inhibitor)


hepatocellular carcinoma (2nd-line) Phase IIwww.pfizer.com

PV-10(rose bengal disodium)ORPHAN DRUG

Provectus BiopharmaceuticalsKnoxville, TN

liver metastases(see also skin)

Phase Iwww.pvct.com

refametinib(MEK inihibitor)

Bayer HealthCare PharmaceuticalsWhippany, NJ

RAS mutant hepatocellular carcinoma(see also solid tumors)

Phase IIwww.bayerpharma.com



liver cancer(see also hematological, leukemia, ovarian)




advanced liver cancer(see also colorectal, pancreatic, stomach)


SPI-1620(endothelin B receptor agonist)


biliary cancer(see also lung)

Phase IIwww.sppirx.com



Liver Cancer


Stivarga®


hepatocellular carcinoma (2nd-line)(see also colorectal, kidney, solid tumors)


ThermoDox®

doxorubicin liposomalORPHAN DRUG

CelsionLawrenceville, NJ

primary liver cancer(Fast Track)(see also breast, colorectal)-----------------------------------------liver metastases

Phase IIIwww.celsion.com

-----------------------------------------Phase IIIwww.celsion.com

tivantinib(c-Met inhibitor)

ArQule Woburn, MADaiichi Sankyo Parsippany, NJ

hepatocellular carcinoma Phase IIIwww.arqule.comwww.dsi.com

TKM-PLK1(RNA interference)

Tekmira PharmaceuticalsBurnaby, Canada

hepatocellular carcinoma(see also stomach, other)

Phase IIwww.tekmira.com

VT-122(cancer immunotherapy)

Vicus TherapeuticsMorristown, NJ

advanced liver cancer(see also prostate)

Phase IIwww.vicustherapeutics.com

Lung Cancer




non-small-cell lung cancer (NSCLC)(see also breast, lymphoma)

Phase Iwww.lilly.com

ABT-RTA-408 AbbVieNorth Chicago, ILReata PharmaceuticalsIrving, TX

metastatic NSCLC(see also skin)

Phase Iwww.abbvie.comwww.reatapharma.com



small-cell lung cancer (SCLC)(see also breast, leukemia, liver, lymphoma, skin)-----------------------------------------mesothelioma, NSCLC


-----------------------------------------Phase Iwww.polarispharma.com

A� nitor®


neuroendocrine lung tumors(see also breast, lymphoma, stomach)




Lung Cancer


aldoxorubicin(delivery-enhanced doxorubicin)


SCLC(see also brain, sarcoma)

Phase IIwww.cytrx.com

alectinib(ALK inhibitor)

Chugai Pharma USABerkeley Heights, NJRocheBasel, Swizterland

NSCLC(Breakthrough Therapy)

Phase IIwww.chugai-pharm.comwww.roche.com

alisertib(auroura A kinase inhibitor)


SCLC(see also lymphoma, ovarian)


antroquinonol Golden BiotechnologyNew Taipei City, Taiwan

NSCLC Phase IIwww.goldenbiotech.com.tw

AP26113(ALK inhibitor)

ARIAD PharmaceuticalsCambridge, MA

NSCLC Phase IIwww.ariad.com



non-squamous NSCLC, squamous NSCLC(see also bladder, pancreatic, prostate)


ASP8273 Astellas Pharma USNorthbrook, IL

NSCLC Phase I/IIwww.astellas.com

AT13387(HSP90 inhibitor)


NSCLC(see also skin)


Avastin®

bevacizumabGenentechSouth San Francisco, CA

NSCLC (adjuvant)(see also brain, ovarian, other)


AZD9291(EGFR tyrosine kinase inhibitor)


advanced T790M mutation-positive NSCLC(Breakthrough Therapy)

Phase II/IIIwww.astrazeneca.com


NSCLC (2nd-line)(see also breast, liver, skin, other)-----------------------------------------NSCLC (1st-line)

Phase IIIwww.peregrineinc.com-----------------------------------------Phase Iwww.peregrineinc.com



NSCLC(see also breast, colorectal, ovarian, skin, stomach)


belinostat Spectrum PharmaceuticalsHenderson, NV

NSCLC Phase I/IIwww.sppirx.com



Lung Cancer


bevacizumab biosimilar ActavisParsippany, NJAmgenThousand Oaks, CA

NSCLC Phase IIIwww.actavis.comwww.amgen.com

BIND-014(PSMA-targeted docetaxel)

BIND TherapeuticsCambridge, MA

NSCLC(see also prostate)

Phase IIwww.bindtherapeutics.com

CRS-207(cancer immunotherapy)

Aduro BioTechBerkeley, CA

mesothelioma (combination therapy)(see also pancreatic)

Phase Iww.adurobiotech.com

custirsen (OGX-111)(antisense oligonucleotide)

OncoGenex PharmaceuticalsBothell, WATeva PharmaceuticalFrazier, PA

NSCLC (Fast Track)(see also prostate)

Phase IIIwww.oncogenex.comwww.tevapharm.com

Cynviloq™paclitaxel polymeric micelle for injection


NSCLC(see also bladder, breast, ovarian, pancreatic)

Phase IIIwww.sorrentotherapeutics.com

Cyramza®


NSCLC (combination therapy)(see also bladder, breast, colorectal, liver, ovarian, solid tumors, stomach)


CYT-6091(nanomedicine)

CytImmune SciencesRockville, MD

NSCLC (2nd-line) Phase IIwww.cytimmune.com

dacomitinib(pan-HER inhibitor)


EGFR-mutant NSCLC (1st-line)(see also solid tumors)

Phase IIIwww.p� zer.com

DKN-01(DKK1 protein inhibitor)

HealthCare PharmaceuticalsCambridge, MA

NSCLC(see also myeloma, other)

Phase Iwww.healthcarepharmaceuticals.com

DPV-001(toll-like receptor agonist)

UbiVacPortland, OR

NSCLC Phase IIwww.ubivac.com



NSCLC (3rd-line monotherapy), NSCLC (RET translocations)(see also brain, liver, skin, other)


EGF816(EGFR TK inhibitor)


NSCLC Phase I/IIwww.novartis.com

� clatuzumab(HGF/c-MET inhibitor)

AVEO OncologyCambridge, MA

NSCLC Phase IIwww.aveooncology.com

foretinib(c-MET kinase inhibitor)


NSCLC Phase IIwww.gsk.com



Lung Cancer




NSCLC (Fast Track)(see also breast, leukemia, ovarian)

Phase IIIwww.syntapharma.com

GI-4000(natural killer cell stimulant)

GlobeImmuneLouisville, CO

NSCLC(see also pancreatic)

Phase IIwww.globeimmune.com



lung, malignant mesothelioma(see also head/neck, solid tumors, other)

Phase Iwww.genelux.com

GSK2879552(LSD1/CoREST inhibitor)


relapsed/refractory SCLC Phase Iwww.gsk.com

Halaven®

eribulinEisaiWoodcli� Lake, NJ

NSCLC(see also breast, sarcoma)


Imprime PGG®


metastatic NSCLC (1st-line)(see also colorectal, leukemia, lymphoma, pancreatic)


INCB24360(IDO1 inhibitor)


NSCLC(see also ovarian, skin, other)

Phase I/IIwww.incyte.com

INCB39110(JAK1 inhibitor)


NSCLC(see also solid tumors)

Phase I/IIwww.incyte.com

ISIS-EIF4ERX(antisense oligonucleotide)

Isis PharmaceuticalsCarlsbad, CA

NSCLC(see also prostate)

Phase IIwww.isispharm.com

Jaka� ® ruxolitinib


NSCLC(see also breast, colorectal, pancreatic, other)


KD019(TKI inhibitor)


NSCLC(see also breast)-----------------------------------------Src-positive NSCLC

Phase IIwww.kadmon.com-----------------------------------------Phase Iwww.kadmon.com

KD032(Ras antagonist)


NSCLC(see also colorectal, pancreatic)

Phase IIwww.kadmon.com

Keytruda®


NSCLC(see also bladder, breast, head/neck, hematological, kidney, myeloma, stomach)

Phase IIIwww.merck.com

Kyprolis®

car� lzomabOnyx PharmaceuticalsSouth San Francisco, CA

SCLC(see also myeloma)

Phase IIwww.onyx.com



Lung Cancer


lifastuzumab vedotin(anti-NaPi2b antibody drugconjugate)(RG7599)


NSCLC(see also ovarian)

Phase Iwww.gene.com

lucitanib(FGFR/VEGFR/PDGFR inhibitor)


squamous NSCLC(see also breast)


lung cancer DNA vaccine(IC 1001)

Immune Cell TherapyChicago, IL

NSCLC Phase Iwww.immcellther.com

LY2510924(CXCR4 peptide antagonist)


SCLC(see also kidney)


LY2875358(c-Met mAb)


NSCLC(see also stomach)


LY2940680(hedgehog/SMO antagonist)


SCLC Phase I/IIwww.lilly.com

MAGE-A3 immunotherapeutic (recombinant antigen-speci� c cancer immunotherapeutic)


NSCLC Phase IIIwww.gsk.com

ME-344(mitochondrial inhibitor)


SCLC(see also ovarian)

Phase Iwww.meipharma.com

MEDI4736(anti-PD-L1 mAb)


stage III NSCLC, NSCLC (3rd-line) Phase IIIwww.medimmune.com

MEDI4736 + Iressa® ge� tinib(anti-PD-L1 mAb/EGFR inhibitor)


NSCLC Phase Iwww.medimmune.com

MGCD265(MET/Axl/VEGFR kinase inhibitor)


NSCLC(see also head/neck)

Phase Iwww.mirati.com



NSCLC(see also breast, ovarian)


MORAb-003(IgG1 mAb)

EisaiWoodcli� Lake, NJMorphotekExton, PA

NSCLC(see also ovarian)


MORAb-009(megakaryocyte potentiatingfactor inhibitor)ORPHAN DRUG


mesothelioma

-----------------------------------------solid tumors

Phase IIwww.eisai.com-----------------------------------------Phase Iwww.eisai.com



Lung Cancer


NC-6004(nanoparticle cisplatin)

NanoCarrierTokyo, Japan

NSCLC Phase I/IIwww.nanocarrier.co.jp

necitumumab(EGFR inhibitor)


squamous NSCLC Phase IIIwww.lilly.com



NSCLC (1st-line), squamous NSCLC (2nd-line), non-squamous NSCLC (2nd-line)(see also brain, colorectal, head/neck, hematological, kidney, liver, lymphoma, skin, solid tumors)-----------------------------------------squamous NSCLC (3rd-line)

-----------------------------------------NSCLC, SCLC


-----------------------------------------Phase IIwww.bms.co-----------------------------------------Phase IIwww.bms.com



NSCLC, SCLC(see also brain, colorectal, head/neck, hematological, kidney, skin)

Phase Iwww.bms.com

patritumab(anti-HER3 antibody)


NSCLC(see also breast)

Phase IIwww.dsi.com

PDT with Photofrin®

por� mer sodiumORPHAN DRUG

Pinnacle BiologicsBannockburn, IL

epithelioid malignant pleural mesothelioma(see also liver)

Phase IIIwww.pinnaclebiologics.com


NSCLC, squamous cell carcinoma of the lung(see also colorectal, head/neck, myeloma, ovarian, pancreatic, skin, solid tumors, other)

Phase IIwww.oncolyticsbiotech.com

pictrelisib(PI3K inhibitor)


metastatic NSCLC(see also breast)


PRAME immunotherapeutic(recombinant antigen-speci� c cancer immunotherapeutic)


resectable NSCLC Phase IIwww.gsk.com

PT 107(allogeneic B7.1/HLA-A1 transfected tumor cell vaccine)

Pique TherapeuticsDurham, NC

NSCLC Phase IIwww.piquetherapeutics.com



Lung Cancer




NSCLC (2nd-line)(see also bladder, kidney, skin)

-----------------------------------------NSCLC (3rd-line)

-----------------------------------------EGFR-positive NSCLC


-----------------------------------------Phase IIwww.gene.com-----------------------------------------Phase Iwww.gene.com

rociletinib (CO-1686)(EGFR inhibitor)


EGFR-mutated NSCLC(Breakthrough Therapy)


roniciclib(CDK inhibitor)


SCLC Phase IIwww.bayerpharma.com



NSCLC(see also hematological, leukemia)


SC16LD6.5(antibody drug conjugate)

Stem CentRxSouth San Francisco, CA

SCLC Phase I/IIwww.stemcentrx.com

seliciclib(CDK inhibitor)


NSCLC(see also other)


selumetinib(MEK inhibitor)

Array BioPharmaBoulder, COAstraZenecaWilmington, DE

KRAS-positive NSCLC (2nd-line)(see also other)

-----------------------------------------KRAS-negative NSCLC (2nd-line)

Phase IIIwww.arraybiopharma.comwww.astrazeneca.com-----------------------------------------Phase IIwww.arraybiopharma.comwww.astrazeneca.com

SPI-1620(endothelin B receptor agonist)


NSCLC(see also liver)

Phase IIwww.sppirx.com

Ta� nlar®

dabrafenibGlaxoSmithKlineResearch Triangle Park, NC

NSCLC(Breakthrough Therapy)

Phase IIwww.gsk.com

tarextumab(anti-NOTCH 2/3)


SCLC(see also pancreatic)

Phase I/IIwww.oncomed.com

Tavocept™ BioNumerik PharmaceuticalsSan Antonio, TX

lung cancer Phase IIIwww.bionumerik.com

tergenpumatucel-L NewLink GeneticsAmes, IA

advanced or metastatic NSCLC Phase II/IIIwww.newlinkgenetics.com



Lung Cancer


TG4010(MVA-MUC1-IL2 cancer vaccine)

TransgeneCambridge, MA

advanced NSCLC Phase II/IIIwww.transgene.fr



NSCLC(see also hematological, pancreatic, skin, sarcoma, solid tumors)

Phase IIwww.emdserono.comwww.thresholdpharm.com

topotecan liposomal Spectrum PharmaceuticalsHenderson, NV

lung cancer(see also breast)


tremelimumab(anti-CTLA-4 mAb)


mesothelioma Phase IIIwww.medimmune.com

tremelimumab(anti-CTLA-4 mAb)/Iressa® ge� tinib combination

AstraZenecaWilmington, DEMedImmuneGaithersburg, MD

NSCLC Phase Iwww.astrazeneca.comwww.medimmune.com

TSR-011(ALK/TRK inhibitor)

TESAROWaltham, MA

NSCLC Phase I/IIwww.tesarobio.com

TUSC2(gene therapy)

GenprexAustin, TX

NSCLC Phase I/IIwww.genprex.com

Vargatef™nintedanib


NSCLC(see also ovarian)-----------------------------------------mesothelioma

Phase IIIwww.boehringer-ingelheim.com-----------------------------------------Phase IIwww.boehringer-ingelheim.com


NSCLC(see also brain, breast)

Phase IIIwww.abbvie.com

viagenpumatucel-L(cancer immunotherapy)

Heat BiologicsDurham, NC

NSCLC Phase IIwww.heatbio.com

vinorelbine liposomal Spectrum PharmaceuticalsHenderson, NV

SCLC(see also ovarian)


vintafolide(folate-DAVLBH)

EndocyteWest Lafayette, IN

NSCLC(see also solid tumors)

Phase IIwww.endocyte.com

VS-6063(FAK inhibitor)ORPHAN DRUG

VerastemCambridge, MA

KRAS-mutant NSCLC, mesothelioma(see also ovarian)

Phase IIwww.verastem.com

Xalkori®crizotinibORPHAN DRUG


ALK-positive NSCLC (1st-line)(see also solid tumors)

Phase IIIwww.pfizer.com



Lung Cancer


Xgeva®

denosumabAmgenThousand Oaks, CA

metastatic NSCLC (1st-line)(see also breast)

Phase IIwww.amgen.com

Yervoy®ipilimumab


SCLC (1st-line), squamous NSCLC (1st-line)(see also ovarian, prostate, skin, stomach)


Zykadia™ceritinib


ALK-positive advanced NSCLC(1st-line, treatment naïve)


Lymphoma




mantle cell lymphoma(see also breast, lung)


abexinostat(HDAC inhibitor)

PharmacyclicsSunnyvale, CA

follicular lymphoma, mantle cell lymphoma

Phase IIwww.pharmacyclics.com



di� use large B-cell lymphoma (DLBCL), Waldenström’s macroglobulinemia(see also leukemia)

Phase Iwww.acerta-pharma.com

Adcetris®

brentuximab vedotinORPHAN DRUG


post-transplant Hodgkin lymphoma prevention, relapsed CD30-positive cutaneous lymphoma, Hodgkin lymphoma (1st-line), CD30-positive mature T-cell lymphoma (1st-line)-----------------------------------------relapsed/refractory CD30-positive non-Hodgkin lymphoma (NHL), Hodgkin lymphoma in the elderly (1st-line), DLBCL (1st-line)-----------------------------------------Hodgkin lymphoma (2nd-line)

Phase IIIwww.seattlegenetics.com

-----------------------------------------Phase IIwww.seattlegenetics.com

-----------------------------------------Phase Iwww.seattlegenetics.com



NHL(see also breast, leukemia, liver, lung, skin)




Lymphoma


AEB071(PKC inhibitor)


CD79-mutant or ABC-subtype DLBCL(combination therapy)(see also other)


A� nitor®


DLBCL(see also breast, lung, stomach)


AFM 13(CD30 antigen modulator)ORPHAN DRUG

A� med TherapeuticsHeidelberg, Germany

Hodgkin lymphoma Phase IIwww.a� med.com



relapsed or refractory peripheralT-cell lymphoma(see also lung, ovarian)

Phase IIIwww.millennium.com

AR-12(PI3K/Akt inhibitor)


lymphoma(see also solid tumors)

Phase Iwww.arnothera.com

Arzerra™ofatumumab


relapsed DLBCL, refractory and relapsed follicular lymphoma(see also leukemia)

Phase IIIwww.gsk.com

bendamustine ready-to-diluteliquid formulationORPHAN DRUG


NHL(see also leukemia, solid tumors)

application submittedwww.eagleus.com


NHL Phase Iwww.boehringer-ingelheim.com

BiovaxID®

dasiprotimut T(personalized cancer vaccine)ORPHAN DRUG

Biovest InternationalMinneapolis, MN

follicular NHL (Fast Track), mantle cell lymphoma

Phase IIIwww.bioinvest.com

blinatumomabORPHAN DRUG


DLBCL(see also leukemia)


CC-122(pleiotropic pathway modulator)

CelgeneSummit, NJ

DLBCL(see also solid tumors)


CC-292(Btk inhibitor)

CelgeneSummit, NJ

lymphoma(see also leukemia)


cerdulatinib(dual Syk-JAK inhibitor)

Portola PharmaceuticalsSouth San Francisco, CA

NHL(see also leukemia)

Phase I/IIwww.portola.com

CMD-003(cell therapy)

Cell MedicaLondon, United Kingdom

Epstein-Barr virus lymphoma Phase IIwww.cellmedica.co.uk



Lymphoma


copanlisib(PI3K inhibitor)


NHL Phase IIwww.bayerpharma.com



lymphoma(see also hematological, leukemia, myeloma)


CUDC-427(IAP inhibitor)

CurisLexington, MA

late-stage lymphoma(see also solid tumors)

Phase Iwww.curis.com

CUDC-907(HDAC, PI3K inhibitor)

CurisLexington, MA

relapsed/refractory lymphoma(see also myeloma)


Debio 1143(IAP inhibitor)

DebiopharmaLausanne, Switzerland

(see also solid tumors) Phase Iwww.debiopharm.com

DI-Leu16-IL2(CD20 antigen inhibitor)

ALOPEXX OncologyConcord, MA

NHL Phase I/IIwww.alopexx.com

DS-3032(MDM2 inhibitor)


(see also solid tumors) Phase Iwww.dsi.com

DS-3078(mTOR inhibitor)


(see also solid tumors) Phase Iwww.dsi.com

DS-8273(anti-DR5 antibody)



Phase Iwww.dsi.com

EPZ-6438(EZH2 inhibitor)

EpizymeCambridge, MA

NHL(see also solid tumors)

Phase I/IIwww.epizyme.com

fenretinide intravenous CerRxLubbock, TX

adult lymphoma(see also solid tumors)

Phase IIwww.cerrx.com

Folotyn®

pralatrexate injectionSpectrum PharmaceuticalsHenderson, NV

peripheral T-cell lymphoma (1st-line) Phase IIIwww.sppirx.com

Gazyva®

obinutuzumabGenentechSouth San Francisco, CA

DLBCL, indolent NHL (1st-line), refractory indolent NHL


GDC-0575(ChK1 inhibitor)


lymphoma(see also solid tumors)

Phase Iwww.gene.com



Lymphoma


Imbruvica®

ibrutinibJanssen Research & DevelopmentRaritan, NJ

relapsed/refractory mantle cell lymphoma, treatment naïve mantle cell lymphoma (combination therapy), relapsed/refractory indolent NHL, non-germinal center B-cell subtype of DLBCL, Waldenström’s macroglobulinemia (Breakthrough Therapy)

Phase IIIwww.janssenrnd.com

IMGN529(CD37 protein inhibitor)

ImmunoGenWaltham, MA

NHL Phase Iwww.immunogen.com

IMMU-114(humanized anti-HLA-DR)



Phase Iwww.immunomedics.com

IMO-8400(TLR antagonist)

Idera PharmaceuticalsCambridge, MA

DLBCL, Waldenström’s macroglobulinemia

Phase I/IIwww.iderapharma.com

Imprime PGG®


advanced indolent NHL(see also colorectal, leukemia, lung, pancreatic)


INCB39110/INCB40093(JAK1 inhibitor/PI3K delta inhibitor)


B-lymphoid malignancies Phase Iwww.incyte.com

INCB40093(PI3K delta inhibitor)


B-lymphoid malignancies Phase Iwww.incyte.com

interleukin-12 gene therapy OncoSec MedicalSan Diego, CA

cutaneous T-cell lymphoma (CTCL), Merkel cell carcinoma(see also skin)

Phase IIwww.oncosec.com

IPI-145(PI3K inhibitor)

In� nity PharmaceuticalsCambridge, MD

indolent NHL(see also leukemia)-----------------------------------------mantle cell lymphoma

Phase IIwww.in� .com-----------------------------------------Phase Iwww.in� .com

Istodax®

romidepsinCelgeneSummit, NJ

peripheral T-cell lymphoma (1st-line) Phase IIIwww.celgene.com



relapsed/refractory follicular lymphoma(see also hematological, myeloma, solid tumors)





Phase I/IIwww.junotherapeutics.com



Lymphoma


JNJ-26481585(HDAC inhibitor)

Janssen Research & DevelopmentRaritan, NJ

CTCL Phase IIwww.janssenrnd.com

JNJ-40346527(Fms inhibitor)


Hodgkin lymphoma Phase II completedwww.janssenrnd.com

JNJ-42756493(FGFR inhibitor)



Phase Iwww.janssenrnd.com

KTE-C19 CAR(chimeric antigen receptor)ORPHAN DRUG

Kite PharmaSanta Monica, CANational Cancer InstituteBethesda, MD

DLBCL Phase I/IIwww.kitepharma.com

Marqibo®

vinCRIStine sulfate LIPOSOME injection


aggressive NHL (1st-line)(see also leukemia, other)


MEDI-551(anti-CD19 mAb)


DLBCL(see also leukemia)

Phase IIwww.medimmune.com

milatuzumab-doxorubicin(antibody drug conjugate)



Phase I/IIwww.immunomedics.com

mocetinostat(HDAC inhibitor)ORPHAN DRUG


DLBCL(see also bladder, hematological)


mogamulizumab(anti-CCR4 humanized antibody)ORPHAN DRUG

Kyowa Hakko PharmaPrinceton, NJ

CTCL (see also leukemia)-----------------------------------------adult T-cell lymphoma

Phase IIIwww.kyowa-kirin.com-----------------------------------------Phase IIwww.kyowa-kirin.com

MOR208(CD19 antigen inhibitor)

MorphoSysMartinsried, Germany


Phase IIwww.morphosys.com



Hodgkin lymphoma (Breakthrough Therapy), NHL (1st-line), DLBCL(see also brain, colorectal, head/neck, hematological, kidney, liver, lung, skin, solid tumors)

Phase IIwww.bms.com

non-Hodgkin’s lymphoma vaccine(personalized cancer vaccine)

XEME BiopharmaMonmouth Junction, NJ

non-Hodgkin’s follicular lymphoma Phase IIwww.xemebiopharma.com

ocaratuzumab(AME-133v)

MENTRIK BiotechDallas, TX

lymphoma Phase I compeletedwww.mentrik.com



Lymphoma


ONO-4059(Btk inhibitor)

Ono PharmaceuticalsOsaka, Japan

B cell lymphoma Phase I

pinatuzumab vedotin(anti-CD22 antibody drug conjugate)


DLBCL, NHL Phase IIwww.gene.com

Pixuvri®pixantrone

CTI BioPharmaSeattle, WA

DLBCL Phase IIIwww.ctibiopharma.com

PNT2258(bcl-2 oncogene inhibitor)

ProNAi TherapeuticsPlymouth, MI

NHL(see also solid tumors)

Phase IIwww.pronai.com

polatuzumab vedotin(anti-CD79b antibody drug conjugate)


DLBCL, NHL Phase IIwww.gene.com

Resimmune™A-dmDT390-bisFv immunotoxin

AngimmuneRockville, MD

CTCL(see also skin)

Phase I/IIwww.angimmune.com

Revlimid®

lenalidomideCelgeneSummit, NJ

DLBCL (maintenance), follicular lymphoma (1st-line), relapsed/refractory indolent lymphoma(see also hematological, leukemia, myeloma)-----------------------------------------DLBCL-ABC subtype (1st-line)


-----------------------------------------Phase IIwww.celgene.com



late-stage lymphoma(see also colorectal, solid tumors)

Phase Iwww.radiorx.com

SAR3419(maytansin-loaded anti-CD19 mAb)

Sano� USBridgewater, NJ

B-cell refractory/relapsed malignancies

Phase IIwww.sano� .com





SGN-CD70A Seattle GeneticsBothell, WA

NHL(see also kidney)


SHAPE(HDAC inhibitor)ORPHAN DRUG


CTCL Phase Iwww.tetralogicpharma.com

SNS-01(nano-encapsulated gene therapy)ORPHAN DRUG

Sevion TherapeuticsBridgewater, NJ

DLBCL, mantle cell lymphoma(see also myeloma)

Phase I/IIwww.seviontherapeutics.com



Lymphoma


TG-1101(anti-CD20 mAb)ORPHAN DRUG


mantle cell lymphoma(see also leukemia)

Phase Iwww.tgtherapeutics.com

Treanda®

bendamustine liquidORPHAN DRUG


advanced indolent NHL, mantle cell lymphoma-----------------------------------------relapsed or refractory mantle cell lymphoma(combination therapy)

Phase IIIwww.tevapharm.com-----------------------------------------Phase IIwww.tevapharm.com

trifarotene GaldermaLausanne, Switzerland

CTCL Phase Iwww.galderma.com

Velcade®

bortezomibMillennium PharmaceuticalsCambridge, MA

mantle cell lymphoma (1st-line)

-----------------------------------------DLBCL

Phase IIIwww.millennium.com-----------------------------------------Phase IIwww.millennium.com

veltuzumab(humanized anti-CD20 mAb)


NHL (combination therapy) Phase IIwww.immunomedics.com

VS-5584(PI3K/mTOR inhibitor)



Phase Iwww.verastem.com

Zydelig®

idelalisibGilead SciencesFoster City, CA

indolent NHL(see also leukemia)


Myeloma


afuresertib(AKT protein kinase inhibitor)


multiple myeloma(see also ovarian)

Phase Iwww.gsk.com



multiple myeloma(see also bladder, hematological, skin)


AT7519(CDK inhibitor)


multiple myeloma Phase IIwww.astx.com



Myeloma


BT062(tubulin polymerisation inhibitor)

Biotest PharmaceuticalsDreieich, Germany

multiple myeloma Phase I/IIwww.biotest.de

CB-5083(P97 ATPase inhibitor)

Cleave BiosciencesBurlingame, CA

multiple myeloma Phase Iwww.cleavebio.com



multiple myeloma(see also hematological, leukemia, lymphoma)


CUDC-907(HDAC, PI3K inhibitor)

CurisLexington, MA

multiple myeloma(see also lymphoma)


daratumumab Janssen Research & DevelopmentRaritan, NJ

relapsed/refractory multiple myeloma (Breakthrough Therapy)




multiple myeloma(see also lung, other)

Phase I/IIww.healthcarepharmaceuticals.com

elotuzumab AbbVieNorth Chicago, ILBristol-Myers SquibbPrinceton, NJ

multiple myeloma (1st-line)(combination therapy), relapsed/refractory multiple myeloma (combination therapy)(Breakthrough Therapy)-----------------------------------------multiple myeloma (2nd-line) (combination therapy)

Phase IIIwww.abbvie.comwww.bms.com-----------------------------------------Phase IIwww.abbvie.comwww.bms.com

� lanesib(KSP inhibitor)

Array BioPharmaBoulder, CO

multiple myeloma Phase IIwww.arraybiopharma.com

FPI-01(multi-peptide immunotherapeutic)

Formula PharmaceuticalsBerwyn, PA

multiple myeloma(see also leukemia)

Phase Iwww.formulapharma.com

GSK2110183(AKT protein kinase inhibitor)


multiple myeloma Phase Iwww.gsk.com



relapsed/refractory multiple myeloma(see also hematological, lymphoma, solid tumors)

Phase IIIwww.millennium.com

Keytruda®


multiple myeloma(see also bladder, breast, head/neck, hematological, kidney, lung, stomach)


Kyprolis®

car� lzomibORPHAN DRUG


multiple myeloma (combination therapy)(see also lung)

Phase IIIwww.onyx.com



Myeloma




multiple myeloma(see also hematological, leukemia)


marizomib intravenousORPHAN DRUG

Triphase AcceleratorSan Diego, CA

multiple myeloma Phase IIwww.triphaseco.com

melphalan intravenous(Captisol-Enabled® melphalan)


multiple myeloma Phase IIwww.sppirx.com

MOR202(anti-CD38 antibody)

CelgeneSummit, NJMorphoSysMartinsried, Germany

multiple myeloma Phase Iwww.celgene.comwww.morphosys.com

NY-ESO-1/LAGE-1-speci� c T-cells AdaptimmuneOxon, United Kingdom

advanced myeloma(see also ovarian, sarcoma, skin)

Phase I/IIwww.adaptimmune.com

panobinostat(HDAC inhibitor)


multiple myeloma application submittedwww.novartis.com


relapsed multiple myeloma(see also colorectal, head/neck, lung, ovarian, pancreatic, skin, solid tumors, other)


plitidepsinORPHAN DRUG

PharmamarMadrid, Spain

relapsed refractory multiple myeloma Phase IIIwww.pharmamar.com

PVX-410(therapeutic cancer vaccine)

OncoPepNorth Andover, MA

smoldering multiple myeloma Phase I/IIwww.oncopep.com

Revlimid®


CelgeneSummit, NJ

multiple myeloma (1st-line)(see also hematological, leukemia, lymphoma)-----------------------------------------multiple myeloma (maintenance)

application submittedwww.celgene.com

-----------------------------------------Phase IIIwww.celgene.com

rocilinostat(HDAC inhibitor)

Acetylon PharmaceuticalsBoston, MA

multiple myeloma Phase I/IIwww.acetylon.com

SAR650984(anti-CD38 naked mAb)


multiple myeloma Phase IIwww.sano� .com

SNS-01(nano-encapsulated gene therapy)ORPHAN DRUG

Sevion TherapeuticsBridgewater, NJ

multiple myeloma(see also lymphoma)

Phase I/IIwww.seviontherapeutics.com



Myeloma


Sylvant®

siltuximabJanssen Research & DevelopmentRaritan, NJ

smoldering multiple myeloma Phase IIwww.janssenrnd.com

TAS-120(FGF/FGFR inhibitor)


multiple myeloma(see also solid tumors)

Phase Iwww.taihooncology.com

TG02(CDK inhibitor)

Tragara PharmaceuticalsCarlsbad, CA

multiple myeloma(see also leukemia)

Phase Iwww.tragarapharma.com

Zolinza®

vorinostatMerckWhitehouse Station, NJ

multiple myeloma (combination therapy)

Phase IIIwww.merck.com

Ovarian Cancer


afuresertib(Akt protein kinase inhibitor)


ovarian cancer(see also myeloma)

Phase IIwww.gsk.com



ovarian cancer(see also lung, lymphoma)


Avastin®

bevacizumabORPHAN DRUG


platinum-resistant relapsed ovarian cancer(see also brain, lung, other)-----------------------------------------metastatic ovarian cancer (1st-line), platinum-sensitive relapsed ovarian cancer

application submittedwww.gene.com

-----------------------------------------Phase IIIwww.gene.com

AZD1775(WEE-1 inhibitor)


ovarian cancer Phase IIwww.astrazeneca.com



ovarian cancer(see also breast, colorectal, lung, skin, stomach)




ovarian cancer(combination therapy)(see also colorectal, hematological)

Phase I/IIwww.tetralogicpharm.com

BNC105(vascular disrupting agent)

BionomicsThebarton, Australia

ovarian cancer(see also kidney)

Phase Iwww.bionomics.com.au

CVac™cancer vaccine MUC-1ORPHAN DRUG

Prima BioMedSydney, Australia

ovarian cancer(Fast Track)

Phase IIwww.primabiomed.com.au



Ovarian Cancer




ovarian cancer(see also bladder, breast, lung, pancreatic)


Cyramza®


ovarian cancer(see also bladder, breast, colorectal, liver, lung, solid tumors, stomach)


demcizumab(DLL4 inhibitor)


ovarian cancer Phase I/IIwww.oncomed.com

DPX-Survivac(peptide cancer vaccine)

ImmunovaccineHalifax, Canada

ovarian cancer Phase I/IIwww.imvaccine.com

EGEN-001(IL2-based immunotherapy)ORPHAN DRUG

EGENHuntsville, AL

ovarian cancer (monotherapy)

-----------------------------------------ovarian cancer (combination therapy)

Phase IIwww.egeninc.com-----------------------------------------Phase Iwww.egeninc.com

elesclomol(HSP70 heat shock protein)


recurrent, persistent ovarian cancer Phase IIwww.syntapharma.com

ENMD-2076(aurora A/angiogenic kinase inhibitor)ORPHAN DRUG


ovarian clear cell carcinoma(see also breast, sarcoma)


etirinotecan pegol (PEGylated irinotecan)ORPHAN DRUG


platinum-resistant ovarian cancer(see also breast, colorectal)

Phase II completedwww.nektar.com


GradalisDallas, TX

high-risk stage IIIc ovarian cancer(see also colorectal, skin, solid tumors)


GALE-301(folate binding proteinimmunotherapy)


ovarian cancer(see also other)

Phase IIwww.galenabiopharma.com



ovarian cancer(see also breast, leukemia, lung)




ovarian cancer(see also lung, skin, other)

Phase IIwww.incyte.com



Ovarian Cancer




ovarian cancer(see also liver, pancreatic)


Karenitecin®

cositecanBioNumerik PharmaceuticalsSan Antonio, TX

advanced ovarian cancer Phase IIIwww.bionumerik.com

lifastuzumab vedotin(anti-NaPi2b antibody drugconjugate)(RG7599)


platinum-resistant ovarian cancer(see also lung)-----------------------------------------platinum-sensitive ovarian cancer

Phase IIwww.gene.com-----------------------------------------Phase Iwww.gene.com



BRCA 1/2 mutation ovarian cancer, high grade ovarian cancer(see also breast, solid tumors)


ME-344(mitochondrial inhibitor)


ovarian cancer(see also lung)

Phase Iwww.meipharma.com

MEK162(MEK inhibitor)

Array BioPharmaBoulder, CONovartis PharmaceuticalsEast Hanover, NJ

low-grade serious ovarian cancer(see also skin, solid tumors)

Phase IIIwww.arraybiopharma.comwww.novartis.com



ovarian cancer(see also breast, lung)


MORAb-003(IgG1 mAb)ORPHAN DRUG


platinum-sensitive ovarian cancer(see also lung)



TESAROWaltham, MA

ovarian cancer(see also breast, sarcoma)

Phase IIIwww.tesarobio.com


ovarian cancer(see also myeloma, sarcoma, skin)




BRCA-mutated platinum-sensitive relapsed ovarian cancer (see also breast, stomach)-----------------------------------------BRCA-mutation ovarian cancer (1st-line)

application submittedwww.astrazeneca.com

-----------------------------------------Phase IIIwww.astrazeneca.com



Ovarian Cancer


oregovomabORPHAN DRUG

Quest PharmaTechEdmonton, Canada

ovarian cancer (1st-line) Phase IIwww.questpharmatech.com

OVax®

autologous tumor cell-based vaccineORPHAN DRUG

AVAX TechnologiesPhiladelphia, PA

relapsed ovarian cancer Phase I/IIwww.avax-tech.com


persistant or recurrent ovarian cancer(see also colorectal, head/neck, lung, myeloma, pancreatic, skin, solid tumors, other)


pimasertib/SAR245509(MEK inihibitor/dual PI3K, mTOR inhibitor)

EMD SeronoRockland, MASano� USBridgewater, NJ

ovarian cancer(see also solid tumors)

Phase IIwww.emdserono.comwww.sano� .com

RG7458(MUC16 antibody drug conjugate)


ovarian cancer(see also pancreatic)

Phase Iwww.gene.com

RG7882(antibody drug conjugate)


ovarian cancer Phase Iwww.gene.com

rucaparib(PARP inhibitor)ORPHAN DRUG


ovarian cancer (see also pancreatic)

Phase IIIwww.clovisoncology.com



ovarian cancer(see also leukemia, liver)


trebananib(recombinant peptide-Fc fusion protein)


ovarian cancer Phase IIIwww.amgen.com

Vargatef™nintedanib




VB-111(vascular targeting system)


ovarian cancer(see also brain, other)

Phase I/IIwww.vblrx.com

vinorelbine liposomal Spectrum PharmaceuticalsHenderson, NV



VS-6063(FAK inhibitor)


ovarian (combination therapy)(see also lung)

Phase IIwww.verastem.com



Ovarian Cancer


VTX-2337(TLR8 agonist immunotherapy)ORPHAN DRUG

VentiRx PharmaceuticalsSeattle, WA

ovarian cancer(see also head/neck)

Phase IIwww.ventirx.com

Yervoy®


ovarian cancer(see also lung, prostate, skin, stomach)

Phase IIwww.bms.com

Yondelis®

trabectedinJanssen Research & DevelopmentRaritan, NJ

relapsed ovarian cancer(see also sarcoma)


Zybrestat®

fosbretabulinORPHAN DRUG

OXiGENESouth San Francisco, CA

ovarian cancer Phase IIwww.oxigene.com

Pancreatic Cancer


177Lu-DOTATATE(somatostatin receptor antagonist)ORPHAN DRUG

Advanced Accelerator ApplicationsNew York, NY

metastatic gastro-entero-pancreatic neuroendocrine tumors

Phase IIIwww.adacap.com

Abraxane®

paclitaxel protein-bound particles for injectable suspension (albumin-bound)ORPHAN DRUG

CelgeneSummit, NJ

pancreatic cancer (adjuvant)(see also breast)


algenpantucel-L(cancer immunotherapy vaccine)ORPHAN DRUG


pancreatic cancer (resected)(Fast Track)-----------------------------------------pancreatic cancer (borderline resectable of locally advanced unresectable)

Phase IIIwww.newlinkgenetics.com-----------------------------------------Phase IIIwww.newlinkgenetics.com

anti-MUC1 AR20.5 mAb (chemotherapy-enhanced immunotherapy)

Quest PharmaTechEdmonton, Canada

pancreatic cancer Phase I/IIwww.questpharmatech.com



metastatic pancreatic cancer (see also bladder, lung, prostate)




pancreatic cancer(see also brain, prostate)

Phase Iwww.advantagene.com



Pancreatic Cancer


CPI-613ORPHAN DRUG

Cornerstone PharmaceuticalsCranbury, NJ

pancreatic cancer Phase IIwww.cornerstonepharma.com

CRS-207(cancer immunotherapy)ORPHAN DRUG

Aduro BioTechBerkeley, CA

pancreatic cancer in combination with GVAX Pancreas(Breakthrough Therapy)(see also lung)

Phase IIwww.adurobiotech.com



pancreatic cancer(see also bladder, breast, lung, ovarian)


ensituximab(neoplasm antigen inhibitor)ORPHAN DRUG

Precision BiologicsDallas, TX

advanced pancreatic cancer(see also colorectal)

Phase IIwww.precision-biologics.com

FG-3019(connective tissue growth factor inhibitor mAb)

FibrogenSan Francisco, CA

advanced pancreatic cancer Phase IIwww.� brogen.com



pancreatic cancer(see also brain, liver)

Phase I/IIwww.lilly.com

GI-4000(mutated Ras cancer vaccine)


resected pancreatic cancer(see also lung)

Phase IIwww.globeimmune.com

glufosfamideORPHAN DRUG

Eleison PharmaceuticalsSt. Petersburg, FL

metastatic pancreatic cancer(2nd-line)(Fast Track)

Phase IIIwww.eleison-pharma.com

Imprime PGG®


advanced pancreatic ductal cancer (see also colorectal, leukemia, lung, lymphoma)

Phase Iwww.biothera.com



pancreatic cancer(see also liver, ovarian)


IRT-102(methionine enkephalin)

TNI BiotechOrlando, FL

pancreatic cancer Phase IIwww.tnibiotech.com



pancreatic cancer(see also breast, colorectal, lung, other)


KD032(Ras antagonist)ORPHAN DRUG


pancreatic cancer(see also colorectal, lung)

Phase IIwww.kadmon.com



Pancreatic Cancer


masitinib AB Science USAShort Hills, NJ

metastatic pancreatic cancer(see also stomach)

Phase IIIwww.ab-science.com

MLN0264(GCC antibody drug conjugate)


advanced pancreatic cancer(see also stomach)


MM-398(irinotecan/nanotherapeutic)


pancreatic cancer (2nd-line)(see also brain, colorectal)


momelotinib(JAK inhibitor)


pancreatic cancer(see also hematological)


necuparanibORPHAN DRUG

Momenta PharmaceuticalsCambridge, MA

pancreatic cancer Phase I/IIwww.momentapharma.com

PCI-27483(FVIIa inhibitor)

PharmacyclicsSunnyvale, CA

pancreatic cancer Phase IIwww.pharmacyclics.com

PEGPH20(pegylated recombinant human hyaluronidase)

Halozyme TherapeuticsSan Diego, CA

metastatic pancreatic cancer(Fast Track)

Phase IIwww.halozyme.com


advanced pancreatic cancer(see also colorectal, head/neck, lung, myeloma, ovarian, skin, solid tumors, other)


pimasertib(MEK inhibitor)


pancreatic cancer(see also skin)


PLX7486(TRK inhibitor)


pancreatic cancer (combination therapy)(see also solid tumors)


PRI-724(CBP/ß-catenin inhibitor)

Prism PharmaTokyo, Japan

pancreatic cancer(see also leukemia, solid tumors)

Phase Iwww.prismbiolab.com

RG7458(MUC16 antibody drug conjugate)


pancreatic cancer(see also ovarian)

Phase Iwww.gene.com

RG7600(mesothelin antibody drug conjugate)


unresectable pancreatic cancer Phase Iwww.gene.com

rucaparib(PARP inhibitor)


BRCA-mutated pancreatic cancer (see also ovarian)




Pancreatic Cancer




advanced pancreatic cancer(see also colorectal, liver, stomach)


Sprycel®dasatinib

Bristol-Myers SquibbPrinceton, NJOtsuka America PharmaceuticalRockville, MD

pancreatic cancer(see also other)

Phase IIwww.bms.comwww.otsuka.com

tarextumab(anti-NOTCH 2/3)


pancreatic cancer(see also lung)

Phase I/IIwww.oncomed.com

TH-302(hypoxia-activated prodrug)ORPHAN DRUG


pancreatic cancer (see also hematological, lung, sarcoma, skin, solid tumors)

Phase IIIwww.emdserono.comwww.thresholdpharm.com

TL-118 Tiltan PharmaJerusalem, Israel

metastatic pancreatic cancer (1st-line)

Phase IIwww.tiltanpharma.com

Y-90 clivatuzumab tetraxetanORPHAN DRUG


relapsed pancreatic cancer(Fast Track)-----------------------------------------pancreatic cancer (1st-line)(Fast Track)

Phase IIIwww.immunomedics.com-----------------------------------------Phase I/IIwww.immunomedics.com

Prostate Cancer


abituzumab(anti-integrin mAb)


prostate cancer(see also colorectal)


AE37(immunotherapeutic)


prostate cancer(see also breast)

Phase IIwww.antigenexpress.com

APC100(androgen antagonist)

Adamis PharmaceuticalsSan Diego, CA

prostate cancer Phase I/IIwww.adamispharmaceuticals.com



prostate cancer(see also bladder, lung, pancreatic)


ATL101(177 Lu-labeled anti-PSMA mAb)

ATLAB PharmaNantes, FranceWeill Medical CollegeNew York, NY

metastatic prostate cancer Phase IIwww.atlab-pharma.com



Prostate Cancer


AZD5312(androgen receptor inhibitor)

AstraZenecaWilmington, DEIsis PharmaceuticalsCarlsbad, CA

prostate cancer Phase Iwww.astrazeneca.comwww.isispharm.com

BIND-014(PSMA-targeted docetaxel)

BIND TherapeuticsCambridge, MA

prostate cancer(see also lung)

Phase IIwww.bindtherapeutics.com

BPX-201(dendritic cell vaccine)

Bellicum PharmaceuticalsHouston, TX

metastatic castration-resistantprostate cancer(combination therapy)

Phase I/IIwww.bellicum.com



newly diagnosed prostate cancer(Fast Track)(see also brain, pancreatic)

Phase IIIwww.advantagene.com

CFG920(steroid 17-alpha-hydroxylase inhibitor)


metastatic castration-resistantprostate cancer


custirsen (OGX-111)(antisense oligonucleotide)

OncoGenex PharmaceuticalsBothell, WATeva PharmaceuticalFrazier, PA

metastatic castration-resistant prostate cancer (2nd-line) (Fast Track)(see also lung)

Phase IIIwww.oncogenex.comwww.tevapharm.com

DCVax®-Prostatedendritic cell vaccine


late-stage hormone independentprostate cancer

Phase II completedwww.nwbio.com

EC1169(PSMA-tubulysin)


prostate cancer Phase Iwww.endocyte.com

galeterone(androgen receptor antagonist)

Tokai PharmaceuticalsCambridge, MA

metastatic castration-resistant prostate cancer (Fast Track)

Phase IIwww.tokaipharma.com

GTx-758(ER alpha agonist)

GTx Memphis, TN

secondary hormone therapy for castration-resistant prostate cancer

Phase IIwww.gtxinc.com

indoximod(IDO pathway inhibitor)


metastatic castration-resistant prostate cancer(see also breast)

Phase IIwww.newlinkgenetics.com

ipatasertib (GDC-0068)(Akt inhibitor)


prostate cancer(see also breast, stomach)

Phase IIwww.arraybiopharma.comwww.gene.com

IRX4204(retinoid X receptor agonist)

Io TherapeuticsSanta Ana, CA

prostate cancer Phase IIwww.io-therapeutics.com



Prostate Cancer


ISIS-EIF4ERX(antisense oligonucleotide)

Isis PharmaceuticalsCarlsbad, CA

castration-resistant prostate cancer(see also lung)

Phase IIwww.isispharm.com

Jevtana®

cabazitaxelSano� USBridgewater, NJ

metastatic prostate cancer Phase IIIwww.sano� .com

JNJ-56021927/ARN-509(androgen receptor antagonist)


hormone refractory prostate cancer Phase IIIwww.janssenrnd.com



metastatic prostate cancer(see also brain, hematological, leukemia, solid tumors)


LFA102(anti-prolactin receptor mAb)

Novartis PharmaceuticalsEast Hanover, NJXOMABerkeley, CA

prolactin receptor-positive castration-resistant prostate cancer(see also breast)

Phase Iwww.novartis.comwww.xoma.com

MER-104(oral acyline)

Merrion PharmaceuticalsDublin, Ireland

prostate cancer Phase Iwww.merrionpharma.com

mipsagargin(ATPase inhibitor)


prostate cancer(see also brain, liver)


MVA-BN-PRO(immunotherapy vaccine)


hormone refractory prostate cancer Phase I/IIwww.bavarian-nordic.com

MVI-816(PAP plasmid DNA)

Madison VaccinesMadison, WI

prostate cancer Phase IIwww.madisonvaccinesinc.com

NX-1207(apoptosis stimulant)

Nymox PharmaceuticalHasbrouck Heights, NJ

early-stage prostate cancer Phase II completedwww.nymox.com

ODM-201(androgen receptor inhibitor)

Bayer HealthCare PharmaceuticalsWhippany, NJOrionEspoo, Finland

prostate cancer Phase IIwww.bayerpharma.comwww.orion.�

ONC1-13B(androgen receptor antagonist)

AllaChemHallandale Beach, FL

metastatic prostate cancer Phase I/IIwww.allachem.com

orteronel(lyase inhibitor)


metastatic castration-resistant prostate cancer-----------------------------------------non-metastatic prostate cancer

Phase IIIwww.millennium.com-----------------------------------------Phase IIwww.millennium.com

ozarelix(GnRH inhibitor)


hormone dependent prostate cancer Phase IIwww.sppirx.com



Prostate Cancer


prostate cancer vaccine(PSA/IL-2/GM-CSF vaccine)

OncBioMuneBaton Rouge, LA

prostate cancer Phase Iwww.oncbiomune.com

Prostvac®

targeted immunotherapyBavarian NordicMountain View, CANational Cancer InstituteBethesda, MA

metastatic castration-resistant prostate cancer

Phase IIIwww.bavarian-nordic.com

Provenge®

sipuleucel-TDendreonSeattle, WA

recurrent early stage prostate cancer

-----------------------------------------recurrent hormone refractory, metastatic prostate cancer

Phase IIIwww.dendreon.com-----------------------------------------Phase IIwww.dendreon.com

PSMA ADC therapeutic(antibody drug conjugate)

Progenics PharmaceuticalsTarrytown, NY

prostate cancer Phase IIwww.progenics.com

RG7450(anti-STEAP1 antibody drug conjugate)


prostate cancer Phase Iwww.gene.com

TAK-385(GnRH angonist)


prostate cancer Phase IIwww.millennium.com

tasquinimod Active BiotechLund, Sweden

metastatic hormone refractoryprostate cancer

Phase IIIwww.activebiotech.com

TeloB-VAX(telomerase vaccine)

Adamis PharmaceuticalsSan Diego, CA

prostate cancer Phase Iwww.adamispharmaceuticals.com

VT-122(cancer immunotherapy)

Vicus TherapeuticsMorristown, NJ

pre-chemotherapy prostate cancer(see also liver)

Phase IIwww.vicustherapeutics.com

Xo� go®

radium-223 dichlorideBayer HealthCare PharmaceuticalsWhippany, NJ

bone metastases in castration-resistant prostate cancer(1st-line) (combination therapy)-----------------------------------------bone metastases in castration-resistent prostate cancer (combination therapy)


-----------------------------------------Phase IIwww.bayerpharma.com

Xtandi®enzalutamide (androgen receptor inhibitor)

Astellas Pharma USNorthbrook, IL MedivationSan Francisco, CA

metastatic castration-resistant prostate cancer (treatment naïve)(see also breast)-----------------------------------------non-metastatic castration-resistant prostate cancer

application submittedwww.astellas.comwww.medivation.com-----------------------------------------Phase IIIwww.astellas.comwww.medivation.com



Prostate Cancer


Yervoy®


prostate cancer (1st-line)(see also lung, ovarian, skin, stomach)


zoptarelin doxorubicin(type II DNA topoisomerase inhibitor)

AEterna ZentarisQuebec, Canada

castration-/taxane-resistant prostate cancer(see also other)

Phase IIwww.aezsinc.com

Sarcoma


aldoxorubicin(delivery-enhanced doxorubicin)ORPHAN DRUG


soft tissue sarcoma (2nd-line)(see also brain, lung)-----------------------------------------Kaposi’s sarcoma, soft tissue sarcoma (1st-line)

Phase IIIwww.cytrx.com-----------------------------------------Phase IIwww.cytrx.com

ENMD-2076(aurora A/angiogenickinase inhibitor)


soft tissue sarcoma(see also breast, ovarian)


Halaven®

eribulinORPHAN DRUG


soft tissue sarcoma(see also breast, lung)


inhaled lipid cisplatin Eleison PharmaceuticalsSt. Petersburg, FL

prevention of pulmonary metastases from osteosarcoma (pediatric)

Phase IIwww.eleison-pharma.com

MORAb-004(CD248 antigen inhibitor)ORPHAN DRUG


soft tissue sarcoma(see also colorectal, skin)



TESAROWaltham, MA

Ewing’s sarcoma(see also breast, ovarian)

Phase Iwww.tesarobio.com


synovial sarcoma(see also myeloma, ovarian, skin)

Phase Iwww.adaptimmune.com

sarcoma vaccine MabVax TherapeuticsSan Diego, CA

metastatic sarcoma Phase IIwww.mabvax.com



soft tissue sarcoma(see also hematological, lung, pancreatic, skin, solid tumors)

Phase IIIwww.emdserono.comwww.thresholdpharm.com



Sarcoma




advanced soft tissue sarcoma(see also brain, kidney)

Phase I/IIwww.traconpharma.com

Yondelis®

trabectedinJanssen Research & DevelopmentRaritan, NJ

soft tissue sarcoma(see also ovarian)


Skin Cancer


ABT-RTA-408 AbbVieNorth Chicago, ILReata PharmaceuticalsIrving, TX

relapsed/refractory melanoma(see also lung)

Phase Iwww.abbvie.comwww.reatapharma.com

ADI-PEG 20(PEG arginine deiminase)ORPHAN DRUG


metastatic melanoma(see also breast, leukemia, liver, lung, lymphoma)

Phase Iwww.polarispharma.com

Ad-RTS-IL-12(interleukin-12 gene therapy)

ZIOPHARM OncologyBoston, MA

malignant melanoma(see also breast)

Phase IIwww.ziopharm.com

AE-M(immunotherapeutic)


melanoma Phase Iwww.antigen-express.com

alpha-gal glycosphingolipid(immunotherapy)

AgalimmuneIrvine, CA

melanoma Phase I completedwww.agalimmune.com



metastatic melanoma(see also bladder, hematological, myeloma)


AT13387(HSP90 inhibitor)


melanoma(see also lung)



advanced melanoma(see also breast, liver, lung, other)




melanoma(see also breast, colorectal, lung, ovarian, stomach)


Cavatak™coxsackievirus A21ORPHAN DRUG

ViralyticsSydney, Australia

malignant melanoma Phase IIwww.viralytics.com



Skin Cancer




advanced melanoma(see also colorectal, solid tumors)

Phase IIwww.tevapharm.com

cobimetinib(MEK inhibitor)


metastatic melanoma(see also solid tumors)


dorgenmeltucel-L NewLink GeneticsAmes, IA

malignant melanoma Phase IIwww.newlinkgenetics.com

E7016(PARP inhibitor)


malignant melanoma Phase IIwww.eisai.com



melanoma(see also brain, liver, lung, other)


Erivedge®

vismodegibGenentechSouth San Francisco, CA

operable basal cell carcinoma(see also leukemia)



GradalisDallas, TX

advanced melanoma(see also colorectal, ovarian, solid tumors)


G100(TLR4 agonist)

Immune DesignSeattle, WA

Merkel cell carcinoma Phase Iwww.immunedesign.com

glembatumumab vedotin(antibody drug conjugate)

CellDex TherapeuticsHampton, NJ

stage III or IV melanoma(see also skin)

Phase IIwww.celldex.com

GM-CT-01(galectin inhibitor)

Galectin TherapeuticsNorcross, GA

melanoma Phase Iwww.galectintherapeutics.com

GR-MD-02(galectin-3 inhibitor)

Galectin TherapeuticsNorcross, GA

melanoma Phase Iwww.galectintherapeutics.com

heat shock protein vaccineORPHAN DRUG

AgenusLexington, MA

melanoma (see also brain)

Phase Iwww.agenusbio.com

IMCgp100(CD3 antigen inhibitor)

ImmunocoreOxon, United Kingdom

malignant melanoma Phase Iwww.immunocore.com



metastatic melanoma(see also lung, ovarian, other)


interleukin-12 gene therapy OncoSec MedicalSan Diego, CA

malignant melanoma(see also lymphoma)

Phase IIwww.oncosec.com



advanced basal cell carcinoma(see also brain, solid tumors)




Skin Cancer




BRAF-mutant melanoma(see also colorectal, solid tumors)


MAGE-A3 immunotherapeutic(recombinant antigen-speci� ccancer immunotherapeutic)


melanoma Phase IIIwww.gsk.com

MEDI4736 + dabrafenib + trametinib (anti-PD-L1 mAb/BRAF inhibitor/MEK inhibitor)


melanoma Phase Iwww.medimmune.com


Array BioPharmaBoulder, CONovartis PharmaceuticalsEast Hanover, NJ

NRAS-mutant melanoma(see also ovarian, solid tumors)

Phase IIIwww.arraybiopharma.comwww.novartis.com

MEK162 and LGX818(MEK inhibitor/BRAF inhibitor)


BRAF-mutant melanoma Phase IIIwww.novartis.com

Mekinist® trametinib +Ta� nlar® dabrafenib combinationORPHAN DRUG


metastatic melanoma (adjuvant) Phase IIIwww.gsk.com

melapuldencel-T(dendritic cell immunotherapy)ORPHAN DRUG

NeoStem OncologyNew York, NY

malignant melanoma(Fast Track)

Phase IIwww.neostem.com

MORAb-004(CD248 antigen inhibitor)


melanoma(see also colorectal, sarcoma)


MSB0010445(NHS-IL2 cancer immunotherapy)


melanoma Phase IIwww.emdserono.com

MSB0010718C(anti-PD-L1 mAb)


Merkel cell carcinoma(see also solid tumors)


MVax®

autologous tumor cell-based vaccine

AVAX technologiesPhiladelphia, PA

melanoma Phase IIIwww.avax-tech.com



melanoma (1st-line),melanoma, (2nd-3rd-line)(see also brain, colorectal, head/neck, hematological, kidney, liver, lung, lymphoma, solid tumors)-----------------------------------------melanoma


-----------------------------------------Phase I/IIwww.bms.com



Skin Cancer




melanoma (1st-line)(see also brain, colorectal, head/neck, hematological, kidney, lung)-----------------------------------------melanoma

Phase II/IIIwww.bms.com



melanoma(see also myeloma, ovarian, sarcoma)


Ontak®denileukin diftitox


melanoma Phase IIwww.eisai.com


metastatic melanoma(see also colorectal, head/neck, lung, myeloma, ovarian, pancreatic, solid tumors, other)


Picato®

ingenol mebutateLEO PharmaParsippany, NJ

basal cell cancer Phase IIwww.leo-pharma.us

pimasertib(MEK inhibitor)


melanoma(see also pancreatic)




melanoma(combination therapy)(see also brain, leukemia)-----------------------------------------melanoma

Phase IIwww.dsi.comwww.plexxikon.com-----------------------------------------Phase Iwww.dsi.comwww.plexxikon.com

POL-103A(immunotherapy vaccine)ORPHAN DRUG

PolynomaSan Diego, CA

late-stage malignant melanoma Phase IIIwww.polynoma.com

PV-10(rose bengal sodium)ORPHAN DRUG

Provectus BiopharmaceuticalsKnoxville, TN

malignant melanoma(see also liver)

Phase IIwww.pvct.com

Resimmune™A-dmDT390-bisFv immunotoxin

AngimmuneRockville, MD

metastatic melanoma(see also lymphoma)

Phase I/IIwww.angimmune.com



malignant melanoma(see also bladder, kidney, lung)

Phase Iwww.gene.com

RG7636(ETBR antibody drug conjugate)


metastatic melanoma Phase Iwww.gene.com



Skin Cancer


talimogene laherparepvec(cancer immunotherapy)


regionally and distantly metastatic melanoma

application submittedwww.amgen.com



advanced melanoma(see also hematological, lung, pancreatic, sarcoma, solid tumors)

Phase IIwww.emdserono.comwww.thresholdpharm.com

TRX518(GITR receptor antagonist)

GITRCambridge, MA

advanced melanoma(see also solid tumors)

Phase Iwww.gitrrx.com

tumor in� ltrating lymphocyte(autologous cell therapy)

Lion BiotechnologiesWoodland Hills, CANational Cancer InstituteBethesda, MD

malignant melanoma (2nd-line)

-----------------------------------------malignant melanoma (1st-line combination therapy)

Phase IIwww.lbio.com-----------------------------------------Phase Iwww.lbio.com

Yervoy®


melanoma (adjuvant), metastatic melanoma (dose optimization)(see also lung, ovarian, prostate, stomach) -----------------------------------------melanoma (adolescents)


-----------------------------------------Phase IIwww.bms.com

Zelboraf®

vemurafenibGenentechSouth San Francisco, CA

BRAF-mutation-positive metastatic melanoma (adjuvant)(see also other)-----------------------------------------BRAF-mutation-positive metastatic melanoma


-----------------------------------------Phase Iwww.gene.com

Solid Tumors


ABBV-399(antibody drug conjugate)



ABC294640(sphingosine kinase-2 inhibitor)

Apogee BiotechnologyHummelstown, PA

advanced solid tumors Phase Iwww.apogee-biotech.com

ABT-414(antibody drug conjugate)


squamous cell solid tumors(see also brain)

Phase IIwww.abbvie.com

ABT-700(c-Met inhibitor)


advanced solid tumors Phase Iwww.abbvie.com



Solid Tumors


ABT-767(PARP inhibitor)



ABT-806(epidermal growth factor receptor [EGFR] mAB)


advanced solid tumors Phase Iwww.abbvie.com

AG-120(IDH1 inhibitor)


(see also hematological) Phase Iwww.agios.comwww.celgene.com

AGS-22ME(antibody drug conjugate)

AgensysSanta Monica, CASeattle GeneticsBothell, WA

Phase Iwww.agensys.comwww.seattlegenetics.com

ALB-109564(a)(tubulin inhibitor)

Bessor-Brightwaters PharmaFramingham, MA

Phase Iwww.bessor-brightwaterspharma.com

ALT-836(recombinant chimeric anti-tissue factor antibody)



altiratinib(MET/TIE2/VEGFR2kinase inhibitor)

Deciphera PharmaceuticalsCambridge, MA

Phase Iwww.deciphera.com

AM0010(PEGylated recombinant human IL-10)

ARMO BioSciencesRedwood City, CA

Phase Iwww.armobio.com

AMG 208(c-Met inhibitor)



AMG 232 AmgenThousand Oaks, CA

(see also leukemia) Phase Iwww.amgen.com



advanced solid tumors(see also stomach, other)


AMG 780(angiogenesis inhibitor)


advanced solid tumors Phase Iwww.amgen.com

AMG 820(c-fms antagonist)





Solid Tumors


AMG 900(aurora kinase inhibitor)


late-stage solid tumors (see also hematological)


anti-LAG3 antibody Bristol-Myers SquibbPrinceton, NJ

(combination therapy) (see also other)

Phase Iwww.bms.com

APS001F(recombinant anaerobic bacteria)

Anaeropharma ScienceTokyo, Japan

Phase Iwww.anaeropharma.co.jp

APTO-253(KLA4 activator)

Aptose BiosciencesToronto, Canada

late-stage solid tumors Phase Iwww.aptose.com

AR-12(PI3K/Akt inhibitor)


(see also lymphoma) Phase Iwww.arnothera.com

AR-42(pan-DAC inhibitor)


(see also hematological) Phase Iwww.arnothera.com

ARQ 087(FGFR antagonist)

ArQuleWoburn, MA

Phase Iwww.arqule.com

ARQ 092(AKT inhibitor)

ArQuleWoburn, MA


ARQ 761(E2F1 transcription factor stimulant)

ArQuleWoburn, MA


ATI-1123(docetaxel liposomal)

Azaya TherapeuticsSan Antonio, TX

Phase Iwww.azayatherapeutics.com

AT13148(ROCK inhibitor)


Phase Iwww.astx.com

AUY922(Hsp90 inhibitor)



AZD2014(mTOR serine/threoninekinase inhibitor)


Phase IIwww.astrazeneca.com

AZD4547(FGFR tyrosine kinase inhibitor)


Phase IIwww.astrazeneca.com

AZD8186(PI3K beta inhibitor)



BAX-69(anti-MIF antibody)

Baxter HealthcareDeer� eld, IL

metastatic solid tumors Phase Iwww.baxter.com



Solid Tumors


BAY949343(mesothelin antibody drug conjugate)


advanced solid tumors Phase Iwww.bayerpharma.com

BAY1129980(C4.4a antibody drug conjugate)


solid tumors with LYPD3 protein expression

Phase Iwww.bayerpharma.com




bendamustine ready-to-diluteliquid formulation


(see also leukemia, lymphoma) Phase Iwww.eagleus.com

BGB-283(BRAF inhibitor)

BeiGeneBeijing, ChinaEMD SeronoRockland, MA

Phase Iwww.beigene.comwww.emdserono.com

BGB-290(tankyrase inhibitor)

BeiGeneBeijing, ChinaEMD SeronoRockland, MA

Phase Iwww.beigene.comwww.emdserono.com



advanced solid tumors (see also brain, hematological, liver)


BI 836845(somatomedin inhibitor)


Phase Iwww.boehringer-ingelheim.com

BI 853520(PTK2 protein inhibitor)





BKM120(PI3K inhibitor)


(see also breast) Phase Iwww.novartis.com

BLY719(PI3K-alpha inhibitor)



BPM 31510 intravenous(ubidecarenone IV)

Berg PharmaFramingham, MA

Phase Iwww.berghealth.com

briciclib(cyclin D1 modulator)

Onconova TherapeuticsNewtown, PA

metastatic solid tumors Phase Iwww.onconova.com

BVD-CNV(C. novyi-NT)

BioMed Valley DiscoveriesKansas City, MO

Phase Iwww.biomed-valley.com



Solid Tumors


CB-839(glutaminase inhibitor)

Calithera BiosciencesSouth San Francisco, CA

advanced solid tumors(see also hematological)

Phase Iwww.calithera.com

CBL0137(curaxin)

Cleveland BioLabsBu� alo, NY

advanced solid tumors Phase Iwww.cbiolabs.com

CC-115(dual TORK/DNA PK inhibitor modulator)

CelgeneSummit, NJ


CC-122(pleiotropic pathway modulator)

CelgeneSummit, NJ

(see also lymphoma) Phase Iwww.celgene.com

CC-223(dual TORK inhibitor)

CelgeneSummit, NJ



(see also hematological) Phase Iwww.celgene.com

CDX-1401(NY-ESO-1 cancer vaccine)


solid tumors expressing NY-ESO-1 protein

Phase Iwww.celldex.com



advanced solid tumors(see also colorectal, skin)

Phase Iwww.tevapharm.com

CEP-37250/KHK2801 Kyowa Hakko Kirin PharmaPrinceton, NJTeva PharmaceuticalFrazier, PA

advanced solid tumors Phase Iwww.kyowa-hakko-kirin.comwww.tevapharm.com

CEP-37440(dual anaplastic lymphoma kinase and PTK2 protein inhibitor)


late-stage solid tumors Phase Iwww.tevapharm.com

CGM097(p53/MDM2-interaction inhibitor)


late-stage solid tumors Phase Iwww.novartis.com

CLR457(PI3K inhibitor)


advanced solid tumors Phase I/IIwww.novartis.com

cobimetinib(MEK inhibitor)


(see also skin) Phase Iwww.gene.com

crolibulin Immune PharmaceuticalsCambridge, MA

Phase IIwww.immunepharmaceuticals.com

CUDC-427(IAP inhibitor)

CurisLexington, MA

late-stage solid tumors(see also lymphoma)




Solid Tumors


Cyramza®


combination therapy(see also bladder, breast, colorectal, liver, lung, ovarian, stomach)


dacomitinib(pan-HER inhibitor)


(see also lung) Phase Iwww.pfizer.com

DCVax®-Directdendritic cell vaccine


inoperable solid tumors Phase I/IIwww.nwbio.com

Debio 1143(IAP inhibitor)

DebiopharmaLausanne, Switzerland

(see also lymphoma) Phase Iwww.debiopharm.com

Debio 1347(FGFR inhibitor)

DebiopharmLausanne, Switzerland

late-stage solid tumors Phase Iwww.debiopharm.com

DFP-11207(thymidylate synthase expression inhibitor)

Delta-Fly PharmaTokushima, Japan

advanced solid tumors Phase I

DLYE-5953A GenentechSouth San Francisco, CA

refractory solid tumors Phase Iwww.gene.com

DS-3032(MDM2 inhibitor)


(see also lymphoma) Phase Iwww.dsi.com

DS-3078(mTOR inhibitor)


(see also lymphoma) Phase Iwww.dsi.com

DS-7423(PI3K/mTOR inhibitor)


Phase Iwww.dsi.com

DS-8273(anti-DR5 antibody)



Phase Iwww.dsi.com

E6201(MEK-1/MEKK-1 kinase inhibitor)


advanced solid tumors Phase Iwww.eisai.com

E7389(liposomal eribulin)


Phase Iwww.eisai.com

EC1456(folate-tubulysin)


Phase Iwww.endocyte.com

enoticumab Regeneron PharmaceuticalsTarrytown, NY

advanced solid malignancies Phase Iwww.regeneron.com



Solid Tumors


entolimod(cancer immunotherapy)

Cleveland BioLabsBu� alo, NY

advanced solid tumors Phase Iwww.cbiolabs.com

EP-100(membrane disrupting peptide)

Esperance PharmaceuticalsSan Francisco, CA

(see also hematological) Phase IIwww.esperancepharma.com

EPZ-6438(EZH2 inhibitor)

EpizymeCambridge, MA

INI1-de� cient solid tumors(see also lymphoma)

Phase Iwww.epizyme.com


GradalisDallas, TX

advanced solid tumors(see also colorectal, ovarian, skin)

Phase Iwww.gradalisinc.com

fenretinide intravenous CerRxLubbock, TX

adult solid tumors, pediatric mixed tumors(see also lymphoma)

Phase IIwww.cerrx.com

G305(GLA-SE+NY-ESO-1 protein)


Phase Iwww.immunedesign.com

GDC-0575(ChK1 inhibitor)


(see also lymphoma) Phase Iwww.gene.com

GDC-0994(ERK inhibitor)


Phase Iwww.gene.com

GI-6301(brachyury peptide vaccine)

GlobeImmuneLousiville, CO

Phase I completedwww.globeimmune.com



(see also head/neck, lung, other) Phase IIwww.genelux.com

GO-203-2c(MUC1 inhibitor)

Genus OncologyVernon Hills, IL

Phase Iwww.genusoncology.com

GS-5745(MMP9 mAb inhibitor)


Phase Iwww.gilead.com

HDM201(tumor suppressor proteinp53 modulator)


(see also hematological) Phase Iwww.novartis.com

I-131-CLR1404(molecular radiotherapeutic)

Cellectar BiosciencesMadison, WI

advanced solid tumors Phase I completedwww.cellectar.com

IMGN289(EGFR kinase inhibitor)


EGFR-positive solid tumors Phase Iwww.immunogen.com



Solid Tumors


IMGN853(folate receptor 1 inhibitor)


folate receptor alpha-positive solid tumors

Phase Iwww.immunogen.com

IMMU-132(antibody drug conjugate)


metastatic solid tumors Phase IIwww.immunomedics.com

INC280(c-Met inhibitor)


late-stage solid tumors Phase IIwww.novartis.com

INCB39110(JAK1 inhibitor)


advanced solid tumors(see also lung)


IT-139(GRP78 protein inhibitor)

IntezyneTampa, FL

Phase Iwww.intezyne.com



(see also hematological, lymphoma, myeloma)


JNJ-42756493(FGFR inhibitor)



Phase Iwww.janssenrnd.com



advanced solid tumors(see also brain, leukemia, prostate, other)

Phase Iwww.karyopharm.com

KTN3379(ErbB3 kinase inhibitor)

Kolltan PharmaceuticalsNew Haven, CT

late-stage solid tumors Phase Iwww.kolltan.com

LB-100(protein phosphatase 2A inhibitor)

Lixte BiotechnologyEast Setauket, NY

Phase Iwww.lixte.com



(see also brain, skin) Phase IIwww.novartis.com

LEE011(CDK4/6 inhibitor)


(see also breast) Phase Iwww.novartis.com



BRAF-mutant tumors (see also colorectal, skin)


LJM716(ERBB-3 receptor antagonist)



lirilumab(anti-KIR mAb)


solid tumors (with nivolumab), solid tumors (with Yervoy® )

Phase Iwww.bms.com



Solid Tumors


LOXO-101(PanTrk inhibitor)

Array BioPharmaBoulder, COLoxo OncologyStamford, CT

Phase Iwww.arraybiopharma.comwww.loxooncology.com

LV305(NY-ESO-1 Zvex)


Phase Iwww.immunedesign.com



advanced solid tumors(see also breast, ovarian)




(see also breast, other) Phase Iwww.macrogenics.com

ME-143(NADH oxidase inhibitor)


refractory solid tumors Phase Iwww.meipharma.com

MEDI-565(anti-CEA BITE mAb)


Phase Iwww.medimmune.com

MEDI0639(anti-DLL-4 mAb)



MEDI0680(anti-PD-1 mAb)



MEDI3617(anti-ANG-2 mAb)



MEDI4736 + MEDI0680(anti-PD-L1 mAb/anti-PD-1 mAb)



MEDI4736 + tremelimumab(anti-PD-L1 mAb/anti-CTLA-4 mAB)



MEDI6469(murine anti-OX40 mAb)

AgonOxPortland, ORMedImmuneGaithersburg, MD

Phase Iwww.agonox.comwww.medimmune.com



(see also ovarian, skin) Phase I/IIwww.novartis.com

MGA271(CD276 protein inhibitor)

MacogenicsRockville, MD

Phase Iwww.macrogenics.com

MGCD516(multi kinase inhibitor)


late-stage solid tumors Phase Iwww.mirati.com



Solid Tumors


MGN 1703(TLR9 agonist)

MologenBerlin, Germany

Phase I completedwww.mologen.com

MINT1526A(integrin-alpha-5/beta-1 inhibitor)


Phase Iwww.gene.com

MK-2206(AKT inhibitor)

MerckWhitehouse Station, NJ

Phase IIwww.merck.com

MK-4166(TNFRSF18 protein stimulant)



MK-8033(c-Met/RON inhibitor)


Phase I completedwww.merck.com

MK-8242(MDM-2 inhibitor)





(see also breast, hematological) Phase Iwww.millennium.com

MLN1117(PI3K alpha inhibitor)



MLN2480(Pan-Raf kinase inhibitor)



MLN4924(Nedd8-activating enzyme inhibitor)


(see also hematological) Phase Iwww.millennium.com

MLN7243(UAE inhibitor)



MM-151(EGFR oligoclonal antibody)


refractory advanced solid tumors Phase Iwww.merrimackpharma.com

MNK-010 Mallinckrodt PharmaceuticalsSt. Louis, MO

Phase Iwww.mallinckrodt.com

MSB0010718C(anti-PD-L1 mAb)


(see also skin) Phase Iwww.emdserono.com

MSC2156119(c-Met kinase inhibitor)


Phase Iwww.emdserono.com

MSC2363318A(p70S6K/Akt inhibitor)





Solid Tumors


MUC-1 dendritic cancer vaccine MicroVAXManassas, VA

Phase I

MVA-BN Brachyury(immunotherapy vaccine)

Bavarian-NordicMountain View, CA

Phase Iwww.bavarian-nordic.com

NC-4016(DACH platin micelle)

NanoCarrierChiba, Japan

Phase Iwww.nanocarrier.co.jp

NHS-IL12(cancer immunotherapy)





solid tumors (signal detection)(see also brain, colorectal, head/neck, hematological, kidney, liver, lung, lymphoma, skin)

Phase Iwww.bms.com

NLG919(immune checkpoint inhibitor)


Phase Iwww.newlinkgenetics.com

OMP-52M51(NOTCH-1 inhibitor)


(see also hematological) Phase Iwww.oncomed.com

ONT-10(cancer immunotherapy)

OncothyreonSeattle, WA

Phase Iwww.oncothyreon.com

OPB-111077 Otsuka PharmaceuticalRockville, MD

Phase Iwww.otsuka.com

oprozomib(proteasome inhibitor)


(see also hematological) Phase Iwww.onyx.com

oraxol(oral paclitaxel)

Kinex PharmaceuticalsBu� alo, NY

Phase Iwww.kinexpharma.com

OTS167(MELK protein inhibitor)

OncoTherapy ScienceKanagawa Prefecture, Japan

Phase Iwww.oncotherapy.co.jp

PB272(neratinib)

Puma BiotechnologyLos Angeles, CA

HER2-mutated solid tumors(see also breast)

Phase IIwww.pumabiotechnology.com

PEG-SN38(angiogenesis inhibitor)


(see also brain, breast) Phase Iwww.belrosepharma.com


relapsed or refractory solid tumors (pediatric)(see also colorectal, head/neck, lung, myeloma, ovarian, pancreatic, skin, other)




Solid Tumors


PFK-158(PFKFB3 inhibitor)

Advanced Cancer TherapeuticsLouisville, KY

Phase Iwww.advancedcancertherapeutics.com

pimasertib/SAR405838(MEK inhibitor/hDM2 inhibitor)


Phase Iwww.emdserono.comwww.sano� .com

pimasertib/SAR245409(MEK inhibitor/dual PI3K, mTOR inhibitor)


(see also ovarian) Phase Iwww.emdserono.comwww.sano� .com

PLX7486(TRK inhibitor)


TRK-driven tumors(see also pancreatic)


PLX8394(BRAF inhibitor)


(see also leukemia) Phase Iwww.dsi.comwww.plexxikon.com

PM060184(tubulin polymerisation inhibitor)

PharmamarMadrid, Spain

Phase Iwww.pharmamar.com

PNT2258(bcl-2 oncogene inhibitor)

ProNAi TherapeuticsPlymouth, MI

(see also lymphoma) Phase IIwww.pronai.com

PRI-724(CBP/β-catenin inhibitor)

PRISM PharmaTokyo, Japan

(see also leukemia, pancreatic) Phase Iwww.prismbiolab.com

PT-112(apoptosis stimulant)

Phosplatin TherapeuticsNew York, NY

late-stage solid tumors Phase Iwww.phosplatin.com

purine nucleoside phosphorylasegene therapy

PNP TherapeuticsBirmingham, AL

Phase Iwww.pnptherapeutics.com

QBI-139(RNA inhibitor)

Quintessence BiosciencesMadison, WI

Phase Iwww.quintbio.com

rebastinib(TRK, BCR-ABL, TIE2 inhibitor)

Deciphera PharmaceuticalsCambridge, MA

Phase I/IIwww.deciphera.com



Solid Tumors


refametinib(MEK inhibitor)


advanced metastatic solid tumors Phase I/IIwww.bayerpharma.com



Phase Iwww.gene.com



(see also hematological) Phase Iwww.gene.com

RG7841(antibody drug conjugate)


Phase Iwww.gene.com



late-stage solid tumors(see also colorectal, lymphoma)

Phase Iwww.radiorx.com

RX-3117(cancer speci� c cytotoxic agent)

RexahnRockville, MD

late-stage solid tumors Phase Iwww.rexahn.com

RX-5902(p68 RNA helicase)

RexahnRockville, MD

late-stage solid tumors Phase Iwww.rexahn.com

RXDX 101(selective tyrosine kinase inhibitor)

IgnytaSan Diego, CA

Phase I/IIwww.ignyta.com

SAR125844(c-Met kinase inhibitor)


Phase Iwww.sano� .com

SAR245408(PI3K inhibitor)


Phase Iwww.sano� .com

SAR260301(PI3K-beta inhibitor)


PTEN-de� cient tumors Phase Iwww.sano� .com

SAR307746(anti-ANG2 mAb)

Regeneron PharmaceuticalsTarrytown, NYSano� USBridgewater, NJ

Phase Iwww.regeneron.comwww.sano� .com

SAR405838(HDM2/p53 antagonist)

Ascenta TherapeuticsMalvern, PASano� USBridgewater, NJ

Phase Iwww.ascenta.comwww.sano� .com

SAR566658(maytansin-loaded anti-CA6 mAb)


Phase Iww.sano� .com



Solid Tumors


sEphB4-HSA(recombinant albumin fusion protein)

VasGene TherapeuticsLos Angeles, CA

Phase I

SF1126(pan-PI3K inhibitor)

SignalRx PharmaceuticalsSan Diego, CA

Phase I completedwww.signalrx.com

SP-02(darinaparsin)

Solasia PharmaTokyo, Japan

(see also hematological) Phase Iwww.solasia.co.jp

Stivarga®


advanced solid tumors(see also colorectal, kidney, liver)

Phase I/IIwww.bayerpharma.com



(see also colorectal, head/neck) Phase Iwww.emdserono.com

TAK-659(SYK kinase inhibitor)


(see also hematological) Phase Iwww.millennium.com

TAK-733(MEK inhibitor)



TAS-114(dUTPase/DPD inhibitor)



TAS-119(aurora kinase A inhibitor)


late-stage solid tumors Phase Iwww.taihooncology.com

TAS-120(FGF/FGFR inhibitor)


advanced solid tumors(see also myeloma)


taselisib(PI3K inhibitor)


(see also breast) Phase Iwww.gene.com

TCN-P(triciribine)

Cahaba PharmaceuticalsPrinceton, NJ

Phase IIwww.cahabapharma.com

TEN-010(BET inhibitor)

Tensha TherapeuticsCambridge, MA

advanced solid tumors Phase Iwww.tenshatherapeutics.com

TEW-7197(TGF-beta type I receptor antagonist)

MedPactoSuwon, South Korea

Phase Iwww.medpacto.com



(see also hematological, lung, pancreatic, sarcoma, skin)

Phase Iwww.emdserono.comwww.thresholdpharm.com



Solid Tumors


thioureidobutyronitrile(tumor suppressor protein p53 stimulant)

CellceutixBeverly, MA

Phase Iwww.cellceutix.com

TKI258(multi-targeted receptor tyrosine kinase inhibitor)

Novartis PhamaceuticalsEast Hanover, NJ


TRX518(GITR receptor antagonist)

GITRCambridge, MA

advanced solid tumors(see also skin)

Phase Iwww.gitrrx.com

TVB-2640(FASN inhibitor)

3-V BiosciencesMenlo Park, CA

advanced solid tumors Phase Iwww.3vbio.com

U3-1565(anti-HB-EGF antibody)


Phase Iwww.dsi.com

urelumab Bristol-Myers SquibbPrinceton, NJ

(see also hematological) Phase Iwww.bms.com

vanticitumab(anti-Fzd7 antibody)


advanced solid tumors Phase Iwww.oncomed.com

varlilumab(CD27 inhibitor)


(see also hematological) Phase Iwww.celldex.com

VGX 100(VEGF-C antibody)

Ceres OncologyVictoria, Australia

Phase Iwww.ceresoncology.com

vintafolide(folate-DAVLBH)


advanced solid tumors(combination therapy)(see also lung)

Phase Iwww.endocyte.com

VS-4718(FAK inhibitor)


late-stage solid tumors Phase Iwww.verastem.com

VS-5584(PI3K/mTOR inhibitor)



Phase Iwww.verastem.com

VX15(CD100 antigen inhibitor)

VaccinexRochester, NY

Phase Iwww.vaccinex.com

VX-970(ATR protein inhibitor)

Vertex PharmaceuticalsBoston, MA

Phase Iwww.vrtx.com

WT2725(cancer immunotherapy)

Sunovion PharmaceuticalsMarlborough, MA

(see also hematological) Phase Iwww.sunovion.com



Solid Tumors


X396(ALK inhibitor)

XcoveryWest Palm Beach, FL

advanced solid tumors Phase Iwww.xcovery.com

X82(VEGFR/PDGFR antagonist)

TyrogenexWest Palm Beach, FL

Phase Iwww.xcovery.com/tyrogenex-inc

Xalkori®crizotinib


(see also lung) Phase Iwww.pfizer.com

Stomach Cancer


A� nitor®


non-functioning gastrointestinal neuroendocrine tumors(see also breast, lymphoma, stomach)




gastric cancer(see also solid tumors, other)


BBI608(cancer stem cell inhibitor)


gastric cancer(see also breast, colorectal, lung, ovarian, skin, other)-----------------------------------------gastrointestinal cancers(combination therapy)


-----------------------------------------Phase Iwww.bostonbiomedical.com

crenolanib(PDGFR inhibitor)

AROG PharmaceuticalsDallas, TX

GIST(see also brain)

Phase IIwww.arogpharma.com

Cyramza®

ramucirumabORPHAN DRUG


gastric cancer (2nd-line combination therapy)(see also bladder, breast, colorectal, liver, lung, ovarian, solid tumors)

application submittedwww.lilly.com

Iclusig®

ponatinibARIAD PharmaceuticalsCambridge, MA

GIST (2nd-line)(see also leukemia)

Phase IIwww.ariad.com

ipatasertib(GDC-0068)(Akt inhibitor)


gastric cancer(see also breast, prostate)

Phase IIwww.arraybiopharma.comwww.gene.com



Stomach Cancer


Kadcyla®

ado-trastuzumab emtansineGenentechSouth San Francisco, CA

advanced HER2-positive gastric cancer(see also breast)


Keytruda®


gastric cancer(see also bladder, breast, head/neck, hematological, kidney, lung, myeloma)


LY2875358(c-Met mAb)


advanced gastric cancer(see also lung)


masitinibORPHAN DRUG

AB Science USAShort Hills, NJ

late-stage GIST (1st-line) (see also pancreatic)

Phase IIIwww.ab-science.com

Minnelide™ 001HSP70 inhibitor

Minneamrita TherapeuticsMoline, IL

advanced gastrointestinal tumors Phase I

MLN0264(GCC antibody drug conjugate)


advanced gastrointestinal cancer(see also pancreatic)


MM-111(ErbB3/ErbB2 receptor antagonist)ORPHAN DRUG


gastric cancer (2nd-line) Phase IIwww.merrimackpharma.com

NeuVax®

nelipepimut-SGalena BiopharmaPortland, OR

gastric cancer(see also breast, kidney, liver, lung, lymphoma, skin, other)

Phase I completedwww.galenabiopharma.com



gastric cancer (2nd-line)(see also breast, stomach)

Phase IIIwww.astrazeneca.com

Perjeta®

pertuzumabRocheNutley, NJ

advanced HER2-positive gastric cancer(see also breast)

Phase IIIwww.roche.com

rilotumumab(hepatocyte growth factor/scatter factor inhibitor)


MET-positive gastric cancer Phase IIIwww.amgen.com

S-1(gimeracil/oteracil/tegafur � xed-dose combination)


gastric cancer Phase IIIwww.taihooncology.com



advanced gastric cancer(see also colorectal, liver, pancreatic)


Somatuline® Depotlanreotide auto-gel formulation

Ipsen BiopharmaceuticalsBasking Ridge, NJ

gastroenteropancreatic neuroendocrine tumors

application submittedwww.ipsenus.com



Stomach Cancer


telatinib(VEGFR inhibitor)ORPHAN DRUG

Eddingpharm Westlake Village, CA

gastric cancer Phase II completedwww.eddingpharm.com


Tekmira PharmaceuticalsBurnaby, Canada

gastrointestinal neuroendocrine tumors(see also liver, other)


Yervoy® ipilimumab


gastric cancer(see also lung, ovarian, prostate, skin)

Phase IIwww.bms.com

Other Cancers


Adcetris®

brentuximab vedotinSeattle GeneticsBothell, WA

CD30-positive non-lymphomamalignancies(see also lymphoma)

Phase IIwww.seattlegenetics.com

ADXS-HPV(live, attenuated Listeria monocytogenes (Lm)-based immunotherapy vaccine)ORPHAN DRUG

AdvaxisPrinceton, NJ

cervical cancer (see also head/neck, other)-----------------------------------------anal cancer

Phase IIwww.advaxis.com-----------------------------------------Phase I/IIwww.advaxis.com

AEB701(PKC inhibitor)


uveal melanoma (combination therapy) (see also lymphoma)




gastroesophageal junction cancer,esophageal cancer(see also stomach, solid tumors)


ATR-101(sterol O-acyltransferase inhibitor)ORPHAN DRUG

AtterocorAnn Arbor, MI

adrenocortical carcinoma Phase Iwww.atterocor.com

Avastin®

bevacizumabGenentechSouth San Francisco, CARocheNutley, NJ

recurrent cervical cancer(see also brain, lung, ovarian)

-----------------------------------------metastatic or locally advanced high-risk neuroendocrine tumor

application submittedwww.gene.comwww.roche.com-----------------------------------------Phase IIIwww.gene.comwww.roche.com



Other Cancers



rectal cancer(see also breast, liver, lung, skin)


BBI608(cancer stem cell inhibitor)


gastroesophageal junction cancer(see also breast, colorectal, lung, ovarian, skin, stomach)


DN24-02(active cellular immunotherapy)

DendreonSeattle, WA

HER2-positive urothelial carcinoma Phase IIwww.dendreon.com



esophageal cancer, gastroesophageal junction cancer, gastroesophageal cancer(see also lung, myeloma)

Phase Iwww.healthcarepharmaceuticals.com



thyroid cancer(see also brain, liver, lung, skin)-----------------------------------------endometrial cancer

Phase III completedwww.eisai.com-----------------------------------------Phase IIwww.eisai.com

GALE-301(folate binding proteinimmunotherapy)


endometrial cancer(see also ovarian)

Phase IIwww.galenabiopharma.com

GI-6207(natural killer cell stimulant)


medullary thyroid cancer Phase IIwww.globeimmune.com



peritoneal carcinomatosis(see also head/neck, lung, solid tumors)

Phase I/IIwww.genelux.com

GSK525762(bromodomain inhibitor)


NUT gene midline carcinoma Phase Iwww.gsk.com



fallopian tube cancer, peritoneal cancer(see also lung, ovarian, skin)


INO-3106(therapeutic vaccine)

Inovio Plymouth Meeting, PA

HPV-6-associated aerodigestivemalignancies


INO-3112(cancer therapeutic vaccine)

Inovio PharmaceuticalsPlymouth Meeting, PA

cervical cancer caused by HPV types 16/18(see also head/neck)




Other Cancers




polycythemia vera(see also breast, colorectal, lung, pancreatic)-----------------------------------------advanced malignancies

Phase IIIwww.incyte.com

-----------------------------------------Phase Iwww.incyte.com



gastroesophageal cancer(see also breast, solid tumors)

Phase IIwww.macrogenics.com

Marqibo®

vinCRIStine sulfate LIPOSOME injection


pediatric cancers(see also leukemia, lymphoma)

Phase I/IIwww.sppirx.com

milciclib(CDK inhibitor)

Nerviano Medical SciencesNerviano, Italy

malignant thymoma Phase IIwww.nervianoms.com

MORAb-066(tissue factor antigen inhibitor)


advanced TF-expressing malignancies Phase Iwww.eisai.com

NTO-1151(ribonuclease inhibitor)

NanotherapeuticsAlachua, FLNational Cancer InstituteBethesda, MD

cervical cancer, vaginal cancer Phase IIwww.nanotherapeutics.com


fallopian tube cancer, primary peritoneal cancer(see also colorectal, head/neck, lung, myeloma, ovarian, pancreatic, skin, solid tumors)


seliciclib(CDK inhibitor)


nasopharyngeal cancer(see also lung)


selumetinib(MEK inhibitor)

Array BioPharmaBoulder, COAstraZenecaWilmington, DE

di� erentiated thyroid cancer, uveal melanoma(see also lung)

Phase IIIwww.arraybiopharma.comwww.astrazeneca.com

SOR-C13(TRPV6 protein inhibitor)

Soricimed BiopharmaSackville, Canada

cancers that overexpress the TRPV6calcium channel

Phase Iwww.soricimed.com

Sprycel®dasatinib

Bristol-Myers SquibbPrinceton, NJOtsuka America PharmaceuticalRockville, MD

pediatric cancer(see also pancreatic)

Phase IIwww.bms.comwww.otsuka.com



Other Cancers


telotristat etiprate(TPH1 inhibitor)ORPHAN DRUG

Lexicon PharmaceuticalsThe Woodlands, TX

malignant carcinoid syndrome(Fast Track)

Phase IIIwww.lexicon-genetics.com

tirapazamine(type II DNA topoisomerase inhibitor)

SRI InternationalMenlo Park, CA

cervical cancer Phase IIIwww.sri.com


Tekmira PharmaceuticalsNurnaby, Canada

adrenocortical carcinoma(see also liver, stomach)


V503(human papillomavirus vaccine 9-valent)


prevention of HPV-related cancers application submittedwww.merck.com

VB-111(vascular targeting system)


thyroid cancer(see also brain, ovarian)

Phase IIwww.vblrx.com

Zelboraf®

vemurafenibGenentechSouth San Francisco, CA

BRAF-mutation-positive papillary thyroid cancer(see also skin)


zoptarelin doxorubicin(type II DNA topoisomerase inhibitor)

AEterna ZentarisQuebec, Canada

endometrial cancer (2nd-line)(see also prostate)

Phase IIIwww.aezsinc.com

Unspecifi ed Cancer


AGS-67E(antibody drug conjugate)

AgensysSanta Monica, CAAstellas PharmaTokyo, Japan

Phase Iwww.agensys.com

AMG 110(recombinant bi-speci� c antibody)



BAY1082439(PI3K inhibitor)



BAY1125976(allosteric AKT1/2 inhibitor)





Unspecifi ed Cancer


BAY1143572(PTEFb inhibitor)



BAY1161909(MPS1 inhibitor)



BAY1163877(Pan-FGFR inhibitor)



BAY1179470(FGFR2 antibody)



BAY2010112(PSMA BiTE antibody)



BVD-523(ERK 1/2 kinase inhibitor)

BioMed Valley DiscoveriesKansas City, MO

Phase I/IIwww.biomed-valley.com

CHML(cytotropic heterogeneous molecular lipids)

Glory PharmaceuticalsVienna, VA

Phase Iwww.anticancerdrug.com

CSF-1R mAb LillyIndianapolis, IN


G1T28-1 (IV)(CDK 4/6 inhibitor)

G1 TherapeuticsResearch Triangle Park, NC

Phase Iwww.g1therapeutics.com

GCS-100(galectin-3 protein inhibitor)

La Jolla PharmaceuticalSan Diego, CA

Phase I completedwww.lajollapharmaceutical.com

GPX-150 Gem PharmaceuticalsBirmingham, AL

Phase Iwww.gempharmaceuticals.com

GSK2256098(focal adhesion kinase inhibitor)


Phase Iwww.gsk.com

GSK2636771(PI3K inhibitor)


Phase Iwww.gsk.com

GSK2816126(EZH2 enzyme inhibitor)


Phase Iwww.gsk.com

GSK2849330(ErbB3 mAb)


Phase Iwww.gsk.com

GSK3052230(FGF ligand trap)


Phase Iwww.gsk.com



Unspecifi ed Cancer


HBI-8000(HDAC inhibitor)

Huya BioscienceSan Diego, CA

Phase Iwww.huyabio.com

high ADCC anticancer mAb Boehringer Ingelheim PharmaceuticalsRidge� eld, CTXencorMonrovia, CA

Phase Iwww.boehringer-ingelheim.comwww.xencor.com

IGF-methotrexate conjugate IGF OncologyBirchwood, MN

Phase Iwww.igfoncology.com

IP-112 InnoPharmaPiscataway, NJ

application submittedwww.innopharmainc.com

LGK974(WNT signaling pathway inhibitor)



LY2228820(p38 MAP kinase inhibitor)



LY2874455(FGF receptor inhibitor)



LY2780301(p70S6/AKT dual inhibitor)



LY2801653(c-Met inhibitor)



LY3009120(pan-Raf inhibitor)



LY3023414(PI3K/mTOR dual inhibitor)



LY3039478(NOTCH inhibitor)

LillyIndianapolis, In


MM-141(IGF-1R/ErbB3 bispeci� ctetravalent antibody)


Phase Iwww.merrimackpharma.com

notch inhibitor Bristol-Myers SquibbPrinceton, NJ

Phase Iwww.bms.com

olaratumab(PDGF-alpha receptor antagonist)


Phase IIww.lilly.com



Unspecifi ed Cancer


OMN54(signal transduction pathway inhibitor)

Omnitura TherapeuticsRedwood Shores, CA

Phase Iwww.omnitura.com

OPB-51602(molecular targeting drug)

Otsuka PharmaceuticalRockville, MD

Phase Iwww.otsuka.com

PBI-05204(triple kinase inhibitor)

Phoenix BiotechnologySan Antonio, TX

Phase Iwww.phoenixbiotechnology.com

PD-0325901(MEK1/2 inhibitor)



PF-03084014(gamma secretase inhibitor)



PF-05082566(CD137 antigen agonist)



PF-06263507(auristatin-based antibody drug conjugate)



PF-06439535(bevacizumab biosimilar)



PF-06463922(dual ALK/ROS1 inhibitor)



PF-06664178 P� zerNew York, NY


PF-06647263(immunoconjugate)



PF-06650808 P� zerNew York, NY


REGN1400(ERBB-3 receptor modulator)

Regeneron PharmaceuticalsTarrytown, NY

Phase Iwww.regeneron.com

rintatolimod Hemispherx PharmaPhiladelphia, PA

adjuvant therapy Phase I/IIwww.hemispherx.net

SNX-5422(HSP90 heat-shock protein inhibitor)

EsanexIndianapolis, IN

Phase I

TGF-BR2 mAb LillyIndianapolis, IN




Unspecifi ed Cancer


trametinib + GSK2141795(MEK1/2 inhibitor + AKT proteinkinase inhibitor)


Phase Iwww.gsk.com

VEGFR-3 mAb LillyIndianapolis, IN



Defi nitionsApplication Submitted—An application for marketing has been submitted by the company to the U.S. Food and Drug Administration (FDA).

Breakthrough Therapy—Upon request by a sponsor, the FDA can grant this designation to expedite the development and review of a drug or biologic intended, alone or in combination with one or more other drugs, to treat a serious or life threatening disease or condition and preliminary clinical evidence indicates that it may demonstrate substan-tial improvement over existing therapies on one or more clinically-signi� cant endpoints, such as substantial treatment e� ects observed early in clinical development. If a drug or biologic is designated as a breakthrough therapy, the FDA will expedite the development and review. With this designation, all Fast Track features convey to the medicine.

Fast Track—Upon request by a sponsor, the FDA can grant this designation to facilitate the development and expedite the review of a drug or biologic to treat a serious condition and � ll an unmet medical need. When considering a biopharmaceutical company’s request for Fast Track designation for an investigational drug or biologic, the FDA evaluates whether it will a� ect factors such as survival, day-to-day functioning, or the likelihood that the disease, if left untreated, will progress from a less severe condition to a more serious one, and whether a condition can be adequately addressed by available therapy. With Fast Track designation, early and frequent communication between the FDA and the biopharmaceutical company is encouraged throughout the entire drug development and review process to help to quickly resolve any questions or issues that arise, potentially leading to an earlier approval and access by patients.

Orphan Designation—Upon request by a sponsor, the FDA can grant special status (“orphan status”) to a drug or biologic to treat a rare disease or condition. In order to receive an orphan designation, a qualifying drug or biologic must be intended for the treatment, diagnosis, or prevention of a rare disease or condition that affects fewer than 200,000 people in the United States, or that affects more than 200,000 persons but is not expected to recover the costs of its development and marketing.

Phase I—Researchers test the investigational drug or biologic in a small group of people, usually between 20 and 100 healthy adult volunteers, to evaluate its initial safety and tolerability profile, determine a safe dosage range, and identify potential side effects.

Phase II—The investigational drug or biologic is given to volunteer patients, usually between 100 and 500, to determine whether it is e� ective, identify an optimal dose, and to further evaluate its short-term safety.

Phase III—The investigational drug or biologic is given to a larger, more diverse patient population, often involving between 1,000 and 5,000 patients (but sometimes many more), to generate statistically signi� cant evidence to con� rm its safety and e� ectiveness. Phase III studies are the longest studies and usually take place in multiple sites around the world.

The content of this report has been obtained through public, government and industry sources, and the Adis “R&D Insight” database based on the latest information. Report current as of September 26, 2014. The medicines in this report include medicines being developed by U.S.-based companies conducting trials in the United States and abroad, PhRMA-member companies conducting trials in the United States and abroad, and foreign companies conducting clinical trials in the United States. The information in this report may not be comprehensive. For more specifi c information about a particular product, contact the individual company directly or go to www.clinicaltrials.gov. The entire series of Medicines in Development is available on PhRMA’s website.

A publication of PhRMA’s Communications & Public Affairs Department (202) 835-3460

www.phrma.org | www.innovation.org | www.pparx.org

Provided as a public service by PhRMA. Founded in 1958 as the Pharmaceutical Manufacturers Association.

Copyright © 2014 by the Pharmaceutical Research and Manufacturers of America. Permission to reprint is awarded if proper credit is given.

Pharmaceutical Research and Manufacturers of America • 950 F Street, NW, Suite 300, Washington, DC 20004


The Drug Discovery, Development and Approval Process

The U.S. system of new drug approvals is perhaps the most rigorous in the world. It takes 10-15 years, on average, for an investigational drug to travel from the lab to U.S. patients, according to the Tufts Center for the Study of Drug Development. Tens of thousands of compounds may be screened early in development, but only one ultimately receives approval. Even medicines that reach clinical trials have only a 16 percent chance of being approved.

On average, it costs a company $1.2 billion, including the cost of failures, to get one new medicine from the laboratory to U.S. patients, according to a 2007 study by the Tufts Center for the Study of Drug Development. More recent studies estimate the cost to be even higher.

Once a new compound has been identifi ed in the laboratory, medicines are usually developed as follows:

Preclinical Testing—A pharmaceutical company conducts laboratory and animal studies to show biological activity of the compound against the targeted disease, and the compound is evaluated for safety.

Investigational New Drug Application (IND)—After completing preclinical testing, a company

fi les an IND with the U.S. Food and Drug Administration (FDA) to begin to test the drug in people. The IND shows results of previous experiments; how, where and by whom the new studies will be conducted; the chemical structure of the compound; how it is thought to work in the body; any toxic effects found in the animal studies; and how the compound is manufactured. All clinical trials must be reviewed and approved by the Institutional Review Board (IRB) where the trials will be conducted. Progress reports on clinical trials must be submitted at least annually to FDA and the IRB.

Clinical Trials, Phase I—Researchers test the drug in a small group of people, usually between 20 and 100 healthy adult volunteers, to evaluate its initial safety and tolerability profi le, determine a safe dosage range, and identify potential side effects.

Clinical Trials, Phase II—The drug is given to volunteer patients, usually between 100 and 500, to see if it is effective, identify an optimal dose, and to further evaluate its short-term safety.

Clinical Trials, Phase III—The drug is given to a larger, more diverse patient population, often involving between 1,000 and 5,000 patients

(but sometimes many more), to generate statistically signifi cant evidence to confi rm its safety and effectiveness. They are the longest studies, and usually take place in multiple sites around the world.

New Drug Application (NDA)/Biologic License Application (BLA)—Following the completion of all three phases of clinical trials, a company analyzes all of the data and fi les an NDA or BLA with FDA if the data successfully demonstrate both safety and effectiveness. The applications contain all of the scientifi c information that the company has gathered. Applications typically run 100,000 pages or more.

Approval—Once FDA approves an NDA or BLA, the new medicine becomes available for physicians to prescribe. A company must continue to submit periodic reports to FDA, including any cases of adverse reactions and appropriate quality-control records. For some medicines, FDA requires additional trials (Phase IV) to evaluate long-term effects.

Discovering and developing safe and effective new medicines is a long, diffi cult, and expensive process. PhRMA member companies invested an estimated $51.1 billion in research and development in 2013.

Developing a new medicine takes an average of 10-15 years. Of the tens of thousands of compounds screened, only one is approved.

The Drug Development and Approval Process

f u l l c o lo r

b l ac k

w h i t e Pharmaceutical Research and Manufacturers of America 950 F Street, NW, Washington, DC 20004

www.phrma.org

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