Q2 2016 Outlook Report - Extract

Q2 2016 OUTLOOK REPORT

2 / April 2016 © Informa UK Ltd 2016 (Unauthorized photocopying prohibited.)

April 2016 / 3© Informa UK Ltd 2016 (Unauthorized photocopying prohibited.)

SummaryOur Early 2016 Outlook Report highlighted several high-impact catalysts. The results of these catalysts can be found on Page 5 of this report. Biomedtracker Likelihood of Approval (LOA) opinions successfully predicted 78% of catalyst outcomes that occurred in Early 2016.

In this report, we cover catalysts from 21 drugs, devices and diagnostics expected to occur in Q2 2016. We have also included a list of Large Impact catalysts in the drug and device/diagnostic areas through Q2 2016 from our sister product, CatalystTracker. The catalyst calendars are provided in Excel by downloading the supplemental material at the top of this page. In addition, each drug’s likelihood of Phase/PDUFA review success and overall Likelihood of Approval (LOA) given their particular phase, drug class, and disease group are provided.

About the AuthorBiomedtracker is an independent research service that offers proprietary clinical assessments and patient-based revenue forecasts of developmental drugs within a comprehensive and intuitive drug information database. Clients from the pharmaceutical, biotech, and investment industries rely on Biomedtracker for its insight on the likelihood of approval, commercial potential, and future data and regulatory catalysts for drugs within the competitive landscape of every important disease and indication. Over the last several years, Biomedtracker has become the leader in providing objective information alongside evidence based clinical assessments and investment research on pipeline drugs worldwide. For more information on getting direct access to Biomedtracker, please email [email protected].

Meddevicetracker is an all new medtech intelligence platform that provides clients with real-time data and analysis on medical devices and diagnostics. From the people behind Biomedtracker, comes an event-driven research service for the medical device and diagnostic marketplace. For access to Meddevicetracker please contact your sales representative or email [email protected].

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DisclaimerCopyright © 2016 Sagient Research

This report is published by Sagient Research (the Publisher). This report contains information from reputable sources and although reasonable efforts have been made to publish accurate information, you assume sole responsibility for the selection, suitability and use of this report and acknowledge that the Publisher makes no warranties (either express or implied) as to, nor accepts liability for, the accuracy or fitness for a particular purpose of the information or advice contained herein. The Publisher wishes to make it clear that any views or opinions expressed in this report by individual authors or contributors are their personal views and opinions and do not necessarily reflect the views/opinions of the Publisher.


ContentsEarly 2016 Outlook Catalyst Results ............................................................................................................6

Drugs ...................................................................................................................................................................8

NurOwn for Amyotrophic Lateral Sclerosis (ALS) (BCLI) .........................................................................8

LEE011 for Breast Cancer (NVS) ....................................................................................................................9

Victoza for Diabetes Mellitus, Type II (NVO) ........................................................................................... 11

Semaglutide for Diabetes Mellitus, Type II (NVO) ................................................................................. 11

Sofosbuvir/Velpatasvir FDC for Hepatitis C (HCV) (Antiviral) (GILD) .................................................. 12

Tivantinib for Hepatocellular (Liver) Cancer (HCC) (ARQL) .................................................................. 13

Austedo for Huntington’s Disease (TEVA) ............................................................................................... 14

ZS-9 for Hyperkalemia (AZN) ...................................................................................................................... 15

Epidiolex for Lennox-Gastaut Syndrome (GW)....................................................................................... 16

Eteplirsen for Muscular Dystrophy (SRPT) ............................................................................................... 17

Rociletinib for Non-Small Cell Lung Cancer (NSCLC) (CLVS) ................................................................. 18

Niraparib for Ovarian Cancer (TSRO) ......................................................................................................... 19

Polyvalent Antigen-KLH Conjugate Vaccine for Ovarian Cancer (MBVX).......................................... 20

Aldoxorubicin for Sarcoma (CYTR) ............................................................................................................ 21

Devices & Diagnostics .................................................................................................................................. 22

Fantom Bioresorable Scaffold for Coronary Artery Disease (REVA) ................................................... 22

DIAM Spinal Stablization System for Disc and Spine Repair (MDT) .................................................... 23

Cartiva SCI for Osteoarthritis (Cartiva) ..................................................................................................... 24

AMPLATZER PFO Occluder for Patent Foramen Ovale Repair (STJ) .................................................... 25

Q2 2016 Large Impact Drug/Device Catalyst Calendar ....................................................................... 26


Large Impact Catalysts from the Biomedtracker Early 2016 Outlook ReportOccurred Date Lead Company Product Market Catalyst Did LOA Predict

Outcome1-Day Perf 1-Week Perf 1-Month Perf

2/10/2016 AbbVie Elagolix Obstetrics/ Gynecology Phase III Solstice - Top-Line Results Yes -2.43% 2.86% 5.14%

3/28/2016 Alder ALD403 Neurology Phase IIb Chronic Migraines - Top-Line Results

Yes 42.67% 43.83% N/A

2/22/2016 Amgen Romosozumab Endocrine Phase III FRAME (PMO) - Top Line Data Yes -1.91% -5.22% 0.33%

3/14/2016 Celator Vyxeos Oncology Phase III 301 - OS Data Yes 484.31% 534.64% N/A

2/9/2016 Celltrion Inflectra Autoimmune/Immunology FDA Advisory Panel Meeting N/A1 N/A N/A N/A

3/17/2016 CymaBay MBX-8025 Cardiovascular Phase II HoFH - Top-Line Results N/A1 -23.53% -19.41% N/A

3/22/2016 Eli Lilly Taltz Autoimmune/Immunology PDUFA for BLA - First Review Yes 1.31% 2.06% N/A

2/16/2016 Flexion Zilretta Rheumatology Phase III - vs. TCA - Topline Data Yes 22.64% -1.70% -21.89%

3/1/2016 Gilead Odefsey Infectious Disease PDUFA for NDA - First Review N/A1 1.78% 1.62% 7.87%

3/29/2016 Newron Zadago Neurology PDUFA For NDA - First Review No N/A N/A N/A

3/30/2016 Opko Rayaldee Endocrine PDUFA for NDA - First Review No -6.14% N/A N/A

3/31/2016 PharmaMar Aplidin Oncology Phase III ADMYRE - Top-Line Results N/A1 N/A N/A N/A

3/24/2016 Portola Betrixaban Hematology Phase III APEX - Top-Line Data N/A1 -6.21% -6.80% N/A

2/26/2016 Roche Gazyva Oncology PDUFA for sBLA - Rituximab-Refractory Yes 0.49% 0.49% -3.35%

1No Previous LOA adjustment


Large Impact Catalysts from the Biomedtracker Early 2016 Outlook ReportOccurred Date Lead Company Product Market Catalyst Did LOA Predict

Outcome1-Day Perf 1-Week Perf 1-Month Perf

2/10/2016 AbbVie Elagolix Obstetrics/ Gynecology Phase III Solstice - Top-Line Results Yes -2.43% 2.86% 5.14%

3/28/2016 Alder ALD403 Neurology Phase IIb Chronic Migraines - Top-Line Results

Yes 42.67% 43.83% N/A

2/22/2016 Amgen Romosozumab Endocrine Phase III FRAME (PMO) - Top Line Data Yes -1.91% -5.22% 0.33%

3/14/2016 Celator Vyxeos Oncology Phase III 301 - OS Data Yes 484.31% 534.64% N/A

2/9/2016 Celltrion Inflectra Autoimmune/Immunology FDA Advisory Panel Meeting N/A1 N/A N/A N/A

3/17/2016 CymaBay MBX-8025 Cardiovascular Phase II HoFH - Top-Line Results N/A1 -23.53% -19.41% N/A

3/22/2016 Eli Lilly Taltz Autoimmune/Immunology PDUFA for BLA - First Review Yes 1.31% 2.06% N/A

2/16/2016 Flexion Zilretta Rheumatology Phase III - vs. TCA - Topline Data Yes 22.64% -1.70% -21.89%

3/1/2016 Gilead Odefsey Infectious Disease PDUFA for NDA - First Review N/A1 1.78% 1.62% 7.87%

3/29/2016 Newron Zadago Neurology PDUFA For NDA - First Review No N/A N/A N/A

3/30/2016 Opko Rayaldee Endocrine PDUFA for NDA - First Review No -6.14% N/A N/A

3/31/2016 PharmaMar Aplidin Oncology Phase III ADMYRE - Top-Line Results N/A1 N/A N/A N/A

3/24/2016 Portola Betrixaban Hematology Phase III APEX - Top-Line Data N/A1 -6.21% -6.80% N/A

2/26/2016 Roche Gazyva Oncology PDUFA for sBLA - Rituximab-Refractory Yes 0.49% 0.49% -3.35%

1No Previous LOA adjustment


NurOwn for Amyotrophic Lateral Sclerosis (ALS) (BCLI)

Drug Company Partner(s) Indication(s) Date Range Expected Catalyst(s)

NurOwn BrainStorm Cell Therapeutics Inc.

Amyotrophic Lateral Sclerosis

4/01/2016-06/30/2016

Phase II BCT-001-US - Topline Results

Phase Disease Group

Drug Class Group/Class Phase Success

Group/Class LOA (PTS)

BMT LOA Opinion

II Neurology Biologic 30.77% 16.67% Above

BrainStorm Cell Therapeutics (BCLI) expects top-line results from their Phase II BCT-001-US study of NurOwn for the treatment of amyotrophic lateral sclerosis (ALS) in the second quarter of 2016. NurOwn is BCLI’s only therapy in clinical development and positive results from this study would have a significant impact on their ability to further expand their development into other CNS disease such as Parkinson’s and Hungtington’s Disease. Currently the only U.S. approved therapy in ALS is riluzole.

The NurOwn technology functions by processing adult human mesenchymal stem cells that are present in the bone marrow and are capable of self-renewal and differentiation into many cell types. The mesenchymal stem cell which secrete neurotrophic factors (MSC-NTF) are then implanted back into the patient.

In context to this data readout, the Company has reported mixed but encouraging results from their single-arm, Phase IIa MSC-NTF-002 clinical study of 12 patients in Israel. For the trial’s secondary endpoint, the study demonstrated that at the six months post-treatment, forced vital capacity declined from -5.1% per months pre-treatment to -1.2% per month post-treatment (p=0.036) while ALS Functioning Rating Score-Revised declined from -1.2 points per month pre-treatment to -0.6 points per month post treatment (p=0.052). Thus, because the Phase II BCT-001-US study also includes ALS Functional Rating Score Scale slopes as its secondary endpoint, topline results at the second quarter of 2016 will further elucidate NurOwn’s efficacy in amyotrophic lateral sclerosis in a larger, placebo-controlled trial consisting of 48 patients.

Drugs

http://www.biomedtracker.com/catalystdetail.cfm?catid=110462


LEE011 for Breast Cancer (NVS)


LEE011 Novartis AG Otsuka Breast Cancer 01/01/2016-6/30/2016

Phase III MONALEESA - Top-Line Results

Phase Disease Group



BMT LOA Opinion

III Oncology NME 42.48% 34.66% Average

LEE011 (ribociclib) is an orally administered cyclin-dependent kinase (CDK) 4/6 inhibitor currently in Phase III development for the treatment of breast cancer. CDKs are involved in cell cycle regulation. Novartis announced that they expect to see top-line Phase III data in the first half of 2016 for LEE011 in breast cancer.

In January 2014, a randomized double-blind, placebo-controlled Phase III study (MONALEESA-2) of LEE011 in combination with letrozole for the treatment of postmenopausal women with hormone receptor positive (HR+), HER2 negative (HER2-), advanced breast cancer who have received no prior therapy for advanced disease was initiated as part of the larger MONALEESA program. This study, which enrolled 667 women by early 2015, is the first of many trials of LEE011 in combination with other agents that Novartis expects to form the bases of several regulatory applications over the coming years.

Novartis expects the first submission for LEE011 to occur in late 2016 or early 2017, with final analysis based on the predefined progression free survival (PFS) data from the MONALEESA-2 trial, provided that the requisite number of events occur no later than early in the third quarter of 2016. We look forward to the initial data from the Phase III MONALEESA-2 study in first line HR+/HER2- postmenopausal advanced breast cancer, as this will likely pave the way for further study and regulatory filings of additional combinations of LEE011 for the treatment of breast cancer.

http://www.biomedtracker.com/CatalystDetail.cfm?CatID=104440


Victoza for Diabetes Mellitus, Type II (NVO)


Victoza Novo Nordisk A/S

Diabetes Mellitus, Type II

06/10/2016-06/14/2016

Phase IIIb – LEADER - Updated Results at ADA

Phase Disease Group



BMT LOA Opinion

Approved Endocrine Non-NME N/A N/A N/A

Semaglutide for Diabetes Mellitus, Type II (NVO)


Semaglutide Novo Nordisk A/S


4/1/2016-6/30/2016

Phase III SUSTAIN 6 - Top-Line Results

Phase Disease Group



BMT LOA Opinion

III Endocrine Non-NME 67.95% 58.56% Above

Semaglutide for Diabetes Mellitus, Type II (NVO)


Semaglutide Novo Nordisk A/S


06/10/2016-6/14/2016

Phase III - Results at ADA

Phase Disease Group



BMT LOA Opinion

III Endocrine Non-NME 67.95% 58.56% Above





VictozaVictoza is Novo Nordisk’s daily injected glucagon-like peptide-1 (GLP-1) analogue, which leads the segment commercially. Interestingly, in March 2016, top-line results from its LEADER trial, which was required by the FDA to demonstrate cardiovascular (CV) safety, showed a statistically significant reduction in major adverse CV events (MACE).

Details were not released, however, but will be presented at the upcoming American Diabetes Association (ADA) conference. It will be important to see how large the reduction in MACE was – especially since the trial was statistically overpowered due to the need for certain safety data – as well as whether anything else could have impacted the results, such as blood pressure reduction.

Officials did say with the top-line release that all three components of the MACE endpoint contributed to the benefit (CV death, myocardial infarction, and stroke). They are hoping this impact on atherosclerotic events will differentiate it from the oral SGLT2 inhibitor Jardiance, which showed a benefit primarily on CV death and heart failure hospitalization. Some have postulated that, in addition to a benefit in heart failure patients, Jardiance’s effect could be more related subclinical left ventricular dysfunction. This can be common in diabetics, though it is not certain what type of dysfunction could be impacted by the drug (eg a large percent is diastolic dysfunction, and another question is whether patients would need to have a degree of dysfunction caused by a myocardial infarction to benefit).

It is not entirely clear how Victoza impacts atherosclerosis, so it will be interesting to see if there is more information at the conference. Officials have touted the potential multiple mechanisms that Victoza has shown preclinically, including an impact on a number of metabolic parameters, inflammation, pro-coagulant activity, and other effects.

SemaglutideSemaglutide is Novo Nordisk’s an injected once-weekly glucagon-like peptide-1 (GLP-1) analogue for type 2 diabetes, intended to meet the challenge from other weekly GLP-1 agonists to Victoza.

Five of the SUSTAIN trials have reported top-line data, with signs (from indirect comparisons) that semaglutide could be more effective than Victoza, possibly without substantially increasing nausea, though more details are needed on other symptoms, such as vomiting. Per a company representative, data from at least one of the trials may be at the upcoming ADA meeting, so it will be useful to get more details on its profile.

SUSTAIN 6’s has a much larger patient population that will yield relatively more long term results. The study involves patients with clinical or subclinical cardiovascular disease and was initiated to demonstrate CV safety for the FDA. Top-line results are expected in the second quarter.


Since semaglutide is fairly similar in structure to Victoza, it will be interesting to see if there are any signs of similar trends for a CV benefit, though officials may not present that much detail unless it shows superiority. A company official acknowledged that SUSTAIN 6 study does not have the statistical power that LEADER did and a larger trial is likely to needed to demonstrate a CV benefit. Based on a simple statistical estimate, BMT estimates it could possibly be significant if the risk reduction is more than around 14%.

Novo Nordisk also has an oral formulation of semaglutide in development, though some doses have shown a somewhat high rate of vomiting.

Sofosbuvir/Velpatasvir FDC for Hepatitis C (HCV) (Antiviral) (GILD)


Sofosbuvir/ Velpatasvir FDC

Gilead Sciences, Inc.

Hepatitis C (HCV) Antiviral

6/28/2016 PDUFA for NDA - First Review

Phase Disease Group



BMT LOA Opinion

NDA Infectious diseases

NME 87.84% 75.00% Above

Sofosbuvir/GS-5816 is a single pill combination of the prodrug Sofosbuvir and the NS5A replication inhibitor GS-5816. Sofosbuvir/Velpatasvir FDC(SOF/VEL) was granted Breakthrough Therapy designation as well as priority review for the New Drug Application submitted October 28, 2015. The U.S. Food and Drug Administration (FDA) has set a target action date under the Prescription Drug User Fee Act (PDUFA) of June 28, 2016 for Gilead’s New Drug Application (NDA) for SOF/VEL for the treatment of chronic genotype 1-6 hepatitis C virus (HCV) infection.

The NDA for SOF/VEL is supported by data from four Phase III ASTRAL trials, which evaluated the fixed-dose combination in hepatitis C genotypes 1-6. Of the 1,035 patients treated with SOF/VEL for 12 weeks in the ASTRAL-1, ASTRAL-2 and ASTRAL-3 studies, 1,015 (98 percent) achieved the primary efficacy endpoint of SVR12. The ASTRAL-4 study randomized 267 patients with decompensated cirrhosis (Child-Pugh class B) to receive 12 weeks of SOF/VEL with or without ribavirin (RBV), or 24 weeks of SOF/VEL. Those who received SOF/VEL plus RBV for 12 weeks achieved an SVR12 rate of 94 percent, while those who received SOF/VEL for 12 weeks and 24 weeks achieved SVR12 rates of 83 percent and 86 percent, respectively. Overall, treatment with SOF/VEL for 12 weeks was well tolerated and resulted in high SVR4 rates in patients with genotype 1-6 HCV infection.



Due to its earlier successes in virtually all patient populations, we anticipate that the U.S. Food and Drug Administration will grant approval to Sofosbuvir/GS-5816 on the first review.

Tivantinib for Hepatocellular (Liver) Cancer (HCC) (ARQL)


Tivantinib ArQule, Inc. Daiichi Sankyo, Kyowa Hakko Kirin

Hepatocellular (Liver) Cancer (HCC)

04/01/2016-05/15/2016

Phase III METIV -HCC - Interim Analysis

Phase Disease Group



BMT LOA Opinion

III Oncology NME 42.11% 34.30% Above

Tivantinib (ARQ 97) is a small molecule, orally available, selective inhibitor of c-Met, a tyrosine kinase receptor for hepatocyte growth factor that is thought to play multiple key roles in human cancer, including cancer cell growth, survival, angiogenesis, invasion and metastasis. Hepatocellular cancer (HCC) is the latest indication in which tivantinib is being developed.

In a Phase II study in unresectable HCC patients who had failed one prior systemic therapy (predominantly sorafenib), MET-diagnostic high cohort patients showed significant improvements in progression free survival (PFS) and overall survival (OS). Median PFS and OS in the tivantinib versus placebo arms were 2.4 versus 1.5 months, and 7.2 versus 3.8 months, respectively.

Given the large unmet need in sorafenib-refractory patients, and the increase in survival in a clearly defined patient population, tivantinib is likely to be approved for Met-high patients in the refractory setting if the Phase II results are confirmed. The Phase III study of tivantinib in a larger Met-high population is thus pivotal in determining whether tivantinib will be approved in HCC, which is one of seven pursued indications. Phase III interim results are expected early in the second quarter of 2016.


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Q2 2016 Outlook Report - Extract

Healthcare