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December 7, 2020

COVID-19 Update…2

Industry Briefs …4

Up and Coming…5

Drug & Device Pipeline News…11Seventy-seven drugs and devices have entered a new trial phase this week.

JobWatch…16

CenterWatchWeekly

By Leslie Ramsey

T here’s no getting around mak-ing careful coverage analysis (CA) determinations. It’s an essential part of what sites need to do to stay compliant on billing practices. However, questions do arise in areas where there is little to no guid-ance. Here is an overview of some of those problem areas and what experts suggest are possible solutions. COVID-19

Under pandemic conditions, guidelines are changing from week to week. Missing an update could result in charging a pa-tient’s insurance carrier for something the sponsor should pay for or vice versa.

“We started with no [COVID-19] guide-lines or very limited guidelines,” says Amanda

Miller, manager of quality and development at WCG PFS Clinical, “and now there are more guidelines available. And whether it was having no guidelines or now where there are guidelines available, they haven’t solved all of the coverage analysis questions.”

Sites should make sure they are using the most recent version of guidelines rather than relying on documents they may have downloaded in previous weeks, Miller says. “Keep checking and checking back again and again,” she says, “because it is changing that fast.”

Making Coverage Analysis Determinations for Outside-the-Box Situations

By Charlie Passut

A chain-of-custody data map should be a central part of every clinical trial site.The map draws a picture that shows the

central data storage location, with links to the data sources on one side and backup storage options on the other. For example, the center of the map could be the site’s electronic case report form (eCRF) system, with arrows to the left pointing to nightly transmittal systems, direct entry, manual transcription and randomization coding, and arrows to the right pointing to backup sys-tems hosted at the CRO or sponsor, remote reviews by IRBs, an electronic trial master file (eTMF) system hosted by the sponsor and other archives.

“I tell my clients it’s really in their best interest to create this type of chain-of-custody map for your data in a clinical trial because that gives you control over what’s available,” says John Avellanet, founder of Cerulean Associates. “I strongly suggest you be able to create this before you make any submission, and ideally before even a trial gets started. You should create this as part of your trial planning to show the data is under control,” Avellanet told at-tendees at a WCG CenterWatch workshop last week.

Clinical trial investigators can take the map and make their own notes on it, Avel-lanet said. This is vastly better than them going off and creating their own maps. “At

Chain-of-Custody Maps a Valuable Visual Aid for Illustrating Data Flow and Integrity to Regulators

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COVID-19 Update

COVID-19 Drug Research Roundup

COVID-19 Vaccines:After preliminary trial data showed As-

traZeneca’s one-and-a-half-dose COVID-19 vaccine candidate produced 90 percent effi-cacy, the British drugmaker announced it will initiate a new global clinical trial to confirm its efficacy data in a one-and-a-half-dose regimen. The company has said that some participants in its initial phase 3 vaccine trial in the UK and Brazil only received half doses instead of full doses in the first injection, which warrants the additional investigation. AstraZeneca expects it may receive results of its U.S. vaccine trial in late January, which the company would then use to file for an Emergency Use Authorization (EUA).

The Medicines and Healthcare products Regulatory Agency in the UK is conducting a rolling review of the latest efficacy and safety data on Pfizer/BioNTech’s COV-ID-19 vaccine. The FDA could issue an EUA for the vaccine in just a few weeks, with initial doses being deployed to vulnerable populations as soon as Dec. 11. While the Pfizer/BioNTech COVID-19 vaccine can-didate demonstrated 95 percent efficacy in its late-stage trial, temperature-related storage requirements pose significant logistical challenges in its distribution.

BioNTech recently joined forces with Fosun Pharma to begin a phase 2 trial of its mRNA COVID-19 vaccine BNT162b2 in China per an agreement signed by the two companies in March. The trial will evaluate the safety and immunogenicity of the vac-cine in 960 healthy participants between the ages of 18 and 85.

Russia’s Sputnik V COVID-19 vaccine, which has drawn criticism and controver-sary since it landed on the scene earlier this year, is reportedly 95 percent effective. This is according to Russian scientists who have commented on interim data from a study of 22,000 people. Russia says it will sell the vaccine, developed by Gamaleya Research

Institute of Epidemiology and Microbiol-ogy, for less than $10 per dose. In a recent updated analysis of a phase 3 trial, the Rus-sian Direct Investment Fund announced that the Sputnik V vaccine has an efficacy of 91.4 percent, contradicting reports of 95 percent efficacy. The new data were obtained from a second interim analysis of the trial, which showed eight confirmed cases of COVID-19 in the vaccine group vs. 31 cases in the placebo group.

The Ministry of Food and Drug Safety in South Korea has approved trials studying different COVID-19 vaccine and treatment candidates. The clinical trials approved to start in South Korea include a phase 1 trial of vaccine candidate NBP 2001 from SK Biosci-ence and a phase 2 trial of drug candidate DW2008 from DongWha Pharm.

Novavax has said it will start a phase 3 trial of NVX-CoV2373, the company’s COVID-19 vaccine, across more than 100 trial sites in the U.S. and Mexico. The company also announced that its late-stage trial of 15,000 participants in the UK is now fully enrolled. Interim data from this trial are expected as early as the first quarter of 2021. The data from the UK trial are expected to provide a basis for licensure application in the UK, in addition to the European Union and other neighboring countries. Also, Novavax has fully enrolled its South Africa phase 2b trial of NVX-CoV2373. The study includes 4,422 volunteers, including 245 medically stable HIV-positive participants.

Moderna has been a leading contender in the global race to receive the first EUA for

a COVID-19 vaccine. On Nov. 30, the compa-ny submitted an EUA request to the FDA for its COVID-19 vaccine candidate mRNA-1273. This follows promising findings from Mod-erna’s late-stage clinical trial showing the vaccine was 95 percent effective at prevent-ing infection. The company plans to test the vaccine in up to 3,000 children between the ages of 12 and 17, but no announcements have been made as to when the study will begin recruiting participants.

An investigational vaccine developed by scientists at the City of Hope generated strong protective immunity against SARS-CoV-2, the virus responsible for COVID-19, in a preclinical trial. In the study, researchers found the vaccine produced neutralizing antibodies that prevented the virus from in-fecting healthy cells. The vaccine is currently undergoing further investigation in a phase 1 trial at City of Hope.

COVID-19 Therapies:A large clinical trial in Africa organized

by the 26-member ANTICOV coalition will investigate several treatments for mild COVID-19 across 13 countries. In the adap-tive platform trial, investigators will study treatments currently approved for malaria, HIV, hepatitis C, parasitic infections and different cancers.

Synairgen and Parexel Biotech are collaborating on a phase 3 trial study-ing SNG001, an inhaled formulation of an interferon-beta therapy, as a potential treatment for hospitalized patients with CO-VID-19. The randomized controlled trial will

continues on next page »

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COVID-19 Update (continued from page 2)

enroll 900 patients across 20 countries and will evaluate whether SNG001 accelerates recovery from severe COVID-19 compared with placebo.

A phase 3 clinical trial led by the Mumbai Hospital shows that Glenmark Pharma-ceuticals’ antiviral drug favipiravir prevents replication of SARS-CoV-2 in patients with mild-to-moderate COVID-19. The 150-pa-tient study also found that treatment with favipiravir delayed the need for supportive oxygen therapy in these patients. Currently, favipiravir is used in China and India on an emergency basis for COVID-19, and Russia and some countries in Eastern Europe have approved the treatment for the disease. Health Canada and the FDA have approved Canadian firm Appili Therapeutics’ request to conduct a phase 3 trial investigating the use of favipiravir tablets as a post-exposure prophylaxis therapy for COVID-19.

Another study suggests convalescent plasma offers little to no clinical or mortal-ity benefit for patients with COVID-19 and severe pneumonia. The trial, which was con-ducted at the Hospital Italiano de Buenos Aires in Argentina, found that there was no difference between patients who received convalescent plasma therapy vs. those who received placebo in terms of improving pneumonia or reducing the risk of death.

Allero Therapeutics and Abbreos are jointly working to develop anti-inflamma-tory drugs for patients with COVID-19 who are at risk of developing acute respiratory distress syndrome. Allero’s OroMucosal ImmunoGlobulin technology will be evalu-ated in clinical trials in Miami and Flanders, Belgium, in 2021.

A University of Oxford study titled the Platform Randomized Trial of Interven-tions against Covid-19 In Older People (PRINCIPLE) trial is currently investigating the efficacy of inhaled corticosteroids in treating COVID-19 in people over the age of 50. In addition to inhaled budesonide, other therapies being studied in this trial include doxycycline and azithromycin. So far, the PRINCIPLE trial has enrolled more than 2,100 people across the UK.

The University of New Mexico has enrolled a patient in a clinical trial investigat-ing the safety and tolerability of SBI-101, a novel investigational therapy developed for acute breathing and kidney problems, in the treatment of patients with COVID-19 and acute kidney injury. The University of Mexico is currently listed as the sole trial site for this early-stage phase 1/2 study.

Takeda’s colchicine, an inexpensive anti-inflammatory drug used for gout and other inflammatory conditions, is now

being studied as a potential COVID-19 therapy in the UK’s RECOVERY trial. Investi-gators believe the drug could treat severe COVID-19 in patients requiring mechani-cal ventilation. The trial will investigate colchicine’s impact on length of hospital stay, supplemental oxygen requirements and mortality at 28 days.

Amgen, Takeda and UCB have enrolled their first patient in the COMMUNITY trial, which is investigating the use of three drugs for reducing the severity of COVID-19 in hospitalized patients. The organizations, all of which are members of the COVID R&D Alliance, will first test Amgen’s PDE4 inhibi-tor apremilast followed by Takeda’s plasma kallikrein inhibitor lanadelumab and UCB’s investigational C5 inhibitor zilucoplan.

The National Institute of Allergy and Infectious Diseases has started enrolling hospitalized patients with COVID-19 in its fourth iteration of the Adaptive COVID-19 Treatment Trial (ACTT-4). The ACTT-4 is ex-pected to enroll up to 1,500 patients across 100 sites in the U.S. and other countries. Patients will be randomly assigned to either a dexamethasone and remdesivir combina-tion or a remdesivir and baricitinib dual approach. Researchers will look to identify which combination strategy prevents dis-ease progression.

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Industry Briefs

FDA Finalizes Requirements for Drug Development Tools Qualification ProcessThe FDA yesterday issued a final guidance outlining the process for sponsors and researchers to submit plans for new drug development tools (DDT) to the agency for approval.

The agency’s DDT qualification program reviews biomarker, clinical outcomes assess-ment and animal model tools to be used in clinical trials. If accepted — or “qualified” — the tools can be used by the developer or other researchers in future trials without seeking prior FDA approval.

The guidance is virtually unchanged from the draft issued in December 2019, which divides the qualification process into three stages (CenterWatch Weekly, Dec. 15, 2019). The applicant first submits a letter of intent that describes the scientific rationale for the tool. If after a three-month review, the FDA accepts the letter, the applicant submits a qualification plan that includes all basic in-formation about the tool, which the agency also will take three months to review. If invited by the agency, the applicant submits a full qualification package that includes detailed descriptions of all studies, analyses and results requested by the FDA. The final review stage takes 10 months.

Examples of FDA-qualified DDTs and definitions are listed in the Biomarkers, Endpoints and other Tools (BEST) glossary, created and regularly updated by the FDA-NIH Biomarker Working Group.

Read the final guidance here: https://bit.l y/3gkb33V.

View the BEST glossary at https://bit.ly/2 LevRyk.

Reaction of Investigational Drugs to Acid-Reducing Agents Should Be StudiedSponsors of drug trials should determine what effect the use of acid-reducing agents (ARA), such as antacids, proton pump inhibi-

tors and histamine blockers, could have on the solubility of an orally administered drug, according to a new FDA draft guidance released Monday.

Because ARAs are widely available over the counter, the FDA says, sponsors need to know if their use could decrease the efficacy or increase the risk of adverse events of an investigational drug. The guidance offers recommendations for determining when drug interaction studies are indicated, the design of such trials, how to interpret study results and how to communicate them in product labeling.

Standalone studies using healthy sub-jects could be conducted, and crossover trial designs are preferred, the guidance says. Results of studies using one ARA can be ex-trapolated to other ARAs of the same type.

Comments on the draft guidance are due Feb. 28.

Read the guidance here: https://bit.ly/3g urf2N.

NIAID Restructures HIV Research NetworksThe National Institute of Allergy and Infec-tious Diseases (NIAID) has chosen four clini-cal trial networks to lead HIV research over the next seven years.

NIAID selected the HIV Vaccine Trials Net-work, HIV Prevention Trials Network, AIDS Clinical Trials Group and the International Maternal Pediatric Adolescent AIDS Clinical Trials Network. The networks will work together to direct, coordinate and conduct NIH-funded global clinical research, as well as collaborating with NIAID and industry and nongovernment research organizations.

The institute also has awarded grants to 35 U.S. and international institutions selected as HIV clinical trial units (CTU). Each CTU will support up to eight clinical research sites and the CTUs will collectively support 101 clinical research sites in 18 countries across North America, South America, Africa and Asia.

NIAID and co-funding NIH institutes will provide up to $375.3 million in the first year to support the HIV networks.

WCG Acquires Trial Communications and Training PlatformWCG Clinical has acquired the technology-enabled trial solutions company Trifecta Clinical, the company announced last week.

Trifecta brings with it the Investigator-Space Clinical Qualifications Management System for communicating about and track-ing investigator and rater training.

The company’s other services include live and web-based investigator meetings, safety letter distribution, site startup document exchange and automated study closeout.

“This acquisition is part of our effort to pursue strategic opportunities that allow us to expand and enhance our offerings in anticipation of our customers’ needs,” said Donald Deieso, Ph.D., executive chairman and CEO of WCG.

The company will be renamed WCG Tri-fecta and will retain its leadership structure and locations in Los Angeles, Indianapolis, Philadelphia and Osaka, Japan.

Majority of ICR Scientists Say Pandemic is Making Trial Enrollment ImpossibleSixty percent of respondents to an Institute of Cancer Research, London (ICR) survey said the pandemic has made it impossible to enroll patients in their trials.

Almost half of the 239 survey respon-dents said the pandemic in particular im-pacted their ability to access clinical samples needed for trials and lab experiments.

Approximately 36 percent of respon-dents to the survey said the pandemic had a “moderate” impact on their research, and an additional 36 percent of scientists said CO-VID-19 had a “substantial” impact. About 5 percent said the pandemic had an “extreme” impact on their work.

https://www.centerwatch.com/articles/24489-industry-briefshttps://bit.ly/3gkb33Vhttps://bit.ly/3gkb33Vhttps://bit.ly/2LevRykhttps://bit.ly/2LevRykhttps://bit.ly/3gurf2Nhttps://bit.ly/3gurf2N


This feature highlights changes in clinical trial organizations’ personnel.

4D Molecular Therapeutics4D Molecular Therapeutics has announced several key personnel appointments, including Robert Fishman to the role of chief medical officer and therapeutic area head of pulmonology, Raphael Schiffmann to senior vice president and therapeutic area head of cardiology, and Robert Kim to senior vice president and clinical therapeutic area head of ophthal-mology. Previously, Fishman served as chief medical officer at Xoc Pharmaceuti-cals, Schiffmann served as director of the Institute of Metabolic Disease at the Baylor Research Institute and Kim served as chief medical officer at Viewpoint Therapeutics.

Actinium PharmaceuticalsActinium Pharmaceuticals has appointed Mary Mei Chen vice president of clinical development. Prior to joining Actinium, Chen was senior medical director of medical oncology at GlycoMimetics.

AdageneAdagene has appointed Hua Gong chief operating officer and head of precision medicine. Gong most recently held the position of senior director of the genomics biomarker department at Novartis Navigate Biopharma. Adagene has also named JC Xu as chief scientific officer and Qinghai Zhao as chief manufacturing officer. Xu most recently served as head of China strategy for research and early development at Celgene, and Zhao was vice president of technical development and manufacturing at Forty Seven prior to joining Adagene.

Allena PharmaceuticalsDavid Clark has been named chief medical officer of Allena Pharmaceuticals. In his most recent role, Clark was chief medical officer of Aldeyra Therapeutics.

Antios Therapeutics Gregory Mayes, former CEO of Engage Therapeutics, has been named CEO of Antios Therapeutics.

ApotexApotex has appointed Peter Hardwick as the company’s new CEO. Previously, Hardwick was the company’s chief com-mercial officer.

Arena Pharmaceuticals Arena Pharmaceuticals has named Paul Streck as senior vice president of clinical de-velopment and chief medical officer. Streck most recently served as chief medical officer at Alder Biopharmaceuticals.

Cosmo PharmaceuticalsCosmo Pharmaceuticals has found its new-est chief medical officer in H. Jurgen Lenz, a gastroenterologist who currently serves as chief of the division of gastroenterology at Scripps Memorial Hospital.

Dragonfly TherapeuticsAlex Lugovskoy has joined Dragonfly Therapeutics in the role of chief operating officer. Prior to this appointment, Lugov-skoy was chief development officer of Morphic Therapeutic.

Elevar Therapeutics Elevar Therapeutics has hired Gus Aromin to take on the role of vice president of regu-latory affairs. Aromin most recently served as senior director of regulatory affairs at Avanir Pharmaceuticals.

Entera BioSpiros Jamas, founding CEO of AoBiome, has been appointed CEO of Entera Bio.

FibroGenFibroGen has appointed Mark Eisner chief medical officer of the company. Eisner previously served as senior vice president

and global head of product development immunology, infectious disease and oph-thalmology at Genentech.

Gossamer BioFaheem Haisnan is set to rejoin Gossamer Bio as CEO just two years after he left the company. Haisnan was previously the CEO of Receptos and currently serves on the board of Aspen Neuroscience.

HumacyteLaura Niklason, founder of Humacyte, has been appointed president and CEO of the company. Niklason was most recently vice chair of anesthesiology at Yale University.

IntraBio Jim Meyers, a former executive vice presi-dent and chief commercial officer at Gilead Sciences, has been appointed president and CEO of IntraBio.

Inversago PharmaGlenn Crater has been appointed chief medical officer of Inversago Pharma. Most recently, Crater was vice president of respira-tory portfolio at Theravance Biopharma.

Mereo BioPharmaSuba Krishnan has been named senior vice president of clinical development of Mereo BioPharma. Most recently, Krishnan was the global program head of immuno-oncology at Genmab.

Mersana Therapeutics Mersana Therapeutics has named Arvin Yang senior vice president and chief medical officer. Prior to joining Mersana, Yang was vice president and head of clinical hematol-ogy at Bristol Myers Squibb.

NeuBase Therapeutics Curt Bradshaw has been appointed chief scientific officer of NeuBase Therapeutics.


Up and Coming




Up and Coming (continued from page 5)

Bradshaw’s most recent role was as chief sci-entific officer at Arrowhead Pharmaceuticals.

PainReform Ilan Hadar has been chosen to lead PainReform as the company’s newest CEO. Most recently, Hadar was the country manager Israel and chief financial officer at Foamix Pharmaceuticals.

Penrose TherapeuTx Penrose TherapeuTx has found its new-est CEO in Mark de Souza, former CEO of Revolution Global.

PureIMS PureIMS has named Bram van Dijck as its new CEO. Van Dijck most recently served as global head portfolio management at the Grünenthal Group.

Revive TherapeuticsJoel Moody has joined Revive Therapeu-tics to assist the company in expanding its clinical studies in Canada. Moody previously served as a medical and clinical adviser for clinical trials, including research in oncology and infectious disease.

SamumedDarrin Beaupre has been hired as Sa-mumed’s new chief medical officer of oncol-

ogy. Most recently, Beaupre was a senior vice president of Pfizer.

Summit Therapeutics Mahkam Zanganeh, former CEO of Maky Zanganeh and Associates, has been appointed chief operating officer of Summit Therapeutics.

Tarus TherapeuticsTarus Therapeutics has brought on Brian Schwartz as acting chief medical officer. Schwartz previously served as chief medical officer for arQule.

TrialbeeMatt Walz has taken the helm of Trialbee as its new CEO. Most recently, Walz was the general manager of life sciences at Aurea Software. Trialbee also recently named Lollo Eriksson as the company’s chief strategy officer. Eriksson has served as the company’s CEO over the past three years.

TYME TechnologiesEmerging biotechnology company TYME Technologies has announced the appointment of Richie Cunningham to CEO. Cunningham was most recently president and CEO of Icagen.

Venatorx Pharmaceuticals Venatorx Pharmaceuticals has named Robert Waltermire as senior vice president

of chemistry, manufacturing and controls. Waltermire joins Venatorx from Palatin Technologies, where he served as senior vice president of product development.

VistaGen TherapeuticsLouis Montiy has been appointed vice president of translational medicine at VistaGen Therapeutics. Montiy previously served as president and CEO of Pherin Pharmaceuticals.

Vividion TherapeuticsJeffrey Hatfield has been named CEO of Vividion Therapeutics. Hatfield was formerly the CEO of Vitae Pharmaceuticals.

WugenRyan Sullivan has been appointed head of Wugen’s natural killer-cell research target-ing its leukemias and lymphomas program. Sullivan joins Wugen from the Novartis Institutes for Biomedical Research, where he was a principal scientist and investigator.

Zai Lab Alan Sandler has been named president and head of global development for oncology at Zai Lab. Previously, Sandler served as vice president and global head of lung and head/neck cancer franchise at Genentech.

By John W Mitchell

Frustration about clinical trial start-up costs is not unlike Sisyphus’ dilemma. A host of long-standing expense and such uphill headwinds for the clinical trial sector. According to some sources, such problems even threaten the viability of the sector. Sites complain they incur more over-head costs driven by regulatory documen-tation, antiquated data collection and the demands of precision medicine to name a few — all without increased compensation from sponsors and CROs. A recent study supports this concern. Researchers at the

-opment found that the study start-up phase

remained unchanged for the past decade. -

ing investments in technology are getting trials done faster. None of this bodes well for smaller, independent players.

“Sites are doing more work for [fewer] patients. We used to enroll about eight pa-

-sley, CEO at IACT Health. His company

-ger, and there are more procedures per pa-tient per day. So, you’re doing loads more work, but you’re only paid when you put

themselves — you put fewer patients in tri-als today.”

According to Kingsley, the only way to -

pate in three times as many concurrent tri-

from an overhead standpoint to have one trial that places eight patients, rather than three trials that place eight patients.

Also, at a time when technology is con-

technology is compounding problems in the clinical trial sector. In each of the three studies he cites above, it’s likely that three

sponsors. When each sponsor uses their own networks and devices for patient-re-ported outcomes, it adds to site workload and time.

“We have no ability to standardize tech-nology,” Kingsley says “We have to do so many trials with so many sponsors, and they have their own decision-making…

-bia (standardization between sponsors and sites).”

As an example, he cites the advantages of sites adopting electronic platforms such as eSource. Using such a platform could save billions compared to the aggregate cost of

documents. Clinically, an electronic plat-form also prevents errors such as entering a blood pressure incorrectly or performing a patient procedure out of order from the test protocol.

“We’re trying to convince the industry

Start-up Costs Can Be an Uphill Slog in Need of Change

Status Quo is Not a Viable Option: A Host of Challenges Strain Clinical Trial Site Viability

September 2018 A CenterWatch Publication Volume 25, Issue 09

By John W Mitchell

C roblem gish payments, contract viability, preferred site status, interconnectivity and something less tangible — a lack of consideration in the

-ry care sites in Canada.

see Start-up Costs on page 6

In this issue3-4 In Review

Regulatory Update

5 Action Items

Part 4: How to Identify Candidate Fraudulence Early in The Recruitment CycleBy Angela Roberts

Top Considerations for Using Digital Health Devices in Clinical TrialsBy Vicki Gashwiler

Risk-based Quality Management: The New NormalBy Brion Regan

10-11 Pipeline NewsFDA Actions

The CenterWatch MonthlyISSN 1556-3367

Editorial DirectorProduction

© 2018 CenterWatch, LLC. All rights reserved.No part of this publication may be distributed orreproduced in any form or by any means without the express written consent of the publisher. Permission requests can be obtained via fax at (617) 948-5101 or emailed at [email protected]. Single-user annual subscriptions start at $399.

For inquiries on multi-reader and corporate

InReview

Rare, Adrenal Gland Tumors

drug Azedra for rare cancers of the adre-

therapy OK’d for these tumors. -

apy drug that attacks tumors with a high,

with inoperable locally advanced or metas-tic cancers called phenochromocytoma and paraganglioma.

Pheochromocytoma forms inside and paraganglioma grows outside the adrenal gland(s).

Both tumor types release hormones that can cause symptoms including high blood pressure, rapid heartbeat and anxiety.

University of Pennsylvania research-

percent who received at least one dose and

license is held by Progenics Pharmaceuticals.

Japan Greenlights Parkinson’s Trial

scientists have won approval from Japanese regulators to test adult stem cells as a possible treatment for Parkinson’s disease.

Induced pluriopotent stem cells (iPS) are derived from skin or blood cells and induced back into an embryonic-like plu-ripotent state that can divide into more stem cells or become any type of cell in the body, leading to a potentially unlimited source of any type of human cell needed

-ered promising for regenerative research

-man cells and, also, avoid controversy sur-rounding stem cells from human embryos.

Researchers plan to transplant iPS cells into the brains of Parkinson’s patients in the hope they will help repair or replace

Regulatory Update

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Features

that point, you have no insight into what’s going into the map and the flow.”

The map focuses attention on the data flow within a trial and keeps data-handling consistent, he said, which is key to main-taining data integrity, “because if you don’t know this information, you can’t be in control of this information by default.”

There have been too many data integrity breaches in recent years, says Steve Niedel-man, lead quality systems and compliance consultant at King & Spalding. The FDA has taken numerous enforcement actions based on data integrity issues, Niedelman told Cen-terWatch Weekly. “Having this chain of custody provides greater assurance” to a regulator.

Avellanet said FDA inspectors — called “investigators” by the agency — are trained to look for certain red flags within the data chain of custody. Chief among these is any contract a site has with a vendor that doesn’t address data integrity. Agency investigators also will spot sites that assume their vendors’ subcon-tractors have the same procedures and proto-cols for compliance with the FDA’s electronic systems regulations in 21 CFR Part 11.

The FDA also will not accept sites that say they cannot validate a computerized data system because it’s cloud-based or is pro-vided by a vendor. The FDA also expects sites to have a cross-functional vendor selection and evaluation process as well as consistently audit or otherwise monitor their suppliers.

Sites should challenge their data management vendors’ control by “jumping into the system at different points to assure that the data is being maintained and is accurate,” Niedelman says. “They need to be able to demonstrate that at some point they challenged the system to ensure that.”

On the vendor side, IT hosting and software vendors are also subject to being flagged if they don’t currently have any customers regulated by the FDA or other regulatory agencies, or if their security does

not meet minimum requirements, such as ISO 27001, an international standard for information security management. Vendors that have periodic backup of network failures that go unnoticed are also subject to being flagged by the FDA, as are vendors that don’t have any long-term data corruption avoid-ance plans, don’t test the security of their systems or have inexperienced technicians.

The FDA, according to Avellanet, “is very well aware” that cloud computing carries more risks, and that the agency’s investigators also know sponsors “do not understand this concept of risk-based data integrity controls.” He called the situation “deeply troubling.”

“It is imperative for you to understand that it isn’t wrong moving your data to cloud providers. All it does is engenders greater risk. You will be the one that suffers, not Amazon.”

Avellanet added that ransomware attacks are a growing issue in the clinical trials space because the return on investment is huge. But even if the ransom is paid and criminals produce a decryption key, “there’s perma-nent damage to one out of every eight files that it encrypts, because they are using brute force encryption. So there’s always going to be data loss.”

“This is why one of the things FDA is starting to do is to ask about your ransom-ware protections. Because they’re under the understanding at this point that if you used a clinical site that got swept up in a ransom-ware attack, they’re just going to exclude the data. This is why it is imperative to get ahead of this.”

Susan Schniepp, distinguished fellow at Regulatory Compliance Associates, said sites and vendors alike need to pay close atten-tion to cybersecurity, especially if they work in the cloud. “You can’t just assume that if it’s in the cloud, it’s safe and secure,” Schniepp told CenterWatch Weekly. “You have to take measures to prevent against infiltration or somebody hacking into your system to get the data.”

Schniepp says some clinical trial sites are “probably not in tune with the cybersecurity as much as they should be. Some of them are little clinics, little sites, they’re not the Mayo Clinic or Mount Sinai. I think there’s a niche segment of the industry where cybersecurity would be a concern and could potentially be easier to hack.” Schniepp says she doubts small sites are actually able to afford putting a ton of money into cybersecurity. “They’re probably just taking the basic steps.”

According to Avellanet, sites should initial-ly request several records from their regulated suppliers, chief among them: a data integrity compliance plan that shows progress to date. Such plans are required if the supplier is a contract lab or a contract manufacturer, he said. “Nobody does this in 30 days. Most firms take three years to put this all into place. If they don’t have a plan, you’ve got a problem.” He also said suppliers should provide a list of computerized data system validations per-formed since their last audit, as well as a list of data integrity SOPs and policies they employ. Those could include good documentation practices; computerized system validations; change control and management; records re-tention and archiving; computerized system security; and backups and disaster recovery.

“These are things that you want to ask, because these are things that the FDA and other regulatory agencies look at during the PAI [pre-approval inspection] program.”

Avellanet said that before the pandemic, sites would typically “throw everybody in a room who might have insight into anything along this path, not just the clinical trial managers,” and spend a day mapping the entire process out on a whiteboard. “It’s basi-cally recreating a huge part of the PAI from a system standpoint. If the investigators can-not put together some semblance of this on their own, then it really begs the question of whether or not your trial is being conducted under a state of control. That all leans more toward data rejection, and we all know the path that goes down.”

Chain-of-Custody(continued from page 1)



Features

There is still very little information about coverage for post-hospitalization treatment, Miller says. “We’ve been waiting for more information to become available because as more time has passed there have been more patients discharged from the hospital [and] there doesn’t seem to be kind of a standard guideline or frequency available yet,” she says.

Repeated COVID-19 testing is another area lacking guidance. “Scientifically, it makes sense why they’re doing this, why it’s important to monitor the viral load, but at this point in time, there are not guide-lines that support repeat testing,” she says. “And testing [guidelines] may vary at the local level as well, so watch out for those.”

Miller advises documenting the version of guidelines used in the CA. “By the time you finish negotiating the budget, a new set of guidelines could be available at that point, so you want to make sure it’s clear what version was being used.”

Sponsors also are supporting sites by adjusting protocols to accommo-date changes to the CA guidelines. “The sponsors are delivering a lot of it into the protocol design and schedules, and on the site side, sites are starting to recognize changes to the way that they see patients currently or new requirements they might not have had prior to COVID-19,” she adds, especially as they move operations off-site.

“The biggest change we’ve seen is tele-health being incorporated into trials and being allowed by sponsors,” she says. For-tunately, Medicare has updated guidelines to cover telehealth visits for all beneficia-ries at the same rates as in-person visits, she adds. Sites should be sure to include the new telehealth codes in their claims.

Sites also should keep their billing de-terminations as consistent as possible with the trial’s original CA if patients already are enrolled. Even if guidelines have changed,

new participants shouldn’t be billed differ-ently. “It can create kind of a compliance headache,” Miller says.

And COVID-19 is having an impact on nonCOVID trials, forcing sites to use new methods, in many cases without clear guide-lines. “Whether it’s through amendments or new studies opening up, we’re seeing sites and sponsors take COVID-19 into account when they’re reviewing and opening new studies,” she says.Keeping Up with CAR-T Cell Rules

New types of research can lead to coverage questions, especially in oncology, where CAR-T cell therapy is blurring the line between research and standard of care.

Sites and researchers are still learn-ing, and guidelines, rules, etc., are being frequently updated, Miller says. Sponsors, too, are adapting to the new information.

Hospitalization of CAR-T study partici-pants has become increasingly acceptable as a billable item. Today, most guidelines state that hospitalization is recommended for many of these patients.

Miller says some sponsors don’t require hospitalization, but many sites have adopt-ed this as their standard with CAR-T therapy. It’s important to be aware of the difference. If a site hospitalizes a patient even when the protocol doesn’t mandate it for all patients, it should document the rationale for the decision and factor it into the CA.

Post-treatment testing follows a com-pletely different pattern than most oncology studies, which use cycles and repetitive testing. In CAR-T, most protocols are the one-time treatment followed by a period of intense monitoring and frequent testing.

Some frequent testing is justified by Medicare guidelines, and post-treatment testing falls under conventional care and/or monitoring of complications. However, that

does not mean every post-treatment test in the protocol is billable to insurance.

Prescribing information on package inserts for FDA-approved products include some monitoring recommendations. Some may recommend an initial test, but not subsequent testing. Such testing may hap-pen in a hospital setting, so some of those considerations may come into play.

According to Miller, many sites are comfortable billing more frequently than for other oncology treatments due to the intensity of the therapy.

One aspect of CAR-T studies is fairly straightforward. Most sites consider T-cell harvesting, or leukapheresis, to be research because it is used to develop the study prod-uct. And sponsors don’t usually push back on this, Miller says. In fact, many offer to pay for it at the start.Phase 1 Oncology

Even when there are guidelines readily available, there still are gray areas. Making coverage determinations for phase 1 studies in particular can prove complicated.

Protocols include various tests to monitor the patient for potential side effects and complications. But in phase 1 trials, there’s limited or no product-risk data available, so that eliminates the NCD 310.1 “prevention of complications” umbrella that would poten-tially support billing insurance.

The question becomes, should you bill a patient for such monitoring when there is limited side effect data for phase 1 studies? Many sites choose to only bill for tests and procedures included in the clinical practice guidelines. Some sites reference back to other internal standard practices related to the indication that have been developed for use outside of clinical trials. For example, routine labs during chemo infusion visits

Making Coverage Analysis(continued from page 1)


Hospitalization of CAR-T study participants has become increasingly

acceptable as a billable item.

Another area of complication in phase 1 is oncology trials that study

multiple tumor types.



Features

may be standard for certain patients.Another area of complication in phase 1

is oncology trials that study multiple tumor types. When a trial only focuses on one type of cancer, you can focus analysis on one set of clinical guidelines. When working across tumor types in one study, you may have to consider several different guidelines. This becomes even more complicated as clinical recommendations across cancer types are not always consistent. Is it reasonable to check all sets of guidelines? That may not be possible, because some protocols don’t define the types of cancer that will be studied. They simply specify any solid tumor expressing a particular biomarker.

How do sites manage this complex mix-and-match of recommendations? Ap-proaches vary. Some sites will want to ana-lyze based on each disease. Most, however, will want it to fit into one billing grid, in one pattern. They want to keep it simple; they want to come to a standard determination for billing consistency.

One option is to categorize it all as sponsor-paid research. Many sites would conclude that, because there’s no standard recommendation for all cancer types enroll-ing, they’ll just make it research and ask the sponsor to pay for it. But it’s difficult to justify billing for a CT scan if it’s not recom-mended in central nervous system cancers, for example.

Some phase 1 studies will require a hospitalization for the first dose of the study product, or for the first two or three doses. Technically, you could say this falls under “prevention of complications” in NCD 310.1, Miller says. You’re monitoring participants closely to catch any serious reaction quickly. After all, outside of research, most patients aren’t hospitalized for the first dose of a drug.

When deciding whether to bill for a hospital stay, she says sites should consider whether there is any indication in the guide-

lines that patients would be hospitalized, absent the trial. Sometimes in blood cancer studies, they are. But in many phase 1 stud-ies, most sites aren’t comfortable billing that hospitalization because, outside of research, that’s just not something that would hap-pen, she says.Making Determinations on Off-Label Drug Use

Many protocols include off-label medica-tions, but who pays for them? Medicare will cover an off-label drug on a case-by-case basis under the following conditions:

} There is support in a compendium;} There is support in a peer-reviewed

journal (for anti-cancer medica-tions, the journal must be on an approved list);

} Use of the medication is an accepted standard of medical practice (non-anti-cancer medications only).

Medicare provides multiple options for off-label use, but in many cases, there may not be support for all the drugs in a particu-lar trial. A good way to start researching off-label medications is to check the protocol for journal citations.

Before researching off-label drug resources, Miller says, it’s important to know the following about the protocol:

} Inclusion criteria details;} Whether the drug is being given in

combination with other medications; sometimes, even the order in which the medications are administered makes a difference;

} Why the drug is being given: to treat the underlying condition; as a pre-medication with one of the other study medications; in preparation for medi-cal intervention (e.g., conditioning therapy in a CAR-T or stem cell study).

It can be difficult to identify resources to make billing determinations for unlabeled use of a drug, says Miller, who recommends several tactics. Start by finding out if the study department considers the drug to be standard of care and usually bills the drug outside of research. The study team may have the documentation needed.

Another approach is to ask the sponsor for more details about how it determined the medication was standard of care and what documentation it has beyond what is in the protocol, but be sure to review any documentation provided by the sponsor and confirm it meets guidelines for billing Medicare or private insurance.

It’s also possible to ask the sponsor if it will provide or reimburse for the medication. If you are struggling to find documented support for billing an off-label drug, Miller says, that may be a good indication that it is not medically accepted under Medicare guidelines. In these cases, pushing the sponsor to cover the cost of the drug may be the best option. Coverage Analysis for Nonqualifying Trials

Medicare’s guidelines in NCD 310.1 outline the criteria to determine which trials qualify for coverage of routine costs. “One of the common questions we hear,” Miller says, “is what should we do if a trial does not meet the qualifying criteria?”

Some sites choose to take a relatively conservative approach and decide they will not bill for any items or services required on a nonqualifying trial. This approach, however, presents operational and consistency issues when faced with studies where there is no external fund-ing or when the sponsor refuses to pay for these items. Sites that choose this ap-proach are often faced with the dilemma of either turning down studies or opening studies that don’t have sufficient funding to cover costs.



Medicare provides multiple options for off-label use, but in many cases, there may not be support for all

the drugs in a particular trial.



Features

“More commonly,” Miller says, “we see institutions determine that they may bill Medicare for items and services that would be covered outside the context of a clinical trial.” For example, if a patient with lung can-cer is receiving regular CT scans, an institu-tion following this approach may continue to bill Medicare for those CT scans during a nonqualifying trial as long as the protocol does not require scans more frequently than the patient’s standard care dictates.

The underlying argument is that this patient would have received the scans regardless of enrolling in a clinical trial, Miller says, and there is no regulation lim-iting coverage just because it is also being done as part of a trial.

She notes that, under this approach, a site cannot bill Medicare for any items and services that are only covered based on NCD 310.1. Coverage for services, such as the IV administration of an investiga-tional drug, are only supported by NCD 310.1 and would not normally be covered outside a qualifying clinical trial. There-fore, you would not have any supporting justification to bill Medicare for the IV administration of an investigational item in a nonqualifying clinical trial.

The same logic applies for tests done to monitor potential complications of the study drug. This is expanded coverage

supported only through NCD 310.1. Un-less those tests would have been done as part of the patient’s standard of care, they are typically not billable to insurance if required by a nonqualifying clinical trial. Negotiation Strategies for Coverage Analysis Decisions

Once CA is complete, sites may still need to convince sponsors of the logic behind their determinations. If the CA de-termines something falls under research, Miller asks, how do you address this with the sponsor or CRO that likely thinks most of the items fall under standard of care?

First, she advises, if the CA has discovered no support for billing these items as standard of care, a site should acknowledge them when it sends the CA to the sponsor. Otherwise, she says, you’re waiting for the sponsor to come back and ask, “Why didn’t you make everything research in this study? Why did you switch all of our determinations?”

Sites also should share their information, Miller says. “Don’t simply say, ‘We think ev-erything is research.’ That’s not going to help them. Say something like, ‘We recognize that you thought everything was standard of care. When we did our analysis of this, we found, based on the following reasons, we didn’t feel comfortable billing these and we would like you to pay for them.’”

Miller also offers suggestions for how to approach a sponsor with some spe-cific items for which the site would like to “flip” the sponsor’s coverage determination:

} If the only documented side effects of the investigational drug are from preclinical studies in animals, let the sponsor know the site doesn’t bill patients for monitoring unless there are human side effects.

} If medical necessity can’t be docu-mented for all protocol-required im-aging, it is important to call this out and document in discussions with the sponsor so that unsupported imaging is included in the budget.

} Many phase 1 studies require frequent testing, sometimes more frequent than allowed in cover-age guidelines. A site should know what frequency it is comfortable billing and let the sponsor/CRO know this.

} If it’s a study enrolling multiple solid tumor types with imaging, a site can say it is not confident it will have documentation for every pa-tient and doesn’t feel comfortable billing in that case.

Because flipping determinations can increase the budget considerably, the mat-ter may have to be escalated to a higher sponsor authority, especially if the site is negotiating a budget with a CRO. The more information you have, Miller says, the better everyone along the line will understand why you made the determina-tions you did.


There is no regulation limiting coverage just because it is also being

done as part of a trial.

Because flipping determinations can increase the budget considerably, the mat-

ter may have to be escalated to a higher sponsor authority, especially if the site is

negotiating a budget with a CRO.

LEARN MORE: www.centerwatch.com/sfdagcpi [email protected] 617.948.5100 Order today!

All the resources you need to pass your next inspection with flying colors.This report brings together all the guidances, statistics and instructions you need to successfully get through an FDA inspection. It also explains the inspection process under the FDA’s Bioresearch Monitoring Program (BIMO), which sets out a precise routine for each kind of inspection.

Surviving an FDA GCP Inspection Resources for Investigators, Sponsors, CROs and IRBs




Drug & Device Pipeline News

Company Drug/Device Medical Condition Status Sponsor ContactCOVID-19 Trials and ActionsRhizen Pharmaceutical

RP7214 treatment of SARS-CoV-2 infection

IND approved by the FDA rhizen.com

City of Hope COH04S1 SARS-CoV-2 vaccine initiation of phase 1 trial [email protected] NeoImmuneTech NT-I7 (efineptakin alfa) adult patients with mild

COVID-19first patient dosed in phase 1 trial

neoimmunetech.com

Capricor Therapeutics

intravenous infusion of CAP-1002

patients with SARS-CoV-2 who require supplemental oxygen

first patients dosed in phase 2 trial

capricor.com

Clear Creek Bio brequinar nonhospitalized patients with SARS-CoV-2 who are symptomatic

first patient dosed in phase 2 trial

clearcreekbio.com

Edesa Biotech EB05 hospitalized COVID-19 patients first patient enrolled in phase 2/3 trial

edesabiotech.com

Algernon Pharmaceuticals

NP-120 (Ifenprodil) hospitalized patients with confirmed COVID-19

final patient enrolled in phase 2b/3 trial

algernonpharmaceuticals.com

Apilli Therapeutics Avigan tablets (favipiravir)

post-exposure prophylaxis for COVID-19

initiation of phase 3 trial appilitherapeutics.com

Apilli Therapeutics Avigan tablets (favipiravir)

patients with mild-to-moderate COVID-19 symptoms

initiation of phase 3 trial appilitherapeutics.com

ARCA Biopharma AB201 patients hospitalized with COVID-19

Fast Track designation granted by the FDA

arcabio.com

Mesoblast remestemcel-L acute respiratory distress syndrome (ARDS) due to COVID-19 infection

Fast Track designation granted by the FDA

mesoblast.com

Eli Lilly

Incyte

Olumiant (baricitinib) plus remdesivir

hospitalized patients diagnosed with COVID-19 who require supplemental oxygen or ventilation

Emergency Use Authorization (EUA) granted by the FDA

lilly.com

incyte.com

Pfizer

BioNTech

COVID-19 mRNA vaccine (BNT162b2)

COVID-19 vaccine EUA granted in the UK pfizer.com

BioNTech.deRegeneron casirivimab and

imdevimabmild-to-moderate COVID-19 in adults, as well as in pediatric patients at least 12 years of age and weighing at least 88 pounds, who have received positive results of direct SARS-CoV-2 viral testing and are at high risk for progressing to severe COVID-19 and/or hospitalization

EUA granted by the FDA regeneron.com

Other Trials and ActionsF-star Therapeutics FS222 cancer clinical trial approved in

Spainf-star.com

Orchard Therapeutics

OTL-200 metachromatic leukodystrophy IND approved by the FDA orchard-tx.com

GEMoaB UniCAR-T-PSMA advanced solid tumors initiation of phase 1a trial gemoab.com NeuroTherapia NTRX-07 Alzheimer's Disease completion of phase 1a

trialneurotherapia.com


http://rhizen.com/http://rhizen.com/http://rhizen.com/mailto:covidvaccine%40coh.org?subject=mailto:covidvaccine%40coh.org?subject=http://neoimmunetech.com/http://neoimmunetech.com/https://capricor.com/https://capricor.com/https://capricor.com/https://www.clearcreekbio.com/https://www.clearcreekbio.com/https://www.edesabiotech.com/https://www.edesabiotech.com/https://algernonpharmaceuticals.com/https://algernonpharmaceuticals.com/https://algernonpharmaceuticals.com/https://www.appilitherapeutics.com/https://www.appilitherapeutics.com/https://www.appilitherapeutics.com/https://www.appilitherapeutics.com/https://arcabio.com/https://arcabio.com/http://mesoblast.com/http://mesoblast.com/https://www.lilly.com/https://www.incyte.com/https://www.lilly.com/https://www.incyte.com/https://www.pfizer.com/https://biontech.de/https://www.pfizer.com/https://biontech.de/https://www.regeneron.com/https://www.regeneron.com/https://www.f-star.com/https://www.f-star.com/https://www.orchard-tx.com/https://www.orchard-tx.com/https://www.orchard-tx.com/https://www.gemoab.com/https://www.gemoab.com/https://www.neurotherapia.com/https://www.neurotherapia.com/



Drug & Device Pipeline News (continued from page 11)

Company Drug/Device Medical Condition Status Sponsor ContactAmerican Cryostem ATCell Expanded

Autologous, Adipose-Derived Mesenchymal Stem Cells

post-concussion syndrome in retired military and athletes

initiation of phase 1 trial americancryostem.com

Bio-Path Holdings BP1002 refractory/relapsed lymphoma and chronic lymphocytic leukemia


biopathholdings.com

Codagenix CodaVax-RSV vaccine prevention of respiratory syncytial virus

patient dosing complete in phase 1 trial

codagenix.com

F-star Therapeutics FS120 advanced cancer first patient dosed in phase 1 trial

f-star.com

IGC Pharma IGC-AD1 mild-to-severe dementia due to Alzheimer’s disease

initiation of phase 1 trial igcpharma.com

Junshi Biosciences JS108 advanced solid tumors first patient dosed in phase 1 trial

junshipharma.com

MapLight Therapeutics

ML-004 social deficit in autism spectrum disorder

patient dosing complete in phase 1 trial

maplightrx.com

Nordic Nanovector Betalutin (177Lu lilotomab satetraxetan)

patients with relapsed/refractory diffuse large B-cell lymphoma not eligible for autologous stem cell transplantation

patient enrollment complete in phase 1 trial

nordicnanovector.com

North Sea Therapeutics

SEFA-1024 hypertriglyceridemia first patient dosed in phase 1 trial

northseatherapeutics.com

QPex Biopharma QPX7728 drug resistant bacterial infections

initiation of phase 1 trial qpexbio.com

Silicon Therapeutics SNX281 advanced solid tumors or lymphoma


silicontx.com

Phoenix Molecular Designs

PMD-026 triple negative breast cancer first patients dosed in phase 1b trial

phoenixmd.ca

Ascentage Pharma APG-2575 relapsed/refractory chronic lymphocytic leukemia/small lymphocytic lymphoma

first patient dosed in phase 1/2 trial in Europe

ascentagepharma.com

Inovio INO-3107 recurrent respirator papillomatosis

first patient dosed in phase 1/2 trial

inovio.com

PMV Pharmaceuticals

PC14586 advanced solid tumors with the specific p53 Y220C variant

first patient dosed in phase 1/2 trial

pmvpharma.com

RegenxBio RGX-111 mucopolysaccharidosis Type I first patient dosed in phase 1/2 trial

regenxbio.com

Sio Gene Therapies AXO-AAV-GM1 Type I (infantile) and Type II (late infantile and juvenile onset) GM1 gangliosidosis

first patient dosed in high-dose cohort of phase 1/2 trial

siogtx.com

Turnstone Biologics

Takeda

RIVAL-01/TAK-605 solid tumors initiation of phase 1/2 trial

turnstonebio.com

takeda.comApellis

Swedish Orphan Biovitrum

pegcetacoplan sporadic amyotrophic lateral sclerosis


apellis.com

sobi.com


http://www.americancryostem.com/http://www.americancryostem.com/http://biopathholdings.com/http://biopathholdings.com/https://codagenix.com/https://codagenix.com/https://www.f-star.com/https://www.f-star.com/https://www.igcpharma.com/https://www.igcpharma.com/http://junshipharma.com/en/Index.htmlhttp://junshipharma.com/en/Index.htmlhttps://maplightrx.com/https://maplightrx.com/https://maplightrx.com/https://www.nordicnanovector.com/https://www.nordicnanovector.com/https://www.northseatherapeutics.com/https://www.northseatherapeutics.com/https://www.northseatherapeutics.com/https://www.qpexbio.com/https://www.qpexbio.com/https://silicontx.com/https://silicontx.com/http://phoenixmd.ca/http://phoenixmd.ca/http://phoenixmd.ca/https://www.ascentagepharma.com/https://www.ascentagepharma.com/https://www.inovio.com/https://www.inovio.com/https://www.pmvpharma.com/https://www.pmvpharma.com/https://www.pmvpharma.com/https://regenxbio.com/https://regenxbio.com/https://siogtx.com/https://siogtx.com/https://turnstonebio.com/https://www.takeda.com/https://turnstonebio.com/https://www.takeda.com/https://apellis.com/https://www.sobi.com/https://www.sobi.com/https://apellis.com/https://www.sobi.com/




Company Drug/Device Medical Condition Status Sponsor ContactApnimed AD109 obstructive sleep apnea first patient dosed in

phase 2 trialapnimed.com

Athira Pharma ATH-1017 mild-to-moderate Alzheimer's disease


athira.com

Cara Therapeutics oral Korsuva (difelikefalin tablets)

moderate-to-severe pruritus in atopic dermatitis patients


caratherapeutics.com

Istari Oncology PVSRIPO in combination with Keytruda

recurrent glioblastoma multiforme


istarioncology.com

Oramed oral insulin capsule ORMD-0801

nonalcoholic steatohepatitis initiation of phase 2 trial oramed.com

Rafael Pharma CPI-613 (devimistat) in combination with hydroxychloroquine

clear cell sarcoma of soft tissue initiation of phase 2 trial rafaelpharma.com

Verastem Oncology VS-6766 recurrent low-grade serous ovarian cancer

initiation of phase 2 trial verastem.com

Abivax ABX464 ulcerative colitis patient enrollment complete in phase 2b trial

abivax.com

Ionis Pharma

AstraZeneca

ION449 (AZD8233) dyslipidemia initiation of phase 2b trial ionispharma.com

astrazeneca.com

3S Bio

SOBI

Selecta Biosciences

SEL-212 chronic refractory gout initiation of phase 3 trial 3sbio.com

sobi.com

selectabio.com

Bausch Health and Bausch + Lomb

NOV03 (perfluorohexyloctane)

dry eye disease associated with Meibomian gland dysfunction

initiation of phase 3 trial bausch.com

Bellerophon Therapeutics

INOpulse fibrotic interstitial lung disease first patient enrolled in phase 3 trial

bellerophon.com

Deciphera Pharmaceuticals

Quinlock second-line gastrointestinal stromal tumor


deciphera.com

Insmed brensocatib bronchiectasis first patient dosed in phase 3 trial

insmed.com

Ionis Pharmaceuticals

AKCEA-APOCIII-LRx adult patients with familial chylomicronemia syndrome

initiation of phase 3 trial ionispharma.com

Mycovia Pharmaceuticals

teseconazole (VT-1161) recurrent vulvovaginal candidiasis

last patient visit complete in phase 3 trial

mycovia.com

Oramed oral insulin capsule ORMD-0801

type 2 diabetes initiation of phase 3 trial oramed.com

PTC Therapeutics vatiquinone (PTC743) Friedreich ataxia initiation of phase 3 trial ptcbio.com

Phathom Pharmaceuticals

vonoprazan healing and maintenance of healing of erosive esophagitis; relief of heartburn


phathompharma.com

Pfizer marstacimab (PF-06741086)

severe hemophilia A or B, with or without inhibitors


pfizer.com


https://apnimed.com/https://apnimed.com/https://www.athira.com/https://www.athira.com/https://www.caratherapeutics.com/https://www.caratherapeutics.com/https://istarioncology.com/https://istarioncology.com/https://www.oramed.com/https://www.oramed.com/https://rafaelpharma.com/https://rafaelpharma.com/https://www.verastem.com/https://www.verastem.com/https://www.abivax.com/https://www.abivax.com/https://www.ionispharma.com/https://www.astrazeneca.com/https://www.ionispharma.com/https://www.astrazeneca.com/http://3sbio.com/en/https://www.sobi.com/https://www.selectabio.com/http://3sbio.com/en/https://www.sobi.com/https://www.selectabio.com/https://www.bausch.com/https://www.bausch.com/https://www.bausch.com/https://bellerophon.com/https://bellerophon.com/https://bellerophon.com/https://www.deciphera.com/https://www.deciphera.com/https://www.deciphera.com/https://insmed.com/https://insmed.com/https://www.ionispharma.com/https://www.ionispharma.com/https://www.ionispharma.com/https://www.mycovia.com/https://www.mycovia.com/https://www.mycovia.com/https://www.oramed.com/https://www.oramed.com/https://www.ptcbio.com/https://www.ptcbio.com/https://www.phathompharma.com/https://www.phathompharma.com/https://www.phathompharma.com/https://www.pfizer.com/https://www.pfizer.com/




Company Drug/Device Medical Condition Status Sponsor ContactPrincipa Biopharma rilzabrutinib patients with moderate-to-

severe pemphigus who are either newly diagnosed or relapsing with chronic disease


principiabio.com

RedHill Biopharma RHB-204 pulmonary nontuberculous mycobacteria disease caused by Mycobacterium avium Complex (MAC)

initiation of phase 3 trial redhillbio.com

Zymeworks zanidatamab previously treated HER2 gene-amplified biliary tract cancer

Breakthrough Therapy designation granted by the FDA

zymeworks.com

Moleculin Biotech WP1066 diffuse intrinsic pontine glioma, medulloblastoma and atypical teratoid rhabdoid tumor

Rare Pediatric Disease designation granted by the FDA

moleculin.com

Asklepios BioPharmaceutical

Selecta Biosciences

MMA-101 isolated methylmalonic acidemia due to methylmalonyl-CoA mutase gene mutations

Orphan Drug designation granted by the FDA

askbio.com

selectabio.com

Catalyst Biosciences Marzeptacog alfa episodic bleeding in subjects with hemophilia A or B with inhibitors

Fast-Track designation granted by the FDA

catalystbiosciences.com

RhoVac RV001 prostate cancer Fast-Track designation granted by the FDA

rhovac.com

Protagonist Therapeutics

PTG-300 polycythemia vera Fast-Track designation granted by the FDA

protagonist-inc.com

Theralase Technologies

Theralase Bacillus Calmete Guérin unresponsive nonmuscle-invasive bladder cancer carcinoma In situ

Fast-Track designation granted by the FDA

theralase.com

Alnylam Pharmaceuticals

Oxlumo (lumasiran) primary hyperoxaluria type 1 approved by the FDA alnylam.com

Armis Biopharma VeriFixx Small Bone Implant

fixation of osteotomies and reconstruction of the lesser toes following correction procedures for hammertoe, claw toe and mallet toe

approved by the FDA armisbiopharma.com

Cerus Corporation INTERCEPT Blood System for Cryoprecipitation

treatment and control of bleeding, including massive hemorrhage, associated with fibrinogen deficiency

approved by the FDA cerus.com

Eiger Biopharmaceuticals

Zokinvy (lonafarnib) Hutchinson-Gilford progeria syndrome and processing-deficient progeroid laminopathies

approved by the FDA eigerbio.com

Genentech Xofluza (baloxavir marboxil)

post-exposure prevention of influenza for patients 12 years of age and older after contact with an individual who has the flu

approved by the FDA for expanded indication

gene.com


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Company Drug/Device Medical Condition Status Sponsor ContactGenentech Gavreto (pralsetinib) adults and children over 12

years of age with advanced or metastatic RET-mutant medullary thyroid cancer who require systemic therapy, or with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory

approved by the FDA gene.com

Novartis Xolair (omalizumab) add-on maintenance treatment of nasal polyps in adults over 18 years of age with inadequate response to nasal corticosteroids

approved by the FDA for expanded indication

novartis.com

Profound Medical Group

Sonalleve osteoid osteoma approved by the FDA under a Humanitarian Device Exemption

profoundmedical.com

Rhythm Pharmaceuticals

Imcivree (setmelanotide)

chronic weight management in adults and pediatrics over the age of six with obesity due to proopiomelanocortin, proprotein convertase subtilisin/kexin type 1 or leptin receptor deficiency confirmed by genetic testing

approved by the FDA rhythmtx.com

Viatris

ViiV Healthcare

pediatric dolutegravir tablets for oral suspension, 10 mg

treatment of HIV-1 infected pediatric patients at least 4 weeks old and weighing at least 6.6 pounds

tentative approval granted by the FDA under the U.S. President's Emergency Plan for AIDS Relief

viatris.com

viivhealthcare.com

Y-mAbs Therapeutics

Danyleza (naxitamab-gqgk)

patients with relapsed or refractory high-risk neuroblastoma

approved by the FDA ymabs.com

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Clinical Research CoordinatorCovenant Research and ClinicsFt. Myers and Sarasota, FL

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Chain-of-Custody Maps a Valuable Visual Aid for Illustrating Data Flow and Integrity to RegulatorsMaking Coverage Analysis Determinations for Outside-the-Box SituationsCOVID-19 UpdateIndustry BriefsUp and ComingDrug & Device Pipeline NewsJobWatch

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