Drug & Device Pipeline News

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COVID-19 Update…2 Industry Briefs …4 Trend of Longer Trial Timelines is Likely Up and Coming…6 Drug & Device Pipeline News…11 to Continue Thirty-eight drugs and devices have By Charlie Passut formance will really come from our ability to entered a new trial phase this week. linical trial timelines have been best manage complexity, customization and JobWatch…13 lengthening since 2014 and with fragmentation,” Getz said last week at the C COVID-19 conditions forcing adap- Outsourcing in Clinical Trials USA virtual con- tation and adoption of new technologies ference. “These macro trends are inevitable.” 15TH ANNUAL and processes, the trend is likely to continue, But other complications “are actually FDA INSPECTIONS vSUMMIT if not accelerate. challenging and conspiring against our bold While the primary response to the development ambitions. They all suggest Tuesday, Nov. 17 – Wednesday, Nov. 18, 2020 pandemic represented important, and that our programs are actually getting lon- Please join us in celebrating 15 years of bringing together industry and the FDA in sometimes transformational, changes in ger. If you look at individual tasks and phase- harmonious collaboration. trials, those changes “will likely contribute specific cycle times, you’ll see that they’re all REGISTER TODAY to even greater complexity and increased increasing in length and we’re seeing high levels of customization and operating frag- levels if not higher levels of variation, that is mentation,” says Kenneth Getz, director one of the macro trends that we’re seeing. FREE WEBINAR of the Tufts Center for the Study of Drug Protocol complexity and customization OCTOBER Development (CSDD). and fragmented operating activity — are “Long term, we believe that develop- the primary factors that are impacting our 14 ment speed, efficiency and optimized per- see Trend of Longer Trials on page 8 » Make Real-Time, Real-World Data Work for You: How to Accelerate Clinical T Cells Could Play Crucial Role in COVID-19 Trials and Enhance Market Access Vaccine and Treatment Trials, Experts Say Sponsored by InterSystems REGISTER By Leslie Ramsey especially in older populations with reduced

he first wave of COVID-19 vaccine de- immune capacity.” NEW WHITE PAPERS AVAILABLE

velopment has not monitored the re- Rothman was joined in a WCG Clini- Clincierge – Patient Travel Management | Let’s talk about your trial today! 888.458.8579 / [email protected] PATIENT SUPPORT SOLUTIONS IN RARE DISEASE DURING THE COVID -19 PANDEMIC Keep Your Participants Safe And Your Study On Time With Clincierge’s Travel & Logistics Coordinators sponse of T cells to the virus, a missed cal webinar by former FDA commissioner Patient Support Solutions 38+ Countries Optimized Patient Travel Itineraries T 170+ Trials Supported Strategic Travel Solutions in Rare Disease During opportunity that should be addressed in Scott Gottlieb, who acknowledged the NAVIGATE THE RISKS OF TRAVEL DURING COVID-19 Chartered ights, long-distance drivers professionally trained to work with study patients, and more. Work with a Clincierge Coordinator who understands all of a patient’s travel options. the COVID-19 Pandemic the next wave of COVID-19 research, two potential of T cells to contribute to efforts ENHANCED GROUND TRANSPORTATION Beth is a patient with a rare disease needed to y to Spain for her study visits, but air travel and public transportation were no longer viable options due to From Clincierge COVID-19. To ensure she made it to her appointments, her STRATEGIC TRAVEL Clincierge Coordinator scheduled two extended private SOLUTIONS car trips, with overnight accommodations for both Beth • Convert Air to Ground and her driver. experts said last week. to vanquish the virus but questioned Transportation • Coordinate Overnight Accommodations

• Enhanced Safety & CHARTERED FLIGHTS Sanitation Protocols Meredith is immuno-suppressed and had only two visits left to complete her study visits. Potential exposure to COVID-19 made airports and commercial ights too risky for her. Her Clincierge Coordinator chartered “Ongoing vaccine trials do not appear whether vaccines were the proper applica- private ights to transport Meredith to her visits safely. LEARN MORE to monitor the T cell response in detail, if tion. He said they could possibly be used in at all,” said James Rothman, chair of the COVID-19 treatments. centerwatch.com/whitepapers Department of Cell Biology at Yale University It’s pretty well established, Gottlieb School of Medicine and winner of the 2013 said, that a large percentage of people FREE WEBINAR Nobel Prize for medicine. have in their immune systems T cells OCTOBER The robust response of these cells was that could react with and kill the SARS- discovered after most COVID-19 vaccine CoV2 virus. 27 trials began, he said, adding that “T cell “What we don’t know is whether that responses need to be assessed after the first confers any protection,” he said. “If it’s going Bringing ClinOps Technology wave of approvals [of vaccines] to improve to confer protection, it wouldn’t be against to the Clinical Site Stories from the Frontlines and compare products and their utility, see T Cells Could Play Crucial Role on page 9 » Sponsored by Agatha, Inc. REGISTER Volume 24, Issue 38. © 2020 CenterWatch WCG | CWWeekly October 5, 2020 2 of 13 COVID-19 Update

COVID-19 Drug Research are receiving oxygen or ventilator support. patients with COVID-19 who reside in com- Roundup The monoclonal antibody will be adminis- munity care homes. The trial will inves- COVID-19 Therapies: tered via a single one-hour infusion and will tigate the drug’s anti-inflammatory and The NIH’s National Institute for be compared with placebo plus standard prophylaxis effects against the disease. Allergy and Infectious Diseases has of care in terms of its ability to reduce the Recruitment of 750 patients across the UK halted enrollment of severely ill COVID-19 effects of the infection on the lungs. The will begin in late October. The Wellcome patients in a phase 3 trial of Merck’s Rebif primary endpoint will be the proportion initiative’s COVID-19 Therapeutics Acceler- (interferon beta-1a) with Gilead Sciences’ of patients who are alive and free of lung ator, in addition to the Bill & Melinda Gates remdesivir due to serious adverse events. failure at 28 days. Foundation and Mastercard, is sponsoring An interim review of safety data found Regeneron Pharmaceuticals has the trial. more adverse events among patients on released early data from its 275-patient COVID-19 Vaccines: high-flow oxygen/noninvasive mechanical phase 1/2/3 COVID-19 trial, which show Two leading COVID-19 vaccine develop- ventilation who received Rebif compared that antibody therapy REGN-COV2 ers, AstraZeneca and Moderna, have hit to those who did not receive the drug. reduced the proportion of patients new speed bumps that could delay their No concerns existed among patients who with COVID-19 who required additional promising COVID-19 vaccine candidates, had less severe COVID-19, and the trial will medical visits compared with placebo. reducing the possibility of a vaccine being continue to enroll patients on low-flow Approximately 7.7 percent and 4.9 per- ready ahead of the U.S. presidential elec- oxygen and those not requiring supple- cent of patients given the high and low tion on Nov. 3. The FDA has broadened mental oxygen. dose of the drug, respectively, required its already-significant investigation into The FDA has approved a phase 2 study additional visits, while 15.2 percent of the halted phase 3 trial of AstraZeneca’s of FSD Pharma’s investigational COVID-19 patients assigned to placebo required AZD1222 after a participant in the UK signature drug, FSD201. The primary ob- additional medical attention. A phase 2/3 experienced a serious adverse event. The jective of the randomized, multicenter trial portion of this trial is set to enroll up to agency is reportedly asking for data from will be to determine whether the therapy 1,300 patients who will be followed for 29 previous studies of similar vaccines that improves time to symptom relief faster days to examine the association between the same researchers worked on. Mean- than standard of care alone in 352 hospi- treatment and viral shedding in the upper while, Moderna CEO Stéphane Bancel talized patients with COVID-19. Dosing of respiratory tract. Three other late-stage said this week that he doesn’t expect the the trial’s first patients is expected some trials are also investigating REGN-COV2 for company to ask the FDA for an Emergency time in October 2020. COVID-19. Currently, a U.S.-based phase Use Authorization (EUA) for its vaccine The FDA has also greenlighted Stemed- 3 trial is testing the therapy in hospital- before Nov. 25 at the earliest. He had previ- ica Cell Technologies’ phase 2 trial, which ized patients with COVID-19, and another ously indicated that the vaccine could be will study the company’s intravenous study is testing the therapy for infection ready by early November. Bancel says the ischemic-tolerant mesenchymal stem-cell prevention in people who live with indi- company just does not have enough sup- treatment in patients with moderate- to- viduals who have been infected. porting data to file for an EUA yet. severe COVID-19. Patients in the trial will The University of Oxford is getting Moderna’s COVID-19 vaccine candi- be randomized to receive either the stem- ready to launch a phase 2 trial of AbbVie’s date, mRNA-1273, generated an immune cell therapy plus placebo or placebo with anti-inflammatory drug adalimumab in continues on next page » standard treatment. In emergency use and expanded use programs, Stemedica’s treatment was shown to reduce oxygen requirements within just one to two days CenterWatch Weekly of treatment initiation in 14 critically ill (ISSN 1528-5731) CenterWatch Main and Editorial Offices 300 N. Washington St., Suite 200, Falls Church, VA 22046 Beth Belton Editorial Director patients with COVID-19. Tel: 866.219.3440 • 617.948.5100 Stephanie Akers Production [email protected] / [email protected] A phase 2 trial in the U.S., launched by Russell Titsch Business Development Director Permission requests can be emailed to [email protected]. GlaxoSmithKline, is studying investiga- © 2020 CenterWatch. No part of this publication may be distributed tional rheumatoid arthritis drug otilimab in or reproduced in any form or by any means without the express Advertising packages and reprints are available: written consent of the publisher. Email [email protected] or call 703.538.7651. hospitalized patients with COVID-19 who

response in older adults between the ages a single dose generated antibodies to the A new trial in India has been launched of 56 and 71, according to new phase 1 coronavirus after 29 days, J&J said. to investigate AstraZeneca’s and Oxford trial data. The study administered two 25 CureVac has initiated its phase 2 trial University’s COVID-19 vaccine candidate, mcg or 100 mcg doses of mRNA-1273 one of CVnCoV, its messenger RNA COVID-19 Covishield, in 300 volunteers. The Indian month apart in 40 healthy adult volun- vaccine candidate, in Panama and Peru. Council of Medical Research and the Na- teers. The vaccine was well-tolerated and Enrollment will focus on 690 patients tional Institute for Research in Tuberculosis produced significant binding and neutral- between the ages of 18 and 60 as well as will run the trial. izing antibodies against COVID-19. Im- those who are 61 years and over. Patients Russia has announced completion of mune responses in this older cohort were will receive two vaccine doses 28 days phase 3 clinical trials of Vector Institute’s comparable to those observed in younger- apart. According to CureVac, initial results coronavirus vaccine, EpiVacCorona. The aged groups, the study found. Currently, are expected in the fourth quarter of country’s prime minister has said prepara- Moderna’s COVID-19 vaccine candidate is 2020. Around the time the initial find- tions are being made to approve the vaccine being investigated in 30,000 participants ings are released, the study plans on also for Russia by Oct. 15. in phase 3 testing in the U.S. launching a 30,000-participant global Researchers from two Chilean universi- The FDA has placed a partial clini- phase 2b/3 trial to examine the efficacy ties are gearing up to begin clinical trials cal hold on Inovio Pharmaceutical’s of the vaccine against COVID-19. Phase 1 of Sinovac’s and Johnson & Johnson’s planned phase 2/3 trial of its COVID-19 testing was launched in June in Germany, COVID-19 vaccines following recent ap- vaccine candidate INO-4800, requesting and CureVac is in the final stages of cut- provals made by Chile’s Institute of Public more information before the trial can ting a supply deal with the EU for 225 Health. Chile may also play host to a trial begin. The hold was not because of any million vaccine doses. from AstraZeneca, which has also applied adverse events related to Inovio’s ongo- Kangtai Biologics, which has collaborat- to start trials in the country. ing phase 1 trial of the candidate, the ed with AstraZeneca to develop COVID-19 Dr. Reddy’s Laboratories has not yet company said. Rather, the agency had vaccine candidate AZD1222, is working with filed a formal application to begin phase 3 questions about the late-stage study and the UK-based company to produce up to trials of Russia’s COVID-19 vaccine, Sputnik about the device that will be used in the 200 million doses of the vaccine by the end V, according to India’s Central Drugs and trial to deliver the vaccine. of next year. Standards Control Organization. No plans Single doses of Johnson & Johnson’s COVAXX has safely dosed its first have been made for any Emergency Use (J&J) COVID-19 vaccine candidate AD26. healthy adult volunteers in its phase Authorization of the vaccine without COV2.S generated a strong antibody re- 1, open-label, dose-escalation trial of relevant local data. Still, the first deputy sponse in patients, according to early trial COVID-19 vaccine candidate UB-612. The CEO of the Russian Direct Investment Fund data. The company released interim results study, conducted in Taiwan, is evaluating (RDIF) has said clinical trials will start in of a phase 1/2a trial that compared one the safety, tolerability and immunoge- India soon. Additionally, the CEO has said and two doses of the vaccine vs. a placebo nicity of the vaccine in up to 60 healthy that the RDIF is speaking with Indian com- in 1,045 participants. One group was made adults between the ages of 20 and 55. panies for contract manufacturing of the up of volunteers age 18 to 55 and another Ascending doses of the vaccine will be vaccine. Russia is also looking to partners group was comprised of adults age 65 to delivered in two intramuscular injections in Brazil, China and Korea for a manufac- 75. Nearly all participants who were given 28 days apart. turing deal.

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FDA Enforcement of Clinical investigators per year — mostly for data pelling demonstrations of clinical benefit” Trial Violations Declining falsification — under President Obama. that outweigh the risk. If the investigational Under Trump The FDA did not dispute the figures drug itself has abuse potential, the guidance The FDA has been slacking off on enforce- in the Science article and said that Trump says the FDA will consider its positive and ment of clinical trial regulations, according administration policies were not a factor. negative public health effects, including its to a recent investigation of the agency’s “The number of warning letters can ebb and effect on nonpatients, such as members of inspection statistics that shows a substantial flow,” the agency wrote in an email. the patient’s household. drop in serious citations in the first three The reporting of the story was funded by Drugs intended to provide relief of years of the Trump administration. Science’s Fund for Investigative Journalism. withdrawal symptoms are not covered by Since 2009, the FDA has conducted the guidance, the FDA says, because they about 6,700 inspections of clinical investiga- FDA Encourages Use of New usually are not sufficient to reduce the risk of tors and IRBs, issuing the strongest citation, Endpoints in Trials of Opioid returning to opioid abuse. “Official Action Indicated” (OAI) in only 291 Abuse Treatments To read the guidance, click here: https:// cases (4 percent) for serious, illegal and po- Sponsors of opioid use disorder (OUD) treat- bit.ly/3lcHLWf. tentially dangerous clinical trial infractions. ments should expand their trials beyond col- But in the first three years of the Trump lecting evidence for an NDA submission and Bladder, Renal Cancer Trials administration, that number dropped to 1 gather data on additional clinically mean- Should Use Blinded percent. In comparison, the eight years of ingful outcomes the FDA considers “highly Independent Central Review, the Obama presidency saw the agency issue valuable,” according to a final guidance the FDA Says OAI citations in about 6 percent of cases, agency released last week. Trials of adjuvant treatments for bladder and an article in the October issue of Science A change in drug-use pattern, particular- renal cancer that use disease-free survival magazine points out. ly abstinence, is typically used as a primary (DFS) as the primary endpoint should rely The article’s authors examined 1,600 endpoint for OUD treatment trials, according on a blinded independent central review FDA inspection and enforcement docu- to the guidance, but the FDA is interested in (BICR) to exclude potential participants with ments issued since 2009, including warn- expanding primary and secondary end- metastatic disease, according to two draft ing letters and investigator debarment points to include outcome measures “impor- guidances the FDA released last week. notices, concluding the agency’s regula- tant to patients and their families, clinicians The two guidances provide virtu- tory enforcement has become “lax, slow- and the public.” ally identical recommendations for trial moving and secretive.” Additional endpoints could include eligibility, imaging assessments, data The number of warning letters issued to reduction in such adverse outcomes as analysis and interpretation of results. Both investigators and IRBs also dropped dramati- mortality, need for emergency medical recommend that trials use BICR to define cally during the first three years of the Trump intervention, and hepatitis C infection or DFS and disease recurrence. presidency. The FDA issued only 12 warning reinfection. The guidance also recommends Interim DFS analyses are not encour- letters from 2017 to 2019, while President endpoints focusing on change in disease aged, the guidance says, “because im- Obama’s FDA issued 99 from 2009 to 2011, status as measured by standard diagnostic mature data may lead to over- or underes- the first three years of his first term, and 36 criteria, patient-reported effects on feeling timation of magnitude of improvement.” from 2014 to 2016, the last three years of his or function (e.g., reduction in intensity of the However, trial protocols and statistical second term. urge to use opioids) or reduction of opioid analysis plans should include a formal Warning letters in Obama’s first term re- use below a clinically significant level. interim analysis of overall survival at the sulted in seven trial and IRB shutdowns and Sponsors can propose other endpoints, time of final DFS analysis. restrictions, but the Trump administration the guidance says, and can study several end- Comments on the draft guidances are has not followed through with enforcement points or composite endpoints in one trial. due by Nov. 30. action on any of its 12 cases. The guidance is virtually unchanged To read the bladder cancer guidance, Noncompliant investigators also got off from the August 2018 draft save for a new click here: https://bit.ly/3d4Am8F. light under Trump; only two were barred section on benefit-risk considerations that To read the renal cancer guidance, click from participating in future FDA-regulated suggests trials of products associated with a here: https://bit.ly/2GdaUln. trials. The FDA barred an average of three risk of serious adverse events present “com- continues on next page »

Novo Nordisk, Covance Leukemia Society to Launch COVID-19 Prevention Network Recognized with ACRP-Avoca Global Pediatric Trial Deploys Mobile COVID-19 Quality Awards Collaboration Vaccine Trials Pharma company Novo Nordisk and CRO The Leukemia and Lymphoma Society is The National Institute of Allergy and Infec- Covance topped the list of winners of the partnering with PRA Health Sciences to tious Diseases (NIAID) has partnered with Association of Clinical Research Professionals launch the first global master clinical trial on Matrix Medical Network to help more than (ACRP) and the Avoca Group joint Quality pediatric relapsed acute leukemia. 20 academic medical centers involved Awards last week. The Pediatric Acute Leukemia (pedAL) in NIAID’s COVID-19 Prevention Network Finalists for the sponsor and CRO awards trial will occur at 200 sites in the U.S., Austra- establish mobile health clinics at universi- were selected based on responses to a sur- lia, Canada and New Zealand that are part of ties, hospitals and other locations across vey of sites Avoca conducted in early 2020. the Children’s Oncology Group network of the U.S. Sites were asked to rate CROs and sponsors children’s hospitals and will test a variety of Mobile clinics will serve communities in on a series of attributes related to quality novel treatments simultaneously. PRA will Arkansas, Colorado, Florida, Illinois, Kansas, outcomes, including partnership and com- provide trial management and oversight. Maryland, Missouri, New Jersey, New York, munication skills, as well as quality of clinical PedAL’s goal is to establish a genetic North Carolina, Texas and Washington trial design, execution and oversight. sequencing process to identify patterns and The collaboration, which is part of the Following Novo Nordisk in the sponsor drivers of the disease, consolidate pediatric Operation Warp Speed initiative and is category were GlaxoSmithKline in second cancer data from multiple institutions into supported through an agreement with the place and AstraZeneca in third place. ICON a single data set that will be available to Fred Hutchinson Cancer Research Center, and PRA Health Sciences were awarded researchers worldwide and form a global will reach trial participants who traditionally second place and third place, respectively, in collaborative to standardize pediatric leuke- have difficulty traveling to trial sites, includ- the CRO category. mia drug development efforts. ing those who reside in rural locations.

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This feature highlights changes in clinical Deep Genomics Matrivax Research and trial organizations’ personnel. Ferdinand Massari has been appointed Development chief medical officer of Deep Genomics. Enda Moran has been appointed vice presi- Achieve Life Sciences Massari was most recently the co-founder dent of process development and chemis- John Bencich has been named CEO of and leader of Kintai Therapeutics. try, manufacturing and controls (CMC) at Achieve Life Sciences. Prior to this ap- Matrivax Research and Development. Most pointment, Bencich served as the compa- Enesi Pharma recently, Moran was the vice president of ny’s chief financial and operating officer. Keith Howard has been named Enesi CMC development and clinical manufactur- Cindy Jacobs, former chief medical officer Pharma’s chief scientific officer. Formerly, ing at Northern Biologics. of Achieve, will now serve as president of Howard was chief development officer the company. at Vaccitech. Medable Medable has appointed Mary Costello head Adamas Pharmaceuticals Freeline Therapeutics of its site and investigator network. Costello Adamas Pharmaceuticals has tapped Adrian Freeline Therapeutics has promoted its vice was most recently vice president of clinic de- Quartel to serve as its chief medical officer. president of research, Romuald Corbau, to velopment and patient advocacy. Rasmus Quartel most recently was global head of take on the role of chief scientific officer. Hogreffe, former head of virtual trials at LEO medical affairs at BioMarin. Innovation Lab, has been appointed by Med- H-CYTE able to vice president of decentralized trial Arcus Biosciences Robert Greif has been named CEO of H- innovation. Additional hires include Steve Jennifer Jarrett has returned to her posi- CYTE, a regenerative cellular therapeutics Lesser to vice president of partner strategy tion as chief operating officer at Arcus Biosci- developer. Greif most recently served as and Heidi Pfefferkorn to decentralized ences after a stint as Uber Technologies’ vice chief commercial officer and business devel- solutions leader. Lesser was most recently president of corporate development and opment leader at Atox Bio. the lead solutions architect of real-world capital markets. evidence at SHYFT Analytics, and Pfefferkorn Inotrem was previously the director of external devel- BioAgilytix Labs Biotech company Inotrem has named opment operations at Novartis. The former Kennon Broadhurst has been appoint- Delphine Joyeux director of regulatory af- chief marketing officer of GeoPhy, David ed vice president of global marketing at fairs and quality assurance. Most recently, Swanger, was also named by Medable as its BioAgilytix Labs. Most recently, Broad- Joyeux was head of regulatory affairs at AB new senior vice president of marketing. In hurst served as global marketing lead for Science. Inotrem also appointed Simon addition, Ching Tian, who previously served oncology and hematology biosimilars Lambden, a current clinical lecturer at as general manager of data and digital at at Amgen. Cambridge University, to the role of head Novartis, was appointed senior vice presi- of medical science. dent of strategy and solutions. Boehringer Ingelheim Fremont Lars Dreesman will take the helm at Ipsen Montis Biosciences Boehringer Ingelheim’s (BI) U.S. biopharma Philippe Lopes-Fernandes has been ap- Karen Zinkewich-Péotti has taken the development and manufacturing business pointed executive vice president and chief helm of Montis Biosciences as its CEO. in California. Dreesman leaves his position business officer of Ipsen. Previously, Lopes- Zinkewich-Péotti was most recently a senior as head of BI’s global quality systems and Fernandes was the senior vice president and vice president at Ipsen. product compliance. global head of business development and alliance management at Merck. PsychoGenics Day One Biopharmaceuticals Daniela Brunner has been appointed chief Day One Biopharmaceuticals has tapped Kaleido Biosciences innovation officer of PsychoGenics. Brunner Jeremy Bender as its new CEO. Bender Kaleido Biosciences’s new president and joined PsychoGenics in 1999 and previously joins Day One from Gilead Sciences, CEO is Daniel Menichella. Menichella served as its director and senior vice presi- where he served as vice president of comes to Kaleido from his former position dent of behavioral R&D. PsychoGenics has corporate development. as CEO of CureVac. continues on next page »

also appointed Geoffrey Varty executive vice president of clinical research and develop- president and head of global development, president of research operations and Leslie ment to the role of chief medical officer. portfolio, regulatory and business develop- Street vice president of medicinal chemis- ment/licensing at the STADA Group. try. Varty and Street previously served as head Sonoma Biotherapeutics of in vivo pharmacology at Merck and senior Sonoma Biotherapeutics has appointed TriSalus Life Sciences director and head of medicinal chemistry at Leonard Dragone, former vice president for TriSalus Life Sciences has named Steven Cortex Pharmaceuticals, respectively. early clinical development at Janssen Bio- Katz as the company’s chief medical officer. pharma, to chief medical officer. Sejal Hall Katz had served as the chief medical adviser RespireRx Pharmaceuticals has also joined Sonoma as its vice president of TriSalus since 2018. RespireRx Pharmaceuticals has appointed of portfolio, program and alliance manage- David Dickason senior vice president of ment. Most recently, Hall was executive Vertice Pharma preclinical product development. Prior to director, research operations and program Scott Meyer, chief operating officer of this appointment, Dickason held senior management at Audentes Therapeutics. Vertice Pharma, has been promoted to CEO technical roles at iCeutica and Alkermes. Susan Lacy has also been hired by Sonoma of the company. Prior to joining Vertice in to serve as vice president of discovery. Lacy 2016, Meyer was vice president and general Silverback Therapeutics previously served in various director roles manager of Fougera. Valerie Odegard, chief scientific officer of during a 20-year tenure at AbbVie. Silverback Therapeutics, has added presi- Vibalogics dent to her list of duties at the company. STADA Group Tom Hochuli, former cell and gene therapy Silverback has also promoted Naomi Hun- Yann Brun, former managing director of Ex- operations head at Lonza, has been named der from her previous position of senior vice eltis, has assumed the role of executive vice global CEO of Vibalogics.

Keep your up to date Earn up to 18 contact hours accepted In this issue: by ACRP, SoCRA and CCIP organizations 2 CE program information 3 Regulatory update 6 IRB meeting minutes 16 CE post-test Subscribe Today! www.centerwatch.com/get-RP The issue of proper documentation in IRB liability By Sue Coons, MA

he guid ance Minutes of Institutional ing IRB meeting activity not only puts Review Boa Learning Objectives: rd (IRB) Meetings in IRBs in violation of regulatory standards, T Federal Register but also could put them in legal cross- 1. List some of the documentation deficiencies FDA is de finding in its IRB inspections. scribes the requirements for IRB meet- hairs if research participant Exam for Cont s are harmed inuin 2. Des g cribe the claims plai ntiffs i E n R in du ob g minutes and prov cation ertson v McGee ides recommenda- or perceive harm in a clinical trial. made against IRB members. » Learn critical and practical strategies tions for meeting applicabRlee resgeualartory Inadequate doc ch Practitioner 19.1um, e3nt aCtionn in IRB tact Hours3. Explain the importance of proper documentation of IRB activities. during FDA inspections, the guidance 4. Discuss the concept of individ Please note: The website for Research Practitioner Author Remigius N. Nw ual liability in IRB decisions.abueze Which of the following is useful exams and evaluations has changed. Please visit the argued that: to guide the preparation of IRB following website which wFilDl nAav’isga wtee ybous ittoe e afochr e bioresearch monito xam a. IRB rm- embers may have mee » Protections (OHRP) and Fofor completion: ting minutes? od and Drug ing (www .fda.gov/ScienceResearcihn/dividual liability in the case of a. Adm http://store.centerwatch.com/t-exams.aspx inistration (FDA). It is intended SpecialTopics/RunningClinica harm of a research sub If you have any questions, feel free to email lTrials/ bership rostjeercst; quorum issuebs;. pSrtoamndpta rd operating procedure for institutions and IRBs responsible default.htm b. Research institutions protect [email protected] or - reporting of noncompliance, suspoenn msioinutes’ preparation and for IRB me n/ clinical trials oversight of human subjceacllt 86 re6s-2e1a9-r3c4h4 0. cies found during IRB inspec mbers from liability maintenance tions to termination; Subpart D issues; and lack under HHS an c. IRB meeting documentation has c. A d FDA regulations. be: inadequate initial and/or continuing standardized minutes’ Requirements for Successful Com no bearing ofn o pr incorrect risk determination. However, a court case fro pletion otential liability template Tmo r etcheieve e caonrtalyc review; inadequate written procedures; t hours, participants must register, of IRB members d. All of the above read the full journ-al and ithnean dgoe qtou: hatttpe: /m/setoeret.ing minutes adn.d Nmoenme -of the above centerwatch.com/t-exams.aspx An IRB is comprised of seven » Critical self-examination and see IRB liability on page 4 mem Read the instructions on this page to register, select an bers. At the meeting, four internal audits are some of th active exam, successfully complete the post-test with a e best ways to catch e minimum score of 70%, complete the evaluation, and rrors, says attorney including a non-scientist and an Daniel L. view and print your certificate. Icenogle. unaliated member. For one hour The case for patient engagem a. T ent to imprruoe ve clinical trials during the meeting, the non- b. Fal » Subscriptions start at $197 a year By John W. Mitchell, MS se scientist leaves the room for an IRB liability and meeting minutes hour to take a conference call. For Which of -the following commonly n the quest to achieve better Learning Objectives: that hour, a quorum technically is out- tional Coordinator of Health Iins foobrsmerav- ed during inspections of still present because the requisite comes, control costs, and improve IRB o 1. Explain the state of patient engagement in clinical Who did the clinical trial perations? four members were at the start health under a slew of government a. M trials. I plainti s not name in the lawsuit? Pihssairng- minutes of the meeting. erefore, any policies and b. I 2. Describe the barriers to and case for patient laws in recent yearas. (Pi.er.i,n tchiep al inmvaecsetiugtaictaolr Outsourcing, patient innavoccurate account of meeting approval vote on a study ma lve- engagement strategies in clinical trials. de b. IRB c-hair attendance during that hour is valid. 3. c. Nurse coordinator c. Insu cient or inLicsto emxampplleets eo f dmeatrkaeitlin g and salesa s.tr ategies in able Care Act [ACA]), some True SUBSCRIBE TODAY! www .centerwatch.com/get-RP sales@cetnhintge urn-watecnghag.ecmoentm, whi c h h as alt e r n+1atively 617.948patien.t 5eng1age0men0t. d. IRB members in minutes b. False precedented happened: Clinicians of all been known by many names, idn.c Aluldl ionfg t he abov4. eDiscuss the future of patient engagement in clinical stripes began asking: What do patients patient centricit trials. What did the plainti s mentioy na nind patient activation, For studies involving c want? hildren, their complainretf aerbso tuot tthhee pIrRoBc eisns of invo lviRequirements for IRB meeting © 2020 CenterWatch CWW2438 WCG | CWWeekly October 5, 2020 8 of 13 Features

Trend of Longer Trials among non-top 50 companies, the clini- age for the demographic was -65.4 percent (continued from page 1) cal phase was nine months longer (89.9 while the disparity percentage in vaccine executional performance that drive higher months) and regulatory approval grew to trials was -48.2 percent. Latinx was expect- levels of inefficiency and challenge quality in 13 months. Meanwhile, CSDD estimates ed to make up 8.2 percent of clinical trials our research.” that 31 percent of active drugs in the but only accounted for 7.2 percent, with a CSDD found that for an entire new set current R&D pipeline are for rare diseases, disparity percentage of -12.4 percent and of 377 drugs and biologics approved by with 58 percent expected to gain regula- a disparity percentage in vaccine trials of the FDA between 2008 and 2018, the clini- tory approval this year. -31.5 percent. Meanwhile, whites made cal phase took an average of 83.1 months Durations were also increasing in up 75.9 percent of clinical trials, up from from 2008-2013, but that timeframe grew select executional areas. CSDD found that the 67.1 percent expected, with a disparity to 89.8 months for 2014-2018. in 2014-2018, it took an average of 145 percentage of 13.6 percent and a disparity Conversely, the regulatory phase shrank days for the study start-up period, up 13.7 in vaccine trials of 112.8 percent. from 13.6 months to 11.7 months over percent from the prior 2008-2013 period. “Public trust and awareness about clinical the respective timeframes (CenterWatch The pivotal trial execution segment also research have been negatively impacted by Weekly, July 20). “Some of that is due to the grew to an average 854 days and the the pandemic,” Getz said. “This is going to fact that a higher proportion of approvals last-patient-last-visit (LPLV) timeframe contribute to ongoing patient recruitment now are for treatments that are targeting extended to 36 days, up 6.3 and 9 percent, challenges, particularly challenges in recruit- rare diseases,” Getz said. Those remedies, respectively, from 2008-2013. In the area ing underrepresented communities in our plus oncology drugs, represent “one of of data-management and data-cycle development programs.” the dominant areas in our pipelines [that] times, “often what we hear is that the Getz said CSDD, through its assess- typically see expedited or accelerated re- largest challenge is the coordination of ments of performance and interviews with view timeframes.” But he also cited longer multiple data sources, some that include companies, found six strategic operating timeframes through all three clinical trial the electronic data capture as well as the practices that could be applied across phases for the 377 new drugs and biolog- use of wearable devices, specialty labs and all operating models to shorten drug ics studied. “We see that the cycle times a variety of other data sources, and that development timelines — early and open are getting longer, and the variation has presents some challenges.” collaboration; patient-engaged design and not improved. We continue to see inconsis- CSDD also found an alarming disparity execution planning; more time upfront tent practices and performance.” between the expected level of diversity in dedicated to precollaboration qualification Despite an industry perception that clinical trials and the actual rate of minor- and coordination; a commitment to flex- smaller companies tend to enjoy quicker ity participation. For new drugs and bio- ible but consistent execution; anticipated development timelines, Getz said observa- logics approved between 2007 and 2017, and preplanned design and executional tions by CSDD show the opposite is true. the organization found only 5.4 percent adaptation; and frequent, proactive and Of the 377 drugs studied, the clinical phase participation from the Black and African clear input from regulators. “That really for the top 50 pharmaceuticals with active American community, but the group was helps organizations determine whether drugs in the R&D pipeline took an aver- expected to make up 15.6 percent, based they feel comfortable assuming a risk and age 80.9 months in the clinical phase and upon disease prevalence rates and popula- accelerating activity further,” Getz said of 11.8 months for the regulatory phase. But tion census figures. The disparity percent- the final recommendation.

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T Cells Could Play Crucial Role the chance of having a proven vaccine moments like this, he said, when there is an (continued from page 1) before the November election. urgent need for life-saving solutions. getting the infection; it would be against The Emergency Use Authorization (EUA) “What it is, really, is a more flexible developing symptomatic disease from the guidance that was originally set to be standard” that allows the agency to respond infection. But we don’t know a lot about released before the end of September would quickly to a public health crisis. what, if anything, this means.” set the criteria sponsors need to meet to There is nothing surprising in the pro- The question of whether T cells trans- administer their vaccines to the highest-risk posed EUA guidance, Gottlieb said, adding late to clinical protection against the virus populations before formal FDA approval. In that it basically lays out the authorization requires a detailed investigation and that an unusual move, President Trump on Sept. principles the agency has been discussing currently isn’t being done, Rothman said. 23 said the White House would review the with vaccine developers all along, including Of 38 vaccines currently in trials, 34 per- proposed guidance, an action that led many the need for a vaccine trial to include in the cent are studying protein subunits and 16 to conclude Trump would bury the docu- placebo arm at least five severe cases and percent are nonreplicating viral vectors, 16 ment in an effort to keep the FDA from rais- some cases in older patients. The guidance percent are RNA based and 16 percent are ing standards for emergency authorization. also would require sponsors of phase 3 trials inactivated virus. The rest are DNA based In fact, the guidance would do the oppo- to track patients for at least two months. and replicating viral vectors. site, Gottlieb told attendees at the webinar “Not allowing the agency to put forward Gottlieb also strongly defended the last week on the potential of using T cells to that guidance would be problematic at the FDA’s approach to getting a vaccine out fight the virus. end of the day,” he said. to the public in the shortest amount of The guidance is “technically a lower “I think the agency needs to be left to its time, criticizing the Trump administration’s standard” than that needed for formal own devices to outline what the approval apparent attempt to derail a planned guid- FDA approval, Gottlieb said. However, the criteria is,” Gottlieb said. “The political sort of ance the president believes could reduce EUA pathway was basically conceived for intrigue around this process is not helpful.”

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Company Drug/Device Medical Condition Status Sponsor Contact COVID-19 Trials and Actions Covistat/Ensysce oral nafamostat acute respiratory failure due to initiation of phase 1 trial ensysce.com Biosciences COVID-19 Covaxx UB-612 COVID-19 vaccine first patient dosed in phase covaxx.com 1 trial Stemedica Cell intravenous allogeneic patients with moderate to severe IND approved by the FDA for stemedica.com Technologies mesenchymal stem cells lung injury due to COVID-19 phase 2 trial Windtree lyo lucinactant COVID-19 associated lung injury IND approved by the FDA for windtreetx.com Therapeutics and acute respiratory distress phase 2 trial syndrome FSD Pharma FSD201 (ultramicronized hospitalized patients with COVID-19 initiation of phase 2 trial fsdpharma.com palmitoylethanolamide) Novavax NVX-CoV2373 COVID-19 initiation of phase 3 trial novavax.com (COVID-19 vaccine) Organicell Zofin acellular therapy patients with SARS-CoV-2 who have expanded access protocol organicell.com Regenerative mild to moderate COVID-19, or who granted by the FDA Medicine are judged by a healthcare provider to be at high risk of progression to moderate disease ADial Assure/FaStep COVID-19 antibody point-of-care test for Emergency Use Authorization adialpharma.com Pharmaceuticals IgG/IgM Rapid Test COVID-19 (EUA) granted by the FDA Device Cepheid Xpert Xpress SARS- qualitative detection of the viruses EUA granted by the FDA cepheid.com CoV-2/Flu/RSV causing COVID-19, Flu A, Flu B, and molecular diagnostic RSV infections from a single patient test sample Hologic Panther Fusion SARS- testing of individuals without EUA granted by the FDA hologic.com CoV-2 assay symptoms or other reasons to suspect COVID-19 infection Quotient MosaiQ COVID-19 detection of COVID-19 antibodies EUA granted by the FDA quotientbd.com antibody test Other Trials and Actions Aerie AR-15512 (TRPM8 dry eye disease IND approved by the FDA aeriepharma.com Pharmaceuticals agonist) eye drop IRLAB mesdopetam (IRL790) levodopa-induced dyskinesias in IND approved by the FDA irlab.se Parkinson's disease Inversago Pharma INV-101 Prader-Willi syndrome and non- initiation of phase 1 trial inversago.com alcoholic steatohepatitis Kuur Therapeutics KUR-502 relapsed/refractory CD19-positive first patients dosed in phase kuurtx.com malignancies 1 trial Prelude PRT1419 relapsed/refractory hematologic first patients dosed in phase preludetx.com Therapeutics malignancies 1 trial Zucara Therapeutics ZT-01 type 1 diabetes first patient dosed in phase zucara.ca 1 trial BioMarin BMN 307 phenylketonuria first patient dosed in phase biomarin.com 1/2 trial Codiak Biosciences exoSTING advanced metastatic or recurrent, first patients dosed in phase codiakbio.com injectable solid tumors 1/2 trial continues on next page »

Company Drug/Device Medical Condition Status Sponsor Contact Aptinyx NYX-2925 fibromyalgia patient recruitment aptinyx.com recommenced in phase 2 trial Calithera telaglenastat (CB-839) nonsmall-cell lung cancer first patient dosed in phase calithera.com Biosciences 2 trial Concert CTP-692 schizophrenia patient enrollment complete concertpharma.com Pharmaceuticals in phase 2 trial Ocular Therapeutics OTX-CSI (cyclosporine dry eye disease first patients dosed in phase ocutx.com intracanalicular insert) 2 trial Aurinia voclosporin ophthalmic dry eye syndrome last patient study visit in auriniapharma.com solution phase 2/3 trial CiVi Biopharma/ CIVI030 (intravenous systemic sclerosis patient recruitment resumed civibio.com Eicos Sciences iloprost) in phase 3 trial Eisai lorcaserin Dravet syndrome initiation of phase 3 trial eisai.com Harbour BioMed batoclimab (HBM 9161) myasthenia gravis (MG) and adult first patient dosed in phase harbourbiomed.com immune thrombocytopenia 3 trials Infinity eganelisib (IPI-549) in patients with inoperable locally Fast-Track designation infi.com combination with a advanced or metastatic triple- granted by the FDA checkpoint inhibitor and negative breast cancer, in the first- chemotherapy line setting AbbVie elezanumab (ABT-555) spinal cord injury Orphan Drug and Fast-Track abbvie.com designations granted by the FDA Neurophth NR082 (rAAV2-ND4, Leber's Hereditary Optic Neuropathy Orphan Drug designation neurophth.com Therapeutics NFS-0) associated with ND4 mutation granted by the FDA CSL Behring Haegarda (C1 Esterase routine prophylaxis to prevent approved by the FDA for cslbehring.com Inhibitor Subcutaneous hereditary angioedema attacks in expanded indication [human] patients 6 years of age and older Eton Alkindi Sprinkle replacement therapy for approved by the FDA etonpharma.com Pharmaceuticals (hydrocortisone) oral adrenocortical insufficiency in granules children under 17 years of age GlaxoSmithKline Nucala (mepolizumab) hypereosinophilic syndrome (HES) approved by the FDA for gsk.com without an identifiable non-blood- expanded indication related cause of the disease in adults and children 12 years of age and older Janssen Simponi Aria active polyarticular juvenile approved by the FDA janssen.com (golimumab) idiopathic arthritis and active psoriatic arthritis in patients 2 years of age and older Masimo Rad-G Pulse Oximeter pulse oximetry and respiration rate approved by the FDA masimo.com monitoring Pfizer Xeljanz (tofacitinib) children and adolescents 2 years approved by the FDA for pfizer.com and older with active polyarticular- expanded indication course juvenile idiopathic arthritis Shionogi Fetroja (cefiderocol) hospital-acquired bacterial approved by the FDA for shionogi.com pneumonia and ventilator- expanded indication associated bacterial pneumonia Vertex Kalydeco (ivacaftor) children with cystic fibrosis ages approved by the FDA for vrtx.com Pharmaceuticals four months to less than six months expanded indication old who have at least one mutation in their CFTR gene

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