FDA: New Guidance for Expansion Cohort Trials

August 20, 2018

Expansion cohorts can help bring life-saving drugs to market quickly, but sponsors should be sure to update informed consent for patients and convene expert safety panels that meet regularly, the FDA says in new draft guidance.

NIH Delays Basic Brain Research Rules

August 20, 2018

NIH has delayed a controversial new policy that would have required basic brain and behavioral researchers to treat their work as clinical trials.

First Human Zika Vaccine Trial

August 20, 2018

Researchers are launching the first human trial of an experimental live Zika vaccine. The vaccine, which contains a weakened version of the virus, was de­veloped by scientists at the National Institute of Allergy and Infectious Diseases (NIAID). The trial plans to enroll a total of 28 healthy, non-pregnant adults, ages 18 to 50, at Johns Hopkins School of Public Health Center for Immunization Research and at the Vaccine Testing Center at Larner College of Medicine at the University of Vermont. Trial participants will be randomly assigned to receive a single subcutaneous dose of the experimental vaccine (20 participants) or a placebo (eight participants). Neither they nor investigators will know who is receiving the real thing. Participants will keep track of their tem­perature on diary cards at home – and return to the clinic for periodic checkups for about six months. Researchers will take blood samples during visits to see if they’re developing antibodies in response to the vaccine. Anna Durbin, a professor of International health at Hopkins School of Public Health, is leading the Phase I clinical trial. Most people infected with the Zika virus — primarily transmitted by certain mosquitoes — have mild to no symptoms. But it can cause serious birth defects and developmental problems in babies born to women infected during pregnancy. That’s why the Centers for Disease Control and Prevention advises women who are pregnant or planning to become pregnant not to travel to areas with a known Zika risk. “Zika virus infection remains a significant threat to pregnant women and their developing fetuses, and we can expect to see periodic outbreaks and cases in areas where Aedes aegypti mosquitoes thrive,” said NIAID Director Anthony Fauci. “NIAID remains committed to developing safe and effective Zika vaccines, and we are pleased to begin clinical testing of a live attenuated candidate.”

New Pipeline from Ivantis, Genentech, BeiGene and Endomag

August 20, 2018

Company Drug/Device Medical Condition Status
Amicus Therapeutics Galafold (migalastat) Fabry disease Approval granted by the FDA
Arbutus Biopharma Coporation and Alnylam Pharmaceuticals, Inc. ONPATTRO (patisiran) Hereditary transthyretin amyloidosis Approval granted by the FDA
Ivantis, Inc. Hydrus® Microstent, a microinvasive glaucoma surgery (MIGS) device Mild to moderate primary open-angle glaucoma in conjunction with cataract surgery Approval granted by FDA
Genentech Xlair® (omalizumab) Prevention of severe allergic reactions following accidental exposure to foods in people with allergies Breakthrough Therapy Designation granted by the FDA
Samsung Bioepis, Co., Ltd. SB26 ulinastatin-Fc (UTI-Fc) fusion protein Severe acute pancreatitis Phase I trial set to begin
Sesen Bio, Inc. Vicinium™ Non-muscle invasive bladder cancer (NMIBC) Granted Fast Track Designation by the FDA
BeiGene Ltd. Tislelizumab, an investigational anti-PD-1 antibody, combined with chemotherapy Potential first-line treatment for patients with Stage IIIB or IV squamous non-small cell lung
cancer (NSCLC)
Phase III clinical trial initiated
Eiger BioPharmaceuticals, Inc. Avexitide (formerly exendin 9-39) Post-bariatric surgical patients who experience post-bariatric hypoglycemia (PBH) Completion of enrollment in Phase II clinical trial
Izana Bioscience Namilumab Ankylosing spondylitis Phase II proof-of-concept clinical trial initiated
Rafael Pharmaceuticals, Inc. CPI-613 in combination with bendamustine Relapsed or refractory T-Cell Lymphoma Activation of a second clinical trial site for the Phase I study
Endomag Magtrace, the first non-radioactive, dual-tracer Lymphatic mapping in patients with breast cancer undergoing a mastectomy Granted premarket approval granted by the FDA
Cannabics Pharmaceuticals, Inc. Cannabics SR 5mg, daily for 3 months Advanced Cancer and Cancer Anorexia Cachexia Syndrome (CACS) Conclusion of trial announced; officials results are being evaluated
OrthoTrophix, Inc. TPX-100 Osteoarthritis Phase II trial results: improvements in function and cartilage in 55 percent of TPX-100 treated knees
Entasis Therapeutics ETX2514SUL Complicated urinary tract infections, including acute pyelonephritis (kidney infection) in adults Positive topline results from Phase II trial
Realm Therapeutics PR022 Atopic dermatitis Phase II trial failed to reach endpoint goals
INOVA Diagnostics, Inc. QUANTA Flash® HMGCR Aids in the diagnosis of idiopathic inflammatory myopathy (IIM), a group of conditions that affect the skeletal muscles Received FDA clearance

August 20: Job openings, upcoming industry conferences, webinars and training programs

August 20, 2018

Twice monthly, CWWeekly provides featured listings of clinical research job openings, upcoming industry conferences and educational programs from JobWatch, CenterWatch’s online recruitment website for both clinical research employers and professionals.

  For conferences, webinars, training programs and job postings, join the LinkedIn JobWatch group.
More Jobs

Senior Medical Director/PI
Quotient Sciences
Miami, FL

Clinical Research Coordinator
Catalina Research Institute, LLC
Montclair, CA

Clinical Project Manager
GlycoMimetics, Inc.
Rockville, MD

Clinical Trials Analyst
Mount Sinai
New York, NY

Clinical Research Coordinator I
Emory University
Atlanta, GA

Clinical Project Manager
Integrium, LLC
Phoenix, AZ

Clinical Research Coordinator
Allergy Partners of Western North Carolina
Asheville, NC

Infectious Diseases – Tenure leading Position
UNMC College of Pharmacy
Omaha, NE

Upcoming Conference

SEPTEMBER 10-12, 2018
Mastering EU Medical Device Regulation
Philadelphia, PA

SEPTEMBER 27, 2018
Emerging Biopharmaceutical Therapies
Washington, DC

OCTOBER 9-10, 2018
Medical Device Complaint Management
Waltham, MA

OCTOBER 23-25, 2018
FDA Inspections Summit – 13th Annual
Bethesda, MD

Training Programs

SEPTEMBER 1-31, 2018
Program Phlebotomy Training — Two Day Training
Various locations

FDA Suggests Endpoints for Opioid Treatments

August 13, 2018

Sponsors may not have to prove a proposed therapy helps addicts totally kick opioids to win FDA approval. But they will have to show it significantly cuts dependence on the potent pain meds, the agency says in new draft guidance.

FDA: Focus on Hardest to Treat in Cohort Cancer Trials

August 13, 2018

The FDA says sponsors considering adaptive studies for early phase cancer treatments should focus on patients with no other options. The agency notes that so-called expansion cohort trials — which start out relatively small but expand if a potential treatment shows promise — can be a way to get a treatment to market more quickly. The thinking is, the more participants in earlier trials the more likely it is to find and fix (if possible) glitches — ensuring faster approval or scrapping the potential treatment in its infancy if it doesn’t work or if risks outweigh benefits. “A lot of time and cost of clinical development is spent waiting in between the start and end of the phases of trials,” FDA Commissioner Scott Gottlieb said in a statement. “Expansion cohort trials can bring efficiency to drug development, potentially reducing development costs and time.” In a 17-page draft guidance released last week, the FDA says it wants to “establish an infrastructure” designed to help sponsors use cohorts to speed drug approvals without compromising safety. Its top recommendation: Focus on patients who are most seriously ill and don’t have any other treatment options. The agency also encourages researchers to hire centralized data management committees and IRBs and have them meet regularly to discuss latest findings and best next steps. “It is critical that investigators, IRBs and regulators are updated with new safety information so that they can provide the necessary oversight for the protection of human subjects and so that investigators can ensure that patients can provide adequate informed consent,” the draft document says. The jury’s still out on the effect of expansion cohorts. A 2017 study by Texas researchers, published in the Journal of the American Association for Cancer Research, analyzed 533 Phase I cancer trials between 2006 and 2011. They found the ones that used expansion cohorts went on to have successful Phase II trials at nearly twice the rate (48 percent) as studies that didn’t (27 percent). But just a year earlier, a team of Cleveland researchers analyzed 252 cancer trials conducted between 2004 and 2014 and found expansion cohorts made little or no statistical difference between success or failure in Phase II trials. Read the FDA’s draft guidance here:

New Pipeline from BioCryst, Elite, PharmaMar and Vertex

August 13, 2018

Company Drug/Device Medical Condition Status
OncoPep, Inc. PVX-410, investigational multi-peptide cancer vaccine Smoldering multiple myeloma (SMM) Phase Ib trial initiated
SW Safety Solutions, Inc. Nitrile Exam Gloves with Low Dermatitis Potential Claim Skin irritation or sensitization Initial – Looking for Principal Investigator to head clinical study
Kyowa Kirin, Inc. Poteligo (mogamulizumabkpkc) injection Relapsed or refractory mycosis fungoides (MF) or Sezary syndrome after at least one prior systemic therapy Approval granted by FDA
Celltrion, Inc. Bevacizumab biosimilar ‘CT-P16’ Metastatic colorectal cancer, metastatic breast cancer, non-small cell lung cancer, and glioblastoma Phase III trial initiated
Apotex Inc. New generic potassium chloride oral solution Hypokalemia (low potassium blood levels) in patients who are on diuretics Approval granted by the FDA under a novel term called the Competitive Generic Therapy designation
Alector, LLC AL001, a human recombinant monoclonal antibody Frontotemporal dementia (FTD) Granted Orphan Drug Designation by the FDA
Pliant Therapeutics, Inc. PLN-74809 Idiopathic pulmonary fibrosis (IPF) Granted Orphan Drug designation by the FDA
Castle Creek Pharmaceuticals, LLC Diacerein 1% ointment (CCP-020) Epidermolysis bullosa simplex (EBS) Fast Track Designation granted by the FDA
Elite Pharmaceuticals, Inc. Generic methadone hydrochloride 5 mg and 10 mg tablets Severe pain, maintenance treatment of opioid addiction (heroin or other morphine-like drugs) Approval granted by the FDA
Alteogen Inc. ALT-P7, an antibody-drug conjugate (ADC) using a Trastuzumab variant form of antibody Gastric cancer Granted Orphan Drug Designation by the FDA
Vertex Pharmaceuticals Inc. Orkambi (lumacaftor/ ivacaftor) Underlying cause of cystic fibrosis in children ages 2 to 5 with the most common form of the disease Approval granted by the FDA
BioCryst Pharmaceuticals, Inc. BCX7353 Prevention of angioedema attacks in patients with hereditary angioedema Fast Track Designation granted by the FDA
PharmaMar Lurbinectedin Small cell lung cancer Granted Orphan Drug Designation by the FDA
Sensus Healthcare, Inc. SRT-100+, the next-generation Superficial Radiation Therapy solution Non-melanoma skin cancers (NMSC) and keloids Granted 501(k) clearance by the FDA
Armagen Inc. GT-184, an investigational enzyme replacement therapy (ERT) Mucopolysaccharidosis type IIIA (also known as Sanfilippo Syndrome A or MPS IIIA) Granted Orphan Drug Designation by the FDA
Shionogi & Co., Ltd Mulpleta®(lusutrom-bopag) Thrombocytopenia in adult patients with chronic liver disease (CLD) who are scheduled to undergo a procedure Approval granted by the FDA

Vivitrol Helps HIV Patients Reduce Alcohol Intake

August 13, 2018

Monthly injections of the anti-addiction drug naltrexone (brand name Vivitrol) may help HIV-positive patients cut down on heavy drinking but it doesn’t help them stick to their med schedules, Yale researchers found. Researchers recruited 51 HIV patients for a four-year trial; all were heavy drinkers and strayed from their daily treatment plans. During the trial, patients received monthly naltrexone injections and regular counseling. The findings, published the journal AIDS and Behavior: Naltrexone helped patients reduce heavy drinking days. But it didn’t improve their antiretroviral adherence. Antiretroviral drugs have converted HIV and even AIDS from deadly diseases into chronic ones. But they only work if patients take them at least 95 percent of the time, researchers said.