2018

Features

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 But they will have to show it significantly cuts dependence on the potent pain meds, the agency says in new draft guidance.

Briefs

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 In a 17-page draft guidance released last week, the FDA says it wants to “establish an infrastructure” designed to help sponsors use cohorts to speed drug approvals without compromising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: www.fdanews.com/08-10-18-DraftGuidance.pdf.

Pipeline

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
Trial Results

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.

Research Center Profiles

August 2018: Research Center Profiles

August 13, 2018

Research Center Spotlight is a monthly selection of clinical research centers who have Research Center Profile pages posted on CenterWatch.com. Included in their annual subscriptions, company profiles are randomly selected to appear in this section, providing added exposure for their expertise and services in conducting and managing clinical studies. To learn more about becoming a Research Center Profile page subscriber, contact Sales at (617) 948-5100.

Features

WHO: Focus on Biosimilar Differences

August 6, 2018

Clinical trials of biosimilars should focus on how they differ from the drugs they copy rather than on creating a whole new safety or efficacy study, the World Health Organization says.

Briefs

FDA Signs Off on Treatment for Rare Adrenal Gland Tumors

August 6, 2018

The FDA has approved the injectable drug Azedra for rare cancers of the adrenal glands — the first ever non-surgical therapy OK’d for these tumors. Azedra (iobenguane I131) is a radiotherapy drug that attacks tumors with a high, specifically targeted dose. It’s designed to treat adults and children (12 and older) with inoperable locally advanced or metastic 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 researchers gave 68 patients in a trial at least one therapeutic dose of Azedra. The results: 25 percent who received at least one dose and 32 percent of those who received two doses saw their blood pressure drop enough to cut their hypertension meds in half. “This is a true breakthrough. Until today, there were no anti-tumor therapies available for patients with these tumors who were not candidates for surgery,” said principal investigator Daniel Pryma, an associate professor of Radiology and Radiation Oncology. “This therapy not only controls the tumors but also the debilitating symptoms caused by their excess hormone production, meaning it provides dual benefit to patients,“ added Pryma, also chief of Penn’s Nuclear Medicine and Clinical Molecular Imaging. Reported side effects include low white blood cell and platelet counts, fatigue and anemia. The FDA gave Azedra an Orphan Drug designation, Fast Track status and Breakthrough Therapy designation in the U.S. Its license is held by Progenics Pharmaceuticals.