Abbott, BMS, GSK, Janssen, and Sanofi have joined Pfizer, AstraZeneca, and Lilly.
NIH’s National Center for Advancing Translational Sciences (NCATS) has more than doubled its roster of compounds available for repurposing by researchers under its agency-industry Discovering New Therapeutic Uses for Existing Molecules pilot program. The increase in available compounds—to 58 from some two dozen when the program was announced on May 3—occurred after five additional pharmaceutical companies joined the pilot program.
Abbott, Bristol-Myers Squibb, GlaxoSmithKline, Johnson & Johnson’s Janssen Pharmaceutical Research & Development, and Sanofi have joined Pfizer, AstraZeneca, and Eli Lilly in contributing compounds to the agency-industry initiative. The program is aimed at developing new indications for shelved compounds that have already been tested in humans, with acceptable safety profiles in dosing, pharmacologics, and toxicity, but failed to show efficacy or were abandoned for business reasons.
A glance of the roster of compounds finds nine compounds with 10 indications for various forms of arthritis, nine compounds with nine pain indications, seven against chronic obstructive pulmonary disease, six for diabetes treatment, five each against asthma, depression, and schizophrenia, four against anxiety, three for allergic rhinitis and oncology indications, and two each against Alzheimer disease, Gastroesophageal reflux disease, and tumors.
“Each company participating in this innovative collaboration has made substantial research and development investments to advance these compounds to the point where they can be used in clinical studies,” Kathy L. Hudson, Ph.D., NCATS acting deputy director, said.
NCATS also published online a pre-application notice for researchers seeking funding as well as two funding announcements: RFA-TR-12-004 covering Stage 1 (Feasibility)/Stage 2 (Implementation) Cooperative Agreements and RFA-TR-12-005 covering partners with Phase II Exploratory/Developmental Cooperative Agreements. NCATS will make available for the pilot program up to $20 million in the 2013 federal fiscal year toward two- to three-year staged, cooperative agreement research grants. The center envisions issuing eight awards during the pilot program’s first year, with announcements of the first winners expected in April or May 2013.
NCATS has established a two-stage process. Interested researchers must complete pre-applications subject to NIH peer review by August 14. Applicants must include details about how they would explore specific hypotheses related to one of the compounds that might be useful in a specific disease area, according to NCATS. Researchers whose applications pass muster must then complete an application showing that they have access to the compound in question, through the signing of a collaborative research agreement.
To speed up the start of research, NCATS has developed template agreements: Memoranda of understanding will govern NIH’s dealings with drug developers, which will sign collaborative research agreements with researchers, who in turn will be expected to sign confidential disclosure agreements with the companies.
The Therapeutics Discovery program drew a lukewarm response in the Corante blog from Derek Lowe, Ph.D. While wishing NCATS luck, he added: “I’ve no idea what the chances for success might be. On the one hand, having a compound that’s passed all the preclinical stages of development and has then been into humans is no small thing. On that ever-present other hand, though, randomly throwing these compounds against unrelated diseases is unlikely to give you anything (there aren’t enough of them to do that),” Dr. Lowe opined.“My best guess is that they have a shot in closely related disease fields, but then again, testing widely might show us that there are diseases that we didn’t realized were related to each other.”
John L. LaMattina, Ph.D., a former Pfizer R&D president and current senior partner in Pure Tech Ventures, holds a dimmer view. Writing in his Forbes blog, Dr. LaMattina said the Therapeutics Discovery pilot program wasn’t the best use of funds given NIH’s already-tight budget. “Is this really the mission of the NIH? With funding so tight, is this the proper use of its resources? I don’t think so,” Dr. LaMattina said.
“These funds would be better used to support the new generation of biomedical researchers who are going to lay the groundwork for discovering the next wave of treatments for Alzheimer’s disease, cancer, and a host of other diseases” Dr. LaMattina added. “The NIH should let industry do the applied R&D for drug discovery and focus its resources on the crucial basic research that is desperately needed.” However, Dr. LaMattina added, “I do hope that people can have success in this type of work. But I believe successes are going to be rare.”
NCATS and NIH have defended the program as helping fulfill the less-than-year-old translational sciences agency’s purpose of accelerating the nation’s drug development effort through strong, innovative, and strategic partnerships across government, academia, and industry. “Clearly, we need to speed the pace at which we are turning discoveries into better health outcomes,” NIH Director Francis S. Collins, M.D., Ph.D., said in a May 3 statement.