NCATS' Therapeutics Discovery program gains more industry collaborators

Nearly 60 compounds from industry collaborators now publicly available

Five additional pharmaceutical companies have joined a National Institutes of Health-led effort to help scientists research promising new treatments for patients. Funding and molecular compound information is available now for the initial phase of the recently unveiled Discovering New Therapeutic Uses for Existing Molecules program. This NIH-industry collaboration will match researchers with 58 compounds to test ideas for new therapeutic uses. Since the launch of the program last month, the total number of compounds the companies are making available has more than doubled.

Abbott, Bristol-Myers Squibb Company, GlaxoSmithKline, Janssen Pharmaceutical Research & Development, L.L.C., and Sanofi have joined Pfizer, AstraZeneca, and Eli Lilly and Company in this innovative approach to research.

The NIH's new National Center for Advancing Translational Sciences (NCATS) created the Therapeutics Discovery program to help re-engineer the research pipeline. By crowdsourcing compounds that already have cleared several key steps in the development process, including safety testing in humans, scientists nationwide have the opportunity to contribute their expertise to advancing these resources for new disease therapies.

The eight participating companies will provide their compounds and related data, which were determined by the NIH to meet specific eligibility criteria. For example, each compound must have advanced to clinical studies but been unsuccessful in its original therapeutic indication or not pursued for business reasons. Preliminary information about the compounds, including mechanism of action, route of administration, and any limitations in use based on safety and tolerability, are available at http://ncats.nih.gov/therapeutics-directory.html.

"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," said Kathy L. Hudson, Ph.D., NCATS acting deputy director. "If researchers funded through this effort can demonstrate new uses for the compounds, they could significantly reduce the amount of time it takes to get a treatment to patients in need."

For the pilot phase of the program, in fiscal year 2013, NCATS will provide up to $20 million to fund two- to three-year staged, cooperative agreement research grants. If specific milestones are met, funded researchers will conduct pre-clinical validation and clinical feasibility studies in the first stage, and proof-of-concept clinical trials in the second stage, to test whether one of the compounds may be effective against a previously unexplored disease target. The pilot phase also is intended to test the utility of the newly created template agreements by reducing the negotiation time that otherwise could delay the research.

Researchers who are interested in NCATS' Therapeutics Discovery funding must submit a pre-application in response to the NIH Funding Opportunity Announcement at http://grants.nih.gov/grants/guide/pa-files/PAR-12-203 by Aug. 14, 2012. 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. The pre-applications will undergo review by external experts, and while no funding will be provided at this stage, successful applicants will be notified to submit a full application for fiscal year 2013 funding.