NCATS Announces Funding Opportunities to Repurpose Drug Candidates from Industry

NCATS now is accepting applications for its Discovering New Therapeutic Uses for Existing Molecules (New Therapeutic Uses) program. Today, NCATS issued funding announcements and provided information about partially developed therapeutic candidates — referred to as “agents” — for researchers to crowdsource ideas for new uses. Launched in 2012 as a pilot initiative, New Therapeutic Uses matches researchers with a selection of pharmaceutical industry agents to explore new treatments for patients.

For the new funding opportunities, NCATS is collaborating with AstraZeneca, Janssen Research & Development, L.L.C., Pfizer Inc. and Sanofi to make 26 agents available. For the first time, the participating pharmaceutical companies have made available agents that are suitable for exploring pediatric indications. For those agents, NCATS will provide an extra year of support — as compared to agents for adult indications — to complete additional studies evaluating safety, dosage and side effects in healthy volunteers, as well as juvenile toxicity studies, which are required before pediatric clinical trials can begin.

The New Therapeutic Uses program is a breakthrough approach to speeding new therapies to patients in need.  Since the failure rate of new therapeutic development is more than 95 percent, many candidate agents exist at pharmaceutical companies that have cleared many key steps in the drug development process, including safety testing in humans. Repurposing these agents, which have known human safety profiles, for a new use offers the potential for rapid completion of development and ultimate regulatory approval.

Diagram of identifying new therapies for patients through (1) pharma, which create drugs, provide agents, sign agreements with researchers and form alliances with NIH/NCATS; (2) researchers, who provide new therapeutic use ideas, access patient populations, conduct clinical trials, sign agreements with pharma and receive funding from NIH/NCATS; and (3) NIH/NCATS, which posts agent information, develops agreement templates, crowdsources ideas, provides funding to researchers and forms alliances with pharma.


“New Therapeutic Uses addresses the common problem of failure in therapeutic development by turning it into an opportunity for the ‘wisdom of the crowd’ to suggest new conditions that candidate agents might treat,” said NCATS Director Christopher P. Austin, M.D. “By working together with our industry and academic partners to further develop these agents, we can meet the goal of speeding treatments to patients in need.”

New Therapeutic Uses accelerates these partnerships through innovative template agreements designed to streamline the legal and administrative process for research collaboration across organizations. During the New Therapeutic Uses pilot phase, these agreements reduced the time required to establish collaborations between industry and academia to about three months from the more typical 9 months to a year.

In June 2013 as part of the pilot, NIH awarded $12.7 million to nine academic research groups for projects to explore new treatments for patients in eight disease areas, including two rare diseases: Duchenne muscular dystrophy and the progressive lung disease lymphangioleiomyomatosis. Within three months, one third of the project investigators were testing compounds in humans for new uses, including potential treatments for schizophrenia (two agents) and Alzheimer’s disease.

“The progress we have made on our collaborative projects is remarkable given the complexities of initiating clinical trials,” said AstraZeneca Vice President of Emerging Innovations, Scientific Partnering and Alliances Don Frail. “The knowledge of the investigators, together with the tools, resources and expertise made available by our company, allowed the projects to move forward very quickly, and quality preclinical and/or clinical data have already emerged.”

Interested researchers can submit a pre-application by July 15, 2014, in response to the funding announcements on NCATS’ New Therapeutics Funding Information page. Applicants must describe how they would explore specific hypotheses for an agent available through the program and how it might be used in a specific disease. Pre-applications will undergo NIH review, and successful applicants will be placed in contact with the pharmaceutical company providing the agent to discuss the submission of an application for funding that would begin in July 2015. In addition to NCATS, multiple NIH Institutes and Centers and the Food and Drug Administration’s Office of Orphan Products Development are contributing funding for these announcements.

A technical assistance teleconference will be held on May 29, 2014, from 1:00 to 2:30 p.m. ET. All prospective applicants are invited to participate. Participants are encouraged to register via the NCATS Events Web page, and to submit questions on or before May 27 to be addressed during the teleconference.


Posted May 2014