NIH backs pharma giants and academics' projects to revive R&D castoffs

The NIH has advanced an ongoing effort to give castoffs from drug pipelines a new lease on development, committing $12.7 million to fund 9 collaborations between academic and pharma groups to find new treatments in areas of unmet need such as Alzheimer's disease, Duchenne muscular dystrophy and schizophrenia.

The pilot program meets at the intersection of major priorities of NIH Director Dr. Francis Collins: Repurposing pharma compounds as well as translating clinical and lab insights into badly needed therapies. His agency's initiative, dubbed Discovering New Therapeutic Uses for Existing Molecules, could shave significant time off the average 13-year period spent from discovery of a disease target to gaining approval for a drug against that target. It also aims to reduce the 95% or more failure rate of such programs.

"This unique collaboration between academia and industry holds the promise of trimming years from the long and expensive process of drug development," Collins said in a statement.

Rather than starting from scratch, the program's researchers get access to pharma compounds that have already undergone safety studies in humans. With an established safety profile for the compounds, the collaborators have a shot to develop them faster than normally possible. That's the hope, anyway. The FDA has not approved any of the experimental compounds, which drugmakers had sidelined because of business reasons or lack of efficacy in their originally intended use, said Dr. Christine Colvis, a program director for new therapeutic uses at the NIH. 

Big Pharma companies have scrambled to find ways to make drug development faster and cheaper, without much success.

Eight large drug companies have signed on for the pilot phase of the NIH's existing-drug research initiative, including AbbVie ($ABBV), AstraZeneca ($AZN), Bristol-Myers Squibb ($BMY), Eli Lilly ($LLY), GlaxoSmithKline ($GSK), Johnson & Johnson ($JNJ) Janssen Research & Development, Pfizer ($PFE) and Sanofi ($SNY).

Pfizer, for example, has joined forces with Dr. John Krystal of Yale University under the NIH-funded initiative to research GlyT1 inhibition for treating cognitive impairment in schizophrenics. Also, Sanofi is collaborating on a program for the muscle-wasting disorder Duchenne muscular dystrophy with investigators from the University of Washington in Seattle and Baltimore's Kennedy Krieger Institute. And AstraZeneca has teamed up with Yale researchers to study a compound called AZD0530 as a Fyn kinase inhibitor for treating Alzheimer's disease.

In all, the program focuses on 8 disease areas where vast divides lie between the needs of patients and available therapies. The NIH has agreed to support the collaborations for up to three years, giving the industry-academic teams time to complete Phase IIa trials to gain initial signs of efficacy. The NIH would then step aside and leave funding of late-stage trials up to the collaborators, Colvis said.

If the proof-of-concept studies are successful "we're hoping that the companies are going to be interested enough… that they would carry it into Phase III trials," Colvis told FierceBiotech in an interview. "But if they won't, it should be in the agreement with the [academic] investigator that the investigator can license from the company… and work with a third party to do further development."

This program is itself part of Collins' experiment at the NIH, which is known for funding a lot of basic research, to see how well the agency can support cost-effective drug development. In a few years, there could be some interesting results.

- here's the release
- check out Nature's article