A pilot program unveiled recently at National Institutes of Health (NIH) will test a new approach to cut the development time for drug compounds, starting with molecules tested for one disease, but potentially applicable to others. On 12 June, NIH said five more pharma manufacturers signed up, making a total of eight companies in the program, with 58 compounds registered for review.
The program, Discovering New Therapeutic Uses for Existing Molecules, called Therapeutics Discovery for short, enlists drug manufacturers to furnish compounds and biologics they tested to treat one or more diseases, but have not taken to market. The drugs must have passed human safety testing -- usually conducted in phase 1 clinical trials -- but did not achieve their efficacy goals or were not considered commercially viable.
In early May, Pfizer (NYSE:PFE), AstraZeneca (NYSE:AZN), and Eli Lilly and Company (NYSE:LLY) signed on as the first companies to offer up candidates that meet these conditions. On 12 June, pharma makers Abbott Laboratories (NYSE:ABT), Bristol-Myers Squibb (NYSE:BMY), GlaxoSmithKline (NYSE:GSK), Janssen Research & Development division of Johnson & Johnson (NYSE:JNJ), and Sanofi (NYSE:SNY) added their compounds and biologics to the registry.
The registry, as of mid-June, has 58 candidates for further testing. The molecules registered had initial disease targets ranging from generalized conditions, such as pain and obesity, to more specialized indications, such as migraine or obesity in type 2 diabetes. Several compounds were tested for psychiatric disorders, such as depression or schizophrenia. A number of the drugs already list multiple conditions as targets.
Tapping the wisdom of crowds
NIH will use something of a crowd-sourcing approach to further develop these candidate molecules. For the pilot stage of the Therapeutics Discovery program, NIH has allocated $20 million though the National Center for Advancing Translational Sciences to fund two- to three-year cooperative agreement research contracts with academic and other researchers who propose new uses for these compounds.
Researchers interested in proposing further examination of these compounds and biologics will first submit pre-application proposals by 14 August 2012. Letters of intent, while not mandatory, are encouraged, and due by 14 July.
NIH panels will review the pre-applications and decide on finalists, who will then compete for the funds. Researchers receiving these awards will first conduct preclinical, validation, and clinical feasibility studies, and later, proof-of-concept clinical trials to test if the compounds are effective against unexplored disease targets.
A new wrinkle in this project, and one potentially applicable to other research initiatives, is a series of template agreements NIH has negotiated with the participating drug companies, to cut the ramp-up time needed by the researchers. The template agreements spell out in advance intellectual property and administrative processes each company requires.
The template agreements thus make it possible for researchers awarded funds for further testing to already know the ground rules for partnering with each company, and if needed can negotiate from there. In general, says NIH, the drug companies will retain the ownership of their compounds, while academic research partners will own any intellectual property they discover through the research project, with the right to publish the results of their work.
While this pilot program has promise, it is still a pilot program, with many questions still unresolved, such as the level of participation from the research community and the amount of time saved in the drug development process to get new therapies to market.
Nonetheless, the industry -- broadly defined to include NIH, FDA, and academic researchers, as well as biotechnology and drug companies -- face continuing pressure to cut the exorbitant time and expense needed to bring new drugs to market, while ensuring patient safety. The Therapeutics Discovery project is one more step in that direction.