This summer, the National Institutes of Health (NIH) announced another funding opportunity for experimental drugs proven safe but sitting on the shelves of pharmaceutical companies. The NIH-sponsored effort is based on a crowdsourcing strategy to establish collaborations between industrial and academic partners to test such drugs.
An initial programme run by the National Centre for Advancing Translational Sciences (NCATS) proved successful, with nine repurposing projects funded to the tune of $12.7m in June 2013.
“The pharmaceutical companies have these well-developed assets. These are agents that are ready to go to Phase IIa trials, so at least Phase I, and in many cases Phase II or in some cases Phase III trials have been completed,” Christine Colvis at NCATS told us. “But they either failed to show efficacy or they were deprioritized for business reasons.”
A limited amount of company-provided information was provided for each agent by NCATS, along with funding opportunity announcements. Companies looking to hook-up their agents with new repurposing ideas included AstraZeneca, Eli Lilly, BMS, Pfizer, AbbVie, GSK, Janssen and Sanofi.
Academic researchers or small pharma or biotech companies could then propose ideas for use of those assets, so no previous relationship was necessary with the companies. “It is very much driven by the science,” said Colvis. “We were looking for new ideas that the companies wouldn’t have thought of, that would either be a niche area or such a new breakthrough that is not something the companies had caught wind of, at least at the time the agent was under active development.”
Modification of the agent is not an option, either during the previous round or during the new round, which made 12 agents with pediatric indications available (there is a little leeway for pediatrics). The restriction on reformulation is there so that investigators can jump onto the development ramp at an advanced point. “During these early days, we want to keep things as simple as we can and give ourselves the opportunity of starting as far down the pipeline as possible,” explains Colvis, who wrote about the program in a recent review in Drug Repurposing, Rescue and Repositioning .
Hermann Mucke of Pharma Consultancy in Vienna, Austria, and editor of this journal, said everyone stands to benefit. “Large pharma companies because they can tap into the inventiveness and agility of small companies and academia; these it turn would get funding and perhaps undisclosed background data on the compounds; and society would benefit from getting new treatments faster. Even regulators would benefit: in many cases they will already have data on the compounds on file.”
However, he believes reformulation should be considered in future programs. “Frequently repurposing will be difficult or impossible without changing the route of administration,” he explains. “Also, if the compound is marketed, developers will want to differentiate against the older formulation to limit off-label prescription, and to create patentable intellectual property. However, in many cases re-formulation is simply a pharmaceutical necessity.”
Mucke said such a scheme could be introduced and managed though the EU’s multilateral projects for the life sciences. “It would be much more complicated than in the US, but it could be adopted into umbrella programs such as the current Horizon 2020."
Colvis said template agreements were used by the investigators and companies and says the company would retain composition IP, but investigators would potentially have use IP, though NCATS is not party to these agreements.