SAN FRANCISCO—In late April, Bay Area protein therapeutics and discovery company FivePrime Therapeutics Inc. inked a two-year research and license deal with Boehringer Ingelheim focused on the discovery of novel therapeutics in the area of rheumatoid arthritis. The multi-product deal, worth a potential $75 million to Five Prime, will see it screen thousand of proteins for specific cellular activity.
Under the terms of the deal, FivePrime will receive upfront cash and research support, with Boehringer Ingelheim securing exclusive worldwide rights to develop and commercialize products and targets discovered. Five Prime will also receive future milestones and royalties of any targets or therapeutics.
Important to completing the deal with Boehringer was FivePrime's protein screening platform, according to company founder and executive chairman Rusty Williams. "The keys features for us to get this kind of deal are our ability to make thousands of proteins each week that are highly curated--so we know how much protein is in each well--and then the automation for protein screening we apply that is more like what a small molecule company would do."
The screening library Five Prime applies contains essentially all human secreted proteins and their receptors, according to company information, and includes a broad number of proteins that are unavailable in public collections.
For BI, Five Prime will be taking a hypothesis of what kinds of proteins may show activity against rheumatoid arthritis, as well as looking for specific kinds of protein activity. Five Prime will then use the hypothesis to design assays to screen for these activities.
"We believe that the combined capabilities of FivePrime and Boehringer Ingelheim will offer new opportunities to discover important disease targets and develop novel drug candidates," says Michael Dolsten, executive VP of pharma research of Boehringer Ingelheim, in a press release announcing the collaboration.
While in this particular case FivePrime is working with a collaborator to develop protein leads in a specific therapeutic area, the company is actively engaged in its own internal efforts to discover and develop therapies in a broad range of other areas.
"When I founded the company, I figured that big pharma had the small molecule space pretty well covered," says Williams. "So this was one area where there appeared to be opportunity.
"Our platform lends itself to working in multiple therapeutic areas. Our lead program is in cancer and we also have a program in diabetes and metabolic disease and also a number of cardiovascular programs. We plan to build a robust pipeline in a number of different areas and anticipate receiving IND for our lead candidate some time next year."
With a highly automated operation from protein production through the screening process, Williams believe FivePrime is the first company to apply such a high level of automation in the screening of potential protein-based therapeutics.
One hurdle the company had to overcome to make its high-throughput screening platform valid, was to minimize the drop out rate of proteins through the multistep process of creating proteins from a cDNA clone through to functioning protein.
"Each step has a drop out rate and the effect is cumulative or multiplicative," Williams notes. "We have been able to minimize the drop out rate in each step, so in the end you have an assay that delivers multiple hits."
While getting hits on a particular assay is by no means guaranteed, Williams notes that typical hit rates per assay average around one percent. "Our experience, having gone through a number of therapeutic areas, is that we always have at least some hits," he says.
