WASHINGTON, D.C.—The National Institutes of Health (NIH), 10 biopharmaceutical companies and several non-profit organizations have launched an unprecedented partnership to transform the current model for identifying and validating the most promising biological targets of disease for new diagnostics and drug development.
The Accelerating Medicines Partnership (AMP) aims to distinguish biological targets of disease most likely to respond to new therapies and characterize their biomarkers. Through the Foundation for the NIH (FNIH), AMP partners will invest more than $230 million over five years in the first projects, which focus on Alzheimer’s disease, type 2 diabetes and the autoimmune disorders rheumatoid arthritis and systemic lupus erythematosus.
A critical and groundbreaking element of the partnership is the agreement that the data and analyses generated will be made publicly available to the broad biomedical community. The three- to five-year, milestone-driven pilot projects in these disease areas could set the stage for broadening AMP to other diseases and conditions.
In his blog on the NIH website, NIH Director Dr. Francis S. Collins noted that lack of efficacy currently accounts for more than half of all drug failures in Phase 2 clinical studies. “If AMP’s target validation efforts improve efficacy by 90 percent,” he said, “the success rate will rise significantly.”
Collins continued: “It would seem like there’s never been a better time for drug development. Recent advances in genomics, proteomics, imaging and other technologies have led to the discovery of more than a thousand risk factors for common diseases—biological changes that ought to hold promise as targets for drugs.
“But this deluge of new opportunities has to be put in context: drug development is a terribly difficult business, he added. “To the dismay of researchers, drug companies and patients alike, the vast majority of drugs entering the development pipeline fall by the wayside … Why is this happening? One major reason is that we’re not selecting the right biological changes to target from the start…
“So I was thrilled to help launch the Accelerating Medicines Partnership. This unprecedented public-private effort will strive to identify better targets so we can develop better drugs at a faster pace. Besides NIH and Foundation for the NIH, the partners include 10 biopharmaceutical firms and a number of non-profits, including patient advocacy groups…
“Let me share an example of how this approach can lead to success: the recent identification of a drug target called PCSK9. Researchers were tuned in to this target by the discovery that people with a rare gene variant that greatly reduces production of the PCSK9 protein have very low levels of cholesterol, along with a dramatically decreased risk of heart disease. After some additional studies to validate that PCSK9 is a solid target, a half-dozen pharmaceutical firms are now racing to develop drugs that lower cholesterol by blocking this protein.
“I think there are a whole lot more PCSK9’s out there waiting to be discovered. We just need to assemble the right teams to help us find the right targets—and AMP has the right stuff to do that,” Collins wrote.
How will AMP work? A spokesperson at Eli Lilly shared some thoughts about that with DDNews.
Although the structure of the Lilly team is still in the process of being finalized, the company will be involved with two of the three AMP initiatives: Alzheimer’s disease and type 2 diabetes. The Alzheimer’s disease arm of the AMP initiative will be managed by an Alzheimer’s disease steering committee, comprising representatives from NIH, FNIH, the U.S. Food and Drug Administration (FDA) and participating companies and patient advocacy organizations. After AMP research grant awards are made, investigators carrying out the research will be added to the disease steering committees. The steering committee will operate under the direction of the overall AMP executive committee, which includes representatives from NIH, participating industry partners, FDA and patient advocacy organizations. The steering committee is responsible for reporting project plans and milestones to the executive committee for review and approval and will meet on a regular basis to assess project progress. The steering committee will be similar in structure and representation for type 2 diabetes.
Lilly is contributing resources, including funding, scientific expertise and biomarkers. In the area of Alzheimer’s disease, for example, Lilly is contributing both its amyloid and tau imaging agents.
Dr. Jan Lundberg, Lilly’s chief scientific officer, will serve on the executive committee. Lilly will also participate on the steering committees on the Alzheimer’s disease and Type 2 Diabetes initiatives.
“We look forward to our top scientists working side by side with academia. The design of these projects best leverages our collective expertise, network and resources with the ultimate goal of improved disease deciphering,” the Lilly spokesperson stated.
Who’s in the partnership?
AMP participants include government, biopharmas and non-profits
Johnson & Johnson
American Diabetes Association
Lupus Foundation of America
Foundation for the NIH
Geoffrey Beene Foundation
Rheumatology Research Foundation