The Alzheimer’s Drug Discovery Foundation’s venture philanthropy model has a singular mission: to invest money and resources in bold ideas that can speed development of drugs for Alzheimer’s. Our impact has been significant. The ADDF has invested in 20% of the drugs currently in clinical development for Alzheimer’s disease.
We concentrate our investments in early clinical stage trials, as well as preclinical drug discovery research. This underfunded area is often called the “valley of death” because it is where “good ideas go to die.” Drug development is expensive, and researchers struggle to get early ideas funded. Failure is frequent, if not the norm. Our funding often helps attract other early investors and serves as a bridge to the point where the research has advanced enough to attract financial support from the pharmaceutical industry, government agencies like the National Institutes of Health, investors, and other major partners.
Our due diligence process seeks out ideas that have great promise of success, but we don’t shy away from the risk that comes with early investing. Like all investors, success turns our risks into financial rewards. But in the ADDF venture philanthropy model, those rewards are not our profit, they’re directly reinvested into additional cutting-edge research.
That’s why all of us at the ADDF are doubly excited when our investments pay off. First, it means a drug we have supported is moving into the next exciting phase of development bringing it that much closer to helping patients with Alzheimer’s and their families. Second, we have more money available to boost the next great idea.
I want to share two recent examples with you, one academic investment and the other in a biotech company.
Vanderbilt University recently signed a license agreement with Acadia Pharmaceutical to develop and commercialize novel drug candidates that Vanderbilt has been researching, with ADDF support, since 2014. The molecules Vanderbilt is developing, and hoping to bring to market with its new commercial partner, hold great promise for Alzheimer’s. They activate muscarinic M1 receptors, which help process signals that cross the synapses in our brains, affecting memory and cognition. We have long thought these drugs could be effective in treating Alzheimer’s and other CNS disorders, but up until now, they have caused intolerable side effects. Principal investigator Jerri Rook, PhD of Vanderbilt’s Warren Center for Neuroscience Drug Discovery has developed molecules that appear to have the desired treatment benefits without the unwanted serious side effects.
Drug discovery and development, plus commercialization — the work of bringing them to market — is a long and complex process that requires not only funding, but the specialized expertise of many professionals. The ADDF also provides in-kind support through our partnership with the Harrington Discovery Institute University Hospitals in Cleveland, Ohio. Through our partnership with the Harrington Discovery Institute University Hospitals in Cleveland, Ohio, the ADDF is able to ensure access to in-kind support for our awardees.
Dr. Rook received the 2015 ADDF-Harrington Scholar Award, which provided her with strategic consulting and management support from experienced drug development professionals, as well as regulatory, intellectual property and business development assistance. Recently, the investigations led to a $515 million licensing agreement with the biotechnology company Acadia Pharmaceuticals to further develop and hopefully commercialize their drug.
Many amazing ideas come from researchers in academia, but biotech companies are also a rich source of researcher innovation. Tetra Therapeutics, another of the ADDF’s long-standing investments, recently announced an agreement to be acquired by Shionogi, a major research-driven pharmaceutical company, for $500 million in milestone payments. Tetra developed a novel therapeutic agent that enhances early and late stages of memory formation. The experimental compound, called BPN14770, inhibits production of an enzyme called PDE4D that starts a cascade of events resulting in impairment of signals moving across synapses in the brain. By inhibiting this enzyme, BPN14770 protects these synaptic connections, which otherwise are lost in patients with Alzheimer’s.
The ADDF has already reaped returns for advancing Tetra drugs into phase 2/3 clinical trials. As the financial disclaimer goes, past performance may not be an indicator of future returns, but in the case of Tetra, we tripled our initial investment.
In another example, the ADDF continues to receive returns today on an investment made starting in 2002 at the University of Pennsylvania for development of a PET amyloid scan. The Amyvid® PET scan was approved by the FDA in 2012 and is now used to estimate the density of beta-amyloid plaques in the brain, a hallmark indicator of Alzheimer’s disease. This first-of-its-kind biomarker test has been invaluable in clinical trials, such as Biogen’s investigational treatment for people with Alzheimer’s, aducanumab, which the company recently submitted to the FDA for U.S. approval.
But as I said earlier, our model is not about financial windfalls, it’s about advancing the research so we can get effective treatments into the hands of patients and their families. These financial returns reflect scientific success towards the final approval and patient access to a new drug. Today, the ADDF watches with much enthusiasm as Tetra and Vanderbilt take the next bold steps toward their futures. We will eagerly anticipate more positive news as they seek to improve the outcomes for men and women living with Alzheimer’s. As we wish them well, we turn our attention to finding the next exciting research idea and look forward to taking our next risk.