SAN DIEGO – A team of top researchers at UC San Diego landed a $6.9 million grant from the NIH’s National Institute on Aging to advance an Alzheimer’s disease drug that was effective in restoring brain health in mice models. The drug – CNDR-51997 – was discovered in a joint program with researchers at the University of Pennsylvania, who will share the funding.
Professor
Skaggs School of Pharmacy & Pharmaceutical Sciences
University of California San Diego
“The NIH has supported this program since its early discovery stages,” Lead UC San Diego Researcher Carlo Ballatore, Ph.D. told the San Diego Business Journal. “This ongoing support has been crucial to the program’s development and reflects the consistent enthusiasm grant reviewers have had for our strategy.” The latest award marks the team’s third NIH grant.
The researchers’ long-term goal is to prepare the drug ready for trials in anticipation for human use. By the end of the the three-year grant period, researchers hope to submit an Investigational New Drug (IND) application to the Food and Drug Administration (FDA). If the FDA approves it, it paves the way for Phase 1 clinical studies.
It’s still unclear who would reap the benefits of an IND approval. Questions remain as to whether the two universities would file the IND, or whether the rights would be licensed to another company.
“As of now, we have not licensed the program to a company,” shared Dr. Ballatore. “If a license agreement were completed before the end of the IND-enabling studies, it is possible that the IND would be filed by the licensee. Otherwise, the [Principal Investigators] of the grant will file the IND.”
The objective is ultimately to license the program, but there’s enough grant money to get researchers to the IND stage. “[It would] provide considerable additional validation and value to the program. Any license would be through UCSD and the University of Pennsylvania, so these institutions would benefit financially upon completion of a license.”
Taking a Tau Approach
Patients with Alzheimer’s disease often have so-called amyloid plaques and tau tangles – abnormal structures inside brain cells. Most experts think they somehow play a critical role in disrupting the communication between nerve cells, according to the Alzheimer’s Association. Dr. Ballatore and his team have discovered a staggering reduction in both plaques and tangles in mouse models – about 50% less.
Current medications on the market have been designed to target plaques. This July, the FDA approved Kisunla from Eli Lilly and Company (NYSE: LLY) to help slow cognitive and functional decline. Last year, the FDA cleared Leqembi from Biogen (NYSE: BIIB). Yet there’s no FDA-cleared medication that attacks the brain’s tangles, which are directly correlated to cognitive decline.
UC San Diego researchers aim to change that by honing in on tau tangles. “This compound has been designed to combat tau-mediated neurodegeneration and our preclinical data suggest that it could be beneficial for the treatment of Alzheimer’s and related dementias,” shared Dr. Ballatore. “Because tau pathology increases during the symptomatic stage of [the disease] and likely is the cause of the cognitive decline, our hope would be that [the drug] could have a meaningful impact on tau pathological burden and patient outcomes if administered to patients with early signs of cognitive decline (e.g., mild cognitive impairment).”
Director, Drug Discovery
Center for Neurodegenerative Disease Research
University of Pennsylvania
Dr. Ballatore explained that drugmakers Bristol Myers Squibb and Cortice Biosciences are developing similarly acting drug compounds. “However, both studies were largely inconclusive due to the short duration of treatment (i.e., 9 weeks) which is considerably shorter than the typical 24-month {Alzheimer’s disease] trials.”
There’s also hope that UCSD and U of Penn’s drug may treat myriad other brain-related diseases. “[Our findings] suggest that the compound holds considerable promise for Alzheimer’s disease. However, there is also a great unmet need for disease-modifying drugs for the other tauopathies,” stated U of Penn’s Kurt Brunden, Ph.D., research professor in the Perelman School of Medicine and director of drug discovery at Penn’s Center for Neurodegenerative Disease Research.
The drug is still far from reaching the commercial market, but researchers continue forging forward following the promising initial results. “Assuming all goes as planned, and assuming that a suitable licensee is identified with the means to support the next stages of clinical development (Phase I, II and III), it is possible that the new drug may become available in approximately 10 years,” added Dr. Ballatore.
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