At a time when uncertainty again swirls around the development of Alzheimer’s disease drugs, research continues in new ways. Through an alliance this morning, Voyager Therapeutics will team with AbbVie to try to use gene therapy tools to help attack the disease with a potential one-time, long-lasting treatment.
Voyager (NASDAQ: VYGR) gets $69 million in cash up front from AbbVie (NYSE: ABBV) in the deal. Voyager will develop adeno-associated viruses—gene therapy delivery tools known as viral vectors—that shuttle genetic instructions into the brain to help treat Alzheimer’s and other neurodegenerative diseases.
AbbVie will decide whether to license one or more of these therapies after Phase 1 testing. These drugs are gene therapies meant to instruct the body to destroy clumps of tau, a misfolded protein implicated in the buildup of brain plaques in patients with Alzheimer’s and other neurodegenerative diseases. AbbVie mentioned progressive supranuclear palsy and frontotemporal dementia as additional possible diseases to treat.
Developing drugs for Alzheimer’s is notoriously difficult for a variety of reasons, and recent months have provided fresh reminders of how daunting the task can be. Since last September, drugs from Axovant Sciences (NASDAQ: AXON), Biogen (NASDAQ: BIIB) and Eisai, and Merck (NYSE: MRK) have all failed human trials, adding to an increasingly long list of clinical failures for a disease that hasn’t seen a new treatment in well over a decade. One of the remaining therapies in late-stage testing is Biogen’s aducanumb, with data expected next year from a pair of closely watched Phase 3 trials. (Biogen gave investors jitters last week when it added more than 500 patients to the two studies, citing “variability” in early, blinded data from the trials. CEO Michel Vounatsos called the ensuing 7 percent stock sell-off an “overreaction” in this Wall Street Journal report.)
Several drugs that failed clinical trials are antibodies that target the buildup of amyloid or tau. AbbVie believes one of the reasons these approaches fail is only small amounts of drug are getting to the brain. AbbVie hopes to solve this problem by using a gene therapy approach from Voyager. An AAV virus infused into the body would head to the brain and deliver genetic instructions to produce antibodies that target tau. These so-called vectorized antibodies could be administered via a one-time treatment, AbbVie says. (A Voyager spokesperson said these therapies would be given through intravenous infusions, unlike the experimental Voyager gene therapy for Parkinson’s that is delivered straight into the brain.)
Multiple gene therapies, which offer long-lasting, if not permanent treatments for disease, are now approved in the U.S. and Europe, though none for neurological diseases as of yet. Voyager’s Parkinson’s treatment has shown promise in early testing. It also has an alliance with Sanofi on preclinical gene therapies for Friedreich’s ataxia and Huntington’s disease.
“Voyager’s [technology] presents an innovative approach to addressing challenges in treating neurological disorders associated with the administration of biologic therapies,” said Jim Sullivan, AbbVie’s vice president of pharmaceutical discovery, in a statement.
Voyager could also get up to $155 million in additional payments through Phase 1 testing, and as much as $895 million per program in downstream payments that AbbVie licenses.