Merck just dashed the last remaining hopes for its industry-leading BACE drug for Alzheimer’s

The Alzheimer’s field  is keeping its perfect record for failure in pivotal Phase III studies.

Merck today scrapped the last remaining Phase III study of its leading BACE therapy verubecestat, the second of two collapsed studies, after independent monitors concluded that the drug wasn’t working for early-stage, prodromal patients.

Roger Perlmutter

Back in February Merck signaled its lack of success with the drug, which moves upstream to reduce the production of amyloid beta, when it punted its Phase III trial in mild-to-moderate patients.

Once one of Merck’s top late-stage drugs in the pipeline, its failure here will likely cast a dark shadow over other BACE drugs, including the one that Eli Lilly obtained from AstraZeneca as the UK giant backed out of CNS disease R&D.

Merck had little to say in detail Tuesday evening. A spokesperson noted that the company had just received the news and it would now have to move to shutter the work and begin the process of analyzing the results. And now the analysts are writing it off. Noted Leerink’s Seamus Fernandez:

While a disappointing development, we had always viewed this trial as a high risk endeavor given the previous suspension of the Phase 2/3 EPOCH study in mild-to-moderate AD patients, along with historic failures of other drugs in this disease. Nevertheless, we had included $750M in peak 2026E probability-adjusted sales for the drug in our model. Removing this brings our price target to $66/shr (from $67/shr).

Alzheimer’s research has been an unrelenting disaster zone for 15 years, with nothing available to treat this disease aside from some marginal symptomatic therapies. Pfizer just abandoned neurosciences entirely, dropping its Alzheimer’s work along the way. Yet Merck and most of the rest of the players aren’t stepping back. J&J, which had one of the biggest failures in the field, is forging ahead with gene therapies and other technologies. Denali pulled off a record IPO last year promising a new approach to conquer Alzheimer’s. And Merck is pressing ahead on tau, the other toxic protein often figured as a trigger for the memory-wasting ailment.

“We are disappointed with this outcome, especially given the lack of treatment options for patients suffering from Alzheimer’s disease,” said Roger Perlmutter, president, Merck Research Laboratories. “We are grateful to the patients and caregivers who participated in this study, and despite this outcome, Merck remains committed to developing novel therapies for the treatment of Alzheimer’s and other neurodegenerative diseases.”

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Recursion Pharmaceuticals Salt Lake City, UT
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Atlas Venture Cambridge, MA

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