After all the setbacks and failures we’ve seen in Alzheimer’s research over the past 15 years, can a startup boasting better technology beat the terrible odds against them and score with a new drug?
We’re at the early stages of getting an answer to that question.
Denali $DNLI announced today that it is now trying just that, launching a Phase I clinical study of its Alzheimer’s drug DNL747, its small molecule inhibitor for RIPK1. It’s also started a second human study of its LRRK2 inhibitor for Parkinson’s disease, which is its lead indication. And the South San Francisco biotech reported that it’s banked a $155 million upfront from Takeda’s billion-dollar partnership while scoring positive early results from a study involving monkeys for BACE1 — a mechanism in Alzheimer’s that recently failed decisively for Merck in Phase III.
That primate study is particularly important, as it helps validate Denali’s belief that its Antibody Transport Vehicle — or ATV — technology can get past the blood brain barrier and deliver a therapy into the brain — a hurdle that has stymied developers in the past.
Alzheimer’s has proven to be the hardest target in biopharma R&D, defeating everything thrown at it for well over a decade in pivotal trials. Merck’s recent failure on BACE, eliminating production of amyloid beta in patients, proved to their researchers that it wouldn’t work in mid-to-moderate patients. And they’re in the process of evaluating the numbers for prodomal patients, where verubecestat also failed.
Despite the litany of failures, though, the ex-Genentech gang — led by CEO Ryan Watts — that runs Denali have pieced together one of the most closely-watched efforts in the field, starting over with genetically defined patient groups and using new tech from companies like F-star. And despite the skepticism over targets and disease modification, the biotech has been able to store $467 million in cash by the end of December, building a market cap close to $2 billion — before the Takeda payment came through. And the global pharma company is on the hook for another $85 million in preclinical milestones.
Image: Ryan Watts. UNIVERSITY OF UTAH via YOUTUBE
The best place to read Endpoints News? In your inbox.
Comprehensive daily news report for those who discover, develop, and market drugs. Join 42,300+ biopharma pros who read Endpoints News by email every day.Free Subscription