The approval on Monday of the first new drug for Alzheimer’s disease in nearly two decades — Biogen’s aducanumab — helps shine a light on a path forward for other companies developing Alzheimer’s therapies, including Seattle-based Athira Pharma, which saw its stock rise more than 4% off the Biogen news.
The FDA cleared aducanumab (also known as Aduhelm) on an accelerated, conditional approval program, and is requiring the company to conduct a new clinical trial to test effectiveness while it markets the drug. Approval came despite the near-unanimous objection of the agency’s independent advisory committee that there was not enough evidence that the drug had a significant patient benefit.
The FDA’s accelerated approval program is designed to advance therapies for a serious condition that is not addressed adequately by available therapies. Criteria include that the therapy “generally provides a meaningful advantage over available therapies and demonstrates an effect on a surrogate endpoint that is reasonably likely to predict clinical benefit,” according to the FDA.
That the FDA deployed this framework suggests that it may similarly ease the approval process for other Alzheimer’s therapies.
“The optimism that this brings is important for the field overall,” said Leen Kawas, CEO and co-founder of Athira, in an interview with GeekWire.
Founded in 2011, Athira is in late-stage development for its lead therapeutic candidate that could halt or reverse the nerve damage that causes Alzheimer’s disease and other illnesses including Parkinson’s and ALS, or Lou Gehrig’s Disease. It uses regenerative technology that rebuilds connections between neurons.
The therapeutic candidate, ATH-1017, targets hepatocyte growth factor (HGF) and its receptor (MET), which can foster neuronal survival and growth, among other actions. In 2019 the company reported what it said were promising initial results from a phase 1 trial testing effects on two surrogate endpoints, one biomarker for brain circuitry activity and another for working memory access and cognitive processes in the brain.
Athira, which went public in September, is now conducting a phase 2 trial examining biomarker response and a phase 2/3 trial that also is assessing clinical response. This set-up, Kawas said, has some parallels with the data used to support Monday’s approval — which relied in part on positive data from one trial, supporting data from another, and biomarker data on the drug’s target, beta-amyloid, which accumulates in clumps in the brains of affected individuals. Biogen’s data showed that its drug reduced levels of amyloid.
“This is going to be helpful for everyone in Alzheimer’s disease drug development as a guide of what is becoming part of the clinical endpoints for approval,” said Kawas.
Athira’s phase 2/3 trial is designed to assess if ATH-1017 can reverse cognitive decline, and Kawas is expecting data to emerge from the phase 2/3 trial as early as next year. Biogen’s trials asked if its drug could slow decline in patients at early stages of the condition, and so took many years to yield results.
Therapeutic candidates that target beta-amyloid have been favored by pharmaceutical companies for decades, which have spent billions testing candidates that ultimately failed in trials; the approval of aducanumab on Monday stands out in this area.
More recently, Athira and other drug companies have turned to other approaches and fresh targets. Anavex Life Sciences Corp., for instance, is clinically testing an agent that targets two receptors present in the brain. Cassava Sciences is testing an agent targeting another protein in the brain. The stock prices of all three biotech companies got a boost today upon the approval of the new drug.
