The approval on Monday of the first new Alzheimer’s drug in nearly two decades, Biogen’s aducanumab, helps illuminate the way forward for other companies developing Alzheimer’s disease therapies, including the one in Seattle. Athira Pharma, which saw its shares rise more than 4% on the Biogen news.
The FDA licensed aducanumab (also known as Aduhelm) in a conditional and accelerated approval program, and requires the company to conduct a new clinical trial to test efficacy while marketing the drug. The approval came despite almost unanimous objection from the agency’s independent advisory committee that there was insufficient evidence that the drug had a significant benefit to the patient.
The FDA’s accelerated approval program is designed to promote therapies for a serious condition that is not adequately addressed by available therapies. The criteria include that the therapy “generally provides a significant advantage over available therapies and demonstrates an effect on a surrogate endpoint that is reasonably likely to predict clinical benefit,” according to the FDA.
The fact that the FDA has implemented this framework suggests that it may also facilitate the approval process for other therapies for Alzheimer’s disease.
“The optimism that this brings is important for the field in general,” he said. They read Kawas, CEO and co-founder of Athira, in an interview with GeekWire.
Founded in 2011, Athira is in an advanced stage of development of its main therapeutic candidate that could stop or reverse the nerve damage that causes Alzheimer’s disease and other diseases, such as Parkinson’s and ALS, or Lou Gehrig’s disease. It uses regenerative technology that rebuilds the connections between neurons.
The therapeutic candidate, ATH-1017, targets hepatocyte growth factor (HGF) and its receptor (MET), which can promote neuronal growth and survival, among other actions. In 2019, the company reported what it said were promising early results from a phase 1 trial testing effects on two surrogate endpoints, a biomarker for brain circuit 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 that examines biomarker response and a phase 2/3 trial that also assesses clinical response. This setup, Kawas said, has some parallels with the data used to support Monday’s approval, which was based in part on positive data from one trial, supporting data from another, and biomarker data on the drug’s target, amyloid, It accumulates in clumps in the brains of affected people. Data from Biogen showed that their drug lowered amyloid levels.
“This will be useful to everyone in Alzheimer’s disease drug development as a guide to what is becoming part of the clinical endpoints for approval,” Kawas said.
Athira’s phase 2/3 trial is designed to assess whether ATH-1017 can reverse cognitive decline, and Kawas expects data from the phase 2/3 trial to emerge as soon as next year. Biogen’s trials asked whether their drug could slow the decline in patients in the early stages of the condition, taking many years to produce results.
Therapeutic candidates that target amyloid have been favored by drug companies for decades, spending billions on testing candidates who ultimately failed trials; In this area, the approval of aducanumab on Monday stands out.
More recently, Athira and other pharmaceutical companies have turned to other approaches and new targets. Anavex Life Sciences Corp, for example, is clinically testing an agent that targets two receptors in the brain. Cassava Sciences is testing an agent to restore the form and function of a protein involved in neuronal cell structure. The share prices of the three biotech companies received a boost today following the approval of the new drug.