The company is using positive data generated last year from its phase Ia/b trial in patients with mild to moderate Alzheimer’s disease as a springboard to the clinic. The randomized, placebo-controlled, double-blind study evaluated NDX-107 by assessing quantitative electroencephalography as a brain circuitry activity biomarker and an event-related potential as a measure of the brain’s working memory access and cognitive processes. The data showed dose-dependent and consistent changes in brain activity in all treated cohorts. The study produced quantitative electroencephalography data showing NDX-1017 stimulated higher frequency gamma activity in the brain compared to baseline one and three hours after dosing on days four and eight in healthy subjects and in Alzheimer’s disease patients.
The study of 88 healthy young and elderly individuals and patients with Alzheimer’s disease dementia evaluated single and multiple ascending doses. The small molecule is designed to enhance hepatocyte growth factor and MET, its receptor, to address neurodegeneration and to regenerate brain tissue.
Seattle-based Athira, the former M3 Biotechnology Inc., which rebranded in April 2019, is also in the IND-enabling phase of using NDX-1017 for treating Parkinson’s disease and is in the discovery phase for treating CNS and non-CNS indications that include major depression and peripheral neuropathy. The new name comes from the Arabic word “athir,” which President and CEO Leen Kawas said “means the sound and energy of everyone.”
The technology is based on the work of Joe Harding, of Washington State University’s department of integrative physiology and neuroscience. His laboratory focuses on developing small-molecule therapeutics targeting growth factors that include activators and antagonists. Harding was a co-founder of M3.
The original company was incorporated in 2009 and geared up to full operations in 2014. In 2016, it identified NDX-107. The company keeps in touch with potential partners as it develops its candidate.
Going forward, Kawas said COVID-19 should not pose problems to entering the clinic later this year. If anything, she said, the pandemic has a silver lining because it has accelerated many efficiencies in clinical trials that she felt should have been implemented a while ago.
“I’ve heard a lot in the past 10 years, that they would do this and do that to remove redundancies,” Kawas told BioWorld. “[COVID-19] pushed trials to become more efficient, and that’s something we’ll take advantage of.”
The upcoming phase II/III was planned between the end of the phase Ia/b and the pandemic’s beginning. The efficiencies that Kawas expects include using validated home testing kits and patients using local labs instead of blood draws at hospitals. She noted that the new FDA guidelines are more open-minded about handling the supply chain.
“It’s nice to see that a lot of it has moved into the virtual world, with a lot becoming more green,” she said. “It shifted very quickly, immediately the clinical science shifted. This saves days and time. I’m more surprised it wasn’t implemented sooner.”
Kawas grew up in Jordan, where her mother ran the University of Jordan Hospital. With a doctorate in pharmacy, she became a pharmacist but left it behind her when she came to the U.S. to earn a PhD in molecular cancer pharmacology. It was as a Washington State student that she encountered Harding, who encouraged her to lead M3 right out of school. She said it’s the best decision she ever made.
The series B financing was led by Perceptive Advisors new investors RTW Investments, Viking Global Investors, Venrock Healthcare Capital Partners, Franklin Templeton, Rock Springs Capital, Lifesci Venture Partners, Surveyor Capital, Highside Capital Management, Logos Capital, funds managed by Janus Henderson Investors, Sofinnova Investments, Avidity Partners, and existing investors including Rick and Suzanne Kayne and Sahsen Ventures.