Athira Aims for the Nasdaq to Advance Alzheimer’s Drug to Phase 2 Tests

Xconomy Seattle — 

Many of the drugs tested in Alzheimer’s disease so far, and some still in development, aim to block or break up proteins associated with the progression of the memory-robbing disorder. Athira Pharma is taking a different approach—neuron regeneration. And it’s doing so by a tapping a protein first discovered in the liver.

Seattle-based Athira has encouraging results from early clinical trials, and it’s now looking ahead to larger tests in Alzheimer’s as well as Parkinson’s disease. With those studies expected to start in the coming year, Athira is turning to the public markets to fund its clinical development plans. In paperwork filed with securities regulators, the company set a preliminary $100 million target for its IPO.

Athira designed its drugs to promote the ability of a protein called hepatocyte growth factor (HGF) to activate a signaling pathway that promotes regeneration. HGF was first discovered in the liver, but further research uncovered its role in multiple organs, including the brain, the company says in its IPO filing. Activating this pathway is challenging for biologic drugs or gene therapies because they can’t cross the blood-brain barrier. But Athira says its small molecules can cross that nearly impermeable membrane, tapping into the pathway’s regenerative properties once inside the brain. The company adds that this approach has potential applications in a wide range of central nervous system disorders, such as Alzheimer’s and Parkinson’s, as well as peripheral conditions such as neuropathies.

Alzheimer’s is the first target of Athira’s lead drug, ATH-1017. At the Clinical Trials on Alzheimer’s Conference in San Diego last December, Athira reported results from an 88-patient Phase 1 study that included 11 patients with mild-to-moderate Alzheimer’s. The results showed that the drug was well tolerated and produced no adverse events. The results also showed improvement in brain network activity, which indicates “potentially positive effects on brain function,” the company says in its filing.

Now Athira is preparing to advance ATH-1017 to two studies. The first is a Phase 2/3 clinical trial enrolling 240 to 300 patients with mild-to-moderate Alzheimer’s. The placebo-controlled study will assess the safety, tolerability, and efficacy of two different doses of the Athira drug. The company plans to start that study by the end of this year; preliminary data are expected by the end of 2022.

Athira plans to run a separate Phase 2 clinical trial in parallel to the Phase 2/3 study. The placebo-controlled Phase 2 study will test the same two doses in the same type of Alzheimer’s patients. But this study, which is targeting enrollment of 60 to 75 patients, aims to assess the overall effects of the Athira drug on memory processing speed and other cognitive measures. The company expects preliminary results from this study will become available by early 2022.

In addition to the Alzheimer’s studies, Athira is planning a Phase 2 study testing its lead drug candidate as a treatment for Parkinson’s disease dementia. That study is expected to start by the end of next year.

Athira’s drugs are based on research licensed from Washington State University. CEO Leen Kawas (pictured above) founded the company in 2011 while earning her PhD in molecular pharmacology and toxicology at the university. Initially called M3 Biotechnology, the company last year adopted the new name Athira, derived from the Arabic word “athir.” Athira says the word loosely translates as “a force” or “an energy” or “something that is part of everything.”

Since its launch, Athira has raised more than $100 million, most recently an $85 million Series B round of financing in June. The company’s largest shareholder is Perceptive Life Sciences, which owns an 11.6 percent stake, according to the filing. RTW Investments owns 10.9 percent of the company; Kawas owns 9.4 percent.

In addition to financing the two Alzheimer’s clinical trials and one Parkinson’s study, the company plans to apply some of the IPO proceeds toward preclinical research leading up to an application to begin human testing of another drug, ATH-1019, in neuropsychiatric indications and neuropathy. Upon completing the IPO, Athira must also pay the Washington Life Sciences Discovery Fund $1.5 million. According to terms of matching grant awards that the fund made in 2014 and 2015, the company must pay twice the amount it received from the fund upon a triggering event, such as an IPO.

If Athira completes the stock offering, the company expects its shares will trade on the Nasdaq under the stock symbol “ATHA.”

 

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