Companies To Watch: Athira Pharma
By Wayne Koberstein, Executive Editor, Life Science Leader
Follow Me On Twitter @WayneKoberstein
To protect and restore neuronal networks by enhancing natural repair processes.
Snapshot
Athira Pharma takes a small-molecule approach to targeting the neurotrophic hepatocyte growth factor (HGF) system for neurodegenerative diseases. Athira has reached late-stage development of its lead drug, fosgonimeton, now in a Phase 2/3 trial for treating mild-to-moderate Alzheimer’s disease (AD), after encouraging top-line data for a Phase 2 trial in Parkinson’s disease and dementia in 2023. The company has several other candidates earlier in its pipeline: ATH-1020, undergoing a Phase 1 trial for neurodegenerative diseases; ATH-1105, in a Phase 1 study for amyotrophic lateral sclerosis (ALS); and others in discovery.
What’s At Stake
Aging … well, it’s complicated. Yet a common denominator of aging exists. The many-splendored mechanisms by which a body repairs itself wear down over time, and when the inevitable damage builds, it does so with ever-increasing ease and permanency, finally leading to disease-like conditions caused not by some outside agent but by our own inherent biology. In the neurology of the brain, we know them by many names, such as Parkinson’s and dementia. Athira is developing agents to reverse such diseases by helping neurons to stay alive and functioning properly.
The company’s president and CEO, Mark Litton, was a business-bound Ph.D. when he got his first industry job in 1990. He joined Athira in 2019 after many years in executive company management and business development. “What really got me excited about this company was this: Athira is all about enhancing a natural repair mechanism our bodies use all the time. We believe, if we can keep these brain cells alive and healthy, that will translate into benefit and functional improvement,” he says.
Athira’s lead drug, like every drug in its pipeline, is a small-molecule therapeutic. Small molecules seem to be making a comeback currently, but in original ways, thanks to advances in understanding disease mechanisms. The change potentially reduces manufacturing costs compared to bio-produced drugs, but it also raises the stakes in R&D, especially discovery, where Athira has placed much of its bets to power key functions.
For each investigative-drug program, Athira must identify and delineate the precise disease mechanism it wants to target, design a molecule that will hit that target, and still satisfy all of the scientific and regulatory requirements for the drug’s safety. It must also conduct clinical trials that meet the same standards as required for biopharmaceuticals and therapies such as CAR-T. Innovation in treating many diseases may no longer be the sole province of biotech.
“We spend a lot of time understanding the biology behind our molecule and its action in detail,” says Litton. “When you enhance this natural repair mechanism with a small molecule, it does help keep these cells alive in culture and animal models, and it significantly reduced patients’ cell damage in our Phase 2 Alzheimer’s trial. Our next trial of fosgonimeton will show us this year whether the biology translates into improvement of cognition, function, and independence.”
One interesting fact is easy to see: with the new small-molecule drugs, Big Pharma is leaving most of the heavy lifting in development to small startups. If you still define biopharma only as artificial products of bio-organisms, you may need to update your lexicon.
Vital Statistics
Employees: ~70
Headquarters: Bothell, WA
Finances: $16M Series A/A-1, February 2016; $85M Series B, June 2020; IPO, $208 million, September 2020; SPO: $97 million, February 2021
Significant Investors: Perceptive Advisors, Baker Bros Advisors, Kayne Anderson Capital Advisors, The Vanguard Group, Propel Bio Partners, Simplify Asset Management
Latest Updates:
January 2024: Completed enrollment in Phase 2/3 study of fosgonimeton in mild-to-moderate Alzheimer’s disease; topline data in second half of 2024.
February 2024: Published preclinical data highlighting therapeutic potential of ATH-1105 in ALS.
June 2024: Initiated Phase 1 study of ATH-1105 in healthy volunteers.