Athira Pharma Presents Preclinical Data Highlighting Neuroprotective Effects of ATH-1105 in Models of ALS at Motor Neurone Disease Association’s 35th International Symposium on ALS/MND

Rhea-AI Summary

Athira Pharma (NASDAQ: ATHA) presented preclinical data for ATH-1105 at the Motor Neurone Disease Association's 35th International Symposium on ALS/MND. The research demonstrated the drug's neuroprotective effects in human models of ALS, specifically in human iPSC-derived motor neurons expressing the SOD1-A4V mutation.

Key findings showed that ATH-1105 promoted activation of MET receptor in ALS patient-derived motor neurons, enhanced motor neuron survival, and preserved neurite networks following glutamate challenge. The drug demonstrated neuroprotective activity through the MET receptor, with effects diminishing when MET was knocked down. In a neuromuscular junction model, ATH-1105 showed protective effects on motor neuron survival and neurite networks.

Positive

• Successful demonstration of neuroprotective effects in human ALS models
• Positive results in preserving neurite networks and motor neuron survival
• Confirmed mechanism of action through MET receptor activation

Negative

• Results to preclinical stage, requiring further clinical validation
• No human clinical trial data available yet

BOTHELL, Wash., Dec. 06, 2024 (GLOBE NEWSWIRE) -- Athira Pharma, Inc. (NASDAQ: ATHA), a clinical-stage biopharmaceutical company focused on developing small molecules to restore neuronal health and slow neurodegeneration, today presented preclinical data highlighting target engagement and neuroprotective effects of ATH-1105 in human motor neurons at the Motor Neurone Disease Association’s 35^th International Symposium on ALS/MND, taking place Dec. 6-8, 2024, in Montreal, Canada.

“The preclinical data presented demonstrate for the first time the neuroprotective effects of ATH-1105 in human models of ALS, including human iPSC-derived motor neurons expressing the SOD1-A4V mutation,” said Kevin Church, Ph.D., Chief Scientific Officer of Athira. “These findings add to our significant body of preclinical evidence for ATH-1105 that have previously demonstrated improvements in nerve and motor function, biomarkers of inflammation and neurodegeneration, and survival in animal models of ALS.”

Presentation Details:
Title: ATH-1105, a small-molecule positive modulator of the neurotrophic HGF system, is neuroprotective in co-culture of human iPSC-derived motor neurons and muscle​
Poster: # HCB-28
Date/Time: Friday, December 6, 5:30 p.m. EST
Presenter: Sherif Reda, Ph.D., Associate Director, Discovery Biology, Athira Pharma

Highlights of this presentation include:

• ATH-1105 promoted activation of MET (HGF receptor) in ALS patient-derived motor neurons​.
• ATH-1105 enhanced motor neuron survival and preserved neurite networks following glutamate challenge in primary rat spinal motor neurons.
• ATH-1105 demonstrated neuroprotective activity through the MET receptor; following siRNA-mediated knockdown of MET, the neuroprotective effects of ATH-1105 on neuronal survival and neurite networks were abolished.
• In a neuromuscular junction model consisting of human iPSC-derived SOD1^A4V motor neurons and human muscle, ATH-1105 enhanced motor neuron survival and preserved neurite networks following glutamate challenge.

About ATH-1105
ATH-1105 is an oral, brain-penetrant, small-molecule positive modulator of the neurotrophic HGF system in development for the potential treatment of amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases. ATH-1105 is currently in a Phase 1 (NCT 06432647) double-blind, placebo-controlled trial in volunteers to evaluate single and multiple oral ascending doses of ATH-1105. The study is evaluating the safety and tolerability of ATH-1105 and includes measurements of pharmacokinetic outcomes. 

About Athira Pharma, Inc.
Athira Pharma, Inc., headquartered in the Seattle, Washington area, is a clinical-stage biopharmaceutical company focused on developing small molecules to restore neuronal health and slow neurodegeneration. Athira aims to alter the course of neurological diseases by advancing its pipeline of drug candidates that modulate the neurotrophic HGF system. For more information, visit www.athira.com. You can also follow Athira on Facebook, LinkedIn, X (formerly known as Twitter) and Instagram.

Forward-Looking Statements
This communication contains “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933, Section 21E of the Securities Exchange Act of 1934 and the Private Securities Litigation Reform Act of 1995. These forward-looking statements are not based on historical fact and include statements regarding: Athira’s drug candidates as potential treatments for amyotrophic lateral sclerosis and other neurodegenerative diseases; future development plans; the potential learnings from preclinical studies and other nonclinical data and their ability to inform and improve future clinical development plans; expectations regarding the potential efficacy and commercial potential of Athira’s drug candidates; and Athira’s ability to advance its drug candidates into later stages of development. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as “may,” “will,” “should,” “on track,” “would,” “expect,” “plan,” “believe,” “intend,” “pursue,” “continue,” “suggest,” “potential,” “target,” and similar expressions. Any forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the data from preclinical and clinical trials may not support the safety, efficacy and tolerability of Athira’s drug candidates; development of drug candidates may cease or be delayed; regulatory authorities could object to protocols, amendments and other submissions; future potential regulatory milestones for drug candidates, including those related to current and planned clinical studies, may be insufficient to support regulatory submissions or approval; whether Athira’s trials are sufficiently powered to meet the planned endpoints; Athira may not be able to recruit sufficient patients for its clinical trials; the outcome of legal proceedings that have been or may in the future be instituted against Athira, its directors and officers; possible negative interactions of Athira's drug candidates with other treatments; Athira’s assumptions regarding its financial condition and the sufficiency of its cash, cash equivalents and investments to fund its planned operations may be incorrect; adverse conditions in the general domestic and global economic markets; the impact of competition; the impact of new or changing laws and regulations; risks related to Athira’s exploration of strategic alternatives; as well as the other risks detailed in Athira’s filings with the Securities and Exchange Commission from time to time. These forward-looking statements speak only as of the date hereof and Athira undertakes no obligation to update forward-looking statements. Athira may not actually achieve the plans, intentions, or expectations disclosed in its forward-looking statements, and you should not place undue reliance on the forward-looking statements.

Investor & Media Contact:

Julie Rathbun
Athira Pharma
Julie.rathbun@athira.com
206-769-9219

Corporate Development Contact:
Maya Kneip
Program Manager, Portfolio & Program Management
Maya.kneip@athira.com
(206) 412-9078

FAQ

What were the key findings of ATH-1105 in Athira Pharma's (ATHA) preclinical ALS study?

ATH-1105 showed neuroprotective effects in human ALS models, enhanced motor neuron survival, and preserved neurite networks through MET receptor activation in ALS patient-derived motor neurons.

How did ATH-1105 perform in the SOD1A4V motor neuron model for ATHA?

ATH-1105 enhanced motor neuron survival and preserved neurite networks in human iPSC-derived SOD1A4V motor neurons when tested in a neuromuscular junction model with human muscle.

What was the mechanism of action demonstrated for Athira's (ATHA) ATH-1105?

ATH-1105 worked through the MET receptor pathway, with its neuroprotective effects being abolished when MET was knocked down through siRNA-mediated intervention.