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
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.
Athira Pharma (NASDAQ: ATHA) ha presentato dati preclinici per ATH-1105 al 35° Simposio Internazionale sulla SLA/MND dell'Associazione per le Malattie del Motoneurone. La ricerca ha dimostrato gli effetti neuroprotettivi del farmaco in modelli umani di SLA, in particolare in motoneuroni derivati da iPSC umane che esprimono la mutazione SOD1-A4V.
I risultati chiave hanno mostrato che ATH-1105 promuoveva l'attivazione del recettore MET nei motoneuroni derivati da pazienti con SLA, migliorava la sopravvivenza dei motoneuroni e preservava le reti neuritiche dopo una sfida al glutammato. Il farmaco ha dimostrato un'attività neuroprotettiva attraverso il recettore MET, con effetti che diminuivano quando il MET veniva abbattuto. In un modello di giunzione neuromuscolare, ATH-1105 ha mostrato effetti protettivi sulla sopravvivenza dei motoneuroni e sulle reti neuritiche.
Athira Pharma (NASDAQ: ATHA) presentó datos preclínicos para ATH-1105 en el 35° Simposio Internacional sobre SLA/MND de la Asociación de Enfermedades de la Motoneurona. La investigación demostró los efectos neuroprotectores del fármaco en modelos humanos de SLA, específicamente en motoneuronas derivadas de iPSC humanas que expresan la mutación SOD1-A4V.
Los hallazgos clave mostraron que ATH-1105 promovía la activación del receptor MET en motoneuronas derivadas de pacientes con SLA, mejoraba la supervivencia de motoneuronas y preservaba las redes de neuritas tras un desafío con glutamato. El fármaco demostró actividad neuroprotectora a través del receptor MET, con efectos que disminuían cuando se eliminaba el MET. En un modelo de unión neuromuscular, ATH-1105 mostró efectos protectores sobre la supervivencia de motoneuronas y las redes de neuritas.
아티라 파마(Athira Pharma) (NASDAQ: ATHA)는 운동신경질환협회의 제35회 국제 ALS/MND 심포지엄에서 ATH-1105에 대한 연구 결과를 발표했습니다. 연구 결과는 인간 ALS 모델에서 약물의 신경 보호 효과를 입증하였으며, 특히 SOD1-A4V 변이를 발현하는 인간 iPSC 유래 운동신경세포에서의 효과가 나타났습니다.
주요 발견으로는 ATH-1105가 ALS 환자로부터 유래한 운동신경세포에서 MET 수용체의 활성화를 촉진하고, 운동신경세포의 생존을 향상시키며, 글루타메이트 도전 후 신경 가지 네트워크를 보호한다는 것이었습니다. 이 약물은 MET 수용체를 통해 신경 보호 활동을 나타냈으며, MET이 제거되면 효과가 감소했습니다. 신경근접합부 모델에서는 ATH-1105가 운동신경세포의 생존과 신경 가지 네트워크에 대한 보호 효과를 보였습니다.
Athira Pharma (NASDAQ: ATHA) a présenté des données précliniques sur ATH-1105 lors du 35e Symposium International sur la SLA/MND de l'Association des Maladies du Motoneurone. La recherche a démontré les effets neuroprotecteurs du médicament dans des modèles humains de SLA, en particulier dans des motoneurones dérivés de iPSC humaines exprimant la mutation SOD1-A4V.
Les résultats clés ont montré qu'Ath-1105 favorisait l'activation du récepteur MET dans les motoneurones dérivés de patients atteints de SLA, améliorait la survie des motoneurones et préservait les réseaux de neurites après un défi au glutamate. Le médicament a démontré une activité neuroprotectrice à travers le récepteur MET, avec des effets diminuant lorsque le MET était inactivé. Dans un modèle de jonction neuromusculaire, ATH-1105 a montré des effets protecteurs sur la survie des motoneurones et les réseaux de neurites.
Athira Pharma (NASDAQ: ATHA) präsentierte präklinische Daten zu ATH-1105 auf dem 35. Internationalen Symposium über ALS/MND der Motor Neuron Disease Association. Die Forschung zeigte die neuroprotektiven Effekte des Medikaments in menschlichen ALS-Modellen, insbesondere in von menschlichen iPSC abgeleiteten Motoneuronen, die die SOD1-A4V-Mutation exprimieren.
Wichtige Ergebnisse zeigten, dass ATH-1105 die Aktivierung des MET-Rezeptors in aus ALS-Patienten abgeleiteten Motoneuronen förderte, die Überlebensrate der Motoneuronen erhöhte und die Neuritnetze nach einer Glutamatbelastung schützte. Das Medikament zeigte neuroprotektive Aktivitäten über den MET-Rezeptor, wobei die Wirkung abnahm, wenn MET ausgeschaltet wurde. In einem neuromuskulären Junction-Modell zeigte ATH-1105 schützende Effekte auf die Überlebensrate der Motoneuronen und die Neuritnetze.
- 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
- Results to preclinical stage, requiring further clinical validation
- No human clinical trial data available yet
Insights
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 35th 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 SOD1A4V 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?
How did ATH-1105 perform in the SOD1A4V motor neuron model for ATHA?
What was the mechanism of action demonstrated for Athira's (ATHA) ATH-1105?
