Athira Pharma to Present Data from First-in-Human Phase 1 Clinical Trial of ATH-1105 at the 4th Annual ALS Drug Development Summit
Athira Pharma (NASDAQ: ATHA) announced the presentation of Phase 1 clinical trial data for ATH-1105, their novel oral drug candidate for ALS treatment, at the 4th Annual ALS Drug Development Summit in Boston. The first-in-human trial demonstrated favorable safety and tolerability profiles in healthy volunteers, with dose proportional pharmacokinetics and CNS penetration.
The company plans to initiate clinical trials in ALS patients in late 2025. ATH-1105 has shown promising results in preclinical studies, including significant improvements in nerve and motor function, reduced inflammation, and increased survival in various ALS models. The drug works by modulating the neurotrophic HGF system and has demonstrated effectiveness in reducing TDP-43 pathology in preclinical ALS models.
Preclinical data has been previously presented at major scientific meetings including AAN, AAIC, NEALS, and MNDA, and published in Frontiers in Neuroscience, 2024.Athira Pharma (NASDAQ: ATHA) ha annunciato la presentazione dei dati della sperimentazione clinica di Fase 1 per ATH-1105, il loro nuovo candidato farmaco orale per il trattamento della SLA, al 4° Summit Annuale sullo Sviluppo di Farmaci per la SLA a Boston. Lo studio di prima somministrazione nell'uomo ha evidenziato profili favorevoli di sicurezza e tollerabilità in volontari sani, con farmacocinetica proporzionale alla dose e penetrazione nel sistema nervoso centrale.
L'azienda prevede di avviare le sperimentazioni cliniche su pazienti con SLA entro la fine del 2025. ATH-1105 ha mostrato risultati promettenti negli studi preclinici, inclusi miglioramenti significativi nella funzione nervosa e motoria, riduzione dell'infiammazione e aumento della sopravvivenza in diversi modelli di SLA. Il farmaco agisce modulando il sistema neurotrofico HGF e ha dimostrato efficacia nella riduzione della patologia TDP-43 nei modelli preclinici di SLA.
I dati preclinici sono stati presentati in precedenza in importanti convegni scientifici come AAN, AAIC, NEALS e MNDA, e pubblicati su Frontiers in Neuroscience nel 2024.
Athira Pharma (NASDAQ: ATHA) anunció la presentación de datos del ensayo clínico de Fase 1 para ATH-1105, su nuevo candidato a fármaco oral para el tratamiento de la ELA, en la 4ª Cumbre Anual de Desarrollo de Medicamentos para ELA en Boston. El ensayo en humanos por primera vez mostró perfiles favorables de seguridad y tolerabilidad en voluntarios sanos, con farmacocinética proporcional a la dosis y penetración en el sistema nervioso central.
La compañía planea iniciar ensayos clínicos en pacientes con ELA a finales de 2025. ATH-1105 ha mostrado resultados prometedores en estudios preclínicos, incluyendo mejoras significativas en la función nerviosa y motora, reducción de la inflamación y aumento de la supervivencia en varios modelos de ELA. El fármaco actúa modulando el sistema neurotrófico HGF y ha demostrado efectividad en la reducción de la patología TDP-43 en modelos preclínicos de ELA.
Los datos preclínicos se han presentado previamente en importantes reuniones científicas como AAN, AAIC, NEALS y MNDA, y se publicaron en Frontiers in Neuroscience en 2024.
Athira Pharma (NASDAQ: ATHA)는 보스턴에서 열린 제4회 연례 ALS 약물 개발 서밋에서 ALS 치료를 위한 새로운 경구용 약물 후보 ATH-1105의 1상 임상시험 데이터를 발표했습니다. 최초 인간 대상 시험에서 건강한 지원자들에게서 안전성과 내약성이 우수하게 나타났으며, 용량에 비례하는 약동학과 중추신경계 침투가 확인되었습니다.
회사는 2025년 말에 ALS 환자를 대상으로 임상시험을 시작할 계획입니다. ATH-1105는 전임상 연구에서 신경 및 운동 기능의 유의미한 개선, 염증 감소, 다양한 ALS 모델에서 생존율 증가 등의 유망한 결과를 보였습니다. 이 약물은 신경영양인자 HGF 시스템을 조절하여 전임상 ALS 모델에서 TDP-43 병리를 감소시키는 효과를 입증했습니다.
전임상 데이터는 이전에 AAN, AAIC, NEALS, MNDA 등 주요 과학 학회에서 발표되었으며, 2024년 Frontiers in Neuroscience에 게재되었습니다.
Athira Pharma (NASDAQ : ATHA) a annoncé la présentation des données de l’essai clinique de phase 1 pour ATH-1105, leur nouveau candidat médicament oral pour le traitement de la SLA, lors du 4e Sommet annuel sur le développement de médicaments pour la SLA à Boston. L’essai de première administration chez l’homme a démontré des profils favorables de sécurité et de tolérance chez des volontaires sains, avec une pharmacocinétique proportionnelle à la dose et une pénétration dans le système nerveux central.
L’entreprise prévoit de lancer des essais cliniques chez des patients atteints de SLA fin 2025. ATH-1105 a montré des résultats prometteurs lors d’études précliniques, notamment des améliorations significatives des fonctions nerveuses et motrices, une réduction de l’inflammation et une augmentation de la survie dans divers modèles de SLA. Le médicament agit en modulant le système neurotrophique HGF et a prouvé son efficacité dans la réduction de la pathologie TDP-43 dans des modèles précliniques de SLA.
Les données précliniques ont déjà été présentées lors de grandes conférences scientifiques telles que l’AAN, l’AAIC, le NEALS et la MNDA, et publiées dans Frontiers in Neuroscience en 2024.
Athira Pharma (NASDAQ: ATHA) gab die Präsentation der Phase-1-Klinikstudien-Daten für ATH-1105, ihren neuen oralen Wirkstoffkandidaten zur Behandlung von ALS, auf dem 4. jährlichen ALS Drug Development Summit in Boston bekannt. Die erste Studie am Menschen zeigte günstige Sicherheits- und Verträglichkeitsprofile bei gesunden Probanden sowie dosisproportionale Pharmakokinetik und ZNS-Durchdringung.
Das Unternehmen plant, Ende 2025 klinische Studien bei ALS-Patienten zu starten. ATH-1105 zeigte vielversprechende Ergebnisse in präklinischen Studien, darunter signifikante Verbesserungen der Nerven- und Motorikfunktionen, reduzierte Entzündungen und eine erhöhte Überlebensrate in verschiedenen ALS-Modellen. Der Wirkstoff wirkt durch Modulation des neurotrophen HGF-Systems und zeigte Wirksamkeit bei der Reduktion der TDP-43-Pathologie in präklinischen ALS-Modellen.
Präklinische Daten wurden zuvor auf bedeutenden wissenschaftlichen Tagungen wie AAN, AAIC, NEALS und MNDA präsentiert und 2024 in Frontiers in Neuroscience veröffentlicht.
- Favorable safety and tolerability profile demonstrated in Phase 1 trials
- Successful dose proportional pharmacokinetics and CNS penetration achieved
- Preclinical data shows statistically significant improvements in nerve and motor function
- On track for ALS patient trials in late 2025
- None.
Insights
Athira's ATH-1105 shows promising Phase 1 results with good safety profile and CNS penetration, advancing toward ALS patient trials in late 2025.
Athira Pharma has shared encouraging data from its first-in-human Phase 1 trial of ATH-1105, an oral, brain-penetrant small molecule targeting the hepatocyte growth factor (HGF) system. The data revealed a favorable safety and tolerability profile in healthy volunteers, with dose-proportional pharmacokinetics and confirmed central nervous system penetration - critical factors for any neurological drug candidate.
ATH-1105's mechanism targeting the neurotrophic HGF system is particularly relevant for ALS, where neuronal loss is a hallmark feature. The HGF system regulates neuronal survival and function, making it a biologically plausible approach to slowing neurodegeneration in ALS. The company appears to be following a methodical development path, with plans to advance to ALS patient trials in late 2025, where they will evaluate neurofilament light chain (NfL) as a biomarker - an important validated measure of neuronal damage.
The preclinical package appears robust, with data showing improvements in nerve and motor function, reduced neuroinflammation, and extended survival in ALS models. These findings have been peer-reviewed and published in Frontiers in Neuroscience, adding scientific credibility. While Phase 1 data in healthy volunteers cannot predict efficacy in patients, the favorable safety profile and confirmed CNS penetration are important technical milestones that support continued development.
The field of ALS therapeutics desperately needs new approaches, as current treatment options provide minimal benefit. If ATH-1105 can demonstrate meaningful effects on NfL and clinical outcomes in upcoming patient trials, it could potentially address this significant unmet need. However, investors should recognize that the path from successful Phase 1 to approved therapy remains long and uncertain, with efficacy in ALS patients yet to be demonstrated.
Data from Phase 1 clinical trial of ATH-1105 in healthy volunteers showed a favorable safety and tolerability profile; dose proportional pharmacokinetics and CNS penetration support continued clinical development
On-track to enable initiation of a clinical trial in ALS patients in late 2025
BOTHELL, Wash., May 13, 2025 (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 announced that the Company will present data from a Phase 1 clinical trial of ATH-1105 in healthy volunteers at the 4th Annual ALS drug Development Summit taking place from May 12-14, 2025 in Boston, Massachusetts.
ATH-1105 is the Company’s novel, orally available, brain-penetrant, next-generation small molecule drug candidate designed to positively modulate the neurotrophic HGF system for the potential treatment of neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS).
“We are very encouraged by these first-in-human safety and pharmacokinetic data and how they reinforce ATH-1105’s continued development as a potential treatment for ALS,” noted Javier San Martin, M.D., Chief Medical Officer at Athira. “We look forward to enabling the initiation of a clinical trial in ALS patients in late 2025 and to evaluating ATH-1105's effect on an ALS validated biomarker (NfL).”
The data will be highlighted in an oral presentation titled, “Advancing ATH-1105 for ALS Through Early Clinical and PK Data,” on Tuesday, May 13, 2025, at 11 am ET and a poster presentation titled, “ATH-1105 Enhances Motor Neuron Survival and Reduces TDP-43 Pathology in Preclinical ALS Models,” on Tuesday, May 13, 2025, at 3 p.m. ET. The data, to be presented by Sherif Reda, Ph.D., Director, Discovery Research at Athira Pharma, and Kai-Bin Ooi, Director, Drug Development and Operations at Athira Pharma, will feature preclinical and Phase 1 data supporting the continued clinical development of ATH-1105 in ALS.
Key Highlights from the Presentation
- ATH-1105 has demonstrated consistent and robust beneficial effects in preclinical models of ALS
- ATH-1105 showed a favorable safety profile and was well tolerated in both single and multiple ascending dose studies in healthy volunteers
- ATH-1105 showed dose proportional pharmacokinetics and central nervous system (CNS) penetration
ATH-1105’s potential is supported by a growing body of preclinical evidence demonstrating statistically significant improvements in nerve and motor function, biomarkers of inflammation and neurodegeneration, and survival in various models of ALS. These data have been presented at a variety of key scientific and medical meetings including the American Association of Neurology (AAN), the Alzheimer’s Association International Congress (AAIC), the Northeast Amyotrophic Lateral Sclerosis Consortium® (NEALS), and the Motor Neurone Disease Association (MNDA), and published in Frontiers in Neuroscience, 2024.
About the Phase 1 Clinical Trial
The first-in-human Phase 1 (NCT 06432647) double-blind, placebo-controlled clinical trial enrolled 80 healthy volunteers to evaluate single and multiple oral ascending doses of ATH-1105. The trial was completed in November 2024 and evaluated the safety and tolerability of ATH-1105 and included measurements of pharmacokinetic outcomes. The results of the Phase 1 trial showed that ATH-1105 demonstrated a favorable safety profile and was well-tolerated in healthy volunteers, supporting continued clinical development.
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 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 and the timing thereof; the potential learnings from preclinical studies and other nonclinical data and from our Phase 1 ATH-1105 clinical trial and their ability to inform future clinical development plans; expectations and timing regarding the initiation of a clinical trial of ATH-1105 in ALS patients; 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 may in the future be instituted against Athira, its directors and officers; possible negative interactions of Athira's drug candidates with other treatments; FDA regulatory delays and uncertainty and new policies implemented under the current administration, including executive orders, changes in the leadership of federal agencies such as the FDA and SEC, staff layoffs, budget cuts to agency programs and research, and changes in drug pricing controls; 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, including as a result of tariffs; the impact of competition; the impact of drug candidate development and clinical activities on operating expenses; 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
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