Athira Pharma Presents ATH-1105 Phase 1 Trial Results at ALS Nexus 2025
Athira Pharma (NASDAQ: ATHA) presented Phase 1 clinical trial results for ATH-1105, their novel oral drug candidate for ALS treatment, at the ALS Nexus 2025 conference. The trial demonstrated a favorable safety and tolerability profile, along with dose-proportional pharmacokinetics and CNS penetration in healthy volunteers.
The drug candidate, designed to modulate the neurotrophic HGF system, has shown consistent and robust beneficial effects in preclinical ALS models. Chief Medical Officer Dr. Javier San Martin emphasized the importance of developing new ALS treatments to prolong survival and improve patients' quality of life.
Athira Pharma (NASDAQ: ATHA) ha presentato i risultati dello studio clinico di Fase 1 per ATH-1105, il loro nuovo candidato farmaco orale per il trattamento della SLA, alla conferenza ALS Nexus 2025. Lo studio ha mostrato un profilo di sicurezza e tollerabilità favorevole, oltre a una farmacocinetica proporzionale alla dose e alla penetrazione nel SNC in volontari sani.
Il candidato, progettato per modulare il sistema neurotrofico HGF, ha evidenziato effetti benefici coerenti e robusti nei modelli preclinici di SLA. Il Direttore Medico, Dr. Javier San Martin, ha sottolineato l'importanza di sviluppare nuove terapie per la SLA per prolungare la sopravvivenza e migliorare la qualità di vita dei pazienti.
Athira Pharma (NASDAQ: ATHA) presentó en la conferencia ALS Nexus 2025 los resultados del ensayo clínico de Fase 1 de ATH-1105, su nuevo candidato oral para el tratamiento de la ELA. El estudio mostró un perfil de seguridad y tolerabilidad favorable, así como farmacocinética proporcional a la dosis y penetración en el SNC en voluntarios sanos.
El candidato, diseñado para modular el sistema neurotrófico HGF, demostró efectos beneficiosos coherentes y robustos en modelos preclínicos de ELA. El Director Médico, Dr. Javier San Martin, destacó la importancia de desarrollar nuevos tratamientos para la ELA con el fin de prolongar la supervivencia y mejorar la calidad de vida de los pacientes.
Athira Pharma (NASDAQ: ATHA)는 ALS Nexus 2025 학회에서 근위축성측삭경화증(ALS) 치료용 새로운 경구 약물 후보 ATH-1105의 1상 임상시험 결과를 발표했습니다. 이 연구에서는 안전성 및 내약성 프로파일이 양호했으며, 용량 비례 약동학과 건강한 지원자에서의 중추신경계(CNS) 침투가 확인되었습니다.
신경영양 인자 HGF 시스템을 조절하도록 설계된 이 후보 물질은 전임상 ALS 모델에서 일관되고 강력한 유익한 효과를 보였습니다. 최고의료책임자(Chief Medical Officer) Dr. Javier San Martin은 생존 기간을 연장하고 환자의 삶의 질을 향상시키기 위한 새로운 ALS 치료제 개발의 중요성을 강조했습니다.
Athira Pharma (NASDAQ: ATHA) a présenté lors de la conférence ALS Nexus 2025 les résultats de l'essai clinique de phase 1 pour ATH-1105, son nouveau candidat-médicament oral pour le traitement de la SLA. L'essai a révélé un profil de sécurité et de tolérabilité favorable, ainsi qu'une pharmacocinétique proportionnelle à la dose et une pénétration dans le SNC chez des volontaires sains.
Le candidat, conçu pour moduler le système neurotrophique HGF, a montré des effets bénéfiques constants et robustes dans des modèles précliniques de SLA. Le directeur médical, Dr Javier San Martin, a souligné l'importance de développer de nouveaux traitements pour la SLA afin de prolonger la survie et d'améliorer la qualité de vie des patients.
Athira Pharma (NASDAQ: ATHA) präsentierte auf der ALS Nexus 2025 die Ergebnisse der Phase‑1‑Studie zu ATH-1105, ihrem neuen oralen Wirkstoffkandidaten zur Behandlung von ALS. Die Studie zeigte ein günstiges Sicherheits‑ und Verträglichkeitsprofil sowie dosisproportionale Pharmakokinetik und ZNS‑Penetration bei gesunden Probanden.
Der Kandidat, der das neurotrophe HGF‑System modulieren soll, zeigte in präklinischen ALS‑Modellen konsistente und robuste positive Effekte. Chief Medical Officer Dr. Javier San Martin betonte die Bedeutung der Entwicklung neuer ALS‑Therapien, um das Überleben zu verlängern und die Lebensqualität der Patienten zu verbessern.
- Favorable safety and tolerability profile demonstrated in Phase 1 trial
- Successful dose-proportional pharmacokinetics and CNS penetration achieved
- Consistent and robust beneficial effects shown in preclinical ALS models
- None.
Insights
Athira's Phase 1 ATH-1105 trial shows promising safety profile and CNS penetration for potential ALS treatment, advancing their neurotrophic approach.
Athira Pharma has reached a significant milestone with its novel ALS drug candidate ATH-1105, demonstrating favorable safety and tolerability in Phase 1 healthy volunteer trials. The drug showed dose-proportional pharmacokinetics and, crucially, CNS penetration - essential for any neurological treatment to cross the blood-brain barrier and reach its target.
ATH-1105 represents a mechanistically distinct approach to ALS treatment by positively modulating the HGF (Hepatocyte Growth Factor) system. This neurotrophic pathway promotes neuronal survival and has shown potential to protect neurons from degeneration. The oral availability of ATH-1105 is particularly significant, as it simplifies administration compared to injectable therapies.
These early results justify continued development, though it's important to recognize this was only a Phase 1 study in healthy volunteers. The "consistent and robust beneficial effects in preclinical models of ALS" mentioned provide additional confidence, but the true test will come in Phase 2 trials involving actual ALS patients.
Given the limited treatment options for ALS and the disease's devastating progression, even incremental advances generate significant interest. The safety profile reported here represents the first crucial hurdle in development, especially for CNS-targeted therapeutics where side effects can be prohibitive. Athira will now need to demonstrate efficacy in patients while maintaining this safety profile across longer treatment durations.
BOTHELL, Wash., Aug. 14, 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, presented results from its Phase 1 clinical trial of ATH-1105 in healthy volunteers at the ALS Nexus 2025 conference in Dallas, Texas. ALS Nexus convenes leading experts from the ALS community, including researchers, healthcare professionals, advocates and individuals living with amyotrophic lateral sclerosis (ALS), to connect and learn about advances in research, clinical care and advocacy.
ATH-1105 is the Company’s novel, orally available, CNS-penetrant, next-generation small molecule drug candidate designed to positively modulate the neurotrophic HGF system for the potential treatment of neurodegenerative diseases, including ALS.
“The advancement of new treatment options for ALS is of vital importance to help prolong survival and improve quality of life of people living with ALS, and we are excited to be developing a potential novel approach with ATH-1105,” said Javier San Martin, M.D., Chief Medical Officer at Athira. “Our Phase 1 safety, tolerability, and pharmacokinetic data are encouraging and support continued development of ATH-1105.”
Results from the Phase 1 trial of ATH-1105 demonstrated a favorable safety and tolerability profile as well as dose-proportional pharmacokinetics and CNS penetration. ATH-1105 has also demonstrated consistent and robust beneficial effects in preclinical models of ALS.
The clinical data were highlighted in a poster presentation titled, “Safety, Tolerability, and Pharmacokinetics of ATH-1105 in Health Volunteers,” presented by Kai-Bin Ooi, Director, Drug Development and Operations at Athira Pharma.
About the ATH-1105 Phase 1 Clinical Trial
The first-in-human Phase 1 (NCT06432647) 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 their ability to inform and improve future clinical development plans; the implications of learnings from our Phase 1 ATH-1105 clinical trial for future development plans, including the timing and our plans to investigate ATH-1105 in a future clinical trial in people living with ALS either by us or in conjunction with a partner; expectations regarding the potential efficacy and commercial potential of Athira’s drug candidates; Athira’s ability to advance its drug candidates into later stages of development; and Athira’s plans and expectations regarding Athira’s exploration of strategic alternatives and partnering options. 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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