Alterity Therapeutics to Deliver an Oral Presentation of the Positive ATH434 Phase 2 Trial Results at the American Academy of Neurology Annual Meeting
Alterity Therapeutics (NASDAQ: ATHE) has announced upcoming presentations of its ATH434 Phase 2 clinical trial results for Multiple System Atrophy (MSA) at the American Academy of Neurology Annual Meeting in San Diego (April 5-9, 2025).
The presentations include an oral session featuring topline data from the randomized, double-blind, placebo-controlled Phase 2 study, to be presented by Dr. Daniel Claassen from Vanderbilt University Medical Center. Additionally, a poster presentation will discuss wearable sensor data correlation with clinical scores in early-stage MSA patients.
ATH434 is an oral agent designed to inhibit pathological protein aggregation in neurodegeneration by restoring brain iron balance. The drug has received Orphan Drug Designation for MSA treatment from both the FDA and European Commission. Besides the completed Phase 2 trial showing positive results, a second Phase 2 open-label biomarker trial in advanced MSA patients is currently ongoing.
Alterity Therapeutics (NASDAQ: ATHE) ha annunciato le prossime presentazioni dei risultati del suo trial clinico di fase 2 ATH434 per l'Atrofia Sistemica Multipla (MSA) durante l'Annual Meeting dell'Accademia Americana di Neurologia a San Diego (dal 5 al 9 aprile 2025).
Le presentazioni includeranno una sessione orale con i dati principali dello studio di fase 2, randomizzato, in doppio cieco e controllato con placebo, che sarà presentata dal Dr. Daniel Claassen del Vanderbilt University Medical Center. Inoltre, una presentazione poster discuterà la correlazione dei dati dei sensori indossabili con i punteggi clinici nei pazienti con MSA in fase iniziale.
ATH434 è un agente orale progettato per inibire l'aggregazione proteica patologica nella neurodegenerazione ripristinando l'equilibrio del ferro nel cervello. Il farmaco ha ricevuto la Designazione di Farmaco Orfano per il trattamento della MSA sia dalla FDA che dalla Commissione Europea. Oltre al trial di fase 2 completato che ha mostrato risultati positivi, attualmente è in corso un secondo trial di fase 2 con biomarcatori in pazienti con MSA avanzata.
Alterity Therapeutics (NASDAQ: ATHE) ha anunciado las próximas presentaciones de los resultados de su ensayo clínico de fase 2 ATH434 para la Atrofia Sistémica Múltiple (MSA) en la Reunión Anual de la Academia Americana de Neurología en San Diego (del 5 al 9 de abril de 2025).
Las presentaciones incluirán una sesión oral con los datos principales del estudio de fase 2, aleatorizado, doble ciego y controlado con placebo, que será presentada por el Dr. Daniel Claassen del Vanderbilt University Medical Center. Además, se presentará un póster que discutirá la correlación de los datos de sensores portátiles con las puntuaciones clínicas en pacientes con MSA en etapas tempranas.
ATH434 es un agente oral diseñado para inhibir la agregación proteica patológica en la neurodegeneración al restaurar el equilibrio de hierro en el cerebro. El medicamento ha recibido la Designación de Medicamento Huérfano para el tratamiento de la MSA tanto de la FDA como de la Comisión Europea. Además del ensayo de fase 2 completado que mostró resultados positivos, actualmente se está llevando a cabo un segundo ensayo de fase 2 abierto con biomarcadores en pacientes con MSA avanzada.
Alterity Therapeutics (NASDAQ: ATHE)는 ATH434 2상 임상 시험의 다계통 위축증(MSA) 결과를 샌디에이고에서 열리는 미국 신경학회 연례 회의(2025년 4월 5-9일)에서 발표할 예정이라고 발표했습니다.
발표에는 랜덤화, 이중 맹검, 위약 대조 2상 연구의 주요 데이터가 포함된 구두 세션이 있으며, 이는 밴더빌트 대학교 의료센터의 다니엘 클라센 박사가 발표할 예정입니다. 또한, 포스터 발표에서는 초기 MSA 환자에서 임상 점수와 웨어러블 센서 데이터 간의 상관관계에 대해 논의할 것입니다.
ATH434는 신경퇴행성 질환에서 병리학적 단백질 응집을 억제하도록 설계된 경구 투여제입니다. 이 약물은 MSA 치료를 위해 FDA와 유럽연합으로부터 고아약 지정(Ophan Drug Designation)을 받았습니다. 긍정적인 결과를 보여준 완료된 2상 시험 외에도, 현재 진행 중인 MSA 말기 환자를 위한 두 번째 2상 공개 바이오마커 시험이 있습니다.
Alterity Therapeutics (NASDAQ: ATHE) a annoncé les prochaines présentations des résultats de son essai clinique de phase 2 ATH434 pour l'Atrophie Systémique Multiple (MSA) lors de la Réunion Annuelle de l'Académie Américaine de Neurologie à San Diego (du 5 au 9 avril 2025).
Les présentations comprendront une session orale présentant les données principales de l'étude de phase 2, randomisée, en double aveugle et contrôlée par placebo, qui sera présentée par le Dr. Daniel Claassen du Vanderbilt University Medical Center. De plus, une présentation par affiche discutera de la corrélation des données des capteurs portables avec les scores cliniques chez les patients atteints de MSA à un stade précoce.
ATH434 est un agent oral conçu pour inhiber l'agrégation protéique pathologique dans la neurodégénérescence en restaurant l'équilibre du fer dans le cerveau. Le médicament a reçu la désignation de médicament orphelin pour le traitement de la MSA à la fois de la FDA et de la Commission Européenne. En plus de l'essai de phase 2 complété montrant des résultats positifs, un deuxième essai de phase 2 à étiquetage ouvert sur des biomarqueurs chez des patients atteints de MSA avancée est actuellement en cours.
Alterity Therapeutics (NASDAQ: ATHE) hat die bevorstehenden Präsentationen der Ergebnisse seiner ATH434 Phase 2 klinischen Studie zur Multisystematrophie (MSA) auf dem Jahreskongress der American Academy of Neurology in San Diego (5.-9. April 2025) angekündigt.
Die Präsentationen umfassen eine mündliche Sitzung mit den Hauptdaten der randomisierten, doppelblinden, placebokontrollierten Phase-2-Studie, die von Dr. Daniel Claassen vom Vanderbilt University Medical Center präsentiert wird. Darüber hinaus wird eine Posterpräsentation die Korrelation von tragbaren Sensordaten mit klinischen Scores bei MSA-Patienten im Frühstadium erörtern.
ATH434 ist ein orales Mittel, das entwickelt wurde, um die pathologische Proteinaggregation bei neurodegenerativen Erkrankungen zu hemmen, indem es das Eisen-Gleichgewicht im Gehirn wiederherstellt. Das Medikament erhielt die Orphan Drug Designation für die Behandlung von MSA sowohl von der FDA als auch von der Europäischen Kommission. Neben der abgeschlossenen Phase-2-Studie, die positive Ergebnisse zeigte, läuft derzeit eine zweite Phase-2-Studie mit offenen Biomarkern bei fortgeschrittenen MSA-Patienten.
- Positive topline results from Phase 2 clinical trial in early-stage MSA
- Orphan Drug Designation granted by both FDA and European Commission
- Multiple ongoing clinical development programs for different MSA stages
- None.
Insights
Alterity's announcement of positive Phase 2 results for ATH434 in Multiple System Atrophy (MSA) represents a significant milestone for this small-cap biotech. The upcoming oral presentation at the American Academy of Neurology Annual Meeting elevates the visibility of these results within the scientific community.
The clinical trial design is particularly robust - randomized, double-blind, and placebo-controlled - which strengthens the credibility of their positive findings. This methodological rigor is essential when evaluating novel treatments for complex neurodegenerative disorders.
Their development strategy demonstrates comprehensiveness by simultaneously running a second Phase 2 open-label biomarker trial in more advanced MSA patients. This two-pronged approach allows them to gather complementary datasets across the disease spectrum.
The Orphan Drug Designation from both FDA and European Commission provides substantial strategic advantages, including extended market exclusivity upon approval (7 years US, 10 years EU), tax credits for clinical trials, and potential regulatory fee reductions - critical benefits for a company with Alterity's market capitalization (
ATH434's mechanism targeting α-synuclein pathology via iron regulation represents a differentiated approach in the neurodegenerative space. Their exploration of wearable sensor technology for patient assessment also shows innovation in endpoint measurement, potentially providing more objective and continuous efficacy data.
Multiple System Atrophy is a rare, progressive neurodegenerative disorder characterized by autonomic dysfunction, parkinsonism, and cerebellar ataxia. With an estimated prevalence of 3-4 per 100,000 population, MSA represents a significant unmet medical need with no disease-modifying treatments currently available.
ATH434's mechanism addressing iron dyshomeostasis and α-synuclein accumulation tackles fundamental pathophysiological processes in MSA. Iron accumulation contributes to oxidative stress and neuronal damage, while α-synuclein aggregation forms characteristic glial cytoplasmic inclusions - hallmarks of MSA pathology.
The incorporation of wearable sensor technology is particularly valuable for MSA assessment, as traditional clinical rating scales may not capture subtle changes in motor function. This technology could provide more sensitive measures of disease progression and treatment response, addressing a key challenge in MSA clinical trials.
The positive Phase 2 results suggest ATH434 may be affecting disease pathology rather than just providing symptomatic relief. If substantiated in larger trials, this would represent a significant advance for MSA patients who currently only receive symptomatic management.
While encouraging, these results will need confirmation in larger Phase 3 studies. The path from Phase 2 to approval remains challenging, particularly for neurodegenerative conditions. However, for a devastating disorder with no disease-modifying treatments, even modest efficacy could prove clinically meaningful.
MELBOURNE, Australia and SAN FRANCISCO, April 03, 2025 (GLOBE NEWSWIRE) -- Alterity Therapeutics (ASX: ATH, NASDAQ: ATHE) (“Alterity” or “the Company”), a biotechnology company dedicated to developing disease modifying treatments for neurodegenerative diseases, today announced that an oral presentation and a poster presentation related to Alterity’s clinical programs in Multiple System Atrophy (MSA) will be delivered at the American Academy of Neurology (AAN) 2025 Annual Meeting taking place April 5 - 9, 2025 in San Diego, CA.
David Stamler, M.D., Chief Executive Officer of Alterity, commented, “We are excited to present the positive topline data along with new analyses from our ATH434-201 clinical trial via an oral presentation at AAN, one of the premier global neurology meetings. In addition, data will be presented on the use of wearable sensor technology to assess patient outcomes, an important component of evaluating novel treatments for individuals with MSA.”
| Type: | Oral Presentation |
| Title: | Topline Data from a Randomized, Double Blind, Placebo Controlled Phase 2 Study of ATH434 in Multiple System Atrophy |
| Presenter: | Daniel Claassen, M.D., M.S., Professor of Neurology at Vanderbilt University Medical Center |
| Date/Time: | Wednesday, April 9, 2025 at 2:24 PM PDT (USA) |
| Type: | Poster Presentation |
| Title: | Association Between Wearable Sensor Data and Clinical Scores in Individuals with Early-stage Multiple System Atrophy |
| Presenter: | Ashkan Vaziri, PhD, BioSensics LLC |
| Date/Time: | Saturday, April 5, 2025 at 11:45 AM PDT (USA) |
About ATH434
Alterity’s lead candidate, ATH434, is an oral agent designed to inhibit the aggregation of pathological proteins implicated in neurodegeneration. ATH434 has been shown preclinically to reduce α-synuclein pathology and preserve neuronal function by restoring normal iron balance in the brain. As an iron chaperone, it has excellent potential to treat Parkinson’s disease as well as various Parkinsonian disorders such as Multiple System Atrophy (MSA). ATH434 successfully completed Phase 1 studies demonstrating the agent is well tolerated and achieved brain levels comparable to efficacious levels in animal models of MSA. ATH434 recently announced positive results from the randomized, double-blind, placebo-controlled Phase 2 clinical trial in patients with early-stage MSA. A second Phase 2 open-label 2 Biomarker trial in patients with more advanced MSA is ongoing. ATH434 has been granted Orphan Drug Designation for the treatment of MSA by the U.S. FDA and the European Commission.
About ATH434-201 Phase 2 Clinical Trial
The ATH434-201 Phase 2 clinical trial is a randomized, double-blind, placebo-controlled investigation of 12 months treatment with ATH434 in patients with MSA. The study evaluated the efficacy, safety and pharmacokinetics of ATH434 as well as the effect of ATH434 on neuroimaging and protein biomarkers. Wearable sensors were employed to evaluate motor activities outside of the clinic. The study enrolled 77 adults who were randomly assigned to receive ATH434 50 mg or 75 mg twice daily or matching placebo. The topline data showed that ATH434 produced clinically and statistically significant improvement on the modified UMSARS Part I, a functional rating scale that assesses disability on activities of daily living affected in MSA. In addition to the robust efficacy demonstrated on the UMSARS Part I, trends in improved motor performance were observed on the Parkinson’s Plus rating scale and overall benefit was shown on the Clinical Global Impression of Severity at the 50 mg dose. Wearable sensor data indicated that both dose levels of ATH434 led to increased activity in an outpatient setting as compared to placebo. Biomarkers were used to evaluate potential drug effect and target engagement. Both dose levels reduced iron accumulation in MSA affected brain regions and trends in preservation of brain volume were observed relative to placebo. Additional information on the Phase 2 trial can be found by ClinicalTrials.gov Identifier: NCT05109091.
About Multiple System Atrophy
Multiple System Atrophy (MSA) is a rare, neurodegenerative disease characterized by failure of the autonomic nervous system and impaired movement. The symptoms reflect the progressive loss of function and death of different types of nerve cells in the brain and spinal cord. It is a rapidly progressive disease and causes profound disability. MSA is a Parkinsonian disorder characterized by a variable combination of slowed movement and/or rigidity, autonomic instability that affects involuntary functions such as blood pressure maintenance and bladder control, and impaired balance and/or coordination that predisposes to falls. A pathological hallmark of MSA is the accumulation of the protein α-synuclein within glia, the support cells of the central nervous system, and neuron loss in multiple brain regions. MSA affects at least 15,000 individuals in the U.S., and while some of the symptoms of MSA can be treated with medications, currently there are no drugs that are able to slow disease progression and there is no cure.1
1Multiple System Atrophy | National Institute of Neurological Disorders and Stroke (nih.gov)
About Alterity Therapeutics Limited
Alterity Therapeutics is a clinical stage biotechnology company dedicated to creating an alternate future for people living with neurodegenerative diseases. The Company is initially focused on developing disease modifying therapies in Parkinson’s disease and related disorders. Alterity recently reported positive data for its lead asset, ATH434, in a Phase 2 clinical trial in participants with Multiple System Atrophy (MSA), a rare and rapidly progressive Parkinsonian disorder. ATH434 is also being evaluated in a Phase 2 clinical trial in advanced MSA. In addition, Alterity has a broad drug discovery platform generating patentable chemical compounds to treat the underlying pathology of neurological diseases. The Company is based in Melbourne, Australia, and San Francisco, California, USA. For further information please visit the Company’s website at www.alteritytherapeutics.com.
Authorisation & Additional information
This announcement was authorized by David Stamler, CEO of Alterity Therapeutics Limited.
Investor and Media Contacts:
Australia
Ana Luiza Harrop
we-aualteritytherapeutics@we-worldwide.com
+61 452 510 255
U.S.
Remy Bernarda
remy.bernarda@iradvisory.com
+1 (415) 203-6386
Forward Looking Statements
This press release contains "forward-looking statements" within the meaning of section 27A of the Securities Act of 1933 and section 21E of the Securities Exchange Act of 1934. The Company has tried to identify such forward-looking statements by use of such words as "expects," "intends," "hopes," "anticipates," "believes," "could," "may," "evidences" and "estimates," and other similar expressions, but these words are not the exclusive means of identifying such statements.
Important factors that could cause actual results to differ materially from those indicated by such forward-looking statements are described in the sections titled “Risk Factors” in the Company’s filings with the SEC, including its most recent Annual Report on Form 20-F as well as reports on Form 6-K, including, but not limited to the following: statements relating to the Company's drug development program, including, but not limited to the initiation, progress and outcomes of clinical trials of the Company's drug development program, including, but not limited to, ATH434, and any other statements that are not historical facts. Such statements involve risks and uncertainties, including, but not limited to, those risks and uncertainties relating to the difficulties or delays in financing, development, testing, regulatory approval, production and marketing of the Company’s drug components, including, but not limited to, ATH434, the ability of the Company to procure additional future sources of financing, unexpected adverse side effects or inadequate therapeutic efficacy of the Company's drug compounds, including, but not limited to, ATH434, that could slow or prevent products coming to market, the uncertainty of obtaining patent protection for the Company's intellectual property or trade secrets, the uncertainty of successfully enforcing the Company’s patent rights and the uncertainty of the Company freedom to operate.
Any forward-looking statement made by us in this press release is based only on information currently available to us and speaks only as of the date on which it is made. We undertake no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise.
FAQ
What are the key presentations for ATHE's ATH434 at the AAN 2025 meeting?
What regulatory designations has ATH434 received for MSA treatment?
How does ATHE's ATH434 work in treating Multiple System Atrophy?
What clinical trials are currently ongoing for ATHE's ATH434?
