Alterity Therapeutics Announces Multiple Oral and Poster Presentations to be Featured at the International MSA Congress
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Insights
Alterity's positive Phase 2 MSA data and FDA Fast Track designation strengthen its position in developing disease-modifying treatments for a rare neurological disorder.
Alterity Therapeutics is gaining scientific attention for ATH434, its lead candidate for Multiple System Atrophy (MSA), a rare and progressive neurodegenerative disorder affecting the autonomic nervous system and motor control. The company will present its clinical progress at the 2025 International MSA Congress with multiple presentations highlighting their clinical programs.
The press release references completed Phase 2 clinical trial results that reportedly demonstrated "robust clinical efficacy, target engagement on key biomarkers, and a favorable safety profile." This represents a significant milestone in ATH434's development, though specific efficacy measurements aren't detailed in this announcement.
The FDA Fast Track Designation for ATH434 is particularly noteworthy as it can accelerate both development and regulatory review processes. This designation, combined with Orphan Drug Designation from both U.S. FDA and European Commission, provides regulatory advantages including enhanced agency interactions, potential for accelerated approval pathways, and market exclusivity periods if eventually approved.
Mechanistically, ATH434's approach as an iron chaperone that reduces α-synuclein pathology addresses fundamental disease processes in MSA. This mechanism targets the pathological proteins implicated in neurodegeneration while restoring normal iron balance in the brain.
The collaboration with Vanderbilt University Medical Center on biomarker development, including the MSA Atrophy Index and studies on phosphorylated alpha-synuclein deposition, demonstrates a comprehensive approach to disease measurement. Such objective biomarkers could potentially improve diagnosis and monitoring of disease progression.
For MSA patients, who currently have no disease-modifying treatment options, these developments represent potential hope. The company's dual clinical approach—with both the completed Phase 2 trial and ongoing biomarker study in more advanced patients—provides complementary data streams to inform future development decisions.
ATH434 Phase 2 Data to be Presented at Opening Plenary Session
MELBOURNE, Australia and SAN FRANCISCO, May 07, 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 multiple oral and poster presentations related to Alterity’s clinical programs in Multiple System Atrophy (MSA) will be featured at the 2025 International MSA Congress taking place May 9 - 11, 2025 in Boston, MA, USA. The Congress is presented by Mission MSA, a non-profit organization dedicated to improving the quality of life and building hope for people affected by MSA through support services, educational resources, research funding, and community engagement.
David Stamler, M.D., Chief Executive Officer of Alterity, commented, “We are excited to play a prominent role at the MSA Congress which brings together a noteworthy group of physicians and scientists as well as patients and care partners, all of whom are focused on advancing MSA research and care. Mission MSA is one of the primary patient education and advocacy organizations for the disease in the U.S., and we are also proud to support the event as a sponsor. We look forward to presenting our double-blind Phase 2 data for ATH434 as well as our bioMUSE natural history study with our colleagues at Vanderbilt University Medical Center. Our clinical advancements in MSA continue to generate significant interest throughout the community, bolstered by the recent Fast Track Designation for ATH434 that was granted by the US FDA.”
| Type: | Oral Presentation |
| Session: | Opening Plenary: Toward Disease Modifying Therapies of MSA |
| Title: | ATH434 Slowed Disease Progression in a Phase 2 Study in Multiple System Atrophy |
| Presenter: | David Stamler, M.D., Chief Executive Officer of Alterity |
| Date/Time: | Friday, May 9, 2025 at 9:10 AM EDT (USA) |
| Type: | Oral Presentation |
| Session: | Biomarkers in MSA: Imaging |
| Title: | MSA Atrophy Index (MSAai): A Quantitative Imaging Marker for Diagnosis and Monitoring of Multiple System Atrophy |
| Presenter: | Amy Brown, M.D., M.S., Assistant Professor, Movement Disorders Division, Department of Neurology, Vanderbilt University Medical Center |
| Date/Time: | Saturday, May 10, 2025 at 2:55 PM EDT |
| Type: | Poster Session |
| Title: | ATH434 Slowed Disease Progression in a Phase 2 Study in Multiple System Atrophy |
| Presenter: | David Stamler, M.D., Chief Executive Officer of Alterity |
| Date/Time: | Saturday, May 10, 2025 at 12:00 PM EDT |
| Poster: | #85 |
| Type: | Poster Session |
| Title: | Cutaneous Phosphorylated Alpha-Synuclein Deposition Informs Autonomic Function in Individuals with Early-Stage Multiple System Atrophy |
| Presenter: | Leah Mann, PhD, Postdoctoral Research Fellow, Vanderbilt University Medical Center |
| Date/Time: | Saturday, May 10, 2025 at 12:00 PM EDT |
| Poster: | #86 |
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 in preclinical models. 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). Phase 1 studies have demonstrated the agent is well tolerated and achieved brain levels comparable to efficacious levels in animal models of MSA. Positive results from the randomized, double-blind, placebo-controlled Phase 2 clinical trial in patients with MSA demonstrated robust clinical efficacy, target engagement on key biomarkers, and a favorable safety profile. A second Phase 2 open-label biomarker trial in patients with more advanced MSA is ongoing. ATH434 has been granted Fast Track Designation by the U.S. FDA, and Orphan Drug Designation by the U.S. FDA and the European Commission for the treatment of MSA.
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 data showed that, compared to placebo, ATH434 produced clinically and statistically significant improvement on the modified Unified Multiple System Atrophy Rating Scale (UMSARS) Part I, a functional rating scale that assesses disability on activities of daily living affected in MSA. Additional efficacy assessments demonstrated improvement consistent with the positive UMSARS Part I findings including trends in improved motor performance on the Parkinson’s Plus rating scale, the Clinical Global Impression of Severity Scale, and the Orthostatic Hypotension Symptom Assessment (a patient reported outcome). Wearable sensor data indicated that ATH434 also led to increased activity in an outpatient setting. Biomarkers were used to evaluate potential drug effect and target engagement relative to placebo. Both dose levels stabilized or reduced iron accumulation in MSA affected brain regions with trends in preservation of brain volume. ATH434 was well tolerated with similar adverse event rates compared to placebo and no serious adverse events attributed to ATH434. Additional information on the Phase 2 trial can be found by ClinicalTrials.gov Identifier: NCT05109091.
About bioMUSE
Biomarkers of progression in Multiple System Atrophy (bioMUSE) is a natural history study that aims to track the progression of individuals with MSA, a Parkinsonian disorder without approved therapy. The study is being conducted in collaboration with Vanderbilt University Medical Center in the U.S. under the direction of Daniel Claassen, M.D., M.S., Professor of Neurology and Principal Investigator. Natural history studies are important for characterizing disease progression in selected patient populations. The study has provided rich data for optimizing the design of Alterity’s randomized ATH434-201 Phase 2 clinical trial and enrolled approximately 20 individuals with clinically probable or clinically established MSA. BioMUSE continues to provide vital information on early stage MSA patients, informs the selection of biomarkers suitable to evaluate target engagement and preliminary efficacy, and delivers clinical data to characterize disease progression in a patient population that mirrors those currently enrolling in the Phase 2 clinical trial.
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
Millie Macdonald
Head of Investor Relations and Business Development
mmacdonald@alteritytherapeutics.com
+61 468 304 742
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.
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