Alterity Therapeutics Recognizes Multiple System Atrophy (MSA) Awareness Month in March and the Urgent Need for Disease-Modifying Treatments
– Company is advancing ATH434 in late-stage clinical development for Multiple System Atrophy, a rapidly progressive neurodegenerative disease with no approved therapies –
MELBOURNE, Australia and SAN FRANCISCO, March 02, 2026 (GLOBE NEWSWIRE) -- Alterity Therapeutics ((ASX: ATH, NASDAQ:ATHE) ("Alterity" or "the Company"), a biotechnology company dedicated to developing disease modifying treatments for neurodegenerative diseases, recognizes Multiple System Atrophy (MSA) Awareness Month this March and the importance of raising awareness for a devastating and often misunderstood rare neurological disorder with no approved treatment options.
MSA is a progressive neurodegenerative disease characterized by a combination of motor impairment, autonomic dysfunction, and rapid loss of independence. Symptoms can include problems with balance, coordination, movement, and blood pressure regulation, and the disease typically progresses quickly following diagnosis. Because early symptoms can resemble Parkinson's disease, MSA is often difficult to diagnose in its initial stages, contributing to delays in care. Currently, treatment options only address symptom management, as there are no approved disease-modifying therapies available.
"MSA Awareness Month is an important opportunity to recognize the resilience of patients and care partners living with this devastating condition and to highlight the urgent need for therapies that can change the course of disease," said David Stamler, M.D., Chief Executive Officer of Alterity Therapeutics. "At Alterity, we are committed to advancing research that targets the underlying pathology of MSA and are very encouraged by our data that demonstrate the potential of ATH434 as a disease-modifying therapy. We are grateful to the patients, families, and clinical investigators who make this work possible, and we remain focused on developing treatments that may offer hope for slowing disease progression and improving quality of life."
Alterity is advancing ATH434, an investigational oral therapy designed to redistribute excess iron in the brain, which contributes to the aggregation, or clumping, of the α-synuclein protein and the progression of neurodegeneration in MSA. The Company has reported data from its Phase 2 clinical development program evaluating ATH434 in individuals with MSA, demonstrating meaningful clinical benefit alongside favorable safety and tolerability profiles.
The Company's clinical program has featured the incorporation of biomarkers and advanced neuroimaging to better characterize disease progression and assess therapeutic impact. These efforts are intended to support the development of disease-modifying approaches and contribute to a deeper understanding of this complex and devastating condition.
Throughout MSA Awareness Month, Alterity will share educational information about the disease on its social media accounts, highlight the experiences of the MSA community, and underscore the importance of continued research to address the significant unmet need for effective treatments. Alterity is committed to supporting patient advocacy efforts and organizations working to raise awareness and improve outcomes for the MSA community.
About ATH434
Alterity's lead candidate, ATH434, is an oral agent designed to reduce iron accumulation and inhibit abnormal protein aggregation associated with neurodegeneration. ATH434 has been shown 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). Positive results from the randomized, double-blind, placebo-controlled Phase 2 clinical trial in patients with MSA demonstrated robust clinical efficacy, target engagement as indicated by key biomarkers, and a favorable safety profile. Positive data from a second Phase 2 open-label biomarker trial in patients with more advanced MSA reinforced these results. ATH434 has been granted Fast Track Designation by the U.S. Food and Drug Administration (FDA), and Orphan Drug Designation by the FDA and the European Commission for the treatment of MSA.
About ATH434-201 Phase 2 Clinical Trial
The ATH434-201 Phase 2 clinical trial was 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 and assessed neuroimaging measures of brain volume and iron-related parameters, together with 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 used to assess outpatient activity levels indicated that patients receiving ATH434 also experienced less decline compared with placebo. Biomarkers were used to evaluate potential drug effect and target engagement relative to placebo. Both dose levels 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 at 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 that causes profound disability. MSA is a Parkinsonian disorder characterized by a variable combination of slowed movement and/or rigidity, autonomic dysfunction affecting involuntary functions such as blood pressure maintenance and bladder control, and impaired balance and/or coordination that predispose patients to falls. A pathological hallmark of MSA is the accumulation of abnormal clumping of the protein α-synuclein within oligodendrocytes, the myelin-producing support cells of the central nervous system, along with progressive neuronal loss in multiple brain regions. MSA affects up to 50,000 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 focused on developing disease modifying therapies in Multiple System Atrophy (MSA) and related Parkinsonian disorders. Alterity is preparing to initiate a Phase 3 pivotal trial in MSA, a rare and rapidly progressive disease. ATH434, the Company's lead asset, has demonstrated clinically meaningful efficacy in a randomized, double-blind, placebo-controlled Phase 2 clinical trial in participants with MSA. Alterity has further reported positive data in its open label Phase 2 clinical trial in participants with 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.
Authorization & Additional information
This announcement was authorized by David Stamler, CEO of Alterity Therapeutics Limited.
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