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Alterity Marks Completion of Patient Dosing in Landmark Study for Rare Neurodegenerative Disease
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Melbourne and San Francisco-based biotech Alterity Therapeutics Ltd (ASX: ATH; NASDAQ: ATHE) has announced a major step forward in its mission to combat rare neurodegenerative diseases. On March 27, the company confirmed the completion of all patient visits in its ATH434-202 open-label Phase 2 clinical trial for individuals with advanced Multiple System Atrophy (MSA), a devastating and rapidly progressive Parkinsonian disorder.
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The study enrolled 10 patients, each treated with Alterity’s lead drug candidate ATH434 over a 12-month period. With all clinical evaluations now finalised, the company is set to deliver topline data by mid-2025—data that could shape the future of treatment for MSA, which currently lacks any disease-modifying therapies.
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Dr. David Stamler, CEO of Alterity, highlighted the importance of the development: “The 202 study gives us the opportunity to evaluate the effects of ATH434 treatment in a population that faces severe challenges due to the stage of their illness. We are incredibly grateful to the participants and look forward to reporting data that will help guide our development strategy moving forward.”
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Dual Phase 2 Strategy Aims to Establish ATH434 as a First-in-Class Treatment
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ATH434 is a novel small molecule designed to inhibit the aggregation of toxic α-synuclein proteins—pathological hallmarks of MSA and related disorders—by restoring iron balance in the brain. The drug has already demonstrated encouraging safety and efficacy outcomes in a previous randomized, double-blind, placebo-controlled Phase 2 trial for early-stage MSA patients. The current open-label study (NCT05864365) is specifically targeting individuals with more advanced disease.
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The primary focus of this 12-month trial is to assess ATH434’s impact on brain volume and key biomarkers, including iron levels and protein aggregation, using neuroimaging and biochemical techniques. Secondary objectives include safety, pharmacokinetics, and clinical measures to evaluate disease progression.
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With ATH434 already granted Orphan Drug Designation by both the U.S. FDA and the European Commission, success in this trial could fast-track regulatory pathways and commercial interest. The candidate’s ability to demonstrate disease-modifying potential in both early and advanced stages of MSA places Alterity in a unique and potentially lucrative position in the neurodegeneration market.
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MSA remains a high-unmet-need condition with an estimated 15,000 affected individuals in the U.S. alone. Characterised by autonomic dysfunction, rigidity, and severe motor impairment, the disease progresses rapidly and currently has no cure. If successful, ATH434 could be the first therapy to slow its advance.
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Despite its microcap status—market capitalisation currently sits at $63.2 million with over 6.6 billion shares issued—Alterity has delivered a remarkable 137.5% return over the past year. Trading at $0.01, the stock has shown resilience amid broader market volatility and continues to attract investor interest, outperforming both its healthcare sector peers and the ASX 200 benchmark
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