Alterity Unveils Promising Phase 2 Data for ATH434 at International MSA Congress
Alterity Therapeutics is set to present compelling Phase 2 clinical trial results for ATH434 at the 2025 International Multiple System Atrophy Congress, highlighting slowed disease progression and biomarker engagement in MSA patients.
- Positive Phase 2 data for ATH434 showing slowed progression in Multiple System Atrophy
- Presentations include oral and poster sessions at the 2025 International MSA Congress
- ATH434 granted Fast Track and Orphan Drug Designations by US FDA and European Commission
- Ongoing bioMUSE natural history study collaboration with Vanderbilt University Medical Center
- Clinical data demonstrates safety, efficacy, and biomarker target engagement
Alterity’s Growing Presence at the 2025 MSA Congress
Alterity Therapeutics (ASX: ATH, NASDAQ: ATHE) is taking centre stage at the upcoming International Multiple System Atrophy (MSA) Congress in Boston, scheduled for May 9-11, 2025. The company will showcase multiple oral and poster presentations that underscore its advancing clinical programs targeting this rare and debilitating neurodegenerative disorder.
Chief Executive Officer Dr. David Stamler will present the headline data from the ATH434-201 Phase 2 clinical trial during the opening plenary session. This pivotal study demonstrated that ATH434, an oral agent designed to inhibit pathological protein aggregation, significantly slowed disease progression in MSA patients. The presentation is highly anticipated by clinicians, researchers, and patient advocates alike, given the urgent need for disease-modifying therapies in this space.
Clinical Efficacy and Biomarker Insights
The Phase 2 trial enrolled 77 adults with MSA and employed a rigorous randomized, double-blind, placebo-controlled design over 12 months. ATH434 showed statistically significant improvement on the modified Unified Multiple System Atrophy Rating Scale (UMSARS) Part I, which assesses daily living disability. Additional measures, including motor performance and patient-reported outcomes, aligned with these positive findings.
Importantly, biomarker analyses revealed that ATH434 stabilized or reduced iron accumulation in affected brain regions, a key pathological hallmark of MSA. Trends toward preservation of brain volume were also observed. The drug’s safety profile was favourable, with adverse events comparable to placebo and no serious events attributed to ATH434.
Supporting Research and Regulatory Momentum
Alongside the clinical data, Alterity will present findings from the bioMUSE natural history study conducted in partnership with Vanderbilt University Medical Center. This study tracks disease progression in early-stage MSA patients and informs biomarker selection and trial design, reinforcing the robustness of Alterity’s clinical approach.
The company’s efforts have been recognised by regulatory authorities, with ATH434 receiving Fast Track Designation from the US FDA and Orphan Drug Designations from both the US FDA and European Commission. These designations facilitate expedited development and regulatory review, underscoring the unmet medical need and the promise of ATH434.
Looking Ahead
Alterity’s prominent role at the MSA Congress, including sponsorship and multiple scientific presentations, signals its commitment to advancing therapies for neurodegenerative diseases. As the company progresses toward later-stage trials, the data presented will be closely scrutinised by the medical community and investors eager for breakthroughs in MSA treatment.
Bottom Line?
Alterity’s Phase 2 results position ATH434 as a leading candidate in the race to modify MSA’s relentless progression.
Questions in the middle?
- Will Phase 3 trials confirm ATH434’s efficacy and safety in larger, diverse patient populations?
- How will regulatory agencies respond to the Phase 2 data in terms of accelerated approval pathways?
- What impact will ATH434 have on the broader Parkinsonian disorder market if approved?
