USFDAJun 9, 2026, 06:07 AM
Alterity & FDA Agree on Pivotal Phase 3 Design for ATH434 in MSA
AI Summary
Alterity Therapeutics announced successful alignment with the U.S. FDA on the key elements of its registrational Phase 3 program for ATH434 in Multiple System Atrophy (MSA). The FDA agreed on the proposed trial design, including the study population, treatment duration, and the 11-item UMSARS Part I rating scale as the primary endpoint. The 50 mg twice daily dose, which demonstrated 48% slowing of disease progression in Phase 2, was also concurred upon. This agreement de-risks the development pathway, with pivotal Phase 3 trial activities expected to commence by year-end 2026.
Key Highlights
- Alterity and FDA aligned on pivotal Phase 3 program for ATH434 in Multiple System Atrophy (MSA).
- FDA agreed on study population, 50mg twice daily dosing regimen, and treatment duration.
- Primary endpoint for Phase 3 will be the 11-item UMSARS Part I rating scale.
- Phase 2 study showed 48% slowing of disease progression vs. placebo with 50mg dose.
- Key secondary endpoints include Swallowing Disturbance Questionnaire and Clinical Global Impression of Severity.
- Pivotal Phase 3 trial activities are on track to initiate by year-end 2026.
- ATH434 holds FDA Fast Track and Orphan Drug Designations for MSA.
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