FDA Confirms Single Pivotal Phase 3 Trial for Alterity’s ATH434, Year-End 2026 Start
ATHE•Official FDA minutes confirm Alterity’s Phase 3 protocol for ATH434 in MSA, endorsing a single pivotal trial supported by confirmatory evidence to underpin an NDA filing. The 200-patient, 12-month study (50 mg BID) with UMSARS Part II primary endpoint is on track to start by year-end 2026.
1. EOP2 Meeting Outcomes
Official FDA minutes from the End-of-Phase 2 meeting validate key elements of the registrational strategy for ATH434 in Multiple System Atrophy, including the acceptability of a single pivotal trial plus confirmatory evidence to support an NDA application.
2. Phase 3 Trial Design Details
The agreed Phase 3 program will randomize approximately 200 patients 1:1 to receive ATH434 50 mg twice daily or placebo over 12 months, with the 11-item UMSARS Part II as the primary efficacy endpoint and secondary measures including SDQ, OHSA and CGI-S.
3. Timeline and NDA Pathway
Alterity is finalizing the Phase 3 protocol and plans to initiate trial activities by year-end 2026, with an open-label extension to bolster the safety database and align with the pathway toward potential approval.
