Alterity says FDA confirmed registrational path for ATH434 in MSA

Alterity Therapeutics released its Appendix 4C report for the quarter ended 30 June 2026 (Q4 FY26) on 30 July 2026, describing progress on its lead drug ATH434 for multiple system atrophy (MSA).

The company said it reached alignment with the FDA at its End-of-Phase 2 meeting on the registrational pathway for a potential New Drug Application for ATH434 in MSA.¹ According to Alterity, the FDA indicated that one pivotal trial together with confirmatory evidence could form the basis for approval, and the company expects that data from its ongoing ATH434-201 Phase 2 trial will serve as that confirmatory evidence.¹

Design terms were also agreed: the study population, treatment regimen, and use of the 11-item UMSARS Part I rating scale as the primary endpoint were aligned with the FDA, along with key secondary endpoints and an anticipated safety database size the agency called reasonable.¹ The trial is expected to enroll roughly 200 patients randomized evenly between ATH434 at 50 mg and placebo, dosed twice a day over a one-year treatment period, and Alterity intends to offer participants who finish the trial the chance to continue into an open-label extension that would also add to the safety data.

Separately, Alterity said it received positive FDA feedback in April 2026 on chemistry, manufacturing and controls matters for the Phase 3 program, and has since manufactured its first registration batch of ATH434 for the pivotal trial.¹

Financially, Alterity held A$37.3 million in cash as of 30 June 2026, after using A$7.72 million in operating cash during the quarter.¹ After quarter-end, on 9 July 2026, the company received an Australian R&D tax refund of A$3,982,992, including A$43,117 in interest.¹