Alterity confirms FDA-aligned Phase 3 design for ATH434 in MSA, reports A$41.3M cash

Alterity Therapeutics released its Appendix 4C quarterly cash flow report and corporate update for the quarter ending September 30, 2026, on October 30, 2026. The company said it received minutes during the quarter from the FDA's End-of-Phase 2 meeting regarding the Phase 3 study design and registrational pathway for a potential NDA for ATH434 in MSA, with the FDA agreeing with the proposed design including study population, treatment regimen, and the primary endpoint, the 11-item UMSARS Part I rating scale.¹ The FDA indicated a single pivotal trial plus confirmatory evidence could support approval, and the company expects data from its ATH434-201 Phase 2 trial to serve as that confirmatory evidence.¹

The planned Phase 3 trial calls for roughly 200 patients randomized evenly between ATH434 at 50 mg or a matching placebo, dosed twice a day over a 12-month period.¹ Alterity said it has started operational work for Phase 3, covering protocol finalization, vendor selection, site identification and qualification, and manufacture of the first registration batch of ATH434 for the pivotal study.¹

At an October 2026 poster presentation at the MDS Congress, new analyses using the statistical methods planned for Phase 3 showed ATH434 50 mg slowed functional decline on the 11-item UMSARS1 by about 52% versus placebo at Week 52.¹

In August 2026, the USPTO granted a new composition of matter patent covering the crystalline mesylate salt form of ATH434 used in its Phase 2 trials and planned for Phase 3, with an expiry in 2045 and expected Orange Book listing upon approval.¹

Financially, Alterity held A$41.3 million in cash as of September 30, 2026, with operating cash outflows of A$1.34 million for the quarter.¹ The company raised approximately A$5.5 million after costs through an underwriting agreement with MST Financial Services Pty Ltd covering the exercise of ATHO listed options.¹