ATH434-202 - (biomarker data)

July 30, 2026: Alterity Therapeutics (ASX: ATH; NASDAQ: ATHE) reported quarterly activities and regulatory updates for ATH434 in Multiple System Atrophy (MSA). FDA End-of-Phase 2 meeting minutes confirmed a registrational pathway in which a single pivotal Phase 3 trial plus confirmatory evidence could support approval; Alterity expects confirmatory evidence to come from its ATH434-201 Phase 2 trial data. The company aligned with FDA on key Phase 3 design elements, including use of the 11-item UMSARS Part I as the primary endpoint, agreed secondary endpoints, and a reasonable planned safety database. The pivotal study is expected to enroll ~200 patients randomized 1:1 to ATH434 50 mg vs placebo twice daily for 12 months, with an open-label extension; Phase 3 trial activities are on track to initiate by year-end 2026. The company also cited positive April 2026 FDA Type C feedback on CMC and said it has manufactured the first registration batch for Phase 3.

Scientific updates included an April 2026 AAN late-breaking analysis using the MuSyCA composite showing ATH434 slowed functional decline vs placebo at Week 52, and a May 2026 NeuroImage publication validating QSM MRI iron mapping as an MSA biomarker. Alterity continues evaluating strategic funding/partnering options. Cash was A$37.3M at June 30, 2026; it later received a ~A$3.98M Australian R&D tax refund (July 9, 2026).