Alterity Therapeutics

Alterity Therapeutics presented encouraging Phase II data for ATH434, with the 50 mg dose slowing progression of multiple system atrophy by 48% on the UMSARS‑Part‑1 scale. The FDA indicated that a single pivotal Phase III study, supported by these data, could be sufficient for an approval filing; the protocol is largely complete and the study is planned to test 50 mg over 12 months versus placebo. Alterity has produced initial drug batches, plans to begin recruitment in 2025 and expects a study duration of around 2.5 years, but still requires additional funding despite approximately AUD 30 million in cash. A new patent would extend protection for ATH434 to 2045 and, the company says, improves the commercial prospects, including potential use in Parkinson’s disease. » Więcej na de.investing.com

Alterity Therapeutics presented phase‑II data on ATH434 that the company says slowed progression of multiple system atrophy (MSA) versus placebo by 34–46%. The firm has agreed with the FDA on the study design, the primary endpoint (UMSARS) and a dose of 50 mg twice daily, and plans a randomized 12‑month phase‑III study with around 200 patients. Secondary endpoints, biomarkers and imaging also signal positive effects, and the safety profile was comparable to placebo. Management sees large commercial potential given the lack of an approved therapy and orphan/priority status (conservatively estimated peak sales of USD 2.4bn), but warns of the usual late‑stage development risks given a market capitalization of about USD 80m. » Więcej na de.investing.com

A peer-reviewed study in NeuroImage shows that quantitative susceptibility mapping (QSM) can detect iron deposits in patients with multiple system atrophy (MSA). Analyses of data from the bioMUSE study found higher iron content in the lentiform nucleus in MSA patients; iron levels in the globus pallidus distinguished MSA from Parkinson with an accuracy of 0.76–0.79 and correlated with disease severity. Alterity has already used QSM in a Phase 2 study of ATH434 and is advancing development into a Phase 3 program; ATH434 has Fast Track and Orphan Drug status, and the company plans an end-of-Phase-2 meeting with the FDA in mid‑2026. Investor reaction: stock up 37.5% year-to-date, market capitalization about $86.5 million. » Więcej na de.investing.com

Alterity Therapeutics presented positive phase-2 data on ATH434 at the AAN meeting, showing a slowing of functional decline in multiple system atrophy on the new MuSyCA scale (relative treatment effect up to 41% at 50 mg, p=0.034). Alternative analyses on the modified UMSARS‑I confirmed significant effects (up to 53% relative, p=0.029). The results recently pushed the share price markedly higher and the company is preparing a registration‑enabling phase‑3 trial; ATH434 has Fast‑Track and orphan‑drug status. » Więcej na de.investing.com