Alterity Therapeutics (ASX:ATH) reported positive topline results from the randomized, double-blind, placebo-controlled phase two clinical trial following 12 months of treatment using the ATH434 drug candidate in patients with early-stage multiple system atrophy, according to a Thursday Australian bourse filing.
ATH434 is designed to inhibit the aggregation of pathological proteins implicated in neurodegeneration, the filing said.
Topline data showed that the candidate produced clinically and statistically significant improvement on the modified Unified MSA Rating Scale Part I (UMSARS I), a functional rating scale that assesses disability in activities of daily living affected by multiple system atrophy.
It demonstrated a 48% slowing of clinical progression at the 50-milligram dose and a 29% slowing of clinical progression at the 75-mg dose at the end of 52 weeks when compared with a placebo.
The firm said it also observed trends of improved motor performance on the Parkinson's Plus rating scale.
It observed trends in preservation of brain volume were observed in the groups that received the 50 mg and 75 mg doses, relative to placebo at both 26 and 52 weeks of treatment.
The 50 mg dose reduced iron accumulation in multiple system atrophy-affected brain regions and the 75 mg dose reduced iron accumulation in the globus pallidus.
The firm's shares soared 125% in recent trade on Thursday, the highest point since Sept. 23, 2022.
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