Alterity Therapeutics (NASDAQ: ATHE) shares moved up at warp-speed Thursday, on announcing positive topline results from the ATH434-201 randomized, double-blind, placebo-controlled Phase 2 clinical trial in patients with early-stage multiple system atrophy (MSA).
The topline data showed that ATH434 produced clinically and statistically significant improvement on the modified UMSARS Part I, a functional rating scale that assesses disability on activities of daily living affected in MSA1. On this important clinical measure, ATH434 demonstrated 48% slowing of clinical progression at the 50 mg dose (p=0.03)^ and 29% slowing of clinical progression at the 75 mg dose (p=0.2) at Week 52 when compared with placebo. The 75 mg dose group showed a 62% slowing of progression (p=0.05) at Week 26. In addition to the robust efficacy demonstrated on the UMSARS I, trends of improved motor performance were observed on the Parkinson’s Plus rating scale2 and overall benefit was shown on the Clinical Global Impression of Severity at the 50 mg dose (p=0.009).
Biomarkers were used to evaluate potential drug effect and target engagement. Regarding iron content by MRI, the 50 mg dose reduced iron accumulation in MSA affected brain regions (substantia nigra, putamen, and globus pallidus) and the 75 mg dose reduced iron accumulation in the globus pallidus. The reduced accumulation of iron was significant for the 50 mg dose group at 26 weeks (putamen, P=0.025) and approached statistical significance at 52 weeks.
ATHE shares leaped $2.06, or 68.9% to $5.05.
