On January 30th 2025, the pharmaceutical company Alterity Therapeutics announced positive data from their phase 2 placebo-controlled trial in early-stage MSA. MSA is a progressive neurological condition leading to impaired co-ordination, slowed movement and/or rigidity. There are two main types of MSA, MSA-P (Parkinson’s-like subtype) and MSA-C (cerebellar subtype).
In this trial, ATH434 was compared to placebo in participants with early-stage MSA, including MSA-C. People with MSA-C are often misdiagnosed with cerebellar ataxia, because the symptoms can be similar in both conditions. This trial had sites in the UK.
The data show that ATH434 produced significant improvement on a number of clinical scales, and slowing of disease progression compared to placebo.
The data showed significant improvement on the modified UMSARS Part I, a functional rating scale that assesses disability on activities of daily living affected in MSA. On this important clinical measure, ATH434 demonstrated 48% slowing of clinical progression at the 50 mg dose and 29% slowing of clinical progression at the 75 mg dose at Week 52 when compared with placebo. Trends of improved motor performance were observed on the Parkinson’s Plus rating scale and overall benefit was shown on the Clinical Global Impression of Severity at the 50 mg dose.
Read the press release here.
