Alterity Therapeutics (ASX:ATH) is set to approach the FDA for fast-track approval of its ATH-434 treatment for multiple system atrophy (MSA) after reporting positive top-line Phase II trial results. The study, involving 77 patients with early-stage MSA, demonstrated statistically significant outcomes, with up to 48% showing a slowing of the disease's progression.
Promising Results from Phase II Trial
The Phase II trial assessed the efficacy of ATH-434 in slowing the progression of MSA, a Parkinsonian disorder affecting approximately 50,000 individuals in the US. The results indicated that a 50-milligram dose of ATH-434 led to a 48% slowing of clinical progression at week 52, as measured by the Unified Multiple System Atrophy Rating Scale (UMSARS), a tool used to assess patients' ability to perform daily activities. Interestingly, a 75mg dose showed a 62% slowing at week 26, but only a 29% response at week 52. According to Alterity chief Dr. David Stamler, the reasons for this are not definitively known and will be explored further.
Mechanism of Action and Biomarker Data
ATH-434 is designed to target excess iron in the brain, which is believed to cause neurons to clump and lose their ability to communicate. The drug acts as a chaperone, facilitating the removal of excess iron to less harmful areas of the body. Biomarker results from the trial suggest that ATH-434 reduces iron accumulation in MSA-affected regions of the brain and preserves brain volume.
Expert Commentary and Future Plans
Dr. Stamler expressed enthusiasm about the trial results, highlighting the drug's safety profile and significant impact on slowing clinical progression. Professor Daniel Claassen from Vanderbilt University Medical Centre, the trial’s lead investigator, described the results as "incredibly compelling". Alterity plans to engage with the FDA to discuss accelerating the development of ATH-434, given the substantial unmet need for effective MSA treatments.
Potential Applications Beyond MSA
Beyond MSA, ATH-434 may also have relevance for other neurological diseases, including Friedreich’s ataxia, Parkinson’s disease, and Alzheimer’s disease.
