Alterity Therapeutics Reports Positive ATH434 Phase 2 Trial Results in MSA

January 30, 2025

DENVER, Colo., Jan 30, 2025 (247marketnews.com)- Alterity Therapeutics (NASDAQ:ATHE) reported positive Phase 2 ATH434-201 trial topline results, for patients with early-stage Multiple System Atrophy (MSA). The data highlights clinical and biomarker-based improvements that could pave the way for a promising new treatment option for MSA.

The key findings are impressive:

  • Clinical Benefit on UMSARS: ATH434 showed statistically significant slowing of disease progression as measured by the modified Unified MSA Rating Scale (UMSARS I), which is the key clinical endpoint. At the 50 mg dose, there was a 48% slowing of clinical progression over 52 weeks, and the 75 mg dose showed a 62% slowing at 26 weeks, both of which demonstrate substantial potential for improving the quality of life for MSA patients.
  • Biomarker Evidence of Target Engagement: The reduction in iron accumulation in affected brain regions (such as the substantia nigra and putamen) at both the 50 mg and 75 mg doses suggests that ATH434 is directly addressing one of the hallmark pathophysiological features of MSA. The MRI data also showed trends of brain volume preservation, further supporting the drug’s neuroprotective effects.
  • Motor and Symptom Improvements: Although the 75 mg dose didn’t show statistically significant results in all secondary motor performance measures, there were clear trends suggesting improvement, including increased activity levels on wearable sensors, which is a novel and practical way to track functional outcomes.
  • Safety Profile: Importantly, ATH434 was well-tolerated across both dose groups, with no serious adverse events related to the drug. This is critical, given the challenges of treating neurodegenerative diseases, where safety concerns are paramount.

David Stamler, M.D., Alterity’s CEO, commented, “We are thrilled that ATH434 has demonstrated significant slowing of clinical progression and an excellent safety profile in this rare, rapidly progressive disease. Currently, there are no approved treatments that slow the progression of MSA and these results show that ATH434’s targeted iron engagement may truly have a disease modifying effect. The fact that we achieved statistical significance on the UMSARS is extremely meaningful because it assesses the functional areas affected in MSA and is the endpoint needed to support drug approval by the U.S. Food and Drug Administration (FDA). Based on the strength of these Phase 2 data, we look forward to engaging with the FDA as quickly as possible to discuss the path forward for accelerating the development of ATH434 given the tremendous unmet need for treating MSA. We are very grateful for the invaluable contributions of the study participants and the clinical sites who contributed to the study.

“We now have evidence that targeting excess labile iron in neurodegenerative disease can be achieved. By redistributing this reactive form of iron that contributes to disease pathogenesis, not only can we target α synuclein aggregation, but we can also break the vicious cycle underlying disease progression. This has implications for developing disease modifying treatments for orphan diseases such as MSA and Friedreich’s ataxia as well as major neurodegenerative disorders such as Parkinson’s disease and Alzheimer’s disease.”

Overall, the combination of clinical efficacy, biomarker evidence, and a favorable safety profile is quite promising. The ability of ATH434 to reduce iron accumulation—something that is not commonly targeted by other MSA therapies—could represent a major step forward in addressing the underlying pathology of the disease.

Daniel Claassen, M.D., M.S., Professor of Neurology at Vanderbilt University Medical Center and Coordinating Investigator for the ATH434-201 Phase 2 study, added “The findings from the study are compelling because ATH434 appears to have meaningfully slowed MSA progression and stabilized motor function. To date, no treatment has altered the progression of this devastating disease. The slowing of clinical progression in this study, particularly at 50 mg, is impressive. I look forward to continue working with Alterity to bring this therapy to patients, and I know the MSA community welcomes this exciting advancement.”

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