FDA Grants Fast Track to Alterity's ATH434 for Multiple System Atrophy Treatment

Alterity Therapeutics, a biotechnology company focused on developing treatments for neurodegenerative diseases, has announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to its lead therapeutic candidate, ATH434. This designation is awarded to expedite the development and review of drugs aimed at treating serious conditions with unmet medical needs, such as Multiple System Atrophy (MSA). MSA is a progressive neurodegenerative disorder for which no approved therapy currently exists.

ATH434 is an oral agent designed to prevent the aggregation of proteins associated with neurodegeneration, including α-synuclein, a pathological protein that accumulates in the brains of MSA patients. By targeting pathological protein aggregation and maintaining normal iron balance in the brain, ATH434 aims to preserve neuronal function. Preclinical studies have indicated its efficacy in reducing α-synuclein pathology and protecting neurons.

The Fast Track designation complements ATH434’s existing Orphan Drug Designation from the FDA and the European Commission, underscoring its potential to address the urgent need for disease-modifying treatments in MSA. David Stamler, M.D., CEO of Alterity, emphasized that the Fast Track status allows for more frequent interactions with the FDA, potentially accelerating the development and approval process for ATH434. The designation also provides opportunities for a rolling review of the New Drug Application (NDA) and eligibility for Accelerated Approval and Priority Review, if certain criteria are met.

The ATH434-201 Phase 2 clinical trial, a randomized, double-blind, placebo-controlled study, evaluated the safety, efficacy, and pharmacokinetics of ATH434 over 12 months in individuals with MSA. The trial engaged 77 adults who received either 50 mg or 75 mg of ATH434 twice daily or a placebo. Results demonstrated clinically significant improvements on the modified Unified Multiple System Atrophy Rating Scale (UMSARS) Part I, which assesses daily living activities affected by MSA. Improvements were consistent across several efficacy measures, including motor performance and orthostatic symptoms.

The trial utilized wearable sensors to assess motor activity outside clinical settings, providing additional data on ATH434's effects in real-world scenarios. Biomarkers were employed to evaluate drug impact and target engagement, with observations indicating that ATH434 reduced or stabilized iron accumulation in affected brain regions. The treatment was well tolerated, with adverse event rates similar to the placebo group and no serious adverse events attributed to ATH434.

MSA is characterized by the dysfunction and death of various nerve cells within the brain and spinal cord, leading to symptoms like autonomic instability, impaired movement, and loss of coordination. This rare disorder affects at least 15,000 people in the United States and currently lacks treatments that can alter disease progression. While some symptoms can be managed with medications, there is no cure for MSA.

Alterity Therapeutics, headquartered in Melbourne, Australia, and San Francisco, USA, is committed to advancing therapies for neurodegenerative diseases. With ATH434, Alterity seeks to provide a disease-modifying treatment option for MSA patients. Beyond MSA, ATH434's potential application is also being explored in Parkinson’s disease and related disorders, supported by its promising Phase 2 trial results and ongoing research in more advanced stages of MSA.

The Fast Track designation for ATH434 marks a significant step forward in Alterity’s mission to develop innovative therapies for debilitating neurodegenerative conditions, bringing hope to patients and families affected by MSA.