Alterity Therapeutics has been granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for its lead candidate, ATH434.
ATH434 is an orally administered compound that targets the abnormal build-up of proteins linked to neurodegeneration. In preclinical studies, the drug has shown the ability to reduce α-synuclein pathology and maintain neuronal function by restoring normal iron regulation in the brain. Clinical trials have also demonstrated its ability to reach effective levels in the brain while maintaining a favourable safety profile.
MSA is a progressive disorder affecting the autonomic nervous system and movement control. Common symptoms include difficulty with coordination, posture, and bladder function, as well as blood pressure regulation issues. The disease is marked by the accumulation of α-synuclein in glial cells and neuronal loss in various parts of the brain. An estimated 15,000 people in the United States are affected by the condition.
The designation applies to the treatment of Multiple System Atrophy (MSA), a rare and debilitating neurodegenerative disease for which no approved therapy currently exists.
The Fast Track designation is designed to speed up the development and regulatory review process for drugs that address serious medical conditions with high unmet needs.
Biomarker analysis demonstrated that ATH434 was associated with stabilisation or reduction of iron accumulation in affected areas of the brain and potential preservation of brain volume. The treatment was generally well tolerated, with adverse events comparable to those seen in the placebo group and no treatment-related serious adverse events reported.
In addition to the Fast Track designation, ATH434 has already received Orphan Drug Designation from both the U.S. FDA and the European Commission for the treatment of MSA. A second open-label Phase 2 trial focusing on patients with more advanced MSA is currently ongoing.