Alterity Therapeutics meeting with US FDA provides development pathway for ATH434
The company recently met with the FDA following the successful completion of its Phase 1 clinical trial last year and further data analysis. The pre-IND (Investigational New Drug) meeting was to obtain input on the clinical development plan for ATH434, including feedback on the Phase 2 study design.
Alterity reached agreement with the FDA on the non-clinical investigations required to support the Phase 2 study. In addition, the FDA agreed to key aspects of the Company's Phase 2 study design including the proposed patient population, safety monitoring plan, and strategy for evaluating drug exposure during the study.
As there are currently no approved treatments for MSA and, therefore, no regulatory precedent regarding accepted efficacy endpoints, the FDA and Alterity will work together to develop an endpoint that is best suited for the MSA patients to be studied. The FDA has also encouraged Alterity to utilise data from a natural history study that Alterity has planned with clinical and neuroimaging experts at
This natural history study, referred to as bioMUSE, or biomarkers of Progression in Multiple System Atrophy, will enroll early stage MSA patients and track change in clinical parameters and biomarkers for up to one year. Natural history studies are important for characterizing disease progression over time in selected patient populations. Well-conducted, these studies can provide vital information to optimize clinical trial design and inform the selection of biomarkers to evaluate target engagement of drug candidates.
In parallel with the US strategy, Alterity is also pursuing a regulatory pathway in
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