Syntara Limited (ASX: SNT), a clinical-stage drug development company targeting extracellular matrix dysfunction to develop novel medicines for blood cancers and conditions linked to inflammation and fibrosis, today announced positive feedback from the U.S. Food and Drug Administration (FDA). The constructive in-person Type C meeting focused on the planned Phase 2b clinical trial of its lead candidate, amsulostat, for treating myelofibrosis (MF) patients who have shown an inadequate response to standard of care.
Following a review of amsulostat’s development, the FDA supported Syntara’s proposed Phase 2b study design, providing guidance on the study’s details and the overall development pathway for amsulostat. This feedback marks a significant milestone, enabling the progression into late-stage clinical development and enhancing opportunities for engagement with potential commercial partners. The planned Phase 2b study will be a double-blind, placebo-controlled trial, investigating amsulostat in combination with standard of care (JAK inhibition) for approximately 100 patients. The primary endpoint will measure a 50% reduction in total symptom score (TSS50) after nine months of treatment.
Syntara Chief Executive Officer Gary Phillips expressed delight at the positive FDA review, highlighting amsulostat’s differentiated and competitive safety and efficacy profile, which positions it as a potential breakthrough therapy for MF patients with an inadequate response to current treatments. Mr. Phillips also noted the advancement of amsulostat’s development into myelodysplastic syndrome (MDS), with two ongoing clinical studies. The company anticipates further clinical milestones in 2026, including top-line data from its Phase 2 study of SNT-4728 for isolated REM sleep behaviour disorder, a prodromal feature of Parkinson’s disease, and results from a placebo-controlled study of SNT-9465 for hypertrophic scarring.
