Syntara Limited (ASX: SNT), a clinical-stage drug development company targeting extracellular matrix dysfunction, has received guidance from the US Food and Drug Administration (FDA) regarding the clinical development of amsulostat for myelofibrosis (MF). The FDA reviewed data from the ongoing open label trial (MF-101) of amsulostat in combination with ruxolitinib, as well as a proposal for a pivotal registrational study, during a Type C meeting.
FDA Recommends Phase 2 Controlled Trial
The FDA has recommended that Syntara undertake a Phase 2 trial with a control arm. This trial aims to acquire additional safety and efficacy data, focusing on improvements in symptoms and spleen volume reductions. The data will then be used to optimize the design and efficiency of a subsequent Phase 3 trial.
Amsulostat, also known as SNT-5505, is Syntara's lead candidate for the bone marrow cancer myelofibrosis, which causes a build-up of scar tissue. The drug is currently being evaluated in combination with ruxolitinib in the ongoing MF-101 trial.
Company Response and Strategic Direction
Syntara CEO Gary Phillips commented that the company will use the FDA guidance to refine its clinical development plan for amsulostat and continue discussions with potential partners. He added that Syntara is in a strong financial position, with a cash runway extending into 2027, and anticipates sharing results from ongoing clinical trials in the coming months.
Pipeline and Upcoming Milestones
The company's pipeline includes programs targeting myelodysplastic syndrome, hypertrophic scars, keloid scars, IRBD, and Parkinson's disease. Syntara's updated clinical pipeline development plan indicates several upcoming milestones, including top-line Phase 1c/2 data for amsulostat in myelofibrosis expected in Q3 2025, and interim Phase 1c/2 data for amsulostat in myelodysplastic syndrome expected in H1 2026.
