Syntara (ASX:SNT) has announced receipt of crucial feedback from the US Food and Drug Administration (FDA) regarding the clinical development of amsulostat for myelofibrosis (MF) treatment.
In a Type C meeting, the FDA reviewed interim data from Syntara's ongoing open-label trial (MF-101) of amsulostat in combination with ruxolitinib, as well as a proposal for a pivotal registrational study. This data was previously presented at the European Haematology Association congress in June 2025.
The FDA uses Type C meetings with companies to allow for general questions and discussions about drug development programs. Typical subjects include study design, safety considerations, regulatory requirements, or any other aspect of drug development that needs clarification.
Syntara said the FDA has recommended that it conduct a Phase 2 trial with a control arm to gather additional safety and efficacy data. This trial will focus on improvements in symptoms and spleen volume reductions, aiming to optimise the design and efficiency of a subsequent pivotal Phase 3 trial.
Syntara CEO Gary Phillips said, "We will use the FDA guidance to refine our clinical development plan for amsulostat and continue discussions with partners based on the FDA recommended path forward."
Phillips also said Syntara's strong position, citing the company's robust pipeline and a cash runway extending into 2027. He added, "We look forward to sharing results from our ongoing clinical trials over the coming months."