The Phase 2 open-label study by Syntara Limited, focusing on the safety and efficacy of SNT-5505 over 52 weeks, has enrolled 16 patients with intermediate-2 or high-risk myelofibrosis (MF). Key findings from the interim data include:
- Safety and Tolerability: SNT-5505, used alongside a stable dose of RUX, was well-tolerated with no serious adverse events attributed to it.
- Symptom Relief: Significant symptom improvement was observed, with 46% of patients achieving a ≥50% reduction in symptom scores by Week 12, increasing to 80% by Week 38.
- Spleen Volume Reduction: 82% of patients experienced stable or reduced spleen volume, with notable improvements from Week 24 to Week 38.
- Haematological Stability: Haemoglobin levels and platelet counts remained stable, with one transfusion-dependent patient showing a 70% reduction in transfusion needs.
Professor Claire Harrison highlighted the potential long-term benefits of SNT-5505, noting its unique mechanism and the sustained improvements in symptoms and spleen volume over time. Syntara CEO Gary Phillips expressed optimism about the drug's differentiation in the market and its potential for durable benefits, with plans to seek FDA feedback in Q2 2025.
The study, conducted across 19 sites in Australia, South Korea, Taiwan, and the USA, is expected to complete patient visits by July 2025. Despite some discontinuations, the trial's progress is consistent with expectations for a high disease burden patient group. Syntara continues to advance its research in MF and other fibrotic and inflammatory diseases, with SNT-5505 also achieving FDA Orphan Drug Designation.
