73% Symptom Improvement and 44% Spleen Reduction Reported for SNT-5505 at 24 Weeks

A New Hope for Myelofibrosis Patients

Myelofibrosis, a rare and debilitating bone marrow cancer, disrupts normal blood cell production and leads to severe symptoms such as fatigue, infections, and abdominal pain. Current standard treatments, primarily JAK inhibitors like ruxolitinib, offer limited survival benefits and are often accompanied by significant side effects and high discontinuation rates.

Syntara Limited’s investigational drug, SNT-5505, aims to address these shortcomings by targeting the root cause of the disease, bone marrow fibrosis driven by elevated lysyl oxidase activity. Unlike existing therapies that mainly manage symptoms, SNT-5505 seeks to clear fibrosis and restore healthy blood cell production, potentially modifying the disease course.

Interim Phase 2a Data Reveal Encouraging Signals

Presented at the European Hematology Association Congress 2025, interim results from the ongoing Phase 2a trial of SNT-5505 as an add-on to ruxolitinib show promising efficacy and safety in a patient population with high disease burden and suboptimal response to existing therapy.

Among patients evaluated at or beyond 24 weeks, 73% achieved a 50% or greater reduction in total symptom score (TSS50), indicating substantial relief from debilitating symptoms. Additionally, 44% of patients experienced at least a 25% reduction in spleen volume (SVR25), a key clinical marker linked to disease severity and patient quality of life.

Importantly, hematology parameters such as hemoglobin and platelet counts remained generally stable, and no treatment-related serious adverse events were reported. The safety profile suggests that SNT-5505 is well tolerated when combined with ruxolitinib, without requiring dose modifications of the standard therapy.

Positioning in a Competitive Landscape

SNT-5505’s novel mechanism, blocking lysyl oxidase to reduce fibrosis and growth factor signaling, sets it apart from other agents in development. While other drugs targeting myelofibrosis have shown mixed results and tolerability challenges, SNT-5505’s sustained symptom and spleen volume improvements, even in a heavily pretreated population, suggest it could fill a significant unmet need.

The company plans to seek FDA guidance on progressing to pivotal Phase 2/3 trials by Q3 2025, with final Phase 2a data anticipated in the second half of the year. This regulatory milestone will be critical in defining SNT-5505’s path toward potential approval and commercialisation in a market valued at approximately US$1 billion.

Broader Pipeline and Future Outlook

Beyond myelofibrosis, Syntara is advancing a diversified pipeline targeting other high-value indications such as myelodysplastic syndrome, hypertrophic and keloid scars, and neurodegenerative diseases. The interim success of SNT-5505 bolsters confidence in the company’s approach to addressing fibrotic and inflammatory conditions with novel mechanisms.

As the trial progresses and more data emerge, investors and clinicians alike will be watching closely to see if SNT-5505 can deliver on its promise of disease modification and improved patient outcomes in a challenging therapeutic area.