Syntara (SNT) Study Update summary

Study design and patient population

• Phase 2A open-label study enrolled 16 patients with severe myelofibrosis, most previously treated with ruxolitinib for an average of three years.

• Patients had high disease burden and limited treatment options.

• Seven patients completed 52 weeks; withdrawal rates were consistent with expectations for this disease severity.

• Six of nine discontinuations had efficacy data; none withdrew due to drug-related adverse effects.

• Next phase likely to be a phase 2B, with protocol discussions ongoing with FDA and EMA.

Efficacy outcomes

• 73% of patients on drug for six months or more achieved TSS50, nearly double rates seen with competitor drugs.

• Mean symptom score improvement was 56% at nine months and 68% at 52 weeks.

• 44% of patients achieved at least 25% spleen volume reduction at week 24 and beyond; one retained SVR25 at week 52.

• Two patients completing 52 weeks had 100% symptom resolution; six of seven completers chose to continue treatment.

• Early withdrawals with efficacy data showed symptom and spleen volume improvements, including one with 61% SVR at 38 weeks.

Safety and tolerability

• Amsulastat plus ruxolitinib was safe and well tolerated, with no treatment-related serious adverse events.

• Drug demonstrated a favorable tolerability profile, allowing long-term use without cytopenia.

• Haemoglobin and platelet counts remained generally stable throughout the study.