Clinical TrialJun 11, 2026, 04:06 PM
Kyntra Bio Roxadustat Phase 3 Data Shows Improved Transfusion Independence
AI Summary
Kyntra Bio announced additional post hoc analysis data from the Phase 3 MATTERHORN trial for roxadustat, demonstrating durable and clinically meaningful improvements in transfusion independence (TI) for patients with lower-risk myelodysplastic syndromes (LR-MDS) and high transfusion burden (HTB). The data showed similar TI rates in both ring sideroblast positive (RS+) and ring sideroblast negative (RS-) disease, an important finding given limited options for RS- patients. The company is finalizing the pivotal Phase 3 trial protocol based on FDA feedback and expects to initiate it in the second half of 2026, with these findings to be presented at the EHA Congress 2026.
Key Highlights
- Roxadustat showed durable and clinically meaningful improvement in transfusion independence (TI) in LR-MDS patients with high transfusion burden (HTB).
- Similar rates of TI were observed in both ring sideroblast positive (RS+) and ring sideroblast negative (RS-) disease.
- In RS- patients (84 of 140), roxadustat achieved ">="8 weeks TI in 48% vs. 28% for placebo.
- In the HTB subgroup (n=37), roxadustat showed higher rates of ">="8-, 12-, 16-week RBC TI vs placebo.
- The pivotal Phase 3 trial protocol for roxadustat in LR-MDS is being finalized based on FDA feedback.
- The pivotal Phase 3 trial is expected to initiate in the second half of 2026.
- No new safety signals were observed; treatment-emergent adverse events (TEAEs) were generally lower grade.
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