Wolfgang Miesbach: Paediatric Phase 3 BASIS KIDS Data for Marstacimab at ISTH 2026
Wolfgang Miesbach, Professor of Medicine at Frankfurt University Hospital, shared a post on LinkedIn:
“A once-weekly, subcutaneous anti-TFPI rebalancing agent has paediatric data – across both haemophilia A and B, with and without inhibitors.
ISTH 2026, Paris – at the Late-Breaking session on bleeding disorders, Gili Kenet presented interim Phase 3 BASIS KIDS data on once-weekly subcutaneous marstacimab in children aged 6–17 years with haemophilia A or B, with and without inhibitors.
Efficacy:
Substantial reductions in annualised bleeding rates vs historical regimens (mean treated ABR 1.8 vs 3.6 without inhibitors; 1.4 vs 18.9 with inhibitors), with 96.4% of baseline target joints resolved – consistent across haemophilia type, inhibitor status and age.
Safety:
Predominantly mild and moderate AEs, no treatment-related discontinuations, injection-site reactions the most frequent event of special interest, no thromboembolic events, PD biomarkers within physiological ranges, and transient anti-drug antibodies without impact on efficacy or safety.
These data build on the adult and adolescent BASIS results and underpin marstacimab’s approvals on both sides of the Atlantic:
- US: now 6 years of age and older, with or without inhibitors (expanded from 12 years of age and older, June 2026).
- EU: severe HA or HB 12 years of age and older (at least 35 kg), now including inhibitors post-CHMP ad EC.”
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