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July, 2026
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Wolfgang Miesbach: Paediatric Phase 3 BASIS KIDS Data for Marstacimab at ISTH 2026
Jul 15, 2026, 06:35

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.”

Wolfgang Miesbach

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