Pegtarazimod limits acute graft-versus-host disease mortality and severity in mice and is tolerated in patients.
J Clin InvestAug 4, 2026 (epub)
Verena Holzmüller, Jana Gawron, Ann-Cathrin Burk et al.
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In 30 seconds
This phase II clinical trial evaluated the efficacy and tolerability of pegtarazimod in patients with corticosteroid-refractory acute graft-versus-host disease (aGVHD). In mouse models, pegtarazimod significantly reduced aGVHD-related mortality and severity. In the clinical trial, 4 out of 7 patients showed an overall response by day 28, indicating potential therapeutic benefits.
Key findings
- Pegtazarimod reduced aGVHD-related mortality and histological severity in mouse models.
- In the clinical trial, 4 out of 7 patients achieved an overall response by day 28.
- Pegtazarimod was well tolerated in patients with corticosteroid-refractory aGVHD.
Why it matters
Acute graft-versus-host disease remains a significant complication following allogeneic hematopoietic cell transplantation. Effective management strategies are crucial for improving patient outcomes, particularly in cases resistant to standard treatments.