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December 8, 2025BloodOpen Access

Efficacy and safety of HSK39297 monotherapy in patients with paroxysmal nocturnal hemoglobinuria (PNH)

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Authors

YGYuemin GongLZLina ZhangJHJianda Hu

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Overview

Phase 2 trial demonstrates HSK39297 reduces hemolysis while improving hemoglobin in complement inhibitor-naïve PNH patients, suggesting robust safety.

Key Points

  • At week 24, 89.4% of patients achieved a significant hemoglobin increase without transfusions, indicating strong efficacy.
  • Patients experienced a mean hemoglobin increase of 50.7 g/L, reflecting impressive improvements in hematological outcomes.
  • The trial involved a phase 2, open-label design with 47 complement inhibitor-naïve PNH patients receiving varying dosages of HSK39297.
  • No thromboembolic events occurred, highlighting the favorable safety profile of HSK39297 monotherapy compared to existing therapies.

Cite This Study

Gong et al. (2025) studied this question.

synapsesocial.com/papers/69362f694fa91c937236df75https://doi.org/10.1182/blood-2025-4969
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Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1LP-005, a bifunctional C5 antibody fusion protein, efficacy and safety in PNH patients with active hemolysis: Updated results from a Phase 2 study at 12/24 weeks2025
  2. 2Efficacy and safety of NTQ5082 monotherapy in complement-inhibitor-Naïve patients with paroxysmal nocturnal hemoglobinuria: A proof-of-concept, multicenter, randomized, open-label phase 2 study2025
  3. 3[New treatment strategies for paroxysmal nocturnal hemoglobinuria: drug selection in the era of novel complement inhibitors].2025
  4. 4KP104, a bifunctional C5 mAb–Factor h fusion protein, demonstrates sustained long-term efficacy and safety in complement inhibitor–naïve PNH patients: 2-year results from A phase 2 study with 8-week post-treatment safety follow-up.2025 · 1 citations
  5. 5VSA012, a CFB-targeted siRNA, demonstrates a favorable safety profile and sustained efficacy in PNH patients: Interim Analysis from a Phase Ib Study2025