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December 8, 2025BloodOpen Access

Efficacy and safety of NTQ5082 monotherapy in complement-inhibitor-Naïve patients with paroxysmal nocturnal hemoglobinuria: A proof-of-concept, multicenter, randomized, open-label phase 2 study

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Authors

HTHongyan TongLXLinghui XiaLLLin Liu

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Overview

Multicenter trial shows NTQ5082 improves hemolysis and Hb levels in paroxysmal nocturnal hemoglobinuria, indicating a favorable safety profile.

Key Points

  • Treatment with NTQ5082 resulted in significant hemolysis control and transfusion-free improvement in Hb levels among patients.
  • At Week 12, all 25 patients met the primary endpoint, showing Hb concentration increases by at least 20 g/L compared to baseline.
  • This open-label, phase 2, multicenter study assessed NTQ5082 monotherapy in complement inhibitor-naïve patients with PNH.
  • Results suggest that NTQ5082 has a well-tolerated safety profile, supporting its competitive potential as a CFB inhibitor.

Cite This Study

Tong et al. (2025) studied this question.

synapsesocial.com/papers/69362f764fa91c937236e378https://doi.org/10.1182/blood-2025-982
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Also Consider

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

  1. 1Efficacy and safety of HSK39297 monotherapy in patients with paroxysmal nocturnal hemoglobinuria (PNH)2025
  2. 2LP-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
  3. 3VSA012, a CFB-targeted siRNA, demonstrates a favorable safety profile and sustained efficacy in PNH patients: Interim Analysis from a Phase Ib Study2025
  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. 5The role of red blood cell lifespan in evaluating the condition of patients with paroxysmal nocturnal hemoglobinuria treated with complement inhibitors2025