NTQ5082: A Comprehensive Clinical and Pharmacological Profile of a Novel Complement Factor B Inhibitor

Executive Summary

This report provides a comprehensive analysis of NTQ5082, an investigational, orally administered small molecule medication developed by Nanjing Chia Tai Tianqing Pharmaceutical. NTQ5082 is a potent and specific inhibitor of Complement Factor B (CFB), a critical enzyme in the alternative pathway of the complement system. By targeting CFB, NTQ5082 is designed to modulate the dysregulated immune activity that underpins a range of severe hematological and renal diseases.

The clinical development program for NTQ5082 is robust and strategically focused on two primary therapeutic areas. The lead indication is Paroxysmal Nocturnal Hemoglobinuria (PNH), a rare and life-threatening blood disorder. The PNH program has advanced to a pivotal Phase III clinical trial (NCT07177859), which is designed as a head-to-head, active-controlled study against Eculizumab, the established intravenous standard of care. This ambitious trial design, coupled with a long-term safety and efficacy extension study (NCT07177872), signals a clear intent to establish NTQ5082 as a competitive, patient-friendly alternative in the PNH treatment landscape.

Concurrently, NTQ5082 is being developed for complement-mediated renal disorders, with Primary IgA Nephropathy (IgAN) as the secondary lead indication. A multicenter, randomized, placebo-controlled Phase II trial (NCT06982040) is underway to evaluate multiple dose levels in patients with persistent proteinuria, a key marker of disease progression. The broader development pipeline also includes early-phase investigations into other rare conditions such as Atypical Hemolytic Uremic Syndrome (aHUS), C3 glomerulopathy, and Cold Agglutinin Disease, underscoring a "pipeline-in-a-pill" strategy to maximize the therapeutic potential of the asset.