Nuvectis Pharma Announces China Approval Of Ciprocopan For Treatment-Naive PNH Patients

Nuvectis Pharma, Inc., a clinical-stage biopharmaceutical company developing therapies for complement-mediated diseases and oncology, has announced that ciprocopan (NXP100), its once-daily oral complement Factor B inhibitor, has received marketing approval from China's National Medicinal Products Administration (NMPA). The approval is for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) who have not previously received complement inhibitor therapy.

The approval marks the first regulatory authorization for ciprocopan and establishes it as the world's first approved once-daily oral complement Factor B inhibitor. According to the Company, the approval provides important clinical and regulatory validation for the therapeutic approach of Factor B inhibition and supports the potential use of ciprocopan not only in PNH but also in other complement-mediated diseases. The once-daily oral administration is expected to offer patients a more convenient treatment option compared with currently available therapies.

Ron Bentsur, Chairman and Chief Executive Officer of Nuvectis Pharma, congratulated the Company's partner, Haisco, on achieving the first marketing approval for ciprocopan. He stated that the approval represents an important milestone for patients with PNH who have not previously been treated with complement inhibitors. Bentsur added that Factor B inhibitors are expected to become an increasingly important class of therapies in the global PNH market and believes ciprocopan's once-daily oral dosing could position it to gain significant market share if it receives regulatory approvals in additional countries. He also noted that the therapy has the potential to be developed for several other complement-mediated disorders where convenient oral dosing may improve patient adherence and long-term treatment outcomes.

Dr. Pangke Yan, Chief Executive Officer of Haisco, described the approval as an important advancement for patients with PNH in China. He stated that Haisco looks forward to continuing its collaboration with Nuvectis to expand the availability of ciprocopan to patients in international markets.

The NMPA's approval was primarily supported by results from a head-to-head Phase 3 clinical trial comparing ciprocopan with Soliris (eculizumab) in patients with PNH who had not previously received complement inhibitor therapy. The study successfully achieved all primary and secondary endpoints and demonstrated superior clinical outcomes for ciprocopan compared with eculizumab.

Clinical trial results showed that 59.5% of patients treated with ciprocopan achieved a hemoglobin level of at least 12 g/dL, compared with 8.3% of patients receiving eculizumab. Patients receiving ciprocopan also experienced an average increase in hemoglobin levels of approximately 5.0 g/dL from baseline, compared with an increase of about 2.2 g/dL in the eculizumab group. In addition, 94.6% of patients treated with ciprocopan did not require blood transfusions during the study, compared with 69.4% of patients receiving eculizumab. The study also demonstrated a favorable safety profile, with no adverse events leading to treatment discontinuation or withdrawal from the trial among patients receiving ciprocopan.

Unlike terminal complement inhibitors such as Soliris and Ultomiris, which target complement component C5, ciprocopan inhibits complement Factor B, blocking amplification of the alternative complement pathway while preserving the classical and lectin pathways. According to the Company, this mechanism is designed to provide broader control of both intravascular and extravascular hemolysis, potentially resulting in improved clinical efficacy compared with C5 inhibitors, as demonstrated in comparative clinical studies.

Ciprocopan was independently developed by Haisco, which is responsible for its clinical development, regulatory approvals, and commercialization within China. Under an exclusive licensing agreement signed in June 2026, Nuvectis Pharma holds the exclusive rights to develop and commercialize ciprocopan outside Greater China, India, and selected Southeast Asian countries, supporting the Company's plans to pursue additional regulatory approvals in global markets.