Nuvectis Pharma’s Partner Wins China Approval for Ciprocopan in PNH, Validating the World’s First Once-Daily Oral Factor B Inhibitor

Nuvectis Pharma, Inc. (NASDAQ: NVCT), a clinical-stage biopharmaceutical company developing therapies for complement-related conditions and oncology, announced on July 23, 2026 that its partner Haisco Pharmaceutical Group received marketing approval from China’s National Medical Products Administration (NMPA) for ciprocopan (NXP100) in adult PNH patients who have not previously received complement inhibitor therapy. The approval, based on a Phase 3 trial in which ciprocopan demonstrated superiority over eculizumab, makes ciprocopan the world’s first approved once-daily oral complement Factor B inhibitor and directly validates the asset Nuvectis licensed for exclusive development and commercialization outside Greater China, Southeast Asia, and India just one month ago.

PNH Is a Severe Rare Disease With a Rapidly Shifting Treatment Landscape

Paroxysmal nocturnal hemoglobinuria is a rare, life-threatening disorder caused by clonal mutations in hematopoietic stem cells. Affected red blood cells lack key complement-regulating proteins, leaving them vulnerable to destruction by the body’s own immune system. That destruction drives chronic anemia, dangerous blood clots, and bone marrow failure. The standard of care—eculizumab (Soliris) and ravulizumab (Ultomiris), both C5 inhibitors marketed by AstraZeneca’s Alexion unit—requires intravenous infusion every one to two weeks, a significant burden for a condition requiring lifelong treatment. Novartis’ Fabhalta (iptacopan), a twice-daily oral Factor B inhibitor, won FDA approval in 2023 and posted $169 million in Q1 2026 sales, more than doubling year over year as patients and physicians demonstrated clear preference for oral therapy over infusion. Ciprocopan targets the same Factor B mechanism but requires only once-daily dosing, a potential differentiator that the NMPA approval now confirms works in a pivotal trial setting.

Superiority Over Eculizumab Anchors the Clinical Case

China’s NMPA granted approval based on Haisco’s Phase 3 trial in complement-inhibitor-naïve PNH patients, in which ciprocopan met all primary and secondary endpoints and demonstrated superiority over eculizumab. Key outcomes included improved hemoglobin levels and reduced transfusion requirements—the two metrics that most directly reflect disease burden for PNH patients. In the trial, 94.6% of ciprocopan patients avoided transfusions versus 69.4% in the eculizumab arm, and 59.5% of ciprocopan patients reached the 12 g/dL hemoglobin target versus 8.3% on eculizumab. A second NMPA application, covering patients who have already failed a C5 inhibitor such as eculizumab or ravulizumab, remains under review. Beyond PNH, a Phase 2 trial in lupus nephritis is also ongoing in China. Outside China, Nuvectis holds ex-territory rights and intends to leverage the NMPA dataset and Haisco’s development work to support its own regulatory program, which could include an FDA submission strategy built on the existing clinical package.

A $10 Billion Market and a Best-in-Class Convenience Profile

The global PNH treatment market stood at approximately $5.75 billion in 2024 and analysts project it will nearly double to approximately $9.8 billion by 2030, driven largely by the shift from injectable C5 inhibitors toward oral therapies. Soliris sales fell 14% year over year in Q1 2026 as Fabhalta eroded its share—a direct signal that oral dosing drives switching behavior in this market. Ciprocopan’s once-daily profile gives it a potential convenience edge over Fabhalta’s twice-daily regimen, though head-to-head data do not yet exist. For Nuvectis, which held just $25.1 million in cash as of March 31, 2026, the Haisco license agreement—which also includes NXP200, a paradox-breaker BRAF inhibitor—transforms the company from a single-drug oncology story into a late-stage rare disease developer with a partner-validated, approved asset underpinning its ex-China development program.

Strategic Investment Summary

• China Approval: Partner Haisco received NMPA marketing approval for ciprocopan (NXP100) in complement-inhibitor-naïve PNH patients on July 23, 2026, based on a Phase 3 trial demonstrating superiority over eculizumab on all primary and secondary endpoints.
• World’s First Once-Daily Oral Factor B Inhibitor: Ciprocopan’s NMPA approval makes it the first approved once-daily oral complement Factor B inhibitor globally, with a potential convenience edge over Novartis’ twice-daily Fabhalta, the only other approved oral agent in the class.
• Nuvectis License Rights: Under the June 2026 agreement with Haisco, Nuvectis (NASDAQ: NVCT) holds exclusive rights to develop and commercialize ciprocopan outside Greater China, Southeast Asia, and India, with the NMPA approval validating the asset one month after the deal closed.
• Second Application Pending: A separate NMPA application covering PNH patients who previously failed a C5 inhibitor remains under review, with a decision expected in late 2026 or early 2027, expanding ciprocopan’s potential China label further.
• Market Opportunity: The global PNH market is projected to grow from approximately $5.75 billion in 2024 to approximately $9.8 billion by 2030, with oral therapies driving the shift as evidenced by Fabhalta’s $169 million Q1 2026 sales and concurrent 14% decline in Soliris revenue.
• Pipeline Context: The Haisco agreement also includes NXP200, a paradox-breaker BRAF inhibitor for BRAF-mutated malignancies, giving Nuvectis a two-asset ex-China platform alongside its existing NXP900 oncology program currently in Phase 1b.

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