Hengrui Pharmaceutical (01276): Licancopam fumarate (HRS-5965) capsules approved for drug registration

Zhitong Finance App News, Hengrui Pharmaceutical (01276) issued an announcement. Recently, Chengdu Shengdi Pharmaceutical Co., Ltd., a subsidiary of Jiangsu Hengrui Pharmaceutical Co., Ltd. (hereinafter referred to as the “Company”), received a notice from the State Drug Administration (hereinafter referred to as the “State Drug Administration”) to approve the launch of the new class 1 drug, rikangkopam fumarate (HRS-5965) capsules independently developed by the company.

PNH is an acquired hemolytic disease. Its incidence rate/prevalence rate is low, and it has been included in the national “First Batch of Rare Diseases List”. It is characterized by the absence of complement regulatory proteins CD55 and CD59. Among them, lack of CD59 protein causes uncontrolled formation of membrane attack complexes (MAC). PNH red blood cells cause complement-mediated intravascular hemolysis due to lack of nuclei and long-term exposure to plasma complement; at the same time, large amounts of C3b are deposited on the surface of PNH red blood cells and can be recognized and removed by hepatosplenic macrophages, leading to extravascular hemolysis. The amplification ring of the complement bypass pathway can produce 80% of downstream complement activation products, which is a key factor driving the development of PNH hemolysis. Dual hemolysis can induce patients to develop a series of clinical symptoms such as anemia, fatigue, thrombosis, and organ damage.

Although C5 complement inhibitors have promoted progress in PNH treatment by blocking the terminal complement pathway, even when used for initial treatment, 25%-50% of patients still have extravascular hemolysis, about 74% of patients suffer from fatigue, and only 21.3% of patients achieve complete hematological remission (defined as patients without blood transfusion and no anemia).

Licancopam fumarate capsules are a complement factor B inhibitor that targets proximal complement inhibition of the complement bypass pathway. By inhibiting abnormal activation of the bypass pathway and complement amplification cycle, it simultaneously inhibits intravascular hemolysis and extravascular hemolysis at the mechanistic level, and increases hemoglobin levels. In response to this indication, Novartis's Ipcopam Hydrochloride Capsules (Fabhalta®) were approved for marketing in 2024, and Lanlai Technology's ranocopam hydrochloride tablets (Etenine®) and Hisco's sipcopam succinate tablets (Ecopan®) were approved for marketing in 2026. According to the EvaluatePharma database, Fabhalta®'s global sales volume in 2025 was approximately US$505 million. At the same time, the marketing license application for this product to treat paroxysmal nocturnal hemoglobinuria (PNH), which is still anemic after previous treatment with C5 complement inhibitors, is also under review. Up to now, the total R&D investment for projects related to licankopam fumarate capsules is about 277.5 million yuan (unaudited).