Maralixibat Chloride

SUZHOU, China I May 16, 2025 I CANbridge Pharmaceuticals Inc. (1228.HK), a global biopharmaceutical company, with a foundation in China, committed to the research, development and commercialization of transformative therapies to treat rare disease, announced today that velaglucerase-beta for injection (Gaurunning), with its wholly – owned subsidiary, CANbridge (Shanghai) Life Sciences Ltd. as the holder, has obtained marketing approval from the National Medical Products Administration (NMPA). Gaurunning is the first domestically independently developed long-term enzyme replacement therapy for adolescents aged 12 and above and adults with type I and type III Gaucher disease. As a Class 1 innovative drug, it can fully replace similar imported products and will significantly enhance medication accessibility for domestic patients. In August of 2024, CANbridge announced positive top-line results from the pivotal clinical trial of Gaurunning in adult and pediatric Gaucher disease patients. In September, the Center for Drug Evaluation (CDE) of NMPA granted it priority review status. In October of the same year, CANbridge successfully passed the Manufacturing Authorization Holder (MAH) production and quality management system audit and on-site inspection and obtained the Drug Manufacturing License Type B certificate. In November, the New Drug Application (NDA) for Gaurunning was accepted by NMPA. In March 2025, it successfully passed the Pre-approval inspection and pre-marketing GMP compliance inspection for the pilot biological product of Divided manufacturing. Velaglucerase-beta for injection is the first innovative biological product in China to pass the inspection of Divided manufacturing of biological products. James Xue, Ph.D., CANbridge Founder, Chairman, and CEO, stated: “Enzyme replacement therapy (ERT) is a critical component of CANbridge’s global pipeline strategy. The approval of Gaurunning in China is a milestone. The six-month timeframe from NDA acceptance to approval not only reflects the strong safety, effectiveness and reliability of our product, as well as the professionalism in pharmaceutical regulatory affairs but also demonstrates the recognition and support of China’s drug regulatory authorities for the urgent medical needs of Gaucher disease patients. The innovative technology adopted in Gaurunning is enzyme modification and directed evolution, serving as a typical case of new quality productive forces. In the future, combined with continuous flow production process technology, it can be extended to other similar drugs beyond Gaucher disease to achieve large-scale, batch production. Going forward, the platform-based technology of Gaurunning will significantly reduce development costs, enabling patients to access safe, effective, and affordable domestic enzyme replacement therapies. The successful development and approval of Gaurunning embody the original entrepreneurial aspiration of CANbridge. It owes much to the concerted efforts of numerous partners including WuXi Biologics, benefits from the support of national and local regulatory authorities, relevant departments, and medical institutions. Above all, it carries the hope that patients with Gaucher disease across China will soon gain access to sustainable treatment. Gaurunning is the first collaborative project between CANbridge and WuXi Biologics ( 2269.HK ) in the rare disease field. We thank our partners, including WuXi Biologics, for enabling the development and production of this product. This is not only the third rare disease treatment launched by CANbridge’s Hunterase and Livmarli but also the first self-developed product to enter commercialization. We will continue to deepen our focus on localized R&D, production, launch, and accessibility of rare disease drugs to bring innovative treatment solutions to Chinese patients.” Dr. Chris CHEN, Chief Executive Officer of WuXi Biologics, said: “We are extremely honored to have empowered CANbridge to achieve this key milestone of Gaurunning, which also represents a significant breakthrough in China’s rare disease drug innovation. The journey of rare disease drug development is long and arduous, requiring the overcoming of countless technical hurdles and challenges from early laboratory discovery to ultimately benefiting patients. The successful realization of this product from concept to commercial production not only testifies to CANbridge’s decade-long commitment to deepening its presence in the rare disease field but also serves as a successful application of WuXi Biologics’ integrated technology platform and CRDMO business model. The shared vision of both parties in addressing the unmet clinical needs of rare disease patients has led to the achievement of China’s first domestically developed enzyme replacement therapy for Gaucher disease. We look forward to empowering more global partners to accelerate the development of high-quality rare disease treatments, driving rare disease drug innovation, and benefiting more rare disease patients.” About Velaglucerase-beta for Injection (Gaurunning) Velaglucerase-beta for injection is the first domestically developed recombinant human glucocerebrosidase ERT in China, now approved for clinical use in treatment of Type I and Type III Gaucher disease patients. Most Gaucher disease patients are Type I and Type III, which are chronic non-neuronopathic and chronic neuronopathic forms, respectively. velaglucerase-beta is administered as an intravenous infusion and is intended to supplement the lack of glucocerebrosidase in the lysosomes of GD patients. The pivotal clinical trial of velaglucerase-beta achieved positive top-line data in August 2024. The velaglucerase-beta pivotal trial is a randomized, double-blind, dose-comparison study designed to evaluate the efficacy, safety, and pharmacokinetics of intravenous velaglucerase-beta administered every other week in newly treated Gaucher Disease (GD) patients, with an extension period. The results demonstrate that the study successfully met its primary efficacy endpoint, showing a statistically significant mean percentage reduction from baseline in spleen volume at nine months for both the 60 U/kg dose (P<0.0001) and the lower 30 U/kg dose (P<0.001).  The primary endpoint of this trial protocol has been agreed upon by the CDE. About Gaucher disease (GD) Gaucher disease, one of the most common lysosomal storage disorders, is a rare inherited genetic metabolic disease caused by autosomal recessive mutations in the GBA gene located on chromosome 1q22 and affects both males and females equally. Gaucher disease is a clinical spectrum that comprises Type 0 (perinatal-lethal), Type I (chronic non-neuronopathic), Type II (acute neuronopathic), and Type III (chronic neuronopathic) forms, with Types I and III surviving into adulthood. Gaucher disease is caused by a deficiency of glucocerebrosidase (acid b-glucosidase), an enzyme that helps break down a cellular membrane glycosphingolipid called glucocerebroside (glucosylceramide) inside lysosomes. As a result, glucocerebroside accumulates primarily in cells of the monocyte-macrophage lineage (Gaucher cells) within certain organs, leading to splenomegaly, hepatomegaly, anemia, thrombocytopenia, bone pain and fractures, and in the most severe forms (perinatal-lethal, Types II and III), early neurological symptoms.  For 30 years, recombinant human glucocerebrosidase enzyme replacement therapy (ERT) has been the standard of care for Gaucher disease, with clinical trials and real-world data demonstrating significant improvement in the major non-neurological signs and symptoms of disease and quality of life. There were 3,000 patients with Gaucher disease in China in 2020, according to Frost & Sullivan. About CANbridge Pharmaceuticals Inc. CANbridge Pharmaceuticals Inc. (HKEX:1228) is a global biopharmaceutical company, with a foundation in China, committed to the research, development and commercialization of transformative therapies for rare disease. CANbridge has a differentiated drug portfolio, with 3 approved drugs and a pipeline of 8 assets, targeting prevalent rare disease indications that have unmet needs and significant market potential. These include Hunter syndrome and other lysosomal storage disorders, complement-mediated disorders, hemophilia A, metabolic disorders, rare cholestatic liver diseases and neuromuscular diseases. The CANbridge Next-Generation Innovation and Process Development Facility is developing novel, potentially curative, gene therapies for rare genetic diseases, including Pompe disease, Fabry disease, spinal muscular atrophy (SMA), Duchenne muscular dystrophy (DMD) and other neuromuscular conditions, and collaborates with world-leading researchers and biotech companies. CANbridge global partners include: Apogenix, GC Pharma, Mirum, Wuxi Biologics, Privus, UMass Chan Medical School, the University of Washington School of Medicine and Scriptr Global. For more on CANbridge Pharmaceuticals Inc., please go to: www.canbridgepharma.com . SOURCE: CANbridge Pharmaceuticals

Enabled by WuXi Biologics' industry-leading integrated technology platform, velaglucerase-beta for injection has successfully advanced from concept to commercialization. Velaglucerase-beta for injection, as the first and the only locally developed enzyme replacement therapy (ERT) for Gaucher disease in China, marks a significant breakthrough for CANbridge Pharmaceuticals and WuXi Biologics in advancing R&D of innovative rare disease therapeutics in China. Velaglucerase-beta for injection is the first innovative biological product in China to pass segmented manufacturing inspection. WUXI, China and SHANGHAI, May 16, 2025 /PRNewswire/ -- WuXi Biologics (2269.HK), a leading global Contract Research, Development and Manufacturing Organization (CRDMO), congratulates its partner CANbridge Pharmaceuticals on the approval of its innovative velaglucerase-beta for injection (Gaurunning) by the China National Medical Products Administration (NMPA) for the treatment of adolescents aged 12 and above and adults with type I and type III Gaucher disease. This marks China's first and only locally developed enzyme replacement therapy (ERT) for Gaucher disease. Gaucher disease is a rare inherited genetic lysosomal storage disorder characterized by clinical manifestations such as hepatosplenomegaly, anemia, bone pain, and neurological symptoms, which can be life-threatening in severe cases. Recombinant human glucocerebrosidase ERT has been the standard treatment for Gaucher disease for 30 years, significantly improving major non-neurological signs and symptoms, as well as improving the quality of life for patients. It is estimated that the global prevalence of Gaucher disease is 0.7 to 1.75 per 100,000 people. Gaucher disease has been included in China's "First List of Rare Diseases". Velaglucerase-beta for injection is the first collaboration project on rare diseases between CANbridge and WuXi Biologics, advancing from concept to commercialization by leveraging WuXi Biologics' leading integrated technology platform. The project utilized innovative technology to construct high-expression cell line and achieved significant improvements in both yield and quality through WuXiUPTM, an ultra-high productivity continuous bioprocessing platform of WuXi Biologics. The overall yield increased by more than 110 times, and the specific enzyme activity increased by more than 50%, ensuring the affordability for Gaucher disease patients. Additionally, WuXi Biologics developed cell-based bioassay methods for commercial product release and stability testing, addressing the technical challenges of enzyme testing and effectively ensuring product quality control. Additionally, velaglucerase-beta for injection is the first innovative biological product in China to pass segmented manufacturing inspection. Dr. Chris Chen, CEO of WuXi Biologics, stated, "We are honored to enable CANbridge to achieve the significant milestone of approval for velaglucerase-beta for injection, which represents a critical advancement in rare disease drug innovation in China. Developing therapies for rare diseases is a long and challenging journey, requiring relentless joint efforts to bridge the gap between early-stage discovery and patients' access to medication. The successful commercialization of velaglucerase-beta for injection not only reflects CANbridge's decade-long dedication to rare diseases but also exemplifies the value of WuXi Biologics' integrated platform and CRDMO business model. Furthermore, our shared commitment to addressing the unmet clinical needs of rare disease patients has resulted in China's first locally developed ERT for Gaucher disease. We look forward to enabling more global partners to accelerate the development of high-quality rare disease therapies, and making them accessible and affordable for more patients suffering from rare diseases." Dr. James Xue, Founder, Chairman, and CEO of CANbridge Pharmaceuticals, stated, "Enzyme replacement therapy is a crucial part of CANbridge' global pipeline. The approval of velaglucerase-beta for injection in China is a milestone. By leveraging the integrated technology platform, velaglucerase-beta for injection will significantly reduce development costs, making safe and effective domestically produced ERT accessible and affordable for patients. This product is also the first collaboration project between CANbridge and WuXi Biologics in rare diseases. We extend our gratitude to our partners, including WuXi Biologics, who have empowered the development and manufacturing of velaglucerase-beta for injection. This milestone is not only CANbridge' third rare disease drug to be launched following Hunterase® and Livmarli®, but also the first in-house developed commercialized product. In the future, we will continue to focus on the localized research, manufacturing, commercialization, and accessibility of rare disease drugs, bringing innovative treatments to patients in China." Since its establishment, WuXi Biologics has been committed to enabling global innovative biotech companies to accelerate the rare disease drugs from concept to commercialization, benefiting rare disease patients worldwide. As ofthe end of 2024, there are 21 rare disease projects on the company's platform. Additionally, the company actively promotes public awareness and supports the rare disease patient community through various philanthropic initiatives. Its global "Run for Health" campaign has engaged over 2,500 participants to raise awareness for rare diseases. In China, the company's "Rare Disease Student Education Support Program" has provided scholarships to over 100 students affected by rare diseases over the past four years, fostering their academic growth. About Velaglucerase-beta for Injection (Gaurunning) Velaglucerase-beta for injection is the first domestically developed recombinant human glucocerebrosidase ERT in China, now approved for clinical use in treatment of Type I and Type III Gaucher disease patients. Most Gaucher disease patients are Type I and Type III, which are chronic non-neuronopathic and chronic neuronopathic forms, respectively. velaglucerase-beta is administered as an intravenous infusion and is intended to supplement the lack of glucocerebrosidase in the lysosomes of GD patients. The pivotal clinical trial of velaglucerase-beta achieved positive top-line data in August 2024. The velaglucerase-beta pivotal trial is a randomized, double-blind, dose-comparison study designed to evaluate the efficacy, safety, and pharmacokinetics of intravenous velaglucerase-beta administered every other week in newly treated Gaucher Disease (GD) patients, with an extension period. The results demonstrate that the study successfully met its primary efficacy endpoint, showing a statistically significant mean percentage reduction from baseline in spleen volume at nine months for both the 60 U/kg dose (P<0.0001) and the lower 30 U/kg dose (P<0.001). The primary endpoint of this trial protocol has been agreed upon by the CDE. About Gaucher Disease (GD) Gaucher disease, one of the most common lysosomal storage disorders, is a rare inherited genetic metabolic disease caused by autosomal recessive mutations in the GBA gene located on chromosome 1q22 and affects both males and females equally. Gaucher disease is a clinical spectrum that comprises Type 0 (perinatal-lethal), Type I (chronic non-neuronopathic), Type II (acute neuronopathic), and Type III (chronic neuronopathic) forms, with Types I and III surviving into adulthood. Gaucher disease is caused by a deficiency of glucocerebrosidase (acid b-glucosidase), an enzyme that helps break down a cellular membrane glycosphingolipid called glucocerebroside (glucosylceramide) inside lysosomes. As a result, glucocerebroside accumulates primarily in cells of the monocyte-macrophage lineage (Gaucher cells) within certain organs, leading to splenomegaly, hepatomegaly, anemia, thrombocytopenia, bone pain and fractures, and in the most severe forms (perinatal-lethal, Types II and III), early neurological symptoms. For 30 years, recombinant human glucocerebrosidase enzyme replacement therapy (ERT) has been the standard of care for Gaucher disease, with clinical trials and real-world data demonstrating significant improvement in the major non-neurological signs and symptoms of disease and quality of life. There were 3,000 patients with Gaucher disease in China in 2020, according to Frost & Sullivan. Gaucher disease has been included in China's "First List of Rare Diseases." About WuXi Biologics WuXi Biologics (stock code: 2269.HK) is a leading global Contract Research, Development and Manufacturing Organization (CRDMO) offering end-to-end solutions that enable partners to discover, develop and manufacture biologics – from concept to commercialization – for the benefit of patients worldwide. With over 12,000 skilled employees in China, the United States, Ireland, Germany and Singapore, WuXi Biologics leverages its technologies and expertise to provide customers with efficient and cost-effective biologics discovery, development and manufacturing solutions. As of December 31, 2024, WuXi Biologics is supporting 817 integrated client projects, including 21 in commercial manufacturing (excluding COVID CMO projects). WuXi Biologics regards sustainability as the cornerstone of long-term business growth. The company continuously drives green technology innovations to offer advanced end-to-end Green CRDMO solutions for its global partners while consistently achieving excellence in Environment, Social and Governance (ESG). Committed to creating shared value, it collaborates with all stakeholders to foster positive social and environmental impacts and promote responsible practices that empower the entire value chain. For more information about WuXi Biologics, please visit: . About Canbridge CANbridge Pharmaceuticals Inc. (HKEX:1228) is a global biopharmaceutical company, with a foundation in China, committed to the research, development and commercialization of transformative therapies for rare disease. CANbridge has a differentiated drug portfolio, with 3 approved drugs and a pipeline of 8 assets, targeting prevalent rare disease indications that have unmet needs and significant market potential. These include Hunter syndrome and other lysosomal storage disorders, complement-mediated disorders, hemophilia A, metabolic disorders, rare cholestatic liver diseases and neuromuscular diseases. The CANbridge Next-Generation Innovation and Process Development Facility is developing novel, potentially curative, gene therapies for rare genetic diseases, including Pompe disease, Fabry disease, spinal muscular atrophy (SMA), Duchenne muscular dystrophy (DMD) and other neuromuscular conditions, and collaborates with world-leading researchers and biotech companies. CANbridge global partners include: Apogenix, GC Pharma, Mirum, Wuxi Biologics, Privus, UMass Chan Medical School, the University of Washington School of Medicine and Scriptr Global. For more on CANbridge Pharmaceuticals Inc., please go to: . Contacts Business [email protected] Media [email protected] SOURCE WuXi Biologics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED