About CANBRIDGE-B Company

Established in 2012, Beihai Kangcheng is a global biopharmaceutical company headquartered in China, dedicated to the research, development and commercialization of innovative treatments for rare diseases. As of June 30, 2024, we have built a comprehensive product line of 12 active drug assets, with high unmet demand and huge market potential for the most common rare disease indications. This strong product line includes four marketed products and three drug candidates in advanced clinical stages. In view of the continuing challenging compensation environment in mainland China, volatile capital markets, and limited biotechnology funding, Beihai Kangcheng has further prioritized key projects, which will achieve major development and regulatory milestones in the coming year. We are led by a management team with extensive experience in the rare disease industry, whose experience covers R&D, clinical development, regulatory affairs, business development and commercialization. As of June 30, 2024, we have reduced the number of employees to 93 full time employees, 11 of whom have doctorates and/or medical doctorates, and over 70% of our employees have experience working for multinational biopharmaceutical companies. As of mid-August 2024, we have further reduced our workforce, with 79 full time employees to reduce operating costs. Our management team has a strong track record of successfully approving and commercializing rare disease treatments in major markets, including China, the US, Europe, Latin America, and Southeast Asia. With management expertise, we play an active role in promoting the development of China's rare disease industry and building a rare disease ecosystem. For example, our founder, Dr. Xue, currently serves as the vice chairman of the China Rare Disease Alliance (CHARD). Since our establishment in 2012, we have built a rich portfolio of therapies specifically targeting diseases with proven mechanisms of action, including biologics, small molecules, and gene therapy. We will continue to optimize our business product line through external licensing partnerships and collaboration with academic institutions and in-house R&D. In the field of rare diseases, we have seven kinds of biologics and small molecule products for various indications. Such indications include MPSII (i.e. Hunter syndrome) and other lysosomal storage diseases (LSDs), complement-mediated disorders, hemophilia A, metabolic disorders, and rare cholestatic liver diseases, including ALGS and PFIC. In September 2020, we obtained marketing approval for the treatment of mucopolysaccharide storage disease type II (CAN101) in mainland China. In May 2023, the China Drug Administration approved Millet for the treatment of ALGS; the Hong Kong Pharmacy and Poison Administration and the Taiwan Food and Drug Administration issued the same approval in September 2023 and October 2023, respectively. In July 2021, we obtained new drug clinical study (IND) approval from the National Drug Administration for the CAN106 study for the treatment of PNH conducted in China; in February 2022, the CAN106I single-dose escalation study in Singapore recorded positive top-line results; in June 2023, we reported the initial positive results of the CAN106iB phase dose escalation trial study for Chinese PNH patients. The results showed good efficacy and safety. LDH showed a dose-dependent decrease in hemoglobin levels, and confirmed clinically significant improvement in hemolysis suppression and transfusion-dependent anemia. Furthermore, in July 2022, the first patient administration was completed in the CAN103I trial for Gaucher disease conducted in China, and the first patient administration was completed in the phase II trial for Gaucher disease in January 2023. Positive top-line data from the CAN103 key trial for Gaucher disease in China was reported in August 2024. In addition to biologics and small molecules, we are investing in next-generation gene therapy technology. Gene therapy provides a possible one-time, long-lasting treatment for rare genetic diseases with limited treatment options. As of June 30, 2024, we are using an AAV sL65 capsid carrier to develop treatments for Fabray's disease and Pompeii disease, and we have obtained authorization from LogicBio Therapeutics for these two indications. In January 2023, we announced that we have exercised our option to obtain an exclusive global license from the Massachusetts State University Chan School of Medicine to develop, produce, and commercialize a novel second-generation gene therapy to treat SMA. Additionally, we have obtained an authorization from ScriptR Global for a dual-vector technology called “StitChR” to treat DMD. StitchR technology can achieve greater gene load transmission through two independent AAVs, and is the foundation of our DMD gene therapy program currently in the research and discovery stage. As of June 30, 2024, we have internally generated proof of concept data for DMD preclinical studies.