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BEIJING & CAMBRIDGE, Mass.--(BUSINESS WIRE)-- CANbridge Pharmaceuticals, Inc. (“CANbridge,” stock code 1228.HK), a global biopharmaceutical company, with a foundation in China, committed to the research, development and commercialization of transformative therapies to treat rare diseases and oncology, today announced financial results for the twelve months ended 2022 and corporate update. “We have made significant progress building CANbridge and advancing our pipeline that consists of 14 drug assets targeting rare diseases, each with significant market potential,” said James Xue, Ph.D., CANbridge founder, chairman and CEO. “We are anticipating an exciting year ahead and we look forward to sharing a number of important program updates that we believe will drive both near-and long-term value to our stockholder’s. Specifically, over the course of the year, we are planning for important data readouts, including data from key registrational trials for CAN008 for the treatment of glioblastoma and CAN106 for the treatment of paroxysmal nocturnal hemoglobinuria. In addition to these important milestones, we are anticipating the potential approval of LIVMARLI® (maralixibat/CAN108) for the treatment of Alagille syndrome, a rare liver disease in the first half of 2023.” Upcoming Milestones CAN108 – Potential approval for Alagille syndrome (ALGS) in the first half of 2023 CAN008 – Anticipate interim read out from the Phase 2 trial for glioblastoma (GBM) in the second half of 2023 CAN106 – Plan to announce top-line data from the Phase 1b trial for paroxysmal nocturnal hemoglobinuria (PNH) mid-year 2023 CAN103 – Anticipate filing an New Drug Application (NDA) by mid-2024 for the treatment of Gaucher disease (GD) Gene Therapy – The Company plans to Investigational New Drug (IND) with the U.S. Food and Drug Administration (FDA) for its novel second-generation scAAV9 gene therapy for the treatment of spinal muscular atrophy (SMA) by the fourth quarter of 2024 Financial Highlights Revenue increased by RMB47.8 million, or 153.2%, from RMB31.2 million for the year ended December 31, 2021, to RMB79.0 million, for the year ended December 31, 2022, which was mainly attributable to the increase of sales from Hunterase® and Nerlynx®. Loss for the year decreased by approximately RMB593.5 million, or 55.1%, from RMB1,077.0 million for the year ended December 31, 2021, to RMB483.5 million for the year ended December 31, 2022, which was primarily attributable to the decrease of loss on fair value changes of convertible redeemable preferred shares and R&D costs. The adjusted loss for the year decreased by RMB124.6 million, or 21.4%, from RMB581.3 million, for the year ended December 31, 2021, to RMB456.7 million for the year ended December 31, 2022. The adjusted loss for the year is arrived at by adjusting the IFRS loss for the year of RMB483.5 million (2021: RMB1,077.0 million) from excluding the effect of (i) a one-time, non-cash, IFRS fair value changes to pre-IPO convertible redeemable preferred shares and derivative financial instruments, (ii) the share-based payment expenses, and (iii) the listing expenses. Research and development (“R&D”) expenses decreased by approximately RMB116.5 million, or 27.2%, from RMB427.7 million for the year ended December 31, 2021 to RMB311.2 million for the year ended December 31, 2022, which was primarily attributable to decreased upfront and milestone payments made to licensing partners, partially offset by increased testing and clinical trial expenses. R&D expense excluding milestone payments increased by RMB37.8 million, from RMB213.9 million for the year ended December 31, 2021 to RMB251.7 million for the year ended December 31, 2022. The Company’s cash and bank balances as of December 31, 2022 were RMB463.1 million. Recent Highlights Hunterase (CAN101), an enzyme replacement therapy for the treatment of MPS II, also known as Hunter syndrome. MPS II has been included in the “First National List of Rare Diseases” since May 2018. Launched in May 2021 in mainland China as the first and the only enzyme replacement therapy (ERT) for MPS II. Identification of new patients is accelerating as is the expansion of commercial insurance coverage. Since the launch, the Company has identified 667 patients as of December 31, 2022. In addition, in China, Hunterase has entered into 78 cities’ commercial insurance program (“Huiminbao”). LIVMARLI, an oral, minimally absorbed reversible inhibitor of the ileal bile acid transporter (IBAT) that is under development to treat rare cholestatic liver diseases. CANbridge has the exclusive rights to develop, commercialize, and under certain conditions, manufacture LIVMARLI in Greater China. LIVMARLI is approved to treat ALGS in the United States (U.S.) and Europe and is under investigation for the treatment of progressive familial intrahepatic cholestasis (PFIC) and biliary atresia (BA)-. Dosed the first patient in the Phase 2 study of LIVMARLI in biliary atresia (BA) in China-. This clinical trial in BA is being conducted by Mirum and supported by CANbridge under the license agreement with Mirum-. LIVMARLI has also been granted priority review by the National Medical Products Administration (NMPA) with the potential to be approved in the first half of 2023 CAN106, a novel, long-acting monoclonal antibody for the treatment of complement-mediated diseases, including PNH, myasthenia gravis (MG) and various other complement-mediated diseases that are targeted by anti-C5 antibodies. PNH has been included in the “First National List of Rare Diseases” in China since May 2018. Received FDA Orphan Drug Designation for the treatment of MG in November 2022 Reported positive top-line Phase 1 data from the Singapore trial first reported in February 2022. Results suggest complete blockade of complement function at safe and well-tolerated doses Dosed the first patient in the CAN106 Phase 1b/2 trial for treatment of PNH in China in March 2022. The ongoing Phase 1b part consists of 3 cohorts and is on track for top-line data mid-year 2023 CAN008, a glycosylated CD95-Fc fusion protein being developed for the treatment of glioblastoma multiforme (GBM). Completed Phase 2 trial enrollment in March 2023 and the potential interim read out is anticipated in the second half of 2023 Expect to commercialize CAN008 in China as a combination therapy with the standard of care for GBM (radiotherapy plus chemotherapy) CANbridge owns the rights to CAN008 for all potential indications in Greater China, including as a potential novel treatment option to treat COVID-19 CAN103, an ERT for the treatment of GD. GD has been included in the “First National List of Rare Diseases” in China since May 2018. Completed Part A of the ongoing Phase 1/2 clinical trial in China and initiated Part B in the first quarter of 2023, which will serve as a potential registrational trial. CANbridge expects to NDA by mid-2024 CAN103 is the first clinical stage ERT being developed for GD in China Gene Therapy, advanced the world-class CANbridge gene therapy platform, focusing on adeno-associated virus (AAV) as a gene delivery vehicle, with potential as a one-time durable therapy for many genetic diseases. Programs in Fabry disease and SMA have been included in the “First National List of Rare Diseases” since May 2018. Opened the CANbridge Next-Generation Innovation and Process Development Facility in July 2022, a full in-house gene therapy research and development facility to develop novel potentially curative, gene therapies for rare genetic diseases, including Pompe disease, Fabry disease, SMA, and other neuromuscular conditions. Presented initial gene therapy data from the research agreement with the Horae Gene Therapy Center, at the UMass Chan Medical School, at the European Society of Gene and Cell Therapy (ESGCT) 29th Annual Congress in October 2022 and at the 2022 American Society of Gene and Cell Therapy (ASGCT) Annual Meeting in May 2022. Data from a novel second-generation scAAV9 gene therapy, expressing co-hSMN1 from an endogenous hSMN1 promoter, demonstrated superior potency, efficacy and safety in mice with SMA, compared to the benchmark vector, scAAV9-CMVen/CB-hSMN1, which is similar to the vector used in the gene therapy approved by the FDA for the treatment of SMA. Announced an in-license agreement with the UMass Chan Medical School for the global development and commercialization rights to a novel second-generation scAAV9 gene therapy, expressing co-hSMN1 from an endogenous hSMN1 promoter, for the treatment of SMA. CANbridge plans to U.S. IND by the fourth quarter of 2024. Obtained non-exclusive worldwide rights to the LogicBio (a wholly owned subsidiary of Alexion, AstraZeneca Rare Disease) proprietary manufacturing process for the development of Fabry and Pompe gene therapies in the second half of 2022. CANbridge completed the full technology transfer of these gene therapy products in late 2022. Organizational Updates Formed a world-class Complement Disease Scientific Advisory Board (the Board) focused on the global development of CAN106, a novel, long-acting monoclonal antibody directed against C5 complement. The Board will offer guidance on the CAN106 clinical development program, as well as explore the potential for CAN106 in other indications. About CANbridge Pharmaceuticals Inc. CANbridge Pharmaceuticals Inc. (HKEX:1228) is a China and U.S.-based global biopharmaceutical company committed to the research, development and commercialization of transformative therapies for rare disease and rare oncology. CANbridge has a differentiated drug portfolio, with three approved drugs and a pipeline of 11 assets, targeting prevalent rare disease and rare oncology 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, as well as glioblastoma multiforme. CANbridge is also building next-generation gene therapy development capability through a combination of collaboration with world-leading researchers and biotech companies and internal capacity. CANbridge global partners include: Apogenix, GC Pharma, Mirum, Wuxi Biologics, Privus, the UMass Chan Medical School, the University of Washington School of Medicine, Scriptr Global and LogicBio. For more on CANbridge Pharmaceuticals Inc., please go to: . Forward-Looking Statements The forward-looking statements made in this article relate only to the events or information as of the date on which the statements are made in this article. Except as required by law, we undertake no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise, after the data on which the statements are made or to reflect the occurrence of unanticipated events. You should read this article completely and with the understanding that our actual future results or performance may be materially different from what we expect. In this article, statements of, or references to, our intentions or those of any of our Directors or our Company are made as of the date of this article. Any of these intentions may alter in light of future development. View source version on businesswire.com: Contacts U.S. Investor Relations: Chris Brinzey ICR Westwicke Chris.brinzey@westwicke.com China Investor Relations CANbridge Pharmaceuticals Inc. ir@canbridgepharma.com Media: Deanne Eagle Planet Communications deanne@planetcommunications.nyc 917.837.5866 Source: CANbridge Pharmaceuticals, Inc. View this news release online at:

BEIJING & BURLINGTON, Mass.--(BUSINESS WIRE)-- 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 diseases and oncology, announced that CAN106, a clinical-stage recombinant humanized monoclonal antibody targeting C5, has been granted Orphan Drug Designation by the United States Food and Drug administration (FDA) for the treatment of myasthenia gravis (MG), an autoimmune neuromuscular disease that causes weakness in skeletal muscles. CAN106 is in an ongoing Phase 1b/2 trial in paroxysmal nocturnal hemoglobinuria (PNH) in China. “Orphan Drug Designation for CAN106 in MG is both a validation of CANbridge innovation and a major milestone as our first US FDA regulatory designation,” said James Xue, Ph.D., CANbridge Founder, Chairman and CEO. “We continue to advance our global development strategy for CAN106 and look forward to developing CAN106 for myasthenia gravis and other complement-mediated diseases.” About CAN106 CAN106 is a clinical-stage investigational novel, long-acting recombinant humanized monoclonal antibody that binds to and neutralizes C5, a key component of the complement system. By preventing the cleavage of C5 into C5a and C5b, CAN106 is intended to prevent the C5b-dependent formation and activation of the membrane attack complex (MAC) on susceptible cell surfaces, with resulting cell lysis (destruction). CAN106 acts downstream of C3 in the complement pathway, preserving the generation of C3a and C3b, which are important for innate immunity. CAN106 has demonstrated a favorable PK/PD profile, safety, and tolerability, indicating that CAN106 has the potential to effectively inhibit C5 in patients with certain complement-mediated diseases. About Myasthenia Gravis Myasthenia gravis (MG) is a rare, acquired, neuromuscular disease that causes weakness in the muscles under voluntary control, including those involved in breathing, swallowing, eye movement and moving the arms and legs. MG is caused when the immune system blocks or destroys muscle fiber sites that normally receive acetylcholine, a brain chemical that activates muscle contraction. MG can result in hospitalization and the need for ventilation. MG can occur at any age, although it most commonly affects women under 40 and men over 60. According to the Myasthenia Gravis Foundation of America, it is estimated that 20 in 100,000 Americans have MG, or approximately 36,000 to 60,000 cases, but given that the disease is underdiagnosed, these numbers could be much higher. There is no cure. About CANbridge Pharmaceuticals Inc. CANbridge Pharmaceuticals Inc. (HKEX:1228) global biopharmaceutical company, with a foundation in China, committed to the research, development and commercialization of transformative therapies for rare disease and rare oncology. CANbridge has a differentiated drug portfolio, with three approved drugs and a pipeline of 10 assets, targeting prevalent rare disease and rare oncology 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, as well as glioblastoma multiforme. 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) and other neuromuscular conditions, and collaborates with world-leading researchers and biotech companies. Animal data from the SMA gene therapy was presented at the American Society for Gene and Cell Therapy (ASCGT), the European Society for Gene and Cell Therapy (ESCGT) and the World Muscle Congress. CANbridge global partners include: Apogenix, GC Pharma, Mirum, Wuxi Biologics, Privus, the UMass Chan Medical School, the University of Washington School of Medicine and Scriptr Global. For more on CANbridge Pharmaceuticals Inc., please go to: . Forward-Looking Statements The forward-looking statements made in this article relate only to the events or information as of the date on which the statements are made in this article. Except as required by law, we undertake no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise, after the data on which the statements are made or to reflect the occurrence of unanticipated events. You should read this article completely and with the understanding that our actual future results or performance may be materially different from what we expect. In this article, statements of, or references to, our intentions or those of any of our Directors or our Company are made as of the date of this article. Any of these intentions may alter in light of future development.