Beijing, China; Cambridge, Mass., August 30, 2023 — 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 for rare diseases and oncology indications, today announced its financial results for the six months ended June 30, 2023 plus corporate updates.

“On the product front, it had been a very productive first half year for CANbridge. We achieved multiple data and regulatory milestones that we believe will continue to drive both near- and long-term value for our stockholders,” said James Xue, Ph.D., CANbridge founder, chairman, and CEO. “Regarding new data, we have encouraging updates on several programs, notably CAN106 for treating paroxysmal nocturnal hemoglobinuria (PNH), as well as CAN203, our gene therapy candidate for SMA. On the regulatory front, we are pleased to have received approval for LIVMARLI® (maralixibat/CAN108) in China for treating Cholestatic Pruritus in patients with the rare liver disease Alagille syndrome (ALGS). Livmarli is the first and only approved product in China for the disease, thus marked a pivotal milestone for CANbridge, given the sizeable commercial opportunity and the potential to expand the Livmarli label to cover multiple other rare liver disease indications. In the second half year, we look forward to achieving continuous progress in developing our pipeline of leading therapies for rare diseases.”

Upcoming Milestones

• CAN108 – Plan to launch Livmarli for patients with ALGS in China in Q1 2024.
• CAN108 – Anticipate New Drug Application (NDA) approval for Livmarli's use in ALGS patients aged one year and older in Taiwan and Hong Kong by the end of 2023.
• CAN108 – Data from the Mirum-sponsored EMBARK Phase 2 trial inbiliary atresia patients is expected in H2 2023.
• CAN008 – Planto report data from the Phase 2 clinical trial in H1 2024.
• CAN106 – Begin enrolling a Phase 2 clinical trial in PNH in China that will commence in H2 2023 with data potentially ready in H2 2024.
• Gene Therapy – Advancenovel second-generation scAAV gene therapy for the treatment of spinal muscular atrophy (SMA)

Financial Highlights

• Revenue increased by RMB8.4 million, or 24.2%, to RMB43.1 million for the six months ended 30 June 2023 from RMB34.7 million for the same period last year, mainly attributable to the increase in sales of Hunterase® and Livmarli®.
• Research and development (“R&D”) expenses decreased by approximately RMB15.3 million, or 9.7%, to RMB143.0 million for the six months ended 30 June 2023 from RMB158.3 million for the same period last year, primarily attributable to the decrease in upfront and milestone payments made to CANbridge’s licensing partners and in technical service fees, partially offset by the increase in depreciation and amortization costs.
• Loss for the Reporting Period decreased by approximately RMB30.8 million, or 12.4%, to RMB218.2 million for the six months ended 30 June 2023 from RMB249.0 million for the same period last year, primarily attributable to the increase in revenue and decrease in selling and distribution expenses, R&D expenses and administrative expenses.

Recent Highlights

Hunterase (CAN101), an enzyme replacement therapy for treating MPS II, also known as Hunter syndrome, has been on the “First National List of Rare Diseases” since May 2018.

• It was launched in May 2021 in mainland China as the first and only ERT for MPS II, and since then more new patients have been identified, up to 739 as of 30 June 2023.
• Expanded relevant commercial insurance programs (Huiminbao) to 109 cities, covering a population of 586 million in China.

LIVMARLI is an oral, minimally absorbed reversible inhibitor of the ileal bile acid transporter (IBAT) being developed for treating rare cholestatic liver diseases. CANbridge has the exclusive rights to develop, commercialize, and under certain conditions, manufacture LIVMARLI in Greater China.

• On May 29, 2023, the Chinese National Medical Products Administration (NMPA) approved CAN108, making Livmarli the first and only approved product in China to be marketed for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) aged one year and older.  
• CANbridge plans to launch Livmarli for ALGS in Q1 2024.
• Completed enrollment of the EMBARK Phase 2 study of Livmarli in biliary atresia (BA) in China inMay 2023. The clinical trial in BA is conducted by Mirum and supported by CANbridge under the license agreement with Mirum.
• Expected to obtain NDA approval for Livmarli's use in ALGS patients aged one year and older in Taiwan and Hong Kong by the end of 2023.
• Planto submit NDA for Livmarli’s use in PFIC patients in Taiwan and mainland China in the second half of 2023.

CAN106 is a novel, long-acting monoclonal antibody for treating 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 on the “First National List of Rare Diseases” in China since May 2018.

• Reported positive preliminary top-line Phase 1 data from the ongoing Phase 1b study of CAN106 in China for PNH. The results suggest complete blockade of complement function at safe and well-tolerated doses.
• Based on the positive results from the Phase 1b study, CANbridge will begin enrolling a registrational clinical trial in PNH in China that will commence in the second half of 2023 with data amassed potentially in the second half of 2024.

CAN008, a glycosylated CD95-Fc fusion protein being developed for the treatment of glioblastoma multiforme (GBM).

• The IDMC (independent data monitoring committee) completed an interim analysis and review of the ongoing Phase 2 study of CAN008 in China in patients with newly diagnosed GBM and recommended the study to continue without any changes to the current trial design.

CAN103 is an ERT for treating Gaucher Disease (GD) and GD has been included on 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, with the latter to serve as a potential registrational trial.
• CAN103 is the first clinical stage ERT being developed for GD in China.

In Gene Therapy, CANbridge advanced its world-class 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 on the “First National List of Rare Diseases” since May 2018.

• Appointed Jason West, Ph.D., as Vice President, Head of Gene Therapy Research. Dr. West possesses expertise in areas such asgene therapy development, platform innovation and clinical candidate development.
• Presented preclinical data at the 2023 American Society of Gene and Cell Therapy (ASGCT) Annual Meeting. The data shared evidenced the potential of the novel, second-generation scAAV in treating SMA as it expresses a codon optimized (co)-hSMN1 from an endogenous hSMN1 promoter. Low-dose intracerebroventricular delivery of the gene therapy was able to achieve superior potency, efficacy and safety in mice with SMA, compared to the benchmark vector, scAAV-CMVen/CB-hSMN1, which is similar to the FDA-approved gene therapy vector for SMA.

CANbridge will also host a conference call to discuss the 2023 interim results and recent corporate progress. To register for the English session, please click here. To register for the Chinese session, please click here. This webcast will be English, and translation will be provided on the Chinese channel.

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 and rare oncology. CANbridge has a differentiated drug portfolio, with 4 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 in 2022 at the American Society for Gene and Cell Therapy (ASGCT), the European Society for Gene and Cell Therapy (ESGCT) and the World Muscle Congress. 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.

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CANbridge Pharmaceuticals Inc.

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