BEIJING & BURLINGTON, Mass.--(BUSINESS WIRE)--CANbridge Pharmaceuticals, Inc. (�CANbridge,� 1228.HK), a China and U.S.-based global biopharmaceutical company committed to the research, development and commercialization of transformative therapies to treat rare diseases and oncology, today announced financial results for the six months ended 2022 and a corporate update.

�We have made significant progress over the first half of the year advancing the development and commercializing of transformative products to treat debilitating rare diseases,� said James Xue, Ph.D., CANbridge Founder, Chairman and CEO. �We have successfully built a high-value pipeline of assets, with validated mechanisms of action, that reduce, in our opinion, the risks associated with developing these drugs, each of which targets large underserved markets, thereby improving the probability of commercial success. So far this year, we have initiated three Phase II clinical trials in China, for CAN008, CAN108, and CAN103 in China; announced positive Phase I results for CAN106, and presented initial data from a novel second-generation scAAV9 gene therapy at the prestigious American Society of Gene & Cell Therapy annual meeting. We also continue to build our organization, with recent additions to both the board and our management team, and with the creation of a scientific advisory board of experts to help us maximize the development and commercial opportunity of CAN106. We look forward to a prosperous second half, as CANbridge continues to shape the emerging rare disease infrastructure in China and to provide total solutions to patients globally.�

Recent Highlights

Hunterase� (CAN101), an enzyme replacement therapy and the only approved targeted therapy approved to treat MPS II (Hunter�s disease) in China.

• CANbridge commercial team has made good progress in Hunterase�, launched in May 2021, in mainland China.
• Identification of new patients is accelerating, as is the expansion of commercial insurance coverage. Since launch, 539 patients have been identified, as of June 30, 2022. In addition, in China, Hunterase has been entered into the government endorsement commercial insurance programme (Huiminbao) in 5 provinces and 42 cities.

CAN108 (maralixibat), an oral, minimally absorbed reversible inhibitor of the ileal bile acid transporter (IBAT) that is under development to treat rare cholestatic liver diseases. Maralixibat (LIVMARLI�) is approved to treat Alagille syndrome (ALGS), in the US, and is under investigation for the treatment of progressive familial intrahepatic cholestasis (PFIC) and biliary atresia (BA). CANbridge has the exclusive rights to develop, commercialize, and in some cases, manufacture CAN108 in Greater China.

• Dosed the first patient in the Phase 2 EMBARK1 study of CAN108 (maralixibat oral solution (LIVMARLI�)) in biliary atresia (BA) in China, at Children�s Hospital of Capital Institute of Pediatrics (CIP), in Beijing. The clinical trial in China is part of the global EMBARK study in BA. The multi-center randomized controlled Phase 2 study to evaluate the efficacy and safety of CAN108 in the treatment of patients with BA after Kasai surgery is expected to enroll up to 20 patients in China and 72 patients globally.
• Announced that CAN108 (maralixibat oral solution (LIVMARLI�)) has been approved for the treatment for ALGS under the Early and Pilot Implementation Policy in Boao Lecheng International Medical Tourism Pilot Zone. This allows CAN108 to be imported and used as an urgently needed drug in the region.
• Announced that the Chinese National Medical Products Administration (NMPA) and the Taiwan Food and Drug Administration (TFDA) have accepted the New Drug Application/Orphan Drug Registration (NDA/ODR) for CAN108 (maralixibat oral solution (LIVMARLI�)) for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) 1 year of age and older. LIVMARLI (maralixibat) oral solution was approved in 2021 by the US Food and Drug Administration (FDA) for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) 1 year of age and older. CAN108 has also been granted priority review by China�s NMPA and could be approved in the first half of 2023.

CAN106, a humanized monoclonal antibody for the treatment of complement-mediated diseases, including paroxysmal nocturnal hemoglobinuria (PNH), and various other complement-mediated diseases, that are targeted by anti-C5 antibodies.

• Reported positive top-line CAN106 Phase 1 data from Singapore trial 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 paroxysmal nocturnal hemoglobinuria (PNH) in China. The Phase 1b/2 study is a multi-center, open-label study to evaluate the tolerability, efficacy, safety and PK/PD of CAN106 administered intravenously to complement inhibitor treatment-na�ve PNH patients. CAN106 was previously shown to be safe and well-tolerated, with dose-dependent and linear pharmacokinetic exposure, in a study of healthy volunteers in Singapore. The data also showed that free C5 and CH50 could be effectively inhibited. Based on these results, China�s National Medical Products Administration approved the CAN106 Phase 1b/2 trial for the treatment of patients with PNH. The trial is composed of three cohorts. Enrollment in cohorts 1 and 2 is expected to be completed by Q3 2022. Depending on the results from cohorts 1 and 2, initiation of cohort 3 could take place by the end of 2022, with a potential interim data readout in the fourth quarter of 2022 or in the first quarter of 2023.

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

• Enrollment continues in the CAN008 Phase 2 trial in patients with newly diagnosed GBM in China. This multi-center, randomized, double-blind, placebo-controlled trial will evaluate the efficacy of CAN008 and explore the correlation between different biomarkers and treatment outcome. The Company anticipates completing trial enrollment at the end of 2022, with a potential interim read out in the second half of 2023.
• CANbridge expects to commercialize CAN008 in China as a combination therapy with the standard-of-care for GBM (radiotherapy plus chemotherapy).

CAN103, an enzyme replacement therapy (ERT) for the treatment of Gaucher disease (GD).

• Dosed the first patient in the CAN103 Phase 1/2 for the treatment of patients with Gaucher disease (GD) Types I and III in China. The multi-center Phase 1/2 clinical trial will consist of two parts: Part A (Phase 1) is an open-label study to evaluate the safety, tolerability and pharmacokinetics of different dose levels of CAN103 in a small number of treatment-na�ve subjects with Gaucher disease Type I. Part B (Phase 2) is a randomized, double-blind, parallel group, dose comparison study to assess the safety and efficacy of CAN103 in a larger number of subjects with Gaucher disease Type I or III.
• According to Frost & Sullivan, there were approximately 3,000 GD patients in 2020 in China.

Advanced lead 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.

• The Company presented initial data from a novel second-generation scAAV9 gene therapy, expressing co-hSMN1 from an endogenous hSMN1 promoter, that demonstrated superior potency, efficacy and safety in mice with spinal muscular atrophy (SMA), compared to the benchmark vector, scAAV9-CMVen/CBhSMN1, which is similar to the vector used in the gene therapy approved by the US Food and Drug Administration for the treatment of SMA. This is the first data to be presented from the gene therapy research collaboration between CANbridge and the Gao Lab at the Horae Gene Therapy Center.

Organizational Updates

• Formed a world-class Complement Disease Scientific Advisory Board focused on the global development of CAN106, a novel, long-acting monoclonal antibody directed against C5 complement. CANbridge is developing CAN106 for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and other complement-mediated diseases. CAN106 is currently in a Phase 1b/2 PNH trial in China. The Board will offer guidance on the CAN106 clinical development program, as well as explore the potential for CAN106 in other indications.
• Appointed Edward Hu as non-executive director of the Board of Directors and member of the Remuneration Committee, replacing Xiao Le, who resigns from the CANbridge Board. Edward Hu brings to the Company a deep and varied C-level biopharmaceutical experience, having served as both Co-Chief Executive Officer and Chief Financial Officer at WuXi AppTec before his current position of Vice Chairman, Global Chief Investment Officer, Executive Director, Strategy Committee Member.
• Appointed Pauline Li, MD, to the position of Senior Vice President of Clinical Development and Operations. Dr. Li brings to CANbridge a wealth of international clinical development experience, in both small molecule and biologic products, across multiple indications and markets. Most recently, she was at Connect Biopharma, where she held the position of Vice President of Clinical Development and was responsible for creating and implementing the clinical development strategy.
• Appointed Lan Hu, Ph.D. as an independent, nonexecutive Director of the Board of Directors and member of the remuneration committee, effective as of February 16, 2022. Dr. Hu has a rich background in healthcare investment, operations and administrative management, and is a seasoned entrepreneur, having founded Beijing Amcare Women�s & Children�s Hospital Co. in 2004, where she also served as Director, Chairman of the Board and General Manager.

Upcoming Milestones

• CAN106 � Interim data from the first two cohorts in the Phase 1b/2 clinical trial in PNH patients in China expected in the fourth quarter of 2022 or in the first quarter of 2023
• CAN008 � Expects to complete enrollment in the Phase 2 clinical trial of CAN008 for the treatment of GBM in 2022, with a potential interim data read out in the second half of 2023
• CAN108 � Expects to receive approval of the NDA in ALGS in the first half of 2023 and to continue to enroll patients in the Phase 2a EMBARK trial for the treatment of BA
• Gene Therapy � Plans to share additional data in SMA in the second half of 2022

Financial Highlights

• Bank balances and cash amounted to approximately RMB604.6 million as of June 30, 2022. Cash balance reflects proceeds raised for a pre-IPO financing of RMB332.3 million, in May 2021, and proceeds raised in the December 2021 IPO of RMB562.3.0 million, less expenses incurred.
• Revenue increased by RMB22.5 million, from RMB12.2 million for the six months ended June 30, 2021, to RMB34.7 million for the six months ended June 30, 2022, which was mainly attributable to the increase of sales from Hunterase� and Nerlynx�.
• Research and development expenses decreased by approximately RMB116.5 million, from RMB274.8 million for the six months ended June 30, 2021, to RMB158.3 million for the six months ended June 30, 2022, which was primarily attributable to decreased upfront and milestone payments made to our licensing partners, partially offset by our increased testing and clinical trial expenses and ,increased R&D employee costs.
• Loss for the Reporting Period decreased by approximately RMB95.2 million, from RMB344.2 million for the six months ended June 30, 2021, to RMB249.0 million for the six months ended June 30, 2022, which was primarily attributable to the decrease of research and development costs.
• The adjusted loss for the period decreased by RMB113.7 million, from RMB342.6 million for the six-months ended June 30, 2021, to RMB228.9 million for the six months ended June 30, 2022. The adjusted loss for the period is arrived at by adjusting the IFRS loss for the Reporting Period of RMB249.0 million (for the six months ended June 30, 2021: RMB344.2 million) from excluding the effect of (i) a one-time, non-cash, IFRS fair value changes of our pre-IPO convertible redeemable preferred shares and derivative financial instruments, (ii) the share-based payment expenses, and (iii) the listing expenses. Please refer to the section headed �Non-IFRS Measures� of this announcement, for details.

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 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. 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: www.canbridgepharma.com.

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.

Contacts

U.S. Investor Relations:
Chris Brinzey

ICR Westwicke

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China Investor Relations:
CANbridge Pharmaceuticals Inc.

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Media:
Deanne Eagle

Planet Communications

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917.837.5866

China Media:
Mandy Go, Brigid Lee, Yvonne Lee

Strategic Financial Relations Limited

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