CANbridge Announces Financial Results and Corporate Updates for the Twelve Months Ended December 31, 2021

BEIJING & CAMBRIDGE, Mass.–(BUSINESS WIRE)–CANbridge Pharmaceuticals, Inc. (“CANbridge,” stock code 1228.HK), a China-based global biopharmaceutical company committed to the research, development and commercialization transformative therapies to treat rare diseases and oncology, today announced 2021 year-end results and corporate update.

“2021 was a transformative year for CANbridge as we made progress in our global mission of developing treatments for rare diseases along all fronts, advancing our clinical pipeline and reporting promising results, commercializing our first rare disease product, deepening our next-generation gene therapy capacity and completing our IPO,“ said James Xue, Ph.D., CANbridge Founder, Chairman and CEO. “We are building a pipeline of assets with validated mechanisms of action, which potentially reduces development risks, as we target large markets with few treatment options and great need. Late in the year, we initiated the launch of our first product, Hunterase®, for the treatment of Hunter syndrome (MPS II), establishing a beachhead in China from which we intend to expand our commercial efforts. Our pipeline has also progressed successfully, with the initiation of two, Phase II clinical trials in China, for CAN008 and CAN108, positive Phase I results for CAN106, and significant progress in building our gene therapy efforts. Finally, with a successful IPO, we are well funded to advance our rare disease therapeutics across multiple modalities and indications in 2022. We look forward to working with all stakeholders to help shape the emerging rare disease infrastructure in China and to provide total solutions to patients globally.”

Recent Highlights

Completed successful listing on The Stock Exchange of Hong Kong Limited, raising HKD$604 million to develop and commercialize the Company’s rare disease and gene therapy pipeline.

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

  • The Company launched the product in mainland China in May 2021 into a non reimbursed market.
  • The Company plans to expand its dedicated, in-house commercial team and expects to assemble a full-fledged rare disease commercial team in China, with over 300 members, in the next five years, with the ability to commercialize multiple rare disease products.

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

  • Amended the Investigational New Drug (IND) application to allow CAN008 to be studied in a first-line Phase II trial, based on the positive preliminary efficacy results obtained in the Phase 1 trial in Taiwan, which suggested that CAN008 has the potential to become a standard-of-care treatment.
  • Initiated a Phase II trial of CAN008 in China in patients with GBM in April 2021 and dosed the first patient in China in October 2021. The Phase II clinical trial is designed to be multi-center, randomized, double-blind and placebo-controlled to investigate the efficacy of CAN008 and explore the correlation of biomarkers to treatment outcome.
  • CANbridge expects to commercialize CAN008 in China as a combination therapy with the GBM standard-of-care (radiotherapy plus chemotherapy).

CAN108 (maralixibat) an oral, minimally absorbed reversible inhibitor of the ileal bile acid transporter (IBAT) and is being developed to treat rare cholestatic liver disease, including Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC) and biliary atresia (BA).

  • Obtained, in April 2021, an exclusive license from Mirum Pharmaceuticals (“Mirum”) to develop, manufacture and commercialize CAN108 (maralixibat) in Greater China for ALGS, PFIC and BA. Maralixibat is currently the first and only approved targeted drug for ALGS by the United States Food and Drug Administration (FDA) in USA.
  • In May 2021, CANbridge’s development partner, Mirum, began patient recruitment and clinical site management in China for a Phase II global multi-center clinical trial evaluating CAN108 for the treatment of BA after Hepatoportoenterostomy.
  • Submitted, in December 2021, a New Drug Application (NDA) for CAN108 for the treatment of cholestatic pruritus in patients with ALGS in mainland China , based on data from global studies conducted by our collaboration partner, Mirum.
  • CAN108 New Drug Application (NDA) for ALGS accepted by China’s National Medical Product Administration and granted priority review in January 2022.

CAN106, a humanized monoclonal antibody for the treatment of complement-mediated diseases, including paroxysmal nocturnal hemoglobinuria (PNH), and various other complement-mediated diseases.

  • Completed a Phase I clinical trial in healthy volunteers for CAN106 in Singapore in February 2022. This first-in-human study is designed to be a randomized, double-blind, placebo controlled and single ascending dose study in 31 healthy volunteers to evaluate the safety, pharmacokinetics, pharmacodynamics and development of anti-drug antibodies of CAN106.
  • Obtained the IND approval from the NMPA for PNH in July 2021.
  • Reported positive top line CAN106 Phase 1 data from Singapore trial on Feb 2022. CAN106 was safe and well-tolerated. Results also suggest that CAN106 achieved a complete blockade of complement function.

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

  • Obtained the IND approval for CAN103 from the NMPA in October 2021. Currently preparing to begin a Phase 1 trial in adult and adolescent Gaucher disease patients.

Advanced 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.

  • Obtained an exclusive worldwide license from LogicBio Therapeutics for a next- generation capsid platform, for use in gene editing and gene therapy, for two targets for the treatment of Fabry and Pompe disease, thereby expanding CANbridge’s rare disease portfolio. The agreement also includes worldwide options to license the platform for development for two additional indications, as well as an option to license LB-001, an investigational gene editing technology potentially for the treatment of methylmalonic acidemia (MMA), in Greater China.
  • In October 2021, entered into a research collaboration and license agreement with Scriptr Global, Inc. for the exclusive worldwide rights to develop, manufacture and commercialize a gene therapy candidate for the treatment of dystrophinopathies, using Scriptr Global’s Stitchr™ platform, a proprietary ribozyme-mediated RNA assembly technology.
  • Identified two gene therapies to advance into clinical development targeting rare neuromuscular diseases: CAN201, for treatment of Fabry disease and CAN202, for the treatment of Pompe disease.

Upcoming Milestones

  • CAN106 – Following the positive readout of the Phase 1 data in Singapore, CANbridge plans to initiate a Phase 1b/2 clinical trial in PNH patients in China in the first half of 2022.
  • CAN008 – Continue to enroll patients in the Phase 2 clinical trial of CAN008 for the treatment of glioblastoma multiforme (GBM). Expect to have CAN008 Phase 2 interim readout in 2023.
  • CAN108 – Receive NDA approval in ALGS and dose the first patient in a Phase 2 clinical trial in BA.
  • CAN201 – Schedule pre-IND meeting with the U.S. Food and Drug Administration in the second half of 2022 and submit IND application in 2023. Complete the development of pilot scale manufacturing process, and tech transfer in the second half of 2022.
  • Gene Therapy – Advance in-house gene therapy global program and generate non-human proof-of-concept data during the year. Announce pre-clinical lead candidate in DMD in 2023.

Financial Highlights

  • Bank balances and cash amounted to approximately Rmb745.8 million as of December 31, 2021. Cash balance reflects proceeds raised for a pre-IPO financing in May 2021, and proceeds raised in the December 2021 IPO, less expenses incurred.
  • The Company reported revenue of Rmb31.2 million. Revenue in 2021 was made up of sales of two marketed products: Hunterase®, Nerlynx®.
  • The research and development expenses increased by approximately RMB318.1 million, to approximately RMB427.7million.
  • The loss before taxes increased by approximately RMB231.0 million, to approximately RMB1,077.0 million.
  • The adjusted net loss increased by approximately RMB370.7million, to approximately RMB581.3 million.

About CANbridge Pharmaceuticals Inc.

CANbridge Pharmaceuticals Inc. (“CANbridge,” stock code 1228.HK) is a China-based global biopharmaceutical company committed to the research, development and commercialization of transformative therapies for rare disease and rare oncology.

CANbridge has a comprehensive and differentiated pipeline of 13 drug assets with significant market potential, targeting some of the most prevalent rare diseases and rare oncology. These include Hunter syndrome (MPS II) and other lysosomal storage disorders (LSDs), complement mediated disorders, hemophilia A, metabolic disorders, rare cholestatic liver diseases and neuromuscular diseases, as well as glioblastoma multiforme (GBM).

CANbridge strategically combines global collaborations and internal research to build and diversify its drug portfolio and invest in next-generation gene therapy technologies for rare disease treatments. CANbridge global partners include, but are not limited to, Apogenix, GC Pharma, Mirum, Wuxi Biologics, Privus, the University of Massachusetts Medical School (UMass) and LogicBio.

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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.


U.S. Investor Relations:
Chris Brinzey

ICR Westwicke

China Investor Relations
CANbridge Pharmaceuticals Inc.

Deanne Eagle

Planet Communications

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