SUZHOU and BURLINGTON, Mass. – 28 March, 2024— 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 disease and rare oncology, today announced financial results for the twelve months ended 2023 and corporate update.

“2023 was a productive year for CANbridge as we achieved multiple regulatory approvals and commercial milestones for our approved rare disease therapy, Livmarli, across multiple geographies, including mainland China, Hong Kong and Taiwan,” said James Xue, Ph.D., CANbridge founder, chairman, and CEO. “We continue to focus on driving adoption of these programs and achieving broader profitability within our commercial operations. Additionally, we continue to make significant progress advancing our deep rare disease pipeline, including generating positive data from our Phase 1b study of CAN106 in PNH, completing enrollment in the potentially registrational trial for CAN103 in Gaucher disease, and reporting promising preclinical updates for our gene therapy programs targeting Fabry disease and SMA. We look forward to driving further growth and executing on our vision of delivering innovative treatments to underserved patient populations worldwide in 2024 and beyond.”

Upcoming Milestones

• CAN008 – Plan to report data from the Phase 2 clinical trialfor newly diagnosed GBM patients in H1 2024
• CAN103– Plan to submit NDA of CAN103 for Gaucher Disease in H2

Financial Highlights

• Revenue increased by RMB23.9 million or 30.3%, from RMB79.0 million for the year ended December 31, 2022 to RMB102.9 million for the year ended December 31, 2023, which was mainly attributable to the increase of sales from Hunterase® and Livmarli®.
• Research and development (“R&D”) expenses decreased by approximately RMB54.0 million or 17.4%, from RMB311.2 million for the year ended December 31, 2022 to RMB257.2 million for the year ended December 31, 2023, which was primarily attributable to the decrease in upfront and milestone payments made to our licensing partners, the decrease intesting and clinical trial expenses, the decrease in the R&D employee costs and partially offset by the increase of depreciation and amortization costs. 
• Loss for the year decreased by RMB104.7 million or 21.7% from RMB483.5 million for the year ended December 31, 2022 to RMB378.8 million for the year ended December 31, 2023, which was primarily attributable to the increase of our revenue and the decreases of R&D expenses and administrative expenses.
• The adjusted loss for the year decreased by RMB97.4 million, or 21.4%, from RMB456.7 million for the year ended December 31, 2022, to RMB358.9million, for the year ended December 31, 2023. The adjusted loss for the year was arrived at by adjusting the IFRS loss for the year of RMB378.8 million (2022: RMB483.5 million) through excluding the effect of share-based payment expenses.

Recent Highlights

Hunterase^® (idursulfase beta, formerly known as CAN101), an enzyme replacement therapy (ERT) for the treatment of Mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome. MPS II is number 73 in the “First National List of Rare Diseases” in China published in May 2018.

• Launched in May 2021 as the first and only ERT for MPS II in mainland China. The identification of new patients accelerated, with 757 identified as of December 31, 2023.
• Implemented commercial insurance programs (Huiminbao) in 103 cities, covering a population of500 million in China.

• Livmarli^® (CAN108, maralixibat oral solution), an oral, minimally absorbed, reversible inhibitor of the ileal bile acid transporter (IBAT) that is under development to treat rare cholestatic liver diseases including Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC). CANbridge has the exclusive rights to develop, commercialize, and under certain conditions, manufacture Livmarli in Greater China. ALGS is number 5 in the “Second National List of Rare Diseases” in China published in September 2023.

• In 2023, CANbridge received multiple marketing approvals for Livmarli in mainland China, Hong Kong, and Taiwan. The broad marketing approvals make Livmarli the first and only approved product marketed for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) in these regions.

CAN106 (omoprubart), a novel, long-acting monoclonal antibody for the treatment of complement- mediated diseases, including paroxysmal nocturnal hemoglobinuria (PNH), myasthenia gravis (MG) and other diseases that may benefit from treatment with an anti-C5 antibody. PNH is number 88 in the “First National List of Rare Diseases” in China published in May 2018.

• Reported positive preliminary top-line data on June 25, 2023 from the ongoing Phase 1b study of CAN106 being conducted in PNH patients in China. Results suggest complete blockade of complement function at safe and well-tolerated doses. The data also show a dose-dependent reduction of lactate dehydrogenase (LDH) and increased hemoglobin levels, demonstrating clinically meaningful hemolysis inhibition.
• Complement-mediated diseases amenable to treatment with an anti-C5 antibody remain an area of broad interest, demonstrating potential for CAN106 in multiple indications beyond PNH.

CAN008, a glycosylated CD95-Fc fusion protein being developed for the treatment of glioblastoma multiforme (GBM). GBM is number 38 in the “Second National List of Rare Diseases” in China published in September 2023.

• An independent data monitoring committee (IDMC) performed an interim analysis of the ongoing Phase 2 study of CAN008 being conducted in China in patients with newly diagnosed GBM and recommended that the study continue without any changes to the current trial design.
• Data from the Phase 2 study of CAN008 is anticipated in the first half of 2024. Depending on the outcome of this trial, the Company may plan to request accelerated regulatory responsein the Greater China.

CAN103, an ERT for the treatment of Gaucher Disease (GD). GD is number 31 in the “First National List of Rare Diseases” in China published in May 2018.

• CAN103 is the first clinical stage ERT being developed for GD in China.
• On October 16, 2023, CANbridge announced that the core part of the ongoing CAN103 Phase 2 trial in treatment-naïve patients aged 12 and above with Gaucher disease GD Types I and III reached full enrollment. The randomized, double-blind and dose-comparison Phase 2 study is designed to evaluate the efficacy, safety and pharmacokinetics of CAN103 in newly treated GD patients over 9 months, and is followed by a long-term extension period. This trial will serve as a potential registrational trial for CAN103.
• We expect to submitnew drug application (NDA) in the second half of 2024.

Gene Therapy, a CANbridge-developed area of excellence, is a therapeutic modality that includes adeno-associated virus (AAV) as a gene delivery vehicle due to its potential to be a one-time, durable treatment for many genetic diseases. Fabry disease and spinal muscular atrophy (SMA) are number 27 and number 110, respectively, in the “First National List of Rare Diseases” in China published in May 2018.

• Presented preclinical data in May 2023 on CAN203 at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting. Data shared at the ASGCT highlights the potential of this novel, second-generation vectorthat expresses a codon-optimized hSMN1 transgene expressed under the control of an endogenous hSMN1 promoter, to treat SMA. The data demonstrated that 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, which is similar to the U.S. Food and Drug Administration (FDA)-approved gene therapy vector for SMA.
• Presented preclinical data in October 2023 on CAN201, a potential gene therapy for the treatment of patients with Fabry disease, at the European Society of Gene and Cell Therapy (ESGCT) 30th Annual Congress. CAN201 utilizes a novel AAV vector (sL65) that specifically targets the liver to produce the enzyme, α-galactosidase A (α-GAL), that is deficient in patients with Fabry disease. In preclinical studies involving Fabry mice and a PXB mouse model containing a humanized liver, CAN201 showed a dose-dependent increase in α-GAL enzyme levels across various tissues with a corresponding reduction in disease-causing Gb3 lipid levels. The gene therapy was well tolerated with no significant adverse effects observed in Fabry mice.
• In February 2024, our pioneering work, in collaboration with the Horae Gene Therapy Center at the UMass Chan Medical School, on developing a novel AAV-based gene therapy for SMA was published in the prestigious EMBO Molecular Medicine journal, accompanied by a commentary highlighting its scientific significance. Compared to the benchmark vector with an identical design to the vector used in the FDA-approved gene therapy for treating SMAthat drove high, ubiquitous tissue expression of SMN, this second-generation vector restored SMN expression close to physiological levels in the central nervous system and major systemic organs of a severe SMA mouse model. Remarkably, it demonstrated superior safety without liver toxicity seen with the benchmark vector and markedly improved therapeutic efficacy over benchmark vector. Compared to benchmark vector, it prolonged longer survival, more efficiently rescued motor function and neuromuscular junction integrity, more effectively rescued heart and respiratory function and reduced peripheral tissue disease manifestations.  This body of work is the basis of our CAN203 gene therapy program.

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  at the American Society for Gene and Cell Therapy (ASGCT) in 2022 and 2023, the European Society for Gene and Cell Therapy (ESGCT) in 2022 and the World Muscle Congress in 2022. 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.

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.

Contact:

U.S. Investor Relations:

Chris Brinzey

ICR Westwicke

Chris.brinzey@westwicke.com

China Investor Relations

CANbridge Pharmaceuticals Inc.

ir@canbridgepharma.com