CANbridge Pharmaceuticals CAN108 New Drug Application (NDA) for Alagille Syndrome Accepted by China’s National Medical Products Administration

Priority Review Expected to be Granted

BEIJING & CAMBRIDGE, Mass.–(BUSINESS WIRE)–CANbridge Pharmaceuticals Inc., (“CANbridge,” stock code 1228.HK) a leading China-based global biopharmaceutical company committed to the research, development and commercialization of transformative rare disease and rare oncology therapies, announced today that the Chinese National Medical Products Administration (NMPA) has accepted the New Drug Application (NDA) for CAN108 (maralixibat oral solution (LIVMARLI ^TM)) for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) 1 year of age and older. CANbridge anticipates that the application will be granted priority review, upon the close of the publicity period of the priority review process, on January 17th. LIVMARLI ^TM (maralixibat) oral solution was recently approved 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.

CANbridge and Mirum Pharmaceuticals signed an exclusive license agreement for the development and commercialization of maralixibat (CAN108) in Greater China last year. Under the terms of the agreement, CANbridge has the right to develop and commercialize CAN108 for three indications: Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC) and biliary atresia (BA) in Greater China.

“That the NMPA has accepted the NDA, which we anticipate will also receive priority review, for CAN108 for Alagille syndrome, our first candidate in rare liver disease, only 3 months after Maralixibat received FDA market approval, showcases our regulatory expertise and highlights the expanding list of indications we target,” said James Xue, Ph.D., CANbridge Founder, Chairman and CEO. “We look forward to developing this treatment in China, where it could bring much-needed relief to patients and their families.”

About Alagille syndrome (ALGS）

Alagille syndrome (ALGS) is a rare autosomal dominant disorder that affects multiple organs, including the liver, heart, bones and eyes. Cholestatic liver disease is the most common form of it and pruritus (severe itching) is the most severe clinical manifestation, in addition to jaundice, xanthomas and hepatomegaly, which usually occur in infancy¹. Pruritus may lead to skin mutilation, emotional disorder, interruption of sleep and school learning activities², seriously affecting children’s growth, development and quality of life³. Severe cholestatic pruritus is also an indication for liver transplantation⁴. According to Frost & Sullivan, there were 68,000 ALGS patients in the world, and 7400 patients in China in 2020.

About Maralixibat

LIVMARLI™ (maralixibat) is a barely absorbed ileal bile acid transporter (IBAT) inhibitor that blocks the enterohepatic circulation of bile acids, reduces bile acid levels in the liver and serum, reduces the resultant liver injury and relieves pruritus. LIVMARLI ^TM is the first and, currently, only FDA-approved medication to treat cholestatic pruritus associated with Alagille syndrome.

Maralixibat is an oral drug that, in addition to ALGS, is under advanced clinical development for the treatment of other cholestatic liver diseases including progressive familial intrahepatic cholestasis (PFIC) and biliary atresia (BA) and has been granted Breakthrough Therapy and Innovative Drugs for Rare Diseases designations by the FDA. Mirum Pharmaceuticals Inc. is conducting a Phase 3 clinical trial of maralixibat in PFIC in North America, Europe, Asia, and South America. A global maralixibat phase 2b clinical trial (EMBARK) in BA is also underway, for which CANbridge is managing the China sites.

About CANbridge Pharmaceuticals Inc.

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

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 including Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC) and biliary atresia (BA) 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), the University of Washington School of Medicine, LogicBio and Scriptr.

For more on CANbridge Pharmaceuticals Inc., please go to: www.canbridgepharma.com.

¹ Binita M. Kamath et al. JPGN. 2018;67: 148-156

² Elisofon et al. JPGN. 2010;51: 759-765

³ Abetz-Webb et al. Hepatology. 2014, 60(4), 526-527

⁴Guidelines for the Management of Cholestatic Liver Diseases (2021)

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CANbridge Pharmaceuticals Inc.

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Deanne Eagle

Planet Communications

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