CANbridge Announces Marketing Approval of CAN108 (迈芮倍®/LIVMARLI®) in China for the Treatment of Cholestatic Pruritus in Patients with Alagille Syndrome

LIVMARLI First and Only Treatment Approved in Mainland China for Cholestatic Pruritus in ALGS

CANbridge to Hold Investor Call

BEIJING & BURLINGTON, Mass.–(BUSINESS WIRE)–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 diseases and oncology, announced that the Chinese National Medical Products Administration (NMPA) has approved CAN108 (Maralixibat Chloride Oral Solution /迈芮倍^®/LIVMARLI^®) to be marketed for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) 1 year of age and older.

LIVMARLI is approved in the United States for the treatment of cholestatic pruritus in patient with ALGS in patients three months of age and older and is authorized in Europe for patients two months and older. LIVMARLI was approved for cholestatic pruritus last year under the Early and Pilot Implementation Policy in the Boao Lecheng International Medical Tourism Pilot Zone in China. CANbridge has an exclusive license with Mirum Pharmaceuticals, Inc. for the development, commercialization and manufacturing, under certain conditions, of LIVMARLI in Greater China for three rare liver disease indications: Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC) and biliary atresia (BA), as well as other select indications. LIVMARLI is currently being investigated in an ongoing Global Phase 2 study (EMBARK) for biliary atresia.

CANbridge will hold investor calls to discuss the approval. These will be on June 6, at 10:00 AM EDT, for the English-language call. To participate, please go to: https://us02web.zoom.us/webinar/register/WN_ihhf7T8ZRd6hgEqN_hWrdg

The Chinese-language call will take place on June 7, at 10:00 AM Beijing Time. To participate. please go to: https://us02web.zoom.us/webinar/register/WN_F3_gSeZ4R_iO9l5tfUTmZg#/registration

“We are delighted to have received marketing approval for LIVMARLI in mainland China so soon after this novel treatment was approved in the leading Western markets,” said James Xue, Ph.D., founder, chairman and CEO of CANbridge Pharmaceuticals Inc. “The fact that we will be able to offer Chinese rare disease patients the same advanced therapies available in other parts of the world both speaks to our regulatory expertise and our mission to make life-changing therapies accessible to patients worldwide. LIVMARLI is the only approved medication in China to treat cholestatic pruritus associated with ALGS. We look forward to bringing this promising new treatment to thousands of ALGS patients in China and to developing LIVMARLI for other indications. We would also like to thank our partner, Mirum.”

About LIVMARLI

LIVMARLI (CAN108 (maralixibat)) is a minimally 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 (extreme itching). LIVMARLI is the first, and only medication approved in China, the US and EU to treat cholestatic pruritus associated with Alagille syndrome (ALGS).

In addition to ALGS, LIVMARLI is under clinical development by CANbridge for the treatment of other cholestatic liver diseases, including progressive familial intrahepatic cholestasis (PFIC) and biliary atresia (BA), and has been granted Orphan Drug designation by the FDA.

CANbridge acquired the exclusive right to develop, commercialize and manufacture, under certain conditions, LIVMARLI in Greater China from Mirum Pharmaceuticals, Inc. for ALGS, PFIC and BA, and select other indications. LIVMARLI has been approved for marketing by China’s National Medical Products Administration (NMPA) for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) I year of age and older.

About Alagille Syndrome (ALGS)

Alagille syndrome (ALGS) is an autosomal dominant multisystem disorder which can lead to end-stage liver disease and death. Its incidence is between 1/30,000 and 1/50,000 [1]. It has been registered in National Rare Diseases Registry System of China (NRDRS). This disease is characterized by dysplasia of bile ducts and involvement of extrahepatic organs, such as the kidneys and eyes, as well as bones and the cardiovascular system. 100% of patients experience liver involvement [2, 3], which often manifests as chronic cholestasis (slowed or stalled bile flow), usually in the neonatal period or within the first 3 months after birth. In addition to jaundice, skin xanthoma and hepatomegaly, patients will also experience severe pruritus [4], which can lead to skin disfigurement, emotional disorder, sleep deprivation and interruption of school learning, due to scratching in affected children [5]. It seriously affects the growth, development and quality of life of patients [6] and can lead to liver transplantation [7].

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.

IMPORTANT SAFETY INFORMATION, U.S. INDICATION

LIVMARLI can cause side effects, including:

Changes in liver tests. Changes in certain liver tests are common in patients with Alagille syndrome and can worsen during treatment with LIVMARLI. These changes may be a sign of liver injury and can be serious. Your healthcare provider should do blood tests before starting and during treatment to check your liver function. Tell your healthcare provider right away if you get any signs or symptoms of liver problems, including nausea or vomiting, skin or the white part of the eye turns yellow, dark or brown urine, pain on the right side of the stomach (abdomen) or loss of appetite.

Stomach and intestinal (gastrointestinal) problems. LIVMARLI can cause stomach and intestinal problems, including diarrhea, stomach pain, and vomiting during treatment. Tell your healthcare provider right away if you have any of these symptoms more often or more severely than normal for you.

A condition called Fat Soluble Vitamin (FSV) Deficiency caused by low levels of certain vitamins (vitamin A, D, E, and K) stored in body fat. FSV deficiency is common in patients with Alagille syndrome but may worsen during treatment. Your healthcare provider should do blood tests before starting and during treatment.

Other common side effects reported during treatment were gastrointestinal bleeding and bone fractures.

US Prescribing Information

EU SmPC

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.

1. Kamath et al, JPGN 2018; 67: 148-156
2. Turnpenny PD, Ellard S. Eur J Hum Genet, 2012;20:251–57.
3. Saleh M, et al. Appl Clin Genet, 2016;9:75–82.
4. Elisofon SA, et al. J Pediatr Gastroenterol Nutr 2010; 51:759–765.
5. Elisofon et al. JPGN. 2010;51: 759-765.
6. Abetz-Webb et al. Hepatology. 2014, 60(4), 526-527.
7. Kamath BM, et al. Hepatol Comms 2020; 4:387–398.

 

Contacts

U.S. Investor Relations:
Chris Brinzey

ICR Westwicke

Chris.brinzey@westwicke.com

China Investor Relations
CANbridge Pharmaceuticals Inc.

ir@canbridgepharma.com

Media:
Deanne Eagle

Planet Communications

deanne@planetcommunications.nyc
917.837.5866