CANbridge Announces Encouraging Development in Maralixibat/LIVMARLI in Rare Liver Disease

Rhea-AI Summary

Mirum Pharmaceuticals has announced positive topline results from its Phase 3 MARCH study of LIVMARLI® (maralixibat) for treating progressive familial intrahepatic cholestasis (PFIC). This achievement may enhance treatment options for patients with PFIC, a rare liver disease. CANbridge holds exclusive rights to develop maralixibat in Greater China for PFIC and other liver diseases. The drug is also under clinical evaluation for biliary atresia and has received Breakthrough Therapy Designation from the FDA.

Positive

• Positive topline data from Phase 3 MARCH study of LIVMARLI®.
• Potential to expand treatment options for PFIC patients.
• CANbridge's exclusive rights to maralixibat in Greater China for multiple liver diseases.

Negative

• None.

• Congratulates Mirum on Positive Topline Phase 3 Data in progressive familial intrahepatic cholestasis (PFIC)
• CANbridge holds exclusive Greater China license to maralixibat (CAN108) in  liver diseases, China trial ongoing in biliary atresia (BA)

BEIJING & BURLINGTON, Mass.--(BUSINESS WIRE)-- CANbridge Pharmaceuticals Inc. (HKEX:1228), a global biopharmaceutical company, with a foundation in China, committed to the research, development and commercialization of transformative rare disease and rare oncology therapies, announced that the company’s partner Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) reported positive topline data from its Phase 3 MARCH study evaluating the safety and efficacy of LIVMARLI® (maralixibat) oral solution in patients with progressive familial intrahepatic cholestasis (PFIC). PFIC is included in the 121 Rare Disease List published in 2018 by Chinese authorities. CANbridge holds the exclusive right to develop and commercialize maralixibat (CAN108) in Greater China for three liver diseases: biliary atresia (BA), Alagille syndrome (ALGS) and PFIC. CANbridge is overseeing the Phase 2 EMBARK study of CAN108 in BA in China. CAN108 has been approved to treat Alagille syndrome under the Early and Pilot Implementation Policy in the Boao Lecheng International Medical Tourism Pilot Zone, in China.

“We congratulate Mirum on the trial success and look forward to continuing to work together to bring potential new treatments to rare liver disease patients in Greater China,” said James Xue, Ph.D., CANbridge Founder, Chairman and CEO.

About CAN108 (maralixibat)

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. Maralixibat is the first and, currently, only FDA-approved medication to treat cholestatic pruritus associated with Alagille syndrome (ALGS).

In addition to ALGS, Maralixibat is under 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 Designation by the FDA.

CANbridge acquired the exclusive right to develop and commercialize CAN108 in Greater China from Mirum Pharmaceuticals, Inc. for ALGS, PFIC and BA. CAN108’s New Drug Application (NDA) for ALGS has been accepted and granted priority review by China’s National Medical Products Administration (NMPA).

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 three approved drugs and a pipeline of 11 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’s global partners include Apogenix, GC Pharma, Mirum, Wuxi Biologics, Privus, the University of Washington School of Medicine and the UMass Chan Medical School.

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

Forward-Looking Statements

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Investor Relations:

US

Chris Brinzey

ICR Westwicke

Chris.brinzey@westwicke.com

China

CANbridge Pharmaceuticals Inc.

ir@canbridgepharma.com

Media:

Deanne Eagle

Planet Communications

deanne@planetcommunications.nyc

917.837.5866

Source: CANbridge Pharmaceuticals Inc.

FAQ

What are the results of the Phase 3 MARCH study for MIRM?

Mirum Pharmaceuticals reported positive topline results from its Phase 3 MARCH study of LIVMARLI® (maralixibat), indicating safety and efficacy for treating progressive familial intrahepatic cholestasis (PFIC).

What liver diseases is maralixibat being developed for by MIRM?

Maralixibat is being developed for progressive familial intrahepatic cholestasis (PFIC), biliary atresia (BA), and Alagille syndrome (ALGS).

What is the significance of the Phase 3 data released by Mirum Pharmaceuticals?

The positive Phase 3 data for LIVMARLI® may enhance treatment options for PFIC patients and support further clinical developments.

Which company holds the rights to maralixibat in Greater China?

CANbridge Pharmaceuticals holds exclusive rights to develop and commercialize maralixibat in Greater China for PFIC and other liver diseases.

Has maralixibat received any special designation from regulators?

Yes, maralixibat has received Breakthrough Therapy Designation from the FDA for its potential to treat cholestatic pruritus associated with Alagille syndrome.