Mirum Grants CANbridge Exclusive License to Maralixibat in Greater China for Rare Liver Diseases

Rare Daily Staff

Mirum Pharmaceuticals and CANbridge Pharmaceuticals have entered into a licensing agreement, pursuant to which CANbridge has agreed to develop and commercialize maralixibat in Greater China for the treatment of rare liver diseases.

Maralixibat, an experimental, orally administered medication that is being evaluated in the cholestatic liver diseases Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia (BA). It targets the apical sodium dependent bile acid transporter (ASBT), ultimately resulting in lower levels of bile acid systemically, which could mediate liver damage. If left untreated, these conditions can lead to liver failure and inflict life-altering symptoms including stunted growth and severe, non-stop itching. Under current standard of care, patients often require a liver transplant and are at higher risk of many serious health issues.

Under the terms of the licensing agreement, Mirum granted CANbridge an exclusive right to develop and commercialize maralixibat within the Greater China regions (China, Hong Kong, Macau and Taiwan) for ALGS, PFIC, and BA. In exchange, Mirum will receive an $11 million upfront payment, R&D funding, and up to $109 million for the achievement of future regulatory and commercial maralixibat milestones, with significant double-digit tiered royalties based on product net sales.

In collaboration with Mirum, CANbridge has also agreed to oversee Mirum’s clinical study sites in China, with the goal of accelerating enrollment of the global phase 2b EMBARK study, which was recently initiated for patients with biliary atresia. CANbridge will also have the right to manufacture maralixibat in Greater China under certain conditions.

The U.S. Food and Drug Administration has accepted a New Drug Application for maralixibat for the treatment of cholestatic pruritus in patients with ALGS under priority review. The European Medicines Agency is reviewing maralixibat for the treatment of PFIC2. Mirum has commenced a global Phase 2b maralixibat study (EMBARK) for the treatment of BA.

“Maralixibat has the potential to be a transformative medication for certain cholestatic liver diseases and our goal is to ensure its availability to patients globally,” said Chris Peetz, president and chief executive officer at Mirum. “CANbridge is a leading rare disease company in China and with their track record of commercial success, we believe they will be a strategic partner to accelerate the global launch of maralixibat, if approved.”

CANbridge focuses on the commercialization of treatments for orphan diseases and rare cancers to address unmet medical needs in China. Besides a global partnership with WuXi Biologics to develop and commercialize proprietary therapeutics for the treatment of rare genetic diseases, the company has strategic collaborations with GC Pharma to commercialize an enzyme replacement therapy for Hunter syndrome, a gene therapy collaboration with LogicBio Therapeutics for the treatment of Fabry and Pompe diseases, and a collaborative agreement with the Horae Gene Therapy Center at UMass Medical School for the research and development of gene therapies to treat rare genetic diseases.

“We are looking forward to participating in the global development of maralixibat in BA by supporting the China sites for the global Phase 2b EMBARK study, in collaboration with Mirum, and to working closely with Mirum to bring this treatment to patients and families in Greater China, where the need is great,” said James Xue, founder, chairman and CEO of CANbridge.

James Xue, founder, chairman and CEO of CANbridge