Mirum Agrees to Acquire Portfolio of Rare Liver Diseases Therapies from Travere

Rare Daily Staff

Mirum Pharmaceuticals said it agreed to acquire from Travere Therapeutics its bile acid product portfolio that includes Cholbam and Chenodal, two medications addressing rare diseases in high-need settings.

Under the terms of the definitive agreement, Mirum will purchase Travere’s bile acid product portfolio for up to $445 million. Of that amount, $210 million will be paid upfront and $235 million in potential sales-based milestone payments.

Mirum will acquire Travere’s rights to Cholbam, indicated for the treatment of bile acid synthesis disorders due to single enzyme deficiencies and adjunctive treatment of peroxisomal disorders in patients who show signs or symptoms of liver disease, and Chenodal, indicated for the treatment of radiolucent stones in the gallbladder, which is also under phase 3 clinical evaluation for cerebrotendinous xanthomatosis (CTX).

Mirum said it secured $210 million in funding from a syndicate of existing investors led by Frazier Life Sciences and other existing stockholders and new investors that include Avidity Partners, BVF Partners, Longitude Capital, Sofinnova Investments. and a healthcare investment fund to finance the upfront payment for the acquisition.

“The addition of the bile acid replacement therapies from Travere will strengthen our pipeline and offer an opportunity to leverage our unique expertise in the development and commercialization of treatments in rare and underserved liver diseases,” said Chris Peetz, president and chief executive officer at Mirum. “This synergistic acquisition of the bile acid portfolio along with the opportunity to sponsor the genetic testing program will help to reinforce our leadership position in pediatric hepatology.”

The FDA approved Cholbam in March 2015, the first FDA-approved treatment for pediatric and adult patients with bile acid synthesis disorders due to single enzyme defects, and for adjunctive treatment of patients with peroxisome biogenesis disorder-Zellweger spectrum disorder. The effectiveness of Cholbam has been demonstrated in clinical trials for bile acid synthesis disorders and the adjunctive treatment of peroxisomal disorders. An estimated 200 to 300 patients are current candidates for therapy.

Chenodal is a synthetic oral form of chenodeoxycholic acid, a naturally occurring primary bile acid synthesized from cholesterol in the liver. The FDA approved Chenodal for the treatment of people with radiolucent stones in the gallbladder. In 2010, Chenodal was granted orphan drug designation for the treatment of cerebrotendinous xanthomatosis, a rare autosomal recessive lipid storage disease.

While Chenodal is not currently labeled for CTX, it received a medical necessity determination in the United States by the FDA and has been used as the standard of care for more than three decades. Travere is working to obtain FDA approval of Chenodal for the treatment of CTX and initiated a phase 3 clinical trial for this indication in January 2020. The prevalence of CTX is estimated in the literature to be as high as 1 in 70,000 in the overall population.

“The sale of the bile acid portfolio will enable us to further focus our efforts on the ongoing, and successful launch of Filspari for IgA nephropathy, pursuing a potential regulatory path forward for sparsentan in FSGS, and the development of pegtibatinase for the treatment of classical homocystinuria, all of which we believe have the potential to be future treatment standards in their respective indications,” said Eric Dube, president and CEO of Travere Therapeutics. This divestment will also strengthen our financial foundation by meaningfully extending our cash runway and allow us to maximize our growth potential.”