Business mix
- Its lead product candidate is LIVMARLI (maralixibat), an orally administered and minimally absorbed ileal bile acid transporter (IBAT) inhibitor that is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome in the United States.
- The company is also involved in the commercialization of Cholbam, a cholic acid capsule, which is approved as treatment for pediatric and adult patients with bile acid synthesis disorders due to single enzyme defects and for adjunctive treatment of patients.
- In addition, it develops Volixibat, an oral and minimally absorbed agent designed to inhibit IBAT, currently under Phase 2b clinical trial for the treatment of adult patients with cholestatic liver diseases.
- The company has a collaboration agreement with the Shire International GmbH; License Agreement with Pfizer Inc.; Sanofi-Aventis Deutschland GmbH; Novartis Pharma AG; Lonza Ltd.; Enthorin Therapeutics, LLC and Dart Neuroscience LLC.
Current key questions
- Can the company's products, services, or pipeline expand adoption within biotechnology?
- How do reimbursement, regulation, clinical data, patents, and commercial execution affect growth?
- Can margins and cash flow improve while funding research, manufacturing, or go-to-market investment?