Mirum Pharmaceuticals Receives Notice of Allowance for Methods of Use Patent for Maralixibat in Alagille Syndrome

Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that it has received a Notice of Allowance from the United States Patent and Trademark Office (USPTO) for a patent application No. 16/994,368. The allowed patent application covers methods of treating Alagille syndrome using maralixibat, an investigational treatment being evaluated for patients with Alagille syndrome, a rare and life-threatening cholestatic liver disease. The patent application is expected to issue in 2021 and will provide patent protection in the United States of the claimed methods of use of maralixibat into 2040.

Mirum has submitted a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for maralixibat for the treatment of cholestatic pruritus in patients with Alagille syndrome one year of age and older. In March 2021, the NDA was accepted for priority review by the FDA and issued a PDUFA date of September 29, 2021. Maralixibat is also being evaluated in clinical studies for the treatment of patients with progressive familial intrahepatic cholestasis (PFIC) and biliary atresia, both of which are also cholestatic liver diseases.

A notice of allowance is a written notification issued after the USPTO makes a determination that a patent can be granted from an application. The majority of patent applications that receive a notice of allowance will proceed to issue as a U.S. patent. However, a notice of allowance is not a guarantee of patent issuance.

About Maralixibat

Maralixibat is a novel, minimally absorbed, orally administered investigational drug being evaluated in several rare cholestatic liver diseases. Maralixibat inhibits the apical sodium dependent bile acid transporter (ASBT), resulting in more bile acids being excreted in the feces, leading to lower levels of bile acids systemically, thereby potentially reducing bile acid mediated liver damage and related effects and complications. More than 1,600 individuals have received maralixibat, including more than 120 children who have received maralixibat as an investigational treatment for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC). In the ICONIC Phase 2b ALGS clinical trial, patients taking maralixibat had significant reductions in bile acids and pruritus compared to placebo, as well as reduction in xanthomas and accelerated growth long-term. In a Phase 2 PFIC study, a genetically defined subset of BSEP deficient (PFIC2), patients responded to maralixibat with an increase in transplant-free survival. The U.S. Food and Drug Administration has granted maralixibat Breakthrough Therapy designation for the treatment of pruritus associated with ALGS in patients one year of age and older and for PFIC2. Maralixibat was generally well-tolerated throughout the studies. The most frequent treatment-related adverse events were diarrhea and abdominal pain. Until maralixibat is approved and available for prescribing, the medication is available to patients with ALGS through Mirum’s expanded access program. For more information, please visit ALGSEAP.com. For more information about the Phase 3 study for maralixibat in pediatric patients with PFIC, visit PFICtrial.com.