Mirum Pharmaceuticals Announced Data Presented During AASLD Highlighting Durable Improvements in Pruritus and Quality of Life in Children with Alagille Syndrome Treated with Maralixibat
Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) announced data presented at the Annual Meeting of the American Association for the Study of Liver Diseases (AASLD) - The Liver Meeting Digital Experience™. The data were presented by Dr.
· 11/16/2020 05:50
Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) announced data presented at the Annual Meeting of the American Association for the Study of Liver Diseases (AASLD) - The Liver Meeting Digital Experience™. The data were presented by Dr. Benjamin Shneider of Baylor College of Medicine and Texas Children's Hospital presenting on behalf of the Childhood Liver Disease Research Network (ChiLDReN) in a late-breaking oral presentation titled "Preliminary Analysis of ITCH and IMAGINE II – Outcome of Long-term Administration of Maralixibat in Children with Alagille Syndrome." The objective of the studies was to assess pruritus and other markers of cholestasis in patients with Alagille syndrome (ALGS) with up to 220 weeks of treatment with maralixibat. Maralixibat, an apical sodium bile acid transporter (ASBT) inhibitor, has previously been shown to interrupt the enterohepatic circulation of bile acids, reducing pruritus. Of the children enrolled in the ITCH and IMAGINE II studies, 28 of the 37 patients were on study at 48 weeks with 80% of those experiencing clinically meaningful reductions in pruritus (ItchRO[Obs] reduction ≥1.0 point) which were durable beyond four years (Week 220), with 90% of patients who continued on study experiencing a pruritus response at the end of treatment. The mean reduction in ItchRO(Obs) at week 48 was -1.9 points and deepened to -2.3 points at the end of treatment. Maralixibat treatment improved quality of life and led to improved growth parameters. The long-term data suggest that maralixibat has the potential to be an effective treatment and could serve as an alternative to surgery for ALGS patients, if approved. "Maralixibat has the potential to address the severe pruritus experienced by children with Alagille syndrome, resulting in meaningful improvement in quality of life, meeting a major unmet need for this patient population," said Benjamin L. Shneider, M.D., principal study investigator and member of the Childhood Liver Disease Research Network, funded largely by the National Institute of Diabetes and Digestive and Kidney Diseases. "Alagille syndrome often leads to progressive liver disease frequently complicated by severe pruritus, often disrupting sleep, interfering with school, and impacting the quality of life for children and their families so greatly that surgical intervention via external biliary diversion or liver transplant are required. These data show that with maralixibat, we have the potential to positively impact children's health across multiple clinical measures, without resorting to invasive surgeries which can be associated with significant complications and potential mortality." To view the presentation and the complete data, please visit the AASLD section within the Events page on Mirum's website. "These results from the study confirm the potential for maralixibat to address the consequences of cholestasis in Alagille syndrome, supporting the effects seen in the long term analysis of the pivotal ICONIC study," said Chris Peetz, president and chief executive officer of Mirum. "As we progress toward completion of our rolling NDA submission, maralixibat is being offered to eligible patients with Alagille syndrome through an expanded access program until it is available for prescribing."