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Mirum Pharmaceuticals Presents Data Demonstrating Long-term Durability of Treatment Effect of Maralixibat in Children With Cholestatic Liver Diseases
Statistically and clinically significant effects on pruritus, xanthomas and growth maintained for up to four years of treatment in Alagille syndrome Durable
About this update from Mirum Pharmaceuticals, Inc.
[{"type":"text","content":"\nStatistically and clinically significant effects on pruritus, xanthomas and growth maintained for up to four years of treatment in Alagille syndrome \n\n\nDurable multi-parameter treatment response and correlation of response with increased bile acid synthesis in progressive familial intrahepatic cholestasis \n\n FOSTER CITY, Calif.--(BUSINESS WIRE)--\nMirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver diseases, today announced that results from the long-term extension of the Phase 2b ICONIC study demonstrate the durability of treatment effect and disease-modifying potential of maralixibat in children with Alagille syndrome (ALGS). The data will be presented on Monday in a late breaking oral presentation at the annual meeting of the American Association of the Study of Liver Diseases (the Liver Meeting®) in Boston. The company will also present data from the long-term extension of the Phase 2 INDIGO study of maralixibat in children with progressive familial intrahepatic cholestasis (PFIC) demonstrating the durability of multi-parameter treatment response in children with PFIC2, as well as correlation of treatment response to non-truncating bile salt export pump mutations and increased bile acid synthesis as a result of ASBT inhibition.\n\n\n“The new analyses of years of treatment with maralixibat supports the disease-modifying potential of the medicine in children with Alagille syndrome and PFIC2,” said Chris Peetz, president and CEO of Mirum. “Our commitment at Mirum is to work tirelessly to bring maralixibat as quickly as possible to the families who are seeking to reclaim their lives from severe pruritus, sleepless nights, xanthomas and the suppressed growth which their children experience as a result of these diseases.”\n\n\nLong-Term Extension of ICONIC Study in ALGS\n\n\nAt the conclusion of the 48-week treatment period of the placebo-controlled Phase 2b ICONIC study of maralixibat in children with ALGS, 23 participants entered into the long-term extension. At the time of this new analysis, 15 remained on study with a duration of up to 4 years. Consistent with results reported after 48 weeks of treatment with maralixibat, reductions in serum bile acids (sBA) and pruritus (itching), were statistically significan...