ABCB11

Albireo to Present Late Breaking Bylvay® (odevixibat) Data at AASLD 2022

Retrieved on: 
Tuesday, November 1, 2022
Population, Liver disease, GLOBE, EC, Department, Health care, Alagille syndrome, Food, Safety, Albert Einstein College of Medicine, Ageing, BSEP, Institute of Liver and Biliary Sciences, Liver, MHRA, Adult, Primary biliary cholangitis, Abdominal pain, Nasdaq, American Association for the Study of Liver Diseases, Incidence, Diarrhea, Digital, University Medical Center Groningen, PFIC, Vomiting, FSV, Biliary atresia, Abstract, BOLD, AASLD, Pediatrics, Public, Diagnosis, Vitamin, Data analysis, Augmented fifth, European Commission, Dehydration, Medicine, ABCB11, 2021 Essex County Council election, Itch, Association, American Association, Pharmaceutical industry, Dietary supplement, Medical imaging, Patient, Montefiore

BOSTON, Nov. 01, 2022 (GLOBE NEWSWIRE) -- Albireo Pharma, Inc. (Nasdaq: ALBO), a rare disease company developing novel bile acid modulators to treat pediatric and adult liver diseases, today announced the acceptance of two late breakers, including one late breaker with new data from the Phase 3 ASSERT study, to be presented as an oral presentation at the American Association for the Study of Liver Disease (AASLD) The Liver Meeting® 2022, November 4 – 8, 2022. Positive topline results from ASSERT, a global, double-blind, randomized, placebo-controlled trial which evaluated the safety and efficacy of Bylvay in ALGS patients from birth to early adulthood, were announced in October. A second late breaker was also accepted as an oral presentation with data showing Bylvay restored biliary bile acid secretion in treatment-responsive progressive familial intrahepatic cholestasis (PFIC) patients with bile salt export pump (BSEP) deficiency in the PEDFIC 1 trial. A third oral presentation was previously announced, with a pooled data analysis of the PEDFIC trials showing that a decrease in serum bile acids was strongly associated with native liver survival in PFIC patients treated with Bylvay.

Key Points: 
  • A full list of presentations can be found on The Liver Meeting Digital Experience 2022 website .
  • A potent, once-daily, non-systemic ileal bile acid transport inhibitor, Bylvay has minimal systemic exposure and acts locally in the small intestine.
  • The most common adverse reactions for Bylvay are diarrhea, liver test abnormalities, vomiting, abdominal pain, and fat-soluble vitamin deficiency.
  • Bylvay was well tolerated, with an overall adverse event incidence similar to placebo and a low incidence of drug-related diarrhea (11.4% vs. 5.9% placebo).

Albireo Reports Positive Topline Data from Phase 3 Trial of Bylvay® (odevixibat) in Alagille Syndrome

Retrieved on: 
Tuesday, October 11, 2022
Medicine, IND, EMA, Diarrhea, Abdominal pain, OLE, Program, Alagille syndrome, BOLD, Vomiting, EC, European Commission, Primary sclerosing cholangitis, Primary biliary cholangitis, Population, Biliary atresia, Private Securities Litigation Reform Act, Company, Wales, FDA, FSV, Institute of Liver and Biliary Sciences, AstraZeneca, MHRA, EAP, Annual report, Albireo, Security (finance), Itch, Ageing, EDT, U.S. Securities and Exchange Commission, Authorization, Dehydration, Physician, FOC, Medicines and Healthcare products Regulatory Agency, Webcast, Investor, Event, Patient, ABCB11, BA, Vitamin, Adult, Food, Forward-looking statement, COVID-19, Pharmaceutical industry, Dietary supplement, PFIC

With clinical programs in ALGS and BA, Bylvay has the potential to be approved for three pediatric cholestatic liver diseases.

Key Points: 
  • With clinical programs in ALGS and BA, Bylvay has the potential to be approved for three pediatric cholestatic liver diseases.
  • A potent, once-daily, non-systemic ileal bile acid transport inhibitor, Bylvay has minimal systemic exposure and acts locally in the small intestine.
  • The most common adverse reactions for Bylvay are diarrhea, liver test abnormalities, vomiting, abdominal pain, and fat-soluble vitamin deficiency.
  • As a result of risks and uncertainties that Albireo faces, the results or events indicated by any forward-looking statement may not occur.

Mirum Pharmaceuticals Reports Second Quarter 2021 Financial Results and Provides Business Update

Retrieved on: 
Thursday, August 5, 2021
Biotechnology, Pharmaceutical, Health, Clinical Trials, Investor, Primary sclerosing cholangitis, Webcast, Low-density lipoprotein, Abdominal pain, U.S. Securities and Exchange Commission, Prescription Drug User Fee Act, Cholestasis, Primary biliary cholangitis, Liver disease, Biliary atresia, Cholestatic pruritus, Feces, Security (finance), Private Securities Litigation Reform Act, IND, Review, Vistas, European Medicines Agency, Patient, PFIC, ABCB11, COVID-19, Growth, Database, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Volixibat, Spiroplasma mirum, Diarrhea, Food and Drug Administration, Itch, Alagille syndrome, Adult, Pregnancy, NDA, Liver, Recycling, Therapy, Food, Xanthoma, Pharmaceutical industry, Maralixibat, Volixibat, Mirum Pharmaceuticals, MARALIXIBAT, VOLIXIBAT, MIRUM PHARMACEUTICALS

Mirum Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the development and commercialization of a late-stage pipeline of novel therapies for debilitating liver diseases.

Key Points: 
  • Mirum Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the development and commercialization of a late-stage pipeline of novel therapies for debilitating liver diseases.
  • The NDA has been accepted for priority review by the FDA with a PDUFA action date of September 29, 2021.
  • Mirum is also developing volixibat, also an oral ASBT-inhibitor, in primary sclerosing cholangitis, intrahepatic cholestasis of pregnancy, and primary biliary cholangitis.
  • Mirum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

Mirum Pharmaceuticals Reports First Quarter 2021 Financial Results and Provides Business Update

Retrieved on: 
Thursday, May 6, 2021
Biotechnology, Pharmaceutical, Genetics, Health, Medical specialties, Hepatology, Primary sclerosing cholangitis, Cholestasis, PFIC, Primary biliary cholangitis, Ascending cholangitis, ABCB11, Progressive familial intrahepatic cholestasis, Maralixibat, Volixibat, Mirum Pharmaceuticals, MARALIXIBAT, VOLIXIBAT, MIRUM PHARMACEUTICALS

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.

Key Points: 
  • 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 NDA has been accepted for priority review by the FDA with a PDUFA action date of September 29, 2021.
  • Mirum is also developing volixibat, also an oral ASBT-inhibitor, in primary sclerosing cholangitis, intrahepatic cholestasis of pregnancy, and primary biliary cholangitis.
  • Mirum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.\n'

Mirum Pharmaceuticals Announces New Data Being Presented in Late-Breaker Oral and Poster Presentations at the Annual Meeting of the American Association for the Study of Liver Diseases (AASLD)

Retrieved on: 
Monday, November 2, 2020
Biotechnology, Pharmaceutical, Health, Clinical trials, Medical specialties, Hepatology, Cholestasis, Primary sclerosing cholangitis, Primary biliary cholangitis, PFIC, ABCB11, Intrahepatic cholestasis of pregnancy, Liver disease, Liver, Progressive familial intrahepatic cholestasis, Maralixibat, Volixibat, Mirum Pharmaceuticals

Maralixibat is a novel, minimally absorbed, orally administered investigational drug being evaluated in several rare cholestatic liver diseases.

Key Points: 
  • Maralixibat is a novel, minimally absorbed, orally administered investigational drug being evaluated in several rare cholestatic liver diseases.
  • In a Phase 2 PFIC study , a genetically defined subset of BSEP deficient (PFIC2), patients responded to maralixibat.
  • Mirum Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the development and commercialization of a late-stage pipeline of novel therapies for debilitating liver diseases.
  • The company is also developing volixibat, also an oral ASBT-inhibitor, in primary sclerosing cholangitis and intrahepatic cholestasis of pregnancy.

Mirum Pharmaceuticals Presents Maralixibat Five-Year Transplant-Free Survival Data for Patients With PFIC2 at Digital International Liver Congress 2020

Retrieved on: 
Saturday, August 29, 2020
Biotechnology, Pharmaceutical, Health, Clinical trials, Medical specialties, Hepatology, Clinical medicine, ABCB11, Organ transplantation, Enterohepatic circulation, Progressive familial intrahepatic cholestasis, the Maralixibat Phase 2 INDIGO Study, Maralixibat, Mirum Pharmaceuticals

The five-year analysis showed that patients with PFIC2, also known as bile salt export pump (BSEP) deficiency, who achieved sBA control on long-term maralixibat treatment have a significant improvement in transplant-free survival.

Key Points: 
  • The five-year analysis showed that patients with PFIC2, also known as bile salt export pump (BSEP) deficiency, who achieved sBA control on long-term maralixibat treatment have a significant improvement in transplant-free survival.
  • Data presented today demonstrate that, similar to the findings of NAPPED, patients exhibiting sBA control with pharmacological interruption of the enterohepatic circulation with maralixibat have significantly improved transplant-free survival (p=0.0006).
  • INDIGO included 19 PFIC2 patients with non-truncating BSEP mutations who received maralixibat 280 g/kg once or twice daily.
  • In a Phase 2 PFIC study , a genetically defined subset of BSEP deficient (PFIC2), patients responded to maralixibat.