BSEP

Mirum Pharmaceuticals Presents New Data at The Liver Meeting®

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星期一, 十一月 13, 2023
Science, Biotechnology, Research, Pharmaceutical, General Health, Health, Infectious Diseases, Clinical Trials, Sleep, Hepatology, MDR3, Self-harm, Safety, Hepatocellular carcinoma, Bilirubin, Cholestasis, AASLD, Liver, Disease, Risk, Cholestatic pruritus, Hematology, Quality of life (healthcare), MYO5B, PFIC, Patient, Publication, Pregnancy, Nature, TJP2, Ascending cholangitis, GI, Hippeastrum mirum, Liver support system, Volixibat, ICP, IBAT, Progressive familial intrahepatic cholestasis, FIC, Augmented fifth, Growth, Doctor of Philosophy, Quality of life, AND, Itch, Medication, MCID, HIV disease progression rates, SBA, Chloride, Pharmaceutical industry, Nursing, Medical imaging, Dietary supplement, Boston, BSEP

Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced new data from its LIVMARLI and volixibat programs presented at The Liver Meeting®, the American Association for the Study of Liver Diseases (AASLD) annual meeting in Boston, Massachusetts.

Key Points: 
  • Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced new data from its LIVMARLI and volixibat programs presented at The Liver Meeting®, the American Association for the Study of Liver Diseases (AASLD) annual meeting in Boston, Massachusetts.
  • “PFIC is a chronic, debilitating cholestatic liver disease characterized by elevated bile acids and pruritus so severe that patients can be listed for liver transplant even in the absence of disease progression.
  • These data suggest overall improved liver health with maralixibat treatment in patients with PFIC that can be maintained long-term.
  • No clinically meaningful changes in liver enzyme levels or hematology parameters were observed after volixibat treatment.

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

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星期二, 十一月 1, 2022
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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).

Intercept Announces Closing of Transaction with Advanz Pharma to Transfer Rights to Commercialize Ocaliva® for PBC Outside the U.S.

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星期五, 七月 1, 2022
FDA, Constipation, Warfarin, NASH, Patient, LinkedIn, Liver, Colesevelam, Adult, Hepatotoxicity, Abdominal pain, Colestipol, Theophylline, Fatty liver disease, BSEP, INR, GLOBE, CYP1A2, OCA, Population, Prothrombin time, Failure, Alkaline phosphatase, Bilirubin, Primary biliary cholangitis, Rash, Hyperlipidemia, MHRA, Jaundice, Obeticholic acid, UDCA, FXR, Bile, Intercept Pharmaceuticals, EMA, Intercept, Therapy, Autoimmune hepatitis, Ciclosporin, Arthralgia, Dizziness, Fatigue, Absorption, Survival, Nasdaq, Sleep, Ascites, ALP, Cirrhosis, Ursodeoxycholic acid, FLS, Hepatic encephalopathy, Dermatitis, Twitter, Alcoholic liver disease, PBC, Itch, Pharmaceutical industry, Vaccine, Medical imaging, Dietary supplement, Generic drug, Advanz Pharma

As a result of this transaction, Intercepts international business has been divested and its international commercial and medical infrastructure have transitioned to Advanz Pharma.

Key Points: 
  • As a result of this transaction, Intercepts international business has been divested and its international commercial and medical infrastructure have transitioned to Advanz Pharma.
  • Upfront consideration for the transaction is $405 million, subject to working capital, closing costs, France reimbursement liability and other adjustments.
  • OCALIVA, a farnesoid X receptor (FXR) agonist, is indicated for the treatment of adult patients with primary biliary cholangitis (PBC).
  • OCALIVA is contraindicated in PBC patients with decompensated cirrhosis, a prior decompensation event, or with compensated cirrhosis who have evidence of portal hypertension.

LIVMARLI (maralixibat) Presentations Show Improvements in Quality of Life Measures in Alagille Syndrome and PFIC2 at NASPGHAN Annual Meeting 2021

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星期一, 十二月 13, 2021
Biotechnology, Pharmaceutical, Health, Clinical Trials, Institute of Liver and Biliary Sciences, SBA, European Medicines Agency, Quality of life (healthcare), Primary sclerosing cholangitis, Gastrointestinal bleeding, NASDAQ, Fatigue, Week, Breakthrough therapy, Form 10-K, Medication, MCID, Cholestasis, Nausea, Vomiting, Vitamin A, Biliary atresia, Symptoms of COVID-19, VISTAS, Form, FDA, Sleep, Pain, FSV, Stomach, Pregnancy, Quality of life, Liver, Society, Pain scale, BSEP, Vitamin, SEC, IBAT, Research, Form 10-Q, Itch, Food, Cholestatic pruritus, Liver injury, Bone, Spiroplasma mirum, PFIC, Caregiver, Primary biliary cholangitis, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Marketing, Abdomen, Patient, INDIGO, Skin, Diarrhea, Residential care, Dietary supplement, Pharmaceutical industry, Alagille syndrome, LIVMARLI™ (maralixibat) oral solution, Mirum Pharmaceuticals, Inc., LIVMARLI™ (MARALIXIBAT) ORAL SOLUTION, MIRUM PHARMACEUTICALS, INC.

Treatment response to LIVMARLI was defined as a 1 point reduction in caregiver Itch-Reported Outcome (ItchRO) instrument score from baseline to Week 48.

Key Points: 
  • Treatment response to LIVMARLI was defined as a 1 point reduction in caregiver Itch-Reported Outcome (ItchRO) instrument score from baseline to Week 48.
  • These data demonstrate that the significant improvements in pruritus seen with LIVMARLI at Week 48 of the ICONIC study are clinically meaningful and are associated with improvements in patients quality of life.
  • The analysis examined 22 patients with BSEP deficiency, or PFIC2, following treatment with LIVMARLI as part of the INDIGO study.
  • Changes in certain liver tests are common in patients with Alagille syndrome and can worsen during treatment with LIVMARLI.

Mirum Pharmaceuticals and Takeda Enter into Exclusive Licensing Agreement to Develop and Commercialize Maralixibat for Rare Pediatric Liver Diseases in Japan

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星期二, 九月 21, 2021
Health, General Health, Research, Pharmaceutical, Science, Biotechnology, Cholestasis, Food, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Marketing, Liver disease, NDA, Primary sclerosing cholangitis, Biliary atresia, Hematology, Health, European Medicines Agency, TSE, Takeda Pharmaceutical Company, Private Securities Litigation Reform Act, U.S. Securities and Exchange Commission, FDA, Alagille syndrome, Multimedia, Patient, Cholestatic pruritus, Abdominal pain, Feces, Pregnancy, Primary biliary cholangitis, Medication, TAK, Itch, BA, BSEP, Security (finance), COVID-19, Vaccine, PDUFA, Genetics, R, Institute of Liver and Biliary Sciences, Investment, PFIC, Quality of life, Nasdaq, Database, Diarrhea, Pharmaceutical industry, Medical device, Maralixibat, Mirum Pharmaceuticals, Inc., Takeda Pharmaceutical Company Limited, MARALIXIBAT, MIRUM PHARMACEUTICALS, INC., TAKEDA PHARMACEUTICAL COMPANY LIMITED

View the full release here: https://www.businesswire.com/news/home/20210921005502/en/

Key Points: 
  • View the full release here: https://www.businesswire.com/news/home/20210921005502/en/
    Under the terms of the agreement, Takeda will be responsible for regulatory approval and commercialization of maralixibat in Japan.
  • We are excited for Takeda to engage in the development of maralixibat and collaborate in our effort to advance this potentially life-changing therapy.
  • This agreement reinforces Takedas commitment to developing highly differentiated medicines to improve the health and quality of life of patients.
  • Maralixibat is a novel, minimally absorbed, orally administered investigational drug being evaluated in several rare cholestatic liver diseases.