Cholestasis

ProQR Announces Year End 2023 Operating and Financial Results

Retrieved on: 
Wednesday, March 13, 2024
Trident, Eli Lilly, Exercise, ProQR, Patient, IP, Therapy, Lilly, RNA editing, Cholestasis, Rett syndrome, Research, Cardiovascular disease, Partnership, Pseudouridine, NTCP, RNA, Functional, Uridine, MECP2, Oligonucleotide, ACTB, LPL, Annual report, Conference, Patent, Biomarker, Mouse, RSRT, Eli Lilly and Company, Liver, ANGPTL3

(Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today reported its financial and operating results for the year ended December 31, 2023, and provided a business update.

Key Points: 
  • (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today reported its financial and operating results for the year ended December 31, 2023, and provided a business update.
  • At December 31, 2023, ProQR held cash and cash equivalents of €118.9 million, compared to €94.8 million at December 31, 2022.
  • The Company experienced a net positive cash flow from operating activities in 2023 primarily due to the receipt of the Lilly up-front payment of $60 million in February 2023.
  • For further financial information for the period ended December 31, 2023, please refer to our 2023 Annual Report on Form 20-F and our Statutory Annual Report which will be available on our website, www.

Mirum Pharmaceuticals’ LIVMARLI (maralixibat oral solution) Receives Positive Reimbursement Recommendation by Canada’s CADTH for Patients with Cholestatic Pruritus in Alagille Syndrome

Retrieved on: 
Tuesday, April 2, 2024
Biotechnology, Pharmaceutical, Health, Clinical Trials, Itch, Medication, Alagille syndrome, Liver disease, Inflammation, Patient, Cholestasis, Bile, Cholestatic pruritus, CADTH, Liver injury, The Hospital, Liver, CDEC, Genetic disorder, PFIC, Pharmaceutical industry

“The positive reimbursement recommendation for LIVMARLI by CADTH is an important step forward for patients suffering from the debilitating effects of cholestatic pruritus related to Alagille syndrome,” said Chris Peetz, chief executive officer at Mirum.

Key Points: 
  • “The positive reimbursement recommendation for LIVMARLI by CADTH is an important step forward for patients suffering from the debilitating effects of cholestatic pruritus related to Alagille syndrome,” said Chris Peetz, chief executive officer at Mirum.
  • These patients suffer greatly from the debilitating and disruptive effects of Alagille syndrome, which often starts in early childhood.”
    “CADTH’s recommendation of reimbursement for LIVMARLI provides a meaningful advancement in the treatment of cholestatic pruritus for patients with Alagille syndrome.
  • LIVMARLI is also approved for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) in the U.S. and Europe, and for Progressive Familial Intrahepatic Cholestasis (PFIC) in the U.S. and in Europe.
  • Cholestasis in ALGS is associated with pruritus which is among the most common indications for liver transplant in ALGS.

Mirum Pharmaceuticals’ LIVMARLI Receives FDA Approval for Treatment of Cholestatic Pruritus in Patients with Progressive Familial Intrahepatic Cholestasis

Retrieved on: 
Wednesday, March 13, 2024
Health, FDA, Infectious Diseases, General Health, Clinical Trials, Pharmaceutical, Biotechnology, Growth, Medication, Alagille syndrome, Disease, Patient, Shanda, Sleep, ALGS, Cholestatic pruritus, Quality of life, Life, Cholestasis, Multimedia, Itch, Collection, Family, Food, Pharmaceutical industry

Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced that the U.S. Food and Drug Administration (FDA) has approved LIVMARLI® (maralixibat) oral solution for the treatment of cholestatic pruritus in patients five years of age and older with progressive familial intrahepatic cholestasis (PFIC).

Key Points: 
  • Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced that the U.S. Food and Drug Administration (FDA) has approved LIVMARLI® (maralixibat) oral solution for the treatment of cholestatic pruritus in patients five years of age and older with progressive familial intrahepatic cholestasis (PFIC).
  • View the full release here: https://www.businesswire.com/news/home/20240313135184/en/
    LIVMARLI® (maralixibat) oral solution is now available for prescribing in the US for cholestatic pruritus in patients with PFIC.
  • “LIVMARLI has the potential to have a transformational impact for patients with cholestatic pruritus associated with PFIC, and importantly, offers an option for those patients with the rarest of subtypes,” said Chris Peetz, chief executive officer at Mirum.
  • We are hopeful that LIVMARLI will have a measurable impact for patients, potentially offering a new normal and relief from the sleepless nights and disruption to their lives caused by cholestatic pruritus.”

NorthSea Therapeutics Initiates Phase 2A Trial of Orziloben (NST-6179) in Intestinal Failure-Associated Liver Disease (IFALD)

Retrieved on: 
Wednesday, February 21, 2024
Biotechnology, Pharmaceutical, Health, Clinical Trials, Liver, Collagen, Myofibroblast, PD, Liver disease, Cirrhosis, Learning, Short bowel syndrome, Conference, Hepatology, Liver failure, Safety, Steatosis, Rigshospitalet, PN, Therapy, Pharmacokinetics, Cholestasis, Fatty liver disease, Patient, ASPEN, Fibrosis, Disease, NASH, Pharmaceutical industry

The trial is a randomized, double-blind, Phase 2a, placebo-controlled study, which will be conducted at multiple sites across North America.

Key Points: 
  • The trial is a randomized, double-blind, Phase 2a, placebo-controlled study, which will be conducted at multiple sites across North America.
  • It is designed to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of Orziloben in adult subjects with IFALD.
  • Commenting on the milestone, Rob de Ree, NST’s CEO, said: "Dosing the first patient in our Phase 2a trial for Orziloben in IFALD is a significant achievement for NorthSea Therapeutics, and is a testament to our commitment to advance innovative treatments for liver diseases.
  • In one pre-clinical model of PN-induced liver injury, Orziloben treatment completely prevented severe cholestasis and the development of fibrosis.

ProQR Highlights New Platform Data from Presentation on Axiomer™ RNA Editing Technology at Deaminet 2024

Retrieved on: 
Friday, January 19, 2024
EON, RNA-Seq, Oligonucleotide, Knowledge, RNA, Science, Circulatory system, Mouse, NHP, NTCP, ACTB, Cholestasis, ANGPTL3, Cardiovascular disease, Plasma, Conference, B4GALT1, LDL, Liver, Eon, ProQR, ADAR, Nested polymerase chain reaction, CNS, Video game

“We were pleased to present new in vivo data for our proprietary Axiomer RNA editing technology platform at the Deaminet 2024 meeting, demonstrating robust editing of ACTB in the liver of NHP, as well as functional protein data with the liver target ANGPTL3 in mice,” said Gerard Platenburg, Chief Scientific Officer of ProQR.

Key Points: 
  • “We were pleased to present new in vivo data for our proprietary Axiomer RNA editing technology platform at the Deaminet 2024 meeting, demonstrating robust editing of ACTB in the liver of NHP, as well as functional protein data with the liver target ANGPTL3 in mice,” said Gerard Platenburg, Chief Scientific Officer of ProQR.
  • “To date, we have generated robust in vitro and in vivo preclinical platform data across a range of liver and CNS targets.
  • Presentation of new data highlighting up to 70% editing efficiency of ACTB in liver of mice and NHPs at multiple timepoints.
  • These new preclinical platform data further highlight the potential of Axiomer EONs in preparation for clinical development and supporting discovery of potential new therapeutic applications.

Human medicines European public assessment report (EPAR): Livmarli, Maralixibat chloride, Date of authorisation: 09/12/2022, Revision: 4, Status: Authorised

Retrieved on: 
Tuesday, January 2, 2024
Medical Subject Headings, Liver, Diarrhea, Marketing, Bile, INN, Chronic pain, ATC, International, Hepatology, Disease, Ageing, Blood, Language, Mouth, Abdominal pain, Medicine, European Medicines Agency, ALGS, Cholestatic pruritus, Cholestasis, Select, Patient, Safety, Anatomy, IBAT, IIA, Medical device

Human medicines European public assessment report (EPAR): Livmarli, Maralixibat chloride, Date of authorisation: 09/12/2022, Revision: 4, Status: Authorised

Key Points: 


Human medicines European public assessment report (EPAR): Livmarli, Maralixibat chloride, Date of authorisation: 09/12/2022, Revision: 4, Status: Authorised

Ipsen confirms U.S. FDA grants priority review for New Drug Application for elafibranor for the treatment of rare cholestatic liver disease, PBC

Retrieved on: 
Thursday, December 7, 2023
Liver, UDCA, Chapter Two, Woman, FDA, MAA, Fatigue, Nausea, Primary biliary cholangitis, Inflammation, ADR, Patent, Liver disease, Diagnosis, AMF, CHMP, Ascending cholangitis, UCD, Business, ACLF, PDUFA, Itch, Risk, New Drug Application, OA, Common, EASL, MASH, Liver failure, Safety, Ammonia, Elafibranor, Therapy, Epidemiology, History, Patient, Cholangiocarcinoma, Food, Health care, EMA, Ursodeoxycholic acid, MHRA, Cirrhosis, Medicine, NTZ, EVP, Cholestasis, NASH, Research, Diarrhea, Bile, NDA, PBC, Abdominal pain, Clinical trial, European Medicines Agency, IPN, PPAR, P10, CCA, Fibrosis, Pharmaceutical industry

An oral, once-daily dual peroxisome activated receptor alpha/delta (PPAR α,δ) agonist, investigational elafibranor could potentially be the first novel second-line treatment for the rare, cholestatic liver disease, PBC, in nearly a decade.

Key Points: 
  • An oral, once-daily dual peroxisome activated receptor alpha/delta (PPAR α,δ) agonist, investigational elafibranor could potentially be the first novel second-line treatment for the rare, cholestatic liver disease, PBC, in nearly a decade.
  • The trial enrolled 161 patients who were randomized 2:1 to receive either elafibranor 80mg once daily or placebo.
  • ELATIVE also investigated the effect of treatment with elafibranor on pruritus (severe itch), a significant symptom burden amongst people living with PBC.
  • Findings from the secondary endpoint using the PBC Worst Itch NRS score, showed a reduction in pruritis for elafibranor, which was not statistically significant.

Mirum Pharmaceuticals Presents New Data at The Liver Meeting®

Retrieved on: 
Monday, November 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.

CymaBay Announces Publication of Two-year Safety and Efficacy Results of Seladelpar in Patients with Primary Biliary Cholangitis

Retrieved on: 
Wednesday, November 1, 2023
McWherter, Liver injury, Patient, University of Texas Southwestern Medical Center, AST, MD, Conditional sentence, Liver, Professor, ALP, PBC, Bilirubin, Vierling, Alkaline phosphatase, Cholestasis, Primary biliary cholangitis, University, Safety, Alimentary Pharmacology & Therapeutics, Therapy, ALT, Diagnosis, Liver disease, Pharmaceutical industry, DA, RJ, Y, PJ

Patients with PBC were eligible to enroll in this long-term extension study if they had successfully completed a prior study of seladelpar.

Key Points: 
  • Patients with PBC were eligible to enroll in this long-term extension study if they had successfully completed a prior study of seladelpar.
  • The objectives were to evaluate the long-term safety, tolerability, and efficacy of seladelpar at 5 mg and 10 mg once daily throughout 2-years.
  • In addition, ALP normalization was achieved in 23% and 42% in patients at one and two years, respectively.
  • In the study long-term treatment with seladelpar resulted in continued improvement in markers of cholestasis and liver injury in the second year of treatment.

Mirum Pharmaceuticals to Present New Data at The Liver Meeting®

Retrieved on: 
Monday, October 30, 2023
Biotechnology, Pharmaceutical, Health, Clinical Trials, Cholestasis, TJP2, Publication, Itch, Hepatology, Medication, Quality of life, American Association for the Study of Liver Diseases, Pregnancy, MYO5B, Cholestatic pruritus, Safety, Patient, Liver cancer, AASLD, Pharmaceutical industry

Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced that data from its LIVMARLI and volixibat programs will be presented at The Liver Meeting®, the American Association for the Study of Liver Diseases (AASLD) annual meeting, taking place November 10-14, 2023, in Boston, Massachusetts.

Key Points: 
  • Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced that data from its LIVMARLI and volixibat programs will be presented at The Liver Meeting®, the American Association for the Study of Liver Diseases (AASLD) annual meeting, taking place November 10-14, 2023, in Boston, Massachusetts.
  • Poster 4602-C: Improvements in pruritus with maralixibat are associated with improved quality of life for patients with progressive familial intrahepatic cholestasis: Data from the MARCH-PFIC trial
    Poster 4604-C: Maralixibat leads to significant improvement in cholestatic pruritus for children with progressive familial intrahepatic cholestasis due to TJP2 or MYO5B deficiency: Data from the MARCH-PFIC trial
    Poster 4605-C: Maralixibat leads to significant improvements in cholestatic pruritus for children with progressive familial intrahepatic cholestasis without a genetic diagnosis: Data from the MARCH-PFIC trial
    Poster 4547-C: Efficacy, safety and tolerability of volixibat in patients with intrahepatic cholestasis of pregnancy: A case series of 4 patients
    Full data will be available within the Publications and Presentations section on Mirum’s website on November 13.