Primary sclerosing cholangitis

Mirum Pharmaceuticals Reports Fourth Quarter and Year-End 2023 Financial Results and Provides Business Update

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Mittwoch, Februar 28, 2024
Health, General Health, Research, Pharmaceutical, Science, Biotechnology, International, New Drug Application, FDA, Food, CTX, Growth, Primary sclerosing cholangitis, PDUFA, PFIC, Event, Food and Drug Administration, Primary biliary cholangitis, Webcast, Patient, Medication, NDA, Pharmaceutical industry

Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today reported financial results for the fourth quarter and year-end 2023 and provided a business update.

Key Points: 
  • Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today reported financial results for the fourth quarter and year-end 2023 and provided a business update.
  • 2023 LIVMARLI net product sales totaled $141.8 million, representing approximately 89% growth over 2022 net product sales.
  • As of December 31, 2023, Mirum had cash and cash equivalents of $286.3 million.
  • The foregoing financial information is unaudited and subject to change, and actual results may vary from the foregoing.

LISCure Biosciences Announces FDA Clearance of IND Application to Initiate a Phase 2 Study of LB-P8 for Primary Sclerosing Cholangitis (PSC)

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Donnerstag, Februar 29, 2024
PSC, Inflammation, AASLD, FDA, Physician, Food, Drug development, Part, Primary sclerosing cholangitis, Ascending cholangitis, ODD, Safety, Attention, Hepatology, Biomarker, Inflammatory bowel disease, Patient, Fibrosis, Clinical trial, IBD, IND, LBP, Pharmaceutical industry

LISCure anticipates treating the first patient in the upcoming months and expects topline results for Part 1 in the first half of 2025.

Key Points: 
  • LISCure anticipates treating the first patient in the upcoming months and expects topline results for Part 1 in the first half of 2025.
  • PSC is a rare, chronic, cholestatic liver disease with very high unmet medical needs where there are no approved drugs.
  • LISCure confirmed the safety, tolerability, and key biomarkers of LB-P8 in a Phase 1 study and received Orphan Drug Designation (ODD) for the treatment of PSC from the U.S. FDA.
  • LB-P8 was presented in oral presentations at the American Association for the Study of Liver Diseases (AASLD) every year for the past two years.

Chemomab Awarded New Patents for CM-101, Its First-in Class Monoclonal Antibody in Clinical Development for Fibro-Inflammatory Diseases

Retrieved on: 
Dienstag, Februar 20, 2024
Patent office, Fast Track, Therapy, Fibrosis, Doctor of Philosophy, Orphan drug, PSC, Inflammation, TEL, FDA, Patent, Treatment, Use, Primary sclerosing cholangitis, Composition, Liver transplantation, CMMB, Chemokine, Liver, CCL24, Antibody, Pharmaceutical industry

TEL AVIV, Israel, Feb. 20, 2024 /PRNewswire/ -- Chemomab Therapeutics Ltd. (Nasdaq: CMMB), (Chemomab), a clinical stage biotechnology company developing innovative therapeutics for fibro-inflammatory diseases with high unmet need, today announced that the Patent Offices in Brazil and Israel have granted new patents for CM-101, Chemomab's first-in-class monoclonal antibody that neutralizes CCL24, a novel disease target that has been shown to play a critical role in the processes that drive diseases involving fibrosis and inflammation. CM-101 is currently being assessed in the global Phase 2 SPRING trial for the treatment of primary sclerosing cholangitis (PSC). Patient enrollment in the trial has been completed, with a topline data readout expected midyear 2024.

Key Points: 
  • CM-101 is currently being assessed in the global Phase 2 SPRING trial for the treatment of primary sclerosing cholangitis (PSC).
  • Patient enrollment in the trial has been completed, with a topline data readout expected midyear 2024.
  • In clinical and preclinical studies, this distinctive approach has been shown to inhibit fibrogenesis and interfere with core PSC pathways.
  • and the FDA recently awarded CM-101 Fast Track designation for the treatment of PSC in adults.

Caregivers Cheer First Patients at New Intermountain Primary Children’s Hospital, Miller Family Campus in Lehi, Utah

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Montag, Februar 12, 2024
Intermountain West, MOGAD, Liver, Child, Central nervous system, Ulcerative colitis, Caregiver, Gymnastics, Intermountain Health, Courage, Friends, Basketball, Hospital, Primary sclerosing cholangitis, Hope, Communication, Bile, Arthralgia, University, Life flight, Nursing, Patient, Myasthenia gravis, Optic nerve, Biology, Intermountain, Football, Environment

The Miller Family Campus’ Infusion Center opened for one day before the grand opening to give them the treatments they needed, closer to home.

Key Points: 
  • The Miller Family Campus’ Infusion Center opened for one day before the grand opening to give them the treatments they needed, closer to home.
  • Everyone at the new campus has been so amazing to work with!”
    The new Miller Family Campus is a full-service children’s hospital providing nearly all the same specialty pediatric services that patients receive at the Salt Lake Campus.
  • The Miller Family Campus is part of Intermountain Health’s Primary Promise to build the nation’s model health system for children.
  • For more information about the new Miller Family Campus in Lehi, visit primarychildrens.org/lehi .

Pliant Therapeutics Announces Positive Safety and Exploratory Efficacy Data from the INTEGRIS-PSC Phase 2a Trial of Bexotegrast 320 mg in Patients with Primary Sclerosing Cholangitis and Suspected Liver Fibrosis

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Sonntag, Februar 4, 2024
ALP, AGAF, IBD, Ascending cholangitis, Liver failure, Pharmacokinetics, SAN, Washington State University, Inflammation, Fibrosis, Cirrhosis, Itch, PSC, Ursodeoxycholic acid, MRI, Telephone, Magnetic resonance imaging, Inflammatory bowel disease, Patient, Safety, FDA, EMA, FACP, Disease, IPF, SAE, FRCP, ELF, Week, Therapy, Conference call, Alkaline phosphatase, Interest, UDCA, FACG, University, Webcast, Liver, Primary sclerosing cholangitis, Plasma, Liver disease, Conference, Medical imaging

SOUTH SAN FRANCISCO, Calif., Feb. 04, 2024 (GLOBE NEWSWIRE) -- Pliant Therapeutics, Inc. (Nasdaq: PLRX) today announced 12-week interim data from the 320 mg dose group of INTEGRIS-PSC, a multinational, randomized, double-blind, placebo-controlled Phase 2a clinical trial of bexotegrast in patients with primary sclerosing cholangitis (PSC) and suspected moderate to severe liver fibrosis. The 320 mg group met its primary and secondary endpoints demonstrating that bexotegrast was well tolerated over a 12-week treatment period and its plasma concentrations increased with dose. There was no dose relationship for adverse events. Pruritus and cholangitis occurred less frequently on bexotegrast than on placebo.

Key Points: 
  • The trial’s exploratory efficacy endpoints assessed changes in the liver fibrosis markers, Enhanced Liver Fibrosis (ELF) score and PRO-C3 levels, as well as liver biochemistry and magnetic resonance imaging (MRI) of the liver.
  • In addition, MRI imaging continued to show evidence of improved hepatocyte function and bile flow with bexotegrast at the 320 mg dose relative to placebo.
  • INTEGRIS-PSC is a multinational, randomized, dose-ranging, double-blind, placebo-controlled Phase 2a trial evaluating bexotegrast at once-daily oral doses of 40 mg, 80 mg, 160 mg, 320 mg or placebo for 12 weeks in 121 patients with PSC.
  • The 320 mg group enrolled 27 patients in the active arm and added 9 new patients to the pooled placebo arm.

Chemomab Therapeutics Announces New Publication Reinforcing the Clinical Potential of Its CCL24-Neutralizing Antibody CM-101 in Primary Sclerosing Cholangitis

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Dienstag, Januar 30, 2024
CCL24, Pathology, Role, Neutrophil, Patient, European, Therapy, Primary sclerosing cholangitis, Fibrosis, CMMB, Proteomics, In vitro, Potential, Orphan drug, CCL11, PSC, Myofibroblast, Health, TEL, Cell, Disease, CCL26, Fast Track, FDA, Protein, Doctor of Philosophy, Inflammation, Monocyte, Pharmaceutical industry

TEL AVIV, Israel, Jan. 30, 2024 /PRNewswire/ -- Chemomab Therapeutics Ltd. (Nasdaq: CMMB) (Chemomab), a clinical stage biotechnology company focused on the discovery and development of innovative therapeutics for fibro-inflammatory diseases with high unmet need, today announced publication of proteomic analyses that further demonstrate the unique role of the soluble protein CCL24 in driving pathologies associated with the rare fibrotic liver disease primary sclerosing cholangitis (PSC). The new studies reinforce the extensive existing evidence showing that Chemomab's first-in-class CCL24-neutralizing antibody CM-101 can interrupt these destructive processes. The study, "The Role of CCL24 in Primary Sclerosing Cholangitis: Bridging Patient Serum Proteomics to Preclinical Data"1 has been published in the current online version of the peer-reviewed journal Cells.

Key Points: 
  • The new studies reinforce the extensive existing evidence showing that Chemomab's first-in-class CCL24-neutralizing antibody CM-101 can interrupt these destructive processes.
  • The study, " The Role of CCL24 in Primary Sclerosing Cholangitis: Bridging Patient Serum Proteomics to Preclinical Data "1 has been published in the current online version of the peer-reviewed journal Cells.
  • CM-101 is a first-in-class CCL24-neutralizing monoclonal antibody whose dual anti-inflammatory and anti-fibrotic activity has demonstrated disease modifying potential in nonclinical studies of PSC and other fibro-inflammatory disorders.
  • CM-101 has Orphan Drug designation for PSC in the U.S. and the European Union and was recently awarded Fast Track designation by the FDA for the treatment of PSC in adults.

Mirum Pharmaceuticals Announces Preliminary Unaudited 2023 Net Revenue and Provides Corporate Updates

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Montag, Januar 8, 2024
Biotechnology, Pharmaceutical, Health, Clinical Trials, PFIC, Conference, Food, FDA, PDUFA, Medication, Disease, Disclosure, Growth, Webcast, Food and Drug Administration, Primary sclerosing cholangitis, Pharmaceutical industry

Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today provided its preliminary and unaudited estimates for full-year 2023 revenue and net product sales, corporate updates, and full-year 2024 outlook.

Key Points: 
  • Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today provided its preliminary and unaudited estimates for full-year 2023 revenue and net product sales, corporate updates, and full-year 2024 outlook.
  • “2023 was a transformative year for Mirum as we cemented our position as a leader in rare disease and dramatically advanced our operating and financial scale.
  • Additional information and disclosure would be required for a more complete understanding of the company’s financial position and results of operations as of December 31, 2023.
  • Mirum will present at the 42nd annual J.P. Morgan Healthcare Conference in San Francisco on Wednesday, January 10, 2024 at 10:30 a.m. PT.

GENFIT Updates 2024 Outlook Following Acceptance of Elafibranor Filings in Primary Biliary Cholangitis (PBC)

Retrieved on: 
Freitag, Dezember 8, 2023
Hepatic encephalopathy, Ipsen, AASLD, Cell death, Food, Itch, Priority review, Food and Drug Administration, PSC, IND, ACLF, Marketing, FDA, EMA, MAA, PDUFA, Permeability (electromagnetism), Liver, NDA, Inflammation, Primary sclerosing cholangitis, ALP, Capital market, Hyperammonemia, Patient, NTZ, Fibrosis, PBC, ASK1

Launches in the US and Europe will also make GENFIT eligible for royalty payments.

Key Points: 
  • Launches in the US and Europe will also make GENFIT eligible for royalty payments.
  • Revenues will fund the development of GENFIT’s pipeline, now mainly focused on Acute On-Chronic Liver Failure (ACLF) with 5 differentiated assets.
  • Acceptance of filings in the US and Europe have triggered the first milestone payment for GENFIT.
  • Ipsen also indicated that it is developing elafibranor in Primary Sclerosing Cholangitis (PSC) which could lead to further revenues for GENFIT under the licensing agreement.

Pliant Therapeutics Presentations at The Liver Meeting® 2023 Highlight Bexotegrast, an Inhibitor of αvß6 and αvß1 Integrins, in Primary Sclerosing Cholangitis

Retrieved on: 
Montag, November 13, 2023
Primary biliary cholangitis, Hepatology, SAN, Myofibroblast, Therapy, PSC, Primary sclerosing cholangitis, PBC, Cirrhosis, AASLD, Toronto Centre, ALK5, Patient, Wound healing, Liver disease, Gene expression, TGF, Liver, Non-alcoholic fatty liver disease, Fibrosis, Trial of the century, ELF, Integrin, Safety, Pharmaceutical industry

These posters were presented at The Liver Meeting® 2023 of the American Association for the Study of Liver Diseases (AASLD).

Key Points: 
  • These posters were presented at The Liver Meeting® 2023 of the American Association for the Study of Liver Diseases (AASLD).
  • “Our presentations at the Liver Meeting feature preclinical and clinical work supportive of bexotegrast’s antifibrotic mechanism of action in liver fibrosis,” said Éric Lefebvre, M.D., Chief Medical Officer of Pliant.
  • An αVβ1-selective integrin inhibitor was evaluated on fibrotic and non-fibrotic human liver tissue to assess the effects on cell populations present in fibrotic human liver tissue.
  • The effects of bexotegrast (PLN-74809), a dual inhibitor of integrins αVβ6 and αVβ1, were assessed on human liver tissue to characterize the response of unique cell populations in fibrotic primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC).

Pliant Therapeutics Provides Corporate Update and Reports Third Quarter 2023 Financial Results

Retrieved on: 
Donnerstag, November 9, 2023
ALP, Safety, SAN, Therapy, PSC, Primary sclerosing cholangitis, Data monitoring committee, AASLD, Investment, Biotechnology, Serum, Patient, DSMB, Idiopathic pulmonary fibrosis, Tumor microenvironment, Research, Enrollment, Week, IPF, Pharmacokinetics, Liver, TGF, Alkaline phosphatase, Novartis, Duchenne muscular dystrophy, Biomarker, Cirrhosis, Integrin, ELF, DMD, Fibrosis, Pharmaceutical industry

SOUTH SAN FRANCISCO, Calif., Nov. 09, 2023 (GLOBE NEWSWIRE) -- Pliant Therapeutics, Inc. (Nasdaq: PLRX), a clinical-stage biotechnology company and leader in the discovery and development of novel therapeutics for the treatment of fibrotic diseases, today provided a corporate update and reported third quarter 2023 financial results.

Key Points: 
  • SOUTH SAN FRANCISCO, Calif., Nov. 09, 2023 (GLOBE NEWSWIRE) -- Pliant Therapeutics, Inc. (Nasdaq: PLRX), a clinical-stage biotechnology company and leader in the discovery and development of novel therapeutics for the treatment of fibrotic diseases, today provided a corporate update and reported third quarter 2023 financial results.
  • “The third quarter was highlighted by positive interim data from the INTEGRIS-PSC trial of bexotegrast demonstrating a favorable safety profile and encouraging antifibrotic activity in PSC.
  • Positive safety and efficacy data from INTEGRIS-PSC Phase 2a trial in patients with primary sclerosing cholangitis (PSC).
  • These data were selected for an oral late-breaker presentation at next week’s American Association for the Study of Liver Diseases’ (AASLD) The Liver Meeting® 2023.