FGF21

89bio Receives EMA PRIME Status for Pegozafermin in the Treatment of Metabolic Dysfunction-Associated Steatohepatitis (MASH) with Fibrosis and Compensated Cirrhosis

Retrieved on: 
Wednesday, March 27, 2024
MASH, Cirrhosis, Patient, SAN, European Medicines Agency, Marketing, FGF21, Liver, Fibrosis, PRIME, EMA, Pharmaceutical industry

SAN FRANCISCO, March 27, 2024 (GLOBE NEWSWIRE) -- 89bio, Inc. (Nasdaq: ETNB), a clinical-stage biopharmaceutical company focused on the development and commercialization of innovative therapies for the treatment of liver and cardiometabolic diseases, today announced that the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) status to pegozafermin in patients with MASH. The PRIME status was supported by positive data from the Phase 2b ENLIVEN trial of pegozafermin in patients with non-cirrhotic MASH with fibrosis (F2-F3) and MASH with compensated cirrhosis (F4).

Key Points: 
  • The PRIME status was supported by positive data from the Phase 2b ENLIVEN trial of pegozafermin in patients with non-cirrhotic MASH with fibrosis (F2-F3) and MASH with compensated cirrhosis (F4).
  • “The EMA PRIME status further supports pegozafermin’s positioning as a leading FGF21 analog for the treatment of MASH, which has demonstrated robust anti-fibrotic and metabolic benefits as seen in our Phase 2b ENLIVEN trial,” said Rohan Palekar, Chief Executive Officer of 89bio.
  • “This status recognizes the urgent need for effective treatment options for MASH patients with advanced fibrosis and underscores pegozafermin’s potential to address the targeted unmet medical need.
  • For more information on the PRIME status, please visit the EMA website at www.ema.europa.eu .

89bio Initiates Phase 3 ENLIGHTEN-Fibrosis Trial of Pegozafermin in Non-Cirrhotic Metabolic Dysfunction-Associated Steatohepatitis (MASH) Patients with Fibrosis

Retrieved on: 
Tuesday, March 12, 2024
Fatty liver disease, FGF21, Cirrhosis, Patient, SAN, Hepatology, University, MASH, Fibrosis, Safety, Division, NASH, Liver, Pharmaceutical industry

SAN FRANCISCO, March 12, 2024 (GLOBE NEWSWIRE) -- 89bio, Inc. (Nasdaq: ETNB), a clinical-stage biopharmaceutical company focused on the development and commercialization of innovative therapies for the treatment of liver and cardiometabolic diseases, today announced the initiation of its Phase 3 ENLIGHTEN Program evaluating the efficacy and safety of pegozafermin in patients with metabolic dysfunction-associated steatohepatitis (MASH), formerly known as nonalcoholic steatohepatitis (NASH). ENLIGHTEN-Fibrosis, the first of two Phase 3 trials in the program, has initiated and is evaluating non-cirrhotic MASH patients with fibrosis stage F2-F3.

Key Points: 
  • ENLIGHTEN-Fibrosis, the first of two Phase 3 trials in the program, has initiated and is evaluating non-cirrhotic MASH patients with fibrosis stage F2-F3.
  • “ENLIGHTEN-Fibrosis aims to enroll MASH patients, including those on background GLP-1-based therapies, where pegozafermin has demonstrated additional anti-fibrotic and metabolic effects.
  • The trial is designed to employ a three-panel consensus biopsy reading methodology, which was successfully utilized in the ENLIVEN trial.
  • Patients will self-administer pegozafermin using the planned commercial liquid formulation delivered as a single subcutaneous injection.

89bio Reports Fourth Quarter and Full Year 2023 Financial Results and Provides Corporate Updates

Retrieved on: 
Thursday, February 29, 2024
Liver, SAN, FGF21, EMA, Research, AASLD, FDA, Research and development, Cirrhosis, Standard of care, Hepatitis, Safety, European Medicines Agency, Administration, Patient, Fibrosis, NASH, Pharmaceutical industry

SAN FRANCISCO, Feb. 29, 2024 (GLOBE NEWSWIRE) -- 89bio, Inc. (Nasdaq: ETNB), a clinical-stage biopharmaceutical company focused on the development and commercialization of innovative therapies for the treatment of liver and cardiometabolic diseases, today reported its financial results for the fourth quarter and full year ended December 31, 2023, and provided corporate updates.

Key Points: 
  • ENLIGHTEN-Fibrosis is expected to enroll non-cirrhotic patients with fibrosis stage F2-F3 and ENLIGHTEN-Cirrhosis is expected to enroll NASH patients with compensated cirrhosis (F4).
  • ENLIGHTEN-Fibrosis and ENLIGHTEN-Cirrhosis are expected to initiate in the first and second quarter of 2024, respectively.
  • Completed follow-on offering in the fourth quarter of 2023 for $172.5 million in gross proceeds.
  • The foregoing financial information is unaudited and subject to change, and actual results may vary from the foregoing.

Kriya Provides Update on Pipeline Progress Ahead of Company Presentation at 42nd Annual J.P. Morgan Healthcare Conference

Retrieved on: 
Monday, January 8, 2024
Continuity, Diabetes, Entrepreneurship, Risk, C3, Non-alcoholic fatty liver disease, Epilepsy, FDA, CR2, Anticonvulsant, Inflammation, Neuron, Geographic atrophy, Conference, Paratriathlon, Pain, European, Disease, Cmax, Glycated hemoglobin, Trigeminal neuralgia, FGF21, Steatosis, Therapy, Computational biology, Exophthalmos, TED, Diplopia, Varenicline, Smoking, Injection, AAV, CNS, DSM-IV codes, Patient, Graves' ophthalmopathy, Medicine, Seizure, Glucokinase, Neurology, CR1, NASH, Insulin, Brain, GMP, Trigeminal nerve, Ophthalmology, Pathology, Fibrosis, Pharmaceutical industry

PALO ALTO, Calif. and RESEARCH TRIANGLE PARK, N.C., Jan. 08, 2024 (GLOBE NEWSWIRE) -- Kriya Therapeutics, Inc., ("Kriya") a biopharmaceutical company developing gene therapies for common diseases affecting millions of people around the world, today provided an update on its pipeline of gene therapies for prevalent conditions including geographic atrophy, thyroid eye disease, diabetes, NASH, trigeminal neuralgia and epilepsy. Kriya is advancing the first of its gene therapy product candidates into the clinic in 2024 and expects up to five programs in the clinic by the end of 2025.

Key Points: 
  • Kriya’s pipeline includes gene therapies across three major therapeutic areas: ophthalmology, metabolic disease and neurology.
  • Its programs share the following features: direct-to-tissue delivery, validated biology, de-risked clinical and regulatory paths and large commercial markets.
  • Dr. Ramaswamy will share further information on Kriya’s portfolio during his presentation at the J.P. Morgan 42nd Annual Healthcare Conference on Tuesday, January 9th at 9:00 AM PT in San Francisco, California.
  • A significant proportion of patients suffer from refractory epilepsy, many of whom are candidates for invasive neurosurgery.

Boston Pharmaceuticals Presents Data at NASH-TAG 2024 Demonstrating Low Immunogenicity Over Time With Long-acting FGF21 Analogue, BOS-580, for MASH

Retrieved on: 
Friday, January 5, 2024
Health, Clinical Trials, Research, Pharmaceutical, Science, Biotechnology, NAFLD, Risk, ADAS, NASH, Division, Hepatology, Non-alcoholic fatty liver disease, ADAPT, CMO, Prevalence, University of California, Medication, Liver injury, University, Fatty liver disease, MASH, Biomarker, FAST, FGF21, Patient, Fibrosis, Therapy, Aspartic acid

The data were presented at NASH-TAG 2024 (Jan. 4-6, 2024) in Park City, Utah.

Key Points: 
  • The data were presented at NASH-TAG 2024 (Jan. 4-6, 2024) in Park City, Utah.
  • “This low immunogenicity distinguishes BOS-580 from other FGF21 analogues currently in development.”
    Another analysis of data from Boston Pharmaceuticals’ Phase 2a trial demonstrated the impact of BOS-580 treatment on several non-invasive indicators of fibrosis based on markers of liver injury and fibrosis in MASH patients.
  • “Our latest data highlight that BOS-580 has great potential to become a backbone in the treatment of patients suffering from MASH.
  • We believe BOS-580 may offer a compelling and convenient treatment option for people living with MASH,” said Juan Carlos Lopez-Talavera, M.D., Ph.D., CMO of Boston Pharmaceuticals.

89bio Announces New Positive Long-Term Data from the ENLIVEN Phase 2b Trial of Pegozafermin in Patients with Nonalcoholic Steatohepatitis (NASH)

Retrieved on: 
Monday, November 27, 2023
Patient, Liver, NASH, Fatty liver disease, SAN, Cirrhosis, QW, Inflammation, Week, FGF21, Fibrosis

SAN FRANCISCO, Nov. 27, 2023 (GLOBE NEWSWIRE) -- 89bio, Inc. (Nasdaq: ETNB), a clinical-stage biopharmaceutical company focused on the development and commercialization of innovative therapies for the treatment of liver and cardiometabolic diseases, today announced positive topline data from the blinded extension phase of its Phase 2b ENLIVEN trial evaluating treatment with pegozafermin in patients with nonalcoholic steatohepatitis (NASH). At week 48, both the 30mg weekly (QW) and 44mg every-two-week (Q2W) dosing schedules of pegozafermin demonstrated statistically significant improvements across key markers of liver health. The benefits observed at week 48 were consistent with the results observed at week 24, indicating sustained benefits over time.

Key Points: 
  • The benefits observed at week 48 were consistent with the results observed at week 24, indicating sustained benefits over time.
  • A subset of patients in the placebo arm of the Main Study (n=19) were re-randomized to receive pegozafermin 30mg weekly (QW) during the Extension Phase.
  • The efficacy endpoints assessed in the Extension Phase included liver fat, non-invasive markers of fibrosis and inflammation, and metabolic markers.
  • Extension Phase Data at Week 48: Liver NITs Results [marker of]

Alnylam Highlights Significant Progress with Platform Innovation and Clinical Pipeline at R&D Day

Retrieved on: 
Wednesday, December 13, 2023
Biotechnology, Health, Genetics, Pharmaceutical, Clinical Trials, CNS, Hypertension, Cerebral amyloid angiopathy, Hepatocellular carcinoma, Gene, ALN, Cardiomyopathy, RNA interference, IND, Tissue, Liver, Sanofi, Amyloidosis, Gene silencing, Overweight, KHK, Patient, FGF21, CAA, Part, Obesity, T2DM, Neoplasm, Investigational New Drug, Therapy, LNP, Development, Shanda, Growth, HCC, Central nervous system, Type, TTR, Immunotherapy, Polyneuropathy, Haemophilia, Medication, Biology, Coagulopathy, Traffic barrier, Adipose tissue, Vaccine, Medical device, Pharmaceutical industry, Video game, Alnylam Pharmaceuticals, Capella, ATTR

We expect our track record of strong pipeline and commercial execution to continue through the end of 2025 and beyond,” said Akshay Vaishnaw, M.D., Ph.D., Chief Innovation Officer of Alnylam.

Key Points: 
  • We expect our track record of strong pipeline and commercial execution to continue through the end of 2025 and beyond,” said Akshay Vaishnaw, M.D., Ph.D., Chief Innovation Officer of Alnylam.
  • In addition, Alnylam will share an update on delivery advances driving ALN-BCAT, which will soon begin clinical testing in patients with hepatocellular carcinoma (HCC).
  • A peak mean TTR reduction of 97% was achieved at Day 29 and a mean TTR reduction of 93% was sustained at Day 180.
  • The Company’s R&D Day event will be held today, Wednesday, Dec. 13, from 8:30 a.m. to 12:45 p.m.

Boston Pharmaceuticals Announces Positive Phase 2a Data Supporting Once-monthly Dosing With Investigational BOS-580 in NASH and Treatment Effects in Diabetic Subgroups at AASLD The Liver Meeting® 2023

Retrieved on: 
Friday, November 10, 2023
Health, Diabetes, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, Hepatitis, Hepatology, NAFLD, Liver disease, Insulin resistance, AASLD, Cirrhosis, Doctor of Pharmacy, Risk, Fatty liver disease, Patient, C-peptide, Metabolism, FGF21, Liver injury, Non-alcoholic fatty liver disease, Liver, Biomarker, Glycated hemoglobin, Medication, NASH, Fibrosis, Pharmaceutical industry, Boston

Results support a once-monthly dosing schedule for BOS-580 and the positive effects of treatment for people living with NASH who are at risk for or have type 2 diabetes.

Key Points: 
  • Results support a once-monthly dosing schedule for BOS-580 and the positive effects of treatment for people living with NASH who are at risk for or have type 2 diabetes.
  • These data are being presented on Saturday, Nov. 11, 2023, at the American Association for the Study of Liver Diseases (AASLD) The Liver Meeting® in Boston.
  • The normal, pre-diabetic and diabetic sub-populations showed very similar reductions (57-66%) in hepatic fat fraction (HFF) after 12 weeks of BOS-580 treatment.
  • “BOS-580 treatment appears promising in maintaining healthy HbA1c levels among patients with phenotypic NASH, irrespective of their concurrent type 2 diabetes status.

Boston Pharmaceuticals To Present New Phase 2a Data From Investigational BOS-580 NASH Program at AASLD The Liver Meeting® 2023

Retrieved on: 
Tuesday, November 7, 2023
Health, Clinical Trials, Research, Pharmaceutical, Science, Biotechnology, Liver injury, American Association for the Study of Liver Diseases, Biomarker, Hepatology, University of California, Medication, Liver, Diabetes, Fibrosis, Hepatitis, Metabolism, FGF21, Liver disease, Disease, NASH, Division, Doctor of Pharmacy, Fatty liver disease, University, AASLD, Pharmaceutical industry, Medical imaging, Patient, Gastroenterology

Boston Pharmaceuticals , a clinical-stage biopharmaceutical company developing differentiated molecules addressing serious liver diseases, today announced it will present new data supporting its long-acting dosing schedule and an analysis of treatment in diabetic sub-groups from the Phase 2a Part A clinical program evaluating BOS-580 for the treatment of non-alcoholic steatohepatitis (NASH), at the American Association for the Study of Liver Diseases (AASLD) The Liver Meeting®, taking place November 10-14, 2023, in Boston.

Key Points: 
  • Boston Pharmaceuticals , a clinical-stage biopharmaceutical company developing differentiated molecules addressing serious liver diseases, today announced it will present new data supporting its long-acting dosing schedule and an analysis of treatment in diabetic sub-groups from the Phase 2a Part A clinical program evaluating BOS-580 for the treatment of non-alcoholic steatohepatitis (NASH), at the American Association for the Study of Liver Diseases (AASLD) The Liver Meeting®, taking place November 10-14, 2023, in Boston.
  • BOS-580 is a long-acting, highly engineered fibroblast growth factor 21 (FGF21) analog administered via a once-monthly subcutaneous injection.
  • BOS-580 is designed to reduce liver fat content, liver inflammation as evidenced by biomarkers of liver injury and fibrosis and improve metabolism as evidenced by the impact on metabolic biomarkers in patients with NASH.
  • We are especially pleased that our presentation on the impact of BOS-580 treatment in subgroups of NASH patients who also have diabetes was designated as a poster of distinction by AASLD, placing it among the foremost presentations at this year’s meeting,” stated Sophie Kornowski, PharmD, CEO of Boston Pharmaceuticals.

Rejuvenate Bio Announces Preclinical Data for Gene Therapy Candidate RJB-0402 for the Treatment of Arrhythmogenic Cardiomyopathy

Retrieved on: 
Monday, October 9, 2023
Genetics, Clinical Trials, Cardiology, Biotechnology, Other Health, Health, Pharmaceutical, Other Science, Science, Disease, Cardiac cycle, HIV disease progression rates, Arrhythmia, Rejuvenation, Research, Trial of the century, B cell, DSM-IV codes, MD, Liver, ACM, IND, Heart, Sudden death, Mutation, FDA, Heart failure, AAV, Gene, ARVC, Genetic disorder, PVC, DSP, SADS, Arrhythmogenic cardiomyopathy, Cardiac muscle, FGF21, Patient, Quality of life, Vaccine, Medical device, Pharmaceutical industry

Rejuvenate Bio , today announced new preclinical efficacy data for RJB-0402, a gene therapy targeting key disease-mediating pathways.

Key Points: 
  • Rejuvenate Bio , today announced new preclinical efficacy data for RJB-0402, a gene therapy targeting key disease-mediating pathways.
  • RJB-0402 demonstrated efficacy in a mouse model of arrhythmogenic cardiomyopathy (ACM), an inherited disease caused by mutations in one of several genes encoding desmosomal proteins.
  • “What makes RJB-0402 especially promising is its superior performance in the ACM nonclinical model, outperforming gene replacement therapies in reducing PVCs.
  • The initial clinical study for RJB-0402 will focus on patients with Desmoplakin-related arrhythmogenic cardiomyopathy (DSP ACM) at high risk of life-threatening ventricular arrhythmias and sudden cardiac death.