Liver injury

Protara Therapeutics Announces Alignment with FDA on Registrational Path Forward for IV Choline Chloride in Patients Dependent on Parenteral Nutrition

Retrieved on: 
Friday, April 5, 2024
Choline, Plasma, Liver, Nutritional science, Route of administration, Liver disease, Steatosis, Short bowel syndrome, Metabolism, Patient, Choline chloride, Cancer, ESPEN, Parenteral nutrition, Adolescence, Liver injury, Hepatology, Tubing (recreation), Fibrosis, Guideline, Society, Therapy, Copenhagen University Hospital, Safety, Pharmacokinetics, PN, FDA, ASPEN, Rigshospitalet, Food, Feeding tube, Extractive metallurgy

IV Choline Chloride has the potential to become the first FDA-approved IV formulation of choline for the 40,000 PN patients in the U.S.

Key Points: 
  • IV Choline Chloride has the potential to become the first FDA-approved IV formulation of choline for the 40,000 PN patients in the U.S.
  • The Company plans to advance the development of IV Choline Chloride as a source of choline for adult and adolescent patients on long-term PN.
  • The FDA has granted IV Choline Chloride Orphan Drug Designation for the prevention of choline deficiency in PN patients.
  • “We look forward to advancing the clinical development of IV Choline Chloride, which we believe has the potential to become the first FDA approved IV choline therapy for patients dependent on PN.

Inventiva announces positive results from the Phase II, LEGEND, Proof-of-Concept study combining lanifibranor with empagliflozin in patients with MASH/NASH and T2D

Retrieved on: 
Monday, March 18, 2024
Diagnosis, Glucose, Histology, Adipose tissue, Arm, Glycated hemoglobin, Type 2 diabetes, Disease, Connective tissue, Steatosis, Insulin, Weight gain, Diabetes, CT1, Patient, Inflammation, SGLT2, Adiponectin, Table, MASH, MD, Liver injury, Insulin resistance, LEGEND, Fibrosis, HDL, Fatty liver disease, Empagliflozin, Safety, Euronext Paris, Phase, NASH, Hepatitis, Lipid metabolism, Prediabetes, Liver disease

Patients treated with lanifibranor in combination with empagliflozin maintained a stable weight throughout the 24 weeks study, addressing the moderate, metabolically healthy, weight gain that has been observed in some patients treated with lanifibranor.

Key Points: 
  • Patients treated with lanifibranor in combination with empagliflozin maintained a stable weight throughout the 24 weeks study, addressing the moderate, metabolically healthy, weight gain that has been observed in some patients treated with lanifibranor.
  • The treatment with lanifibranor 800mg/once daily alone or in combination with empagliflozin for 24 weeks was well tolerated, with no safety concerns reported.
  • The trial is double-blind for the placebo arm and lanifibranor (800mg daily) arm, and open-label for the combination of lanifibranor (800mg daily) and empagliflozin (10 mg daily) arm.
  • More details on these results are expected to be presented in upcoming scientific conferences and submitted for publication.

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.

Akero Therapeutics Announces Publication of Phase 2b SYMMETRY Cohort D Study in Clinical Gastroenterology and Hepatology

Retrieved on: 
Thursday, March 7, 2024
SAN, EFX, T2D, Diarrhea, Liver injury, Obesity, ALT, Nausea, ELF, DSM-IV codes, Type 2 diabetes, Safety, MASH, Diabetes, Histology, Therapy, Lipid metabolism, Patient, Fibrosis, NASH, Appetite, Hepatology, Pharmaceutical industry

SOUTH SAN FRANCISCO, Calif., March 07, 2024 (GLOBE NEWSWIRE) -- Akero Therapeutics, Inc. (Nasdaq: AKRO), a clinical-stage company developing transformational treatments for patients with serious metabolic disease, today announced publication of results in Clinical Gastroenterology and Hepatology from an expansion cohort (Cohort D, N=31) of the Phase 2b SYMMETRY study.

Key Points: 
  • SOUTH SAN FRANCISCO, Calif., March 07, 2024 (GLOBE NEWSWIRE) -- Akero Therapeutics, Inc. (Nasdaq: AKRO), a clinical-stage company developing transformational treatments for patients with serious metabolic disease, today announced publication of results in Clinical Gastroenterology and Hepatology from an expansion cohort (Cohort D, N=31) of the Phase 2b SYMMETRY study.
  • Tolerability of EFX on top of GLP-1RA (N=21 patients) was generally comparable to GLP-1RA alone (placebo, N=10).
  • The most frequent adverse events for EFX-treated patients were grade 1 or 2 gastrointestinal events (diarrhea, nausea, and increased appetite).
  • Patients treated with EFX plus GLP-1RA showed a similar mean weight loss from baseline relative to patients treated with GLP-1RA alone.

ACTG CROI Presentations Show That Semaglutide Improves Metabolic-Associated Steatotic Liver Disease Among People Living With HIV

Retrieved on: 
Tuesday, March 5, 2024
Infection, Liver, Bangladesh Technical Education Board, Metabolism, Woman, P.T, Fungal infection, NIAID, Prediabetes, CROI, Liver injury, Inflammation, Quality of life, ACTG, ALT, Weight loss, Waist, Weight, Organ dysfunction, University of Southern California, Conference, Health, Glucagon-like peptide-1 receptor, University, SLIM, ART, Triglyceride, BMI, UM1, Insulin resistance, HIV, Non-alcoholic fatty liver disease, Research, University of North Carolina, Dietary supplement

Yesterday, the SLIM LIVER poster “Effects of Semaglutide on Muscle Structure and Function in the SLIM LIVER study” was presented.

Key Points: 
  • Yesterday, the SLIM LIVER poster “Effects of Semaglutide on Muscle Structure and Function in the SLIM LIVER study” was presented.
  • Together, these presentations demonstrate that semaglutide was highly effective in improving, and in some cases, resolving completely, metabolic dysfunction-associated steatotic liver disease (MASLD, formerly known as non-alcoholic fatty liver disease) among people living with HIV.
  • SLIM LIVER is the first study evaluating semaglutide as a treatment for MASLD among people living with HIV.
  • MASLD is common among people living with HIV and likely acts synergistically with HIV to accelerate liver injury and organ dysfunction.

Akero Therapeutics Reports Fourth Quarter and Full Year 2023 Financial Results and Provides Business Update

Retrieved on: 
Thursday, February 29, 2024
SAN, Lipoprotein, EFX, Research, Liver injury, Marketing, FDA, Biopsy, Cirrhosis, Safety, MASH, Histology, Therapy, DSM-IV codes, Patient, Fibrosis, Pharmaceutical industry

SYNCHRONY Histology is evaluating the safety and efficacy of 28 and 50mg doses of efruxifermin (EFX) in patients with biopsy confirmed pre-cirrhotic MASH (F2-F3).

Key Points: 
  • SYNCHRONY Histology is evaluating the safety and efficacy of 28 and 50mg doses of efruxifermin (EFX) in patients with biopsy confirmed pre-cirrhotic MASH (F2-F3).
  • Additional patients will be enrolled and followed for long-term clinical outcomes to support an application for full marketing approval.
  • In the fourth quarter of 2023, Akero reported results for the week 36 analysis of the Phase 2b SYMMETRY study in patients with cirrhosis due to MASH.
  • Akero's cash, cash equivalents, short-term and long-term marketable securities for the year ended December 31, 2023 were $569.3 million.

Systemic Bio to Present Innovative Human-relevant Datasets from h-VIOS™ Platform at SOT and AACR 2024 Annual Meetings

Retrieved on: 
Thursday, February 8, 2024
Abstract, Society of Toxicology, DMPK, Therapy, 3D Systems, CYP, AACR, SOT, Liver injury, Tissue, Liver, Urea, Research, P450, Annual general meeting

HOUSTON, Feb. 08, 2024 (GLOBE NEWSWIRE) -- Systemic Bio™ , a 3D Systems (NYSE:DDD) company, is excited to announce the acceptance of two abstracts for presentation at the prestigious Society of Toxicology (SOT) 2024 Annual Meeting (Salt Lake City, March 10-14, 2024) and American Association for Cancer Research (AACR) 2024 Annual Meeting (San Diego, April 5-10, 2024).

Key Points: 
  • HOUSTON, Feb. 08, 2024 (GLOBE NEWSWIRE) -- Systemic Bio™ , a 3D Systems (NYSE:DDD) company, is excited to announce the acceptance of two abstracts for presentation at the prestigious Society of Toxicology (SOT) 2024 Annual Meeting (Salt Lake City, March 10-14, 2024) and American Association for Cancer Research (AACR) 2024 Annual Meeting (San Diego, April 5-10, 2024).
  • At the SOT 2024 meeting, Systemic Bio will present its latest research on an advanced liver model that demonstrates several-fold enhanced albumin production, CYP (cytochrome P450) activity, and urea production compared to traditional models.
  • The second abstract, accepted for AACR 2024, showcases a novel model that simultaneously assesses on-target anti-tumor therapeutic efficacy and off-target liver injury of therapeutics across different modalities.
  • To delve deeper into this research, all conference attendees and fellow researchers can visit booth #4452 to engage directly with Systemic Bio’s team and discover the full potential of the h-VIOS platform.

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.

Akero Therapeutics Announces First Patients Dosed in Efruxifermin Phase 3 SYNCHRONY Program

Retrieved on: 
Monday, December 18, 2023
NAFLD, Marketing, Histology, FDA, Injection, Liver injury, EFX, SAN, Cirrhosis, Therapy, Lipid, DSM-IV codes, Patient, Safety, Diabetes, Fibrosis

SOUTH SAN FRANCISCO, Calif., Dec. 18, 2023 (GLOBE NEWSWIRE) -- Akero Therapeutics, Inc. (Nasdaq: AKRO), a clinical-stage company developing transformational treatments for patients with serious metabolic diseases marked by high unmet medical need, today reported patients have received their first doses of efruxifermin (EFX) in the SYNCHRONY Phase 3 program. Two parallel, randomized, placebo-controlled trials have commenced: SYNCHRONY Histology evaluating the efficacy and safety of EFX in patients with biopsy-confirmed pre-cirrhotic nonalcoholic steatohepatitis/metabolic dysfunction-associated steatohepatitis (NASH/MASH), and SYNCHRONY Real-World assessing safety and tolerability of EFX in patients with non-invasively diagnosed NASH/MASH or non-alcoholic fatty liver disease/metabolic dysfunction-associated steatotic liver disease (NAFLD/MASLD).

Key Points: 
  • SOUTH SAN FRANCISCO, Calif., Dec. 18, 2023 (GLOBE NEWSWIRE) -- Akero Therapeutics, Inc. (Nasdaq: AKRO), a clinical-stage company developing transformational treatments for patients with serious metabolic diseases marked by high unmet medical need, today reported patients have received their first doses of efruxifermin (EFX) in the SYNCHRONY Phase 3 program.
  • “We are thrilled to have dosed the first patients in our Phase 3 program and are delighted with the current level of engagement by clinicians and patients,” said Kitty Yale, chief development officer of Akero.
  • After 52 weeks, patients will continue treatment as randomized in SYNCHRONY Histology to be followed for long-term clinical outcomes, such as progression to cirrhosis.
  • SYNCHRONY Real-World is expected to enroll approximately 600 patients with NASH/MASH or NAFLD/MASLD diagnosed by non-invasive tests to receive weekly injections of EFX 50mg or placebo.

Cogent Biosciences Announces Positive Data from Ongoing Phase 2 APEX Trial Evaluating Bezuclastinib in Patients with Advanced Systemic Mastocytosis (AdvSM)

Retrieved on: 
Monday, December 11, 2023
University of Alabama, Patient, Associate, Deficit spending, Division, BID, Efficacy, Part Two, Novel, Poster, APEX, American Society of Hematology, Liver injury, PPR, KIT, Society, Stromal cell, TKI, Clinical trial, Mast cell leukemia, ASH, University, VAF, Part, Biomarker, Webcast, Safety, ASM, PEAK, GIST, MCL, Pharmaceutical industry

WALTHAM, Mass. and BOULDER, Colo., Dec. 11, 2023 (GLOBE NEWSWIRE) -- Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, today reported positive data from Part 1 of the Company’s ongoing Phase 2 APEX clinical trial evaluating bezuclastinib in patients with advanced systemic mastocytosis (AdvSM) at the 65th American Society of Hematology (ASH 2023) Annual Meeting & Exposition taking place December 9-12, 2023 in San Diego, CA.

Key Points: 
  • “Advanced systemic mastocytosis is a serious and life-threatening disease,” said Pankit Vachhani, M.D., Associate Professor of Medicine, Division of Hematology and Oncology, University of Alabama at Birmingham.
  • Patients were enrolled with the following sub-types: seven patients with aggressive systemic mastocytosis (ASM), 23 patients with systemic mastocytosis with associated hematologic neoplasm (SM-AHN), and two patients with mast cell leukemia (MCL).
  • In addition, Cogent plans to present data from the completed SUMMIT Part 1 trial (1a and 1b) in the first quarter of 2024.
  • ET (5:00 a.m. PT) to discuss today’s APEX data and the SUMMIT data in NonAdvSM released on December 9.