Alanine transaminase

Vir Biotechnology Completes Enrollment of Phase 2 Chronic Hepatitis Delta SOLSTICE Trial Ahead of Schedule

Retrieved on: 
Tuesday, March 5, 2024

Vir Biotechnology, Inc. (Nasdaq: VIR) today announced that its Phase 2 SOLSTICE clinical trial evaluating the safety, tolerability and efficacy of tobevibart and elebsiran for the treatment of people living with chronic hepatitis delta (CHD) virus has completed enrollment of its current cohorts one month earlier than anticipated.

Key Points: 
  • Vir Biotechnology, Inc. (Nasdaq: VIR) today announced that its Phase 2 SOLSTICE clinical trial evaluating the safety, tolerability and efficacy of tobevibart and elebsiran for the treatment of people living with chronic hepatitis delta (CHD) virus has completed enrollment of its current cohorts one month earlier than anticipated.
  • Initial data are expected in the second quarter of 2024.
  • SOLSTICE is a Phase 2 multi-center, open-label trial designed to evaluate the safety, tolerability, and efficacy of tobevibart and elebsiran in adult participants (age 18 to 69) with CHD infection receiving nucleot(s)ide reverse transcriptase inhibitor therapy.
  • Depending on the cohort, trial participants are receiving multiple doses of tobevibart and elebsiran as either monotherapy or in combination administered via subcutaneous injection for up to 88 weeks.

Xencor Reports Fourth Quarter and Full Year 2023 Financial Results

Retrieved on: 
Tuesday, February 27, 2024

Both programs entered Phase 1 clinical development during the fourth quarter of 2023, and Xencor received $20 million in development milestones.

Key Points: 
  • Both programs entered Phase 1 clinical development during the fourth quarter of 2023, and Xencor received $20 million in development milestones.
  • In the fourth quarter of 2023, Xencor completed enrollment in subcutaneous dose-escalation cohorts of a Phase 1 study evaluating plamotamab.
  • Financial Results for the Fourth Quarter and Full Year Ended December 31, 2023
    Cash, cash equivalents, and marketable debt securities totaled $697.4 million as of December 31, 2023, compared to $584.5 million on December 31, 2022.
  • Total revenue for the fourth quarter ended December 31, 2023 was $44.7 million compared to $21.6 million for the same period in 2022.

Foundation Fighting Blindness Launches GYROS, a Natural History Study for People with Gyrate Atrophy

Retrieved on: 
Tuesday, February 6, 2024

COLUMBIA, Md. , Feb. 6, 2024 /PRNewswire/ -- `The Foundation Fighting Blindness, the world's leading organization committed to finding treatments and cures for inherited retinal diseases and dry age-related macular degeneration, is launching a natural history study for people with gyrate atrophy, a rare, vision-robbing inherited retinal disease. The lead investigators for the study are Mandeep S. Singh, MD, PhD, the Andreas C. Dracopoulos Professor of Ophthalmology and Associate Professor of Ophthalmology and Genetic Medicine at the Wilmer Eye Institute and David Valle, MD, Professor of Genetic Medicine and former director of the McKusick-Nathans Department of Genetic Medicine at Johns Hopkins University.

Key Points: 
  • GYROS results will help researchers design clinical trials for an emerging gyrate atrophy gene therapy.
  • , Feb. 6, 2024 /PRNewswire/ -- `The Foundation Fighting Blindness , the world's leading organization committed to finding treatments and cures for inherited retinal diseases and dry age-related macular degeneration, is launching a natural history study for people with gyrate atrophy, a rare, vision-robbing inherited retinal disease.
  • Known as the Gyrate Atrophy Ocular and Systemic Study (GYROS), the $3.5 million study is informing the design of future clinical trials for gyrate atrophy.
  • Gyrate atrophy is a progressive retinal disease that causes night blindness and loss of peripheral vision, in addition to severe childhood-onset myopia.

Intercept Announces New Phase 2 Data Showing Significant Impact of OCA-Bezafibrate Combination on Normalization of Multiple Key Biomarkers of PBC-Induced Liver Damage at AASLD The Liver Meeting® 2023

Retrieved on: 
Monday, November 13, 2023

These data will be presented on Monday, November 13, 2023, at the American Association for the Study of Liver Diseases’ (AASLD) The Liver Meeting® in Boston (poster #5019-C).

Key Points: 
  • These data will be presented on Monday, November 13, 2023, at the American Association for the Study of Liver Diseases’ (AASLD) The Liver Meeting® in Boston (poster #5019-C).
  • One severe TEAE (pruritus, OCA5/B100 IR) occurred in Study 214; no TEAEs led to study discontinuation in Study 214.
  • The Company expects to have the necessary data from the OCA-bezafibrate combination program to submit a request in 2023 for an End-of-Phase 2 meeting with the FDA.
  • These data include analyses from both Phase 2 studies, in addition to Phase 1 and preclinical data.

CSL Behring's HEMGENIX® (etranacogene dezaparvovec-drlb) Demonstrates at Three Years Post-Treatment Long-Term Durability, Safety and Greater Bleed Protection Versus Prophylactic Treatment in People Living with Hemophilia B

Retrieved on: 
Tuesday, December 12, 2023

The Phase III, open label, single-dose, single arm HOPE-B trial included 54 male participants with severe or moderately severe hemophilia B with or without pre-existing AAV5 neutralizing antibodies.

Key Points: 
  • The Phase III, open label, single-dose, single arm HOPE-B trial included 54 male participants with severe or moderately severe hemophilia B with or without pre-existing AAV5 neutralizing antibodies.
  • HEMGENIX produced mean factor IX levels of 41.5 IU/dL at year one, 36.7 IU/dL at year two and 38.6 IU/dL at year three post-treatment.
  • Additionally, an analysis on the indirect treatment comparison of HEMGENIX and factor IX prophylaxis was recently published in Haemophilia.
  • Earlier this year, CSL announced the first person living with hemophilia B had received HEMGENIX.

FDA Approves Veklury® (Remdesivir) to Treat COVID-19 in People With Mild to Severe Hepatic Impairment With no Dose Adjustment

Retrieved on: 
Thursday, August 24, 2023

The clinical benefit of Veklury in hospitalized populations with COVID-19 is supported by randomized controlled trials, real-world evidence and meta-analyses, but the pharmacokinetics of Veklury had not previously been evaluated in patients with hepatic impairment.

Key Points: 
  • The clinical benefit of Veklury in hospitalized populations with COVID-19 is supported by randomized controlled trials, real-world evidence and meta-analyses, but the pharmacokinetics of Veklury had not previously been evaluated in patients with hepatic impairment.
  • This latest approval is based on results from a Phase 1 study of safety and pharmacokinetics in people with hepatic impairment (GS-US-540-9014).
  • Based upon these results, the label has been updated to reflect that no dose adjustment is required across all stages of liver disease.
  • Furthermore, people with liver disease represent a population that is vulnerable to COVID-19 and are at increased risk of morbidity and mortality.

Intercept Presents New Data Showing Significant Impact of OCA-bezafibrate Combination on Normalization of Multiple Biomarkers of PBC-Induced Liver Damage

Retrieved on: 
Friday, June 23, 2023

MORRISTOWN, N.J., June 23, 2023 (GLOBE NEWSWIRE) -- Intercept Pharmaceuticals, Inc. (Nasdaq: ICPT), a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat rare and serious liver diseases, today announced new results from a planned interim analysis of its ongoing Phase 2 study 747-213 assessing improvements in serum biomarkers of hepatic function, cholestasis and inflammation in patients with primary biliary cholangitis (PBC) after treatment with an investigational combination of obeticholic acid (OCA) and bezafibrate. Results from the full interim data set, shared in a podium presentation today at the European Association for the Study of the Liver (EASL) Congress 2023 in Vienna, Austria, showed that the combination of OCA 5-10 mg and bezafibrate 400 mg was effective in normalizing multiple biochemical markers associated with PBC-induced liver damage.

Key Points: 
  • “We have an opportunity to build on the improved transplant-free survival seen in patients with PBC taking OCA across multiple real-world studies.
  • The lower target for total bilirubin was selected as it is associated with the lowest risk for liver transplant or death.
  • Intercept expects to complete planned interim analyses from both ongoing Phase 2 studies this year.
  • Analyses from these Phase 2 studies, in addition to Phase 1 and preclinical data, will serve as the basis of an end-of-phase 2 meeting with FDA.

Boston Pharmaceuticals Announces Positive Phase 2a Results from Monthly and Bi-weekly Dosing with Investigational BOS-580 NASH Program at EASL 2023

Retrieved on: 
Wednesday, June 21, 2023

Boston Pharmaceuticals today announced positive Phase 2a results for BOS-580, its investigational, proprietary, long-acting fibroblast growth factor 21 (FGF21) analog for the treatment of non-alcoholic steatohepatitis (NASH).

Key Points: 
  • Boston Pharmaceuticals today announced positive Phase 2a results for BOS-580, its investigational, proprietary, long-acting fibroblast growth factor 21 (FGF21) analog for the treatment of non-alcoholic steatohepatitis (NASH).
  • The study suggested promising results for monthly and bi-weekly doses of BOS-580, which achieved statistically significant reduction in liver fat content, an exploratory endpoint in the trial of phenotypic NASH patients.
  • Statistically significant reductions in additional exploratory endpoints, including biomarkers of liver injury and fibrosis, were also observed.
  • The most common adverse events in this Phase 2a trial were gastrointestinal, and included nausea, vomiting, and diarrhea.

Intercept Announces New Data Showing Potential of Fixed-Dose Combination of OCA and Bezafibrate to Normalize Multiple Biomarkers in PBC to be Featured in Podium Presentation at EASL Congress 2023

Retrieved on: 
Wednesday, June 7, 2023

MORRISTOWN, N.J., June 07, 2023 (GLOBE NEWSWIRE) -- Intercept Pharmaceuticals, Inc. (Nasdaq: ICPT), a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat progressive non-viral liver diseases, today announced initial results from a planned interim analysis of its ongoing Phase 2 study 747-213, an active comparator trial demonstrating the therapeutic potential of the combination of obeticholic acid (OCA) and bezafibrate vs. bezafibrate monotherapy on serum biomarkers in primary biliary cholangitis (PBC). These data are part of a larger analysis that will be presented at 08:45 CEST on Friday, June 23 at the European Association for the Study of the Liver (EASL) Congress 2023 in Vienna, Austria.

Key Points: 
  • Safety was assessed by monitoring of adverse events (AEs) and laboratory values.
  • “Multiple real-world studies have demonstrated improved transplant-free survival of OCALIVA (obeticholic acid) following improvements in a specific combination of biomarkers.
  • We are encouraged by the best-in-class potential of our novel combination to build on this evidence with rapid biochemical responses that have predicted improved clinical outcomes.
  • “Results from a Planned Interim Analysis of a Randomized, Double-Blind, Active-Controlled Trial Evaluating the Effects of Obeticholic Acid and Bezafibrate on Serum Biomarkers in Primary Biliary Cholangitis” Abstract #2495

Foghorn Therapeutics Announces FDA Has Lifted Clinical Hold on Phase 1 Study of FHD-286 in Relapsed and/or Refractory AML/MDS Patients

Retrieved on: 
Monday, June 5, 2023

CAMBRIDGE, Mass., June 05, 2023 (GLOBE NEWSWIRE) -- Foghorn® Therapeutics Inc. (Nasdaq: FHTX), a clinical-stage biotechnology company pioneering a new class of medicines that treat serious disease by correcting abnormal gene expression, today announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold on the Phase 1 monotherapy dose escalation study of FHD-286 in acute myelogenous leukemia (AML) and myelodysplastic syndrome (MDS). Foghorn plans to commence a Phase 1 study of FHD-286 in combination with decitabine or cytarabine in relapsed and/or refractory AML patients in the third quarter of 2023.“With a focus on patient safety, we have worked with the FDA to resolve the clinical hold on FHD-286 in AML and MDS,” said Adrian Gottschalk, President and Chief Executive Officer of Foghorn. “Clinical data suggest FHD-286 is a potent, broad-based differentiation therapeutic, and we believe it has significant combination potential as a treatment in AML. We anticipate commencing a Phase 1 combination study focusing on first-line relapsed and/or refractory AML patients in the third quarter of 2023.”

Key Points: 
  • Foghorn plans to commence a Phase 1 study of FHD-286 in combination with decitabine or cytarabine in relapsed and/or refractory AML patients in the third quarter of 2023.
  • Foghorn has amended the protocol and plans to commence a Phase 1 study of FHD-286 in combination with decitabine or low-dose cytarabine (LDAC) in relapsed and/or refractory AML patients.
  • Foghorn plans to commence the Phase 1 combination study of FHD-286 in relapsed and/or refractory AML patients in the third quarter of 2023.
  • The study will enroll relapsed and/or refractory AML patients and the protocol allows for first-line relapsed and/or refractory AML patients.