Hepatology

HistoIndex's AI-based Digital Pathology Platform: A Validated Quantification Tool Recommended in the Guidelines for the Prevention and Treatment of Chronic Hepatitis B in China

Retrieved on: 
Sunday, May 24, 2020
Medical specialties, Medicine, RTT, Clinical medicine, Infectious causes of cancer, Hepatology, Hepatitis, Cirrhosis, Hepatitis B in China, Jade Ribbon Campaign

Hepatitis B has been known as a complex disease to treat, due to the challenge in distinguishing patients with an active infection.

Key Points: 
  • Hepatitis B has been known as a complex disease to treat, due to the challenge in distinguishing patients with an active infection.
  • These patients require intervention as opposed to patients with an inactive infection who require only standard monitoring without treatment.
  • Image Credits: Quantitative assessment of liver fibrosis (qFibrosis) reveals precise outcomes in Ishak "stable" patients on anti-HBV therapy , Scientific Reports [ 2 ].
  • Guidelines for the Prevention and Treatment of Chronic Hepatitis B (2019 Edition) [J].

Agios Announces Publication of TIBSOVO® Phase 3 Data in The Lancet Oncology Demonstrating Significant Improvement in Progression-Free Survival Compared to Placebo in Previously Treated IDH1-Mutant Cholangiocarcinoma Patients

Retrieved on: 
Tuesday, May 19, 2020
Health, Clinical research, Medical statistics, Medicine, Clinical trials, Articles, Hepatology, Design of experiments, Clinical endpoint, Progression-free survival, Cholangiocarcinoma, Blinded experiment

The study met its primary endpoint, demonstrating a statistically significant improvement in progression-free survival (PFS) in patients randomized to TIBSOVO compared with placebo patients.

Key Points: 
  • The study met its primary endpoint, demonstrating a statistically significant improvement in progression-free survival (PFS) in patients randomized to TIBSOVO compared with placebo patients.
  • Thirty-five patients randomized to placebo (57.4%) crossed over to open-label TIBSOVO upon radiographic disease progression and unblinding.
  • TIBSOVOis not approved in any country for the treatment of patients with previously treated advanced IDH1-mutant cholangiocarcinoma.
  • Patients treated with TIBSOVO have experienced symptoms of differentiation syndrome, which can be fatal if not treated.

Can-Fite Reports Additional Findings from Successful Phase II NASH Study: 25 mg Dose of Namodenoson Significantly Reduced Liver Fat and Fibrosis

Retrieved on: 
Monday, May 18, 2020
Oncology, Health, Infectious diseases, Clinical trials, Pharmaceutical, Biotechnology, Medical specialties, Medicine, Hepatitis, Hepatology, RTT, Non-alcoholic fatty liver disease, Cirrhosis, Liver, Fibrosis, Hepatotoxicity, Obeticholic acid, Fatty liver disease

In the Phase II study, 25 mg of Namodenoson was shown to reduce hepatic fibrosis (scar tissue in the liver resulting from the liver trying to repair itself), reduce steatosis (fat buildup in the liver), and improve the FAST score, a measure for NASH (liver stiffness and an enzymatic biomarker of liver damage).

Key Points: 
  • In the Phase II study, 25 mg of Namodenoson was shown to reduce hepatic fibrosis (scar tissue in the liver resulting from the liver trying to repair itself), reduce steatosis (fat buildup in the liver), and improve the FAST score, a measure for NASH (liver stiffness and an enzymatic biomarker of liver damage).
  • Patients treated with 25 mg of Namodenoson had a statistically significant reduction in hepatic fibrosis as measured by the Fibrosis-4 (FIB-4) score, as compared to placebo.
  • The data show that Namodenoson reversed and eliminated NASH, and may prevent the progression of NAFLD into NASH.
  • Patients with evidence of an active inflammation were treated twice daily with 12.5 mg (n=21) or 25 mg (n=19) of oral Namodenoson vs. placebo (n=20).

SEngine Precision Medicine to Present Data Related to the Predictive Value of PARIS® Test, an Organoid-based Drug Response Assay, for Cholangiocarcinoma Patients at 2020 AACR Virtual Meeting Session II

Retrieved on: 
Friday, May 15, 2020
Health, Branches of biology, Academic disciplines, Cholangiocarcinoma, Hepatology, RTT, Precision medicine, Sensitivity and specificity, Clinical Laboratory Improvement Amendments, Organoid

The AACR poster presentation summarizes the strong correlation between genomics,retrospective treatment outcomes and PARIS Test drug sensitivity results based on a retrospective analysis of 17 cholangiocarcinoma patients.

Key Points: 
  • The AACR poster presentation summarizes the strong correlation between genomics,retrospective treatment outcomes and PARIS Test drug sensitivity results based on a retrospective analysis of 17 cholangiocarcinoma patients.
  • This study shows the feasibility of functional testing of organoids derived from cholangiocarcinoma patients tissue in a CLIA-certified diagnostic test.
  • SEngines CLIA certified PARIS Test generates predictive drug sensitivity reports for patients with solid tumors.
  • SEngine is also pursuing drug discovery via strategic collaborations with biopharmaceutical / pharma companies leveraging its precision oncology platform.

Tiziana Lifesciences Announces Online Publication of Two Abstracts at the American Society of Clinical Oncology (ASCO) Reporting Clinical Activity and Safety of Milciclib in Patients with Advanced Hepatocellular Carcinoma

Retrieved on: 
Thursday, May 14, 2020
Chemical compounds, Organic compounds, Aromatic compounds, Chloroarenes, Pyridines, Ureas, Cancer treatments, Hepatology, Sorafenib, Regorafenib, Hepatocellular carcinoma, Protein kinase inhibitor

Abstract #298561: Phase 2a Safety and Efficacy of Milciclib, a Pan-Cyclin Dependent Kinase Inhibitor, in Unresectable, Sorafenib-Refractory or -Intolerant Hepatocellular Carcinoma Patients.

Key Points: 
  • Abstract #298561: Phase 2a Safety and Efficacy of Milciclib, a Pan-Cyclin Dependent Kinase Inhibitor, in Unresectable, Sorafenib-Refractory or -Intolerant Hepatocellular Carcinoma Patients.
  • Positive demonstrated clinical activity included:
    50% (14 out of 28) evaluable patients completed 6-month duration of the trial.
  • Abstract #307309: Safety and Clinical Activity of Combination Treatment with Regorafenib and Milciclib in Liver Transplant Patients with Hepatocellular Carcinoma Recurrence.
  • The American Society of Clinical Oncology (ASCO) was established in 1964 with the sole purpose of improving the care of people with cancer.

AVROBIO Presents New Preclinical Data on Lentiviral Gene Therapy Program for Pompe Disease at ASGCT 2020

Retrieved on: 
Thursday, May 14, 2020
Research, FDA, Genetics, Clinical trials, Biotechnology, Other Health, Health, Other Science, Science, Inborn errors of metabolism, Branches of biology, Rare diseases, Lipid storage disorders, Lysosomal storage diseases, Glycogen storage disease type II, Hepatology, Fabry disease, Gene therapy, Gaucher's disease, Pompe, Pompe disease, lentiviral gene therapy, AVROBIO

(Nasdaq: AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced new preclinical data for AVR-RD-03 for Pompe disease showcasing how its lentiviral gene therapy approach may potentially correct Pompe disease manifestations in the muscle and central nervous system (CNS).

Key Points: 
  • (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced new preclinical data for AVR-RD-03 for Pompe disease showcasing how its lentiviral gene therapy approach may potentially correct Pompe disease manifestations in the muscle and central nervous system (CNS).
  • The data will be presented today at the American Society of Gene & Cell Therapy (ASGCT) 23rd Annual Meeting.
  • Our clinical-stage programs include Fabry disease, Gaucher disease and cystinosis and we also are advancing a program in Pompe disease.
  • AVROBIO is powered by the plato gene therapy platform, our foundation designed to scale gene therapy worldwide.

Mirum Pharmaceuticals Reports Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)

Retrieved on: 
Monday, May 11, 2020
Biotechnology, Pharmaceutical, Health, Clinical trials, Medical specialties, Digestive diseases, Hepatology, Medicine, Progressive familial intrahepatic cholestasis, PFIC, Alagille syndrome, Cholestasis, Liver

The Compensation Committee of Mirums Board of Directors approved the awards as an inducement material to the new employees employment in accordance with Nasdaq Listing Rule 5635(c)(4).

Key Points: 
  • The Compensation Committee of Mirums Board of Directors approved the awards as an inducement material to the new employees employment in accordance with Nasdaq Listing Rule 5635(c)(4).
  • The awards are subject to the terms and conditions of Mirums 2020 Inducement Plan and the terms and conditions of an applicable award agreement covering the grant.
  • Mirum Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the development and commercialization of a late-stage pipeline of novel therapies for debilitating liver diseases.
  • The companys lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC).

Protagonist Therapeutics Announces Initial Phase 2 Results with Hepcidin Mimetic PTG-300 in the Treatment of Polycythemia Vera

Retrieved on: 
Thursday, May 7, 2020
Medical specialties, Branches of biology, Medicine, Hematopathology, Iron metabolism, Hepatology, Polycythemia vera, Polycythemia, Hepcidin, HFE hereditary haemochromatosis

NEWARK, Calif., May 7, 2020 /PRNewswire/ --Protagonist Therapeutics, Inc. (Nasdaq:PTGX) today announced initial data from the ongoing Phase 2 study of PTG-300 in patients with polycythemia vera.

Key Points: 
  • NEWARK, Calif., May 7, 2020 /PRNewswire/ --Protagonist Therapeutics, Inc. (Nasdaq:PTGX) today announced initial data from the ongoing Phase 2 study of PTG-300 in patients with polycythemia vera.
  • In the near term, we are expanding the current study to include additional patients as the Company focuses on these encouraging results.
  • These early results are very encouraging and suggest the potential for a paradigm shift for the treatment of polycythemia vera.
  • PTG-300 is an injectable hepcidin mimetic in development for the treatment of polycythemia vera and hereditary hemochromatosis.

HighTide Therapeutics Announces Positive Topline Results From Phase 2a Study of HTD1801 in NASH Patients

Retrieved on: 
Thursday, May 7, 2020
Health, Diabetes, Other Health, General Health, Clinical trials, Pharmaceutical, Biotechnology, Medical specialties, Medicine, Health, Hepatology, Primary sclerosing cholangitis, Hepatitis, Non-alcoholic fatty liver disease, Clinical trial, NASH, PSC, HighTide Therapeutics and HTD1801

HighTide Therapeutics Inc. (HighTide), a clinical-stage biopharmaceutical company, today announced topline results of the study of HTD1801 in 100 adults with non-alcoholic steatohepatitis (NASH) and type 2 diabetes mellitus (T2DM).

Key Points: 
  • HighTide Therapeutics Inc. (HighTide), a clinical-stage biopharmaceutical company, today announced topline results of the study of HTD1801 in 100 adults with non-alcoholic steatohepatitis (NASH) and type 2 diabetes mellitus (T2DM).
  • This trial was a dose-ranging, double blind, placebo controlled, multi-center Phase 2a clinical trial evaluating the treatment effects of HTD1801 in patients with NASH and T2DM conducted at 17 U.S. clinical sites.
  • As the principal investigator, I was very gratified to see the study results, said Stephen A. Harrison, M.D., Medical Director at Pinnacle Clinical Research.
  • Additionally, HighTide is also providing an update on the on-going Phase 2 study of HTD1801 in adult patients with primary sclerosing cholangitis (PSC) in the U.S. and Canada.

Enanta Announces Results of INTREPID Study of EDP-305 for the Treatment of Primary Biliary Cholangitis

Retrieved on: 
Wednesday, May 6, 2020
Biotechnology, Health, Science, Pharmaceutical, Research, Medical specialties, Hepatology, Medicine, Gastroenterology, Bile acids, Cholanes, Autoimmune diseases, Primary biliary cholangitis, Ursodeoxycholic acid, Obeticholic acid, Primary Biliary Cholangitis, EDP-305, a Farnesoid X Receptor Agonist, Enanta Pharmaceuticals, Inc.

The INTREPID study was a 12-week, randomized, double-blind, placebo-controlled study evaluating the safety, tolerability, pharmacokinetics and efficacy of EDP-305 in subjects with PBC, with or without an inadequate response to ursodeoxycholic acid.

Key Points: 
  • The INTREPID study was a 12-week, randomized, double-blind, placebo-controlled study evaluating the safety, tolerability, pharmacokinetics and efficacy of EDP-305 in subjects with PBC, with or without an inadequate response to ursodeoxycholic acid.
  • Overall, EDP-305 was generally safe in subjects with PBC, with the majority of treatment-emergent adverse events (TEAEs) being mild to moderate.
  • The incidence of treatment discontinuation due to pruritus in INTREPID was approximately 3% for the 1 mg EDP-305 treatment group and 18% for the 2.5 mg EDP-305 treatment group.
  • Primary biliary cholangitis (PBC) is a chronic disease of the liver that slowly destroys the medium-sized bile ducts within the liver.