Liver disease

A considerable roadblock in the successful triaging of new therapeutics for liver and metabolic related diseases is the reliability and effectiveness of pre-clinical disease models. Methods employing animal models may have limited success in replicating liver diseases, primarily because of species-specific differences, furthermore, the utilisation of animal models to simulate liver disease phenotypes frequently leads […]

A considerable roadblock in the successful triaging of new therapeutics for liver and metabolic related diseases is the reliability and effectiveness of pre-clinical disease models. Methods employing animal models may have limited success in replicating liver diseases, primarily because of species-specific differences, furthermore, the utilisation of animal models to simulate liver disease phenotypes frequently leads […]

A considerable roadblock in the successful triaging of new therapeutics for liver and metabolic related diseases is the reliability and effectiveness of pre-clinical disease models. Methods employing animal models may have limited success in replicating liver diseases, primarily because of species-specific differences, furthermore, the utilisation of animal models to simulate liver disease phenotypes frequently leads […]

A considerable roadblock in the successful triaging of new therapeutics for liver and metabolic related diseases is the reliability and effectiveness of pre-clinical disease models. Methods employing animal models may have limited success in replicating liver diseases, primarily because of species-specific differences, furthermore, the utilisation of animal models to simulate liver disease phenotypes frequently leads […]

A considerable roadblock in the successful triaging of new therapeutics for liver and metabolic related diseases is the reliability and effectiveness of pre-clinical disease models. Methods employing animal models may have limited success in replicating liver diseases, primarily because of species-specific differences, furthermore, the utilisation of animal models to simulate liver disease phenotypes frequently leads […]

A considerable roadblock in the successful triaging of new therapeutics for liver and metabolic related diseases is the reliability and effectiveness of pre-clinical disease models. Methods employing animal models may have limited success in replicating liver diseases, primarily because of species-specific differences, furthermore, the utilisation of animal models to simulate liver disease phenotypes frequently leads […]

Madrigal Pharmaceuticals has submitted an application for resmetirom, a drug for nonalcoholic steatohepatitis (NASH) with liver fibrosis, to the US Food and Drug Administration (FDA) and has requested a priority review. Resmetirom is a once daily, oral, thyroid hormone receptor (THR)-β selective agonist designed to target key underlying causes of NASH in the liver. The […]

Sequana Medical, a company targeting fluid overload in liver disease, heart failure and cancer, has enrolled the first patient in its MOJAVE study.  MOJAVE, a randomised controlled Phase I/IIa study in the US, will evaluate the safety and efficacy of the company’s second-generation DSR product (DSR 2.0) in diuretic-resistant chronic heart failure patients with persistent […]

The European Medicines Agency (EMA) has granted PRIME (Priority Medicines) status to the gene therapy, GNT-0003, currently in clinical trials for Crigler-Najjar syndrome, a rare liver disease.   This status, granted only to drug candidates with major therapeutic potential, follows the successful completion of the first phases of the clinical trials.  The manufacturer, Genethon, is a […]

Intercept Pharmaceuticals’ obeticholic acid (OCA) could be first drug to be approved in the US for non-alcoholic steatohepatitis (NASH), according to analysts GlobalData.  The Food and Drug Administration (FDA) accepted the company’s resubmission of its new drug application (NDA) for OCA in patients with pre-cirrhotic liver fibrosis due to NASH in December 2022.  According to […]

China is expected to record the highest diagnosed prevalence of non-alcoholic fatty liver disease (NAFLD) in 2029, representing 44% of cases within the 16 major pharmaceutical markets (16MM).   As a result, the number of clinical trials conducted in the country for this indication is gradually increasing, driven mostly by international pharmaceutical companies.  According to GlobalData’s […]

Enanta Pharmaceuticals, a clinical-stage biotechnology company dedicated to creating small molecule drugs for viral infections and liver diseases, has dosed the first subject in its Phase I clinical trial of EDP-323, a novel, oral L-protein inhibitor in development for the treatment of respiratory syncytial virus (RSV).  Context   RSV is the most common cause of bronchiolitis […]

News round-up for 10-14 October by DDW Digital Content Editor Diana Spencer The top news in the world of drug discovery this week reflects the vital importance of collaboration, partnership and support within this industry.  Partnerships have been forged and renewed to shed light on neuropsychiatric disorders, oncology and cystic fibrosis. Meanwhile, Ochre Bio successfully raised […]

Ochre Bio, a company developing therapies for liver health challenges, has closed $30 million in Series A financing, which will be used to translate the insights from their discovery research into RNA-based drug candidates, and then test these in human livers at their recently launched ‘Liver ICUs’ across the US.   Employing a deep phenotyping […]

MiNA Therapeutics has dosed the first patient in a global Phase II clinical trial (OUTREACH-2) of MTL-CEBPA in combination with second line standard of care sorafenib (a tyrosine kinase inhibitor (TKI)) in advanced hepatocellular carcinoma (HCC or liver cancer).  What is OUTREACH-2?  OUTREACH-2 is a multi-centre, open-label, randomised study of MTL-CEBPA in combination with sorafenib, compared to sorafenib […]

Dompé Farmaceutici has entered a strategic collaboration with and investment in Engitix as part of Engitix’s $54m Series A financing to support identification of new treatments against fibrosis and liver-associated solid tumours.  The agreement   Dompé will provide direct access to facilities, resources and expertise including its Exscalate structure-based drug design platform which can design new molecules following the rules of rational drug design to obtain […]