HepaRegeniX GmbH has announced the initiation of a Phase 1 clinical trial for its lead drug candidate, HRX-0215, a small molecule inhibitor of Mitogen-Activated Protein (MAP) Kinase Kinase 4 (MKK4), marking a novel approach to enhancing liver regeneration. The trial, a single-center, double-blind, randomized, placebo-controlled study, will enroll 48 healthy male volunteers to assess the safety, tolerability, and pharmacokinetics of HRX-0215.
The Phase 1 trial is designed as a single and multiple dose escalation study (SAD/MAD). HepaRegeniX anticipates initial results later this year. Preclinical studies have indicated that HRX-0215 demonstrates significant efficacy in models of both acute and chronic liver diseases.
MKK4 Inhibition: A Novel Therapeutic Strategy
HRX-0215 represents a first-in-class approach by targeting MKK4, a key regulator of liver regeneration. Suppression of MKK4 is believed to unlock the regenerative capacity of hepatocytes, even in severely diseased livers. This mechanism offers a potential breakthrough in treating liver diseases, where current therapeutic options are limited.
"It is exciting to see the first MKK4 inhibitor now entering clinical development only four years after the synthesis of our very first small molecule compound," said Dr. Wolfgang Albrecht, Chief Operating Officer and Co-Founder of HepaRegeniX. He further stated, "This start of clinical development is backed by a large dataset showing the potential of our MKK4 inhibitor improving key liver parameters in a number of preclinical models plus an encouraging safety profile in 4-week general toxicology studies."
Unmet Need in Liver Diseases
Liver diseases represent a substantial global health burden, affecting millions and significantly impairing their quality of life. In EU5 countries and the U.S., approximately 250,000 patients suffer from acute liver diseases annually, while 700,000 are affected by chronic liver diseases. Chronic liver diseases are also a major risk factor for hepatocellular carcinoma, a leading cause of cancer-related deaths.
About HRX-0215
HRX-0215 is a potent and selective inhibitor of MKK4 that has demonstrated preclinical efficacy in both acute and chronic liver disease models. The compound was well-tolerated in 4-week general toxicology studies, with no geno-toxicological liabilities detected. The discovery of HRX-0215 was a collaborative effort between HepaRegeniX and Prof. Stefan Laufer’s group from the University of Tuebingen.
HepaRegeniX: Pioneering Liver Disease Therapies
Since its establishment in 2017, HepaRegeniX has focused on discovering and developing preclinical drug candidates for treating acute and chronic liver diseases. The company's approach centers on MKK4, a novel molecular target identified by Prof. Lars Zender and his research group at the University Hospital Tubingen.
