HepaRegeniX Completes Darizmetinib Trial for Liver Cancer Patients

Clinical-stage biotech company HepaRegeniX GmbH has announced the successful completion of the Phase 1b of its clinical trial for darizmetinib, a novel therapy aimed at treating acute and chronic liver diseases. The first patient to undergo major liver resection for liver metastases originating from colorectal cancer has completed the 28-day treatment with darizmetinib, a treatment formerly known as HRX215. This milestone marks a significant step in the company's ongoing Phase 1b/2a study, which is expected to enroll ten patients and be completed in the fourth quarter of 2026.

The Phase 1b study, which is part of the ongoing Phase 1b/2a trial, has demonstrated a favorable safety profile with no darizmetinib-related serious adverse events reported. This progress has led to the seamless advancement of darizmetinib into the randomized Phase 2a portion of the study, which is planned to be conducted in the same patient population. The study's design, which has been reviewed and authorized by all relevant regulatory authorities, including the FDA, EMA, and the Israel Ministry of Health, as well as ethics committees and Institutional Review Boards (IRBs), has been recognized for its streamlined approach.

Major liver resection is a critical part of standard of care for patients with liver metastases, but postoperative recovery of liver function remains a significant challenge.

Darizmetinib, formerly known as HRX215, is an orally available small molecule inhibitor of mitogen-activated protein kinase kinase 4 (MKK4), a key regulator of liver regeneration. In preclinical models, darizmetinib has been shown to selectively inhibit MKK4, stabilizing and protecting hepatocytes while accelerating and enhancing regenerative processes, even in compromised or diseased livers. This therapeutic approach has the potential to expand surgical eligibility to patients requiring extended liver resection who would otherwise be deemed inoperable, offering a new path to potentially lifesaving treatment. Darizmetinib can potentially transform the field of liver transplantation by preventing liver failure after the transplant of small liver grafts and thereby expand living donor transplantations, as well as in acute and chronic late-stage liver diseases that often have limited therapeutic options.

HepaRegeniX is advancing therapies to treat acute and chronic liver diseases based on groundbreaking discoveries of a novel cellular target and small molecules that enable rapid liver regeneration. The company's lead candidate, darizmetinib (HRX215), is an orally available small molecule that selectively inhibits mitogen-activated protein (MAP) kinase kinase 4 (MKK4), a master regulator of liver regeneration. Building on demonstrated safety in clinical trials, HepaRegeniX is advancing darizmetinib in a Phase 1b/2a trial to prevent post-hepatectomy liver failure, with potential applications in living donor liver transplantation and severe alcohol-associated hepatitis. Beyond liver diseases, the company is also developing HRX233 in oncology.

HepaRegeniX is backed by experienced life science investors, including Vesalius Biocapital IV, Novo Holdings A/S, Boehringer Ingelheim Venture Fund (BIVF), Coparion, High-Tech Gründerfonds, Ascenion GmbH, and Wellington Partners.