Company targets Phase 2 trial of MKK4 inhibitor that demonstrates ability to enhance liver regeneration without promoting tumorigenesis.
German biotech HepaRegeniX has successfully completed a €21.5 million financing round to advance the clinical development of its lead drug candidate aimed at enhancing liver regeneration in patients with acute and chronic liver conditions. The new investment will specifically be used to finalize the ongoing Phase 1b trial and to initiate and progress a subsequent Phase 2a trial.
HepaRegeniX’s approach is based on the inhibition of Mitogen-Activated Protein Kinase Kinase 4 (MKK4), a critical regulator in the liver’s regenerative process. The approach stems from research led by Professor Lars Zender at the University Hospital of Tübingen, whose team discovered MKK4 as a key molecular target and demonstrated its role in liver regeneration using small RNA molecules. The company collaborated with Zender’s group to design its lead program HRX-215, an orally administered small molecule designed to selectively inhibit MKK4 and promote liver regeneration without triggering uncontrolled cell proliferation.
By targeting MKK4, HRX-215 activates the regenerative potential of hepatocytes in both healthy and damaged livers. According to HepaRegeniX, the mechanism holds therapeutic potential in a range of clinical scenarios, such as enabling surgical resection of tumors in patients previously considered inoperable due to insufficient remaining liver volume, supporting liver regeneration following living donor transplantation, and treating patients suffering from severe alcohol-associated hepatitis. In preclinical models, including an experimental setting of high-risk liver resection in pigs, HRX-215 significantly improved survival and liver tissue regeneration.
The compound has also shown the ability to reverse pathological processes like fibrosis and steatosis in diseased livers, while maintaining a favorable safety profile with no observed risk of tumor formation in knockdown models. A completed Phase 1 clinical study in healthy volunteers confirmed HRX-215’s safety and pharmacokinetics.
“We continue to make significant progress in the clinical evaluation of our lead candidate, HRX-215, and the additional investment is a strong acknowledgment of our achievements and the impressive safety results of HRX-215 in the completed Phase I trials,” said HepaRegeniX CEO Elias Papatheodorou.
With liver disease causing over two million deaths globally each year, liver transplantation is often the only viable option for many late-stage patients. It is hoped that a drug like HRX-215 could expand treatment options, and may also broaden the pool of eligible living donors due to its potential ability to improve the viability of smaller donor liver grafts.
The funding round saw HepaRegeniX secure additional investment from Munch-based Wellington Partners, which joins a syndicate of existing backers including Vesalius Biocapital IV, Novo Holdings A/S, Boehringer Ingelheim Venture Fund, Coparion, High-Tech Gründerfonds and Ascenion GmbH.
“HepaRegeniX’ approach is highly differentiated and has demonstrated efficacy in several in vivo models for liver diseases with extraordinarily high unmet medical need,” said Wellington Managing Partner Dr Rainer Strohmenger. “We look forward to supporting the generation of meaningful clinical efficacy data in human patients in the near future.”
