Inipharm Initiates Dosing in Phase 1 Trial for Small Molecule Inhibitor Targeting HSD17B13
Friday, December 01, 2023
Inipharm, a biopharmaceutical company focused on developing therapies for severe liver diseases, has announced the initiation of dosing in a Phase 1 study for INI-822, its orally-delivered small molecule inhibitor of HSD17B13. INI-822 is believed to be the first small molecule inhibitor of HSD17B13 to advance to clinical development and is being developed for fibrotic liver diseases, including non-alcoholic steatohepatitis (NASH).
Dr. Chuhan Chung, Chief Medical Officer of Inipharm, emphasized the significance of multiple human genetic validation studies showing that inactive forms of HSD17B13 protect against the progression of several liver diseases, including NASH. He pointed out that other development approaches are focused on RNA knockdown of HSD17B13, but these require injection delivery. INI-822, if successful, would provide an oral option more suitable for the chronic treatment of diseases like NASH.
The Phase 1 study is a randomized, double-blind, placebo-controlled trial enrolling 96 participants in three parts. Parts A and B involve approximately 72 healthy volunteers, and Part C enrolls 24 participants with NASH or presumed NASH. The study aims to assess safety, tolerability, pharmacokinetics of INI-822, and biomarkers of target engagement.
In preclinical studies, INI-822 demonstrated potent and selective inhibition of HSD17B13, showing improvements in markers of liver homeostasis in animal models. These improvements included a reduction in liver transaminases, specific bioactive lipids, and the enrichment of hepatic lipid species associated with the protective variant of HSD17B13 observed in patients.
Dr. Chung highlighted that HSD17B13 inhibition represents a genetically validated approach that may improve various aspects of liver injury, such as inflammation and fibrosis. This could be as standalone therapy or in combination with other treatments addressing liver fat but not directly targeting key features of liver injury.
Brian Farmer, Co-founder and CEO of Inipharm, expressed gratitude for the team's dedication and acknowledged the challenges in developing a small molecule inhibitor for HSD17B13. Commencing clinical development for this program is a significant achievement for Inipharm.
Multiple human genetic association studies have confirmed that enzymatically inactive variants of the HSD17B13 protein are associated with a reduced risk of developing more advanced fibrotic disease in metabolic, alcoholic, and viral liver diseases. INI-822, a small molecule inhibitor of HSD17B13, is Inipharm’s first development candidate.
Source: businesswire.com
