BELLEVUE, Wash. & SAN DIEGO--(BUSINESS WIRE)--Inipharm, a biopharmaceutical company focused on discovering and developing therapies for severe liver diseases, today announced that it has begun dosing in a Phase 1 study of INI-822, its orally-delivered small molecule inhibitor of HSD17B13. INI-822, believed to be the first small molecule inhibitor of HSD17B13 to advance to clinical development, is under development for fibrotic liver diseases, including non-alcoholic steatohepatitis (NASH).
“Multiple human genetic validation studies demonstrate that inactive forms of HSD17B13 protect against the progression to more advanced stages of several liver diseases including NASH,” said Chuhan Chung, M.D., chief medical officer of Inipharm. “Other development approaches are focused on RNA knockdown of HSD17B13, but those must be delivered via injection. INI-822, if successful in clinical studies, would offer an oral option that may be more suitable for treating a chronic disease like NASH.”
The randomized, double-blind, placebo-controlled Phase 1 study will enroll 96 participants in three parts. Parts A and B will enroll approximately 72 healthy volunteers, and Part C will enroll 24 participants with NASH or presumed NASH. The study is designed to assess safety and tolerability and pharmacokinetics of INI-822, as well as biomarkers of target engagement.
In preclinical studies, INI-822 was shown to potently and selectively inhibit HSD17B13, and when dosed in animal models, demonstrated improvements in markers of liver homeostasis. These included a reduction in liver transaminases and specific bioactive lipids, and the enrichment of hepatic lipid species that were also identified in patients with the protective variant of HSD17B13.
“HSD17B13 inhibition represents a genetically validated approach that may improve multiple aspects of liver injury, such as inflammation and fibrosis. This could be as standalone therapy or in combination with other therapies that reduce liver fat but may not directly address key features of liver injury,” added Chung.
“I want to applaud the Inipharm team who has worked tirelessly to develop a small molecule that can inhibit HSD17B13,” said Brian Farmer, co-founder and chief executive officer of Inipharm. “This target presents significant challenges with respect to the development of a small molecule inhibitor. I am grateful for the dedication of our team, collaborators and investors. The start of clinical development for this program is a significant milestone for Inipharm.”
About HSD17B13 and INI-822
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.
About Inipharm
Inipharm is a biopharmaceutical company focused on discovering and developing therapies for severe liver diseases. Inipharm’s lead programs are focused on the highly validated genetic target, HSD17B13. Extensive, consistent evidence that genetic variants in expression of HSD17B13 are associated with significantly lower rates and severity of multiple liver diseases. Building on these novel insights into the biologic activity of HSD17B13, Inipharm is advancing a pipeline of small-molecule therapies that target the activity of this protein.
