Inipharm Initiates Dosing in 12-Week Proof-of-Concept Clinical Study in MASH With Its Oral HSD17B13 Inhibitor INI-822

Clinical study to build upon encouraging safety, pharmacokinetic and biomarker data generated in Phase 1c study of INI-822 in MASH patients

BELLEVUE, Wash. & SAN DIEGO--(BUSINESS WIRE)--Inipharm, a clinical-stage biopharmaceutical company focused on developing therapies for fibrotic liver diseases, today announced that it has initiated dosing in a 12-week proof-of-concept clinical study assessing its orally available small molecule, INI-822, for the treatment of MASH.

“Our ongoing clinical trial of INI-822 has generated encouraging biomarker and target engagement data in MASH patients after only 28 days of dosing, demonstrating its potential to meaningfully impact the course of MASH,” said Chuhan Chung, M.D., chief medical officer of Inipharm. “This 12-week study will build on that data and provide us even greater insight into how INI-822 may address MASH features such as fibrosis and is designed to position us to enter Phase 2b studies.”

INI-822 is designed to inhibit the enzymatic activity of HSD17B13 and is the only small-molecule inhibitor of HSD17B13 in the clinic. Protective genetic variants of the HSD17B13 protein that reduce its activity demonstrate a decrease in the risk of fibrotic liver disease, including 50 to 60% reductions in risk of progression from simple steatosis, characterized by liver fat, to MASH with fibrosis or cirrhosis. Importantly, protective HSD17B13 mitigates the risks driven by PNPLA3 I148M, the major genetic risk factor for progression of MASH. Individuals carrying this risk variant may not respond as well to therapies such as GLP-1 agonists.

“Injectable RNAi-based approaches to targeting HSD17B13 have generated encouraging data in MASH, bolstering our confidence in the target and our program,” said Brian Farmer, CEO of Inipharm. “That noted, we believe an oral small molecule that inhibits enzymatic activity of HSD17B13, rather than knocking it down, may better reflect the physiology of the naturally occurring protective variant, in addition to providing more convenient dosing for patients.”

"HSD17B13 is an increasingly well‑validated target in MASH, supported by strong human genetics,” said Rohit Loomba, MD, chief of the Division of Gastroenterology and Hepatology at University of California San Diego School of Medicine. "The results of this study will help clarify the potential role of HSD17B13 inhibition in addressing gaps in treatment strategies for MASH.”

The double-blind, placebo-controlled clinical study will enroll approximately 50 patients with MASH. In addition to pharmacokinetics and safety and tolerability, the study will assess changes in MASH-related biomarkers, biomarkers indicative of INI-822’s target engagement and fibrosis, as assessed by FibroScan.

About HSD17B13 and INI-822

Numerous human genetic association studies have confirmed that enzymatically inactive variants of the HSD17B13 protein are associated with a reduced risk of progressing to 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 clinical-stage biopharmaceutical company focused on discovering and developing therapies for severe liver diseases. Inipharm’s lead program is focused on the highly validated genetic target, HSD17B13. There is 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 small-molecule therapies that target the activity of this protein including INI-822, which is currently in clinical development for the treatment of MASH. For more information, visit https://inipharm.com.