Benzinga | Dec 29, 2020 04:16PM EST

Hepion Pharmaceuticals Announces Top Line Data For Low Dose CRV431 In Phase 2a 'AMBITION' Clinical Trial For Advanced NASH; Clinically Significant Reductions In Important Biomarkers ALT And AST Observed In 28 Days

* CRV431 Demonstrated to be Generally Safe and Well Tolerated

* Clinically Significant Reductions in Important Biomarkers, ALT and AST, Observed in 28 Days

* Study Continuing with Higher Dose

EDISON, NJ / ACCESSWIRE / December 29, 2020 / Hepion Pharmaceuticals, Inc. (NASDAQ:HEPA)("Hepion"), a clinical stage biopharmaceutical company focused on Artificial Intelligence ("AI")-driven therapeutic drug development for the treatment of non-alcoholic steatohepatitis ("NASH") and liver disease, today announced top line data from the low dose cohort in the Company's Phase 2a 'AMBITION' clinical trial of CRV431, an oral, once daily novel cyclophilin inhibitor. This Phase 2a study is continuing with the higher dose of 225 mg CRV431, with NASH patient dosing expected to be completed in Q1-2021.

The AMBITION trial is a placebo-controlled study of CRV431 in NASH patients with evidence of moderate-to-severe fibrosis. In this study, which is being conducted at 10 U.S. sites, 75 mg CRV431 (low dose) was administered orally, once-daily for 28 days. A second dosing cohort of 225 mg CRV431 (high dose) is ongoing. Final results from both dosing cohorts are expected after the high dose group has completed active dosing, followed by a 14-day observation period.

The primary objectives of the AMBITION trial are to assess safety and tolerability of CRV431, as well as to delineate pharmacokinetics ("PK"). The secondary outcome measure of this Phase 2a trial is to evaluate decreases in antifibrotic markers from baseline to the end of study.

The results of the low dose group indicated that CRV431 was generally safe and well tolerated. The pharmacokinetics further indicated that blood concentrations of CRV431 were similar to those observed in earlier Phase 1 studies in healthy volunteers, suggesting that the PK profile in moderate-to-severe NASH patients did not appear to be altered by disease. Biomarkers of efficacy, including declines in alanine aminotransferase ("ALT") and aspartate aminotransferase ("AST") from baseline to day 28 were observed, with mean declines of 18.4% and 12.1%, respectively. At day 28 in the placebo group, the changes observed for ALT and AST from baseline were a 0.65% reduction and a 2.52% increase, respectively. Improvements in mean transaminase values were not powered to be statistically significant, as only 12 subjects received drug.

Dr. Stephen Harrison, Hepion's Consultant Medical Director, commented, "The reductions in the liver chemistry tests, ALT and AST, occurred rapidly and were sustained throughout the 28 days of dosing. These reductions began to return to baseline once the patients stopped taking CRV431 at the end of the study, indicative of a drug effect. As such, these early signals are supportive of positive actions on liver health in a short period of time with the low dose of CRV431, and we eagerly look forward to evaluating the fibrosis biomarkers, as well as the higher dose of CRV431, in the second cohort."

"We are pleased to see this positive movement in liver chemistry tests," commented Dr. Robert Foster, Hepion's CEO. "Although the primary focus of this Phase 2a study was to examine the safety and tolerability of CRV431 in NASH patients, we were hoping to see early signs of potential efficacy and are pleased with the results thus far. We will continue to monitor liver safety lab tests in our higher dose group and will also look at a panel of serum fibrosis biomarkers. Once completed, we will analyze all the data from the Phase 1 and 2a trials, as well as our non-clinical studies to conduct an a priori analysis with our artificial intelligence platform, AI-POWRTM, to enrich our Phase 2b study design and optimize it for outcomes. Our Phase 2b trial is anticipated to start midway through 2021."