Hepion Pharmaceuticals Announces Publication of Phase 2a ‘AMBITION’ Clinical Trial Results

Rhea-AI Summary

Hepion Pharmaceuticals announced that the Phase 2a study of its drug candidate rencofilstat for non-alcoholic steatohepatitis (NASH) successfully met its primary endpoints focusing on safety and pharmacokinetics. The study demonstrated significant reductions in ALT, ProC3, and C6M biomarkers, indicating potential antifibrotic effects. Enrollment for the Phase 2b trial, ‘ASCEND-NASH’, has begun with 336 subjects. The findings underscore the drug's potential effectiveness and support its progression toward clinical development.

Positive

• Successful completion of Phase 2a study for rencofilstat with met safety and pharmacokinetics endpoints.
• Significant reductions in ALT, ProC3, and C6M suggest antifibrotic effects.
• Enrollment initiated for larger Phase 2b trial with 336 subjects.

Negative

• None.

- Study met safety, tolerability, and pharmacokinetics primary endpoints -

- Reductions in ALT, ProC3, and C6M suggest direct antifibrotic effects with longer treatment duration -

- Enrollment initiated in Phase 2b ‘ASCEND-NASH’ trial evaluating the safety and efficacy of rencofilstat in 336 biopsy confirmed F2 / F3 NASH subjects -

EDISON, N.J., Oct. 26, 2022 (GLOBE NEWSWIRE) -- Hepion Pharmaceuticals, Inc. (NASDAQ:HEPA), a clinical stage biopharmaceutical company focused on Artificial Intelligence (“AI”)-driven therapeutic drug development for the treatment of fibrotic diseases, including non-alcoholic steatohepatitis (“NASH”), hepatocellular carcinoma (“HCC”), and other chronic liver diseases, today announced that the peer-reviewed journal, Hepatology Communications, has published a paper by Harrison et al. entitled, “Rencofilstat, a cyclophilin inhibitor: A phase 2a, multicenter, single-blind, placebo-controlled study in F2/F3 NASH,” reviewing the results of Hepion’s ‘AMBITION’ clinical trial.

The Phase 2a, multicenter, single-blind, placebo-controlled study randomized 49 presumed F2/F3 NASH subjects to receive either 75 mg or 225 mg of rencofilstat, or placebo, once daily for 28 days. The aim of the study was to determine safety, tolerability, and pharmacokinetics, while exploring NASH efficacy biomarkers, including multi-omic and AI-POWR™ analyses.

As previously reported, the AMBITION clinical trial demonstrated rencofilstat was safe and well tolerated. A majority of subjects (28/47; 59.6%) who were dosed with rencofilstat, or placebo experienced no adverse events. Of the 36 adverse events recorded in total, 97.2% were graded as mild to moderate, none were serious, and the majority (27/36; 75%) were considered unrelated to administration of rencofilstat. Additionally, blood concentrations of rencofilstat in the NASH subjects were similar to those observed previously in healthy subjects.

The reductions in alanine transaminase (“ALT”), a biomarker of liver damage, were greater in the rencofilstat arms versus the placebo groups and was statistically different in the 225-mg cohort compared to the placebo cohort (−16.3 ± 25.5% versus −0.7 ± 13.4%, respectively). Reductions in Pro-C3, a biomarker of collagen formation and fibrosis, and C6M, a biomarker of tissue remodeling, were statistically significant in rencofilstat subjects with baseline Pro-C3 levels above 15.0 ng/mL. Pro-C3 levels greater than 15-20 ng/mL are generally accepted to represent active NASH disease and a marker of fibrosis in the primary patient population for treatment by many NASH drug candidates. The paper concluded that the reductions in ALT, Pro-C3, and C6M suggest that rencofilstat has direct antifibrotic effects with longer treatment duration, supporting the advancement of rencofilstat into a larger and longer Phase 2b study.

“The findings of this paper further strengthen the rationale behind our ongoing Phase 2b ‘ASCEND-NASH’ trial, which initiated screening at the end of August,” said Robert Foster, PharmD, PhD, CEO of Hepion. “The degree of decline in Pro-C3 observed in the AMBITION trial, which had a duration of only 28 days, was comparable to Pro-C3 declines seen in similar studies with durations of several months, particularly in subjects with Pro-C3 levels indicating more advanced disease. Given this observation, we are very much looking forward to seeing the magnitude of rencofilstat’s antifibrotic effects over a 12-month-period in our ongoing Phase 2b trial.”

The article may be accessed at: https://doi.org/10.1002/hep4.2100.

About Hepion Pharmaceuticals

The Company's lead drug candidate, rencofilstat, is a potent inhibitor of cyclophilins, which are involved in many disease processes. Rencofilstat is currently in clinical-phase development for the treatment of NASH, with the potential to play an important role in the overall treatment of liver disease - from triggering events through to end-stage disease. Rencofilstat has been shown to reduce liver fibrosis and hepatocellular carcinoma tumor burden in experimental models of NASH, and has demonstrated antiviral activities towards HBV, HCV, and HDV through several mechanisms, in nonclinical studies. In November 2021, the U.S. Food and Drug Administration (“FDA”) granted Fast Track designation for rencofilstat for the treatment of NASH. That was followed in June 2022 by the FDA’s granting of Orphan Drug designation to rencofilstat for the treatment of HCC.

Hepion has created a proprietary AI platform, called AI-POWR™, which stands for Artificial Intelligence - Precision Medicine; Omics (including genomics, proteomics, metabolomics, transcriptomics, and lipidomics); World database access; and Response and clinical outcomes. Hepion intends to use AI-POWR™ to help identify which NASH patients will best respond to rencofilstat, potentially shortening development timelines and increasing the delta between placebo and treatment groups. In addition to using AI-POWR™ to drive its ongoing NASH clinical development program, Hepion intends to use the platform to identify additional potential indications for rencofilstat to expand the company's footprint in the cyclophilin inhibition therapeutic space.

Forward-Looking Statements

Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified by the use of forward-looking words such as “anticipate,” “believe,” “forecast,” “estimated,” and “intend,” among others. These forward-looking statements are based on Hepion Pharmaceuticals’ current expectations and actual results could differ materially. There are a number of factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, substantial competition; our ability to continue as a going concern; our need for additional financing; uncertainties of patent protection and litigation; risks associated with delays, increased costs and funding shortages caused by the COVID-19 pandemic; uncertainties with respect to lengthy and expensive clinical trials, that results of earlier studies and trials may not be predictive of future trial results; uncertainties of government or third party payer reimbursement; limited sales and marketing efforts and dependence upon third parties; and risks related to failure to obtain FDA clearances or approvals and noncompliance with FDA regulations. As with any drug candidates under development, there are significant risks in the development, regulatory approval, and commercialization of new products. There are no guarantees that future clinical trials discussed in this press release will be completed or successful, or that any product will receive regulatory approval for any indication or prove to be commercially successful. Hepion Pharmaceuticals does not undertake an obligation to update or revise any forward-looking statement. Investors should read the risk factors set forth in Hepion Pharmaceuticals’ Form 10-K for the year ended December 31, 2021, and other periodic reports filed with the Securities and Exchange Commission.

For further information, please contact:

Stephen Kilmer
Hepion Pharmaceuticals Investor Relations
Direct: (646) 274-3580
skilmer@hepionpharma.com  

FAQ

What are the results of the Phase 2a study for HEPA's rencofilstat?

The Phase 2a study showed that rencofilstat met its primary endpoints related to safety and pharmacokinetics, with notable reductions in ALT, ProC3, and C6M biomarkers.

What is the purpose of the ASCEND-NASH trial for HEPA?

The ASCEND-NASH trial aims to evaluate the safety and efficacy of rencofilstat in a larger cohort of 336 biopsy-confirmed F2/F3 NASH subjects.

How does rencofilstat affect NASH biomarkers according to the study?

The study indicated that rencofilstat resulted in significant reductions of liver damage biomarker ALT and fibrosis indicators ProC3 and C6M.

What are the expectations for rencofilstat in the ongoing clinical trials?

Expectations are high for rencofilstat's antifibrotic effects to become more pronounced over the duration of the Phase 2b trial.