Rencofilstat

Hepion Pharmaceuticals’ shares lost a third of their value after the company decided to halt its mid-stage programme for rencofilstat in metabolic dysfunction-associated steatohepatitis (MASH) due to financial constraints. The drugmaker will seek strategic alternatives to pursue further development of the cyclophilin inhibitor.The Phase IIb ASCEND-NASH study kicked off in August 2022 and planned to investigate the safety and efficacy of rencofilstat versus placebo over 12 months in 336 participants. However, enrolment was stopped in April last year after 150 people were randomised. Hepion noted that nearly 80 participants had currently completed the one-year follow-up, with an additional 40 lined up for safety assessments.“Given the number of enrolled [MASH] patients to date and the low probability of generating relevant efficacy data to support a registrational trial with our current cash resources, we have opted to wind down the programme,” explained Hepion’s executive chairman John Brancaccio. He confirmed there were no safety issues associated with the drug in the study.Hepion had earlier announced a restructuring plan to preserve capital by slashing operating costs by approximately 60%, thus extending its cash runway into the second quarter of 2025. Several players, including big pharmas such as Eli Lilly and Novo Nordisk, are battling it out in the competitive MASH arena. However, the MASH debutant crown was snatched by Madrigal after clinching FDA approval for Rezdiffra (resmetirom) last month (for related analysis, read KOL Views Q&A: Leading endocrinologist lays out challenges ahead as Madrigal launches Rezdiffra in MASH).

EDISON, N.J., April 19, 2024 (GLOBE NEWSWIRE) -- Hepion Pharmaceuticals, Inc. (NASDAQ:HEPA), a clinical stage biopharmaceutical company that has been developing a treatment for non-alcoholic steatohepatitis (“NASH”), hepatocellular carcinoma (“HCC”), and other chronic liver diseases, today announced that it has begun wind-down activities in its ASCEND-NASH Trial, while continuing to explore strategic alternatives, as previously announced in December 2023. ASCEND-NASH is a Phase 2b, randomized, multi-center, double-blinded study with first patient screened in August 2022, to evaluate the safety and efficacy of rencofilstat dosed for 12 months, with a target enrollment of 336 subjects. Enrollment was paused in April 2023, with 151 subjects randomized. To date, approximately 80 subjects have completed their Day 365 visits and are evaluable for both safety and efficacy. An additional 40 subjects will provide significant safety data for evaluation. These patients will be added to our existing safety database. “We are disappointed to announce the wind-down of our Phase 2 NASH trial, which we attribute entirely to resource constraints,” said John Brancaccio, Executive Chairman of Hepion. “The Company remains committed to its strategic alternative efforts previously announced in December 2023. Given the number of enrolled NASH patients to date and the low probability of generating relevant efficacy data to support a registrational trial with our current cash resources, we have opted to wind down the program and assure that patients are transitioned out of the trial in a safe and compliant manner. There were no safety concerns observed for rencofilstat in the ASCEND-NASH trial.” About Hepion Pharmaceuticals Rencofilstat, is a potent inhibitor of cyclophilins, which are involved in many disease processes. Rencofilstat has been shown to reduce liver fibrosis and hepatocellular carcinoma tumor burden in experimental disease models. 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 and maintains a proprietary Artificial Intelligence deep machine learning (“AI/ML”) platform designed to better understand disease processes and identify patients that are rencofilstat responders. This AI/ML has the potential to shorten development timelines and increase the observable differences between placebo and treatment groups. In addition, Hepion’s AI/ML can be used to further NASH and HCC clinical development programs and identify other potential therapeutic indications for cyclophilin inhibition with rencofilstat. 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, 2023, and other periodic reports filed with the Securities and Exchange Commission. For further information, please contact: Hepion Pharmaceuticals 732-902-4000 info@hepionpharma.com