Hepion Pharmaceuticals Receives FDA Orphan Drug Status Designation for Rencofilstat for the Treatment of Hepatocellular Carcinoma

Rhea-AI Summary

Hepion Pharmaceuticals (NASDAQ:HEPA) announced FDA's Orphan Drug Designation for its drug rencofilstat, aimed at treating hepatocellular carcinoma (HCC). This designation highlights its potential to address significant unmet medical needs in an aggressive cancer type, which accounts for 85-90% of liver cancer cases. Hepion plans to initiate a Phase 2a study of rencofilstat in HCC in Q3 2022, alongside ongoing Phase 2 studies for non-alcoholic steatohepatitis (NASH). The Orphan Drug Designation offers various incentives, including potential seven-year market exclusivity and tax credits for clinical trials.

Positive

• FDA granted Orphan Drug Designation for rencofilstat for HCC, enhancing its market potential.
• Rencofilstat targets a significant unmet medical need in HCC, a major cause of mortality.
• Initiation of Phase 2a study in HCC expected in Q3 2022, indicating continued development progress.

Negative

• None.

EDISON, N.J., June 20, 2022 (GLOBE NEWSWIRE) -- Hepion Pharmaceuticals, Inc. (NASDAQ:HEPA), a clinical mid-stage biopharmaceutical company focused on Artificial Intelligence (“AI”)-driven therapeutic drug development for the treatment of non-alcoholic steatohepatitis (“NASH”) and hepatocellular carcinoma (“HCC”), today announced that the U.S. Food and Drug Administration (“FDA”) has granted Orphan Drug Designation to rencofilstat, a liver-targeting, orally administered, novel cyclophilin inhibitor, for the treatment of HCC.

HCC is the most common form of liver cancer, accounting for 85-90% of all cases. NASH, viral hepatitis infection, and alcohol consumption all are major causes of HCC. Worldwide, it is estimated that over 800,000 people died from liver cancer in 2020, second only to lung cancer among all cancer-related deaths.¹ The high mortality is largely due to the fact that only around half of all people who develop HCC (in developed countries) receive the diagnosis early enough to have an opportunity for therapeutic intervention. Additionally, recurrence rates are high, and effective treatment options remain limited.

“Orphan Drug Designation for rencofilstat in HCC represents a significant milestone for Hepion and its recognition by the FDA of the potential for rencofilstat to address a significant unmet medical need for patients suffering from this aggressive cancer,” said Robert Foster, PharmD, PhD, Hepion’s CEO. “In addition to two Phase 2 studies in patients with NASH, we remain on track to initiate patient enrollment in a Phase 2a study of rencofilstat in HCC in the third quarter of 2022.”

The FDA's Orphan Drug Designation program provides orphan status to drugs or biologics intended for the prevention, diagnosis, or treatment of diseases that affect fewer than 200,000 people in the United States. Sponsors of medicines that are granted Orphan Drug Designation are entitled to certain incentives, including tax credits for qualified clinical trials, prescription drug user-fee exemptions, and potential seven-year marketing exclusivity upon FDA approval.

Reference

¹ GLOBOCAN 2020

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 soon followed in December 2021 by the FDA’s acceptance of Hepion’s investigational new drug (IND) application for rencofilstat for the treatment of hepatocellular carcinoma (HCC). In June 2022, rencofilstat was granted Orphan Drug designation 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 is the significance of the FDA's Orphan Drug Designation for HEPA?

The Orphan Drug Designation for rencofilstat represents recognition by the FDA of its potential to meet significant medical needs for HCC patients, which may enhance the drug's marketability and provide financial incentives.

When will Hepion start the Phase 2a study for rencofilstat in HCC?

Hepion Pharmaceuticals plans to initiate patient enrollment in the Phase 2a study of rencofilstat for HCC in the third quarter of 2022.

How does rencofilstat aim to treat hepatocellular carcinoma (HCC)?

Rencofilstat is a liver-targeting, orally administered cyclophilin inhibitor designed to address challenges in treating HCC, which includes limitations in existing treatment options.