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

Hepion Pharmaceuticals, Inc. recently announced the US 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. 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 US. 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.

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 US 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.