Hepion Pharmaceuticals Receives FDA Orphan Drug Status Designation for Rencofilstat for the Treatment of Hepatocellular Carcinoma
June 20 2022 - 7:00AM
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 KilmerHepion Pharmaceuticals Investor RelationsDirect:
(646) 274-3580skilmer@hepionpharma.com
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