Genkyotex’s Setanaxib Granted Orphan Drug Designation by The US FDA For the Treatment of PBC
20 Ottobre 2020 - 6:00PM
Business Wire
Regulatory News:
Genkyotex (Paris:GKTX) (Brussels:GKTX) (Euronext Paris
& Brussels: FR0013399474 – GKTX), a biopharmaceutical
company and leader in NOX therapies, today announced that its lead
drug candidate, setanaxib, has been granted orphan drug designation
(ODD) by the US Food and Drug Administration (FDA) for the
treatment of primary biliary cholangitis (PBC).
Elias Papatheodorou, CEO of Genkyotex, comments:
“Obtaining the ODD from the FDA is of strategic importance for the
development of setanaxib in PBC. The FDA and the European Medicines
Agency (EMA) have also granted ODD for setanaxib in the treatment
of idiopathic pulmonary fibrosis and systemic sclerosis,
highlighting the compound’s therapeutic potential in multiple
fibrotic disorders with high unmet needs.”
Orphan Drug Designation is granted by the FDA to drugs or
biological products intended for the safe and effective treatment
of rare diseases with an unmet medical need, affecting fewer than
200,000 people in the United States. Among other benefits beyond
the clinical development phases of the product, ODD provides 7
years of market exclusivity after obtaining marketing approval in
the United States. In addition, in certain cases, it may provide
tax credits on clinical research expenses, an accelerated
registration procedure, technical assistance to complete the
registration file as well as exemptions from filing fees with the
regulatory agencies.
The company is currently discussing its registration strategy
for setanaxib in PBC with the FDA and the EMA. Genkyotex will
provide an outline of its late stage development plan once final
approval of a common registration strategy has been obtained from
these regulatory agencies.
Next financial press release: Q3 2020 business update and
cash position: October 22, 2020 (after market)
About Genkyotex Genkyotex is the leading
biopharmaceutical company in NOX therapies, listed on the Euronext
Paris and Euronext Brussels markets. Its unique platform enables
the identification of orally available small-molecules which
selectively inhibit specific NOX enzymes that amplify multiple
disease processes such as fibrosis, inflammation, pain processing,
cancer development, and neurodegeneration. Genkyotex is developing
a pipeline of first-in-class product candidates targeting one or
multiple NOX enzymes. The lead product candidate, setanaxib
(GKT831), a NOX1 and NOX4 inhibitor has shown evidence of
anti-fibrotic activity in a Phase II clinical trial in primary
biliary cholangitis (PBC, a fibrotic orphan disease). Based on its
positive Phase II results, a phase 3 trial with setanaxib in PBC is
being planned. Setanaxib is also being evaluated in an
investigator-initiated Phase II clinical trial in Type 1 Diabetes
and Kidney Disease (DKD). A grant from the United States National
Institutes of Health (NIH) of $8.9 million was awarded to Professor
Victor Thannickal at the University of Alabama at Birmingham (UAB)
to fund a multi-year research program evaluating the role of NOX
enzymes in idiopathic pulmonary fibrosis (IPF), a chronic lung
disease that results in fibrosis of the lungs. The core component
of this program is a Phase 2 trial with setanaxib in patients
suffering from IPF for which the first patient has been enrolled in
September 2020. This product candidate may also be active in other
fibrotic indications.
Genkyotex also has a versatile platform well-suited to the
development of various immunotherapies (Vaxiclase). A partnership
covering the use of Vaxiclase as an antigen per se (GTL003) has
been established with Serum Institute of India Private Ltd (Serum
Institute), the world’s largest producer of vaccine doses, for the
development by Serum Institute of cellular multivalent combination
vaccines against a variety of infectious diseases. For further
information, please go to www.genkyotex.com
Disclaimer This press release may contain forward-looking
statements by the company with respect to its objectives. Such
statements are based upon the current beliefs, estimates and
expectations of Genkyotex’s management and are subject to risks and
uncertainties such as the company's ability to implement its chosen
strategy, customer market trends, changes in technologies and in
the company's competitive environment, changes in regulations,
clinical or industrial risks and all risks linked to the company's
growth. These factors as well as other risks and uncertainties may
prevent the company from achieving the objectives outlined in the
press release and actual results may differ from those set forth in
the forward-looking statements, due to various factors. Without
being exhaustive, such factors include uncertainties involved in
the development of Genkyotex’s products, which may not succeed, or
in the delivery of Genkyotex’s products marketing authorizations by
the relevant regulatory authorities and, in general, any factor
that could affects Genkyotex’s capacity to commercialize the
products it develops. No guarantee is given on forward-looking
statements which are subject to a number of risks, notably those
described in the universal registration document filed with the AMF
on April 30, 2020 under number D.20-0434, and those linked to
changes in economic conditions, the financial markets, or the
markets on which Genkyotex is present. Genkyotex products are
currently used for clinical trials only and are not otherwise
available for distribution or sale.
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GENKYOTEX Alexandre Grassin CFO Tel.: +33 (0)5 61 28 70
60 investors@genkyotex.com
NewCap Dušan Orešanský Tel.: +33 1 44 71 94 92
genkyotex@newcap.eu