The European Commission Grants Orphan Drug Designation to Temferon™ for Treatment of Glioma
June 29 2023 - 7:00AM
Genenta Science (NASDAQ: GNTA), a clinical-stage immuno-oncology
company developing a cell-based platform harnessing the power of
hematopoietic stem cells to provide durable and safe treatments for
solid tumors, today announced that the
European
Commission has granted Orphan Drug Designation (ODD) to
Temferon™ for the treatment of
glioma. Glioblastoma Multiforme (GBM) is the first
clinical indication of Temferon.
ODD is granted by the European Commission for
medicines in development to treat rare conditions affecting no more
than five in 10,000 people in the European Union (EU), provided
there is no other satisfactory treatment option or the medicine can
be of significant benefit to those affected by the condition.
Sponsors of with ODD designation can benefit from a number
of incentives in the EU; for example, ODD medicines
benefit from ten years of market exclusivity once they receive a
marketing authorization in the EU.
“European Medicine Agency’s Committee reviewed
Genenta's ODD application for Temferon and agreed on the potential
significant benefit that Temferon could contribute to patients
suffering from GBM if approved. The ODD designation supports and
facilitates the development of our cell therapy-based technology
platform for solid tumors,” said Pierluigi
Paracchi, Chief Executive Officer at
Genenta. “The EMA ODD designation follows the orphan drug
designation granted by the US Food and Drug
Administration to Temferon for the treatment of GBM in
March 2023. The preliminary interim results of Genenta’s ongoing
phase 1/2a trials in newly diagnosed patients with unmethylated
MGMT gene promoter reviewed by EMA included an Overall Survival at
two years, which is longer than the median Overall Survival
described in published reports,” continued Pierluigi
Paracchi.
GBM is the most common malignant primary brain
tumor and the most aggressive diffuse glioma, with unmethylated
MGMT promoter status identified in approximately 60% of the GBM
population.
Temferon is Genenta's product at the most
advanced stage of development and consists of the patient's own
stem progenitor cells modified with Genenta's platform to express
interferon alpha (IFNa) within solid tumors. IFNa is a well-known
immunomodulatory protein that has been used in the clinic for
decades for the treatment of a variety of cancers, but with limited
current use because of the systemic toxicity. Genenta's platform is
designed to avoid systemic toxicity and selectively deliver
therapeutic activity within the solid tumor. From pre-clinical
experiments, it has been observed that Temferon breaks
tumor-induced tolerance, thus allowing the immune system
to recognize the tumor and mount a durable immune
response.
About Genenta and
TemferonGenenta (www.genenta.com) is a clinical-stage
biotechnology company engaged in the development of a proprietary
hematopoietic stem cell therapy for the treatment of a variety of
solid tumor cancers. Temferon™ is based on ex-vivo gene transfer
into autologous Tie2+ hematopoietic stem/progenitor cells (HSPCs)
to deliver immunomodulatory molecules directly via
tumor-infiltrating monocytes/macrophages (Tie2 Expressing Monocytes
- TEMs). Temferon, which is under investigation in a phase 1/2a
clinical trial in newly diagnosed Glioblastoma Multiforme patients
who have an unmethylated MGMT gene promoter (uMGMT-GBM), is
designed to reach solid tumors, induce a durable immune response
not restricted to pre-selected tumor antigens nor type, and avoid
systemic toxicity, which are some of the main unresolved challenges
in immuno-oncology.
Forward-Looking
StatementsStatements in this press release contain
“forward-looking statements,” within the meaning of the U.S.
Private Securities Litigation Reform Act of 1995, that are subject
to substantial risks and uncertainties. All statements, other than
statements of historical fact, contained in this press release are
forward-looking statements. Forward-looking statements contained in
this press release may be identified by the use of words such as
“anticipate,” “believe,” “contemplate,” “could,” “estimate,”
“expect,” “intend,” “seek,” “may,” “might,” “plan,” “potential,”
“predict,” “project,” “suggest,” “target,” “aim,” “should,” "will,”
“would,” or the negative of these words or other similar
expressions, although not all forward-looking statements contain
these words. Forward-looking statements are based on Genenta’s
current expectations and are subject to inherent uncertainties,
risks and assumptions that are difficult to predict, including
risks related to the completion and timing of the phase 1/2a
clinical trial or any studies relating to the treatment of
glioblastoma multiforme patients who have an unmethylated MGMT gene
promoter (uMGMT-GBM). Further, certain forward-looking statements
are based on assumptions as to future events that may not prove to
be accurate. These and other risks and uncertainties are described
more fully in the section titled “Risk Factors” in Genenta's Annual
Report on Form 20-F for the year ended December 31, 2022 filed with
the Securities and Exchange Commission. Forward-looking statements
contained in this announcement are made as of the date of this
announcement, and Genenta undertakes no duty to update such
information except as required under applicable law.
Genenta MediaTiziana Pollio+39 348 23 15
143tiziana.pollio@genenta.com
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