HOUSTON, April 14, 2021 /PRNewswire/ -- Moleculin
Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a
clinical stage pharmaceutical company with a broad portfolio of
drug candidates targeting highly resistant tumors and viruses,
announced today that the U.S. Food and Drug Administration (FDA)
has granted Rare Pediatric Disease Designation (RPD) to its p-STAT3
inhibitor, WP1066, for the treatment of ependymoma.
Ependymoma is a rare type of tumor that can form in the brain or
spinal cord. Ependymoma begins in the ependymal cells in the brain
and spinal cord that line the passageways where the fluid
(cerebrospinal fluid) that nourishes your brain flows. Ependymoma
can occur at any age, but most often occurs in young children.
"The FDA's recognition of the high prevalence and unmet needs in
the treatment landscape for ependymoma, especially in pediatric
patients is a significant milestone as we continue to advance and
expand the WP1066 development program. We currently have Orphan
Drug Designation for WP1066 for the treatment of brain tumors, as
well as RPD designation for three other pediatric indications, and
believe that ependymoma represents another important rare
indication. We continue to be encouraged by the data WP1066 has
demonstrated to date and believe it has the potential to be an
effective therapy for pediatric patients with ependymoma,"
commented Walter Klemp, Chairman and
CEO of Moleculin.
The Rare Pediatric Disease Priority Review Voucher program,
which was created as part of the Food and Drug Administration
Safety and Innovation Act (FDASIA) of 2012, is intended to
incentivize the development of new therapies for rare pediatric
diseases. Under the FDA's rare pediatric disease designation
program, the FDA may grant a priority review voucher to a sponsor
who receives a product approval for a "rare pediatric disease,"
which is defined as a serious or life-threatening disease in which
the serious or life-threatening manifestations primarily affect
individuals aged from birth to 18 years and affects fewer than
200,000 people in the U.S. Subject to FDA approval of WP1066 for
the treatment of ependymoma, Moleculin would be eligible to receive
a voucher that may be redeemed to receive priority review for a
subsequent marketing application for a different product candidate
or which could be sold or transferred.
About WP1066
WP1066 is an immune/transcription modulator capable of directly
inhibiting certain key oncogenic transcription factors, including
the activated form of a protein known as STAT3. The activated form
of STAT3 referred to as p-STAT3, is considered a master regulator
of tumor activity. In addition to inhibiting p-STAT3 and several
other signaling proteins linked to tumor development, WP1066 has
also been shown in animal models to stimulate a natural immune
response and immune memory to fight tumor progression.
About Moleculin Biotech, Inc.
Moleculin Biotech, Inc. is a clinical stage pharmaceutical
company focused on the development of a broad portfolio of oncology
drug candidates for the treatment of highly resistant tumors and
viruses. The Company's clinical stage drugs are: Annamycin, a
next-generation anthracycline, designed to avoid multidrug
resistance mechanisms with little to no cardiotoxicity being
studied for the treatment of relapsed or refractory acute myeloid
leukemia, more commonly referred to as AML, WP1066, an
Immune/Transcription Modulator capable of inhibiting p-STAT3 and
other oncogenic transcription factors while also stimulating a
natural immune response, targeting brain tumors, pancreatic cancer
and hematologic malignancies, and WP1220, an analog to WP1066, for
the topical treatment of cutaneous T-cell lymphoma. Moleculin is
also engaged in preclinical development of additional drug
candidates, including other Immune/Transcription Modulators, as
well as WP1122 and related compounds capable of
Metabolism/Glycosylation Inhibition.
For more information about the Company, please visit
http://www.moleculin.com.
Forward-Looking Statements
Some of the statements in this release are forward-looking
statements within the meaning of Section 27A of the Securities Act
of 1933, Section 21E of the Securities Exchange Act of 1934 and the
Private Securities Litigation Reform Act of 1995, which involve
risks and uncertainties. Forward-looking statements in this press
release include, without limitation, the ability of WP1066 to show
safety and efficacy in ependymoma patients. Although
Moleculin believes that the expectations reflected in such
forward-looking statements are reasonable as of the date made,
expectations may prove to have been materially different from the
results expressed or implied by such forward-looking statements.
Moleculin has attempted to identify forward-looking statements by
terminology including ''believes,'' ''estimates,'' ''anticipates,''
''expects,'' ''plans,'' ''projects,'' ''intends,'' ''potential,''
''may,'' ''could,'' ''might,'' ''will,'' ''should,''
''approximately'' or other words that convey uncertainty of future
events or outcomes to identify these forward-looking statements.
These statements are only predictions and involve known and unknown
risks, uncertainties, and other factors, including those discussed
under Item 1A. "Risk Factors" in our most recently filed Form 10-K
filed with the Securities and Exchange Commission ("SEC") and
updated from time to time in our Form 10-Q filings and in our other
public filings with the SEC. Any forward-looking statements
contained in this release speak only as of its date. We undertake
no obligation to update any forward-looking statements contained in
this release to reflect events or circumstances occurring after its
date or to reflect the occurrence of unanticipated events.
Investor Contact:
JTC Team, LLC
Jenene Thomas
(833) 475-8247
MBRX@jtcir.com
View original content to download
multimedia:http://www.prnewswire.com/news-releases/moleculin-awarded-new-rare-pediatric-disease-designation-from-us-fda-for-wp1066-for-the-treatment-of-ependymoma-301268294.html
SOURCE Moleculin Biotech, Inc.