- Glioblastoma is the most aggressive
malignant primary brain tumor with serious unmet medical
need
HOUSTON, Sept. 6,
2022 /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, today announced that
the U.S. Food and Drug Administration ("FDA") has granted Orphan
Drug Designation of WP1122 for the treatment of Glioblastoma
Multiforme ("GBM").
"The receipt of Orphan Drug Designation represents an important
milestone for our promising WP1122 development program,"
commented Walter Klemp, Chairman and
Chief Executive Officer of Moleculin. "Given the progress of our
Phase 1 clinical trial in healthy volunteers, the strong
preclinical data supporting GBM as one of many potential
indications and the recent clearance by the FDA of IND status for
WP1122 in GBM, we believe this designation further supports the
potential of WP1122 and is another step forward in further
validating our deep pipeline."
WP1122 was developed as a 2-DG prodrug to provide a more
favorable pharmacological profile and was found to have greater
potency than 2-DG alone in preclinical models where tumor cells
require higher glycolytic activity than normal cells. Based on
preclinical data indicating the potential for WP1122 as a treatment
for GBM, Moleculin received Investigational New Drug status and is
evaluating opportunities for collaboration in clinical
development.
GBM is the most aggressive malignant primary brain tumor and
remains as an incurable tumor with a median survival of only 15
months.1 It is the most common malignant primary brain
tumor making up 54% of all gliomas and 16% of all primary brain
tumors,2 and despite advancements, survival rates for
patients with GBM have shown no notable improvement in population
statistics in the last three decades.3 The average
annual age-adjusted incidence rate of GBM is 3.19 per 100,000
persons in the United States.
The FDA grants Orphan Drug Designation to drugs and biologics
intended for the treatment, diagnosis or prevention of rare
diseases or conditions affecting fewer than 200,000 people in
the United States. Orphan Drug
Designation provides Moleculin certain benefits, including
financial incentives to support clinical development and the
potential for up to seven years of market exclusivity for the drug
for the designated orphan indication in the U.S. if the drug is
ultimately approved for its designated indication.
About Moleculin Biotech, Inc.
Moleculin Biotech, Inc. is a clinical stage pharmaceutical
company focused on the development of a broad portfolio of drug
candidates for the treatment of highly resistant tumors and
viruses. The Company's lead program, Annamycin, is a
next-generation anthracycline designed to avoid multidrug
resistance mechanisms with little to no cardiotoxicity. Annamycin
is currently in development for the treatment of relapsed or
refractory acute myeloid leukemia (AML) and soft tissue sarcoma
(STS) lung metastases.
Additionally, the Company is developing WP1066, an
Immune/Transcription Modulator designed to be capable of inhibiting
p-STAT3 and other oncogenic transcription factors while also
stimulating a natural immune response, targeting brain tumors,
pancreatic and other cancers; and WP1220, an analog to WP1066, for
the topical treatment of cutaneous T-cell lymphoma. Moleculin is
also engaged in the development of a portfolio of antimetabolites,
including WP1122, for the potential treatment of COVID-19 and other
viruses, as well as cancer indications including brain tumors,
pancreatic and other cancers.
For more information about the Company, please visit
www.moleculin.com and connect on Twitter, LinkedIn and
Facebook.
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 for WP1122 to be
shown safe and effective in humans. 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
1: Koshy M, Villano JL, Dolecek TA, Howard A, Mahmood U, Chmura
SJ, et al. Improved survival time trends of glioblastoma using the
SEER 17 population-based registries. J Neuro Oncol.
2012;107(1):207-12
2: Ostrom QT, Gittleman H, Farah P, Ondracek A, Chen Y, Wolinsky Y,
et al. CBTRUS statistical report: Primary brain and central nervous
system tumors diagnosed in the United
States in 2006-2010. Neuro Oncol. 2013;15 Suppl:2ii-56.
3: De Vleeschouwer S, editor. Brisbane (AU): Codon Publications; 2017 Sep
27.
4: Thakkar J, Dolecek TA, Horbinski C, Ostrom QT, Lightner DD,
Barnholtz-Sloan JS, et al. Epidemiologic and molecular prognostic
review of glioblastoma. Cancer Epidemiol. Biomarkers Rev.
2014;23(10):1985-96.
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SOURCE Moleculin Biotech, Inc.