Investigational New Drug (IND) application
clearance from FDA opens up potential for additional investigator
funded and led clinical studies
HOUSTON,
April 21,
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 US Food and Drug Administration (FDA) is allowing the Company's
Investigational New Drug (IND) application to study WP1066 for the
treatment of recurrent malignant glioma. With this IND now cleared,
Moleculin plans to evaluate strategic partnerships and
collaborations to conduct a Phase 1 open label, single arm, dose
escalation study of the safety, pharmacokinetics and efficacy of
oral WP1066 in adult patients with recurrent malignant glioma.
WP1066 is the Company's flagship
Immune/Transcription Modulator designed to stimulate the immune
response to tumors by inhibiting the errant activity of Regulatory
T-Cells (TRegs) while also inhibiting key oncogenic transcription
factors, including p-STAT3 (phosphorylated signal transducer and
activator of transcription 3), c-Myc (a cellular signal transducer
named after a homologous avian virus called Myelocytomatosis) and
HIF-1α (hypoxia inducible factor 1α). These transcription factors
are widely sought targets that are believed to contribute to an
increase in cell survival and proliferation, and the angiogenesis
(coopting vasculature for blood supply), invasion, metastasis and
inflammation associated with tumors. They may also play a role in
the inability of immune checkpoint inhibitors to affect more
resistant tumors.
"WP1066 has demonstrated significant anti-tumor
activity in a wide range of tumor cell lines and increased survival
in a number of animal models to-date. Additionally, the preliminary
results demonstrated in the ongoing trial of WP1066 for pediatric
brain tumors bolster our confidence and this IND clearance provides
further momentum for its continued research and development,"
commented Walter Klemp, Chairman and
CEO of Moleculin. "We expect the clearance of this IND to further
support the ongoing pediatric studies being conducted by the team
at Emory University, and we are
evaluating the potential for additional externally funded
investigator-initiated studies."
WP1066 is currently being evaluated in
collaboration with Emory University for
the treatment of pediatric brain tumors, including Diffuse
Interstitial Pontine Glioma (DIPG). The Emory trial for pediatric
brain tumors has now treated three subjects in the first two
cohorts of the Phase 1 dose escalation portion of
physician-sponsored clinical trial. In that trial, one of the
subjects in the first cohort with DIPG showed an apparent response
to the treatment with both clinical improvement and radiologic
reduction of tumor size. The Company cautions that this is
preliminary data, and no conclusions should be drawn from this
single event. One subject has been treated in the third cohort at
the dose level of 8mg/kg. Two more subjects will be treated at this
dose level. Emory University has
amended its protocol to allow dosing at 16 mg/kg after these two
additional subjects have been dosed, and the third cohort dosing
has been deemed safe.
The Company has received Orphan Drug Designation
for WP1066 for the treatment of brain tumors, as well as Rare
Pediatric Disease designation for three other pediatric
indications. Additionally, WP1066 + radiation is being evaluated,
pre-clinically, in the treatment of Glioblastoma Multiforme
(GBM).
Glioma is a common type of tumor originating in
the brain. About 33% of all brain tumors are gliomas, which
originate in the glial cells that surround and support neurons in
the brain, including astrocytes, oligodendrocytes and ependymal
cells. 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.[4]
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 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.
______________________________
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.
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 release include, without limitation, Moleculin's ability to
secure a strategic partnership or collaboration to conduct a Phase
1 trial. 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:https://www.prnewswire.com/news-releases/moleculin-receives-ind-clearance-to-conduct-phase-1-study-of-wp1066-for-the-treatment-of-recurrent-malignant-glioma-301530189.html
SOURCE Moleculin Biotech, Inc.