– Next-generation
non-cardiotoxic anthracycline Annamycin with notable signs of
clinical efficacy in AML in combination with Cytarabine achieves
preliminary CRc rate of 60% in 2nd line AML
subjects in a European clinical trial
– There are
approximately 160,000 people with AML worldwide
– Annamycin
continues to show no signs of cardiotoxicity (N=82 across multiple
studies); Lower toxicity profile than traditional intensive
therapy
– Annamycin is
advancing towards pivotal AML study in 2024 and may qualify for an
accelerated approval pathway
HOUSTON, April 18,
2024 /PRNewswire/ -- Moleculin Biotech,
Inc., (Nasdaq: MBRX) (Moleculin or the Company), a
clinical stage pharmaceutical company with a broad portfolio of
drug candidates targeting hard-to-treat tumors and viruses, today
announced that the European Medicines Agency (EMA) has granted
Orphan Drug Designation to Annamycin for the treatment
of AML.
Annamycin is the Company's next-generation anthracycline that
has been designed to be non-cardiotoxic (unlike currently
prescribed anthracyclines) and has shown to be non-cardiotoxic in
the 82 subjects treated in multiple studies in the U.S. and in
Europe. Furthermore, Annamycin has
recently shown in Moleculin's European clinical study for the
treatment of AML using Annamycin in combination with Cytarabine
(MB-106) a preliminary 60% composite complete response (CRc) rate
in 2nd line subjects (N=10) and an overall interim CRc
of 39% in all subjects (N=18), regardless of the prior number of
therapies, in the European trial. Durability of the CRc's is
developing. One subject has surpassed the one-year mark with a
durable complete response. Recruitment in MB-106 has ended for
2nd line subjects while recruitment for 1st
line and 3rd line subjects continue. Annamycin is
currently in development for the treatment for AML and STS lung
mets, and the Company believes the drug may have the potential to
treat additional indications.
"We are pleased to receive Orphan Drug Designation from the EMA
for Annamycin, which further supports our ongoing efforts to
advance this compelling next-generation anthracycline for the
treatment of hard-to-treat cancers," commented Walter Klemp, Chairman and Chief Executive
Officer of Moleculin. "Combined with the Orphan Drug Designation we
already have in the US and with the new composition of matter
patent just awarded by the US Patent and Trademark Office with
coverage through 2040, we believe the commercial exclusivity of
Annamycin is now very well protected. We continue to be encouraged
by the growing body of promising interim clinical data demonstrated
by Annamycin. We remain focused on advancing our clinical and
regulatory strategies toward our next phase of development,
including the planned commencement of a pivotal registration study
as a 2nd line therapy in AML before year end."
The EMA grants orphan drug designation to drugs and biologics
intended for the treatment, diagnosis or prevention of rare,
life-threatening or chronically debilitating diseases or conditions
that affect fewer than five in 10,000 people in the European Union.
Orphan designation allows companies certain benefits, including
reduced regulatory fees, clinical protocol assistance, research
grants and up to 10 years of potential market exclusivity in the
European Union if approved.
Annamycin currently has Fast Track Status and Orphan Drug
Designation from the U.S. Food and Drug Administration for the
treatment of relapsed or refractory AML, in addition to Orphan Drug
Designation for the treatment of STS lung mets.
About Moleculin Biotech, Inc.
Moleculin Biotech, Inc. is a clinical stage pharmaceutical
company with a growing pipeline, including Phase 2 clinical
programs, for hard-to-treat 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. All interim and
preliminary data discussed above is subject to change.
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 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 timing of the commencement
of a pivotal registration study of Annamycin as a
2nd line therapy in AML before year end. 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
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SOURCE Moleculin Biotech, Inc.