Company on track for first subject treated in
pivotal, adaptive Phase 3 clinical trial (the "MIRACLE" trial) in
the first quarter of 2025
Trial designed for possible accelerated
approval of Annamycin in combination with cytarabine for the
treatment of relapsed or refractory AML
HOUSTON, Nov. 12,
2024 /PRNewswire/ -- Moleculin Biotech, Inc.,
(Nasdaq: MBRX) ("Moleculin" or the "Company"), a late-stage
pharmaceutical company with a broad portfolio of drug candidates
targeting hard-to-treat tumors and viruses, today announced it has
received Institutional Review Board (IRB) approval for its Phase 3
pivotal trial protocol evaluating Annamycin in combination with
Cytarabine (also known as "Ara-C" and for which the combination of
Annamycin and Ara-C is referred to as "AnnAraC") for the treatment
of AML patients who are refractory to or relapsed after induction
therapy (R/R AML) (MB-108). This Phase 3 "MIRACLE" trial (derived
from Moleculin R/R AML AnnAraC
Clinical Evaluation) will be a global trial,
including sites in the US.
"IRB approval marks an important milestone towards the launch of
our MIRACLE pivotal Phase 3 trial of Annamycin in AML patients. Our
team is focused on getting clinical trial sites up and running, and
we believe we will be in a position to commence enrollment in the
first quarter of 2025," commented Walter
Klemp, Chairman and Chief Executive Officer of Moleculin.
"We have been extremely busy meeting with potential investigators
for this study and are highly encouraged by these conversations.
The positive feedback from the clinicians on the strength of our
data generated to date, along with our alignment with FDA on the
strategic design of the MIRACLE study, gives us a high level of
confidence as we take these next steps toward potentially bringing
a much needed solution to AML patients."
The MIRACLE study, subject to appropriate future filings with
and potential additional feedback from the FDA and their foreign
equivalents, is expected to initially utilize an adaptive design
whereby the first 75 to 90 subjects will be randomized in Part A of
the trial to receive high dose cytarabine (HiDAC) combined with
either placebo, 190 mg/m2 of Annamycin, or 230 mg/m2 of Annamycin,
such doses were specifically recommended by the FDA in our end of
Phase 1B/2 meeting. At that point,
the trial will be unblinded to select the optimum dose for
Annamycin. For Part B of the trial, approximately 240 additional
subjects will be randomized to receive either HiDAC plus placebo or
HiDAC plus the optimum dose of Annamycin. The selection of the
optimum dose will be based on the overall balance of safety,
pharmacokinetics and efficacy, consistent with the FDA's new
Project Optimus initiative.
Annamycin currently has Fast Track Status and Orphan Drug
Designation from the FDA for the treatment of relapsed or
refractory acute myeloid leukemia, in addition to Orphan Drug
Designation for the treatment of soft tissue sarcoma. Furthermore,
Annamycin has Orphan Drug Designation for the treatment of relapsed
or refractory acute myeloid leukemia from the European Medicines
Agency (EMA).
About Moleculin Biotech, Inc.
Moleculin Biotech, Inc. is a Phase 3 clinical stage
pharmaceutical company advancing a pipeline of therapeutic
candidates addressing hard-to-treat tumors and viruses. The
Company's lead program, Annamycin, is a next-generation
anthracycline designed to avoid multidrug resistance mechanisms and
to eliminate the cardiotoxicity common with currently prescribed
anthracyclines. Annamycin is currently in development for the
treatment of relapsed or refractory acute myeloid leukemia (AML)
and soft tissue sarcoma (STS) lung metastases.
The Company is initiating the MIRACLE (Moleculin
R/R AML AnnAraC Clinical Evaluation)
Trial (MB-108), a pivotal, adaptive design Phase 3 trial evaluating
Annamycin in combination with cytarabine, together referred to as
AnnAraC, for the treatment of relapsed or refractory acute myeloid
leukemia. Following a successful Phase 1B/2 study (MB-106), with input from the FDA, the
Company believes it has substantially de-risked the development
pathway towards a potential approval for Annamycin for the
treatment of AML. This study is subject to appropriate future
filings with potential additional feedback from the FDA and their
foreign equivalents.
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. Moleculin is also engaged in the development of a
portfolio of antimetabolites, including WP1122 for the potential
treatment of pathogenic viruses, as well as certain cancer
indications.
For more information about the Company, please visit
www.moleculin.com and connect on X, 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 enrollment of the MIRACLE 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
(908) 824-0775
MBRX@jtcir.com
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SOURCE Moleculin Biotech, Inc.