First of many site approvals, including sites
in the US, Europe and the
Middle East, expected throughout
Q1-Q3 2025
Company remains on track for first subject
treated in pivotal, adaptive Phase 3 clinical trial (the "MIRACLE"
trial) in the first quarter of 2025
Recently amended protocol accelerates
unblinding of preliminary primary efficacy data (CR) and
safety/tolerability of the three arms at first 45 subjects,
expected in the second half of 2025
HOUSTON, Feb. 11,
2025 /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 first country regulatory approval in Europe to begin recruiting 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). In Ukraine the final necessary regulatory
approvals from the Ministry of Health were received last week. This
Phase 3 "MIRACLE" trial (derived from Moleculin
R/R AML AnnAraC Clinical Evaluation)
will be a global trial, including sites in the US, Europe and the Middle East.
"We continue to make solid progress across our site initiation
and enrollment efforts. Achieving this important milestone of
receiving our first country's approval puts us another step closer
to getting this study well underway and further bolsters our
confidence that we're on track for unblinded preliminary data from
the first 45 subjects in the second half of this year. Our team
remains committed to operational execution and face-to-face
meetings with investigators, over 30 investigators in 11 countries
of which I personally have met," commented Walter Klemp, Chairman and Chief Executive
Officer of Moleculin. "Building on this momentum, we are focused on
getting more of the initial 25 sites we have selected to date
on board and enrolling for the study."
The MIRACLE study, subject to appropriate future filings with
and potential additional feedback from the FDA and their foreign
equivalents, utilizes an adaptive design whereby the first 75 to 90
subjects will be randomized (1:1:1) 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, which Annamycin
doses were specifically recommended by the FDA in the Company's end
of Phase 1B/2 meeting. The amended
protocol allows for the unblinding of preliminary primary efficacy
data (Complete Remission or CR) and safety/tolerability of the
three arms at 45 subjects, in addition to the conclusion of Part A
(at 75 to 90 subjects). This early unblinding will yield 30
subjects with Annamycin (190mg/m2 and 230/m2) and HiDAC and 15
subjects with just HiDAC. The Company expects to reach the first
unblinding (45 subjects) in the second half of 2025, in addition to
the second unblinding, which is expected in the first half of 2026.
This accelerated estimated timeline is due to the positive response
the Company received in meetings during December with potential
investigators regarding recruitment for the trial.
For Part B of the trial, approximately 244 additional subjects
will be randomized to receive either HiDAC plus placebo or HiDAC
plus the optimum dose of Annamycin (randomized 1:1). 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. This increase from 240 to 244 subjects
represents the statistical "cost" of the additional interim
unblinding.
For more information about the MIRACLE trial, visit
clinicaltrials.gov and reference identifier NCT06788756.
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 achievements
of each of the milestones in this press release. Moleculin will
require significant additional financing, for which the Company has
no commitments, in order to conduct its clinical trials as
described in this press release, and the milestones described in
this press release assume the Company's ability to secure such
financing on a timely basis. 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.
