Company remains on track for first subject
treated in pivotal, adaptive Phase 3 clinical trial (the "MIRACLE"
trial) in the first quarter of 2025
HOUSTON, Feb. 13,
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 U.S. Food and Drug Administration ("FDA") feedback and
guidance on its IND amendment that has allowed a reduction in the
size of 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, Europe and the Middle East.
"Given the importance of our Miracle trial as a pivotal
registration clinical trial, we were extremely pleased to receive
detailed follow up from the FDA regarding our IND amendment to
enable the trial. While all of the major aspects of the trial
remain unchanged, guidance from FDA recommended an alteration to
the statistical plan that will allow us to reduce the size of Part
B of our trial by approximately 10%. Moreover, the nature of
the feedback helps us move forward quickly to open sites in the US,
in addition to the sites we are expecting to open in Europe and the Middle East. All of this supports the pursuit
of an accelerated timeline for new drug approval," commented
Walter Klemp, Chairman and Chief
Executive Officer of Moleculin.
Mr. Klemp added, "The spirit of collaboration surrounding the
Miracle trial is, frankly, inspiring. From investigating
clinicians to regulatory authorities around the world, people are
starting to realize the potential significance of approving the
first-ever non-cardiotoxic anthracycline. Annamycin's
approval in AML alone would bring the potential to save thousands
of lives every year, and the opportunity to eliminate the threat of
cardiotoxicity in a wide range of tumors equates to as much as 20
times that in long term potential. It's critical to remember
that nearly half of all cancers are treated with an
anthracycline…and 60% of all childhood cancers. One of the
things that drives us every day is our belief that no child should
ever have to be subjected to a cardiotoxic anthracycline once
Annamycin is approved."
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 220 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.
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.
