Amended protocol accelerates unblinding of
preliminary primary efficacy data (CR) and safety/tolerability of
the three arms at first 45 subjects
Company remains on track for first subject
treated in pivotal, adaptive Phase 3 clinical trial (the "MIRACLE"
trial) in the first quarter of 2025
Company releases Virtual Investor "What This
Means" segment discussing the amended protocol; Available
here
HOUSTON, Nov. 14,
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 amended the clinical trial protocol with the U.S.
Food and Drug Administration ("FDA") 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. Additionally, the Company released a
Virtual Investor "What This Means" segment to discuss the amended
protocol. Access the segment here.
"Our team has been thoughtful and strategic with the design of
the MIRACLE trial, which may allow for possible accelerated
approval of Annamycin in combination with cytarabine for the
treatment of relapsed or refractory AML. This amended protocol
enables us to share definitive data earlier, which helps to
partially de-risk financing the trial and potentially accelerates
the timeline for strategic partnering. We believe that the
unblinding of data at 45 subjects will enable us to begin assessing
all three arms of the study and provide us with a clear path
forward in understanding the potential of Annamycin for AML
patients. This change now puts us potentially less than 12 months
away from definitive unblinded data that could be a strong
indicator of our likelihood of approval, and the kind of data that
is likely to drive advanced partnering discussions," commented
Walter Klemp, Chairman and Chief
Executive Officer of Moleculin.
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 the
Company's end of Phase 1B/2 meeting.
The amended protocol will allow for the unblinding of preliminary
primary efficacy data (CR) and safety/tolerability of the three
arms at 45 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 45 subjects in the second
half of 2025, in addition to the planned unblinding expected in
2026 of the next 30-45 subjects.
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. 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 unblinding.
The amended protocol is currently being reviewed by the
Institutional Review Board (IRB). Once approved, the amended
protocol will be filed with the amendment for the Company's Initial
New Drug (IND) application in the US with the FDA.
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 and the timing of the release of
the unblinded data. 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.