Preclinical data accepted for online
publication at ASH Annual Meeting reveal significant activity of
Annamycin in Venetoclax resistant AML model
New preliminary clinical results show
Annamycin plus Ara-C achieved 60% CR/CRi in subjects who were
relapsed from or refractory to Venetoclax regimens; more than 4
times greater than published historical rates
Annamycin demonstrates an even greater
potential than previously reported to address a significant AML
patient population for which treatment options are extremely
limited
New data from MB-106 trial show median overall
survival of 11.6 months in subjects receiving AnnAraC as 2nd line
therapy
HOUSTON, Nov. 18,
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 new
findings supporting the ability of Annamycin to overcome resistance
to Venetoclax in acute myeloid leukemia ("AML"). This
includes data from preclinical in vitro studies recently accepted
for online publication at the upcoming American Society of
Hematology ("ASH") Annual Meeting, and correlates with efficacy
demonstrated by recent preliminary clinical data in subjects who
were relapsed from or refractory to first line Venetoclax regimens
and were then treated with Annamycin in combination with Ara-C
("AnnAraC").
Jorge Cortes, MD, Director of the
Georgia Cancer Center at Augusta University and a member of the
Company's Scientific Advisory Board, commented, "Although
Venetoclax has been an important improvement in first line therapy
for AML patients who are unfit for intensive chemotherapy, the rate
of relapse is high and overall survival post relapse is just a few
months. This turns out to be a large percentage of AML patients in
total and we clearly need a better treatment option."
Michael Andreeff, MD, PhD,
Professor of Medicine, Department of Leukemia, Division of Cancer
Medicine, The University of Texas MD
Anderson Cancer Center and a member of the Company's Scientific
Advisory Board, added, "Many patients get Ven-Aza, not because they
are 'unfit' but because of the high initial response rates. When
they relapse, however, our options are very limited. Annamycin
combined with Ara-C could significantly advance the standard of
care and provide better outcomes for these high-risk patients. I am
excited to be a part of the next step in the development of this
important asset."
A prior publication, Outcomes of relapsed or refractory acute
myeloid leukemia after frontline hypomethylating agent and
venetoclax regimens1, reported that the
CR/CRi2 rate for salvage therapy using available
standard of care in AML subjects who relapsed from or were
refractory to Venetoclax regimens was 12.5% (n=24, 4% CR). The new
preliminary clinical findings announced today in the MB-106
clinical trial indicate that relapsed or refractory ("R/R") AML
subjects previously treated with Venetoclax regimens achieved a 60%
CR/CRi rate (n=5, 40% CR), more than 4 times the rate that would be
expected based on the above referenced paper. As previously
disclosed in MB-106, there was an overall CR/CRi rate of 60% (50%
CR) in 2nd line subjects (n=10) and 41% (36% CR) in all
subjects, 1st-7th line (n=22).
An abstract titled, "Annamycin, a non-cardiotoxic
anthracycline, demonstrates unique organotropism and activity
against Ara-C and Venetoclax resistant AML," supporting the
clinical activity of Annamycin was accepted for online publication
at the ASH Annual Meeting being held December 7-10, 2024, in San Diego, CA. The abstract will be published
in a supplemental issue of Blood, expected in late
November, and will become part of the permanent ASH and
Blood abstracts archive following the conclusion of the
Annual Meeting.
Additionally, new preliminary data from the Company's Phase
1B/2 clinical trial evaluating
AnnAraC for the treatment of subjects with AML as both first line
therapy and for subjects who were refractory to or relapsed after
induction therapy (MB-106) demonstrated median overall survival
("OS") of 9.1 months for subjects with a wide range of (0-6) prior
lines of therapy (n=22) and 11.6 months (n=10) for subjects with 1
prior line of therapy (second line therapy).
"Moleculin is focusing on development of Annamycin to address
the significant unmet need in R/R AML. The growing body of
preliminary data continue to bolster our confidence in the safety
and efficacy of Annamycin, and its potential to provide patients
and physicians with a promising new treatment option in AML,"
commented Walter Klemp, Chairman and
Chief Executive Officer of Moleculin. "We believe the latest
preliminary OS data we are seeing in our MB-106 trial can now be
considered exceptional and we look forward to the initiation of our
pivotal registration study, especially now that our recent protocol
amendment allows for disclosing unblinded data for the first 45
subjects, which we expect within the next 12 months."
The Company is advancing the development of Annamycin in a Phase
3 pivotal trial evaluating 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. The Company remains on track to initiate
patient treatment in the first quarter of 2025.
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
1 A. Maiti, C. Rausch, J. Cortes, Et al,
"Outcomes of relapsed or refractory acute myeloid leukemia after
frontline hypomethylating agent and venetoclax regimens,
Haematologica online, vol. 106 No.3 (2021)
2 CR = complete remission; CRi = complete remission
with incomplete hematologic recovery
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SOURCE Moleculin Biotech, Inc.