HOUSTON, March 26, 2019 /PRNewswire/ -- Moleculin
Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a
clinical stage pharmaceutical company with a broad portfolio of
drug candidates targeting highly resistant tumors, today announced
positive interim safety and efficacy data from two ongoing open
label, single arm Phase 1/2 studies of Annamycin. In the
first study, being conducted in the US, four patients have
completed treatment at 100 mg/m2 with no significant
adverse events related to Annamycin, and the study will now proceed
to the next higher dose of 120 mg/m2. The second trial,
taking place in Poland, started at
a 120 mg/m2 dose of Annamycin and has treated three
patients. The first patient treated in that trial received a single
course of Annamycin and his bone marrow blasts have reduced from
60% to 11%. Our principal investigator considers this response
sufficient for the patient to proceed to consolidation therapy,
with the goal of receiving a potentially curative bone marrow
transplant. To date in Poland, one
patient experienced grade 2 mucositis (which resolved to grade 1
within 2 days) and no other adverse events related to Annamycin
have been reported. Trial results for the other two patients
treated in Poland will not be
known until the second quarter of this year.
"We are very pleased to have completed the treatment of patients
in cohort 1 of the US trial and move to the next higher dose,"
commented Walter Klemp, Moleculin's
Chairman and CEO. "In Poland, we are pleased to have such a
positive response at the starting dose level in this trial. Of
course the response of a single patient doesn't necessarily predict
the outcome of the trial, but this is a great way to begin and it's
consistent with our expectations for Annamycin."
Mr. Klemp continued: "One of the advantages we believe Annamycin
will offer is a lack of cardiotoxicity. We have seen no
evidence of cardiotoxicity in any of the patients treated thus
far. We intend to advance the clinical study of Annamycin
with the goal of ultimately demonstrating the drug's safety and
effectiveness to support regulatory approval in the US and European
Union."
Dr. Robert Shepard, Moleculin's
Chief Medical Officer for Annamycin added: "A prior clinical trial
for Annamycin in acute leukemia demonstrated activity at its
Maximum Tolerable Dose of 150 mg/m2, so we are pleased
for the US trial to move to the next higher dose in cohort 2
of 120 mg/m2. In Poland, we consider reducing bone marrow
blasts for Patient 1 down to 11% with a single course of Annamycin
to be very encouraging. Of course, significant
additional study is necessary to definitively demonstrate
causality."
"We have begun a second course of Annamycin as a consolidation
phase for Patient 1," commented Dr. Lidia
Gil, Principal Investigator in the Polish Annamycin clinical
trial. "Considering that this patient was refractory to
standard of care induction therapy, I am very pleased to see that
Annamycin appears to be showing activity. While this is
considered a 'Partial Response,' I believe it's enough of a
reduction to serve as a 'bridge to transplant' for this
patient. Importantly, with the first course of Annamycin, no
toxicities have been observed that would limit continued dosing
with Annamycin."
Study Design
The Company is studying Annamycin in both the US and
Poland in open label, single arm
clinical trials to assess the safety and efficacy of Annamycin for
the treatment of adults with relapsed or refractory acute myeloid
leukemia. Both the US and Polish trials have the same study
design, providing for a Phase 1 intended to establish a
"Recommended Phase 2 Dose," ("RP2D") with cohorts of 3 patients
each where the first cohort starts at a low beginning dose and each
successive cohort receives the next higher dose level until "dose
limiting toxicities" prevent further increases. In the case
of cohort 1 in the US, one patient did not complete the evaluation
protocol, so a fourth patient was added to complete that
cohort.
A key difference in the US is that the starting dose was 100
mg/m2, whereas, in Poland, the starting dose was 120
mg/m2. Having completed the first cohort in the
US, the Company is seeking patients for the second cohort at a dose
level of 120 mg/m2. Once 3 patients have completed
the safety evaluation period of the first cohort in Poland, the second cohort will begin there at
a dose level of 150 mg/m2. Once the Company
establishes an RP2D, the intent is for each trial to advance to a
Phase 2 arm planned to assess the safety and efficacy of Annamycin
in 21 additional patients.
The US trial also differs from the Polish trial in that the FDA
would like to review safety data relating to cardiotoxicity from
patients treated prior to advancing beyond 120
mg/m2. The Company believes that the additional
patient safety data gained from the Polish trial may also assist in
the FDA's review of cardiac safety.
About Moleculin Biotech, Inc.
Moleculin Biotech, Inc. is a clinical stage pharmaceutical
company focused on the development of a broad portfolio of oncology
drug candidates for the treatment of highly resistant tumors. The
Company's clinical stage drugs are: Annamycin, a Next Generation
Anthracycline, designed to avoid multidrug resistance mechanisms
with little to no cardiotoxicity being studied for the treatment of
relapsed or refractory acute myeloid leukemia, more commonly
referred to as AML, 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 cancer and hematologic malignancies, and
WP1220, an analog to WP1066, for the topical treatment of cutaneous
T-cell lymphoma. Moleculin is also engaged in preclinical
development of additional drug candidates, including additional
Immune/Transcription Modulators, as well as compounds capable of
Metabolism/Glycosylation Inhibition.
For more information about the Company, please visit
http://www.moleculin.com.
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 ability of the Company to
successfully recruit patients to complete its clinical trials and
the ability of Annamycin to show safety and efficacy in patients.
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 Biotech 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. The
Company cautions investors not to place undue reliance on the
interim results announced today.
Contacts
Joe Dorame,
Robert Blum or Joe Diaz
Lytham Partners, LLC
602-889-9700
mbrx@lythampartners.com
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SOURCE Moleculin Biotech, Inc.