HOUSTON, May 7, 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
additional positive interim safety and efficacy data from its
ongoing open label, single arm Phase 1/2 study of Annamycin in
Poland. After receiving a single starting dose of 120
mg/m2 in the first cohort of the dose escalation phase
of the trial, 2 of 3 patients treated responded sufficiently to
qualify for a potentially curative bone marrow transplant.
The results for all 3 patients were reviewed by the Safety Review
Committee, which determined that no drug-related adverse events
were observed that would prevent advancing the trial to the next
higher dose level of 150 mg/m2. To date in the
European trial, 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. No additional
patient data have been developed in the Company's parallel US
clinical trial, which is currently recruiting its second cohort to
be given a dose level of 120 mg/m2 (the US trial started
at a lower initial dose of 100 mg/m2).
"The progress in Europe,
specifically Poland, continues to
be encouraging," commented Walter
Klemp, Moleculin's Chairman and CEO. "In just a few
months, we have completed the first cohort and we've seen a partial
response from 2 out of 3 patients sufficient to qualify them for a
potentially curative bone marrow transplant. It's important
to remember that these are relapsed or refractory patients for whom
the standard of care failed. So, to see this kind of response
at the starting dose level in the dose-ranging phase, we believe is
quite remarkable. And, although this data is still
preliminary and may not be indicative of the ultimate outcome of
the trial, the improvement in activity at 120 mg/m2 in
Poland as compared with the 100
mg/m2 cohort in the US is consistent with our
expectations for Annamycin."
Mr. Klemp continued: "Recognizing that cardiotoxicity is a
significant limitation of existing therapies, we are pleased that
we continue to see no evidence of cardiotoxicity in any of the
patients treated thus far. Specifically, there was no
observed reduction in left ventricle ejection fraction, which is
the standard metric for acute cardiotoxicity, nor any change in
biomarkers that would indicate the potential for long-term
cardiovascular impairment. This is an important step in the
ongoing clinical study of Annamycin and toward our goal of
ultimately demonstrating the drug's safety and effectiveness to
support regulatory approval in both the US and European
Union."
Dr. Robert Shepard, Moleculin's
Chief Medical Officer for Annamycin added: "With the Polish trial
now progressing to 150 mg/m2, we expect to see even
better results. And, although 150 mg/m2 was the
maximum tolerable dose established by the prior developer of
Annamycin due to the incidence of mucositis in patients above that
dose level, now that the cryotherapy protocol is well understood to
mitigate the potential for dose-limiting mucositis, there is a good
opportunity for dose levels to progress even beyond 150
mg/m2, so the potential to help patients is very
exciting."
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. The US and Polish trials have the same study
design, consisting of a Phase 1 intended to establish a
"Recommended Phase 2 Dose" ("RP2D"), to which the studies will then
proceed. The Phase 1 studies provide for escalating doses in
cohorts of 3 patients each, with each successive cohort receiving
the next higher dose level until "dose limiting toxicities" prevent
further increases. Cohort 1 in Poland received a dose of 120
mg/m2, and the results permit moving to 150
mg/m2. Cohort 1 in the US started at 100
mg/m2, and the results support moving to 120
mg/m2, and the Company is now seeking patients for the
second cohort in both studies. (Because one patient in US
cohort 1 did not complete the evaluation protocol, a fourth patient
was added to complete that cohort.) 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.
We plan to report top-line results by cohort in each trial, with
each announcement also including an update on the other
trial. Top-line results will include reporting of any
drug-related adverse events ("AEs") and assessment of
cardiotoxicity, including Echo or MUGA scans measuring change in
ejection fraction and measuring blood troponin level, which is
considered a biomarker for potential long-term cardiovascular
impairment. To date, one patient experienced grade 2
mucositis (which resolved to grade 1 within 2 days) and no other
drug-related AEs have been reported. Also, no loss of
ejection fraction or reduction in Troponin levels has been
reported. Top-line results will also include the number of
partial responses ("PRs"), complete responses ("CRs") and patients
deemed capable of progressing to a potentially curative bone marrow
transplant, which we term "bridge to transplant" ("BTs"), each of
which is essentially a function of the magnitude of reduction in a
patient's bone marrow blasts. For purposes of these clinical
trials, a CR means, primarily, that the patient's bone marrow
blasts reduced to 5% or less, a PR means the patient's bone marrow
blasts reduced, but not to the level of a CR, and a BT means
patients are deemed capable of progressing to a potentially
curative bone marrow transplant. To date, there have been no
PRs, CRs or BTs in the US trial, which has treated 4 patients at
100 mg/m2, and 2 PRs and BTs out of 3 patients treated
in the Polish trial at 120 mg/m2.
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.