HOUSTON, April 2, 2020 /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
it has completed the latest (210 mg/m2) cohort in its
European open label, single arm Phase 1/2 clinical trial of
Annamycin for the treatment of relapsed or refractory acute myeloid
leukemia ("AML"). A total of 19 patients have been treated in
the US and Europe, and all results
continue to show Annamycin to be safe, and, especially, all have
shown Annamycin to be free of cardiotoxicity. Of those, 10
have been treated at or above the FDA lifetime maximum
anthracycline exposure.
The Phase 1 portion of this clinical trial, which is described
in more detail later in this press release, is designed to
establish the safety of Annamycin and to determine the Recommended
Phase 2 Dose to be used in the Phase 2 portion of the trial.
While the Primary Endpoint of the Phase 1 portion is safety, a
Secondary Endpoint is the assessment of efficacy generally defined
as an improvement in bone marrow biopsy results sufficient to
qualify patients for a potentially curative bone marrow
transplant. The Company cautions not to place undue reliance
on interim results.
The fourth cohort in Poland
receiving a single dose of 210 mg/m2 in the Phase 1 dose
escalation portion of the trial was completed with no adverse
events and the trial will continue to the next cohort of 240
mg/m2. To date in the European trial, only one
adverse event related to Annamycin has been reported; a patient
experienced grade 2 mucositis (which resolved to grade 1 within 2
days). In the Company's recently completed Phase 1 portion of
a parallel US Phase 1/2 clinical trial, there were no unexpected
serious adverse events (SAE) and no dose limiting toxicities (DLT)
at any dose tested.
We refer to Annamycin as a "next generation anthracycline,"
because it is designed to provide enhanced therapeutic benefits
when compared with traditional anthracyclines while reducing the
potential for unwanted cardiotoxicity, or damage to the heart. This
design intent has previously been validated with preclinical
toxicology studies in animal models (as required by FDA)
demonstrating Annamycin has little to no cardiotoxicity when
compared with doxorubicin. Of the 19 patients treated thus far in
both trials, none has shown any evidence of cardiotoxicity.
This includes 10 patients in Poland who were treated at levels above the US
maximum allowable cumulative anthracycline dose level (550
mg/m2), a limitation not imposed on our trial in
Europe. If upheld in further studies, we believe this lack of
toxicity would be an important differentiator between Annamycin and
the currently approved anthracyclines, for which cardiotoxicity is
a well-known treatment limitation.
Walter Klemp, Chairman and CEO of
Moleculin commented, "Now that we are relying upon the European
trial to establish an RP2D, and now that it is becoming apparent
that the safety profile of Annamycin is even better than we
expected, we will explore opportunities to increase the dosing
increment between cohorts to speed up the establishment of the
Recommended Phase 2 Dose or RP2D. We continue to target
establishing the RP2D before the end of the year, but of course
this will depend upon the continued rate of recruitment and the
actual point at which we begin to see dose limiting
toxicities."
The U.S. trial met its primary endpoint, demonstrating the
safety of Annamycin in AML patients. Most importantly, all patients
reflected the absence of cardiotoxicity (potential damage to the
heart), as determined by echocardiograms, as well as cardiac health
biomarkers, principally blood troponin levels. Based on testing to
date, no patients in either the US or European trial have exhibited
evidence of cardiotoxicity. Additionally, there were no
unexpected serious adverse events (SAE) and no dose limiting
toxicities (DLT) at any dose tested. Although a primary
objective of the Phase 1 trial was to evaluate safety, the study
also gathered data to support a preliminary assessment of the
product's efficacy. Among other things, the study recorded
complete response (CR), partial response (PR), event-free survival
(EFS), overall survival (OS; Kaplan-Meier), and time to and
duration of remission/response. The Company reported efficacy
in 33% of the US patients, even though the drug was dosed at what
was expected to be sub-therapeutic levels. The evidence of
efficacy consisted of 1 patient who achieved a "morphologically
leukemia-free state," which the protocol defined as a CR with
incomplete recovery of platelets or neutrophils, and another
patient who had a substantial remission of leukemia cutis (a
somewhat rare leukemia symptom), from diffuse to 3 lesions.
In all but one of the 7 relapsed patients treated in our
Poland trial, a reduction in
blasts in the bone marrow aspirate occurred. Of those 7, 2
qualified as PRs and 1 qualified as a bridge-to-transplant.
In the last cohort, only 1 patient was relapsed, and that patient
had a 33% reduction in bone marrow blasts. In all three
patients in the 210 mg/m2 cohort (1 relapsed and 2 refractory), a
reduction of circulating blasts to <2% was achieved during
treatment.
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, under investigation for
brain tumors, pancreatic cancer and hematologic malignancies; and
WP1220, an analog to WP1066, being developed 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, the
ability of Annamycin to show safety and efficacy in patients, and
the ability for Annamycin to be an alternative to currently
approved anthracyclines for treating cancers other than AML.
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
James Salierno
/ Carol Ruth
The Ruth Group
646-536-7028 /
7000
jsalierno@theruthgroup.com
cruth@theruthgroup.com
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SOURCE Moleculin Biotech, Inc.