HOUSTON, Feb. 5, 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
intends to discuss with the FDA and EMA (European Medicines Agency)
plans to conduct a single arm Phase 2 trial that would serve as the
basis for accelerated approval of Liposomal Annamycin ("Annamycin")
to treat relapsed or refractory acute myeloid leukemia ("AML").
This will follow the establishment of a recommended Phase 2 dose
("RP2D") in the Company's ongoing Phase 1 dose escalation trial in
Europe. The FDA has already
granted Annamycin Fast Track status and Orphan Drug Designation for
AML. FDA grants Fast Track designation to drugs intended to treat
serious conditions that demonstrate the potential to address unmet
medical needs, which can include providing efficacy comparable to
available therapy while avoiding toxicity associated with the
existing treatment. The benefits of Fast Track include FDA actions
to expedite development and review, including "rolling review,"
where the agency reviews portions of a marketing application before
the complete application is submitted.
The announcement follows continuing positive results from
Moleculin's open label, single arm Phase 1 trials in AML patients
in Europe and the US. Most
recently, the US Phase 1 study met its primary objective of
demonstrating the safety of Annamycin at a dose that was
cumulatively at or below the lifetime maximum anthracycline dose.
Those results are consistent with results achieved with the
parallel Phase 1 study being conducted in Europe, which has demonstrated the safety of
escalating doses of Annamycin in AML patients, including doses that
significantly exceed the maximum lifetime dose of anthracyclines
imposed in the US. In both trials, the primary endpoints are aimed
at demonstrating the product's safety, primarily the lack of
cardiovascular risk. This is a key characteristic that, if borne
out, could significantly differentiate Annamycin from other
anthracyclines, which generally are well-known to have
treatment-limiting cardiotoxicity.
Based on these results, Moleculin will continue to focus the
Company's efforts on the European trial to establish an RP2D. Once
that is complete, the Company intends to enter discussions with the
FDA and EMA about conducting a single arm Phase 2 study that would
be the pivotal trial supporting US and European approval of
Annamycin for relapsed or refractory AML.
Walter Klemp, Chairman and CEO of
Moleculin commented, "We believe relying upon the European trial to
establish an RP2D is the fastest and most efficient way to reach a
pivotal Phase 2 trial. Recruitment in Europe has been faster than in the US and the
trial is progressing well. The US Phase 1 trial was designed to
demonstrate that Annamycin is indeed non-cardiotoxic when delivered
to patients at or below the lifetime maximum anthracycline dose,
and it has served that purpose. Beyond that, we have now treated 9
patients in the European trial above the lifetime maximum, also
without any evidence of cardiotoxicity."
The U.S. trial met its primary endpoint, demonstrating the
safety of Annamycin in AML patients, most importantly 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.
Dr. Rob Shepard, Chief Medical
Officer – Annamycin, added, "Because of this early success in the
US trial and our continued progress in the parallel European trial,
and given our new Fast Track status, we have made a strategic
decision on how best to move forward. Once we have established an
RP2D with the European trial, we intend to discuss with the FDA
conducting a Phase 2 single arm registration trial that would form
the basis for accelerated approval. This should allow us to combine
efforts between the U.S. and Europe, creating one global Phase 2 trial. The
European trial continues with its dose escalation, and although
dosing had now taken patients well beyond the lifetime maximum
anthracycline limit with no evidence of cardiotoxicity, we are
still below what we believe to be a therapeutic dose. We look
forward to reaching a therapeutic dose and a recommended Phase 2
dose in the coming quarters."
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.
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SOURCE Moleculin Biotech, Inc.