Full Press Release Details
Moleculin Receives Orphan
Drug Designation for Annamycin for the Treatment of Acute Myeloid Leukemia
HOUSTON - March 22, 2017 -
Moleculin Biotech, Inc., (NASDAQ: MBRX) ("Moleculin" or the "Company"), a preclinical pharmaceutical company
focused on the development of anti-cancer drug candidates, some of which are based on license agreements with The University of
Texas System on behalf of the M.D. Anderson Cancer Center, today announced its lead candidate, Annamycin (also known as "Liposomal
Annamycin"), an anthracycline, has received Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for the
treatment of acute myeloid leukemia (AML).
Moleculin's Chairman and CEO, Walter
Klemp, commented, "We are pleased to report this key milestone and the FDA's decision to grant Annamycin orphan drug
designation. We look forward to announcing additional milestones in regard to our clinical pathway as we make further progress."
The FDA grants orphan drug
designation to drugs and biologics that are intended for the treatment of rare diseases that affect fewer than 200,000 people in
the U.S. Orphan drug status is intended to facilitate drug development for rare diseases and may provide several benefits to drug
developers, including tax credits for qualified clinical trials costs, exemptions from certain FDA application fees, and seven
years of market exclusivity upon regulatory product approval.
is a cancer of the white blood cells and the acute forms of leukemia can manifest quickly and leave patients with limited treatment
options. AML is the most common type of acute leukemia in adults. It occurs when a clone of leukemic progenitor white blood cells
proliferates in the bone marrow suppressing the production of normal blood cells. In order to qualify for a curative bone marrow
transplant, patients must first undergo induction therapy. The current standard of care is the combining of 2 chemotherapeutic
drugs, always including an anthracycline intended to induce a CR or complete response, which has not improved since it was first
used in the 1970's. We estimate that it has the same cure rate of about 20% as then. Currently, the only viable long term
option for acute leukemia patients is a bone marrow transplant for those 20%, which is successful in a significant number of patients.
For more information on AML click: http://www.moleculin.com/technology/about-acute-myeloid-leukemia/.
Annamycin is an anthracycline intended
for the treatment of relapsed or refractory AML. Annamycin is a unique liposome formulated anthracycline (also referred to in literature
as "L-Annamycin") that has been designed to produce little to no cardiotoxicity and avoid the multidrug resistance
mechanisms that often defeat current anthracyclines. It has been tested in 114 patients in 6 clinical trials, 3 of which focused
on leukemia, with little to no cardiotoxicity and 3 of those clinical trials focused on leukemia. The Company is working with the
FDA on an investigative new drug application for a Phase I/II trial for second line treatment of relapsed or refractory AML, for
which no approved therapy currently exists.
About Moleculin Biotech,
Moleculin Biotech, Inc. is a preclinical
pharmaceutical company focused on the development of anti-cancer drug candidates, some of which are based on discoveries made at
M.D. Anderson Cancer Center. Our lead product candidate is Annamycin, an anthracycline for the treatment of relapsed or refractory
acute myeloid leukemia, more commonly referred to as AML. We also have two pre-clinical small molecule portfolios, one of
which is focused on the modulation of hard-to-target tumor cell signaling mechanisms and the recruitment of the patient's
own immune system. The other portfolio targets the metabolism of tumors.
more information about Moleculin, 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. These statements relate
to future events, future expectations, plans and prospects. Although Moleculin Biotech 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 the heading "Risk Factors" in our
Registration Statement on Form S-1 originally filed with the Securities and Exchange Commission on February 7, 2017, as amended
(Registration No. 333-214898). 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.
Chief Operating Officer