SAN DIEGO and TORONTO, June 2,
2015 /CNW/ - Aptose Biosciences Inc. (NASDAQ: APTO;
TSX: APS), a clinical-stage company developing new therapeutics
that target the underlying mechanisms of cancer, today announced
that the U.S. Food and Drug Administration (FDA) has granted the
company orphan drug designation for APTO-253 for the treatment of
acute myeloid leukemia (AML). APTO-253, a first-in-class inducer of
the KLF4 gene, is the company's lead product candidate in a Phase
Ib clinical trial in patients with AML, high-risk myelodysplastic
syndrome (MDS) and other hematologic malignancies in which KLF4
silencing is reported as operative.
"AML is a particularly challenging cancer of the blood and bone
marrow for which there are currently few treatment options," said
William G. Rice, Ph.D., Chairman,
President and CEO. "APTO-253, with its unique mechanism of action,
has the potential to emerge as an entirely new therapeutic approach
for this patient population, and receiving orphan drug designation
is a key regulatory milestone along the path."
Epigenetic suppression of the Krüppel-like factor 4 (KLF4) gene
has been reported in the scientific literature as a key
transforming event in AML. APTO-253 is a first-in-class, targeted
inducer of the KLF4 tumor suppressor gene, and has demonstrated a
favorable safety profile with no evidence of suppression of the
normal bone marrow. Preclinical studies have shown potent
single-agent activity to kill AML cells and strong synergy as part
of a combination strategy with various marketed and investigational
agents. APTO-253 is currently in a Phase 1b clinical study in
patients with relapsed or refractory hematologic
malignancies.
Orphan drug designation is granted by the FDA to encourage
companies to develop therapies for the treatment of diseases that
affect fewer than 200,000 individuals in the United States. Orphan drug status provides
research and development tax credits, an opportunity to obtain
grant funding, exemption from FDA application fees and other
benefits. If APTO-253 is approved to treat AML, the orphan drug
designation provides Aptose with seven years of marketing
exclusivity.
About Acute Myeloid Leukemia
Acute myeloid leukemia (AML) is a cancer derived from myeloid
progenitor or stem cells that typically mature into red blood
cells, white blood cells or platelets. AML initiates in the bone
marrow when stem or progenitor cells lose cell cycle control,
anti-apoptotic factor or other means to limit rampant
proliferations. Leukemic cells have the ability to rapidly
spread from the marrow to the bloodstream. Further, these rapidly
proliferating cells quickly crowd out normal cells as they
infiltrate other organs and tissue systems.
AML is the most common type of acute leukemia among adults, with
an annual incidence of more than 18,000 patients, and causing more
than 10,000 deaths each year in the U.S. It is a particularly
devastating blood cancer, with less than 25 percent of newly
diagnosed patients surviving beyond five
years.
About Aptose
Aptose Biosciences is a clinical-stage biotechnology company
committed to discovering and developing personalized therapies
addressing unmet medical needs in oncology. Aptose is advancing new
therapeutics focused on novel cellular targets on the leading edge
of cancer research, coupled with companion diagnostics to identify
the optimal patient population for our products. The Company's
small molecule cancer therapeutics pipeline includes products
designed to provide enhanced efficacy with existing anti-cancer
therapies and regimens without overlapping toxicities. Aptose
Biosciences Inc. is listed on NASDAQ under the symbol APTO and on
the TSX under the symbol APS.
This press release contains forward-looking statements within
the meaning of Canadian and U.S. securities laws. Such statements
include, but are not limited to, statements relating to Aptose's
plans, objectives, expectations and intentions and other statements
including words such as "continue", "expect", "intend", "will",
"should", "would", "may", and other similar expressions. Such
statements reflect our current views with respect to future events
and are subject to risks and uncertainties and are necessarily
based upon a number of estimates and assumptions that, while
considered reasonable by us are inherently subject to significant
business, economic, competitive, political and social uncertainties
and contingencies. Many factors could cause our actual results,
performance or achievements to be materially different from any
future results, performance or achievements described in this press
release. Such expressed or implied factors include, among others:
changes in our stock price; our ability to meet listing
requirements; our ability to obtain the capital required for
research and operations; the inherent risks in early stage drug
development including demonstrating efficacy; development time/cost
and the regulatory approval process; the progress of our clinical
trials; our ability to find and enter into agreements with
potential partners; our ability to attract and retain key
personnel; changing market conditions; stock market volatility; and
other risks detailed from time-to-time in our ongoing quarterly
filings, annual information forms, annual reports and annual
filings with Canadian securities regulators and the United States
Securities and Exchange Commission.
Should one or more of these risks or uncertainties materialize,
or should the assumptions set out in the section entitled "Risk
Factors" in our filings with Canadian securities regulators and the
United States Securities and Exchange Commission underlying those
forward-looking statements prove incorrect, actual results may vary
materially from those described herein. These forward-looking
statements are made as of the date of this press release and we do
not intend, and do not assume any obligation, to update these
forward-looking statements, except as required by law. We cannot
assure you that such statements will prove to be accurate as actual
results and future events could differ materially from those
anticipated in such statements. Investors are cautioned that
forward-looking statements are not guarantees of future performance
and accordingly investors are cautioned not to put undue reliance
on forward-looking statements due to the inherent uncertainty
therein.
SOURCE Aptose Biosciences Inc.
