Aprea Therapeutics Receives FDA Breakthrough Therapy Designation for APR-246 in Combination with Azacitidine for the Treatmen...
January 30 2020 - 7:30AM
Aprea Therapeutics, Inc. (NASDAQ: APRE), a biopharmaceutical
company focused on developing and commercializing novel cancer
therapeutics that reactivate mutant tumor suppressor protein p53,
today announced that the U.S. Food and Drug Administration (FDA)
has granted Breakthrough Therapy Designation for APR-246 in
combination with azacitidine for the treatment of myelodysplastic
syndromes (MDS) with a susceptible TP53 mutation.
MDS represents a spectrum of hematopoietic stem cell
malignancies in which bone marrow fails to produce sufficient
numbers of healthy blood cells. Approximately 30-40% of MDS
patients progress to acute myeloid leukemia (AML) and mutation of
the p53 tumor suppressor protein is thought to directly contribute
to disease progression and a poor overall prognosis.
“Breakthrough Therapy Designation further supports our
development program for APR-246 in combination with azacitidine in
MDS patients with a TP53 mutation,” said Christian S. Schade, Chief
Executive Officer of Aprea. “Outcomes for MDS patients with a TP53
mutation are poor and there are no current therapeutic options
specifically for these patients. We look forward to continued
interaction with FDA regarding our ongoing Phase 3 clinical study
and our clinical development program to advance APR-246.”
The FDA’s Breakthrough Therapy Designation is intended to
expedite the development and review of a drug candidate that is
planned to treat a serious or life-threatening disease or condition
when preliminary clinical evidence indicates that the drug may
demonstrate substantial improvement over available therapies on one
or more clinically significant endpoints.
About p53 and APR-246
The p53 tumor suppressor gene is the most frequently mutated
gene in human cancer, occurring in approximately 50% of all human
tumors. These mutations are often associated with resistance
to anti-cancer drugs and poor overall survival, representing a
major unmet medical need in the treatment of cancer.
APR-246 is a small molecule that has demonstrated reactivation
of mutant and inactivated p53 protein – by restoring wild-type p53
conformation and function – and thereby induce programmed cell
death in human cancer cells. Pre-clinical anti-tumor activity
has been observed with APR-246 in a wide variety of solid and
hematological cancers, including MDS, AML, and ovarian cancer,
among others. Additionally, strong synergy has been seen with
both traditional anti-cancer agents, such as chemotherapy, as well
as newer mechanism-based anti-cancer drugs and immuno-oncology
checkpoint inhibitors. In addition to pre-clinical testing, a Phase
1/2 clinical program with APR-246 has been completed, demonstrating
a favorable safety profile and both biological and confirmed
clinical responses in hematological malignancies and solid tumors
with mutations in the TP53 gene.
A pivotal Phase 3 clinical trial of APR-246 and azacitidine for
frontline treatment of TP53 mutant MDS is ongoing. APR-246 has
received Orphan Drug and Fast Track designations from the FDA for
MDS, and Orphan Drug designation from the EMA for MDS, AML and
ovarian cancer.
About Aprea Therapeutics
Aprea Therapeutics Inc., (NASDAQ: APRE) is a biopharmaceutical
company headquartered in Boston, Massachusetts with research
facilities in Stockholm, Sweden, focused on developing and
commercializing novel cancer therapeutics that reactivate the
mutant tumor suppressor protein p53. The Company’s lead product
candidate is APR-246, a small molecule in clinical development for
hematologic malignancies, including myelodysplastic syndromes (MDS)
and acute myeloid leukemia (AML). For more information, please
visit the company website at www.aprea.com.
The Company may use, and intends to use, its investor relations
website at www.ir.aprea.com as a means of disclosing material
nonpublic information and for complying with its disclosure
obligations under Regulation FD.
Forward-Looking Statements
Certain information contained in this press release includes
“forward-looking statements”, within the meaning of Section 27A of
the Securities Act of 1933, as amended, and Section 21E of the
Securities Exchange Act of 1934, as amended, related to our
clinical trials and regulatory submissions. We may, in some cases
use terms such as “predicts,” “believes,” “potential,” “continue,”
“anticipates,” “estimates,” “expects,” “plans,” “intends,” “may,”
“could,” “might,” “likely,” “will,” “should” or other words that
convey uncertainty of the future events or outcomes to identify
these forward-looking statements. Our forward-looking statements
are based on current beliefs and expectations of our management
team that involve risks, potential changes in circumstances,
assumptions, and uncertainties. Any or all of the
forward-looking statements may turn out to be wrong or be affected
by inaccurate assumptions we might make or by known or unknown
risks and uncertainties. These forward-looking statements are
subject to risks and uncertainties including risks related to the
success and timing of our clinical trials or other studies and the
other risks set forth in our filings with the U.S. Securities and
Exchange Commission, including our Quarterly Report on Form
10-Q. For all these reasons, actual results and developments
could be materially different from those expressed in or implied by
our forward-looking statements. You are cautioned not to place
undue reliance on these forward-looking statements, which are made
only as of the date of this press release. We undertake no
obligation to publicly update such forward-looking statements to
reflect subsequent events or circumstances.
Corporate Contacts:
Scott M. CoianteSr. Vice President and Chief Financial
Officer617-463-9385
Gregory A. KorbelVice President of Business
Development617-463-9385
Source: Aprea Therapeutics, Inc.
Aprea Therapeutics (NASDAQ:APRE)
Historical Stock Chart
From Jun 2024 to Jul 2024
Aprea Therapeutics (NASDAQ:APRE)
Historical Stock Chart
From Jul 2023 to Jul 2024