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 positive results from its Phase 2 trial evaluating
eprenetapopt with azacitidine for post-transplant maintenance
therapy in patients with TP53 mutant MDS and AML.
In 33 patients enrolled in the trial, the
relapse free survival (RFS) at 1 year post-transplant was 58% and
the median RFS was 12.1 months. The overall survival (OS) at 1 year
post-transplant was 79%, with a median OS of 19.3 months. Prior
clinical trials evaluating post-transplant outcomes in TP53 mutant
MDS and AML patients have reported a 1-year post-transplant RFS of
~30% and a median OS of ~5-8 months. In addition, the post-
transplant regimen of eprenetapopt and azacitidine was well
tolerated among patients in the clinical trial. The Company plans
to discuss the data from this Phase 2 clinical trial with the U.S.
Food and Drug Agency (FDA) in the second half of 2021 and expects
to present data at a future scientific or medical conference.
“The post-transplant RFS and OS data with
eprenetapopt and azacitidine maintenance therapy in these very
difficult-to-treat TP53 mutant MDS and AML patients are incredibly
exciting,” said trial principal investigator Asmita Mishra, M.D.,
of the H. Lee Moffitt Cancer Center and Research Institute.
“Although transplant is currently the only potentially curative
treatment for patients with TP53 mutant MDS and AML, the risk of
relapse with current standard of care remains unacceptably high and
the median OS post-transplant is very limited at 8 months or less.
Post-transplant maintenance therapy with eprenetapopt and
azacitidine could, if approved, represent a new treatment paradigm
that meaningfully improves outcomes for these patients with limited
treatment options.”
About Aprea Therapeutics,
Inc.
Aprea Therapeutics, Inc. is a biopharmaceutical
company headquartered in Boston, Massachusetts with research
facilities in Stockholm, Sweden, focused on developing and
commercializing novel cancer therapeutics that reactivate mutant
tumor suppressor protein, p53. The Company’s lead product candidate
is eprenetapopt (APR-246), a small molecule in clinical development
for hematologic malignancies and solid tumors. Eprenetapopt has
received Breakthrough Therapy, Orphan Drug and Fast Track
designations from the FDA for myelodysplastic syndromes (MDS),
Orphan Drug and Fast Track designations from the FDA for acute
myeloid leukemia (AML), and Orphan Drug designation from the
European Commission for MDS and AML. APR-548, a next generation
small molecule reactivator of mutant p53, is being developed for
oral administration. 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 https://ir.aprea.com/ as a means of
disclosing material nonpublic information and for complying with
its disclosure obligations under Regulation FD.
About p53, eprenetapopt and
APR-548
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.
Eprenetapopt (APR-246) is a small molecule that
has demonstrated reactivation of mutant and inactivated p53 protein
– by restoring wild-type p53 conformation and function – thereby
inducing programmed cell death in human cancer cells. Pre-clinical
anti-tumor activity has been observed with eprenetapopt 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
eprenetapopt 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 eprenetapopt
and azacitidine for frontline treatment of TP53 mutant MDS has been
completed and failed to meet the primary statistical endpoint of
complete remission. A Phase 1/2 clinical trial of eprenetapopt with
venetoclax and azacitidine for the frontline treatment of TP53
mutant AML met the primary efficacy endpoint of complete remission.
Additional clinical trials in hematologic malignancies and solid
tumors are ongoing. Eprenetapopt has received Breakthrough Therapy,
Orphan Drug and Fast Track designations from the FDA for MDS,
Orphan Drug and Fast Track designations from the FDA for AML, and
Orphan Drug designation from the European Medicines Agency for MDS
and AML.
APR-548 is a next-generation small molecule p53
reactivator. APR-548 has demonstrated high oral bioavailability,
enhanced potency relative to eprenetapopt in TP53 mutant cancer
cell lines and has demonstrated in vivo tumor growth inhibition
following oral dosing of tumor-bearing mice.
About MDS
Myelodysplastic syndromes (MDS) represent 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 contribute to disease progression. Mutations in p53 are
found in up to 20% of MDS and AML patients and are associated with
poor overall prognosis. There are no currently approved therapies
specifically for TP53 mutant MDS or AML patients.
About AML
AML is the most common form of adult leukemia,
with the highest incidence in patients aged 60 years and older. AML
is characterized by proliferation of abnormal immature white blood
cells that impairs production of normal blood cells. AML can
develop de novo or may arise secondary to progression of other
hematologic disorders or from chemotherapy or radiation treatment
for a different, prior malignancy; secondary AML carries a worse
prognosis than de novo AML. Mutations in TP53, which are associated
with poor overall prognosis, occur in approximately 20% of patients
with newly diagnosed AML, more than 30% of patients with
therapy-related AML and approximately 70-80% of patients with
complex karyotype.
Forward-Looking Statement
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 study analyses, clinical trials, regulatory
submissions, and projected cash position. We may, in some cases use
terms such as “future,” “predicts,” “believes,” “potential,”
“continue,” “anticipates,” “estimates,” “expects,” “plans,”
“intends,” “targeting,” “confidence,” “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, risks associated with the coronavirus pandemic and the
other risks set forth in our filings with the U.S. Securities and
Exchange Commission. 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.
Source: Aprea Therapeutics, Inc.
Corporate Contacts:
Scott M. CoianteSr. Vice President and Chief
Financial Officer617-463-9385
Gregory A. KorbelSr. Vice President and Chief
Business Officer617-463-9385
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