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 Phase 1/2 trial evaluating the frontline
treatment of eprenetapopt with venetoclax and azacitidine in
patients with TP53 mutant AML has met the pre-specified primary
efficacy endpoint of complete remission (CR) rate.
In 30 patients who were evaluable for efficacy at the time of
the analysis, the CR rate was 37% and the composite response rate
of CR plus CR with incomplete hematologic recovery (CRi), CR/CRi,
was 53%. The trial met the primary efficacy endpoint of CR, which
is based on a Simon 2-stage design. As of the data cut, 11 patients
remain on study treatment and continue to be followed for safety
and efficacy. The Company plans to discuss the dataset with the
U.S. Food and Drug Agency (FDA) in the second half of 2021 and
expects to present data from the trial at a future scientific or
medical conference.
“We are pleased with these results from the combination of
eprenetapopt with venetoclax and azacitidine in this very
difficult-to-treat TP53 mutant AML population, a patient group with
significant unmet medical need,” said Eyal Attar, M.D., Chief
Medical Officer of Aprea Therapeutics. “These data, which follow
the recent granting of Fast Track and Orphan Drug designations by
FDA, provide further demonstration of the potential for
eprenetapopt in the treatment of myeloid malignancies. We continue
to make excellent progress across our myeloid malignancies program
and look forward to providing an update in July 2021 on our Phase 2
trial evaluating eprenetapopt with azacitidine as maintenance
therapy in TP53 mutant MDS and AML patients who have received
allogeneic stem cell transplant.”
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. 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 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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