Aprea Therapeutics, Inc. (Nasdaq: APRE), a biopharmaceutical
company focused on developing and commercializing novel cancer
therapeutics that reactivate the mutant tumor suppressor protein,
p53, today announced results of the primary data cut from its Phase
3 clinical trial evaluating the safety and efficacy of eprenetapopt
with azacitidine (AZA) versus AZA alone in TP53 mutant
myelodysplastic syndromes (MDS). The trial did not meet the
predefined primary endpoint of complete remission (CR)
rate. Analysis of the primary endpoint at this data cut
demonstrated a higher CR rate in the experimental arm receiving
eprenetapopt with AZA versus the control arm receiving AZA alone,
but did not reach statistical significance. In the
intention-to-treat population of 154 patients, the CR rate in the
eprenetapopt with AZA arm was 33.3% (95% CI: 23.1% - 44.9%)
compared to 22.4% (95% CI: 13.6% - 33.4%) in the AZA alone arm (P =
0.13).
While analysis of certain secondary endpoints (ORR
and duration of responses) appears to favor the experimental arm at
this data cut, they are not significantly different. The median
duration of overall survival at the primary data cut was similar
between the arms. Additional patients in the study who have not
achieved a CR remain on study treatment and the data will be
analyzed at future pre-specified timepoints as set forth in the
statistical analysis plan. The combination of eprenetapopt with AZA
appeared well-tolerated, with an adverse event profile that was
similar to the Company’s prior Phase 2 clinical trials. Subsequent
analyses of the trial data, including secondary endpoints, will be
conducted as the duration of patient follow-up increases. The
Company expects to present the data at a future scientific
conference.
“Though we are disappointed the topline results did
not reach statistical significance, we continue to believe that
eprenetapopt can offer clinical benefit to patients with TP53
mutant malignancies,” said Dr. Eyal Attar, Chief Medical Officer of
Aprea. “We will continue to analyze data as it matures and follow
patients who are still receiving study treatment. Our other
clinical trials continue to progress and we remain committed to
pursuing our clinical development programs.”
About the Phase 3 Trial in
TP53 Mutant MDS
The Phase 3 trial enrolled 154 TP53 mutant MDS
patients, randomized 1:1 to either the eprenetapopt with AZA arm or
the AZA alone arm. Response criteria are those defined by
International Working Group 2006 (IWG 2006) and include measures of
peripheral blood counts and bone marrow blasts.
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), Fast
Track designation from the FDA for acute myeloid leukemia (AML),
and Orphan Drug designation from the European Commission for MDS,
AML and ovarian cancer. 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 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, Fast Track designation from the
FDA for AML, and Orphan Drug designation from the European
Medicines Agency for MDS, AML and ovarian cancer.
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. Enrollment in a Phase
1 clinical trial of APR-548 is anticipated to begin in the first
quarter of 2021.
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. KorbelVice President of Business
Development617-463-9385
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