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APRE - Aprea Therapeutics Inc News Story

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Aprea Therapeutics Announces Results of Primary Endpoint from Phase 3 Trial of Eprenetapopt in TP53 Mutant Myelodysplastic Syndromes (MDS)

Mon 28th December, 2020 1:00pm
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* The trial failed to meet its primary endpoint of complete remission (CR)

* CR rate was 53% higher in eprenetapopt with AZA arm compared to AZA alone,
but did not reach statistical significance
BOSTON, Dec. 28, 2020 (GLOBE NEWSWIRE) -- 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

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

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. Coiante
Sr. Vice President and Chief Financial Officer

Gregory A. Korbel
Vice President of Business Development


GlobeNewswire, Inc. 2020
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