Full Press Release Details
Therapeutics Announces Results of Primary Endpoint from
Phase 3 Trial of Eprenetapopt in TP53 Mutant Myelodysplastic
MA, December 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).
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.
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."
the Phase 3 Trial in TP53 Mutant MDS
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.
Aprea Therapeutics, Inc.
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.
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.
p53, eprenetapopt and APR-548
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 - 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.
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.
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.
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
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assumptions, and uncertainties. Any or all of the forward-looking statements may turn out to be wrong or be affected by inaccurate
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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
Aprea Therapeutics, Inc.
Vice President and Chief Financial Officer
President of Business Development