Aprea Therapeutics Completes Full Enrollment of Phase 3 Clinical Trial in TP53 Mutant Myelodysplastic Syndromes (MDS)
June 03 2020 - 8:00AM
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 patient enrollment in its Phase 3 clinical
trial evaluating eprenetapopt with azacitidine for the treatment of
front-line TP53 mutant myelodysplastic syndromes (MDS) has been
completed. Topline results are expected by year-end 2020. Aprea
plans to include the results of the trial in a New Drug Application
(NDA) to the U.S. FDA and a Marketing Authorization Application
(MAA) to the EMA in 2021.
“Completion of enrollment in this Phase 3 trial is an important
milestone for Aprea and our first-in-class p53 reactivator,
eprenetapopt,” said Christian S. Schade, President and Chief
Executive Officer of Aprea. “We continue to advance the development
of eprenetapopt with the goal of providing an urgently needed
therapeutic option to patients with p53 mutated MDS.”
The randomized, controlled pivotal Phase 3 trial is designed to
evaluate eprenetapopt with azacitidine compared with azacitidine
alone as front-line therapy in intermediate, high, and very high
risk TP53 mutant MDS patients. The multi-center trial enrolled 154
patients, randomized 1:1 to the two arms with a primary endpoint of
CR rate. The trial has 90% power with P-value < 0.05 to detect a
difference in CR rates of 50% in the eprenetapopt-containing arm
versus 25% in the azacitidine-only control arm.
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 APR-246 (eprenetapopt), a small molecule
in clinical development for hematologic malignancies, including
myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML).
APR-246 has received Breakthrough Therapy, Orphan Drug and Fast
Track designations from the FDA for MDS, and Orphan Drug
designation from the European Commission for MDS, AML and ovarian
cancer. 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 and APR-246 (eprenetapopt)
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.
APR-246 (eprenetapopt) is a small molecule that has demonstrated
reactivation of mutant and inactivated p53 protein – by restoring
wild-type p53 conformation and function – and thereby induce
programmed cell death in human cancer cells. Pre-clinical
anti-tumor activity has been observed with APR-246 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 APR-246 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.
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.
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 clinical trials, regulatory
submissions and projected cash position. We may, in some cases use
terms such as “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
617-463-9385
Gregory A. Korbel
Vice President of Business Development
617-463-9385
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