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Aprea Therapeutics Receives FDA Orphan Drug Designation for Eprenetapopt for the Treatment of Acute Myeloid Leukemia (AML)

Thu 8th April, 2021 4:15pm
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BOSTON, April 08, 2021 (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 that the U.S. Food and Drug Administration (FDA) has
granted Orphan Drug designation to eprenetapopt for treatment of AML.

“We are pleased to have been granted Orphan Drug designation by FDA for
eprenetapopt in AML, building on the Fast Track designation in AML that was
granted in November 2020,” said Christian S. Schade, Chairman and Chief
Executive Officer of Aprea. “We look forward to continued productive
dialogue with FDA and bringing eprenetapopt to patients as soon as

Orphan Drug designation is granted by the FDA Office of Orphan Products
Development to advance the evaluation and development of safe and effective
therapies for the treatment of rare diseases or conditions affecting fewer
than 200,000 people in the U.S. The designation can provide development and
commercial incentives for designated compounds and medicines, including
eligibility for a seven-year period of market exclusivity in the U.S. after
product approval, FDA assistance in clinical trial design, tax credits related
to clinical trial expenses, and an exemption from FDA user fees.

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, 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 failed to meet the primary
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, 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
early in the second quarter of 2021.

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. Coiante

Sr. Vice President and Chief Financial Officer


Gregory A. Korbel

Chief Business Officer



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