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 the oral presentation of updated data from its
French Phase 1b/2 clinical trial at the 25th European Hematology
Association Annual Meeting (EHA). The trial is evaluating the
safety and efficacy of APR-246 (eprenetapopt) in combination with
azacitidine (AZA) for the treatment of TP53 mutant myelodysplastic
syndromes (MDS) and acute myeloid leukemia (AML). The clinical
trial is sponsored by the Groupe Francophone des Myélodysplasies
(GFM).
As of the April 1, 2020 data cutoff, the overall response rate
(ORR) in 28 evaluable MDS patients was 75%, with a 57% complete
remission (CR) rate, by International Working Group (IWG) criteria.
With a median duration of follow-up of 9.7 months, the median
overall survival (OS) for all enrolled patients (n=52) was 12.1
months and in MDS patients (n=34) was 12.1 months. For patients who
remained on treatment for 3 or more cycles of treatment the median
OS was higher at 13.7 months versus 2.8 months for patients who
were on treatment for fewer than 3 cycles. Relative to baseline,
mutant TP53 variant allele frequency (VAF) was decreased in
responding patients by 3 cycles of treatment, including 20 (51%)
patients who achieved mutant TP53 negativity by next-generation
sequencing (NGS).
“The data from this ongoing trial of eprenetapopt with
azacitidine continue to be very encouraging in these most
difficult-to-treat TP53 mutant MDS and AML patients, who not only
have at least one TP53 mutation but the majority of whom also have
high risk cytogenetic abnormalities,” said Thomas Cluzeau, M.D.,
co-lead investigator for the GFM trial. “We continue to observe ORR
and CR rates in these patients that are substantially higher than
the GFM’s experience with azacitidine monotherapy. Furthermore,
with increased duration of follow-up, we now also see the emergence
of highly encouraging overall survival that appears to be better
than azacitidine alone or in combination with others agents in this
very high-risk molecular group of patients with a TP53
mutation.”
Details of the on-demand oral presentation are as follows:
Title: APR-246 Combined with Azacitidine in TP53 Mutated
Myelodysplastic Syndromes (MDS) and Acute Myeloid Leukemia. A Phase
2 Study by the Groupe Francophone des Myélodysplasies (GFM)
Oral Abstract Session: Novel treatments for MDS I
Abstract: S181
About the Clinical Trial
Eligible patients in the Phase Ib/II clinical trial include
hypomethylating agent (HMA) naïve, TP53 mutated MDS and AML. All
enrolled patients were to receive APR-246 as a 4,500 mg fixed dose
IV daily for 4 days and AZA over 7 days in 28-day cycles. The
primary endpoint of the trial is CR rate.
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 Myelodysplastic Syndromes
Myelodysplastic syndromes (MDS) represents 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.
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.
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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