Aprea Therapeutics Presents Results From French Phase Ib/II Clinical Trial of APR-246 and Azacitidine (AZA) in Patients with ...
December 09 2019 - 11:00AM
Aprea Therapeutics, Inc. (Nasdaq: APRE), a biopharmaceutical
company focused on developing and commercializing novel cancer
therapeutics that reactivate mutant tumor suppressor protein p53,
presented preliminary results at the 2019 ASH Annual Meeting from
its French Phase Ib/II clinical trial in MDS and AML. The trial is
evaluating the safety and efficacy of APR-246 in combination with
azacitidine for the treatment of patients with TP53 mutated MDS and
AML. The clinical trial is sponsored by the Groupe Francophone des
Myélodysplasies.
As of the data cutoff, the overall response rate (ORR) in 24
evaluable MDS patients was 74%, with a 66% complete remission (CR)
rate, based on International Working Group criteria. With a median
duration of follow-up of 6.4 months, the median overall survival
(OS) for all enrolled patients (n=53) had not been reached.
In addition, all responding patients were alive at data
cutoff. Relative to baseline, mutant TP53 variant allele
frequency (VAF) was significantly decreased in responding patients
and undetectable in all patients who achieved a CR.
About the Clinical Trial
Eligible patients in the Phase Ib/II clinical trial include HMA
naïve, TP53 mutated MDS and acute myeloid leukemia (AML). All
enrolled patients were to receive APR-246 as a 4,500 mg fixed dose
IV daily (days 1-4) and AZA over 7 days (days 4-10 or 4-5 and 8-12)
in 28-day cycles. The primary endpoint of the trial is CR rate.
About Myelodysplastic Syndrome
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
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 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
I/II 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.
A pivotal Phase 3 clinical trial of APR-246 and azacitidine for
frontline treatment of TP53 mutant MDS is ongoing. APR-246 has
received Orphan Drug and Fast Track designations from the FDA for
MDS, and Orphan Drug designation from the EMA for MDS, AML and
ovarian cancer.
About Aprea Therapeutics
Aprea Therapeutics Inc., (NASDAQ: APRE) is a biopharmaceutical
company headquartered in Boston, Massachusetts with research
facilities in Stockholm, Sweden, focused on developing and
commercializing novel cancer therapeutics that reactivate the
mutant tumor suppressor protein p53. The Company’s lead product
candidate is APR-246, a small molecule in clinical development for
hematologic malignancies, including myelodysplastic syndromes (MDS)
and acute myeloid leukemia (AML). 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 www.ir.aprea.com as a means of disclosing material
nonpublic information and for complying with its disclosure
obligations under Regulation FD.
The Company will host a 2019 ASH Clinical Update meeting and
webcast as follows:
Time and Date: Monday, December 9 at 12:00 pm
ETLocation: Hyatt Regency Orlando, Rainbow Spring
2 Room, 9801 International Drive, Orlando,
FloridaWebcast: The Clinical Update meeting will
be webcast live and can be accessed from "Events Calendar" in the
News and Events section of the company's website
at LinkPresentation: The presentation will be
available as a PDF on the Company’s website at Link
Forward-Looking Statements
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 and regulatory submissions. We may, in some cases
use terms such as “predicts,” “believes,” “potential,” “continue,”
“anticipates,” “estimates,” “expects,” “plans,” “intends,” “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 and the
other risks set forth in our filings with the U.S. Securities and
Exchange Commission, including our Quarterly Report on Form
10-Q. 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.
Corporate Contacts:
Christian S. SchadePresident and Chief Executive
Officerchris.schade@aprea.com
Gregory A. KorbelVice President of Business
Developmentgreg.korbel@aprea.com
Source: Aprea Therapeutics, Inc.
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