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 reported financial results for the three months
ended March 31, 2021 and provided a business update.
“During our recent R&D Day presentation, we reported an
analysis of available data from its Phase 3 MDS trial. We
identified an imbalance in dose modifications in the experimental
arm which we believe negatively impacted efficacy, particularly the
primary CR endpoint,” said Christian S. Schade, Chairman and Chief
Executive Officer of Aprea. “We remain confident that eprenetapopt
and our next-generation oral agent, APR-548, represent important
potential therapeutic options for cancer patients and are
encouraged by emerging data from our ongoing clinical trials. We
look forward to sharing data updates from these clinical trials as
well as our continued progress in expanding the opportunity for our
therapies in new indications.”
Business Operations Update:
The Company is conducting, supporting, and planning multiple
clinical trials of eprenetapopt (APR-246) and APR-548:
- Phase 3 Frontline MDS Trial
-- In June 2020, the Company completed full enrollment of
154 patients in a pivotal Phase 3 trial of eprenetapopt with
azacitidine for frontline treatment of patients with TP53 mutant
MDS. The pivotal Phase 3 trial is supported by data from two
Phase 1b/2 investigator-initiated trials, one in the U.S. and
one in France, testing eprenetapopt with azacitidine as frontline
treatment in TP53 mutant MDS and AML patients. The data from the
U.S. and French Phase 1b/2 trials were published in The Journal of
Clinical Oncology in January 2021 and February 2021, respectively.
In December 2020, the Company announced that its pivotal Phase 3
trial failed to meet its predefined primary endpoint of complete
remission (CR) rate. Analysis of the primary endpoint at this data
cut demonstrated a higher CR rate (53% more patients achieving a
CR) in the experimental arm receiving eprenetapopt with azacitidine
versus the control arm receiving azacitidine alone but did not
reach statistical significance. Based on a thorough analysis of the
current Phase 3 trial data and comparisons to the U.S. and French
Phase 1b/2 trials the Company believes that despite similar types
and frequency of adverse events observed in the Phase 3
experimental arm and the Phase 1b/2 trials, patients in the Phase 3
experimental arm experienced substantially more study treatment
dose modifications compared to the experience in the U.S. and
French Phase 1b/2 trials. The Company believes that dose
modifications of eprenetapopt and azacitidine led to undertreatment
in the Phase 3 experimental arm that negatively impacted efficacy,
particularly the primary endpoint of CR rate. The Company continues
to follow patients who remain on-study and anticipates discussing
with FDA the Phase 3 data and future possible regulatory pathways
in the second half of 2021.
- Phase 2 MDS/AML
Post-Transplant Trial – The Company has completed
enrollment of 33 patients in a single-arm, open-label Phase 2
clinical trial evaluating eprenetapopt with azacitidine as
post-transplant maintenance therapy in TP53 mutant MDS and AML
patients who have received an allogeneic stem cell transplant. The
primary endpoint of the trial is the rate of relapse-free survival
(RFS) at 12 months, with a published benchmark of ~30%. An interim
analysis in April 2021 showed a 62% rate of RFS at 12 months, with
a median RFS of 462 days. An interim analysis of overall survival
(OS) showed a 77% OS at 1 year, with a median number of events not
yet reached. The Company anticipates initial results from the
primary endpoint of RFS at 12 months in the second quarter of
2021.
- Phase 1/2 AML
Trial – The Company is currently enrolling a Phase 1/2
clinical trial evaluating the safety, tolerability, and preliminary
efficacy of eprenetapopt therapy in TP53 mutant AML patients. The
lead-in portion of the trial evaluated the tolerability of
eprenetapopt with venetoclax, with or without azacitidine, and no
dose-limiting toxicities were observed in 12 patients receiving
either regimen. Based on these results, the Company has expanded
the trial to treat 33 additional frontline TP53 mutant AML patients
with the combination of eprenetapopt, venetoclax and azacitidine.
In the 19 frontline AML patients who are evaluable for efficacy
with the triplet regimen, the Company has observed a 63% CR + CRi
composite response rate and a 31% CR rate. The Company anticipates
completion of enrollment in the triplet regimen expansion cohort
during the second quarter of 2021 and availability of preliminary
response rate data from the cohort also in the second quarter of
2021.
- Phase 1 NHL Trial
– The Company is currently enrolling a Phase 1 clinical trial in
relapsed/refractory TP53 mutant chronic lymphoid leukemia (CLL)
assessing eprenetapopt with venetoclax and rituximab and
eprenetapopt with ibrutinib in order to further assess eprenetapopt
in hematological malignancies. The first patient was enrolled in
the first quarter of 2021. The Company is also planning to evaluate
the combination of eprenetapopt with venetoclax in
relapsed/refractory mantle cell lymphoma.
- Phase 1/2 Solid Tumor
Trial – The Company is currently enrolling a Phase 1/2
clinical trial in relapsed/refractory gastric, bladder and
non-small cell lung cancers assessing eprenetapopt with anti-PD-1
therapy. The dose-escalation phase of the trial enrolled 6 patients
with advanced solid tumors and no dose-limiting toxicities were
observed. Based on these results, the Company is enrolling
expansion cohorts for patients with advanced gastric, bladder and
non-small cell lung cancers and has currently enrolled 15 patients
across these expansion arms. A poster presentation for this trial
has been accepted for presentation at the 2021 ASCO Annual Meeting
(abstract TPS3161).
- APR-548 Phase 1 Trial
-- The Company’s second product candidate, APR-548, is a
next-generation p53 reactivator that is being developed in an oral
dosage form. The Company has planned a Phase 1 dose-escalation
clinical trial evaluating the safety, tolerability, and preliminary
efficacy of APR-548 with azacitidine in frontline and
relapsed/refractory MDS patients. The Company anticipates the first
patient to be enrolled in the second quarter of 2021.
First Quarter Financial Results
- Cash and
cash equivalents: As of March 31, 2021, the
Company had $77.6 million of cash and cash equivalents
compared to $89.0 million of cash and cash equivalents as
of December 31, 2020. The Company expects cash burn for
the full year 2021 to be between $30.0 million $35.0 million. The
Company believes its cash and cash equivalents as of March 31,
2021 will be sufficient to meet its current projected
operating requirements into 2023.
- Research and
Development (R&D) expenses: R&D
expenses were $6.8 million for the quarter
ended March 31, 2021, compared to $9.1 million for
the comparable period in 2020. The decrease in R&D expenses was
primarily due to decreases in clinical trial costs for (i) our
pivotal Phase 3 clinical trial of eprenetapopt with azacitidine for
the frontline treatment of TP53 mutant MDS which completed
enrollment in Q2 2020 and (ii) our Phase 2 post-transplant MDS/AML
clinical trial. These decreases were partially offset by increases
in clinical trial costs for our Phase 1/2 clinical trial for the
treatment of TP53 mutant AML with venetoclax and azacitidine, our
Phase 1/2 clinical trial in relapsed/refractory gastric, bladder
and non-small cell lung cancers assessing eprenetapopt with
anti-PD-1 therapy, and our Phase 1 clinical trial in
relapsed/refractory TP53 mutant chronic lymphoid leukemia (CLL)
assessing eprenetapopt with venetoclax and rituximab, and
eprenetapopt with ibrutinib.
- General and
Administrative (G&A) expenses: G&A
expenses were $3.4 million for the quarter
ended March 31, 2021, compared to $2.8 million for
the comparable period in 2020. The increase in G&A
expenses was primarily due to increases in non-cash stock-based
compensation and insurance expense.
- Net
loss: Net loss was $9.7 million, or $0.46
per share for the quarter ended March 31, 2021, compared to a
net loss of $9.4 million, or $0.45 per share for the quarter
ended March 31, 2020. The Company had 21,186,827
shares of common stock outstanding as of March 31, 2021.
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 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, 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 cancer.
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.
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. CoianteSr. Vice President and Chief Financial
Officer617-463-9385
Gregory A. KorbelSr. Vice President and Chief Business
Officer617-463-9385
|
Aprea Therapeutics, Inc.Condensed
Consolidated Balance
Sheets(Unaudited) |
|
|
|
|
|
|
|
|
|
March 31, 2021 |
|
December 31, 2020 |
Assets |
|
|
|
|
|
|
|
Current assets: |
|
|
|
|
|
|
|
Cash and cash equivalents |
$ |
77,616,074 |
|
|
$ |
89,017,686 |
|
Prepaid expenses and other current assets |
|
2,467,443 |
|
|
|
3,399,019 |
|
Total current assets |
|
80,083,517 |
|
|
|
92,416,705 |
|
Property and equipment, net |
|
33,572 |
|
|
|
38,515 |
|
Right of use lease and other
noncurrent assets |
|
277,576 |
|
|
|
349,999 |
|
Total assets |
$ |
80,394,665 |
|
|
$ |
92,805,219 |
|
Liabilities and
Stockholders’ Equity |
|
|
|
|
|
|
|
Current liabilities: |
|
|
|
|
|
|
|
Accounts payable |
$ |
3,456,728 |
|
|
$ |
4,503,619 |
|
Accrued expenses |
|
7,532,473 |
|
|
|
10,571,237 |
|
Lease liability—current |
|
225,537 |
|
|
|
256,309 |
|
Total current liabilities |
|
11,214,738 |
|
|
|
15,331,165 |
|
Lease liability—noncurrent |
|
33,763 |
|
|
|
78,847 |
|
Total liabilities |
|
11,248,501 |
|
|
|
15,410,012 |
|
Commitments and
contingencies |
|
|
|
|
|
|
|
Stockholders’ equity: |
|
|
|
|
|
|
|
Common stock, par value $0.001; 21,186,827 shares issued and
outstanding at March 31, 2021 and December 31, 2020,
respectively. |
|
21,187 |
|
|
|
21,187 |
|
Additional paid-in capital |
|
233,240,918 |
|
|
|
231,418,356 |
|
Accumulated other comprehensive loss |
|
(10,440,111 |
) |
|
|
(10,037,261 |
) |
Accumulated deficit |
|
(153,675,830 |
) |
|
|
(144,007,075 |
) |
Total stockholders’ equity |
|
69,146,164 |
|
|
|
77,395,207 |
|
Total liabilities and stockholders’ equity |
$ |
80,394,665 |
|
|
$ |
92,805,219 |
|
|
|
|
|
|
|
|
|
|
Aprea Therapeutics, Inc.Condensed
Consolidated Statements of Operations and Comprehensive
Loss(Unaudited) |
|
|
|
|
|
Three Months Ended March 31, |
|
|
2021 |
|
2020 |
Operating expenses: |
|
|
|
|
|
|
Research and development |
|
$ |
6,763,848 |
|
|
$ |
9,096,122 |
|
General and administrative |
|
|
3,425,833 |
|
|
|
2,776,468 |
|
Total operating expenses |
|
|
10,189,681 |
|
|
|
11,872,590 |
|
Other income (expense): |
|
|
|
|
|
|
Interest (expense) income |
|
|
(1,057 |
) |
|
|
224,442 |
|
Foreign currency (loss) gain |
|
|
521,983 |
|
|
|
2,247,891 |
|
Total other income (expense) |
|
|
520,926 |
|
|
|
2,472,333 |
|
Net loss |
|
$ |
(9,668,755 |
) |
|
$ |
(9,400,257 |
) |
Other comprehensive income
(loss): |
|
|
|
|
|
|
Foreign currency translation |
|
|
(402,850 |
) |
|
|
(2,424,653 |
) |
Total comprehensive loss |
|
|
(10,071,605 |
) |
|
|
(11,824,910 |
) |
Net loss per share attributable
to common stockholders, basic and diluted |
|
$ |
(0.46 |
) |
|
$ |
(0.45 |
) |
Weighted-average common shares
outstanding, basic and diluted |
|
|
21,186,827 |
|
|
|
21,052,726 |
|
|
|
|
|
|
|
|
|
|
Aprea Therapeutics (NASDAQ:APRE)
Historical Stock Chart
From Mar 2024 to Apr 2024
Aprea Therapeutics (NASDAQ:APRE)
Historical Stock Chart
From Apr 2023 to Apr 2024