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 and year
ended December 31, 2020 and provided a business update.
“Though disappointed the topline complete remission rate from
the Phase 3 clinical trial narrowly missed its primary endpoint, we
continue to analyze the totality of the data from the study to
understand those differences from our prior Phase 2 experience in
frontline MDS patients and expect to present these findings in the
second quarter of 2021,” said Christian S. Schade, Chairman and
Chief Executive Officer of Aprea. “Our dedicated team remains
committed to the clinical development of eprenetapopt and our next
generation, oral p53 reactivator, APR-548, in hematological and
solid tumor malignancies. In 2021, we look forward to sharing data
from our current clinical studies as well as our plans to expand
the clinical pipeline to include new indications.”
Business Operations Update:
The Company is conducting, supporting, and planning multiple
clinical trials of eprenetapopt (APR-246) and APR-548:
- Pivotal Phase 3 MDS
Trial—In December 2020, the Company announced its pivotal
Phase 3 randomized, controlled trial evaluating eprenetapopt with
azacitidine as frontline therapy in HMA-naïve TP53 mutant
myelodysplastic syndromes (MDS) patients failed to meet its
predefined primary endpoint of complete remission (CR) rate.
Analysis of the primary endpoint at this data cut demonstrated a
53% higher number of 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. The Company is completing analysis from this Phase 3
clinical trial and expects to present additional information in the
second quarter 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
Company anticipates initial results from the primary endpoint of
relapse-free survival 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 with 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 8 patients
across these expansion arms.
- APR-548 -- 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
early in the second quarter of 2021.
Fourth Quarter Financial Results
- Cash and
cash equivalents: As of December 31, 2020, the
Company had $89.0 million of cash and cash equivalents
compared to $130.1 million of cash and cash equivalents
as of December 31, 2019. 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 December 31, 2020 will be sufficient to meet its
current projected operating requirements into 2023.
- Research and
Development (R&D) expenses: R&D
expenses were $9.3 million for the quarter
ended December 31, 2020, compared to $8.0
million for the comparable period in 2019. The increase in
R&D expenses was primarily related to the continued development
of the Company’s lead product candidate, eprenetapopt, in the
following ongoing clinical trials; its pivotal Phase 3 clinical
trial of eprenetapopt with azacitidine for frontline treatment of
TP53 mutant MDS, its Phase 1/2 clinical trial for the treatment of
TP53 mutant AML with venetoclax and azacitidine, its Phase 1/2
clinical trial in relapsed/refractory gastric, bladder and
non-small cell lung cancers assessing eprenetapopt with anti-PD-1
therapy, its Phase 1 clinical trial in relapsed/refractory TP53
mutant chronic lymphoid leukemia (CLL) assessing eprenetapopt with
venetoclax and rituximab, and eprenetapopt with ibrutinib and its
Phase 2 post-transplant MDS/AML clinical trial.
- General and
Administrative (G&A) expenses: G&A
expenses were $4.9 million for the quarter
ended December 31, 2020, compared to $3.9
million for the comparable period in 2019. The increase in
G&A expenses was primarily due to increases in non-cash
stock-based compensation, insurance expense and commercial
development expense.
- Net
loss: Net loss was $15.4 million, or $0.73
per share for the quarter ended December 31, 2020, compared to
a net loss of $13.1 million, or $0.64 per share for the
quarter ended December 31, 2019. The Company had
21,186,827 shares of common stock outstanding as of December 31,
2020.
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, including
myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML).
Eprenetapopt has received Breakthrough Therapy, Orphan Drug and
Fast Track designations from the FDA for MDS, Fast Track
designation from the FDA for 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 is ongoing but at the
primary data cut, it failed to meet its predefined primary endpoint
of complete remission rate. Eprenetapopt has received Breakthrough
Therapy, Orphan Drug and Fast Track designations from the FDA for
MDS, 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. A Phase 1 clinical trial of APR-548
in TP53 MDS is planned.
Forward-Looking StatementCertain 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. CoianteSr. Vice President and Chief Financial
Officer617-463-9385
Gregory A. KorbelChief Business Officer617-463-9385
Aprea Therapeutics,
Inc.Condensed Consolidated Balance
Sheets
|
December 31, 2020 |
|
December 31, 2019 |
Assets |
|
|
|
|
|
Current assets: |
|
|
|
|
|
Cash and cash equivalents |
$89,017,686 |
|
|
$130,088,869 |
|
Prepaid expenses and other current assets |
|
3,399,019 |
|
|
|
2,955,878 |
|
Total current assets |
|
92,416,705 |
|
|
|
133,044,747 |
|
Property and equipment, net |
|
38,515 |
|
|
|
41,639 |
|
Right of use lease and other noncurrent assets |
|
349,999 |
|
|
|
521,499 |
|
Total assets |
$92,805,219 |
|
|
$133,607,885 |
|
|
|
|
|
|
|
Liabilities and Stockholders’ Equity |
|
|
Current liabilities: |
|
|
Accounts payable |
$4,503,619 |
|
|
$2,176,852 |
|
Accrued expenses |
|
10,571,237 |
|
|
|
6,642,553 |
|
Lease liability—current |
|
256,309 |
|
|
|
242,329 |
|
Total current liabilities |
|
15,331,165 |
|
|
|
9,061,734 |
|
Lease liability—noncurrent |
|
78,847 |
|
|
|
302,621 |
|
Total liabilities |
|
15,410,012 |
|
|
|
9,364,355 |
|
Commitments and contingencies |
|
|
Stockholders’ equity: |
|
|
Common stock, par value $0.001; 21,186,827 and 21,022,752, shares
issued and outstanding at December 31, 2020 and 2019,
respectively. |
|
21,187 |
|
|
|
21,023 |
|
Additional paid-in capital |
|
231,418,356 |
|
|
|
226,284,548 |
|
Accumulated other comprehensive loss |
|
(10,037,261 |
) |
|
|
(11,533,778 |
) |
Accumulated deficit |
|
(144,007,075 |
) |
|
|
(90,528,263 |
) |
Total stockholders’ equity |
|
77,395,207 |
|
|
|
124,243,530 |
|
Total liabilities and stockholders’ equity |
$92,805,219 |
|
|
$133,607,885 |
|
Aprea Therapeutics,
Inc.Condensed Consolidated Statements of
Operations and Comprehensive
Loss(Unaudited)
|
|
Three Months Ended December 31, |
|
Year Ended December 31, |
|
|
|
2020 |
|
|
2019 |
|
|
2020 |
|
|
2019 |
|
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
|
Research and development |
|
$ |
9,328,079 |
|
|
$ |
8,041,993 |
|
|
$ |
37,879,325 |
|
|
$ |
20,950,672 |
|
|
General and administrative |
|
|
4,895,323 |
|
|
|
3,937,765 |
|
|
|
14,931,887 |
|
|
|
8,593,626 |
|
|
Total operating expenses |
|
|
14,223,402 |
|
|
|
11,979,758 |
|
|
|
52,811,212 |
|
|
|
29,544,298 |
|
|
Other income (expense): |
|
|
|
|
|
|
|
|
|
|
|
|
|
Interest income |
|
|
4,744 |
|
|
|
169,888 |
|
|
|
222,652 |
|
|
|
156,351 |
|
|
Foreign currency (loss) gain |
|
|
(1,173,888 |
) |
|
|
(1,262,868 |
) |
|
|
(890,252 |
) |
|
|
1,328,140 |
|
|
Total other income (expense) |
|
|
(1,169,144 |
) |
|
|
(1,092,980 |
) |
|
|
(667,600 |
) |
|
|
1,484,491 |
|
|
Net loss |
|
$ |
(15,392,546 |
) |
|
$ |
(13,072,738 |
) |
|
$ |
(53,478,812 |
) |
|
$ |
(28,059,807 |
) |
|
Other comprehensive income
(loss): |
|
|
|
|
|
|
|
|
|
|
|
|
|
Foreign currency translation |
|
|
2,333,369 |
|
|
|
2,154,388 |
|
|
|
1,496,517 |
|
|
|
(2,772,453 |
) |
|
Total comprehensive loss |
|
|
(13,059,177 |
) |
|
|
(10,918,350 |
) |
|
|
(51,982,295 |
) |
|
|
(30,832,260 |
) |
|
Net loss per share attributable
to common stockholders, basic and diluted |
|
$ |
(0.73 |
) |
|
$ |
(0.64 |
) |
|
$ |
(2.53 |
) |
|
$ |
(4.67 |
) |
|
Weighted-average common shares
outstanding, basic and diluted |
|
|
21,186,827 |
|
|
|
20,318,040 |
|
|
|
21,133,651 |
|
|
|
6,002,486 |
|
|
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