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 and nine months
ended September 30, 2020 and provided a business update.
“We made solid progress during the third quarter
across our development pipeline as we approach top-line data from
the Phase 3 clinical trial evaluating eprenetapopt with azacitidine
for the treatment of front-line TP53 mutant myelodysplastic
syndromes (MDS) patients by year-end 2020” said Christian S.
Schade, Chairman and Chief Executive Officer of Aprea. “We
continue to execute on our goal of expanding the clinical
opportunities for our p53 reactivator programs, including expansion
of our front-line AML clinical trials, enrollment of the first
patients in our solid tumor trial, initiation of our lymphoma trial
and clearance from FDA to proceed with the Phase 1 trial of our
next-generation p53 reactivator, APR-548.”
Business Operations
Update:
The Company is conducting, supporting, and planning
multiple clinical trials of eprenetapopt (APR-246):
- Pivotal Phase 3 MDS Trial—The Company has
completed the full enrollment of 154 patients in its pivotal Phase
3 randomized, controlled trial evaluating eprenetapopt with
azacitidine as frontline therapy in HMA-naïve TP53 mutant
myelodysplastic syndromes (MDS) patients with a primary endpoint of
complete remission (CR) rate. The Company remains confident it will
have top-line data available by year-end 2020.
- Phase 2 MDS/AML Post-Transplant Trial—The
Company has completed the target enrollment of 31 patients in its
single-arm, open-label Phase 2 trial evaluating eprenetapopt with
azacitidine as post-transplant maintenance therapy in TP53 mutant
MDS and acute myeloid leukemia (AML) patients who have received an
allogeneic stem cell transplant.
- Phase 1/2 AML Trial—The Company is currently
enrolling its Phase 1/2 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 approximately 30 additional
frontline TP53 mutant AML patients with the combination of
eprenetapopt, venetoclax and azacitidine. The Company also plans to
activate a separate cohort in the trial to evaluate the combination
of eprenetapopt with azacitidine in approximately 30 frontline TP53
mutant AML patients.
- Phase 1 NHL Trial—The Company has designed and
plans to conduct 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.
A poster describing the clinical trial has been accepted for
presentation at the 62nd American Society of Hematology (ASH)
Annual Meeting (abstract # 1311) on December 5, 2020 from 10:00 am
to 6:30 pm eastern time. The Company is targeting the first patient
to be enrolled in the fourth quarter of 2020.
- Phase 1/2 Solid Tumor Trial—Based on in vivo
data suggesting synergistic activity between eprenetapopt and
immuno-therapy agents including anti-PD-1 antibody, the Company has
designed and is conducting Phase 1/2 clinical trials in
relapsed/refractory gastric, bladder and non-small cell lung
cancers assessing eprenetapopt with anti-PD-1 therapy. Five
patients have been enrolled in the safety review cohort of this
trial.
- APR-548 -- The Company’s second product
candidate, APR-548, is a pre-clinical, next-generation p53
reactivator with the potential for oral administration. APR-548
exhibits high oral bioavailability in preclinical testing and is
being developed in an oral dosage form. The Company completed
Investigational New Drug, or IND, enabling preclinical studies of
APR-548 and filed an IND with the FDA. The Company received
clearance from the FDA in October 2020 to initiate Phase 1 clinical
trials for APR-548. The Company anticipates enrollment in the Phase
1 clinical trial to begin in the first quarter of 2021.
Third Quarter Financial
Results
- Cash and cash equivalents: As of
September 30, 2020, the Company had $101.1 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 2020 to be between
$35.0 million $40.0 million. The Company believes its cash
and cash equivalents as of September 30, 2020 will be
sufficient to meet its current projected operating requirements
into 2023.
- Research and Development (R&D)
expenses: R&D expenses were $8.8
million for the quarter ended September 30, 2020,
compared to $4.9 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; our pivotal
Phase 3 clinical trial of eprenetapopt with azacitidine for
frontline treatment of TP53 mutant MDS, our Phase 1/2 clinical
trials in relapsed/refractory gastric, bladder and non-small cell
lung cancers assessing eprenetapopt with anti-PD-1 therapy, our
Phase 1 clinical trial in relapsed/refractory TP53 mutant chronic
lymphoid leukemia (CLL) assessing eprenetapopt with venetoclax and
rituximab, and eprenetapopt with ibrutinib and our Phase 2
post-transplant MDS/AML clinical trial.
- General and Administrative (G&A)
expenses: G&A expenses were $3.5
million for the quarter ended September 30, 2020,
compared to $2.3 million for the comparable period in
2019. The increase in G&A expenses was primarily due to
increased non-cash stock-based compensation, increased insurance
expense and increased commercial development expense.
- Net loss: Net loss was $12.3
million, or $0.58 per share for the quarter ended September
30, 2020, compared to a net loss of $6.2 million, or $5.29 per
share for the quarter ended September 30, 2019. The Company
had 21,186,827 shares of common stock outstanding as of September
30, 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, 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. 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 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. CoianteSr. Vice President and Chief
Financial Officer617-463-9385
Gregory A. KorbelVice President of Business
Development617-463-9385
Aprea Therapeutics,
Inc.Condensed Consolidated Balance
Sheets(Unaudited)
|
|
|
|
September 30, 2020 |
|
December 31, 2019 |
Assets |
|
|
Current assets: |
|
|
Cash and cash equivalents |
$ |
101,146,633 |
|
|
$ |
130,088,869 |
|
Prepaid expenses and other current assets |
|
1,257,077 |
|
|
|
2,955,878 |
|
Total current assets |
|
102,403,710 |
|
|
|
133,044,747 |
|
Property and equipment, net |
|
0,696 |
|
|
|
41,639 |
|
Right of use lease and other
noncurrent assets |
|
389,982 |
|
|
|
521,499 |
|
Total assets |
$ |
102,834,388 |
|
|
$ |
133,607,885 |
|
Liabilities and
Stockholders’ Equity |
|
|
Current liabilities: |
|
|
Accounts payable |
$ |
4,349,977 |
|
|
$ |
2,176,852 |
|
Accrued expenses |
|
9,084,389 |
|
|
|
6,642,553 |
|
Lease liability—current |
|
244,310 |
|
|
|
242,329 |
|
Total current liabilities |
|
13,678,676 |
|
|
|
9,061,734 |
|
Lease liability—noncurrent |
|
132,773 |
|
|
|
302,621 |
|
Total liabilities |
|
13,811,449 |
|
|
|
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 September 30, 2020 and December 31,
2019, respectively |
|
21,187 |
|
|
|
21,023 |
|
Additional paid‑in capital |
|
229,986,911 |
|
|
|
226,284,548 |
|
Accumulated other comprehensive loss |
|
(12,370,630 |
) |
|
|
(11,533,778 |
) |
Accumulated deficit |
|
(128,614,529 |
) |
|
|
(90,528,263 |
) |
Total stockholders’ equity. |
|
89,022,939 |
|
|
|
124,243,530 |
|
Total liabilities and stockholders’ equity |
$ |
102,834,388 |
|
|
$ |
133,607,885 |
|
|
|
|
|
|
|
|
|
Aprea Therapeutics,
Inc.Condensed Consolidated Statements of
Operations and Comprehensive
Loss(Unaudited)
|
|
|
|
|
Three Months Ended September 30, |
|
Nine Months Ended September 30, |
|
2020 |
|
2019 |
|
2020 |
|
2019 |
Operating expenses: |
|
|
|
|
|
|
|
|
|
|
|
Research and development |
$ |
8,761,095 |
|
|
$ |
4,910,409 |
|
|
$ |
28,551,246 |
|
|
$ |
12,908,679 |
|
General and administrative |
|
3,473,210 |
|
|
|
2,307,946 |
|
|
|
10,036,564 |
|
|
|
4,655,861 |
|
Total operating expenses |
|
12,234,305 |
|
|
|
7,218,355 |
|
|
|
38,587,810 |
|
|
|
17,564,540 |
|
Other income (expense): |
|
|
|
|
|
|
|
|
|
|
|
Interest income (expense) |
|
(9,212 |
) |
|
|
(6,098 |
) |
|
|
217,908 |
|
|
|
(13,537 |
) |
Foreign currency (loss) gain |
|
(74,565 |
) |
|
|
975,034 |
|
|
|
283,636 |
|
|
|
2,591,008 |
|
Total other income (expense) |
|
(83,777 |
) |
|
|
968,936 |
|
|
|
501,544 |
|
|
|
2,577,471 |
|
Net loss |
$ |
(12,318,082 |
) |
|
$ |
(6,249,419 |
) |
|
$ |
(38,086,266 |
) |
|
$ |
(14,987,069 |
) |
Other comprehensive income
(loss): |
|
|
|
|
|
|
|
|
|
|
|
Foreign currency translation |
|
(168,982 |
) |
|
|
(2,940,174 |
) |
|
|
(836,852 |
) |
|
|
(4,926,841 |
) |
Total comprehensive loss |
|
(12,487,064 |
) |
|
|
(9,189,593 |
) |
|
|
(38,923,118 |
) |
|
|
(19,913,910 |
) |
Net loss per share attributable
to common stockholders, basic and diluted |
$ |
(0.58 |
) |
|
$ |
(5.29 |
) |
|
$ |
(1.80 |
) |
|
$ |
(12.72 |
) |
Weighted-average common shares
outstanding, basic and diluted |
|
21,186,827 |
|
|
|
1,181,726 |
|
|
|
21,115,797 |
|
|
|
1,178,206 |
|
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