Applied Therapeutics Reports Second Quarter 2023 Financial Results
10 Agosto 2023 - 6:00AM
Applied Therapeutics, Inc. (Nasdaq: APLT), a clinical-stage
biopharmaceutical company developing a pipeline of novel drug
candidates against validated molecular targets in indications of
high unmet medical need, today reported financial results for the
second quarter ended June 30, 2023.
“In the second quarter we continued to advance
our programs across Galactosemia, SORD Deficiency and Diabetic
Cardiomyopathy,” said Shoshana Shendelman, PhD, Founder, Chief
Executive Officer, and Chair of the Board. “As we move into the
second half of this year, our efforts will focus on regulatory
progress in Galactosemia and SORD Deficiency, and on Phase 3
clinical readouts in SORD Deficiency and DbCM. We remain steadfast
in our commitment to bring govorestat (AT-007) and caficrestat
(AT-001) to patients with these devastating diseases in need of
treatment.”
Recent Highlights
- Regulatory Progress on Govorestat (AT-007) for the
Treatment of Classic Galactosemia. The Company announced
that the United States Food and Drug Administration (US FDA)
granted a Pre-New Drug Application (Pre-NDA) meeting to be held
this summer to discuss a potential NDA submission for govorestat
(AT-007) for the treatment of Galactosemia. The Company believes
that the clinical efficacy demonstrated to date, combined with
galactitol biomarker data and a favorable safety profile, may
support an NDA submission, and is seeking feedback from the FDA and
alignment on the details of the submission. If the FDA is in
agreement on the potential path forward to approval, the Company
will plan to submit an NDA in the fall. The Company expects to
provide a further update following the meeting. Regarding
regulatory submission plans in Europe, Applied Therapeutics and its
European commercial partner, Advanz Pharma, met with the European
Medicines Agency (EMA) rapporteurs, and plan to proceed with an EMA
Marketing Authorization Application (MAA) submission as
expeditiously as possible. The EMA submission is anticipated in the
fall in order to provide sufficient time for approval of the
Pediatric Investigational Plan (PIP), as well as incorporation of
rapporteur comments and suggestions from the meeting.
- Presented Baseline Data from Ongoing Phase 3 ARISE-HF
Study of AT-001 (caficrestat) in Diabetic Cardiomyopathy at the
2023 Annual Meeting of the American Diabetes Association
(ADA). In June 2023, the Company presented baseline data
at the 2023 Annual Meeting of the ADA from the ongoing Phase 3
ARISE-HF study of AT-001 (caficrestat) in Diabetic Cardiomyopathy
(DbCM). The data, presented in an oral symposium and two poster
presentations, demonstrated a strong statistical correlation
between elevations in the cardiac stress biomarker NT-proBNP,
reduced cardiac functional capacity, and physical activity,
underscoring the negative impact of DbCM on physical function and
quality of life in patients. The ongoing ARISE-HF Phase 3 global
clinical trial is evaluating the safety and efficacy of AT-001
(caficrestat) in improving or preventing worsening of cardiac
functional capacity in Diabetic Cardiomyopathy (DbCM). The Company
expects topline data from the study in the fourth quarter of
2023.
- Presented Baseline Data, Clinical Trial Design, and
3-month Sorbitol Reduction Interim Analysis from Phase 3 INSPIRE
Study of Govorestat (AT-007) in SORD Deficiency and new supporting
preclinical data at 2023 Peripheral Nerve Society Annual
Meeting. Also in June 2023, the Company presented baseline
data and 3-month sorbitol reduction interim data from the Phase 3
INSPIRE study of govorestat for the treatment of Sorbitol
Dehydrogenase (SORD) Deficiency, as well as new preclinical data
supporting the ongoing Phase 3 study, at the 2023 Annual Meeting of
the Peripheral Nerve Society. The preclinical data, showcased in an
oral presentation at the Annual Meeting of the Peripheral Nerve
Society, demonstrated that sorbitol accumulation is a disease
driver in patient-derived cells as well as in animal models,
including a newly presented rat model. The oral presentations also
included the trial design of the Phase 3 INSPIRE study.
- Orphan Medicinal Product Designation Granted by EMA to
Govorestat in SORD Deficiency. In May 2023, the Company
announced that the European Medicines Agency (EMA) granted Orphan
Medicinal Product Designation to govorestat for the treatment of
SORD Deficiency. Orphan Medicinal Product Designation provides
certain benefits and incentives in the EU, including protocol
assistance, fee reductions, and ten years of market exclusivity
once the medicine is on the market.
- New Preclinical Data Published in the Journal
of Clinical Investigation Showing Reduction of Sorbitol in
SORD-Deficient Patient-Derived Cells and Drosophila Disease Model
Treated with Govorestat. Also in May 2023, the Company
announced that new data was published in the Journal of Clinical
Investigation evaluating govorestat treatment in patient-derived
fibroblasts, patient iPSC-derived motor neurons, and SORD-deficient
drosophila. The data demonstrated that govorestat treatment
significantly reduced sorbitol levels in the patient-derived cells
and SORD-deficient drosophila, and also mitigated synaptic
degeneration and significantly improved synaptic transduction,
locomotor activity, and mitochondrial function in the
SORD-deficient drosophila.
Financial Results
- Cash and
cash equivalents and short-term investments totaled $35.6
million as of June 30, 2023, compared with $30.6 million at
December 31, 2022.
- Research
and development expenses for the three months ended June
30, 2023 were $11.9 million, compared to $15.4 million for the
three months ended June 30, 2022. The decrease of approximately
$3.5 million was primarily related to a decrease in clinical and
pre-clinical expense of $3.7 million, primarily due to the decrease
in expense related to CROs, offset by the progression of the SORD
Phase 3 registrational study; an increase in drug manufacturing and
formulation costs of $0.4 million primarily related to purchase of
raw materials in the three months ended June 30, 2023; a decrease
in personnel expenses of $0.2 million due to the decrease in
headcount; a decrease in stock-based compensation of $36,000 due to
decrease in headcount which resulted in options and restricted
stock units being forfeited; and a decrease in regulatory and other
expenses of $0.1 million.
- General
and administrative expenses were $5.3 million for the
three months ended June 30, 2023, compared to $6.1 million for the
three months ended June 30, 2022. The decrease of approximately
$0.8 million was primarily related to an increase in legal and
professional fees of $0.2 million due to higher external legal
fees; a decrease in commercial expenses of $13,000 related to a
decrease in spend for commercial operations; a decrease in
personnel expenses of $0.4 million related to a decrease in
headcount; a decrease in stock-based compensation of $0.4 million
relating to options being forfeited during the current period as
well as decrease in headcount; a decrease in insurance expenses of
$0.3 million related to decreased insurance costs; and a decrease
in other expenses of $30,000 relating to decreased costs of other
office expenses.
-
Net loss for the second quarter of 2023 was $29.6
million, or $0.37 per basic and diluted common share, compared to a
net loss of $25.9 million, or $0.96 per basic and diluted common
share, for the second quarter 2022.
About Applied Therapeutics
Applied Therapeutics is a clinical-stage biopharmaceutical
company developing a pipeline of novel drug candidates against
validated molecular targets in indications of high unmet medical
need. The Company’s lead drug candidate, govorestat, is a novel
central nervous system penetrant Aldose Reductase Inhibitor (ARI)
for the treatment of CNS rare metabolic diseases, including
Galactosemia, SORD Deficiency, and PMM2-CDG. The Company is also
developing AT-001, a novel potent ARI, for the treatment of
Diabetic Cardiomyopathy, or DbCM, a fatal fibrosis of the heart.
The preclinical pipeline also includes AT-003, an ARI designed to
cross through the back of the eye when dosed orally, for the
treatment of Diabetic retinopathy.
To learn more, please visit
www.appliedtherapeutics.com and follow the company on Twitter
@Applied_Tx.
Forward-Looking Statements
This press release contains “forward-looking statements” that
involve substantial risks and uncertainties for purposes of the
safe harbor provided by the Private Securities Litigation Reform
Act of 1995. Any statements, other than statements of historical
fact, included in this press release regarding the strategy, future
operations, prospects, plans and objectives of management,
including words such as “may,” “will,” “expect,” “anticipate,”
“plan,” “intend,” and similar expressions (as well as other words
or expressions referencing future events, conditions or
circumstances) are forward-looking statements. These include,
without limitation, statements regarding (i) our plans to request a
pre-NDA meeting or submit an NDA or MAA for approval and (ii) the
anticipated cash runway of the Company. Forward-looking statements
in this release involve substantial risks and uncertainties that
could cause actual results to differ materially from those
expressed or implied by the forward-looking statements, and we,
therefore cannot assure you that our plans, intentions,
expectations or strategies will be attained or achieved.
Such risks and uncertainties include, without limitation, (i)
our plans to develop, market and commercialize our product
candidates, (ii) the initiation, timing, progress and results of
our current and future preclinical studies and clinical trials and
our research and development programs, (iii) our ability to take
advantage of expedited regulatory pathways for any of our product
candidates, (iv) our estimates regarding expenses, future revenue,
capital requirements and needs for additional financing, (v) our
ability to successfully acquire or license additional product
candidates on reasonable terms and advance product candidates into,
and successfully complete, clinical studies, (vi) our ability to
maintain and establish collaborations or obtain additional funding,
(vii) our ability to obtain and timing of regulatory approval of
our current and future product candidates, (viii) the anticipated
indications for our product candidates, if approved, (ix) our
expectations regarding the potential market size and the rate and
degree of market acceptance of such product candidates, (x) our
ability to fund our working capital requirements and expectations
regarding the sufficiency of our capital resources, (xi) the
implementation of our business model and strategic plans for our
business and product candidates, (xii) our intellectual property
position and the duration of our patent rights, (xiii) developments
or disputes concerning our intellectual property or other
proprietary rights, (xiv) our expectations regarding government and
third-party payor coverage and reimbursement, (xv) our ability to
compete in the markets we serve, (xvi) the impact of government
laws and regulations and liabilities thereunder, (xvii)
developments relating to our competitors and our industry, (xvii)
our ability to achieve the anticipated benefits from the agreements
entered into in connection with our partnership with Advanz Pharma
and (xiv) other factors that may impact our financial results. In
light of the significant uncertainties in these forward-looking
statements, you should not rely upon forward-looking statements as
predictions of future events. Although we believe that we have a
reasonable basis for each forward-looking statement contained in
this press release, we cannot guarantee that the future results,
levels of activity, performance or events and circumstances
reflected in the forward-looking statements will be achieved or
occur at all. Factors that may cause actual results to differ from
those expressed or implied in the forward-looking statements in
this press release are discussed in our filings with the U.S.
Securities and Exchange Commission, including the “Risk Factors”
contained therein. Except as otherwise required by law, we disclaim
any intention or obligation to update or revise any forward-looking
statements, which speak only as of the date they were made, whether
as a result of new information, future events or circumstances or
otherwise.
Contacts
Investors:Maeve Conneighton (212) 600-1902
orappliedtherapeutics@argotpartners.com
Media:media@appliedtherapeutics.com
Applied Therapeutics, Inc.Condensed
Balance Sheets(in thousands, except share and per
share data) |
|
|
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As of |
|
As of |
|
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June 30, |
|
December 31, |
|
|
2023 |
|
|
2022 |
|
|
|
(Unaudited) |
|
|
ASSETS |
|
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|
|
|
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CURRENT ASSETS: |
|
|
|
|
|
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Cash and cash equivalents |
|
$ |
35,616 |
|
|
$ |
16,657 |
|
Investments |
|
|
— |
|
|
|
13,923 |
|
Prepaid expenses and other current assets |
|
|
7,205 |
|
|
|
6,728 |
|
Total current assets |
|
|
42,821 |
|
|
|
37,308 |
|
Operating lease right-of-use asset |
|
|
628 |
|
|
|
857 |
|
Security deposits and leasehold improvements |
|
|
197 |
|
|
|
198 |
|
TOTAL ASSETS |
|
$ |
43,646 |
|
|
$ |
38,363 |
|
LIABILITIES AND
STOCKHOLDERS’ (DEFICIT)/EQUITY |
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CURRENT LIABILITIES: |
|
|
|
|
|
|
Current portion of operating lease liabilities |
|
$ |
486 |
|
|
$ |
477 |
|
Accounts payable |
|
|
4,687 |
|
|
|
4,534 |
|
Accrued expenses and other current liabilities |
|
|
16,023 |
|
|
|
14,756 |
|
Warrant liability |
|
|
25,992 |
|
|
|
13,657 |
|
Total current liabilities |
|
|
47,188 |
|
|
|
33,424 |
|
NONCURRENT LIABILITIES: |
|
|
|
|
|
|
Noncurrent portion of operating lease liabilities |
|
|
170 |
|
|
|
414 |
|
Clinical holdback - long-term portion |
|
|
622 |
|
|
|
464 |
|
Total noncurrent liabilities |
|
|
792 |
|
|
|
878 |
|
Total liabilities |
|
|
47,980 |
|
|
|
34,302 |
|
STOCKHOLDERS’
(DEFICIT)/EQUITY: |
|
|
|
|
|
|
Common stock, $0.0001 par value; 200,000,000 shares authorized as
of June 30, 2023 and December 31, 2022;
62,119,466 shares issued and outstanding as of
June 30, 2023 and 48,063,358 shares issued and
outstanding as of December 31, 2022 |
|
|
6 |
|
|
|
5 |
|
Preferred stock, par value $0.0001; 10,000,000 shares authorized as
of June 30, 2023 and December 31, 2022; 0
shares issued and outstanding as of June 30, 2023 and
December 31, 2022 |
|
|
— |
|
|
|
— |
|
Additional paid-in capital |
|
|
384,197 |
|
|
|
352,828 |
|
Accumulated other comprehensive gain/(loss) |
|
|
— |
|
|
|
51 |
|
Accumulated deficit |
|
|
(388,537 |
) |
|
|
(348,823 |
) |
Total stockholders' (deficit)/equity |
|
|
(4,334 |
) |
|
|
4,061 |
|
TOTAL LIABILITIES AND STOCKHOLDERS’ (DEFICIT)/EQUITY |
|
$ |
43,646 |
|
|
$ |
38,363 |
|
Applied Therapeutics, Inc.Condensed
Statements of Operations(in thousands, except
share and per share data)(Unaudited) |
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Three Months Ended |
|
Six Months Ended |
|
|
June 30, |
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June 30, |
|
|
2023 |
|
|
2022 |
|
|
2023 |
|
|
2022 |
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REVENUE: |
|
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|
|
|
|
|
|
|
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License Revenue |
|
$ |
— |
|
|
$ |
— |
|
|
$ |
10,660 |
|
|
$ |
— |
|
Total Revenue |
|
|
— |
|
|
|
— |
|
|
|
10,660 |
|
|
|
— |
|
OPERATING EXPENSES: |
|
|
|
|
|
|
|
|
|
|
|
|
Research and development |
|
$ |
11,883 |
|
|
$ |
15,396 |
|
|
$ |
27,818 |
|
|
$ |
30,426 |
|
General and administrative |
|
|
5,293 |
|
|
|
6,125 |
|
|
|
10,876 |
|
|
|
14,196 |
|
Total operating expenses |
|
|
17,176 |
|
|
|
21,521 |
|
|
|
38,694 |
|
|
|
44,622 |
|
LOSS FROM OPERATIONS |
|
|
(17,176 |
) |
|
|
(21,521 |
) |
|
|
(28,034 |
) |
|
|
(44,622 |
) |
OTHER INCOME (EXPENSE),
NET: |
|
|
|
|
|
|
|
|
|
|
|
|
Interest income |
|
|
408 |
|
|
|
111 |
|
|
|
628 |
|
|
|
187 |
|
Change in fair value of warrant liabilities |
|
|
(12,804 |
) |
|
|
(4,357 |
) |
|
|
(12,335 |
) |
|
|
(4,357 |
) |
Other expense |
|
|
(5 |
) |
|
|
(90 |
) |
|
|
27 |
|
|
|
(186 |
) |
Total other expense, net |
|
|
(12,401 |
) |
|
|
(4,336 |
) |
|
|
(11,680 |
) |
|
|
(4,356 |
) |
Net loss |
|
$ |
(29,577 |
) |
|
$ |
(25,857 |
) |
|
$ |
(39,714 |
) |
|
$ |
(48,978 |
) |
Net loss attributable to
common stockholders—basic and diluted |
|
$ |
(29,577 |
) |
|
$ |
(25,857 |
) |
|
$ |
(39,714 |
) |
|
$ |
(48,978 |
) |
Net loss per share
attributable to common stockholders—basic and diluted |
|
$ |
(0.37 |
) |
|
$ |
(0.96 |
) |
|
$ |
(0.59 |
) |
|
$ |
(1.84 |
) |
Weighted-average common stock
outstanding—basic and diluted |
|
|
79,041,695 |
|
|
|
26,901,069 |
|
|
|
67,762,501 |
|
|
|
26,560,185 |
|
Applied Therapeutics (NASDAQ:APLT)
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