Applied Therapeutics Reports Fourth Quarter and Year-end 2023 Financial Results
06 March 2024 - 11:00PM
Applied Therapeutics, Inc. (Nasdaq: APLT) (the “Company”), 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 fourth quarter and full year ended December 31,
2023.
“We’ve made significant clinical and regulatory progress,
particularly with the NDA acceptance and MAA validation for
govorestat for the treatment of Galactosemia, achieving key
milestones for our rare disease pipeline. Additionally, we believe
that the recent positive data from the interim analysis of the
INSPIRE study in SORD Deficiency confirms the role of sorbitol as a
key driver of disease progression, and we plan to request a pre-NDA
meeting with the FDA,” said Shoshana Shendelman, PhD, Founder,
Chief Executive Officer, and Chair of the Board. “As Applied enters
into this next stage of growth, we are poised for continued value
generation across our rare disease pipeline, supported by our
recent financing and bolstered cash position.”
Recent Highlights
- Govorestat NDA Accepted and Granted Priority Review by
US FDA for Treatment of Classic Galactosemia, PDUFA Target Action
Date of August 28, 2024; MAA under CHMP Review by EMA. In
February 2024, the Company announced that the U.S. Food and Drug
Administration (FDA) has accepted the filing of the New Drug
Application (NDA) for govorestat (AT-007) for the treatment of
Classic Galactosemia. The NDA was granted Priority Review status
and the FDA assigned a Prescription Drug User Free Act (PDUFA)
target action date of August 28, 2024. The FDA also noted that it
is planning to hold an advisory committee meeting to discuss the
application. Govorestat was previously granted Pediatric Rare
Disease designation and will qualify for a Priority Review Voucher
(PRV) upon approval. The Company has also submitted a Marketing
Authorization Application (MAA) for govorestat for the treatment of
Classic Galactosemia to the European Medicines Agency (EMA), which
was validated in December 2023 and is under review by the EMA’s
Committee for Medicinal Products for Human Use (CHMP). The Company
expects a decision by the EMA in the fourth quarter of 2024. The
NDA and MAA submission packages include clinical outcomes data from
the Phase 3 registrational ACTION-Galactosemia Kids study in
children aged 2-17 with Galactosemia, the Phase 1/2
ACTION-Galactosemia study in adult patients with Galactosemia, and
preclinical data.
- Entered into $100 Million Private Placement, Expected
to Extend Cash Runway to 2026. In February 2024, the
Company entered into a securities purchase agreement for a private
placement of $100 million of equity with participation from new and
existing large, healthcare dedicated institutional and mutual fund
investors including Perceptive Advisors, Janus Henderson Investors,
Venrock Healthcare Capital Partners, Adage Capital Partners,
Frazier Life Sciences, Logos Capital, Vestal Point Capital, and
Rock Springs Capital. The Company intends to use the net proceeds
to fund commercial activities for govorestat and to further develop
other pipeline candidates, and for working capital and general
corporate purposes. With cash of approximately $153.5 million as of
March 1, 2024, the Company is well-capitalized with an expected
runway into the first half of 2026.
- Announced Positive Data from 12-Month Interim Analysis
of Govorestat (AT-007) in Ongoing INSPIRE Phase 3 Trial in Sorbitol
Dehydrogenase Deficiency. In February 2024, the Company
announced that govorestat met primary and several key secondary
endpoints in a pre-specified 12-month interim analysis of the
ongoing INSPIRE Phase 3 Trial in Sorbitol Dehydrogenase (SORD)
Deficiency. The objective of this pre-specified, 12-month interim
analysis was to evaluate trends in clinical outcome measures and
biomarker correlations in order to inform future regulatory
discussions and support a potential NDA submission, due to the
urgent need for treatment and absence of any other options for
patients with SORD Deficiency. At the time of the interim analysis,
a statistically significant correlation between sorbitol levels and
the prespecified CMT-FOM composite clinical endpoint (10-meter
walk-run test, 4 stair climb, sit to stand test, 6-minute walk test
and dorsiflexion) was observed at 12 months of treatment with
govorestat (p=0.05). Govorestat treatment provided sustained
reduction in sorbitol level in patients with SORD Deficiency over
12 months of treatment, which was statistically significant
compared to placebo (p<0.001). In addition, treatment with
govorestat also resulted in a highly statistically significant
effect (p=0.01) on the CMT Health Index (CMT-HI), an important
patient-reported outcome measure of disease severity and well-being
and secondary endpoint in the study. Aspects of the CMT-HI that
demonstrated a treatment effect included lower limb function,
mobility, fatigue, pain, sensory function, and upper limb function.
Govorestat continues to be safe and well tolerated to-date. The
Company believes the results from the 12-month interim analysis of
the ongoing INSPIRE Phase 3 trial confirm the role of sorbitol as a
key driver of disease severity and progression over time. Clinical
outcomes of the ongoing INSPIRE trial are expected to be assessed
again at 24 months, where the 10-meter walk run test serves as the
primary clinical efficacy endpoint. The Company plans to discuss a
potential NDA submission with the FDA based on the clinical data to
date. The ongoing INSPIRE trial is a Phase 3 double-blind
placebo-controlled registrational study evaluating the effect of
once-daily oral govorestat in approximately 50 patients aged 16-55
with SORD Deficiency in the US and Europe.
- Announced Topline Results from ARISE-HF Phase 3 Study
of AT-001 (caficrestat) in Diabetic Cardiomyopathy. In
January 2024, the Company announced topline data from its Phase 3
Study of AT-001 in patients with diabetic cardiomyopathy (DbCM).
AT-001 1500mg twice daily (BID) was generally safe and well
tolerated, and demonstrated a strong trend in stabilizing cardiac
functional capacity. In a pre-specified subgroup analysis of
patients not concomitantly treated with SGLT2 or GLP-1 therapies,
active treatment with AT-001 resulted in a statistically
significant difference in the primary endpoint, and also resulted
in a substantially lower rate of clinically significant worsening
in cardiac functional capacity of 6% or more as compared to
placebo. Full study results will be presented at an upcoming
medical conference, along with results of the Diabetic Peripheral
Neuropathy sub-study, which are still being analyzed. As a result
of the encouraging ARISE-HF Phase 3 data, the Company plans to
focus on identifying an appropriate path forward in order to bring
AT-001 to DbCM patients.
Financial Results
- Cash and
cash equivalents and short-term investments totaled $49.9
million as of December 31, 2023, compared to $30.6 million as of
December 31, 2022. On March 1, 2024, the Company completed the
private placement of shares and pre-funded warrants to purchase
common stock for gross proceeds of approximately $100 million
(before deducing placement agent commissions and other offering
expenses).
- Research
and development expenses for the year ended December 31,
2023 were $53.9 million, compared to $55.6 million for the year
ended December 31, 2022. The decrease of approximately
$1.7 million was primarily related to decreased expenses
related to contract research organization (CRO) costs, partially
offset by an increase in drug manufacturing and formulation
costs.
- General
and administrative expenses were $20.6 million for the
year ended December 31, 2023, compared to $27.3 million for the
year ended December 31, 2022. The decrease of approximately
$6.7 million was primarily related to a decrease in commercial
expense of $2.1 million, an overall decrease in headcount resulting
in a $3.0 million decrease in expense and reduced insurance expense
of $1.4 million.
- Net
loss for the year ended December 31, 2023 was $119.8
million, or $1.42 per basic and diluted common share, compared to a
net loss of $82.5 million, or $2.18 per basic and diluted common
share, for the year ended December 31, 2022.
- Cash
runway: The Company expects that its cash and cash
equivalents, combined with the proceeds from the February 2024
Private Placement and potential milestones expected from its Advanz
European licensing partnership, will fund the business into 2026.
Additionally, the sale of the PRV, which would be granted upon a
potential Galactosemia NDA approval could substantially extend the
Company’s cash runway.
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,” “predicts” 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 the (i) Company’s expectation that its cash
and cash equivalents will extend into 2026; (ii) Company’s plans to
request a pre-NDA meeting with the FDA; and (iii) the likelihood
that the Company’s ongoing NDA and MMA submissions will be approved
and the timing of any approval decision. 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, (xviii) 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.Balance Sheets(in thousands
except share and per share data) |
|
|
|
|
|
|
|
|
|
As of |
|
As of |
|
|
December 31, |
|
December 31, |
|
|
2023 |
|
2022 |
|
|
(Unaudited) |
|
|
ASSETS |
|
|
|
|
|
|
CURRENT ASSETS: |
|
|
|
|
|
|
Cash and cash equivalents |
|
$ |
49,898 |
|
|
$ |
16,657 |
|
Investments |
|
|
— |
|
|
|
13,923 |
|
Current portion of security deposits and leasehold
improvements |
|
|
254 |
|
|
|
57 |
|
Prepaid expenses and other current assets |
|
|
4,234 |
|
|
|
6,671 |
|
Total current assets |
|
|
54,386 |
|
|
|
37,308 |
|
Noncurrent portion of security deposits and leasehold
improvements |
|
|
— |
|
|
|
198 |
|
Operating lease right-of-use asset |
|
|
447 |
|
|
|
857 |
|
TOTAL ASSETS |
|
$ |
54,833 |
|
|
$ |
38,363 |
|
LIABILITIES AND STOCKHOLDERS’
(DEFICIT)/EQUITY |
|
|
|
|
|
|
CURRENT LIABILITIES: |
|
|
|
|
|
|
Current portion of operating lease liabilities |
|
$ |
429 |
|
|
$ |
477 |
|
Accounts payable |
|
|
1,742 |
|
|
|
4,534 |
|
Accrued expenses and other current liabilities |
|
|
15,286 |
|
|
|
14,756 |
|
Warrant liabilities |
|
|
53,725 |
|
|
|
13,657 |
|
Total current liabilities |
|
|
71,182 |
|
|
|
33,424 |
|
NONCURRENT LIABILITIES: |
|
|
|
|
|
|
Noncurrent portion of operating lease liabilities |
|
|
38 |
|
|
|
414 |
|
Clinical holdback - long-term portion |
|
|
759 |
|
|
|
464 |
|
Total noncurrent liabilities |
|
|
797 |
|
|
|
878 |
|
Total liabilities |
|
|
71,979 |
|
|
|
34,302 |
|
STOCKHOLDERS’ (DEFICIT)/EQUITY: |
|
|
|
|
|
|
Common stock, $0.0001 par value; 200,000,000 shares authorized as
of December 31, 2023 and December 31, 2022;
84,869,832 shares issued and outstanding as of
December 31, 2023 and 48,063,358 shares issued and
outstanding as of December 31, 2022 |
|
|
8 |
|
|
|
5 |
|
Preferred stock, par value $0.0001; 10,000,000 shares authorized as
of December 31, 2023 and December 31, 2022; 0
shares issued and outstanding as of December 31, 2023 and
December 31, 2022 |
|
|
— |
|
|
|
— |
|
Additional paid-in capital |
|
|
451,432 |
|
|
|
352,828 |
|
Accumulated other comprehensive income |
|
|
— |
|
|
|
51 |
|
Accumulated deficit |
|
|
(468,586 |
) |
|
|
(348,823 |
) |
Total stockholders' (deficit)/equity |
|
|
(17,146 |
) |
|
|
4,061 |
|
TOTAL LIABILITIES AND STOCKHOLDERS’ (DEFICIT)/EQUITY |
|
$ |
54,833 |
|
|
$ |
38,363 |
|
Applied Therapeutics, Inc.Statements
of Operations(in thousands except share and per
share data)(Unaudited) |
|
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Year Ended |
|
|
|
December 31, |
|
|
|
2023 |
|
2022 |
REVENUE: |
|
|
|
|
|
|
|
License revenue |
|
|
$ |
9,219 |
|
|
$ |
— |
|
Research and development services revenue |
|
|
|
774 |
|
|
|
— |
|
Total revenue |
|
|
|
9,993 |
|
|
|
— |
|
COSTS AND EXPENSES: |
|
|
|
|
|
|
|
Research and development |
|
|
|
53,905 |
|
|
|
55,634 |
|
General and administrative |
|
|
|
20,623 |
|
|
|
27,316 |
|
Total operating expenses |
|
|
|
74,528 |
|
|
|
82,950 |
|
LOSS FROM OPERATIONS |
|
|
|
(64,535 |
) |
|
|
(82,950 |
) |
OTHER INCOME (EXPENSE), NET: |
|
|
|
|
|
|
|
Interest income |
|
|
|
1,372 |
|
|
|
685 |
|
Change in fair value of warrant liabilities |
|
|
|
(56,573 |
) |
|
|
(66 |
) |
Other expense |
|
|
|
(27 |
) |
|
|
(177 |
) |
Total other income (expense), net |
|
|
|
(55,228 |
) |
|
|
442 |
|
Net
loss |
|
|
$ |
(119,763 |
) |
|
$ |
(82,508 |
) |
Net
loss per share attributable to common stockholders—basic and
diluted |
|
|
$ |
(1.42 |
) |
|
$ |
(2.18 |
) |
Weighted-average common stock outstanding—basic and diluted |
|
|
|
84,244,494 |
|
|
|
37,825,431 |
|
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