Applied Therapeutics Reports First Quarter 2024 Financial Results
May 09 2024 - 7:00AM
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 first quarter ended March 31, 2024.
“Preparations are underway for the potential approval and
commercial launch of govorestat for the treatment of Classic
Galactosemia in the US and EU, following the significant regulatory
progress we have already made in 2024,” said Shoshana Shendelman,
PhD, Founder and CEO of Applied Therapeutics. “We are also
discussing a potential NDA submission for SORD Deficiency with the
Neurology Division at FDA, further advancing our objective of
bringing first ever treatment to patients with rare diseases.”
Recent Highlights
- Govorestat NDA Under Priority Review by US FDA for
Treatment of Classic Galactosemia, PDUFA Target Action Date of
November 28, 2024; MAA under CHMP Review by EMA. In March
2024, the Company announced that the U.S. Food and Drug
Administration (FDA) has extended the review period for the New
Drug Application (NDA) for govorestat (AT-007) for the treatment of
Classic Galactosemia to allow more time to review supplemental
analyses of previously submitted data that had been provided by
Applied in response to the FDA’s routine information requests. No
additional data or studies have been requested by the FDA at this
time. The new PDUFA action date is November 28, 2024. The NDA was
granted Priority Review Status, and the FDA also noted that it
plans 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). In April
2024, the EMA granted a 3-month extension to the Day 120 clock stop
period to allow sufficient time for responses to the CHMP’s Day 120
list of questions. As a result, the Company now expects a decision
by the EMA in early first quarter of 2025. 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.
- Appointed Dale Hooks as Chief Commercial
Officer. In April 2024, the Company appointed Dale Hooks
as Chief Commercial Officer to lead Applied’s commercial
preparations for the potential launch of govorestat for the
treatment of Classic Galactosemia and SORD. Mr. Hooks brings over
30 years of biopharmaceutical experience in leadership, rare
disease marketing and sales roles to Applied Therapeutics.
- Presented Full Results from Phase 3 ARISE-HF Study of
AT-001 (caficrestat) in Diabetic Cardiomyopathy at the 2024
American College of Cardiology Annual Scientific Session.
In April 2024, the Company presented full results from the Phase 3
ARISE-HF study of AT-001 in patients with diabetic cardiomyopathy
(DbCM) in an oral presentation at the 2024 American College of
Cardiology (ACC) Annual Scientific Session. In the ARISE-HF study,
treatment with AT-001 demonstrated a strong trend in stabilizing
cardiac functional capacity, and a statistically significant
difference in cardiac functional capacity in a prespecified
subgroup of patients not receiving concomitant treatment with an
SGLT2 or GLP-1 while preventing clinically significant worsening.
Furthermore, AT-001 treatment prevented progression to overt heart
failure in patients with DbCM as compared to placebo (p=0.0285).
Data also demonstrated that AT-001 was safe and well tolerated in a
large cohort of patients, providing proof of concept that the
technology has overcome the selectivity and safety issues of “old”
aldose reductase inhibitors. Following the presentation, the full
study results were published in the Journal of the American College
of Cardiology (JACC).
Financial Results
- Cash and
cash equivalents and short-term investments totaled $146.5
million as of March 31, 2024, compared with $49.9 million at
December 31, 2023.
- Research
and development expenses for the three months ended March
31, 2024, were $12.2 million, compared to $15.9 million for the
three months ended March 31, 2023. The decrease of approximately
$3.7 million was primarily attributed to decreased expenses
related to contract research organizations (CROs) and drug
manufacturing and formulation costs, partially offset by an
increase in regulatory and personnel expenses.
- General
and administrative expenses were $9.1 million for the
three months ended March 31, 2024, compared to $5.6 million for the
three months ended March 31, 2023. The increase of approximately
$3.5 million was primarily related to an increase in legal and
professional fees of $1.2 million and $1.4 million in commercial
expenses to support planned commercialization of govorestat, and an
increase in other expenses of $0.9 million due to an overall
increase in data storage costs to support planned
commercialization.
- Net
loss for the first quarter of 2024 was $83.9 million, or
$0.67 per basic and diluted common share, compared to a net loss of
$10.1 million, or $0.18 per basic and diluted common share, for the
first quarter 2023.
- Cash
runway: The Company expects that its cash and cash
equivalents will fund the business into 2026. Additionally, the
sale of the priority review voucher (PRV), which would be granted
upon a potential NDA approval of govorestat for the treatment of
Galactosemia 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 fund the business into 2026; (ii) the
likelihood that the Company’s ongoing NDA and MMA submissions will
be approved and the timing of any approval decision and (iii)
statements related to the potential commercial launch of
govorestat. 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.Condensed
Balance Sheets(in thousands, except share and per
share data)(Unaudited) |
|
|
|
|
As ofMarch 31,2024 |
|
As ofDecember 31,2023 |
|
ASSETS |
|
|
|
|
|
|
|
|
CURRENT ASSETS: |
|
|
|
|
|
|
|
|
Cash and cash equivalents |
$ |
146,484 |
|
|
$ |
49,898 |
|
|
Security deposits and leasehold improvements |
|
253 |
|
|
|
254 |
|
|
Prepaid expenses and other current assets |
|
4,169 |
|
|
|
4,234 |
|
|
Total current assets |
|
150,906 |
|
|
|
54,386 |
|
|
Operating lease right-of-use asset |
|
328 |
|
|
|
447 |
|
|
TOTAL ASSETS |
$ |
151,234 |
|
|
$ |
54,833 |
|
|
LIABILITIES AND STOCKHOLDERS’
EQUITY/(DEFICIT) |
|
|
|
|
|
|
|
|
CURRENT LIABILITIES: |
|
|
|
|
|
|
|
|
Current portion of operating lease liabilities |
$ |
308 |
|
|
$ |
429 |
|
|
Accounts payable |
|
4,019 |
|
|
|
1,742 |
|
|
Accrued expenses and other current liabilities |
|
14,641 |
|
|
|
15,286 |
|
|
Warrant liabilities |
|
64,937 |
|
|
|
53,725 |
|
|
Total current liabilities |
|
83,905 |
|
|
|
71,182 |
|
|
NONCURRENT LIABILITIES: |
|
|
|
|
|
|
|
|
Noncurrent portion of operating lease liabilities |
|
35 |
|
|
|
38 |
|
|
Clinical holdback - long-term portion |
|
— |
|
|
|
759 |
|
|
Total noncurrent liabilities |
|
35 |
|
|
|
797 |
|
|
Total liabilities |
|
83,940 |
|
|
|
71,979 |
|
|
STOCKHOLDERS’ EQUITY/(DEFICIT): |
|
|
|
|
|
|
|
|
Common stock, $0.0001 par value; 200,000,000 shares authorized as
ofMarch 31, 2024 and December 31, 2023;
114,241,803 shares issued andoutstanding as of
March 31, 2024 and 84,869,832 shares issuedand
outstanding as of December 31, 2023 |
|
11 |
|
|
|
8 |
|
|
Preferred stock, par value $0.0001; 10,000,000 shares authorized as
ofMarch 31, 2024 and December 31, 2023; 0 shares issued
and outstandingas of March 31, 2024 and
December 31, 2023 |
|
— |
|
|
|
— |
|
|
Additional paid-in capital |
|
619,807 |
|
|
|
451,432 |
|
|
Accumulated deficit |
|
(552,524 |
) |
|
|
(468,586 |
) |
|
Total stockholders' equity/(deficit) |
|
67,294 |
|
|
|
(17,146 |
) |
|
TOTAL LIABILITIES AND STOCKHOLDERS’ EQUITY/(DEFICIT) |
$ |
151,234 |
|
|
$ |
54,833 |
|
|
|
|
Applied Therapeutics, Inc.Condensed
Statements of Operations(in thousands, except
share and per share data)(Unaudited) |
|
|
|
|
Three Months EndedMarch 31, |
|
|
2024 |
|
2023 |
|
REVENUE: |
|
|
|
|
|
|
|
|
License revenue |
$ |
— |
|
|
$ |
10,660 |
|
|
Research and development services revenue |
|
190 |
|
|
|
— |
|
|
Total revenue |
|
190 |
|
|
|
10,660 |
|
|
COSTS AND EXPENSES: |
|
|
|
|
|
|
|
|
Research and development |
|
12,217 |
|
|
|
15,935 |
|
|
General and administrative |
|
9,066 |
|
|
|
5,583 |
|
|
Total costs and expenses |
|
21,283 |
|
|
|
21,518 |
|
|
LOSS FROM OPERATIONS |
|
(21,093 |
) |
|
|
(10,858 |
) |
|
OTHER (EXPENSE) INCOME, NET: |
|
|
|
|
|
|
|
|
Interest income |
|
586 |
|
|
|
221 |
|
|
Change in fair value of warrant liabilities |
|
(63,405 |
) |
|
|
469 |
|
|
Other expense |
|
(26 |
) |
|
|
31 |
|
|
Total other (expense) income, net |
|
(62,845 |
) |
|
|
721 |
|
|
Net loss |
$ |
(83,938 |
) |
|
$ |
(10,137 |
) |
|
Net loss per share attributable to common stockholders—basicand
diluted |
$ |
(0.67 |
) |
|
$ |
(0.18 |
) |
|
Weighted-average common stock outstanding—basic anddiluted |
|
125,318,993 |
|
|
|
56,357,983 |
|
|
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