Applied Therapeutics Reports Second Quarter 2024 Financial Results
07 Agosto 2024 - 1:00PM
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 second quarter ended June 30, 2024.
“Momentum continues with our steady regulatory progress in
Classic Galactosemia and SORD Deficiency,” said Shoshana
Shendelman, PhD, Founder and CEO of Applied Therapeutics. “We are
incredibly pleased to share our alignment with the Neurology
Division of the FDA regarding a potential second NDA submission for
govorestat for the treatment of SORD Deficiency. Both Galactosemia
and SORD Deficiency are rare neurological diseases with no
currently approved treatment options. At Applied, we are dedicated
to creating transformative treatments for rare diseases, and we
continue to work closely with regulatory agencies and patient
advocacy groups to ensure that treatments become available for
patients with these debilitating diseases.”
Recent Highlights
- Govorestat PDUFA Target Action Date of November 28,
2024; MAA under CHMP Review by EMA; Updated Cognition Data Included
in Review. In the process of preparing for the United
States Food and Drug Administration (FDA) inspection, it was
discovered that the vendor hired to compile NIH Toolbox data for
the Company used an adult formula for calculation of about one
third of composite cognition and motor skills scores. Adjusting the
formula to the pediatric formula resulted in significantly improved
data for cognition as compared to the prior data, demonstrating
improvement in the govorestat (AT-007) treated group of
approximately 8 points on a standard scale, which was statistically
significant compared to placebo (p=0.032). This also resulted
in a statistically significant effect on the primary endpoint
sensitivity analysis which included cognition (p=0.034). The motor
skills data did not change
substantially. These updates were disclosed
and discussed with the FDA and European Medicines Agency (EMA) and
will be used in the ongoing evaluation of the New Drug Application
(NDA) and Marketing Authorization Application (MAA). As previously
announced, the FDA Prescription Drug User Fee Act (PDUFA) target
action date is November 28, 2024. 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 MAA for govorestat for the treatment of Classic Galactosemia to
the EMA, which was validated in December 2023 and is under review
by the EMA’s Committee for Medicinal Products for Human Use (CHMP).
As previously announced, 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. The Company
expects a decision by the EMA early in the first quarter of 2025.
The NDA and MAA submission packages are supported by rapid and
sustained reduction in galactitol, which resulted in a meaningful
benefit on clinical outcomes across pediatric patients, alongside a
favorable safety profile. The 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. If approved, govorestat would be the first
medication indicated for the treatment of Galactosemia and would be
Applied Therapeutics’ first commercial product.
- FDA Advisory Committee Meeting to Review Govorestat NDA
for the Treatment of Classic Galactosemia Tentatively Scheduled for
October 9, 2024. The FDA notified the Company of their
tentative plans to convene the Genetic Metabolic Diseases Advisory
Committee (GeMDAC) on October 9, 2024, to discuss the Company’s NDA
for govorestat for the treatment of Classic Galactosemia. The date
is tentative and has not yet been confirmed in the federal
register. The newly formed GeMDAC will consist of experts in the
fields of medical genetics, inborn errors of metabolism,
epidemiology, and other related specialties.
- Company Aligned with the Neurology I Division of the
FDA on Potential Submission of an NDA for Govorestat for the
Treatment of SORD Deficiency Under Accelerated
Approval. In July 2024, the Company held a Type C meeting
with the FDA to align on the regulatory path forward for govorestat
for the treatment of SORD Deficiency. The Neurology I Division
confirmed that the data generated to-date was appropriate for a
potential NDA submission under the FDA’s Accelerated Approval
Program, and discussed the design of a new confirmatory study to be
completed as a post-marketing requirement. The Company plans to
hold a pre-NDA meeting to discuss administrative aspects of the
submission in the second half of this year, and expects to submit
an NDA early in the first quarter of 2025. If govorestat is
approved for the treatment of Classic Galactosemia, the regulatory
submission for the treatment of SORD will be submitted as a
supplementary New Drug Application (sNDA). Patients in the INSPIRE
study will be offered open-label govorestat treatment and will be
followed for additional safety data generation. The review and
potential approval of govorestat for SORD is independent of the
ongoing review of govorestat for Classic Galactosemia.
- APLT Added to Russell 3000® Index. In June
2024, as part of the Russell indexes annual reconstitution, the
Company was added to the Russell 3000® Index, a market
capitalization-weighted equity index that tracks the performance of
the largest 3,000 U.S. stocks. Membership of the Russell indexes is
primarily determined by objective, market-capitalization rankings
and style attributes. Russell indexes are widely used by investment
managers and institutional investors for index funds and as
benchmarks for active investment strategies.
- Participated in Multiple Medical and Patient Advocacy
Group Focused Conferences. In the second and third
quarters of 2024, the Company deepened its relationships and
partnership with the patient community, presenting data and giving
keynote addresses at the following medical meetings and patient
advocacy group conferences:
- Hereditary Neuropathy Foundation (HNF) Charcot-Marie Tooth
Syndrome (CMT) Summit, June 7-8 in San Diego, California;
- Charcot-Marie-Tooth Associate (CMTA) Strategy to Accelerate
Research (STAR) Advisory Board meeting held June 21 in Montreal,
Canada;
- Peripheral Nerve Society (PNS) 2024 Annual Meeting, held June
22-25 in Montreal, Canada;
- Sponsored and presented at the 2024 Galactosemia Foundation
Conference, held July 18-20 in Concord, North Carolina.
Financial Results
- Cash and
cash equivalents and short-term investments totaled $122.2
million as of June 30, 2024, compared with $49.9 million at
December 31, 2023.
- Research
and development expenses for the three months ended June
30, 2024, were $10.0 million, compared to $11.9 million for the
three months ended June 30, 2023. The decrease of approximately
$1.9 million was primarily related to decreased expenses
associated with clinical and pre-clinical expenses for the near
completion of AT-001 and AT-007 and drug manufacturing and
formulation costs, partially offset by an increase in regulatory
and personnel expenses.
- General
and administrative expenses were $10.6 million for the
three months ended June 30, 2024, compared to $5.3 million for the
three months ended June 30, 2023. The increase of approximately
$5.3 million was primarily related to an increase in legal and
professional fees of $1.3 million, an increase in commercial
expenses to support planned commercialization of govorestat of $3.5
million, and an increase in personnel expenses of $1.1 million due
to increased headcount and salary increases, offset by a decrease
in stock-based compensation, insurance expenses and other
miscellaneous expense.
- Net
income for the second quarter of 2024 was $2.9 million, or
$0.02 per basic common share and a net loss of $0.13 per diluted
common share, compared to a net loss of $29.6 million, or $0.37 per
basic and diluted common share, for the second quarter of
2023.
- Cash
runway: The Company expects that its cash and cash
equivalents will fund the business into 2026. Additionally, the
Company expects that 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 decision and (iii) statements
related to the scheduling or timing of any potential FDA or EMA
meetings, interactions or submissions. 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:Julie
Seidel/Andrew
Vulis646-970-0543appliedtherapeutics@argotpartners.com
Media:media@appliedtherapeutics.com
|
Applied Therapeutics, Inc.Condensed
Balance Sheets(in thousands, except share and per
share data)(Unaudited) |
|
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|
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As of |
|
As of |
|
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June 30, |
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December 31, |
|
|
2024 |
|
2023 |
ASSETS |
|
|
|
|
|
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CURRENT ASSETS: |
|
|
|
|
|
|
Cash and cash equivalents |
|
$ |
122,197 |
|
|
$ |
49,898 |
|
Security deposits and leasehold improvements |
|
|
253 |
|
|
|
254 |
|
Prepaid expenses and other current assets |
|
|
5,122 |
|
|
|
4,234 |
|
Total current assets |
|
|
127,572 |
|
|
|
54,386 |
|
Operating lease right-of-use asset |
|
|
206 |
|
|
|
447 |
|
TOTAL ASSETS |
|
$ |
127,778 |
|
|
$ |
54,833 |
|
LIABILITIES AND
STOCKHOLDERS’ EQUITY/(DEFICIT) |
|
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|
|
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CURRENT LIABILITIES: |
|
|
|
|
|
|
Current portion of operating lease liabilities |
|
$ |
185 |
|
|
$ |
429 |
|
Accounts payable |
|
|
2,683 |
|
|
|
1,742 |
|
Accrued expenses and other current liabilities |
|
|
10,296 |
|
|
|
15,286 |
|
Warrant liabilities |
|
|
42,192 |
|
|
|
53,725 |
|
Total current liabilities |
|
|
55,356 |
|
|
|
71,182 |
|
NONCURRENT LIABILITIES: |
|
|
|
|
|
|
Noncurrent portion of operating lease liabilities |
|
|
30 |
|
|
|
38 |
|
Clinical holdback - long-term portion |
|
|
— |
|
|
|
759 |
|
Total noncurrent liabilities |
|
|
30 |
|
|
|
797 |
|
Total liabilities |
|
|
55,386 |
|
|
|
71,979 |
|
STOCKHOLDERS’
EQUITY/(DEFICIT): |
|
|
|
|
|
|
Common stock, $0.0001 par value; 250,000,000 shares authorized as
of June 30, 2024 and 200,000,000 shares authorized as of
December 31, 2023; 114,846,271 shares issued and
outstanding as of June 30, 2024 and 84,869,832 shares
issued and outstanding as of December 31, 2023 |
|
|
11 |
|
|
|
8 |
|
Preferred stock, par value $0.0001; 10,000,000 shares authorized as
of June 30, 2024 and December 31, 2023; 0
shares issued and outstanding as of June 30, 2024 and
December 31, 2023 |
|
|
— |
|
|
|
— |
|
Additional paid-in capital |
|
|
622,007 |
|
|
|
451,432 |
|
Accumulated deficit |
|
|
(549,626 |
) |
|
|
(468,586 |
) |
Total stockholders' equity/(deficit) |
|
|
72,392 |
|
|
|
(17,146 |
) |
TOTAL LIABILITIES AND STOCKHOLDERS’ EQUITY/(DEFICIT) |
|
$ |
127,778 |
|
|
$ |
54,833 |
|
|
|
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 |
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|
June 30, |
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June 30, |
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|
2024 |
|
2023 |
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2024 |
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2023 |
REVENUE: |
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License revenue |
|
$ |
— |
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$ |
— |
|
|
$ |
— |
|
|
$ |
10,660 |
|
Research and development services revenue |
|
|
144 |
|
|
|
— |
|
|
|
334 |
|
|
|
— |
|
Total revenue |
|
|
144 |
|
|
|
— |
|
|
|
334 |
|
|
|
10,660 |
|
COSTS AND EXPENSES: |
|
|
|
|
|
|
|
|
|
|
|
|
Research and development |
|
|
10,004 |
|
|
|
11,883 |
|
|
|
22,221 |
|
|
|
27,818 |
|
General and administrative |
|
|
10,580 |
|
|
|
5,293 |
|
|
|
19,646 |
|
|
|
10,876 |
|
Total costs and expenses |
|
|
20,584 |
|
|
|
17,176 |
|
|
|
41,867 |
|
|
|
38,694 |
|
LOSS FROM OPERATIONS |
|
|
(20,440 |
) |
|
|
(17,176 |
) |
|
|
(41,533 |
) |
|
|
(28,034 |
) |
OTHER (EXPENSE) INCOME,
NET: |
|
|
|
|
|
|
|
|
|
|
|
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Interest income |
|
|
628 |
|
|
|
408 |
|
|
|
1,215 |
|
|
|
628 |
|
Change in fair value of warrant liabilities |
|
|
22,744 |
|
|
|
(12,804 |
) |
|
|
(40,660 |
) |
|
|
(12,335 |
) |
Other (expense) income, net |
|
|
(34 |
) |
|
|
(5 |
) |
|
|
(62 |
) |
|
|
27 |
|
Total other income (expense), net |
|
|
23,338 |
|
|
|
(12,401 |
) |
|
|
(39,507 |
) |
|
|
(11,680 |
) |
Net income (loss) |
|
$ |
2,898 |
|
|
$ |
(29,577 |
) |
|
$ |
(81,040 |
) |
|
$ |
(39,714 |
) |
Net income (loss) per share
attributable to common stockholders—basic |
|
$ |
0.02 |
|
|
$ |
(0.37 |
) |
|
$ |
(0.60 |
) |
|
$ |
(0.59 |
) |
Net income (loss) per share
attributable to common stockholders—diluted |
|
$ |
(0.13 |
) |
|
$ |
(0.37 |
) |
|
$ |
(0.60 |
) |
|
$ |
(0.59 |
) |
Weighted-average common stock
outstanding—basic |
|
|
143,934,239 |
|
|
|
79,041,695 |
|
|
|
134,627,942 |
|
|
|
67,762,501 |
|
Weighted-average common stock
outstanding—diluted |
|
|
152,392,748 |
|
|
|
79,041,695 |
|
|
|
134,627,942 |
|
|
|
67,762,501 |
|
|
Grafico Azioni Applied Therapeutics (NASDAQ:APLT)
Storico
Da Lug 2024 a Ago 2024
Grafico Azioni Applied Therapeutics (NASDAQ:APLT)
Storico
Da Ago 2023 a Ago 2024