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Applied Therapeutics Reports Fourth Quarter and Year-end 2020 Financial Results AT-007 ACTION-Galactosemia Kids pediatric clinical study recently resumed following discussions with FDA On track to submit NDA for AT-007 i

Key Takeaway: Applied Therapeutics Reports Fourth Quarter and Year-end 2020 Financial Results AT-007 ACTION-Galactosemia Kids pediatric clinical study recently resumed following discussions with FDA On track to submit NDA for AT-007 in Galactosemia no later than Q3 2021; Commercial prepar

Full Press Release Details

Applied Therapeutics Reports Fourth Quarter
and Year-end 2020 Financial Results
AT-007 ACTION-Galactosemia Kids pediatric
clinical study recently resumed following discussions with FDA
On track to submit NDA for AT-007 in
Galactosemia no later than Q3 2021; Commercial preparations underway
ARISE-HF Phase 3 global registrational
study of AT-001 in Diabetic Cardiomyopathy on track to complete enrollment in mid-2021
On track to initiate rare disease franchise
expansion programs in SORD Deficiency and PMM2-CDG studies in the first half of 2021; Company to host Rare Disease Forum on Tuesday,
NEW YORK, March 18, 2021 - 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 fourth quarter
and full year ended December 31, 2020.
"The fourth quarter was a productive
period of internal planning and execution," said Shoshana Shendelman, PhD, Founder, CEO and Chair of the Board of Applied
Therapeutics. "We are excited for what lies ahead in 2021, where our focus will be on advancing our late stage assets toward
commercialization while initiating clinical development of our two new rare disease programs."
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, AT-007, is a novel central nervous system penetrant aldose reductase
inhibitor (ARI) for the treatment of Galactosemia, a rare pediatric metabolic disease. The Company initiated a pivotal Phase 1/2
clinical trial in June 2019, read out positive top-line biomarker data in adult Galactosemia patients in January 2020 and announced
full data from the trial in April 2020. A pediatric Galactosemia study commenced in June 2020. The Company is also developing AT-001,
a novel potent ARI that is being developed for the treatment of Diabetic Cardiomyopathy, or DbCM, a fatal fibrosis of the heart.
The Company initiated a Phase 3 registrational study in DbCM in September 2019. 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, as well as novel
dual PI3k inhibitors in preclinical development for orphan oncology indications.
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
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) the timing of our NDA submission for potential approval of AT-007, which will include data
from the ACTION-Galactosemia Kids trial and the 90-day safety data in adults with Galactosemia, (ii) the timing of our rare disease
franchise expansion programs in SORD Deficiency and PMM2-CDG, (iii) the timing and effectiveness of our Galactosemia awareness
and education campaign, (iv) the timing of the initiation and completion of our clinical trials, (v) the likelihood that data from
our clinical trials will support future development of our product candidates and (vi) the likelihood of obtaining regulatory approval
of our product candidates. 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 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, (vi) our ability to maintain and establish
collaborations or obtain additional funding, (vii) our ability to obtain regulatory approval of our current and future
product candidates, (viii) our expectations regarding the potential market size and the rate and degree of market acceptance
of such product candidates, (ix) our ability to fund our working capital requirements and expectations regarding the
sufficiency of our capital resources, (x) the implementation of our business model and strategic plans for our business and
product candidates, (xi) our intellectual property position and the duration of our patent rights, (xii) developments or
disputes concerning our intellectual property or other proprietary rights, (xiii) our expectations regarding government and
third-party payor coverage and reimbursement, (xiv) our ability to compete in the markets we serve, (xv) the impact of
government laws and regulations and liabilities thereunder, (xvi) developments relating to our competitors and our industry,
(xvii) the impact of the COVID-19 pandemic on the timing and progress of our ongoing clinical trials and our business in
general and (xviii) 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.
Applied Therapeutics, Inc.
Statements of Operations
(in thousands, except share and per share
Year Ended
December 31,
2020 2019
OPERATING EXPENSES:
Research and development $ 61,788 $ 32,350
General and administrative 32,678 13,232
Total operating expenses 94,466 45,582
LOSS FROM OPERATIONS (94,466 ) (45,582 )
OTHER INCOME (EXPENSE), NET:
Interest income (expense), net 559 93
Other income (expense) (54 ) (24 )
Total other income (expense), net 505 69
Net loss $ (93,961 ) $ (45,513 )
Net loss attributable to common stockholders-basic and diluted $ (93,961 ) $ (45,513 )
Net loss per share attributable to common stockholders-basic and diluted $ (4.28 ) $ (3.55 )
Weighted-average common stock outstanding-basic and diluted 21,966,326 12,831,221
Applied Therapeutics, Inc.
(in thousands, except share and per share
As of As of
December 31, December 31,
2020 2019
ASSETS
CURRENT ASSETS:
Cash and cash equivalents $ 57,466 $ 18,850
Investments 39,363 20,004
Prepaid expenses and other current assets 5,764 7,301
Total current assets 102,593 46,155
Operating lease right-of-use asset 1,712 2,035
Security deposits and leasehold improvements 201 199
TOTAL ASSETS $ 104,506 $ 48,389
LIABILITIES AND STOCKHOLDERS' EQUITY
CURRENT LIABILITIES:
Current portion of operating lease liabilities $ 406 $ 356
Accounts payable 640 8,793
Accrued expenses and other current liabilities 20,189 4,950
Total current liabilities 21,235 14,099
NONCURRENT LIABILITIES:
Noncurrent portion of operating lease liabilities 1,332 1,683
Total noncurrent liabilities 1,332 1,683
Total liabilities 22,567 15,782
STOCKHOLDERS' EQUITY:
Common stock, $0.0001 par value; 100,000,000 shares authorized as of December 31, 2020 and December 31, 2019; 22,493,661 shares and 18,531,560 shares issued and outstanding as of December 31, 2020 and December 31, 2019, respectively 2 1
Additional paid-in capital 242,780 99,378
Accumulated other comprehensive loss (112 ) (2 )
Accumulated deficit (160,731 ) (66,770 )
Total stockholders' equity 81,939 32,607
TOTAL LIABILITIES AND STOCKHOLDERS' EQUITY $ 104,506 $ 48,389
Last updated: Mar 18, 2021