“We made significant progress in the first quarter across all three of our Phase 3 programs,” said
Recent Highlights
- Provided Regulatory Update on Galactosemia Program. In
January 2022 , the Company announced that the FDA would require clinical outcomes data for approval. Clinical outcomes are assessed in the pediatric clinical study every 6 months by a firewalled committee until the study reaches statistical significance. The Company met with the FDA inApril 2022 , and the FDA confirmed that the pediatric study as it is currently designed would support an NDA submission if statistical significance is reached. The 6-month clinical outcomes were assessed by the firewalled data monitoring committee, and it was determined that the safety/benefit profile supports continuing the study. The next committee evaluation will be following completion of the 12-month clinical outcomes assessments. - Presented Data on AT-007 Treatment in SORD Deficiency at the 2022 Annual Meeting of the
Peripheral Nerve Society . InMay 2022 , the Company presented data highlighting the effect of AT-007 treatment on sorbitol levels. In a pilot study in patients with Sorbitol Dehydrogenase (SORD) Deficiency, sorbitol level correlated with disease severity, and AT-007 treatment substantially reduced sorbitol levels. In a Drosophila model of disease, elevated sorbitol resulted in neuronal damage and decline in mobility, and AT-007 treatment prevented the disease phenotype by inhibiting sorbitol production. Taken together, this data significantly advances the understanding of the disease and potential for treatment with AT-007. - Presented Data on Galactosemia Disease Progression at the 2022 43rd Annual Meeting of the
Society for Inherited Metabolic Disorders . InApril 2022 , the Company presented data featuring baseline disease characteristics of 47 pediatric patients with Classic Galactosemia, illustrating the high burden of disease associated with Galactosemia, including neurological complications, due to high galactitol levels. In addition, data presented at the meeting illustrated that higher galactitol levels, but not higher Gal-1p levels, were associated with greater disease severity overall and on each of the four quadrants of central nervous system (CNS) function. Together, these data underscore the urgent need to bring a treatment to people living with Galactosemia. - Presented Data on Galactosemia Disease Progression at the 2022 Annual Clinical Genetics Meeting of the
American College of Medical Genetics and Genomics . InMarch 2022 , the Company presented data featuring the first therapeutic interventional clinical trial in pediatric patients with Classic Galactosemia, demonstrating that AT-007 significantly reduces toxic galactitol in ACTION-Galactosemia children.
Financial Results
- Cash and cash equivalents and short-term investments totaled
$55.7 million as ofMarch 31, 2022 , compared with$80.8 million atDecember 31, 2021 . - Research and development expenses for the three months ended
March 31, 2022 were$15.0 million , compared to$14.4 million for the three months endedMarch 31, 2021 . The increase of$0.6 million was primarily due to an increase in clinical and pre-clinical expense of$5.1 million , primarily related to the progression of the SORD pivotal trial, progression of the AT-007 ACTION-Galactosemia long-term extension adult study, and progression of the AT-007 ACTION-Galactosemia Kids pediatric registrational study; an increase in personnel expenses of$0.8 million due to the increase in headcount in support of our clinical program pipeline; an increase in regulatory and other expenses of$0.2 million ; an increase in stock-based compensation of$0.1 million due to new stock option and restricted stock grants; and offset by a decrease in drug manufacturing and formulation costs of$5.6 million primarily related to the completion and release of AT-001 and AT-007 drug product batches in the three months endedMarch 31, 2021 . - General and administrative expenses were
$8.1 million for the three months endedMarch 31, 2022 , compared to$9.8 million for the three months endedMarch 31, 2021 . The decrease of$1.7 million was due to a decrease in commercial expenses of$0.6 million related to a decrease in spend relating to commercial operations; a decrease in other expenses of$0.3 million relating to decreased costs of other office expenses; a decrease in stock-based compensation of$1.0 million relating to options being forfeited during the current period; a decrease in legal and professional fees of$0.2 million due to lower external legal fees; offset by an increase in personnel expenses of$0.2 million related to an increase in headcount; and an increase in insurance expenses of$0.1 million related to increased insurance costs. - Net loss for the first quarter of 2022 was
$23.1 million , or$0.88 per basic and diluted common share, compared to a net loss of$24.2 million , or$1.00 per basic and diluted common share, for the first quarter 2021.
About
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 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. 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, 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
Contacts
Investors:
(212) 600-1902 or
appliedtherapeutics@argotpartners.com
Media:
media@appliedtherapeutics.com
Statement of Operations
(in thousands, except share and per share data)
Three Months Ended | ||||||||
2022 | 2021 | |||||||
OPERATING EXPENSES: | ||||||||
Research and development | $ | 15,030 | $ | 14,448 | ||||
General and administrative | 8,071 | 9,751 | ||||||
Total operating expenses | 23,101 | 24,199 | ||||||
LOSS FROM OPERATIONS | (23,101 | ) | (24,199 | ) | ||||
OTHER INCOME (EXPENSE), NET: | ||||||||
Interest income (expense), net | 76 | 76 | ||||||
Other income (expense) | (96 | ) | (56 | ) | ||||
Total other income (expense), net | (20 | ) | 20 | |||||
Net loss | $ | (23,121 | ) | $ | (24,179 | ) | ||
Net loss attributable to common stockholders—basic and diluted | $ | (23,121 | ) | $ | (24,179 | ) | ||
Net loss per share attributable to common stockholders—basic and diluted | $ | (0.88 | ) | $ | (1.00 | ) | ||
Weighted-average common stock outstanding—basic and diluted | 26,215,514 | 24,135,735 |
Balance Sheet
(in thousands, except share and per share data)
As of | As of | |||||||
2022 | 2021 | |||||||
(Unaudited) | ||||||||
ASSETS | ||||||||
CURRENT ASSETS: | ||||||||
Cash and cash equivalents | $ | 38,214 | $ | 53,888 | ||||
Investments | 17,437 | 26,935 | ||||||
Prepaid expenses and other current assets | 7,913 | 7,571 | ||||||
Total current assets | 63,564 | 88,394 | ||||||
Operating lease right-of-use asset | 1,190 | 1,298 | ||||||
Security deposits and leasehold improvements | 199 | 200 | ||||||
TOTAL ASSETS | $ | 64,953 | $ | 89,892 | ||||
LIABILITIES AND STOCKHOLDERS’ EQUITY | ||||||||
CURRENT LIABILITIES: | ||||||||
Current portion of operating lease liabilities | $ | 453 | $ | 442 | ||||
Accounts payable | 5,922 | 9,461 | ||||||
Accrued expenses and other current liabilities | 15,842 | 16,559 | ||||||
Total current liabilities | 22,217 | 26,462 | ||||||
NONCURRENT LIABILITIES: | ||||||||
Noncurrent portion of operating lease liabilities | 773 | 891 | ||||||
Total noncurrent liabilities | 773 | 891 | ||||||
Total liabilities | 22,990 | 27,353 | ||||||
STOCKHOLDERS’ EQUITY: | ||||||||
Common stock, | 3 | 3 | ||||||
Preferred stock, par value | - | - | ||||||
Additional paid-in capital | 331,476 | 328,958 | ||||||
Accumulated other comprehensive loss | (80 | ) | (107 | ) | ||||
Accumulated deficit | (289,436 | ) | (266,315 | ) | ||||
Total stockholders' equity | 41,963 | 62,539 | ||||||
TOTAL LIABILITIES AND STOCKHOLDERS’ EQUITY | $ | 64,953 | $ | 89,892 |
Source:
2022 GlobeNewswire, Inc., source