On March 10, 2022 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, reported financial results for the fourth quarter and full year ended December 31, 2021 (Press release, Applied Therapeutics, MAR 10, 2022, View Source [SID1234609906]).
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"We remain committed to bringing new treatments to patients in areas of high unmet need," said Shoshana Shendelman, PhD, Founder and CEO of Applied Therapeutics. "With three ongoing Phase 3 studies across Galactosemia, Sorbitol Dehydrogenase (SORD) Deficiency and Diabetic Cardiomyopathy, we are advancing treatments forward, and expect multiple catalysts and opportunities for value creation in 2022 and 2023."
Recent Highlights
Provided Regulatory Update on Galactosemia Program. In January 2022, the Company provided a regulatory update on the AT-007 Galactosemia program. Following discussions with the FDA at the end of the year, the Company decided to hold on submitting an NDA for AT-007 for treatment of Galactosemia pending additional discussions with the agency. Although the Galactosemia program had previously been discussed in the context of an NDA for Accelerated Approval based on reduction in galactitol, the FDA has now indicated that clinical outcomes data will likely be required for approval. Clinical outcomes are assessed every 6 months by a firewalled committee until the study reaches statistical significance. All children enrolled in the study have completed the 6-month clinical outcomes visits. The Company is in active dialogue with the FDA regarding a potential NDA submission and expects to complete the statistical analysis of the 6-month clinical outcomes in the second quarter. The Company expects to provide an update accordingly.
Announced Initiation of Registrational Phase 2/3 Study of AT-007 in SORD Deficiency. In December 2021, the Company initiated the global registrational phase 2/3 study of AT-007 in Sorbitol Dehydrogenase (SORD) Deficiency. The study, termed INSPIRE (INhibition of Sorbitol Production through Inhibition of the Aldose Reductase Enzyme), will investigate biomarker efficacy, clinical outcomes and safety in people living with SORD Deficiency treated with AT-007 vs. placebo.
Financial Results
Cash and cash equivalents and short-term investments totaled $80.8 million as of December 31, 2021, compared with $96.8 million at December 31, 2020.
Research and development expenses for the year ended December 31, 2021 were $62.6 million, compared to $61.8 million for the year ended December 31, 2020. The increase of approximately $0.8 million was primarily related to an increase in clinical and pre-clinical expense of $11.6 million, primarily related to the progression of the AT-007 ACTION-Galactosemia adult extension and the AT-007 ACTION-Galactosemia Kids pediatric registrational study; a decrease in drug manufacturing and formulation expenses of $12.5 million primarily related to the completion and release of AT-001 and AT-007 drug product batches in the three months ended March 31, 2021; an increase in personnel expenses of $2.2 million due to the increase in headcount in support of our clinical program pipeline; an increase in stock-based compensation of $0.2 million due to new stock option and restricted stock unit grants, offset by forfeitures of stock option and restricted stock unit grants; and a decrease of regulatory and other expenses of $0.6 million primarily related to the UM license fees recognized during the year ended December 31, 2020.
General and administrative expenses were $43.0 million for the year ended December 31, 2021, compared to $32.7 million for the year ended December 31, 2020. The increase of approximately $10.4 million was primarily related to a decrease in professional and legal fees of $2.1 million due to lower external consulting and legal fees; an increase of $5.6 million related to increased commercial expenditures; an increase in personnel expenses of $1.0 million and an increase in stock-based compensation of $3.0 million due to an increase in headcount; an increase of insurance expenses of $0.6 million related to increased directors and officers liability insurance costs; and an increase in other expenses of $2.3 million, primarily relating to increased costs of rent and other office expenses.
Net loss for the year ended December 31, 2021 was $105.6 million, or $4.12 per basic and diluted common share, compared to a net loss of $94.0 million, or $4.28 per basic and diluted common share, for the year ended December 31, 2020.