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a THERAPEUTICS Applied Therapeutics Reports First Quarter 2021 Financial Results May 11 , 2021 Phase 2 pilot study of AT - 007 initiated in patients with SORD Deficiency NDA submission for AT - 007 in Galactosemia expected no later than Q3 2021 ; commercial preparations ongoing Completion of enrollment in ARISE - HF Phase 3 global registrational study of AT - 001 in Diabetic Cardiomyopathy on track for mid - 2021 Ended 1Q21 with a strong balance sheet of $ 148.1 in cash and cash equivalents and short - term investments NEW YORK , May 11 , 2021 ( GLOBE NEWSWIRE ) -- Applied Therapeutics , Inc. ( Nasdaq : APLT ) ( " Applied Therapeutics " or 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 , 2021 . " In the first quarter we continued to progress our late stage programs towards commercialization in Galactosemia and Diabetic Cardiomyopathy , while advancing our additional rare disease programs in SORD and PMM2 - CDG , " said Shoshana Shendelman , PhD , Founder , CEO and Chair of the Board of Applied Therapeutics . " We are looking forward to our planned NDA submission in Galactosemia in the third quarter of this year , and continue to focus on our primary objective - bringing drugs to patients in desperate need of treatment . " Recent Highlights • Initiated Phase 2 Study of AT - 007 in Patients with SORD Deficiency . The Phase 2 pilot study of AT - 007 in patients with Sorbitol Dehydrogenase Deficiency ( SORD Deficiency ) , a progressive , debilitating hereditary neuropathy that affects peripheral nerves and motor neurons , has been initiated . Preclinical findings demonstrate that AT - 007 has the potential to be the first disease - modifying therapy for SORD , targeting the underlying cause of disease . • Presented Data on Galactosemia Disease Progression at the 2021 Annual Clinical Genetics Meeting of the American College of Medical Genetics and Genomics . In April 2021 , the Company presented data featuring a cross sectional analysis of nineteen pediatric patients with Classic Galactosemia , providing meaningful insight on the progressive worsening of the central nervous system phenotype with age . • Hosted Virtual Rare Disease Forum . In March 2021 , the Company hosted a virtual forum highlighting its AT - 007 development programs in Galactosemia , SORD Deficiency , and PMM2 - CDG . A replay of the presentation , which featured several rare disease key opinion leaders , is available at https : //www.applied therapeutics.com/presentations and - publications / . • Announced Restart of Pediatric Galactosemia Study . In February 2021 , the Company announced that the FDA lifted the clinical hold on the AT - 007 ACTION - Galactosemia Kids pediatric clinical study , and the study resumed immediately . Applied Therapeutics worked closely with FDA to modify the trial , with the shared goal of ensuring that all patients have the opportunity to receive clinical benefit , and remains on target to submit an NDA no later than Q3 2021 . • Closed Approximately $ 75 million Public Offering . In February 2021 , the Company completed an underwritten public offering of 3,450,000 shares of its common stock , including the exercise in full of the underwriters ' option to purchase 450,000 additional shares of common stock at a price of $ 23.00 per share , resulting in aggregate net proceeds of approximately $ 74.4 million . Financial Results • Cash and cash equivalents and short - term investments totaled $ 148.1 million as of March 31 , 2021 , compared with $ 96.8 million at December 31 , 2020 . • Research and development expenses for the three months ended March 31 , 2021 were $ 14.4 million , compared to $ 7.3 million for the three months ended March 31 , 2020. The increase of $ 7.2 million was primarily related to an increase in clinical and pre - clinical expense of $ 1.3 million , related to the progression of the AT - 007 ACTION - Galactosemia adult extension study , the AT - 007 ACTION - Galactosemia Kids pediatric registrational study and the AT - 001 Phase 3 ARISE - HF clinical study ; an increase in drug manufacturing and formulation costs of $ 5.2 million related to the progression of the manufacturing campaigns and the completion and release of AT - 001 and AT - 007 drug product batches ; an increase in personnel expenses of $ 0.3 million due to the increase in headcount in support of our clinical program pipeline ; an