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APPLIED a THERAPEUTICS Applied Therapeutics Reports Biomarker Data from Pilot Trial of AT - 007 in SORD Deficiency October 25 , 2021 Substantial and significant reduction in sorbitol Company plans to initiate registrational trial end of 2021 Company to host conference call and webcast today at 8:30 a.m. ET NEW YORK , Oct. 25 , 2021 ( GLOBE NEWSWIRE ) 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 biomarker data from a pilot trial of AT - 007 in patients with SORD Deficiency . Sorbitol Dehydrogenase Deficiency ( SORD Deficiency ) is a rare , progressive , debilitating hereditary neuropathy that affects peripheral nerves and motor neurons . SORD Deficiency affects approximately 3,300 patients in the US and 4,000 patients in Europe . The disease is caused by a lack of the enzyme sorbitol dehydrogenase , responsible for metabolism of sorbitol , which causes sorbitol to accumulate at high levels and become toxic to the body . Sorbitol accumulation results in significant disability , loss of sensory function , and neuromuscular dysfunction . Patients with SORD Deficiency have 100 times higher sorbitol concentration in their blood compared with unaffected individuals . In a pilot open - label study in 8 SORD Deficiency patients , AT - 007 reduced blood sorbitol levels by approximately 66 % from baseline through 30 days of treatment . The range of reduction from baseline in patients was 54 % -75 % . AT - 007 was safe and well tolerated in all treated patients . These results , in addition to preclinical findings , demonstrate that AT - 007 has the potential to be the first disease - modifying the for SORD Deficiency . The Company plans to initiate a registrational study by the end of 2021. In advance of the registrational study start , patients can now pre - screen to determine whether they have SORD and if they may qualify for the upcoming trial . " Reduction in toxic sorbitol is critically important in patients with SORD Deficiency . This data demonstrates a significant effect on the underlying cause of the disease , " said Michael Shy , MD , Professor of Neurology and Director of the Division of Neuromuscular Medicine at the University of lowa Hospital Carver School of Medicine . " AT - 007 represents an important advancement for patients with SORD Deficiency , and a unique opportunity to meaningfully impact patients ' lives . We look forward to beginning our registrational trial for this indication in the coming months , " said Riccardo Perfetti , MD , PhD , Chief Medical Officer of Applied Therapeutics . Increased access to screening and early diagnosis can dramatically improve patients ' lives , and the Company is collaborating with the Charcot Marie Tooth Association and the Hereditary Neuropathy Foundation to improve access to SORD diagnostic testing , and to better understand the perspectives of individuals living with SORD . Conference Call Information Applied Therapeutics will host a conference call today , Monday , October 25 , 2021 , at 8:30 a.m. Eastern Time , to discuss data from a pilot trial of AT - 007 in SORD deficiency . To access the conference call , please dial ( 800 ) 369-8554 ( local ) or ( 409 ) 937-8917 ( international ) at least 10 minutes prior to the start time and refer to conference ID 2437605. A live webcast of the call will be accessible on the Events page under the Investor Relations section of the Applied Therapeutics website at www.appliedtherapeutics.com . A replay will be available on the Company's website approximately two hours after the event . 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 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 , as well as novel dual Pl3k inhibitors in preclinical development for orphan oncology indications . 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 . These include , without limitation , statements regarding ( i ) the Company's plan to initiate a registrational study by the end of 2021 , ( ii ) AT - 007 potential to be the first disease - modifying therapy for SORD Deficiency , ( iii ) the timing of the initiation and completion of our clinical trials , ( iv ) the likelihood that data from our clinical trials will support future development of our product candidates and ( v ) 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