Core Viewpoint - Applied Therapeutics, Inc. has announced key executive appointments, promoting Evan Bailey to Chief Medical Officer and Dottie Caplan to Executive Vice President of Patient Advocacy and Government Affairs, effective June 15, 2025 [1][5]. Group 1: Executive Appointments - Evan Bailey, MD, has been promoted to Chief Medical Officer, succeeding Riccardo Perfetti, MD, PhD, who served since 2018 [5]. - Dottie Caplan has been promoted to Executive Vice President, Patient Advocacy and Government Affairs, expanding her role to include government affairs [5][6]. Group 2: Leadership Contributions - Dr. Bailey has been with Applied Therapeutics for four years and has played a critical role in advancing the company's development programs, particularly in clinical development execution [2][3]. - Ms. Caplan has been instrumental in integrating patient voices into clinical development strategies and will continue to lead advocacy efforts in her new role [2][4]. Group 3: Focus on Rare Diseases - The company is dedicated to developing treatments for rare diseases, with its lead drug candidate, govorestat, targeting conditions such as Classic Galactosemia and CMT-SORD [7]. - Dr. Bailey expressed commitment to advancing the portfolio of supporting data for govorestat to address high unmet medical needs in rare disease patients [2][3].

Core Insights - The company presented full 12-month clinical results and new topline data from 18 and 24 months of the INSPIRE Phase 2/3 trial for govorestat, aimed at treating CMT-SORD, at the Peripheral Nerve Society Annual Meeting in May 2025 [2][4][10] Group 1: Clinical Trial Results - Govorestat treatment demonstrated a statistically significant 37% reduction in sorbitol levels in the sciatic nerve of CMT-SORD rats, indicating its potential effectiveness [1] - MRI data at 24 months showed a slowing of disease progression in patients treated with govorestat compared to 12 months [8][9] - Statistically significant improvements were observed in the CMT-Health Index (CMT-HI) at 12 months, correlating with reductions in blood sorbitol levels [11][12] Group 2: Safety and Tolerability - Govorestat remained generally safe and well tolerated throughout the 24 months of treatment, with similar adverse event rates between active and placebo groups [12][11] Group 3: Regulatory Pathway and Future Plans - The company is committed to submitting a New Drug Application (NDA) for govorestat for CMT-SORD treatment in 2025 [2][10] - 90% of remaining patients from the INSPIRE trial have transitioned to an open-label extension study, indicating strong patient retention and interest in continued treatment [10] Group 4: Disease Background - CMT-SORD is a rare, progressive neuromuscular disease caused by genetic mutations affecting the sorbitol dehydrogenase enzyme, leading to high levels of sorbitol in blood and tissues [3][14] - The disease was officially recognized in 2020, and there are now commercially available tests for sorbitol and genetic testing specific to CMT-SORD [3][4]

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2025 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ______________ to ______________ Commission File Number: 001-38898 Applied Therapeutics, Inc. (Exact Name of Registrant as Specified in its Ch ...

Financial Performance - Cash and cash equivalents totaled $79.4 million as of December 31, 2024, compared to $49.9 million at December 31, 2023, representing a 59% increase[9] - Research and development expenses for the year ended December 31, 2024, were $48.7 million, a decrease of approximately 10% from $53.9 million for the year ended December 31, 2023[9] - General and administrative expenses increased to $56.0 million for the year ended December 31, 2024, from $20.6 million in 2023, marking an increase of approximately 172%[9] - The net loss for the year ended December 31, 2024, was $105.6 million, or $0.76 per share, compared to a net loss of $119.8 million, or $1.42 per share, for the year ended December 31, 2023[9] - Total revenue for the year ended December 31, 2024, was $455,000, a significant decrease from $9.99 million in 2023[16] - The accumulated deficit increased to $574.2 million as of December 31, 2024, compared to $468.6 million as of December 31, 2023[14] Regulatory and Strategic Developments - The company received a Complete Response Letter from the FDA regarding the New Drug Application for govorestat for Classic Galactosemia, and is evaluating its response[3] - The company is focused on preparing for potential regulatory interactions regarding govorestat in both Classic Galactosemia and SORD Deficiency[2] - The company appointed key senior executives to enhance its regulatory, medical, and quality affairs capabilities, including Todd F. Baumgartner as Chief Regulatory Officer[3] Asset Management - The total assets increased to $86.7 million as of December 31, 2024, from $54.8 million as of December 31, 2023[14]

Core Viewpoint - Applied Therapeutics, Inc. is focused on advancing its lead drug candidate, govorestat, for the treatment of rare diseases, particularly CMT-SORD and Classic Galactosemia, while preparing for upcoming regulatory interactions and clinical data presentations [2][6]. Recent Highlights - The company will present full 12-month clinical data and new topline 18-month and 24-month data from the INSPIRE Phase 3 trial of govorestat at the Peripheral Nerve Society 2025 Annual Meeting [5][6]. - Ongoing reviews of govorestat development programs for Classic Galactosemia and CMT-SORD Deficiency are progressing as planned [5][6]. Financial Results - Cash and cash equivalents and short-term investments totaled $50.8 million as of March 31, 2025, down from $79.4 million at December 31, 2024 [6]. - Research and development expenses for Q1 2025 were $7.8 million, a decrease from $12.2 million in Q1 2024, primarily due to reduced clinical and regulatory expenses [6]. - General and administrative expenses increased to $17.7 million in Q1 2025 from $9.1 million in Q1 2024, mainly due to higher legal and professional costs [6]. - The net loss for Q1 2025 was $21.8 million, or $0.15 per share, compared to a net loss of $83.9 million, or $0.67 per share, in Q1 2024 [6][13].

Core Insights - Applied Therapeutics, Inc. announced the presentation of full 12-month clinical results from the INSPIRE Phase 3 trial of govorestat (AT-007) at the Peripheral Nerve Society 2025 Annual Meeting, along with new topline 18-month and 24-month data [1] - The INSPIRE Phase 3 trial is a double-blind, placebo-controlled study evaluating govorestat's effect on patients with Sorbitol Dehydrogenase (SORD) Deficiency, a subtype of Charcot-Marie-Tooth (CMT) disease [1] - Govorestat is a central nervous system penetrant Aldose Reductase Inhibitor being developed for multiple rare diseases, including CMT-SORD, Classic Galactosemia, and PMM2-CDG [2][4] Presentation Details - The oral presentation titled "INSPIRE CMT-SORD Clinical Trial: Month 18 and Month 24 INSPIRE Trial Results and Designing the Next Randomized Study" will be presented by Dr. Evan Bailey on May 18, 2025 [2] - The presentation is scheduled for 12:25 - 12:40 p.m. BST [2] Regulatory Designations - Govorestat has received Orphan Medicinal Product Designation from the European Medicines Agency for both Galactosemia and CMT disease [3] - It has also received Orphan Drug Designation from the U.S. FDA for Galactosemia, PMM2 deficiency, and SORD Deficiency, along with Rare Pediatric Disease designation for Galactosemia and PMM2-CDG, and Fast Track designation for Galactosemia [3] Company Overview - Applied Therapeutics is a clinical-stage biopharmaceutical company focused on developing novel drug candidates targeting rare diseases [4] - The lead drug candidate, govorestat, is aimed at treating CNS rare metabolic diseases, including Classic Galactosemia, CMT-SORD, and PMM2-CDG [4]

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-K (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission file number 001-38898 Applied Therapeutics, Inc. (Exact name of registrant as specified in its charter) Delaware 81-3405262 (State ...

Core Viewpoint - Applied Therapeutics, Inc. reported its financial results for the fourth quarter and full year ended December 31, 2024, highlighting key leadership appointments and ongoing development programs for its lead drug candidate, govorestat, aimed at addressing unmet medical needs in rare diseases [1][2]. Leadership Appointments - John H. Johnson was appointed as Executive Chairman, and Les Funtleyder was named Interim Chief Executive Officer [5]. - Todd F. Baumgartner, MD, MPH was appointed as Chief Regulatory Officer, and Reena Thomas Colacot was appointed as Vice President and Head of Quality [5]. Development Programs - The company is focused on regulatory interactions regarding govorestat for Classic Galactosemia and Sorbitol Dehydrogenase (SORD) Deficiency [2]. - A Complete Response Letter (CRL) was received from the FDA for the NDA submitted for govorestat for Classic Galactosemia, and the company is evaluating its response [5]. Financial Results - Cash and cash equivalents increased to $79.4 million as of December 31, 2024, from $49.9 million at the end of 2023 [5]. - Research and development expenses decreased to $48.7 million in 2024 from $53.9 million in 2023, primarily due to reduced clinical and drug manufacturing costs [5]. - General and administrative expenses rose to $56.0 million in 2024 from $20.6 million in 2023, largely due to increased commercial and legal expenses [5]. - The net loss for 2024 was $105.6 million, or $0.76 per share, compared to a net loss of $119.8 million, or $1.42 per share, in 2023 [5][12]. Revenue Overview - Total revenue for 2024 was $455,000, a decrease from $9.99 million in 2023, with no license revenue reported for the year [12].

Core Viewpoint - Applied Therapeutics, Inc. has filed a Form 12b-25 with the SEC for a late filing of its Annual Report on Form 10-K for fiscal year 2024, granting an automatic 15-day extension to file the report by April 15, 2025 [1] Company Overview - Applied Therapeutics is a clinical-stage biopharmaceutical company focused on developing novel drug candidates targeting validated molecular pathways in rare diseases [3] - The lead drug candidate, govorestat, is an Aldose Reductase Inhibitor designed for treating central nervous system rare metabolic diseases, including Classic Galactosemia, SORD Deficiency, and PMM2-congenital disorder glycosylation [3] Filing Details - The Form 12b-25 allows the company additional time to finalize its financial statements, with the expectation to file the Form 10-K by April 15, 2025 [1] - The filing is accessible on the company's Investor Relations website under "SEC Filings" [2] - A subsequent announcement will be made to disclose the fourth quarter and full-year 2024 earnings results once the Form 10-K filing date is confirmed [2]

Core Insights - Applied Therapeutics, Inc. has appointed Todd F. Baumgartner, MD, MPH as Chief Regulatory Officer to lead the company's global regulatory strategy [1][3] - Dr. Baumgartner brings over 35 years of experience in drug development and has successfully led 12 New Drug Applications and marketing authorizations [2] - The company is focused on advancing its lead drug candidate, govorestat, for the treatment of rare diseases [4] Company Overview - Applied Therapeutics is a clinical-stage biopharmaceutical company dedicated to developing novel drug candidates targeting rare diseases [4] - The lead drug candidate, govorestat, is an Aldose Reductase Inhibitor aimed at treating CNS rare metabolic diseases [4] Leadership Background - Dr. Baumgartner previously served as Senior Vice President of Global Regulatory Affairs at Ovid Therapeutics, overseeing multiple development functions [2] - He has held senior roles at Acorda Therapeutics, Purdue Pharma, AstraZeneca Pharmaceuticals, and Bristol-Myers Squibb [2]