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]

- Company to present full 12-month clinical data and new topline 18-month and 24-month data from INSPIRE registrational Phase 3 trial of govorestat in CMT-SORD Deficiency at the Peripheral Nerve Society 2025 Annual Meeting - Review of govorestat development programs for Classic Galactosemia and CMT-SORD Deficiency ongoing NEW YORK, May 13, 2025 (GLOBE NEWSWIRE) -- Applied Therapeutics, Inc. (Nasdaq: APLT) (the “Company”), a clinical-stage biopharmaceutical company dedicated to creating transformative treatm ...

NEW YORK, May 09, 2025 (GLOBE NEWSWIRE) -- Applied Therapeutics, Inc. (Nasdaq: APLT) (the “Company”), a clinical-stage biopharmaceutical company dedicated to creating transformative treatments for rare diseases, today announced that it will present full 12-month clinical results from the INSPIRE Phase 3 trial of govorestat (AT-007) in a late-breaking abstract selected for oral presentation at the Peripheral Nerve Society (PNS) 2025 Annual Meeting, being held May 17-20, 2025 in Edinburgh, Scotland. The prese ...

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].

The Company will make a subsequent announcement to disclose its fourth quarter and full-year 2024 earnings results once the filing date of its Form 10-K is confirmed. About Applied Therapeutics NEW YORK, March 31, 2025 (GLOBE NEWSWIRE) -- Applied Therapeutics, Inc. (Nasdaq: APLT) (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 announced that it has filed a Form 12b- ...

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]

Core Viewpoint - A securities class action lawsuit has been filed against Applied Therapeutics, Inc. for alleged misconduct related to its drug candidate, govorestat, during the Class Period from January 3, 2024, to December 2, 2024 [1][2]. Group 1: Lawsuit Details - The lawsuit was filed in the United States District Court for the Southern District of New York on behalf of investors who acquired Applied Therapeutics securities during the specified Class Period [1]. - The lead plaintiff deadline for the lawsuit is set for February 18, 2025 [1][3]. - The complaint alleges that the defendants made false or misleading statements and failed to disclose non-adherence to trial protocols and good clinical practices, which posed a significant risk of FDA rejection of trial data [2]. Group 2: Lead Plaintiff Process - Investors in Applied Therapeutics can seek to be appointed as lead plaintiff representatives by the deadline of February 18, 2025, or choose to remain absent class members [3]. - The lead plaintiff will represent all class members in directing the litigation and is typically the investor or small group with the largest financial interest [3]. Group 3: Firm Background - Kessler Topaz Meltzer & Check, LLP is known for prosecuting class actions and has a reputation for recovering billions for victims of corporate misconduct [4]. - The firm encourages investors who have suffered significant losses to contact them for more information regarding the lawsuit [4].