Core Viewpoint - Applied Therapeutics has initiated a process to explore strategic alternatives to maximize shareholder value, which may include mergers, acquisitions, partnerships, and other strategic transactions [1][2][3] Strategic Alternatives - The Board is evaluating a broad range of opportunities to enhance shareholder value, including potential mergers, acquisitions, partnerships, joint ventures, and licensing arrangements [2] - There is no set timetable for the completion of this strategic review process, and the company will not provide further public comments unless necessary [3] Workforce Reduction - The company is reducing its workforce by approximately 46% as part of cost-containment and cash conservation measures [4] Commitment to Patients - The interim CEO emphasized the company's commitment to advancing govorestat for patients with rare diseases while maximizing shareholder value [5] Regulatory Updates - The company completed a Type C meeting with the FDA regarding govorestat for the treatment of Charcot-Marie-Tooth Sorbitol Dehydrogenase Deficiency (CMT-SORD) and plans to submit a request for an additional Type C meeting to discuss a potential Phase 3 trial design [6] - Feedback from the FDA regarding govorestat for Classic Galactosemia is being evaluated, with potential discussions for a Phase 3 trial design to follow [7] Company Overview - Applied Therapeutics is a clinical-stage biopharmaceutical company focused on developing novel drug candidates for rare diseases, with its lead candidate being govorestat, aimed at treating CNS rare metabolic diseases [8]

Core Insights - The company is making significant progress in its regulatory strategy for govorestat, particularly for the treatment of CMT-SORD, with a meeting scheduled with the FDA in Q4 2025 to discuss the potential NDA submission [1][3][7] - New data on govorestat for PMM2-CDG has been published, showing promising results that support further clinical development [2][4][9] - A leadership transition has occurred within the Board of Directors, with John Johnson stepping down as Executive Chairman [10] Regulatory Strategy - The company received Type C meeting minutes from the FDA regarding govorestat for CMT-SORD, which included constructive feedback on the data submitted and requirements for a potential NDA submission [7] - An additional Type C meeting is planned to discuss the design of a potential Phase 3 trial for CMT-SORD [3][7] - A meeting is also scheduled with the FDA to review govorestat for Classic Galactosemia, following a Complete Response Letter received in November 2024 [6][8] Clinical Development - New data from a single-patient trial for PMM2-CDG was published in JIMD and presented at the 2025 ASHG Annual Meeting, indicating a dose-dependent decrease in whole blood sorbitol and a 46% improvement in the Nijmegen Pediatric CDG Rating Scale [9] - Govorestat has received Orphan Drug Designation and Rare Pediatric Disease designation from the FDA for PMM2-CDG [9] Financial Performance - As of September 30, 2025, the company reported cash and cash equivalents of $11.9 million, a decrease from $79.4 million at the end of 2024 [13] - Research and development expenses for Q3 2025 were $9.6 million, down from $14.8 million in Q3 2024, primarily due to reduced clinical and preclinical expenses [13] - The net loss for Q3 2025 was $19.0 million, or $0.13 per share, compared to a net loss of $68.6 million, or $0.48 per share, for the same period in 2024 [13][21]

Core Viewpoint - Applied Therapeutics, Inc. has completed a meeting with the U.S. FDA regarding the potential new drug application for govorestat to treat Charcot-Marie-Tooth Sorbitol Dehydrogenase Deficiency (CMT-SORD) in Q3 2025 [1][2] Company Overview - Applied Therapeutics is a clinical-stage biopharmaceutical company focused on developing transformative treatments for rare diseases [1][6] - The lead drug candidate, govorestat, is an Aldose Reductase Inhibitor aimed at treating multiple rare diseases, including CMT-SORD, Classic Galactosemia, and PMM2-CDG [4][6] Disease Information - CMT-SORD is a rare hereditary neuropathy affecting approximately 3,300 patients in the U.S. and 4,000 patients in the EU, characterized by significant disability and loss of sensory function due to sorbitol accumulation [3] - The disease results from a deficiency of the enzyme sorbitol dehydrogenase, leading to high levels of sorbitol in blood and tissues [3] Regulatory Status - Govorestat has received Orphan Medicinal Product Designation from the EMA for both Galactosemia and CMT-SORD, and Orphan Drug Designation from the FDA for the same conditions [5] - The company is awaiting official meeting minutes from the FDA to determine the next steps for the NDA submission strategy, including potential eligibility for the accelerated approval pathway [2][5]

Company Developments - Innovation Beverage Group Limited (NASDAQ:IBG) experienced a 312% surge in stock price to $2.37, driven by strategic expansion in the beverage sector and a potential merger with BlockFuel Energy Inc. [2] - bioAffinity Technologies, Inc. (BIAFW) saw a 265% jump in stock price to $0.28, linked to the release of three new case studies demonstrating the clinical value of its product, CyPath® Lung, in detecting early-stage lung cancer [3] - MEDIROM Healthcare Technologies Inc. (NASDAQ:MRM) recorded a 116% rise in stock price to $3.13, attributed to the adoption of its REMONY system by the Japan Ground Self-Defense Force [4] - Applied Therapeutics, Inc. (APLT) experienced a 71% increase in stock price to $0.95, driven by progress in clinical trials for its lead product candidate AT-007 and advancements in the CMT-SORD program [5] Market Trends - Recent market trends indicate significant price changes for several companies, reflecting key developments and heightened investor interest [1] - The dynamic nature of the stock market is underscored by how company developments and investor sentiment can significantly influence stock prices [6]

Core Insights - The company is making significant progress in its clinical operations and pipeline, particularly with govorestat for the treatment of CMT-SORD, and is preparing for a meeting with the FDA in Q3 2025 regarding a potential NDA submission [2][3] - A new sponsored Urine Sorbitol Assay has been launched to aid in the identification of patients with CMT-SORD, which is available at no cost to healthcare providers [3] - The company presented full 12-month clinical results from the INSPIRE Phase 2/3 trial of govorestat, showing slowed disease progression and statistically significant improvements in key secondary endpoints [3] CMT-SORD - A meeting with the FDA is scheduled to discuss the NDA submission strategy for govorestat, focusing on its treatment for CMT-SORD [3] - The company launched a Urine Sorbitol Assay to help healthcare providers identify suspected cases of CMT-SORD, removing barriers to diagnosis and treatment [3] - Full clinical results from the INSPIRE trial indicated that govorestat is generally safe and well-tolerated, with significant improvements in various health metrics [3] Classic Galactosemia - The company is reviewing its development program for govorestat in Classic Galactosemia, including responses to the Complete Response Letter from the FDA [4] PMM2-CDG - Results from an ongoing trial of govorestat for PMM2-CDG will be presented at the 2025 ASHG Annual Meeting, highlighting the drug's potential in treating this ultra-rare disease [5] Corporate Developments - The company entered into an out-licensing agreement with Biossil, Inc. for AT-001, gaining upfront payment and potential future royalties [9] - Key leadership changes were made, promoting Evan Bailey, M.D., to Chief Medical Officer and Dottie Caplan to Executive Vice President [9] Financial Results - As of June 30, 2025, cash and cash equivalents totaled $30.4 million, down from $79.4 million at the end of 2024 [7] - Research and development expenses for Q2 2025 were $9.9 million, slightly down from $10.0 million in Q2 2024 [7] - General and administrative expenses increased to $13.2 million in Q2 2025 from $10.6 million in Q2 2024, contributing to a net loss of $21.3 million for the quarter [7]

Core Insights - The company is advancing its CMT-SORD program and has scheduled a meeting with the FDA in Q3 2025 to discuss the New Drug Application (NDA) submission for govorestat [1][4] - Full 12-month clinical results from the INSPIRE Phase 2/3 trial of govorestat for CMT-SORD were presented, showing slowed disease progression observed via MRI at 24 months [1][4] - A new sponsored Sorbitol Assay has been launched to assist in identifying patients suspected of having CMT-SORD [1][4] - The company has entered into an out-licensing agreement with Biossil, Inc. for AT-001, an investigational therapy for Diabetic Cardiomyopathy [1][9] CMT-SORD Program - A meeting with the FDA is planned to align on the NDA submission strategy for govorestat, focusing on comprehensive data for Sorbitol Dehydrogenase Deficiency [4] - The newly launched Urine Sorbitol Assay aims to support healthcare providers in identifying suspected CMT-SORD cases at no cost to patients [4] - Clinical results from the INSPIRE trial indicated that govorestat was generally safe and well-tolerated, with significant improvements in key secondary endpoints at 12 months [4] Other Development Programs - New data on govorestat for PMM2-CDG will be presented at the 2025 ASHG Annual Meeting [2][6] - The review of the govorestat development program for Classic Galactosemia is progressing as planned, including responses to the Complete Response Letter from the FDA [5] Financial Performance - As of June 30, 2025, cash and cash equivalents totaled $30.4 million, down from $79.4 million at the end of 2024 [8] - Research and development expenses for Q2 2025 were $9.9 million, slightly down from $10.0 million in Q2 2024 [8] - General and administrative expenses increased to $13.2 million in Q2 2025 from $10.6 million in Q2 2024 [8] - The net loss for Q2 2025 was $21.3 million, compared to a net income of $2.9 million in Q2 2024 [8][15]

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 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 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]