Welcome to our dedicated page for Applied Therapeutics news (Ticker: APLT), a resource for investors and traders seeking the latest updates and insights on Applied Therapeutics stock.
Applied Therapeutics, Inc. (Nasdaq: APLT) is a clinical-stage biopharmaceutical company that regularly issues news about its progress in developing novel drug candidates for rare diseases. The company’s updates frequently focus on its lead drug candidate, govorestat (AT-007), a central nervous system penetrant Aldose Reductase Inhibitor (ARI) being developed for CNS rare metabolic diseases such as Classic Galactosemia, Charcot-Marie-Tooth Sorbitol Dehydrogenase Deficiency (CMT-SORD), and phosphomannomutase 2 congenital disorder of glycosylation (PMM2-CDG).
On this APLT news page, readers can find company press releases covering clinical trial milestones, including detailed data from the INSPIRE Phase 2/3 and registrational Phase 3 trial of govorestat in CMT-SORD, investigator-initiated studies in PMM2-CDG, and regulatory interactions with the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). These items often describe safety findings, sorbitol reduction, functional and patient-reported outcomes, and plans for potential New Drug Application (NDA) strategies.
News items also report on regulatory designations for govorestat, such as Orphan Drug, Orphan Medicinal Product, Rare Pediatric Disease, and Fast Track designations, as well as corporate developments like leadership appointments, workforce changes, cost-containment measures, and business development activities. Examples include the out-licensing of AT-001 for diabetic cardiomyopathy and partnership arrangements related to the company’s pipeline.
Another key category of APLT news involves strategic and capital markets events. Applied Therapeutics has announced a strategic review process, at-the-market equity offering agreements, Nasdaq listing and bid price notices, and, most notably, an Agreement and Plan of Merger with Cycle Group Holdings Limited. Updates related to the planned tender offer, contingent value rights (CVR) structure, and expected acquisition timeline are central topics for investors following APLT.
By reviewing the Applied Therapeutics news feed, investors and observers can track how the company describes its clinical, regulatory, financial, and strategic developments over time, and how these developments relate to the evolution of APLT stock and the proposed acquisition by Cycle.
Applied Therapeutics (Nasdaq: APLT) announced that the European Medicines Agency (EMA) has granted orphan medicinal product designation for its drug AT-007 (gavorestat) to treat Galactosemia. This designation recognizes the significant medical need and potential of AT-007 to reduce toxic galactitol levels in patients. The company plans to meet with the EMA in Q3 to discuss a potential submission for conditional approval. The orphan status provides various incentives, including a decade of market exclusivity upon approval.
Applied Therapeutics, Inc. (Nasdaq: APLT) is participating in a fireside chat at the UBS Global Healthcare Conference on March 25, 2022, at 10:45 a.m. ET, in New York. The event can be accessed via webcast on their Investor Relations page, with a replay available post-event. The company specializes in developing novel biopharmaceutical candidates targeting significant unmet medical needs, including CNS rare metabolic diseases and diabetic cardiomyopathy. Their leading drug candidates include AT-007 and AT-001, among others.
Applied Therapeutics (APLT) reported progress in three Phase 3 trials in areas of high unmet medical need, with multiple clinical milestones anticipated in 2022. The company achieved alignment with the FDA regarding its Galactosemia program, which requires clinical outcomes data for NDA submission. Financially, as of March 31, 2022, cash and equivalents totaled $55.7 million, down from $80.8 million at year-end. Research and development expenses rose to $15 million, while general and administrative expenses decreased to $8.1 million. The net loss for Q1 2022 was $23.1 million, or $0.88 per share.
Applied Therapeutics, Inc. (Nasdaq: APLT) announced findings at the 2022 Annual Meeting of the Peripheral Nerve Society regarding SORD Deficiency. Data revealed that high levels of sorbitol correlate with disease severity. In a pilot study, treatment with AT-007 significantly reduced sorbitol levels, which are linked to mitochondrial dysfunction and neuronal loss. The findings indicate AT-007 could prevent disease progression by inhibiting sorbitol production. This research highlights the potential of AT-007, a novel Aldose Reductase inhibitor, in treating SORD Deficiency.
Applied Therapeutics (Nasdaq: APLT) announced poster presentations at the 43rd Annual Meeting of the Society for Inherited Metabolic Disorders, held April 10-13, 2022, in Orlando, Florida. The presentations highlighted baseline data from the ACTION-Galactosemia Kids study, revealing significant disease burdens in pediatric patients with Galactosemia, including neurological issues due to elevated galactitol levels. The drug candidate AT-007, designed for treating Galactosemia, is undergoing a Phase 3 clinical trial, showcasing its potential in reducing toxic levels and disease complications.
Applied Therapeutics (Nasdaq: APLT) announced the acceptance of their abstract #OP005 for an oral presentation at the 2022 Annual Clinical Genetics Meeting, scheduled for March 22-26 in Nashville. This presentation will focus on the efficacy of AT-007 in reducing toxic galactitol levels in pediatric patients with Classic Galactosemia. Dr. Riccardo Perfetti highlighted the significant correlation between galactitol levels and CNS deficit severity in patients. Approximately 3,000 individuals are affected by Galactosemia in the U.S., emphasizing the importance of ongoing clinical research and development.
Applied Therapeutics, Inc. (Nasdaq: APLT) reported its fourth-quarter and full-year financial results for 2021, showing a net loss of $105.6 million, or $4.12 per share, up from a loss of $94.0 million in 2020. The company is focused on three ongoing Phase 3 trials in Galactosemia, SORD Deficiency, and Diabetic Cardiomyopathy. A regulatory update indicates the FDA may require clinical outcomes data for the Galactosemia program before NDA submission. Cash reserves are reported at $80.8 million, down from $96.8 million in 2020, while R&D expenses increased to $62.6 million.
Applied Therapeutics, Inc. (Nasdaq: APLT) has announced its participation in two key investor conferences in March 2022. The Cowen 42nd Annual Health Care Conference will be held on March 7, 2022, with a panel discussion scheduled from 9:10 to 10:10 a.m. ET. The Barclays Global Healthcare Conference is set for March 17, 2022, featuring a presentation from 10:15 to 10:40 a.m. ET. Interested investors can access the webcast on the company's Investor Relations page and listen to replays afterward. The company focuses on developing treatments for unmet medical needs.
Applied Therapeutics (Nasdaq: APLT) is marking the 15th Annual Rare Disease Day to enhance awareness of rare diseases, specifically focusing on Galactosemia and SORD Deficiency. The company is actively supporting events like the GLOW for Galactosemia in Knoxville, Tennessee, and collaborating with advocacy partners on various initiatives. Its lead drug candidate, AT-007, aims to treat rare metabolic diseases with a high unmet medical need. The company emphasizes its commitment to developing solutions for these conditions, aligning with its clinical-stage development goals.
Applied Therapeutics, a clinical-stage biopharmaceutical company (Nasdaq: APLT), will present at the 11th Annual SVB Leerink Global Healthcare Conference on February 18, 2022, at 11:20 a.m. ET. The event's webcast can be accessed via the company's Investor Relations page. Applied Therapeutics focuses on developing novel drug candidates for high unmet medical needs, notably with three Phase 3 programs in progress, including lead drug AT-007 for rare CNS diseases and AT-001 for Diabetic Cardiomyopathy.