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 a regulatory update on its AT-007 Galactosemia program. Following discussions with the FDA, the company will delay submitting an NDA for AT-007, as clinical outcomes data will now likely be required for approval. The ongoing Phase 3 ACTION-Galactosemia Kids study assesses AT-007's impact on clinical outcomes, with the first assessment due in Q1 2022. Despite the disappointment, CEO Shoshana Shendelman expressed commitment to work with the FDA on the approval process.
Applied Therapeutics has launched a registrational phase 2/3 study of AT-007 targeting Sorbitol Dehydrogenase (SORD) Deficiency, a hereditary neuropathy affecting around 3,300 patients in the U.S. and 4,000 in Europe. The INSPIRE trial aims to evaluate the safety and efficacy of AT-007 compared to placebo, focusing on sorbitol reduction and clinical outcomes. Initial data from a pilot study showed a 66% reduction in blood sorbitol levels. This milestone could offer new hope for patients suffering from this debilitating condition.
Applied Therapeutics (Nasdaq: APLT) announced its participation in the 14th International Congress of Inborn Errors of Metabolism (ICIEM) from November 21-23, 2021, in Sydney, Australia. The company's CEO, Shoshana Shendelman, highlighted critical data correlating toxic galactitol levels with disease severity in Galactosemia. Key presentations include studies on the impact of Galactosemia on children and adults, and an upcoming symposium on targeting toxic galactitol for treatment. The company develops therapies for rare metabolic diseases and has notable candidates like AT-007 for Galactosemia.
Applied Therapeutics, Inc. (Nasdaq: APLT) reported financial results for Q3 2021, highlighting significant advancements in its AT-007 drug for SORD deficiency and Galactosemia. In a pilot trial, AT-007 reduced blood sorbitol levels by approximately 66%. The ACTION-Galactosemia Kids study showed a statistical 40% reduction in plasma galactitol. The company ended Q3 with $108.8 million in cash. However, the net loss for the quarter was $28.4 million, or $1.09 per share, a slight improvement from the $29.8 million loss a year prior.
Applied Therapeutics (Nasdaq: APLT) announced promising results from a pilot trial of AT-007 for SORD Deficiency, a rare neurological disorder. AT-007 decreased blood sorbitol levels by approximately 66% in patients, with a range of 54%-75% reduction. The drug was safe and well tolerated. The company plans to initiate a registrational trial by the end of 2021. With around 3,300 patients in the US affected by this condition, AT-007 could be a first disease-modifying therapy. The company is also enhancing patient screening and diagnosis.
The press release from Applied Therapeutics (Nasdaq: APLT) highlights significant progress in the ACTION-Galactosemia Kids study. The drug candidate AT-007 demonstrated a 40% reduction in plasma galactitol levels, statistically significant (p<0.001) compared to placebo, indicating potential to improve the quality of life for Galactosemia patients. Furthermore, higher baseline galactitol levels correlated with greater disease severity. The company plans to submit a New Drug Application to the FDA in Q4 2021 and is preparing for a potential product launch.
Applied Therapeutics (Nasdaq: APLT) announced significant progress in its pediatric ACTION-Galactosemia Kids study, showing a 35% reduction in plasma galactitol after 30 days of treatment with AT-007, meeting statistical significance (p<0.001) compared to placebo. The drug was well-tolerated among children aged 2-17. The company is adjusting dosing based on weight and plans to submit a New Drug Application to the FDA in Q4 2021. A baseline analysis revealed a correlation between plasma galactitol levels and disease severity in patients, marking a milestone in understanding Galactosemia.
Applied Therapeutics, Inc. (APLT) reported its Q2 2021 financial results, ending the quarter with $125.6 million in cash and equivalents. The company is preparing for a New Drug Application (NDA) submission for AT-007 in Galactosemia, expected in Q3 2021, and plans to launch commercially in 2022. R&D expenses decreased to $14.8 million, while G&A expenses rose to $11.1 million. The net loss for the quarter was $25.8 million, or $0.99 per share, an improvement from a $28.1 million loss in the same quarter last year.
Applied Therapeutics (Nasdaq: APLT) has officially joined the Russell Microcap® Index, effective June 25, 2021. This inclusion, lasting a year, provides automatic membership in related growth and value style indexes, which are crucial for institutional investors. The Russell indexes are benchmarked by approximately $10.6 trillion in assets, underscoring their market significance. Applied Therapeutics, focused on novel drug development for unmet medical needs, is progressing with its lead candidate, AT-007, among others.
Applied Therapeutics announced data confirming the high prevalence of Diabetic Cardiomyopathy (DbCM) among adults with diabetes or pre-diabetes, with studies showing about 17% prevalence in affected populations. The findings, presented at the 81st American Diabetes Association Scientific Sessions, highlight the urgent need for awareness and screening. The company is actively enrolling participants in its global ARISE-HF study for the investigational drug AT-001, targeting DbCM and its progression to overt heart failure.