Applied Therapeutics, Inc. - APLT STOCK NEWS

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.

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.