Applied TherapeuticsNASDAQ: APLT

Applied Therapeutics is a clinical-stage biotech focused on treating symptoms of galactosemia in the central nervous system. Govorestat (AT-007) is in late-stage regulatory approval for galactosemia, with a PDUFA date set for November 2024. APLT holds $146.5 million in cash, ensuring a sufficient runway to FDA approval and initial product revenues by early 2025.

The U.S. Food and Drug Administration has extended its review of Applied Therapeutics' experimental drug to treat galactosemia, a rare genetic metabolic disease, the company said on Thursday.

Applied Therapeutics (APLT) surges 124% in a month due to encouraging updates from two separate developmental programs for govorestat to treat different rare neurological indications.

Applied Therapeutics (NASDAQ: APLT ) stock is on the rise Wednesday after the clinical-stage biopharmaceutical company got an update from the Food and Drug Administration (FDA). The FDA has accepted the company's New Drug Application (NDA) for govorestat as a treatment for Classic Galactosemia.

Positive 12-month interim results from the phase 3 INSPIRE study using govorestat for the treatment of patients with SORD deficiency achieved. Pre-NDA meeting with FDA to be sought out after to see if an NDA submission of govorestat for SORD deficiency could be done with only the positive 12-month interim data. 24-month interim results from the phase 3 INSPIRE study, using govorestat for the treatment of patients with SORD deficiency, is the next data readout to be expected.

Applied Therapeutics shares plunged almost 35% after the biotechnology firm reported disappointing results from its Phase 3 heart disease trial. The company reported that its ARISE-HF trial of drug candidate AT-001 (caficrestat) in patients with diabetic cardiomyopathy (DbCM) at high risk of progressing to overt heart failure did not meet its primary endpoint of stabilization or improvement in cardiac functional capacity as measured by peak VO2.

Applied Therapeutics (NASDAQ: APLT ) stock is falling on Friday following the release of Phase 3 clinical trial data from the clinical-stage biopharmaceutical company. This data comes from Applied Therapeutics' ARISE-HF Phase 3 trial of AT-001 in patients with Diabetic Cardiomyopathy who have a high risk of overt heart failure.

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Applied Therapeutics is a micro-cap with low cash balance, but has major upcoming catalysts (pre-NDA meeting for galactosemia and phase 3 results in diabetic cardiomyopathy) and a huge upside potential. Despite missing the primary endpoint in galactosemia ph3 trial, APLT still plans for NDA submission. The pre-NDA meeting with the FDA this summer will be a big catalyst if positive. I expect a positive outcome from the meeting considering consistent and meaningful clinical benefit in multiple endpoints, orphan and fast-track designation, no alternative treatments, and the significant burden for affected patients.