FDA Places Partial Clinical Hold On Applied Therapeutics Galactosemia Study

Applied Therapeutics (APLT) received a letter from the Food and Drug Administration that placed a partial clinical hold on the ACTION-Kids Study and requested that the company provide the FDA additional technical information relating to ensuring that every patient in the study has access to the prospect of direct benefit of the drug.

The partial clinical hold does not relate to concerns regarding the safety profile of AT-007, the company said in a regulatory filing. "The study is currently in the dose range finding segment, and the partial clinical hold does not introduce any disruption to ongoing treatment," it added. The adult ACTION-Galactosemia study, which is in the long-term extension phase, is not affected by the partial clinical hold. On June, 15, Applied Therapeutics announced the initiation of the ACTION-Galactosemia Kids pediatric registrational study of AT-007 for treatment of galactosemia to evaluate safety, pharmacokinetics, and reduction in the toxic biomarker, galactitol. The company said it will "work closely with FDA to resume the study as soon as possible and plans to submit the supporting technical information requested by the FDA promptly." The FDA has 30 days thereafter to review the information and to notify the company if the ACTION-Kids Study may be resumed.

Shares of Applied Therapeutics are down 20.5% to $23.24 following the news. Based on this anticipated pediatric study delay, the Company plans to submit a new drug application in the first quarter of 2021.