Applied Therapeutics’ Galactosemia Program Stumbles Again after FDA Requests for More Data

Applied Therapeutics is holding off a New Drug Application for AT-007, a treatment for galactosemia. The decision by the New York biopharma came after the FDA requested that additional clinical data was required for approval. 

Shares of Applied Therapeutics tanked by 27% when trading began on Monday. 

“While disappointed by this change in direction by the FDA, we remain committed to bringing this important treatment to patients with Galactosemia,” said Shoshana Shendelman, CEO of Applied Therapeutics. “We will continue to work with the FDA to determine the most expeditious path forward to regulatory approval and will provide an update on timing and plans accordingly.”

Applied Therapeutics’ galactosemia program has faced issues before. In July 2020, the FDA slapped a clinical hold on the study of AT-007 in children, citing concerns over how the trial was run. The hold was lifted last February, after Applied Therapeutics modified the study design. 

AT-007, Treatment for Galactosemia

Patients with galactosemia cannot efficiently break down the sugar galactose in their bloodstream. The sugar is instead metabolized into toxic galactitol, which builds up in the organs and tissues and causes long-term complications such as delayed development and cognitive impairment. 

There is no cure for galactosemia, and the disease is generally managed by avoiding lactose and galactose in the diet. 

Applied Therapeutics believes their galactosemia program has the chance to transform the lives of patients living with the disease. Their lead candidate AT-007 has received both Orphan Drug and Pediatric Rare Disease and Fast Track designations from the FDA for the treatment of galactosemia. 

AT-007 is an aldose reductase inhibitor that can cross the blood brain barrier. A recent data analysis from Applied Therapeutics’ Phase 3 program on AT-007 showed that the drug significantly reduced baseline galactitol levels by 40% compared to placebo. The reduction was rapid and long-lasting, while the drug was well tolerated by the 47 children taking part in the study. 

According to Applied Therapeutics, lower galactitol levels correlate with better clinical functional outcomes and less severe disease. 

While the program has hit a bump after the FDA decision, the company is still hopeful that their treatment will get approved. The ongoing Phase 3 program will continue to evaluate the effect of AT-007 treatment on clinical outcomes, including how patients feel and function. The results will be assessed every six months, with the first assessment expected to be completed in the first quarter of 2022. 

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Joy Lin

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