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Applied Therapeutics' metabolic disorder drug helps reduce symptoms; stocks soar

Applied Therapeutics Inc. said its medicine AT-007 was better than placebo in treating a rare metabolic disorder called galactosemia.

According to the results of a mid-stage study called Action-Galactosemia, the highest tested dose of AT-007 helped reduce plasma galactitol by between 45% to 54% in patients from the time treatment began. Those on placebo saw no substantial change in the toxic metabolite which is the cause of long-term complications related to growth, intellectual disability, liver disease and the kidneys.

Galactosemia, for which there are no known cures or therapies, affects the body's ability to process a sugar called galactose. High levels of galactose, which is derived from lactose in food, are then converted by the body into a toxic sugar alcohol called galactitol.

AT-007 was well tolerated and no drug-related adverse events were seen. The company said in a Jan. 8 press release that it will present detailed results of the study at a medical conference in April.

The New York-based biopharmaceutical company, which went public in 2019, saw its stock rise by 24.62% to $32.60 in pre-market trading on Jan. 8 on Nasdaq.

Applied Therapeutics plans to file for U.S. regulatory approval of the drug this year, with further plans to evaluate long term safety of AT-007 in adult patients and study its effects in children.

AT-007 holds the FDA's orphan-drug designation for treating galactosemia.