Applied Therap soars on mixed Phase III results with govorestat

25 April 2023

Shares of New York, USA-based rare disease firm Applied Therapeutics (Nasdaq: APLT) closed up more than 16% yesterday at $1.10, despite releasing disappointing results from the ACTION-Galactosemia Kids study of govorestat (AT-007), a novel, oral, small molecule, central nervous system (CNS) penetrant aldose reductase inhibitor (ARI).

The ACTION-Galactosemia Kids Phase III study was designed to evaluate the impact of govorestat treatment versus placebo on clinical outcomes over time in 47 children age 2-17 with galactosemia. Treatment with govorestat demonstrated consistent and sustained clinical benefit on activities of daily living, behavioral symptoms, cognition, adaptive behavior and tremor, the company stated.

Missed primary endpoint

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