Shares of Applied Therapeutics Inc. (APLT), a clinical-stage biopharmaceutical company, are up nearly 40% at $6.50 in premarket trading Wednesday, following a regulatory update on the investigational drug Govorestat, proposed for the treatment of classic galactosemia.
Galactosemia is a rare genetic metabolic disease resulting in an inability to metabolize the simple sugar galactose. If galactose isn't metabolized properly, it transforms into a hazardous metabolite known as galactitol. This, in turn, leads to neurological complications, including deficiencies in speech, cognition, behavior, and motor skills, and also results in juvenile cataracts and ovarian insufficiency (in women).
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