Applied Therapeutics has reported positive results from a Phase II portion of the ACTION-Galactosemia study of AT-007 in adult Galactosemia patients.

Galactosemia is a metabolic disorder that disturbs how a human body processes a simple sugar called galactose. There is currently no approved drug to treat the disorder.

The ACTION-Galactosemia study involved a double-blind placebo-controlled trial to test the safety and pharmacokinetics of AT-007.

It found that the AT-007 treatment led to a significant reduction in galactitol when compared to placebo. Galactitol is an aberrant toxic metabolite of galactose, formed by aldose reductase in Galactosemia patients.

The ACTION-Galactosemia study further found that higher doses of AT-007 resulted in a greater reduction in galactitol. The highest dose of AT-007 tested was 20mg/kg, which lowered plasma galactitol by 45% to 54% from baseline versus placebo.

Study subjects (patients and healthy volunteers) did not exhibit any drug-related adverse events in the first part of the trial.

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By GlobalData

Applied Therapeutics chief medical officer Riccardo Perfetti said: “Galactosemia is a devastating disease with no treatments currently available. We have long known that dietary restriction alone does not prevent chronic complications of disease.

“These results provide hope for patients and families that action through drug treatment with AT-007 can potentially change the course of the disease, transforming patients’ lives.”

Driven by these results, the company now intends to file for regulatory approval of AT-007 in the second half of this year.

AT-007 is a central nervous system (CNS) penetrant aldose reductase inhibitor (ARI). It received orphan designation for Galactosemia in May last year.