Applied Therapeutics Announces Positive Topline Results of Pivotal Phase 2 ACTION-Galactosemia Study of AT-007 in Galactosemia Patients
AT-007 demonstrated a robust and sustained reduction in galactitol vs placebo; significant plasma galactitol reduction of ~50% (p<0.01)
AT-007 was well-tolerated; no drug-related adverse events reported at any dose to date
Company to host conference call and webcast today at
AT-007 treatment resulted in a statistically significant and robust reduction in plasma galactitol vs placebo in adult Galactosemia patients. Reductions in galactitol were dose dependent, with higher concentrations of AT-007 resulting in a greater magnitude of reduction in galactitol. At the highest dose tested (20mg/kg), AT-007 significantly reduced plasma galactitol 45-54% from baseline vs. placebo (p<0.01). Galactitol reduction was rapid and sustained over time. No substantial change from baseline was observed in placebo treated patients. AT-007 was well tolerated, with no drug-related adverse events noted to date in Galactosemia patients or in the 72 healthy volunteers treated in Part 1 of the trial.
The company will present full data from the ACTION-Galactosemia trial at the
Based on these results reported today,
“We are thrilled with these results,” said
“The Galactosemia program is an example of our overall strategy to apply technological breakthroughs to areas of high unmet need,” said
Galactosemia is a rare metabolic disease that affects how the body processes a simple sugar called galactose, and for which there is no known cure or approved treatment available. Galactose is found in foods, but the human body also naturally produces galactose on its own, so dietary restriction can’t prevent complications of disease. It is estimated that the U.S. Galactosemia population is approximately 2,800 patients, based on newborn screening data identifying 2,500 infants through 2014, and the estimated birth rate of 80 patients per year. High levels of galactose circulating in the blood and tissues of Galactosemia patients enables Aldose Reductase to convert galactose to a toxic metabolite, called galactitol, which causes long-term complications ranging from CNS dysfunction to cataracts.
AT-007 is a central nervous system (CNS) penetrant Aldose Reductase inhibitor (ARI) in clinical development for treatment of Galactosemia. AT-007 has been studied in in an animal model of Galactosemia, which demonstrated that AT-007 reduces toxic galactitol levels and prevents disease complications.
Conference Call at
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Source: Applied Therapeutics