Applied Therapeutics Announces Initiation of Phase 1/2 Study of AT-007 in Galactosemia
The multicenter study will primarily enroll patients with Classic Galactosemia, the most common form of Galactosemia in the US, but will also seek to enroll a single cohort of GALK deficient (GALKD) patients, which is more common in Europe.
“Dietary restriction alone does not prevent long term complications of disease because the body makes galactose endogenously,” said
Aldose Reductase (AR) is responsible for formation of a toxic metabolite of galactose, called galactitol, which has been shown to be responsible for many of the long-term complications in Galactosemia. Patients will be treated initially with a single dose of AT-007, followed by 27 days of consecutive dosing. The study endpoints will be safety, tolerability, PK and biomarker-based analyses. The trial will determine the ability of AT-007 to suppress toxic accumulation of galactitol in patients and will explore feasibility of measuring galactitol levels in the brain via MRI.
“The initiation of the first therapeutic clinical trial in Galactosemia marks an important milestone for the Galactosemia community,” 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 Phase 1/2 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.
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If you are a patient or family member interested in receiving information regarding participation in the Phase 1/2 clinical trial, please email: firstname.lastname@example.org.
Source: Applied Therapeutics