Applied Therapeutics Announces Start of AT-007 Pediatric Galactosemia Study; Releases Additional 40mg/kg Data from Adult Galactosemia Study
AT-007 pediatric Galactosemia study (ACTION-Galactosemia Kids) initiated
Additional 40mg/kg adult cohort demonstrates incremental efficacy; data reinforces AT-007 favorable safety and efficacy profile
“From a scientific perspective, it was important to investigate effects of AT-007 at the higher dose, 40mg/kg, to ensure that we are fully informed on the optimal dose for patients,” said
“The initiation of our pediatric study is an important milestone for the Galactosemia community,” said
ACTION-Galactosemia Adult Trial: 40 mg/kg Dose Results
ACTION-Galactosemia is a Phase 1/2 clinical trial of the CNS penetrant Aldose Reductase inhibitor AT-007 in healthy volunteers and adults with Galactosemia. The biomarker-based pivotal study targeted reduction in plasma galactitol, an aberrant toxic metabolite of galactose formed by Aldose Reductase in Galactosemia patients. Accumulation of galactitol causes long-term complications ranging from CNS dysfunction to cataracts. Previously,
90-Day Safety Extension
The 90-day extension study of AT-007 in adult Galactosemia patients remains ongoing. Patients who completed the 40mg/kg cohort have rolled into the 3-month safety extension study. The extension study is open to patients from the core study and to new adult Galactosemia patients. To date, the drug has been shown to be safe and well tolerated, and all patients enrolled in the study remain on treatment.
ACTION-Galactosemia Kids: Pediatric Study Initiated
A placebo-controlled trial of AT-007 in pediatric patients (ACTION-Galactosemia Kids) will begin enrolling this week. Similar to the ACTION-Galactosemia trial in adults, the pediatric study will evaluate safety, pharmacokinetics, and reduction in the toxic biomarker, galactitol. Three age cohorts are being studied in parallel: age 2-6, age 7-12, and age 13-17. An additional cohort of children under 2 years of age may be added following analysis of safety data from the initial pediatric cohorts. The core study is comprised of two parts: a dose range finding segment evaluating up to 7 days of consecutive dosing to determine the optimal dose in children of each age group, followed by a 90 day study evaluating safety and biomarker efficacy. Following completion of the core study, all participants will have the option to participate in a long-term open-label extension (where placebo patients will cross over to active drug). Both the ACTION-Galactosemia adult extension study and the pediatric study are designed to incorporate primarily home health visits in order to limit travel and risk of exposure to COVID-19.
The full ACTION-Galactosemia study data will be available on the Presentations & Publications section of the Applied Therapeutics website at www.appliedtherapeutics.com.
About Applied Therapeutics, Inc.
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. Applied Therapeutics is conducting a biomarker based development program in patients with Galactosemia, based on the recently released industry guidance on drug development for low prevalence, slowly progressing rare metabolic diseases. The company received Orphan Designation for AT-007 for Galactosemia in May 2019 and Pediatric Rare Disease Voucher (PRV) designation in 2020.
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Source: Applied Therapeutics