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AT-007 · 3 trials · 2 indications
The 10MWRT is a timed functional test used to measure walking or running speed over 10 meters by the study population
Patients with SORD Deficiency develop extremely high sorbitol levels in cells and tissues, as aldose reductase converts glucose to sorbitol which then cannot be converted into fructose by SORD. Sorbitol is known to be toxic to many cell types. The measurement of change in sorbitol will provide evidence of the efficacy of the treatment (AT-007) used in the study.
The primary endpoint is a GST of four components: 1) Behavioral Symptoms Index of the Behavioral Assessment Scale for Children 3 (BASC-3); 2) Activities of Daily Living from the BASC-3; 3) Oral Expression from the Oral and Written Language Skills-II (OWLS-II); 4) Listening Comprehension from the OWLS-II. Each individual component is also a secondary endpoint on their own with the test and scoring described below in the secondary endpoint section. For the GST, change from baseline using standardized z-scores is calculated for active versus placebo.
Sensitivity Analysis adding the National Institutes of Health Toolbox Cognition Battery (NIH-CB) test which measures cognition. NIH-CB is described below in the secondary endpoints. For the GST with cognition, change from baseline using standardized z-scores is calculated for active versus placebo.
Sensitivity Analysis adding the National Institutes of Health Toolbox Motor Battery (NIH-MB) 9-Hole Pegboard test which measures fine motor skills. NIH-MB is described below in the secondary endpoints. For the GST with fine motor skills, change from baseline using standardized z-scores is calculated for active versus placebo.
To evaluate the safety and tolerability of AT-007 after administration to healthy subjects, including clinically-significant changes in clinical laboratory test results, physical examination findings, vital sign evaluations, and electrocardiogram results.
| Arm | Type | Description |
|---|---|---|
| AT-007 | ACTIVE_COMPARATOR | AT-007 is an Aldose reductase inhibitor |
| Placebo | SHAM_COMPARATOR | Is an non-active control |
| Experimental: AT-007 | EXPERIMENTAL | AT-007 The starting dose in Part A will be 5 mg/kg for all age groups. For each age group, Part B of the study will not start until the optimum dose evaluated in Part A has been identified |
| Placebo Comparator | PLACEBO_COMPARATOR | Placebo is used as a comparator to the experimental arm. |
| Name | Type | Description |
|---|---|---|
| AT-007 | DRUG | AT-007, aldose reductase inhibitor |
| Placebo | DRUG | Liquid oral suspension |
Inclusion Criteria: 1. Willing and able to provide signed and dated informed consent prior to any study-related procedures and willing and able to comply with all study procedures. 2. Male and non-pregnant, non-lactating female patients between the ages of 18 and 55 years, inclusive. 3. Females mus...
AT-007 is an investigational small molecule being developed for Classic Galactosemia and for hereditary neuropathy caused by SORD deficiency. Both are rare metabolic conditions, and AT-007 is being studied to address the underlying enzyme deficiency in each. It is not yet approved for any indication.
AT-007 targets sorbitol dehydrogenase, an enzyme involved in sorbitol metabolism. By inhibiting this enzyme, AT-007 is designed to reduce the accumulation of sorbitol, which is thought to contribute to the pathology of both Classic Galactosemia and SORD deficiency neuropathy.
AT-007 is being developed by Applied Therapeutics, Inc., which trades on the Nasdaq under the ticker APLT. The company is the sponsor of the clinical trials evaluating AT-007 in Classic Galactosemia and SORD deficiency.
AT-007 is in Phase 2 clinical development. Two Phase 2 trials have been completed, one in Classic Galactosemia and one in SORD deficiency neuropathy. A Phase 1 trial in healthy subjects and adults with Classic Galactosemia has also been completed. AT-007 is investigational and has not been approved by the FDA.
AT-007 has been evaluated in three completed trials. NCT05397665 was a Phase 2 study in hereditary neuropathy caused by SORD deficiency with 56 patients across the United States, Czechia, Italy, and the United Kingdom. NCT04902781 was a Phase 2 study in pediatric Classic Galactosemia with 47 patients in the United States. NCT04117711 was a Phase 1 study with 114 healthy subjects and adults with Classic Galactosemia.