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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 studied for the treatment of hereditary neuropathy caused by SORD deficiency and classic galactosemia. It is being developed by Applied Therapeutics, Inc. (NASDAQ: APLT) and is currently in clinical development, with Phase 2 trials completed for both indications.
AT-007 is a small molecule designed to inhibit aldose reductase, an enzyme involved in the polyol pathway. By targeting this enzyme, AT-007 aims to reduce the accumulation of sorbitol, which is implicated in the pathology of SORD deficiency and classic galactosemia. This mechanism is being evaluated in clinical trials.
AT-007 is being developed by Applied Therapeutics, Inc., a biopharmaceutical company traded on NASDAQ under the ticker symbol APLT. The company is conducting clinical trials to evaluate the safety and efficacy of AT-007 for hereditary neuropathy caused by SORD deficiency and classic galactosemia.
AT-007 is in Phase 2 clinical development. Two Phase 2 trials have been completed: one in pediatric subjects with classic galactosemia (NCT04902781) and one in patients with SORD deficiency (NCT05397665). A Phase 1 trial in healthy subjects and adults with classic galactosemia (NCT04117711) has also been completed.
AT-007 has been studied in three completed clinical trials. NCT04117711 was a Phase 1 safety and pharmacokinetics study in healthy subjects and adults with classic galactosemia. NCT04902781 was a Phase 2 study in pediatric subjects with classic galactosemia. NCT05397665 was a Phase 2 study in patients with SORD deficiency.
AT-007 is also known as govorestat. It is an investigational aldose reductase inhibitor being developed by Applied Therapeutics for the treatment of SORD deficiency and classic galactosemia. Both names refer to the same drug candidate.