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Vatiquinone · 5 trials · 3 indications
An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. AEs included both serious adverse events (SAEs) and non-serious AEs. A TEAE was defined as an AE that had an onset date or date of worsening on or after the first dose of study drug and within 30 days of the date of the last dose of treatment. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'.
mFARS is a 93-item scale; comprised of neurologic component of FARS. For each item, responses categorize the corresponding neurological finding, with a score ranging from 0 to 3, 4, or 5 with 0 being normal and higher numbers indicative of greater impairment. Total mFARS scores for each subscale: bulbar (0 to 5), upper limb coordination (0 to 36), lower limb coordination (0 to 16), and upright stability (0 to 36). mFARS total score was a composite score of all 4 subscales, ranging from 0 (normal) to 93 (greater impairment). A lower score = better neurological function. Missing data was imputed using pattern mix model multiple imputation. Least square (LS) mean and standard error (SE) was calculated using mixed-model repeated measures (MMRM).
mFARS is a 93-item scale; comprised of neurologic component of FARS. For each item, responses categorize the corresponding neurological finding, with a score ranging from 0 to 3, 4, or 5 with 0 being normal and higher numbers indicative of greater impairment. Total mFARS scores for each subscale: bulbar (0 to 5), upper limb coordination (0 to 36), lower limb coordination (0 to 16), and upright stability (0 to 36). mFARS total score was a composite score of all 4 subscales, ranging from 0 (normal) to 93 (greater impairment). A lower score = better neurological function. Missing data was imputed using pattern mix model multiple imputation. LS mean and SE was calculated using MMRM.
| Arm | Type | Description |
|---|---|---|
| Vatiquinone | EXPERIMENTAL | Participants will receive vatiquinone capsule at a dose of either 200 milligrams (mg) orally 3 times a day (TID) if weighing ˂25 kilograms (kg) or 400 mg orally TID if weighing ≥25 kg for 24 months. |
| Placebo | PLACEBO_COMPARATOR | Participants will receive placebo matching to vatiquinone (per age and weight) orally TID for 72 weeks during the placebo-controlled phase and vatiquinone at a dose of either 200 mg orally TID if ˂12 years of age and weighing ˂25 kg or 400 mg orally TID if ≥12 years of age and/or weighing ≥25 kg for 24 weeks during the open-label extension phase. |
| Name | Type | Description |
|---|---|---|
| Vatiquinone | DRUG | Vatiquinone will be administered per dose and schedule specified in the arm. |
| Placebo | DRUG | Placebo will be administered per schedule specified in the arm. |
Key Inclusion Criteria: * mFARS ≥20 to ≤70 at Screening (4 weeks prior to Day 1) and Baseline (Day 1). * Must be ambulatory as defined by a E7 score of 4 or less on the USS at Screening and Baseline. * Documentation that participants reached maximum score on items E4, E5, and E3b on the USS of mFAR...
Vatiquinone is an investigational small molecule being developed for Friedreich Ataxia and Inherited Mitochondrial Disease. It is currently in Phase 3 clinical development for these rare diseases.
Vatiquinone is a small molecule being studied for Friedreich Ataxia and Inherited Mitochondrial Disease. The specific molecular target has not been disclosed in available information.
Vatiquinone is being developed by PTC Therapeutics, Inc., a biopharmaceutical company. PTC Therapeutics is publicly traded under the ticker symbol PTCT.
Vatiquinone is in Phase 3 clinical development. It is an investigational drug and has not been approved by the FDA. It has received Priority Review designation from the FDA.
Vatiquinone has been studied in several clinical trials. NCT04577352 was a Phase 2 study in Friedreich Ataxia. NCT05218655 was a Phase 3 safety study in Inherited Mitochondrial Disease. NCT05515536 is an active Phase 3 study in Friedreich Ataxia. NCT07681713 is a planned Phase 3 long-term efficacy study.
Vatiquinone is also known as PTC743. A clinical trial for Vatiquinone (PTC743) in Inherited Mitochondrial Disease has been completed.