Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as PTC743, EPI-743
Vatiquinone · 8 trials · 4 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.
NPMDS is a validated scale to assess the mitochondrial disease progression.
Low contrast visual acuity
Change from baseline to six months will be compared between subjects in active treatment group and placebo group
| 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. |
| EPI-743 | EXPERIMENTAL | Participants will receive EPI-743 at a dose of 15 milligrams/kilogram (mg/kg) up to a total 200 mg 3 times daily (TID) orally with meal or by using nasogastric (NG) or gastrostomy feeding tubes with liquid food for at least 36 months. |
| EPI-743 400 mg | ACTIVE_COMPARATOR | EPI-743 at a dose of 400 mg three times daily |
| EPI-743 200 mg | ACTIVE_COMPARATOR | EPI-743 at a dose of 200 mg three times daily |
| EPI-743 15 mg/kg | ACTIVE_COMPARATOR | Each subjects dose will be based on their weight. 15 mg/kg with a maximum dose of 200 mg per dose, t.i.d., will be administered in this treatment arm. |
| EPI-743 5 mg/kg | ACTIVE_COMPARATOR | Each subjects dose will be based on their weight. 5 mg/kg with a maximum dose of 100 mg per dose, t.i.d., will be administered in this treatment arm. |
| 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. |
| EPI-743 | DRUG | EPI-743 100 milligrams/milliliter (mg/mL) oral solution will be administered per dose and schedule specified in the arm. |
| EPI-743 400 mg | DRUG | - |
| EPI-743 200 mg | DRUG | - |
| EPI-743 15 mg/kg | DRUG | - |
| EPI-743 5 mg/kg | DRUG | - |
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 rare diseases, specifically Friedreich Ataxia and Inherited Mitochondrial Disease. It is a modulator of the NQO1 target. Vatiquinone is currently in Phase 3 clinical development and has not been approved by the FDA.
Vatiquinone is being studied as a treatment for Friedreich Ataxia, a rare inherited neurodegenerative disorder. It is also being evaluated in Inherited Mitochondrial Disease. The drug remains investigational and is not approved for any indication. Clinical trials are evaluating its safety and efficacy in these patient populations.
Vatiquinone targets NQO1, an enzyme also known as NAD(P)H quinone dehydrogenase 1. It acts as a modulator of this target. This mechanism is being investigated for its potential role in treating Friedreich Ataxia and Inherited Mitochondrial Disease, both rare conditions with significant unmet need.
Vatiquinone is being developed by PTC Therapeutics, Inc., which trades on the Nasdaq under the ticker PTCT. The company is advancing the drug through Phase 3 clinical trials for Friedreich Ataxia and Inherited Mitochondrial Disease. PTC Therapeutics is a biopharmaceutical company focused on rare diseases.
Vatiquinone is in Phase 3 clinical development. It has completed two trials and has two active trials, with a total enrollment of 401 participants across all studies. The drug is investigational and has not received FDA approval. It holds a Priority Review designation from the FDA.
Vatiquinone is being studied in several clinical trials. NCT07681713 is a Phase 3 long-term efficacy study in Friedreich's Ataxia that is not yet recruiting. NCT05515536 is an active Phase 3 safety and efficacy study in Friedreich Ataxia. NCT05485987 is a completed Phase 2 study, and NCT05218655 is a completed Phase 3 safety study in Inherited Mitochondrial Disease.