Approval Probability
TA Base Rate
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ML Risk
Also known as Vatiquinone, PTC743
EPI-743 · 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...
EPI-743 is an investigational small molecule being studied for the treatment of rare mitochondrial and neurodegenerative disorders, specifically Friedreich's Ataxia and Leigh Syndrome. It is a Phase 2 clinical-stage candidate and is not approved for any indication.
EPI-743 is being developed by PTC Therapeutics, Inc., which trades on the Nasdaq under the ticker symbol PTCT. The company is the sponsor of the clinical development program for this investigational small molecule in rare disease indications.
EPI-743 is in Phase 2 clinical development. It is an investigational therapy and has not been approved by the FDA or any other regulatory agency. All completed trials to date have been Phase 2 studies.
EPI-743 has been evaluated in completed Phase 2 trials including NCT02352896, a long-term safety and efficacy study in children with Leigh Syndrome; NCT01728064, a safety and efficacy study in patients with Friedreich's Ataxia; and NCT01721733, a safety and efficacy study in children with Leigh Syndrome. All three trials are completed.
Across the completed Phase 2 clinical trials of EPI-743, a total of 65 patients have been enrolled. The studies were randomized, double-blind, and placebo-controlled, evaluating the drug in Friedreich's Ataxia and Leigh Syndrome patient populations.