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EPI-743 · 3 trials · 2 indications
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 |
|---|---|---|
| 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. |
| Placebo | PLACEBO_COMPARATOR | Placebo capsules three times daily |
| 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 |
|---|---|---|
| EPI-743 | DRUG | EPI-743 100 milligrams/milliliter (mg/mL) oral solution will be administered per dose and schedule specified in the arm. |
| Placebo | DRUG | Placebo capsules |
| EPI-743 400 mg | DRUG | - |
| EPI-743 200 mg | DRUG | - |
| EPI-743 15 mg/kg | DRUG | - |
| EPI-743 5 mg/kg | DRUG | - |
Inclusion Criteria: 1. Diagnosis of Leigh syndrome with genetic confirmation 2. Completion of EPI743-12-002 protocol and initiation of treatment within 14 days of Month 12 visit in EPI743-12-002 study 3. Participant or participant's guardian able to consent and comply with protocol requirements 4. ...
EPI-743 is an investigational small molecule being developed by PTC Therapeutics for Friedreich's Ataxia and Leigh Syndrome, both rare diseases. It has been studied in Phase 2 clinical trials for these conditions.
EPI-743 is being developed by PTC Therapeutics, Inc., a biopharmaceutical company. PTC Therapeutics is publicly traded under the ticker symbol PTCT.
EPI-743 is in Phase 2 clinical development. It has completed two Phase 2 trials, one in Leigh Syndrome and one in Friedreich's Ataxia, as well as a long-term safety and efficacy study in Leigh Syndrome.
EPI-743 has been studied in three completed Phase 2 trials: NCT01721733 in children with Leigh Syndrome, NCT01728064 in patients with Friedreich's Ataxia, and NCT02352896, a long-term safety and efficacy study in children with Leigh Syndrome.
EPI-743 is the sole name provided for this investigational drug. No alternative names have been reported for this compound.