Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
EPI-589 · 1 trial · 1 indication
An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and non-serious AEs. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'.
| Arm | Type | Description |
|---|---|---|
| EPI-589 | EXPERIMENTAL | Participants will receive EPI-589 500 milligrams (mg) (2 tablets of 250 mg each) twice daily (BID) for 3 months, unless discontinued for safety or tolerability issues. |
| Name | Type | Description |
|---|---|---|
| EPI-589 | DRUG | An immediate release film-coated table at a 250 mg dosage strength will be administered per dose and schedule specified in arm. |
Inclusion Criteria: * Diagnosis of possible, probable, laboratory supported probable, or definite ALS by E1 Escorial Criteria * Forced vital capacity (FVC) ≥ 70% of predicted * Weakness onset within 3 years * Agreement to use contraception if within reproductive years * Willingness and ability to c...
EPI-589 is an investigational small molecule being studied for the treatment of Amyotrophic Lateral Sclerosis (ALS). It is currently in Phase 2 clinical development, though the single trial evaluating it has been completed. EPI-589 is not approved by the FDA and remains an experimental therapy.
EPI-589 is being developed by PTC Therapeutics, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol PTCT. The company is conducting clinical research to evaluate the safety and potential efficacy of EPI-589 in patients with Amyotrophic Lateral Sclerosis.
EPI-589 is in Phase 2 clinical development. The Phase 2 trial, identified as NCT02460679, has been completed. This was an uncontrolled, open-label study that enrolled 19 participants with Amyotrophic Lateral Sclerosis in the United States. The drug remains investigational and has not received FDA approval.
EPI-589 has been evaluated in one clinical trial, NCT02460679, titled "Safety and Biomarker Study of EPI-589 in Participants With Amyotrophic Lateral Sclerosis (ALS)." This Phase 2 study was completed and enrolled 19 adults aged 21 years and older in the United States. The trial was uncontrolled and not blinded.
No, EPI-589 is not FDA approved. It is an investigational drug currently in Phase 2 clinical development for Amyotrophic Lateral Sclerosis. The only clinical trial for EPI-589, NCT02460679, has been completed, but the drug has not yet received regulatory approval for any use.