Part 1 (dose escalation) will evaluate the safety and tolerability of 2 doses (100 milligrams/kilogram \[mg/kg\] and 200 mg/kg) of eteplirsen in approximately 10 participants with DMD; Part 2 (dose finding and dose comparison) will evaluate the efficacy and safety of the high doses (100 mg/kg and 200 mg/kg) of eteplirsen compared with that of the 30 mg/kg dose of eteplirsen, in approximately 144 participants with genetically confirmed deletion mutations amenable to treatment by skipping exon 51.
Phase 3 trials are pivotal studies with large patient populations (hundreds to thousands) designed to prove efficacy and safety for FDA submission. A successful Phase 3 readout is typically the last major hurdle before a New Drug Application (NDA) or BLA filing.
Enrollment is complete. The trial is in its follow-up or data collection phase — a readout may occur on or near the primary completion date.
Sarepta Therapeutics (SRPT) is the sponsoring company for this trial. BiotechSign currently grades this company C (54/100) based on composite catalyst signals across its full pipeline. This trial is one data point in that overall catalyst picture.