Eton Pharmaceuticals has launched ET-900, a program evaluating mecasermin injection for the treatment of infantile and epileptic spasms. Mecasermin is a recombinant form of insulin-like growth factor 1 and the active ingredient in INCRELEX, which Eton markets in the United States. The company has licensed patent rights from Baylor College of Medicine covering the use of IGF-1 for the treatment of spasms.
Research led by Dr. John W. Swann at Baylor, funded by CURE Epilepsy, identified impaired IGF-1 signaling as a potential contributor to epileptic spasms. In an animal model, recombinant human IGF-1 reduced daily spasm counts by about 50% and eliminated spasms in 20% of treated animals. Dr. Swann said studies of patients with structural brain abnormalities show reduced IGF-1 levels in their brains and that mecasermin may serve as replacement therapy.
A research IND has been submitted for an investigator-sponsored Phase 2 study sponsored by the University of Chicago. The trial will enroll up to 20 patients ages 2-18 across three sites: the University of Chicago, Baylor College of Medicine and UCLA. Patients must have spasms that have not responded to or not been tolerated with hormonal therapy and vigabatrin. Participants will receive twice-daily subcutaneous injections over three to five weeks. The study will assess spasm resolution, changes in daily spasm frequency, and EEG abnormalities. Initial dosing is targeted for the first quarter of 2027.
Mecasermin is not FDA-approved for infantile or epileptic spasms, and its safety and efficacy for these uses have not been established. Eton retains commercial rights to ET-900.
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