GED-0301 is an antisense oligodeoxynucleotide with a sequence complementary to the Smad7 mRNA transcript.
Smad7 is a negative regulator of transforming growth factor- β, which is increased in the intestinal mucosa of patients with active Crohn’s disease.
Dr Brian Feagan and colleages from Western Univeristy, London, Ontario, Canada, randomly assigned 63 Crohn's Disease patients to 4-, 8-, or 12-week treatment groups receiving oral GED-0301 160 mg/day.
The teams primary objective was to determine GED-0301’s effect on endoscopic Crohn's Disease measures; secondary objectives included effects on clinical activity.
Endoscopic improvement was observed in 37% of participants with evaluable endoscopy results at week 12.
At week 12, 4 weeks, 8 weeks, and 12 weeks of patients receiving GED-0301 were in remission. Crohn's Disease activity index score <150; corresponding reductions from baseline in mean Crohn's Disease activity index scores were −124, −112, and −133 points.
No new safety signals were observed by the doctors.
Dr Feagan's team comments, "These findings support a GED-0301 benefit in active Crohn's Disease."