Investigational Crohn's Disease Drug Shows Favorable Results in Clinical Trial

Specialty Pharmacy Times, November/December 2014, Volume 5, Issue 6

An experimental drug developed by Celgene exhibited strong efficacy and safety results during a recent phase 2 study.

Mongersen, an oral topically active Smad7 antisense oligonucleotide, demonstrated a statistically significant remission rate compared with placebo after 2 weeks of treatment.

The double-blind, placebo-controlled trial evaluated the efficacy of Mongersen as induction therapy in steroid-dependent or steroid-resistant patients with active Crohn’s disease (Crohn’s disease activity index [CDAI] score 220-400). Patients were randomized to Mongersen at doses of 10 mg, 40 mg, or 160 mg per day, or placebo, for 2 weeks.

A greater proportion of patients achieved statistically significant remission rates receiving Mongersen 40 mg per day (55%) and 160 mg per day (65.1%) compared with placebo (9.5%). There was not found to be a significant difference in clinical remission in the 10-mg per day group (12.2%) compared with the placebo group. The clinical response rate was also significantly greater among patients receiving Mongersen at doses of 10 mg per day (36.6%), 40 mg per day (57.5%), or 160 mg per day day (72.1%) compared with placebo (16.7%; P = .039, P = .0001 and P <.0001, respectively).

Adverse events and serious adverse events were similar across all treatment groups. There were 9 serious adverse events in 6 patients, mostly consisting of hospital admissions for complications or symptoms associated with Crohn’s disease.

The study did not report any toxicities previously found with systemically active antisense agents.