Treatment for Severe Crohn's Disease Achieves Clinical Remission

Investigational drug will move to phase 3 studies.

An experimental treatment for moderate-to-severe Crohn’s disease showed promise during a recent phase 2 clinical trial.

The results of a double-blind, placebo-controlled, randomized, multicenter trial of Celgene’s GED-0301 (mongersen) in patients with active Crohn's disease was presented at Digestive Disease Week (DDW) in Washington, DC.

"For patients with Crohn's, disease severity and duration can influence the therapeutic effect of certain medicines," said Professor Giovanni Monteleone, University of Rome Tor Vergata, in a press release. "This subgroup analysis of data from the phase 2 study explored the effects of these factors on clinical response and clinical remission rates with GED-0301 — being investigated as an orally administered antisense therapy with a novel mechanism of action designed to act locally."

The trial enrolled 166 patients with active Crohn's disease who were treated for 2 weeks with either placebo or 1 of 3 once daily doses of GED-0301 in 10 mg, 40 mg, or 160 mg tablets. The patients were subsequently followed for an additional 10 weeks.

Clinical remission rates for patients who received GED-0301 160 mg were similar regardless of disease duration or baseline CDAI or CRP levels. Remission rates ranged from 62.5% to 75% for GED-0301 160 mg, compared with 5% to 24% for placebo.

In patients with a disease duration of at least 5 years, 62.5% treated with GED-0301 160 mg achieved clinical remission at week 2, compared with 15.4% of patients treated with placebo. Similar results were found at week 4 with 66.7% versus 15.4%, respectively.

The clinical response rate was 70.8% with GED-0301 160 mg, compared with 19.2% with placebo at week 2. At week 4, the response rate in the GED-0301 160 mg group was 79.2% versus 26.9% in the placebo group.

The rates of patients with at least 1 adverse event were 49%, 62%, and 49% in the GED-0301 10 mg, 40 mg, and 160 mg doses, respectively, compared with 67% for placebo. The most common adverse events in the GED-0301 treatment groups were abdominal pain, worsening of Crohn's disease, urinary tract infection, and CRP increase.

"The analysis presented at DDW suggests that patients with more severe Crohn's disease or a longer duration of disease were able to achieve clinical response or clinical remission with the 160 mg dose of GED-0301," said Scott Smith, President of Celgene Inflammation and Immunology, in a press release. "Patients with moderate to severe Crohn's disease are in need of new treatment options. Based on these findings, and as part of our commitment to bringing innovative medicines to this patient community, we look forward to continued study of this potentially transformative therapy in phase III trials."