Celgene reports positive data from Phase Ib study of GED-0301 for Crohn’s disease

16th October 2016 (Last Updated October 16th, 2016 18:30)

US-based Celgene's Swiss subsidiary Celgene International has reported positive data from its exploratory Phase Ib CD-001 study of GED-0301 (mongersen) to treat active Crohn’s disease.

US-based Celgene's Swiss subsidiary Celgene International has reported positive data from its exploratory Phase Ib CD-001 study of GED-0301 (mongersen) to treat active Crohn’s disease.

GED-0301 is an orally administered anti-sense therapy and an oligonucleotide designed to target the messenger RNA (mRNA) for Smad7, resulting in the reduction of Smad7 protein levels.

Crohn’s disease is characterised by abnormally high levels of Smad7 that interfere with TGF-β1 anti-inflammatory pathways in the gut, causing an increased inflammation.

The Phase Ib CD-001 study has been conducted as a randomised, double-blind, multi-centre, exploratory study to assess the effects of GED-0301 on endoscopic and clinical outcomes in patients with active Crohn’s disease.

The study involved 63 biologic-exposed and biologic-naïve patients, as well as patients diagnosed with Ileitis, Ileocolitis or colitis.

The patients were randomised in a 1:1:1 ratio to be administered with GED-0301 and placebo in one of three active treatment regimens in a 12-week treatment phase.

This treatment phase was followed by an off-treatment observation phase conducted for 52 weeks.

Eligible patients can also enter an extension phase (on treatment) for an additional 100 weeks.

"We are encouraged that oral GED-0301 showed both meaningful endoscopic improvement and clinical remission at an early time point in this study."

Data suggested an endoscopic improvement across all the treatment groups with highest clinical response and remission rates notes in the 12-week treatment group.

The study has also exhibited a consistent safety and tolerability profile when compared with the previous studies.

Celgene inflammation and immunology president Scott Smith said: “We are encouraged that oral GED-0301 showed both meaningful endoscopic improvement and clinical remission at an early time point in this study.

"The fact that this study included nearly 50% biologic-experienced patients further reflects the potential of GED-0301 as a novel approach for patients with Crohn’s disease searching for alternatives."


Image: CT scan displaying Crohn's disease in the fundus of the stomach. Photo: courtesy of Samir.