Celgene reports positive early-stage data for Crohn's disease therapy mongersen

Celgene announced early-stage study data for the experimental drug mongersen in patients with active Crohn's disease, showing that the oral antisense therapy had a positive effect on both endoscopic response and clinical remission. Last month, the company said that top-line results from the CD-001 trial demonstrated endoscopic improvement and clinical response and remission at week 12.

The Phase Ib study randomised 63 patients with active Crohn's disease to receive mongersen, also known as GED-0301, dosed once daily for either 12, eight or four weeks. The company noted that the treatment phase was followed by an off-treatment observation phase for up to 52 weeks, with endoscopic and clinical assessments reported through week 12.

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Data, which are scheduled to be presented at the United European Gastroenterology Week (UEGW), showed that of the 52 patients with evaluable endoscopies at week 12, 37 percent had an endoscopic response, with no meaningful difference across treatment groups. RBC Capital Markets analyst Michael Yee suggested that experts are looking for a quarter or more of patients to see that improvement.

Celgene added that in patients who had a greater endoscopic disease activity at baseline, 63 percent exhibited a reduction of at least 25 percent in the simple endoscopic score for Crohn's disease and 31 percent had a reduction of at least 50 percent. Further, the company noted that clinical improvement was seen by week 2, and clinical response and remission rates were highest in the 12-week treatment group at 67 percent and 48 percent, respectively, at week 12.

Scott Smith, president of inflammation and immunology at Celgene, remarked "the fact that this study included nearly 50 percent biologic-experienced patients further reflects the potential of GED-0301 as a novel approach for patients with Crohn's disease searching for alternatives." Celgene licensed rights to mongersen from Nogra Pharma in 2014 under a deal potentially worth more than $2.5 billion, including an upfront payment of $710 million.

For related analysis, see KOL Views: Leading gastroenterologist opines on new mongersen results, sets the bar for what Celgene must clear at final readout.

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