Drug/Device Pipeline

Celgene Halts Mongersen Trials in Crohn’s Disease

Celgene announced it has discontinued two trials of drug candidate GED-0301 (mongersen) in Crohn’s disease due to disappointing results.

The company said it decided to stop the phase 3 REVOLVE trial (CD-002) and the extension trial (SUSTAIN, CD-004) based on the Data Monitoring Committee’s recommendation in October following an interim analysis of observed risks and benefits. A press release noted this analysis showed “no meaningful safety imbalances.”

As a result, the company said it will not begin the phase 3 DEFINE trial (CD-003) in Crohn’s disease, and will decide if it will continue developing mongersen for ulcerative colitis after reviewing full data from a phase 2 trial.

This oral antisense therapy, which is investigational and not approved for use in any country, “is an oligonucleotide that decreases Smad7 protein, thereby potentially impacting [TGF-beta 1] signaling,” according to the press release. “In patients with Crohn’s disease, abnormally high levels of Smad7 interfere with [TGF-beta 1] anti-inflammatory pathways in the gut, leading to increased inflammation.”

The company noted that it remains committed to developing novel drugs for inflammatory bowel disease, including ozanimod (RPC1063), for which 2-year results from the phase 2 TOUCHSTONE trial in ulcerative colitis and phase 2 data from the STEPSTONE trial in Crohn’s disease were recently shared at the World Congress of Gastroenterology at ACG 2017. Additionally, the phase 3 TRUENORTH pivotal trial in ulcerative colitis is ongoing, and the company plans to begin a phase 3 pivotal trial in Crohn’s disease in the next few months.

A phase 2 randomized controlled trial (UC-001) of Otezla (apremilast) in ulcerative colitis is also ongoing, with results expected by the end of the year, which the company hopes will support a phase 3 program in 2018, according to the press release.

 

Celgene announced it has discontinued two trials of drug candidate GED-0301 (mongersen) in Crohn’s disease due to disappointing results.

The company said it decided to stop the phase 3 REVOLVE trial (CD-002) and the extension trial (SUSTAIN, CD-004) based on the Data Monitoring Committee’s recommendation in October following an interim analysis of observed risks and benefits. A press release noted this analysis showed “no meaningful safety imbalances.”

As a result, the company said it will not begin the phase 3 DEFINE trial (CD-003) in Crohn’s disease, and will decide if it will continue developing mongersen for ulcerative colitis after reviewing full data from a phase 2 trial.

This oral antisense therapy, which is investigational and not approved for use in any country, “is an oligonucleotide that decreases Smad7 protein, thereby potentially impacting [TGF-beta 1] signaling,” according to the press release. “In patients with Crohn’s disease, abnormally high levels of Smad7 interfere with [TGF-beta 1] anti-inflammatory pathways in the gut, leading to increased inflammation.”

The company noted that it remains committed to developing novel drugs for inflammatory bowel disease, including ozanimod (RPC1063), for which 2-year results from the phase 2 TOUCHSTONE trial in ulcerative colitis and phase 2 data from the STEPSTONE trial in Crohn’s disease were recently shared at the World Congress of Gastroenterology at ACG 2017. Additionally, the phase 3 TRUENORTH pivotal trial in ulcerative colitis is ongoing, and the company plans to begin a phase 3 pivotal trial in Crohn’s disease in the next few months.

A phase 2 randomized controlled trial (UC-001) of Otezla (apremilast) in ulcerative colitis is also ongoing, with results expected by the end of the year, which the company hopes will support a phase 3 program in 2018, according to the press release.