The FDA Data Monitoring Committee recommended Celgene discontinue the mongersen trials.
A phase 3 trial for an investigational Crohn’s disease (CD) therapy has been discontinued following a US Food and Drug Administration (FDA) Data Monitoring Committee recommendation.
The FDA committee recommended a stop be made to trials testing Celgene’s REVOLVE trial and extension trial (SUSTAIN) for GED-0301 (mongersen), an oral compound currently not approved for any uses.
The committee made the recommendation this month, upon overall benefit/risk consideration in a recent interim futility analysis. There were no clinically meaningful safety imbalances identified in the analysis, according to Celgene.
Mongersen, an Smad7 protein-decreasing oligonucleotide, was considered for CD due to its impact on TGF beta-1 signaling. The Smad7 protein interferences with the signaling leads to increased inflammation in CD patients.
At the time of the decision, the phase 3 DEFINE trial had not been initiated, and Celgene is awaiting review from phase 2 trial datasets for mongersen’s treatment of ulcerative colitis (UC). Next steps regarding the drug will be made when the data is reviewed.
Scott Smith, president and chief operating officer for Celgene, thanked the patients and investigators of the REVOLVE trial.
“Crohn's disease is a debilitating condition with few effective long-term treatment options,” Smith said. “While we are disappointed with the results of REVOLVE, we remain committed to advancing our portfolio of novel medicines for patients suffering from this disease and other inflammatory bowel disorders.”
A press release regarding the decision was made available.