$710M Gamble Fails as Celgene Terminates Two Phase III Trials
By Alex Keown
Celgene said it was pulling the plug on two trials studying GED-0301 (mongersen ) after an overall benefit/risk analysis was conducted. Celgene acquired GED-0301in 2014 from privately-held Nogra Pharma Limited. Celgene announced it was terminating the Phase III Resolve trial for Crohn’s disease, as well as the Sustain extension trial based on the recommendation of the data monitoring committee. Additionally, Celgene said it will not initiate the Phase III Define trial in Crohn’s disease. The company is waiting to review the full dataset from the phase II trial with GED-0301 in ulcerative colitis (UC) to determine if the company can continue forward in some way with the drug.
Investors reacted negatively to the news, sending share prices down more than 7 percent overnight. Stocks fell from $135.96 to $125.82.
“Crohn’s disease is a debilitating condition with few effective long-term treatment options. While we are disappointed with the results of Resolve, we remain committed to advancing our portfolio of novel medicines for patients suffering from this disease and other inflammatory bowel disorders,” said Scott Smith, Celgene’s president and chief operating officer.
GED-0301, also known as Mongersen, is an oligonucleotide designed to target the messenger RNA (mRNA) for Smad7, a protein that is a key regulator of TGF-beta (transforming growth factor) type 1 receptors. Celgene said there were no meaningful safety imbalances identified in the analysis.
Leerink analyst Geoffrey Porges said in a note that the GED-0301 mistake will likely cost Celgene about $2 billion in long-term revenue. He said the deal with Nogra was a “painful reminder” of the costs of taking “shots on goal,” Endpoints News reported.
With GED-0301 on the rocks, Celgene will focus its inflammatory bowel disease research onto Ozanimod. Earlier this week, Celgene provided a glimpse of Phase II data showing that the drug demonstrated efficacy in patients with moderately to severely active Crohn’s disease and in patients with moderately to severely active ulcerative colitis. The company said data from the Stepstone study support the potential of Ozanimod to provide continued efficacy in ulcerative colitis at 92 weeks. Data is also promising for Ozanimod in Crohn’s disease, Terrie Curran, head of Celgene’s Inflammation and Immunology programs said in a statement. Data from the Phase II studies will be presented at the World Congress of Gastroenterology meeting later this month.