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left some questions unanswered. Celgene said of the 52 patients whose endoscopies could be evaluated, 37 percent met the 25 percent endoscopic improvement threshold, with no meaningful differences across the three tested groups. The company didn’t specify how the drug performed in each of these groups, however, or what the total remission rates were. Celgene only said that 48 percent of patients on the drug for 12 weeks were in remission. More data will be presented in Vienna this week.
Celgene measured the drug’s effects both via scores on the Crohn’s Disease Activity index (CDAI, a survey used to quantify patients’ Crohn’s symptoms) and the Simple endoscopic score for Crohn’s disease, or SES-CD, in which investigators use endoscopy images to gauge patients’ disease. Patients in the trial started with CDAI scores of between 220 and 450, and those on mongersen for 12 weeks saw those scores drop by an average of 133 points. Of those patients, 67 percent saw their CDAI scores improve by at least 100.
Celgene said the rates of side effects were “low and similar across treatment groups” and that, again, no new safety problems popped up.
Biotech analysts weighing in on the data offered mixed opinions. Some deemed the figures impressive, while others felt the information so far is inconclusive. In a research note, Jefferies analyst Brian Abrahams wrote that given the small size of the trial, the design of the study, and “mixed” endoscopy data—there wasn’t a dose-dependent improvement on patients’ endoscopies—the results are difficult to interpret. Leerink Partners’ Geoffrey Porges wrote that the data suggest mongersen may be a viable drug and its benign safety profile so far bodes well, but it’s “hardly [a] transformative” drug given the modest effectiveness shown so far.
RBC Capital Markets’ Michael Yee was more bullish on the data, calling the results promising, “better than expected,” and helping bolster the idea that mongersen could be a $1 billion to $2 billion in sales per year drug for Celgene over time. Yee expects the drug’s effects on Crohn’s will get better over time. It “could be the [apremilast (Otezla)] of Crohn’s disease,” he wrote, referencing Celgene’s arthritis pill, which won FDA approval in 2014 and climbed to $472 million in sales last year. “Even if [it’s] not the most potent drug, it can still be a $1 billion-plus drug for [Celgene] if well tolerated, good safety, convenient, and good efficacy as the world moves to more orals,” he wrote.
As with apremilast, Celgene’s plan with mongersen is that a pill could be a significant differentiator in a market dominated by injectable biologic drugs. Patients with Crohn’s and ulcerative colitis (another form of IBD) typically end up on injectable biologics such as infliximab or adalimumab, which generate billions of dollars in sales every year. Many patients don’t respond to these biologics, however, and in some cases their effects diminish. Apremilast and mongersen are part of Celgene’s plan to branch out from its flagship blood cancer drugs and amass a second franchise in inflammatory diseases.
Celgene acquired mongersen when it paid Ireland’s Nogra Pharma $710 million up front in a 2014 licensing deal. Mongersen is meant to reduce the levels of a protein called Smad7, high levels of which are implicated in gut inflammation in people with IBD.