Drug Development

Celgene’s update on its big Crohn’s drug? Trust us, we’re doing fine

Celgene insists that an interim analysis of its closely-watched study for GED-0301 (mongersen) was “encouraging,” but it failed to show any of its cards today to back that up.

Scott Smith, President of Global Inflammation and Immunology, Celgene

Scott Smith, President of Global Inflammation and Immunology, Celgene

Celgene $CELG paid a whopping $710 million up front to in-license this drug for Crohn’s disease. Highlighted as a key program in its attempt to build a franchise for inflammation and immunology, GED-0301 is one of the company’s biggest experimental assets. And at this stage, all that Celgene is willing to say is that their investigators tracked an improvement in some patients.

Analysts have been waiting for some clear signs related to the drug’s effect on Crohn’s. Investigators have touted some remarkable advances for patients in an early study, but experts also questioned just how severely afflicted they were going into the study. “For shares to move higher,” BMO noted recently, “the trial needs to demonstrate CDAI benefit (P3 primary endpoint) in line with IGON-1 P2 trial and endoscopic improvement correlated with therapy duration.”

From the statement:

Topline data from CD-001 show that in a proportion of patients treated with oral GED-0301 there was endoscopic improvement (defined as a 25 percent improvement from baseline) and clinical response and remission across all treatment groups at week 12. Findings to date reveal no new safety signals and tolerability is consistent with earlier studies.

It’s not unusual, though, to see a proportion of patients improve in any arm of a study, including the placebo group. The question is the relative rate of improvement, and that’s something that Celgene is not referring to specifically. What it will allow is that the big biotech remains enthusiastic about its prospects.

“Given the high unmet need in Crohn’s disease, we are pleased that oral GED-0301 showed both endoscopic improvements and clinically meaningful responses and remission at an early timepoint in this study,” said Scott Smith, president of Celgene Inflammation and Immunology. “These data are particularly encouraging for several reasons, including the difficult-to-treat patient population evaluated in the trial.”

Analysts have virtually nothing to go on. But some are trying to read this update as best they can. And not everyone is as encouraged as Celgene professes to be. After all, says Baird’s Brian Skorney, “it’s hard to believe an unambiguous success wouldn’t be described in some quantitative detail.”

“In our view,” notes Leerink’s Geoffrey Porges, “while the drug could be active in some patients, it seems that the impressive data from the previous phase 2 trial will not translate to the phase 3 patient population, or to the real world which mainly consists of patients with active, longstanding, treatment resistant disease, similar to today’s endoscopy trial patients. Importantly, however, the relatively clean safety profile reported from previous studies was apparently maintained in this study. We spoke with Celgene this morning, and the company stated that there would be no changes to ongoing and planned phase 3 trials as a result of this analysis.”

Brian Abrahams at Jefferies had this:

KOLs had indicated to us they believed 1/3-1/2 of pts achieving a 50% endoscopic improvement (SES-CD score reduction) by 12wks would suggest true activity of the agent, which recall is still unclear based on the complex prior ph.IIa dataset. CELG had indicated that KOLs and regulators had told them a median 25% SES-CD score improvement for the whole group would be the bar for success. The PR’s statement that a proportion of pts had this 25% reduction appears to suggest activity fell short of both of these thresholds. However, we note that given the lack of substantial historical precedent, the bar for adequate supportive activity on endoscopy is not definitive, and based on our feedback with the company appears to be evolving from their continued discussions with physicians and regulators (and may now be a portion of pts achieving 25% reductions). Historical data has suggested in mod-to-severe Crohn’s pts taking a placebo, median SES-CD score improved by 6wks by a median of 14% and 1/4 of pts had 50% reductions, though this was from a small post-hoc analysis and may have been in less heavily pre-treated pts.

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