Absent a placebo arm for comparison, the GED-0301 Crohn's data presented Tuesday (announced via press release on Monday) are largely uninterpretable.
Celgene is not some dinky micro-cap biotech lacking the resources or knowledge to conduct a rigorous clinical trial, so omitting a placebo arm from the GED-0301 study is inexcusable.
A Celgene spokesperson did not respond to questions.
I'm not the only person frustrated with Celgene. Piper Jaffray analyst Josh Schimmer titled his Celgene note on GED-0301, "That's Why Placebo Controlled Trials are Needed."
I like what Schimmer says here:
While response and remission rates seen with '301 are consistent with rates seen with active drug arms of placebo controlled trials, the rates with placebo in trials can be substantial ... When [approximately] 60 patients are divided across 3 dose arms and subsequent subset analyses are performed, the error bars become extremely wide. We were concerned by how confident CELG was in interpreting the small dataset overall but then fell back on the 'small patient numbers' argument to explain away any inconsistencies in the data. And this ultimately highlights the reason that drugs for Crohn's disease all have to complete P3 trials to gain FDA approval.
In Celgene's study, the 37% of Crohn's patients characterized as endoscopic responders at the 25% cutoff fell to 15% at the more rigorous and meaningful 50% cutoff.
In the randomized phase II study of filgotinib in Crohn's conducted by Gilead Sciences (GILD) - Get Report and Galapagos (GLPG) - Get Report , the placebo arm showed a 14% endoscopic response rate at the 50% cutoff. The endoscopic response rate for filgotinib was 25%.
Celgene and its supporters believe GED-0301 is active against Crohn's disease even without a placebo comparison. They caution against making the cross-trial comparison between the GED-0301 results and those presented for filgotinib.
Okay, that might certainly be true, but it's also hard not to look at the 14% placebo response rate in the filgotinib study and wonder if GED-0301 (15% response rate) is also a placebo. It would certainly be more accurate and reassuring to have placebo data from the Celgene study, but it's missing.
Thanks for nothing, Celgene. Without definitive data, investors are served up rose-tinted pablum masquerading as analysis, courtesy of RBC Capital analyst Michael Yee. (Emphasis below is mine.)
The data today are early and help de-risk but ultimately the Phase III data with full one-year time point will give the complete picture (readout early '18). The investigator helped reassure investors and suggested (1) overall clinical remission (48%) and endoscopy response (37%) are solid and biomarkers all go in right direction in a severe population and not too concerned there was no placebo (said typically placebo would have single digit CDAI remissions and endoscopy nearly zero remissions and thus data appears all better than placebo), (2) data appears "somewhat similar" to GLPG/GILD filgotinib in that they are active and show endoscopy response (with all caveats of cross-trial comparison.)
Do you feel reassured? With all those qualifiers? I certainly don't.
We won't get a complete picture of GED-0301's efficacy and safety profile in Crohn's until 2018, when a phase III study is expected to read out. Before then, Celgene will have results from a phase II study of GED-0301 in ulcerative colitis in 2017. But it's another single-arm study lacking a placebo comparison.
More of the same.
Adam Feuerstein writes regularly for TheStreet. In keeping with company editorial policy, he doesn't own or short individual stocks, although he owns stock in TheStreet. He also doesn't invest in hedge funds or other private investment partnerships. Feuerstein appreciates your feedback; click here to send him an email.