A month after posting an underwhelming top-line assessment of a Phase Ib study of its closely watched Crohn’s drug mongersen (GED-0301) for Crohn’s disease, Celgene posted an update Sunday evening that included a positive snapshot of the actual data.
Investigators for the study say they were able to track a quick response to the drug, which Celgene paid a whopping $710 million in cash to in-license the drug for Crohn’s. The researchers tracked the impact of 160 mg doses of the drug on endoscopic response and clinical remission among 63 patients with active Crohn’s disease over short periods stretching from 4 to 8 to 12 weeks. And they say the 12-week group has responded with the most impressive results — though some might object to what qualifies as impressive at this stage of the game for such an expensive therapy.
Clinical improvement was seen by week 2, and clinical response (CDAI decrease ≥100) and remission (CDAI <150) rates were highest in the 12-week treatment group at 67 and 48 percent respectively, at week 12. The mean CDAI reduction from baseline at week 12 in the 12-week treatment group was 133 points. Of the patients with evaluable endoscopies at week 12 (n=52), 37 percent had an endoscopic response (≥25 percent reduction in SES-CD score from baseline), with no meaningful difference across treatment groups. In addition, of those patients with greater endoscopic disease activity at baseline (SES-CD score of >12; n=16), 63 percent exhibited a reduction ≥25 percent in SES-CD score and 31 percent had a reduction of ≥50 percent.
The investigators touted the results being posted at United European Gastroenterology Week in Vienna as evidence of the kind of efficacy that Celgene was looking for. Analysts concentrated on that response rate and whether it qualifies as the kind of result they were looking for. And they can be a tough crowd.
Baird’s Brian Skorney gave a thumbs up to 48% remission rate, but was unimpressed by the 37% endoscopic response score. His bottom line:
This data appears to be more promising than we expected but it is unlikely to drive a material increase in confidence. The endoscopy results fall short of what we have seen from other studies but the CDAI responses do appear in line with other agents.
Likewise, Leerink’s Geoffrey Porges saw the potential here for blockbuster sales of $1.3 billion in 2023 with the drug a likely winner on frontline approval status. But there’s nothing here that suggests an out-of-the-park home run hit. And Credit Suisse cautions that this is still early data from a small study. Doctors don’t usually do endoscopies at 12 weeks and investigators were really looking for signs of activity.
Investors liked the sounds of that, driving up Celgene’s shares by 1.3% Monday afternoon.
Add it all up and the drug still looks promising though the jury is definitely still out on what kind of market awaits GED-0301.
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 in the pipeline of the big biotech, which has struck a series of bold deals to build its portfolio of experimental therapies. The drug is designed to slice Smad7 protein levels. Spiking levels of Smad7 interfere with TGF-β1 anti-inflammatory pathways in the gut, spurring inflammation.
Celgene, though, was clearly happy to praise the results.
“We are encouraged that oral GED-0301 showed both meaningful endoscopic improvement and clinical remission at an early time point in this study,” said Scott Smith, president of Celgene Inflammation & Immunology, in a statement. “The fact that this study included nearly 50 percent biologic-experienced patients further reflects the potential of GED-0301 as a novel approach for patients with Crohn’s disease searching for alternatives.”
The big biotech also posted some updated ozanimod data from a mid-stage program on ulcerative colitis, noting some fresh signs of efficacy from the extension results. The added data were also released in Vienna.
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