Celgene Corporation (NASDAQ:CELG) reported additional positive trial data for its experimental Crohn’s disease drug, GED-0301on Sunday, reinforcing results from an earlier study and increasing its chances of regulatory approval. The oral drug is particularly significant because it can break a market currently dominated by blockbuster injectable, biologic treatments like Remicade and Humira, and has already reported great results from a mid-stage trial that saw more than 60% of patients reach remission at the highest dose.
However, the former study only measured GED-0301’s effects by the reduction of signs and symptoms of Crohn’s disease, without examining its impact on the ulcers through endoscopies. The data Celgene presented at the United European Gastroenterology Week in Vienna is an early look at an ongoing, 63-patient Phase 1b study of GED-0301 which also measured gut health. 37% of Crohn’s patients saw an improvement of 25% or higher at the 12th week of treatment in a score that measures the healing of patients’ ulcers in the colon and bowel (SES-CD).
The company tested the drug for treatment lengths of 4, 8 and 12 weeks, with the latter providing the best outcome of 48% in remission. Credit Suisse’s analysts said in a research note, “Data from the abstract continues to validate activity of GED-0301 in Crohn’s showing endoscopic improvement at just 12 weeks (which is relatively early in the course of treatment).” They also noted that relative to the drug’s Phase 2 trial, the study had a more severe patient population at baseline (46% have been on anti-TNFs like Humira), while the CDAI (a survey that quantifies patients’ disease symptoms) baseline was higher. The analysts said they looked forward to more data on sub-groups and biomarkets in the full data release on Tuesday, although they expect the results to be “fairly consistent.”
Jefferies’ analysts said that while the latest data increases the likelihood of GED-0301 ultimately developing into a drug, its “mixed” endoscopy data, small trial size and no placebo arm along with absence of full details meant it remained risky going into Phase 3 data. Analyst Brian Abrahams noted that there was no dose-dependent improvement on patients’ endoscopies which was “disappointing.” Leerink Partners’ analyst Geoffrey Porges said in a research note that GED-0301 may be a viable treatment and has a benign safety profile so far, but given its modest efficacy shown so far, it is “hardly transformative.”