Note: This story was first published on July 14. It's been updated and republished with additional information about investor expectations for the GED-0301 study results, which Celgene is likely to announce in early September.
Celgene (CELG) will use a medical meeting this fall to address a lingering investor concern about GED-0301, an experimental pill for Crohn's disease and one of the most important drugs in the company's research pipeline.
GED-0301 is one of three drugs -- Otezla and ozanimod are the others -- which form the core of Celgene's burgeoning immunology and inflammation franchise. Today, Celgene's blood cancer drugs, mainly the multiple myeloma drug Revlimid, account for a majority of the company's revenue and profit. But in the next four to five years, Celgene is counting on "I&I" drugs like GED-0301 to diversify and extend the company's prodigious growth projections.
At the medical meeting, (Celgene isn't being any more specific about which meeting) results will be presented from a small phase II study in which doctors use an endoscope to peer into the gastrointestinal tract of Crohn's patients treated with GED-0301.
The "endoscopy data" assess the ability of GED-0301 to reduce the size and severity of ulcers in the colon and bowel. These ulcers -- a hallmark of Crohn's disease -- occur when a patient's own immune system mistakes the gastrointestinal tract as a foreign body needing to be eliminated.
Investors were first excited about the blockbuster potential of GED-0301 in March 2015, when a mid-stage study of GED-0301 demonstrated high rates of clinical remission in Crohn's patients. Data from this study were called "unprecedented" by a physician in an editorial published in the New England Journal of Medicine. However, the physician also raised a concern that enrolled patients may have had more moderate Crohn's disease, making it easier for GED-0301 to show an outsized treatment effect.
In that study, Celgene defined remission as a durable reduction in the signs and symptoms of the disease, measured by the Crohn's Disease Activity Index (CDAI). The study did not require patients to undergo bowel examination with an endoscope.
Given the concerns raised about the prior phase II study, investors would like more direct evidence that GED-0301 can heal the guts of Crohn's patients. This is why the current endoscopy study is important.
As a pill, GED-0301 could become a preferred Crohn's therapy over the currently approved injectable drugs like AbbVie's Humira or Johnson & Johnson's Remicade, which together generate approximately $3 billion to $5 billion in sales worldwide in Crohn's.
Analysts' estimates for peak sales of GED-0301 in Crohn's range from $2 billion to $3 billion, depending on the drug's ultimate benefit-risk profile.