Celgene Touts Data From Early Study of Oral GI Drug

Xconomy Boston — 

For a few years now, Celgene has been trying to show that the $710 million it shelled out for a gastrointestinal drug from Ireland’s Nogra Pharma was money well spent. Data released this morning from an early study are meant to help boost its case.

Celgene (NASDAQ: CELG), of Summit, NJ, said this morning that an early study of GED-0301, also known as mongersen, helped improve the condition of a group of patients with Crohn’s disease after 12 weeks of treatment. This is an early look at an ongoing, 63-patient Phase 1b study of mongersen; more data from the study are being accumulated, and a larger, potentially more telling Phase 3 trial is ongoing. But the data released today are important for Celgene because they represent the first time Crohn’s patients on mongersen were given endoscopies to show how the drug is impacting the size of their ulcers. Previously, mongersen’s effects were measured only by the reduction of signs and symptoms of the disease.

Celgene said that patients on the drug in the Phase 1b trial showed an endoscopic improvement—defined as a 25 percent improvement from the start of the study—after 12 weeks of treatment. Without giving specific numbers, Celgene said that “a proportion of patients” in all of the different treatment groups (Celgene tested three different doses of mongersen) improved, that there were remissions in each group, and that no new safety problems popped up. Patients in the study will now be followed for 52 weeks without treatment and Celgene will report the effects on their gut, with data coming next year.

Shares of Celgene climbed slightly in pre-market trading Monday. RBC Capital Markets analyst Michael Yee called the data positive and “an initial de-risking step” that suggests that the drug might be working. Yee believes the drug could eventually bring in $1 billion to $2 billion in revenue for Celgene, should it make it to market. Hype for the drug as a possible blockbuster built last year when the New England Journal of Medicine published data from a different Phase 2 trial. Author Severine Vermeire wrote in the NEJM at the time that mongersen led to remission rates that would be “unprecedented” if they held up in further testing.

More will be learned soon when Celgene provides more details from the Phase 1b trial at a medical meeting, possibly next month.

“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 Celgene’s president of inflammation and immunology, Scott Smith, in a statement.

Mongersen is an important drug for Celgene, which is best known for its blood cancer drugs but has been trying to expand and amass a second franchise in inflammatory diseases. The first of those drugs to win approval was apremilast (Otezla), that generated $472 million in sales last year as an arthritis drug and which Celgene is developing for a variety of inflammatory diseases. The next two on tap are mongersen, which Celgene spent $710 million for up front in April 2014, and ozanimod, which Celgene acquired when it paid $7.2 billion for Receptos in July 2015.

Celgene bought into mongersen on the belief that a pill could be a significant differentiator in a market dominated by injectable biologic drugs. Mongersen is being developed for ulcerative colitis and Crohn’s, forms of inflammatory bowel disease, a chronic condition that affects about 1.6 million Americans, according to the Crohn’s & Colitis Foundation of America. Patients typically end up on injectable biologics such as infliximab (Remicade) or adalimumab (Humira), which generate billions of dollars in sales every year. Many patients don’t respond to these biologics, however, and in some cases their effects diminish. They also increase possible risks of infection. Celgene’s drug is meant to reduce the levels of a protein called Smad7, high levels of which are implicated in gut inflammation in people with IBD.