The drug works in a similar way to Novartis' Gilenya in multiple sclerosis—a market with 12 disease-modifying drugs already approved. Analysts say Receptos' medicine may work as well as Gilenya, but could come with fewer side effects.
"1063 may have similar efficacy to Gilenya with less hepatotoxicity and only a mild, benign first-dose heart rate effect," Joseph Schwartz, an analyst with Leerink Partners, wrote in a research note Thursday. Gilenya is associated with increased liver enzymes and requires monitoring for slowed heart rate on the first dose. Still, it draws more than $2 billion annually for the Swiss drugmaker.
Receptos' medicine is in the third and final stage of testing generally required for regulatory approval, with data expected in MS in 2017, CEO Faheem Hasnain said in a telephone interview Thursday. The company is also testing the compound in areas Gilenya isn't approved—ulcerative colitis and Crohn's disease—where it would compete with inflammatory drugs such as Johnson & Johnson's Remicade and AbbVie's Humira.
There, the advantage is that RPC1063 is a pill, rather than an infused drug like existing options.
"That differentiation is obvious," Hasnain said. "But our efficacy looks to be in line with those biologics, if not better."
The data reported in October from a mid-stage study in ulcerative colitis, a chronic inflammatory bowel disease that may affect as many as 700,000 people in the U.S., were "arguably about as good as it gets," Hasnain said. The company is due to present additional data from the study Saturday at the European Crohn's and Colitis Organisation meeting.