REGENERON REPORTS POSITIVE DATA ON CAPS TREATMENT

Regeneron Pharmaceuticals has announced positive data from a Phase III clinical program designed to provide two separate demonstrations of efficacy for the investigational drug Interleukin-1 (IL-1) Trap within a single group of patients suffering from a rare chronic disease known as CIAS1-related autoinflammatory periodic syndromes (CAPS). The Phase III program of the IL-1 Trap (rilonacept) included two studies (Part A and Part B). Both studies met their primary endpoints. The primary endpoint of both studies was the change in disease activity, which was measured using a composite symptom score composed of a daily evaluation of fever/chills, rash, fatigue, joint pain and eye redness/pain.

Regeneron plans to file a biologics license application (BLA) with the FDA in the second quarter of 2007, following completion of a 24-week open-label extension phase. The FDA has granted orphan drug status and fast-track designation to the IL-1 Trap program for the treatment of CAPS.

The first study (Part A) was a double-blind, placebo-controlled six-week trial, in which patients randomized to receive IL-1 Trap had an approximately 85 percent reduction in their mean symptom score compared to an approximately 13 percent reduction in patients treated with placebo. Following a nine-week interval during which all patients received IL-1 Trap, a randomized withdrawal study (Part B) was performed, in which the same patients were re-randomized to either switch to placebo or continue treatment with IL-1 Trap in a double-blind manner. During the 9-week randomized withdrawal period, patients who were switched to placebo had a five-fold increase in their mean symptom score, compared with those remaining on IL-1 Trap who had no significant change. Both the Part A and Part B studies achieved statistical significance in all of their prespecified secondary and exploratory endpoints.