Celgene to initiate apremilast Phase III placebo-controlled trial for ankylosing spondylitis

Celgene International Sàrl, a subsidiary of Celgene Corporation (NASDAQ: CELG), today announced data from a Phase II study of apremilast, its investigational oral immunomodulatory compound, in patients with ankylosing spondylitis (AS) were presented at the American College of Rheumatology Scientific Meeting in Chicago, IL. Based on the data from this study, the company will be initiating a Phase III placebo-controlled trial with apremilast in ankylosing spondylitis during the first half of 2012.

The Spondylitis Trial of Apremilast for better Rheumatic Therapy (START) was a double-blind, placebo-controlled investigator-initiated pilot study conducted in subjects with AS. The primary objective of this study was to detect an efficacy signal using the Bath Ankylosing Spondylitis Disease Activity Index (BASDAI).

In the study, AS patients who had been symptomatic for at least two years, with their disease uncontrolled by conventional non-steroidal anti-inflammatory drugs and with daily spinal pain and stiffness for at least two weeks, were treated for 12 weeks with 30 mg BID apremilast or placebo. The treatment phase was followed by a 4-week observation phase.

Thirty-eight patients were randomized and 36 patients were evaluable for response. These patients were evaluated based on mean change from baseline across multiple parameters including the Bath Ankylosing Spondylitis Disease Activity Index (BASDAI), Functional Index (BASFI) and Metrology Index (BASMI). Additionally, plasma levels of sclerostin and serum levels of receptor activator of nuclear factor kappa-B ligand (RANKL) and osteoprotegerin (OPG) were measured at baseline and after 12 weeks.

More patients receiving apremilast versus placebo reported loose stools (26.3% vs. 10.5%) and headache (42.1% vs. 26.3%). There was no relevant difference in the incidence of diarrhea (10.5% in both arms), nausea (15.8% in both arms) or upper respiratory tract infections (31.6% in both arms) between the groups. There were no serious adverse events reported in the study.

These results are from an investigational study. Apremilast is not approved for the treatment of patients with ankylosing spondylitis or any other indication.