Switzerland-based Celgene International's Phase II trial of oral small-molecule inhibitor of phosphodiesterase 4 (PDE4), Apremilast, has demonstrated statistical significance for primary and secondary endpoints in patients with Behcet's disease (BD).
The primary endpoint of the average number of oral ulcers at day 85 and the number of oral ulcer-free patients at week 12, the complete response rate, were statistically significant.
All secondary endpoints, such as patient-reported outcomes, also achieved statistical significance and clinically meaningful responses.
Celgene I&I clinical research and development vice president Randall Stevens said; "I think that apremilast could potentially be a very useful addition to our armamentarium for the treatment of Behcet's disease, given the high unmet medical need and the severe impact that mucocutaneous ulcerations can have on a patient's quality of life."
The BCT-001 double-blind, parallel-group, placebo-controlled study randomised a total of 111 active BD subjects with either apremilast (APR) 30mg BID or identically appearing placebo (PBO), stratified by gender in 1:1 ratio.
Additionally, the study established considerably increased oral ulcer pain (VAS) improvement with apremilast compared to placebo and complete response at week 12 was demonstrated by 100% patients with genital ulcers at baseline receiving APR against 50% receiving PBO.
While the overall safety and tolerability profile was consistent with earlier apremilast studies conducted in other patient groups, the treatment-emergent adverse events were analogous among 30mg BID and placebo.
With no approved therapies in the US or Europe, Behcet's disease is a rare chronic inflammatory disorder with characteristics of recurrent oral and genital ulcers, skin and eye lesions and joint inflammation, as well as involvement of the brain and GI tract.
Image: Computer illustration of the backbone structure of HLA-B5101 complexed with HIV's immunodominant epitope KM2 1e28, strongly associated with Behcet's disease. Photo: Courtesy of Pdeitiker.