Otezla Shows Promise in Treatment of Rare Inflammatory Disease

Treatment found to improve several measures of disease activity and quality of life in patients with Behçet's disease.

A treatment for a rare, painful inflammatory condition showed promising signs of improving disease activity and quality of life during a recent phase 2 trial.

Results were announced last week for a randomized placebo-controlled trial for apremilast (Otezla) in the treatment of patients with Behçet's disease, which is characterized by recurrent oral and genital ulcers. Patients can also suffer joint inflammation and recurrent skin and eye lesions.

The results of the trial were published April 16, 2015 in The New England Journal of Medicine.

"Painful oral ulcers due to Behçet's disease are the hallmark of the condition and can have a significant impact on the lives of many patients,” Gulen Hatemi, MD, associate professor, said in a press release. “Currently used drugs for this condition may not control oral or genital ulcers in some patients or have potential adverse events that may limit their use.”

The primary endpoint of the study was a reduction in the mean number of oral ulcers after 12 weeks of treatment. At the end of 12 weeks, patients in the placebo cohort crossed over to the apremilast cohort, while patients in the apremilast arm continued with treatment for an additional 12 weeks.

In patients treated with apremilast for the full 24 weeks, the decrease in the mean number of oral ulcers was 0.3 at week 2 compared with 2.7 at baseline. The decrease was sustained through week 24 at 0.6.

Meanwhile, the placebo cohort showed the mean number of oral ulcers to be 2.9 at baseline, compared with 1.7 after 2 weeks. At week 24 in the placebo group after 12 weeks of treatment with apremilast, the mean number of oral ulcers was 0.4.

There was also a decrease in pain associated with oral ulcers that matched the drop in ulcers during the course of treatment. The mean pain scores dropped from 54.3 at baseline to 12 after 2 weeks and 9.7 after 24 weeks in the apremilast arm.

In the placebo cohort, pain scores were 51.7 at baseline and 29.8 after 2 weeks. After 12 weeks of treatment with apremilast, the mean pain score was 9.7. The drug was also found to significantly improve several measures of disease activity and quality of life after 12 weeks.

There were serious adverse events reported in 2 patients in the apremilast group and 4 patients discontinued treatment. Side effects such as nausea, vomiting, and diarrhea were more common in the apremilast arm.

A global phase 3 clinical trial for apremilast is currently enrolling patients.

"Celgene is dedicated to investigating and delivering to patients new treatment options for rare, chronic inflammatory disorders such as Behçet's disease," said Scott Smith, President, Celgene Inflammation & Immunology. "Based on these phase II results, Celgene has filed with regulatory authorities in Turkey and has initiated a global phase III trial of OTEZLA in this debilitating disease."