New Phase 3 Data Shows Significant Improvements in Behcet's Disease

On Saturday, Celgene Corporation announced that data from the Phase 3 RELIEF clinical trial of Otezla (apremilast) in patients with active Behçet’s disease with oral ulcers were presented in a presentation at the 2018 American Academy of Dermatology (AAD) Annual Meeting.

The results showed statistically significant reductions in oral ulcers with apremilast, Celgene’s oral selective inhibitor of phosphodiesterase 4 (PDE4), when administered at 30 mg twice daily (BID) versus placebo through week 12.

Behçet’s disease, or Behçet’s syndrome, is a rare genetic disorder that leads to the inflammation of blood vessels. Behçet’s disease most commonly presents with oral ulcers that can be disabling and severely damage a patient’s quality of life. The study, which randomized 207 patients to apremilast 30 mg BID or placebo, evaluated the effect of apremilast on recurring oral ulcers in patients who were previously treated with at least one topical or systemic medication.

The area under the curve (AUC) assesses the change in the number of oral ulcers over time, accounting for the clinical characteristic that oral ulcers repeatedly remit and recur. A reduction in oral ulcers was the primary endpoint of the 52-week trial, and at week 12, it was observed that the AUC for the number of oral ulcers was statistically significantly reduced by Celgene’s therapy.

Secondary endpoints included: oral ulcer pain, overall disease activity, Behçet’s Disease Current Activity Index, and quality of life.

“Reducing oral ulcers, which are painful and can negatively impact quality of life, is an important goal in the treatment of people with Behçet’s syndrome,” said Gulen Hatemi, M.D., Associate Professor, Istanbul University Cerrahpassa Medical School in a press release. “These findings suggest that apremilast, which reduced oral ulcers and oral ulcer pain, and improved disease activity in this pivotal study, has the potential to be a treatment option for patients with active Behçet’s syndrome with oral ulcers, for which few treatment alternatives exist.”

Adverse events (AEs) observed in patients who were given apremilast throughout the trial included: diarrhea (41.3%), nausea (19.2%), headache (14.4%), and upper respiratory tract infection (11.5%). The safety profile was consistent with what had already been observed in patients who received the drug.

“The positive phase III findings in Behçet’s Disease reflect the unique aspects of the profile of OTEZLA® (apremilast) 30 mg across inflammatory-related diseases,” said Terrie Curran, President, Celgene Inflammation and Immunology. “OTEZLA® (apremilast) 30 mg has the potential to provide a clinically meaningful new treatment option for patients and doctors and to become the first product indicated specifically for the treatment of active Behçet’s Disease with oral ulcers.”

Celgene has plans to submit a supplemental New Drug Application (sNDA) for apremilast 30 mg BID for the treatment of active Behçet’s Disease with oral ulcers in the U.S. in the second half of 2018.

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