Dusquetide Granted Orphan Drug Status for Behçet Disease

The Food and Drug Administration (FDA) has granted Orphan Drug designation to dusquetide (SGX945) for the treatment of Behçet disease, a type of vasculitis characterized by mucocutaneous symptoms (eg, oral and genital ulcers).

Dusquetide is part of a new class of short, synthetic peptides known as innate defense regulators. This novel therapeutic approach is expected to target the underlying innate immune dysfunction associated with Behçet disease, thereby providing an anti-inflammatory, anti-infective, and wound healing response.  

The Orphan Drug designation was supported by data from an open-label phase 2 trial (ClinicalTrials.gov Identifier: NCT06386744) that included 8 adult patients with mild to moderate Behçet disease and active oral and/or genital ulcers. Study participants received dusquetide as a twice-weekly 4-minute intravenous infusion for 4 weeks, followed by 4 weeks of follow-up. 

The efficacy of dusquetide was based on a comparison with the apremilast and placebo arms of a phase 3 study that formed the basis of approval for apremilast in Behçet disease. The primary endpoint of this phase 3 trial was area under the curve (AUC) of the mean number of ulcers vs time.

Findings at 4 weeks showed treatment with dusquetide led to a 40% improvement relative to the placebo group from the phase 3 apremilast trial, indicating a highly comparable result with apremilast (37% improvement relative to placebo). The improvement observed with dusquetide was sustained throughout the 4-week follow-up period; a 32% improvement was reported at week 8 despite patients having stopped treatment at week 4. In the apremilast trial, patients continued treatment through week 12, with 41% improvement reported at week 8. 

During the 4-week treatment period with dusquetide, 1 patient experienced resolution of a punctuated skin ulcer. Additionally, some patients had experienced less ulcers and pain during the 4-week follow-up period. While diarrhea, nausea, and headache were reported with apremilast, these adverse events were not seen with dusquetide.

“Given the clinically meaningful improvements seen in a phase 2 proof-of-concept study in patients with oral aphthous ulcers due to Behçet Disease, we are hopeful dusquetide will have a role to play in helping underserved patients suffering from this difficult to treat and chronic auto-immune disease,” stated Christopher J. Schaber, PhD, President and Chief Executive Officer of Soligenix. “The FDA’s decision to grant Orphan Drug designation to the SGX945 program signifies an important step for Soligenix as we continue to advance the program and adds significantly to the existing intellectual property estate surrounding this novel technology.”

Dusquetide previously received the FDA’s Fast Track designation for the treatment of oral lesions of Behçet Disease.

This article originally appeared on MPR