Soligenix, Inc. has announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to dusquetide, the active ingredient in its product, SGX945, for the treatment of Behçet's Disease. This designation follows a review of positive Phase 2a clinical trial results that showed both biological efficacy and safety in patients. Behçet's Disease is a rare, incurable, and chronic autoimmune disorder affecting up to 18,000 people in the U.S. and one million worldwide. The disease is characterized by painful symptoms like mouth, genital, and skin sores, as well as eye inflammation. Current treatments only manage symptoms and come with significant side effects, highlighting a major unmet medical need.

The FDA's orphan drug designation is a significant step for Soligenix as it provides a path for developing treatments for rare diseases, which affect fewer than 200,000 people in the U.S. This designation not only offers a seven-year term of market exclusivity if the drug is approved but also provides financial and regulatory benefits, including eligibility for government grants, tax credits, and waivers of costly user fees. This new designation adds to the existing Fast-Track designation already received for the treatment of oral lesions of Behçet's Disease. Dr. Christopher J. Schaber, Soligenix’s President and CEO, stated that given the "clinically meaningful improvements" seen in the Phase 2a study, the company is hopeful that dusquetide will help underserved patients.

Dusquetide is part of a new class of synthetic peptides called innate defense regulators (IDRs), which work by modulating the body's response to injury and infection toward anti-inflammatory and tissue-healing responses.

In a Phase 2a pilot study involving eight patients, SGX945 demonstrated biological efficacy and safety that was comparable to a key competitor, apremilast (Otezla®), which is the only currently approved drug for oral ulcers in Behçet's Disease. In the pilot study, SGX945 showed a 40% improvement in the mean number of ulcers relative to the placebo group from the apremilast trial, compared to apremilast's 37% improvement. This improvement was sustained even after treatment with SGX945 stopped, while apremilast required continuous administration. The drug was also well-tolerated, with no treatment-related side effects observed, a significant advantage over apremilast which is associated with common adverse events like diarrhea, nausea, and headaches. These promising results and the new orphan drug designation strengthen Soligenix's intellectual property and position the company to advance the program toward further clinical development.