Soligenix Inc (Nasdaq: SNGX), a US-based late-stage biopharmaceutical company focused on developing and commercialising products to treat rare diseases where there is an unmet medical need, announced on Monday that the Office of Orphan Products Development of the US Food and Drug Administration (FDA) has granted orphan drug designation to dusquetide, the active ingredient in SGX945, for the treatment of Behcet's Disease.

This follows a review of recent Phase 2a clinical results demonstrating biological efficacy and safety in patients with Behcet's Disease.

The US Orphan Drug Act is intended to assist and encourage companies to develop safe and effective therapies for the treatment of rare diseases and disorders, defined as one that affects fewer than 200,000 people in the United States. In addition to providing a seven-year term of market exclusivity upon final FDA approval, orphan drug designation also positions Soligenix to be able to leverage a wide range of financial and regulatory benefits, including government grants for conducting clinical trials, waiver of expensive FDA user fees for the potential submission of a New Drug Application (NDA), and certain tax credits.

"Behcet's Disease is an area of unmet medical need, with up to 18,000 people in the US, 50,000 in Europe, 350,000 people in Turkey and as many as 1 million people worldwide affected by this incurable disease," said Christopher J. Schaber, PhD, Soligenix president and CEO. "Given the clinically meaningful improvements seen in a Phase 2 proof-of-concept study in patients with oral aphthous ulcers due to Behcet's 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."

Dusquetide, the active ingredient in SGX945 (Behcet's Disease) and SGX942 (oral mucositis), is an innate defence regulator (IDR), a new class of short, synthetic peptides. Preclinical efficacy and safety have been demonstrated in numerous animal disease models including mucositis, colitis, macrophage activation syndrome as well as bacterial infections. In addition, potential anti-tumour activity has been demonstrated in multiple in vitro and in vivo xenograft studies.

Solgenix says that Dusquetide has demonstrated safety and tolerability in a Phase 1 clinical study in 84 healthy human volunteers. In Phase 2 and 3 clinical studies with dusquetide in over 350 subjects with oral mucositis due to chemoradiation therapy for head and neck cancer, positive efficacy results were demonstrated, including potential long-term ancillary benefits.