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  Title	Soligenix’s dusquetide receives US FDA orphan drug designation to treat Behçet's disease
  ID	12601214
  External ID	7972703897
  Author	Unknown
  Published Date	Aug. 19, 2025, 5:38 a.m.
  Summary	Soligenix, Inc., a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, announced that the Office of Orp
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  URL	https://www.pharmabiz.com/newsdetails.aspx?aid=180774&sid=2
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  Content ID	8029105
  Key	e53765da-817d-443c-be98-1d6043d16fcf
  Content	Soligenix, Inc., a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, announced that the Office of Orphan Products Development of the United States (US) Food and Drug Administration (FDA) has granted orphan drug designation to dusquetide, the active ingredient in SGX945, for "treatment of Behçet's disease" following review of recent phase 2a clinical results demonstrating biological efficacy and safety in patients with Behçet'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 US. 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. "Behçet'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. Given the clinically meaningful improvements seen in a phase 2 proof-of-concept study in patients with oral aphthous ulcers due to Behçet'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," stated Christopher J. Schaber, PhD, president and chief executive officer of Soligenix. "The FDA's decision to grant orphan drug designation to the SGX945 programme signifies an important step for Soligenix as we continue to advance the programme and adds significantly to the existing intellectual property estate surrounding this novel technology." Dusquetide, the active ingredient in SGX945 (Behçet's disease) and SGX942 (oral mucositis), is an innate defense regulator (IDR), a new class of short, synthetic peptides. It has a novel mechanism of action whereby it modulates the body's reaction to both injury and infection towards an anti-inflammatory, anti-infective, and tissue healing response. IDRs have no direct antibiotic activity but, by modulating the host's innate immune system responses, increase survival after infections caused by a broad range of Gram-negative and Gram-positive bacterial pathogens. Dusquetide also accelerates resolution of tissue damage following exposure to a variety of agents including bacterial pathogens, trauma, and chemo- and/or radiation therapy. 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-tumor activity has been demonstrated in multiple in vitro and in vivo xenograft studies. 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. Dusquetide has also demonstrated biological efficacy and safety in a phase 2a pilot study in 8 patients with Behçet's disease. The phase 2a study was an open-label study designed to be highly comparable (e.g., study endpoints, inclusion-exclusion criteria) to the published phase 3 study which was used to support marketing approval of apremilast (Otezla) for oral ulcers in Behçet's disease. The primary endpoint in the phase 3 apremilast study was the area under the curve (AUC) of the mean number of ulcers versus time. Using this same endpoint after 4 weeks of treatment, the SGX945 treated group had a 40% improvement relative to the placebo group from the phase 3 apremilast study, whereas apremilast had a 37% improvement relative to placebo. This improvement was sustained throughout the 4-week follow-up after treatment with SGX945, with 32% improvement evaluated at Week 8 despite treatment having stopped at Week 4. In contrast, apremilast, which was continuously administered through Week 12, had a 41% improvement at Week 8. One patient began the study with a punctuated skin ulcer and this also resolved during the 4-week treatment with SGX945. Skin ulcers are generally considered very difficult to resolve and usually require protracted treatment. Notably, some patients also explicitly reported experiencing less ulcers and pain during the 4-week follow-up period, as also reflected in the numerical analysis. SGX945 was well-tolerated with no treatment-related adverse events. Common adverse events for apremilast included diarrhoea (41% of patients), nausea (19% of patients) and headache (14% of patients), none of which were observed with SGX945. Soligenix has a strong intellectual property position in the IDR technology platform, including composition of matter for dusquetide and related analogs. Dusquetide was developed pursuant to discoveries made by Professors B. Brett Finlay, PhD and Robert Hancock, PhD of the University of British Columbia, Canada. Dusquetide has been awarded Fast-Track designation for the treatment of oral lesions of Behçet's disease and Orphan Drug designation for the treatment of Behçet's disease by the FDA. Behçet's disease is commonly known as an inflammatory disorder of the blood vessels (vasculitis). Often first diagnosed in young adults, its effects and severity will wax and wane over time. Major signs and symptoms usually include mouth sores (approximately 95% of patients), skin rashes and lesions (approximately 50% of patients), genital sores (approximately 50% of patients), leg ulcers (approximately 40% of patients) and eye inflammation (approximately 15% of patients). It is a painful disease, directly impacting the patient's quality of life and ability to productively engage in life activities, including work. Behçet's disease is thought to be an auto-immune disease with both genetic and environmental factors. It is most common along the "Silk Road" in the Middle East and East Asia, including Turkey, Iran, Japan and China. There are approximately 18,000 known cases of Behçet's disease in the US and over 50,000 in Europe. There are as many as 1,000,000 people worldwide living with Behçet's disease. There is no cure for Behçet's disease, rather treatments are prescribed to manage symptoms. Treatments may include both maintenance therapies and those specifically addressing flares (e.g., mouth ulcers, genital ulcers and leg ulcers). Corticosteroids are generally applied topically to sores and as eyedrops and may also be given systemically to reduce inflammation. Although used frequently, they have limited efficacy over the long-term and have significant side effects that become more concerning with more chronic use. Genital ulcers are often associated with significant genital scarring while leg ulcers can result in a post-thrombotic syndrome. Other treatments for Behçet's disease flares involve suppressing the immune system with drugs (e.g., cyclosporine or cyclophosphamide). These drugs come with a higher risk of infection, liver and kidney problems, low blood counts and high blood pressure. Finally, anti-inflammatory drugs are also used, including anti-TNF medications. The only approved drug in Behçet's disease is apremilast, which is used as a maintenance therapy to prevent formation of oral ulcers. Unfortunately, apremilast must be used continuously to be effective and is associated with both high cost and side effects including diarrhoea, nausea, upper respiratory tract infection and headache. Soligenix is a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need.
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