Karen Krumeich, the company’s senior vice president, will discuss the continuing development of the treatment for cutaneous T-cell lymphoma (CTCL) and oral mucositis at the World Orphan Drug Congress USA on April 21.
Her presentation will be titled “Rare Disease Treatment in Cutaneous T-cell Lymphoma and Oral Mucositis.” The congress is running from April 19-21.
Soligenix, based in Princeton, New Jersey, develops products to treat rare diseases and to counter biological weapons.
The rare diseases it is targeting include CTCL, oral mucositis, children’s Crohn’s disease, and acute radiation enteritis. It is also developing vaccines to protect military personnel and civilians against biological weapons such as ricin, gastrointestinal acute radiation syndrome, and melioidosis.
SGX3o1, one of its lead product candidates, is a synthetic form of the photosensitizer, hypericin. It is used as a gel to treat patients with patch/plaque phase CTCL, also known as mycosis fungoides or Alibert-Bazin syndrome.
In contrast with other photodynamic therapies, which are activated with ultraviolet light, SGX301 is activated with fluorescent light. The drug’s use of visible light is a major advance because it can prevent secondary UV-related malignancies, such as melanoma.
SGX301 inhibited the growth of malignant T-cells isolated from CTCL patients in a lab, Soligenex said. A Phase 1 study in healthy volunteers also indicated it was safe, the company said.
Fifty-six percent of mycosis fungoides patients in a Phase 2 study showed a significant treatment response to SGX301, compared with only 8 percent of placebo-treated patients. Not only did it improve CTCL patients’ skin lesions, but it was also well tolerated, according to the trial results. In addition, the Phase 2 study confirmed the Phase 1 trial’s finding that SGX301 is safe.
Based on the results of the two clinical trials, the United Kingdom’s Medicines and Healthcare Products Regulatory Agency has designated SGX301 a Promising Innovative Medicine (PIM) for treating CTCL.
Soligenex is in the midst of recruiting participants for the Phase 3 FLASH study (NCT02448381). The objective of the multicenter, placebo-controlled clinical trial is to confirm SGX301’s effectiveness as a treatment for CTCL skin lesions.