Phase 3 Study of SGX301 and Fluorescent Light for Cutaneous T Cell Lymphoma

Project Summary Abstractchanges are in Time New Roman fontCutaneous T cell lymphomaCTCLis a rare form of Non Hodgkin s LymphomaNHLaffecting approximatelypatients annually in the United States and can be characterized by either an indolent or aggressive disease progressionEither case is accompanied by significant morbidityparticularly due to the skin effects of the diseaseThe indolent disease may eventually progress to a more aggressive systemic disease associated with significant mortalityWhen the disease is primarily localized to the skin it is referred to as mycosis fungoidesMFand is characterized by abnormal accumulation of T cells in the skincausing lesionswhich increasingly cover the body as the disease progressesThese lesions can be scaly patches of varying color and or eczema like rashes and or psoriasis like rashesThick tumors may develop on the skin that may eventually become openinfected ulcersThe lesions are often associated with severe itchingmaking sleep and other daily activities difficultEarly treatment of MF can prevent progression to the open ulcerative skin tumor stageCTCL is an orphan disease with no approved first line therapyThe active pharmaceutical ingredient of Soligenix s SGXtechnology is synthetic hypericina photo activated agent that intracellularly concentrates in the endoplasmic reticulum andwith activation by visible lightgenerates singlet oxygen that causes localized reactive oxygen species that initiates the mitochondrial apoptotic pathway Because of its lack of DNA interactions and negative Ames testit is anticipated that this treatment will have a substantially lower risk for secondary cancer development than the majority of CTCL treatments thatbecause their anti tumor effects are generated through DNA damage mechanismshave either theoretical or proven long term association with secondary cancers including melanomaImportantlyhypericin is not by itself mutageniclike other treatmentsand is activated by visible lightwhich is not carcinogenicThe two components can be easily combined at the site of a skin lesionresulting in highly selective delivery and killing of tumor cells with minimal systemic impact on the patientPhaseandclinical studies have clearly demonstrated its safety and efficacyincluding a patient response rate rtwith hypericin treatmentorSGXhas been granted Fast Track Designation by the US FDA for the first line treatment of CTCL and hypericin has been granted Orphan Product Designation for the treatment of CTCLDesignationThis proposal seeks to conduct a Phaseregistration trialHPN CTCLevaluating the safety and efficacy ofhypericin in the treatment of CTCLThe Specific Aims of this proposal areTest the hypothesis that in patients with early stage cutaneous T cell LymphomaCTCLtopically applied hypericin in association with standard fluorescent bulb light phototherapy given twice per week forweeks can induce a clinically meaningful improvement in skin lesions when compared to placebo therapyandAssess the utility of repeat treatments of hypericin in early stage CTCL