Biotech company Pharming Group NV ("Pharming" or "the Company") (NYSE Euronext: PHARM) announced today that its wholly-owned subsidiary DNage has initiated an observational study in children suffering from Cockayne Syndrome (CS).
This study is designed to assess the natural (untreated) progression of the disease during a period of 6-12 months and will include detailed data collected from existing medical records as well prospective measurements and assessments. Data collected in the observational study will enable the Company to conduct one or more clinical studies to assess the efficacy of Prodarsan®, a product in development for the treatment of CS. Although the final design of these planned international efficacy studies is not yet completed and also dependent on the outcome of discussions with regulatory authorities, it is likely that in these studies Prodarsan® will be tested, amongst other parameters, for its effect on growth of paediatric patients compared to their growth when untreated.
DNage is developing Prodarsan® as a potential therapy for CS. The product is a combination of small molecules formulated formulated as an oral liquid and is believed to reduce the accumulation of DNA-damage, the underlying biochemical cause of Cockayne Syndrome. Prodarsan® has been successfully tested in animal models of CS and appeared to be safe and well-tolerated in a Phase I study in healthy human volunteers. Prodarsan® has an Orphan Drug Designation from the US Food and Drug Administration (FDA) who recently also approved an Investigational New Drug (IND) application allowing the Company to conduct a clinical trial in children suffering from CS.
The Principal Investigator of the observational study, Dr. Edward G. Neilan, Staff Physician at the Children's Hospital Boston/Harvard Medical School, commented: "The initiation of this observational study is an important milestone in the development of new treatment options for CS-patients and in particular for the development of Prodarsan®. For the first time, we will be collecting detailed measurements of the natural progression of this disease. This study will hopefully provide us with data needed to complete the design of a pivotal clinical study necessary to achieve registration of Prodarsan® as a therapeutic. While our center in Boston will serve as a central site in the current study, I look forward to collaborate with many colleagues in the US and abroad to make this study a success."
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