Pharmaceutical
Prodarsan:
Pre-clinical experiments with the active ingredients of Prodarsan® have shown a significant improvements in Cockayne Syndomre (“CS”) animal models which mimic the disease in many aspects. No toxicity related to the administration of Prodarsan® was noted in GLP toxicity studies in juvenile rats and dogs, even when administered up to ten-fold the maximum intended daily treatment dose for CS patients. A Phase I study has succesfully been completed in which healthy adult volunteers were exposed to Prodarsan® for up to 14 days without any tolerability issues or any clinically relevant changes.
The efficacy of Prodarsan® in pre-clinical studies and the lack of any observed toxicity provide a strong basis to further develop Prodarsan for the treatment of Cockayne syndrome in man. This was underlined by the US FDA, who granted Orphan Drug Designation to Prodarsan® for the treatment of Cockayne syndrome in April 2009. DNage is currently planning a Phase I/II clinical study in patients and intends to start a Phase III study towards the end of 2010. Although CS and related disorders are rare diseases, there are currently no treatments available and the market size is estimated to be larger than € 50 million per year.
CNS products
The technology platform of DNage has resulted in animal models that develop neurodegenerative diseases caused by insufficient repair of DNA-damage. These models can serve as models for important diseases as Alzheimer and Parkinson disease. DNage is currently screening novel compounds in its proprietary models and cell-lines derived from them. These compounds are selected to delay the progressions of these diseases.
Osteoporosis
DNage owns animal models that predictively and spontaneously develop osteoporosis (both females and males) in the context of overall ageing. These models can be used to validate biomarkers and targets as well compounds which may delay the development of osteoporosis.