Biotech company Pharming Group NV announced that its wholly-owned subsidiary, DNage, has had its Investigational New Drug (IND) application for Prodarsan accepted by the US Food and Drug Administration (FDA), allowing DNage to initiate a clinical trial in children suffering from Cockayne Syndrome (CS).

In this short clinical study, which is expected to be conducted in the fourth quarter of 2009, the pharmacokinetics (PK) of single and multiple escalating doses of Prodarsan will be evaluated in a small number of paediatric patients with CS. The individual components of Prodarsan are already known to the FDA and during this study data will be collected to make a bridge to existing data on the individual components of Prodarsan. This will facilitate the future registration of Prodarsan. In addition to data collected in previous studies, the safety and tolerability of the product will also be investigated in this study. The principal investigator is Dr Edward G Neilan, staff physician at the Children's Hospital Boston/Harvard Medical School, a world-renowned clinician and researcher in the field of Cockayne Syndrome.

Prodarsan is a product in development to delay the progression of CS, a premature ageing disease. Premature ageing diseases are a group of rare diseases caused by a genetic defect leading to deficient repair of DNA damage. Patients suffering from these diseases develop multiple 'ageing-related pathologies', normally associated with old age, early on in their lives. Generally, these patients have a strongly reduced quality of life and reduced life expectancy. Prodarsan is a combination of small molecules formulated as a liquid for oral use and is believed to reduce the accumulation of DNA-damage, the underlying biochemical cause of CS. There is currently no effective therapy available for this disease.

In late 2008, a phase-I study of Prodarsan in healthy human volunteers was successfully completed. In this study, single and multiple escalating doses were administered to healthy volunteers to evaluate the PK, safety and tolerability of Prodarsan in adults. Prodarsan appeared to be safe and well tolerated in dosages that had earlier shown to yield significant beneficial effects in animal models of CS. Since Prodarsan will be used as an oral, liquid formulation the effects of food intake on absorption and elimination of the product were also investigated. Based on these data, combined with data generated in the preclinical program, an oral dosing scheme has been established that will be used in the upcoming studies in paediatric CS patients.

After successful completion of the planned clinical study and the ongoing toxicology testing, DNage expects to perform another clinical trial to evaluate the efficacy of Prodarsan in alleviating symptoms of CS. The final design and set-up of this planned international study will be completed after discussions with regulatory agencies and evaluating the results of the currently planned short clinical study.

Dr Rein Strijker, general manager of DNage and chief commercial officer at Pharming, commented, "Prodarsan, as the first product being developed with DNage technology, is testimony to the great potential of this approach in treating diseases related to ageing in humans. The positive data with Prodarsan in preclinical and the earlier completed phase-I studies are encouraging for its further development. We look forward to initiating studies in CS patients and will also continue to develop a number of other product candidates for the treatment of other premature ageing diseases as well as ageing diseases in the elderly population. With the continued increase in average life-expectancy of humans in many parts of the world, there is a great medical need for new products in the field of diseases associated with old age. We believe that our innovative DNage technology can contribute to develop new approaches in this area."

DNage is a wholly-owned subsidiary of Pharming Group NV since its acquisition in 2006. DNage is developing products that interfere with the development of ageing-related pathologies.