GTC Biotherapeutics (fka GTCB)

[DewDiligence]

Pharming Starts Clinical Program in Ageing Diseases

[Is it just a coincidence that this PR comes out one day after SIRT is acquired by GSK?]

http://biz.yahoo.com/iw/080423/0389641.html

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Wednesday April 23, 5:59 am ET

LEIDEN, NETHERLANDS--(MARKET WIRE)--Apr 23, 2008 -- Pharming Group NV ("Pharming") (NYSE Euronext: PHARM) announced today that its wholly owned subsidiary DNage has started a Phase I clinical study to evaluate the pharmacokinetics and tolerability of Prodarsan® in humans.

Premature ageing, the primary target of Prodarsan®, is a group of rare genetic diseases which manifests itself in several forms that are genetically and clinically similar although not identical. Dependent on the specific form of the disease, patients have a strongly reduced life expectancy and exhibit many ageing-related diseases early on in their lives. There is currently no effective therapy available for these patients.

Pharming has demonstrated that Prodarsan® as an oral, liquid formulation has significant effects in animal models for Cockayne Syndrome ("CS"). CS is one of the more common forms of premature ageing and is characterized, amongst others, by growth failure, mental retardation, eye abnormalities and a reduced life expectancy. The positive effects of Prodarsan® in preclinical testing on life expectancy in general and more specifically on the eye abnormalities are promising for further development of the product and its testing in humans.

The Phase I trial that is now being conducted consists of a combined single and multiple dose escalating clinical study in healthy volunteers. By studying the pharmacokinetics and tolerability of Prodarsan® and the effects of food intake on the absorption and elimination of the product, an oral dosing scheme will be determined that targets the pharmacological effective concentration range effectively. It is expected that following a successful completion of this trial the first clinical studies in patients will start later in 2008.

Prodarsan® is a defined combination of small molecules [?] which is being developed to delay the progression of age-related diseases. Prodarsan® is thought to act by reducing DNA-damage accumulation, which has recently been established as an important element in ageing and the development of ageing related pathology. For instance, patients that suffer from premature ageing usually have a genetic defect that results in insufficient DNA-repair. DNage had previously also shown that defected DNA-repair in animals leads to the development of ageing related diseases such as Osteoporosis and Neurodegeneration.
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