Long-term follow-up of patients from a phase I/II clinical trial supports I2S development. The phase I/II program evaluated I2S (0.15mg/kg, 0.5mg/kg and 1.5mg/kg) in 12 patients with Hunter syndrome in a randomized, double-blind, placebo-controlled clinical trial. Within each dose group, study subjects were randomized to receive either intravenous I2S (3 patients) or placebo (one patient) biweekly for 6 months. All 12 patients successfully completed the six-month double-blind phase and elected to participate in the open-label extension phase. The 6-month therapeutic effects were of a magnitude sufficient for advancement to phase III investigation. 12-month data shows that I2S:

• diminished urinary GAG excretion by an average of 45%
• diminished liver and spleen volumes by an average of 27% and 26%, respectively
• stabilized respiratory capacity (measured by forced vital capacity)
• Improved 6-minute walk test endurance (12% and 28% in the mid- and high-dose groups, respectively)
• improved joint range of motion over several parameters
• administration is associated with reassuring safety, tolerability, adverse effect, and immunological profiles.

Hunter syndrome is a lysosomal storage disease that affects approximately 5,000 people in the U.S. In the condition, the lysosomal enzyme I2S is unavailable for or ineffective in breaking down glycosaminoglycans (GAGs). GAGs then accumulate in relevant tissues, leading to the clinical features of skeletal deformities, obstructive airway disease, cardiac failure, and even neurodegeneration. Depending on the severity of the accumulation, life expectancy ranges from pre-teen to 50-55 years of age. Currently, there is no effective treatment. In 2001, I2S was designated an orphan drug in both Europe and the U.S. Approved products with this designation are entitled to a certain duration of market exclusivity. The E.U. grants up to ten years of marketing exclusivity for serious disorders affecting not more than 185,000 people and the U.S. grants up to seven years exclusivity for disorders affecting less than 200,000 people.

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