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Hunter Syndrome (MPS II)

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November 7, 2005

Shire Pharmaceuticals announced positive results of their phase II/III "AIM" trial of idursulfase, their enzyme replacement therapy for the treatment of Hunter syndrome. Trial data met their primary composite efficacy endpoint, with subjects receiving the drug weekly experiencing improvements in both 6-minute walk distance and forced vital volume, compared to placebo (p=0.0049). Further, primary efficacy values for subjects receiving the drug once every two weeks were also significant (p=0.0416). Urinary GAG levels, a measure of disease severity, were normalized in 40.6% of subjects receiving one of the two idursulfase regimens, vs. 0% for placebo (p<0.0001). This double-blind, placebo-controlled study enrolled 94 subjects across 9 sites in the UK, US, Germany and Brazil; subjects received 0.5 mg/kg idursulfase once weekly or once every two weeks, or placebo, for 52 weeks.

June 27, 2005

Transkaryotic Therapies reported preliminary results from a phase III trial of I2S (iduronate-2-sulfatase), their enzyme replacement therapy for the treatment of mucopolysaccharidosis II, also called Hunter syndrome. Trial data met their primary endpoint, improving forced vital capacity by 3.4% and six-minute walk distance by 44 meters, vs. placebo (0.8%, 7 meters; p=0.0049). This double-blind, placebo-controlled study enrolled 96 subjects across 9 sites in the US, UK, Germany and Brazil. Subjects received either 0.5 mg/kg I2S weekly or once every two weeks or placebo for 1 year, followed by an open-label extension. Based on these results, Transkaryotic announced plans for NDA and MAA filing for I2S during Q4 2005.

November 24, 2003

Transkaryotic Therapies reported positive results from a phase I/II trial investigating iduraonate-2-2sulfatase (I2S), an enzyme replacement therapy for the treatment of Hunter syndrome. Results showed that treatment with I2S was clinically active in subjects and that their overall activity level, measured by joint mobility and walking, increased. The randomized, double-blind, placebo-controlled study enrolled 12 subjects with Hunter syndrome and was designed to evaluate safety and clinical activity of I2S. Three dosages of I2S (0.15 mg/kg, 0.5 mg/kg and 1.5 mg/kg) were studied. All subjects elected to participate in an open-label extension study. Results were presented at the American Society of Human Genetics 53rd Annual Meeting being in Los Angeles.

September 8, 2003

Transkaryotic Therapies reported positive results from a phase I/II trial investigating iduronate-2-sulfatase (I2S), an enzyme replacement therapy for the treatment of Hunter syndrome, (MPS II). Data demonstrated a significant reduction in urinary glycosaminglycan (GAG) excretion, significant reductions in liver and spleen volumes, as well as reductions in left ventricular mass and improvements in forced vital capacity. Results also showed greater improvements in joint range of motion and the six-minute walk test at one year. The six-month randomized, double blind, placebo-controlled study enrolled 12 subjects and tested three doses of I2S (0.15, 0.5 and 1.5 mg/kg) bi-weekly for 24 weeks. All subjects also elected to enroll in the open-label extension study. Results will be presented in November 2003 at the American Society of Human Genetics 53rd Annual Meeting.

October 21, 2002

Transkaryotic Therapies reported positive results from a phase I/II trial investigating iduronate-2-sulfatase (I2S), an enzyme replacement therapy as a possible treatment for Hunter syndrome. The drug was generally well tolerated and demonstrated evidence of clinical activity. The randomized, double blind, placebo-controlled study evaluated the safety of I2S and its clinical activity in 12 subjects affected with Hunter syndrome. The data demonstrated mean reductions in glycosaminoglycan (GAG) levels, the toxic substrate that accumulates in patients with Hunter syndrome. Mean urinary GAG reductions of 41%, 51%, and 59% were observed from baseline for subjects in the 0.15 mg/kg, 0.5 mg/kg, and 1.5 mg/kg groups, respectively, compared to a 4% increase in the placebo group. I2S also demonstrated a mean reduction of mass in enlarged organs such as liver, spleen and heart. Transkaryotic Therapies believes the data from this trial support the advancement of this program into pivotal clinical testing.

June 17, 2002

Preliminary phase I/II trial results indicate that treatment with Transkaryotic Therapies' iduronate-2-sulfatase (I2S) is generally well tolerated. I2S is an enzyme replacement therapy being developed for the treatment of Hunter Syndrome (also referred to as mucopolysaccharidosis type II). The double-blind, placebo-controlled trial assessed the safety and clinical activity of I2S in 12 subjects with Hunter syndrome. Subjects were randomized to receive placebo or one of three I2S doses (0.15, 0.50 or 1.5 mg/kg) as an intravenous infusion every other week for six months. Results showed reductions in glycosaminoglycan levels, indicating that the enzyme is biochemically active. Reductions in liver and spleen size were also observed, in addition to evidence of clinical activity in pulmonary function and joint mobility.