28th Oct 2005 14:15
Shire presents positive results of Hunter syndrome pivotal clinical trial atThe American Society of Human Genetics Annual MeetingSalt Lake City, Utah, October 28, 2005 -- Shire Pharmaceuticals Group plc (LSE:SHP, NASDAQ: SHPGY, TSX: SHQ) announced today at the Annual Meeting of TheAmerican Society of Human Genetics, further results of its pivotal clinicaltrial evaluating its investigational human enzyme replacement therapy,idursulfase, for the treatment of Hunter syndrome.The primary efficacy endpoint of the trial was a composite of two clinicalmeasures - forced vital capacity (FVC) and 6-minute walk test (6MWT). Aspreviously reported, patients receiving the weekly dosing regimen of 0.5 mg/kgof idursulfase showed a statistically significant difference (p=0.0049)compared to placebo. Results presented today by Joseph Muenzer, M.D., Ph.D., ofthe University of North Carolina at Chapel Hill, indicated that patientsreceiving the every other week dosing regimen of idursulfase also showed astatistically significant difference (p=0.0416) compared to placebo whenmeasuring the composite. Treatment with idursulfase was generallywell-tolerated by patients in the trial.Other results presented today included urine GAG levels, liver and spleenvolume, cardiac left ventricular volume, and joint range of motion.Commenting on the trial results, Dr. Muenzer said "Data from this study arevery encouraging for patients and families living with Hunter syndrome. I amexcited about the potential of idursulfase as the first treatment option forpatients, once approved."As previously announced, Shire expects to file for regulatory approval ofidursulfase in both the United States and Europe in the fourth quarter of 2005.Trial DesignThe AIM study ("Assessment of I2S in MPS II") was a Phase II/III double-blind,placebo-controlled clinical trial conducted at nine sites around the world,including the United States, the United Kingdom, Germany and Brazil. Theprimary goal of the study was to evaluate the safety and efficacy of 0.5 mg/kgof idursulfase administered weekly compared to placebo. Additionally, the trialevaluated 0.5 mg/kg of idursulfase every other week compared to placebo.Ninety-six patients with Hunter syndrome were randomized to one of three groupswith each patient receiving a total of 52 infusions of either idursulfase,idursulfase alternating weekly with placebo, or placebo. Of the 96 whoenrolled, 94 completed the 52 week study and they all elected to participate inthe open-label extension study of idursulfase at a dose of 0.5 mg/kg weekly.Trial ResultsSix minute walk test (6MWT)In the weekly dosing regimen, the difference in meters walked was 35 meterscompared to placebo (p=0.0131) and in the every other week idursulfase group,the difference in meters walked was 24 meters, compared to placebo (p=0.0732).Forced Vital Capacity (FVC)Model adjusted percent predicted FVC in the weekly group was 4.3% greatercompared to placebo (p=0.0650); the every other week group showed no treatmentdifference in percent predicted FVC over placebo (p=0.9531).Urinary GAG LevelsUrinary GAG levels were significantly lower in patients being treated withidursulfase in both the weekly or every other week dosing regimen compared topatients receiving placebo (pShire Pharmaceuticals Group PLCRelated Shares:
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