Table: BMN-701 Phase 1/2 Results as Compared to LOTS Study*
|Baseline||Change from baseline||% Chg||Baseline||Change from baseline||% Chg|
|at Week 24||at Week 26|
|Avg. 6MWT (meters)||354.5||22.3||6.3%||332.2||28.5||8.6%|
|Super Responders||n= 16||3||18.8%||n=60||4||6.7%|
|Baseline||Week 24||Change||% Chg||Baseline||Week 26||Change||% Chg|
|% predicted||from baseline||% predicted||from baseline|
*The LOTS trial (N Engl J Med 2010;362:1396-406) was a randomized, placebo-controlled trial of alglucosidase alfa, recombinant human GAA, for the treatment of late-onset Pompe's disease. Ninety patients who were eight years of age or older, ambulatory, and free of invasive ventilation were randomly assigned to receive biweekly intravenous alglucosidase alfa (20 mg/kg) or placebo. The two primary endpoints were 6-minute walk distance and percentage of predicted forced vital capacity (FVC). Comparison to LOTS study data roughly estimated at week 26 are presented to provide additional information regarding BioMarin's decision making process for the continued development of BMN-701. As the trials were independently conducted and utilized different protocols, no inference should be made about the comparative efficacy of the products tested.