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BioMarin To Advance BMN-701 For Pompe Disease To Next Phase Of Development

Table: BMN-701 Phase 1/2 Results as Compared to LOTS Study*

    POM-001       LOTS  
    BMN-701       Alglucosidase Alfa  
  Baseline Change from baseline  % Chg   Baseline Change from baseline % Chg
    at Week 24       at Week 26  
Endurance:              
 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%
             
    POM-001       LOTS
    BMN-701       Alglucosidase Alfa
  Baseline Week 24 Change % Chg    Baseline Week 26 Change % Chg 
       % predicted from baseline        % predicted from baseline
Pulmonary                  
 FVC 58.1% 59.3% 1.2% 2.0%   55.4% 56.9% 1.5% 2.7%
 MVV (L/min) 67.6 70.6 2.9 4.3   na na na na
 MEP 36.4% 41.6% 5.1% 14.1%   32.0% 34.6% 2.6% 8.0%
 MIP 40.6% 51.6% 11.0% 27.0%   40.0% 45.0% 5.0% 12.5%

*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.

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