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

Stocks in this article: BMRN

"We are excited by the findings of our first study of BMN-701 in previously untreated Pompe patients. We observed meaningful improvements in mean 6-minute walk distance and very substantial walk improvements for some patients, plus significant improvements in respiratory muscle strength. Importantly, there were improvements in one or more walk or respiratory domains in nearly all patients. Based on discussions with Pompe KOLs who have reviewed this data, we believe there will be broad interest to participate in our planned Phase 2/3 study," said Hank Fuchs, M.D., Chief Medical Officer of BioMarin. 

Next Phase of Development: Phase 2/3 Study

The company expects to initiate a Phase 2/3 switching trial by the end of 2013 in late-onset Pompe patients who have previously been treated with alglucosidase alfa. Subject to discussions with health authorities, the proposed study design is a single arm trial, with treatment at 20 mg/kg administered every other week for 24 weeks. The company intends to use efficacy as measured by the respiratory parameter MIP as the primary endpoint. Secondary objectives include MEP and six-minute walk test, as well as safety. The study will be conducted with full scale material from a revised manufacturing process, which has improved process robustness and increased productivity.

POM-001 Phase 1/2 Study Design

The Phase 1/2 trial is an open-label study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamic and clinical activity of BMN-701 administered as an intravenous infusion every two weeks at doses of 5 mg/kg, 10 mg/kg and 20 mg/kg. The company enrolled 22 patients between the ages of 18 and 65 years old with late-onset Pompe disease for a treatment period of 24 weeks.

Conference Call Details

BioMarin will host a conference call and webcast today, Tuesday, March 19, 2013 at 5:00 p.m. ET. This event can be accessed on the investor section of the BioMarin website at www.BMRN.com.

Date: March 19, 2013 Time: 5:00 p.m. ET

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