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On May 13, 2012 BioMarin provided the IPA with the following update on their clinical trial investigating the safety and efficacy of BMN 701.   

As you will read below, the trial will consist of three dosing groups: 5, 10 and 20 mg/kg.  The 5 and 10 mg/kg groups have been fully enrolled, and the 20 mg/kg group is currently enrolling.  

For more information about the BioMarin trials please contact Sean McCarthy of BioMarin at smccarthy@bmrn.com. The IPA is also available to help answer any questions or concerns. Thank you.

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May 13, 2012

BMN 701 is an experimental agent currently under clinical development for Pompe Disease by BioMarin Pharmaceutical Inc. (BioMarin). BMN 701 contains a unique peptide-tagged form of recombinant human GAA (rhGAA). BioMarin is conducting clinical studies to find out whether rhGAA can increase enzyme delivery to lysosomes; the technique is called Glycosylation Independent Lysosomal Targeting (GILT). The peptide tag on BMN 701 is the insulin-like growth factor-type 2, a naturally occurring protein with high affinity for the M6P receptor, the receptor that recognizes rhGAA.

POM-001 is a phase I/II open-label dose-finding study of the safety and tolerability of BMN 701 in patients with late-onset Pompe Disease that has been open approximately 1 year. Patients are treated by intravenous infusion every 2 weeks for up to 24 weeks. Three dose levels of BMN 701 are being investigated (5, 10 & 20 mg/kg). The study's primary endpoint is to determine the immune response to the drug. Secondary endpoints include pharmacokinetics, and preliminary efficacy assessments. A preliminary report on the POM-001 study was presented by Dr. Bruce Barshop (University of California, San Diego) in February at the 8th Annual WORLD Symposium for Lysosomal Storage Disorders. So far, six patients have been treated at the 5 & 10 mg/kg dose levels for up to 24 weeks. The first subject began dosing at the 20 mg/kg dose level in January 2012 after an independent review board permitted dose escalation. UK study sites were opened in February and March of this year.

Details of the study and enrolling sites can be found at https://www.clinicaltrialsregister.eu/ctr-search/search?query=POM-001 and http://clinicaltrials.gov/ct2/show/NCT01230801?term=pom-001&rank=1. There are plans to open additional sites in France, Germany, Italy and Australia later this spring and summer.

On completion of 24 week participation in POM-001, patients may transition to extension protocol POM-002. Details can be found at: http://clinicaltrials.gov/ct2/show/NCT01435772?term=pom-002&rank=1.