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Highlights from BioMarin's Research & Development Day
Date:12/8/2011

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  • Successful Type C meeting with FDA
  • Reduction in Phe is an acceptable primary endpoint for Phase III
  • Pivotal trial can be conducted in diet non compliant patients
  • Phase 2 Update
  • Weekly dosing (Part A and B): Good safety profile and substantial Phe reduction achieved in the majority of patients continuing in the study at a median maintenance dose of 1.0 mg/kg/week
  • Daily dosing (Part C): Initial rapid Phe reduction achieved in all patients but not sustained in the majority of patients; joint pain, fever and/or rash reliably ten days after study start
  • Initiating Part D of Phase 2 study to find the quickest and safest induction dosing regimen to an efficacious maintenance dose.  The optimal chronic regimen will be one subcutaneous injection per day.
  • Immunology Update
  • A daily dosing induction schedule leads to higher levels of anti PEG IgM titers and both higher and earlier PAL IgG titers compared to weekly dosing
  • Ways to attenuate the hypersensitivity reactions: Limit dosing when IgM responses are highest and titrate to efficacious doses once the IgG response has developed
  • Some patients do not mount a robust IgM Response to PEG and appear to have sustained efficacy and good tolerance
  • BMN-673 for Genetically-Defined Cancers

  • Dose escalation in Phase 1/2 study in solid tumors continuing after 5 steps (25-400 μg/once per day)
  • PK analysis is ongoing: Preliminary data indicate long terminal half-life
  • Once daily oral dosing is feasible
  • Preliminary results are encouraging in terms of toxicity and signs of efficacy
  • BMN-111 for Achondroplasia

  • Demonstrated benefits in moderately and severely affected animal models
  • Positive therapeutic index predicted in vivo
  • Phase 1 clinical trial in healthy volunteers to begin 1Q 2012
  • Phase 2 proof of concept trial in affected pedi
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  • SOURCE BioMarin Pharmaceutical Inc.
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