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Vertex Pharmaceuticals, Orphan Drug Kalydeco, And The J.P. Morgan Healthcare Conference

January 9, 2013

On Monday, January 7, 2013, 10:00 AM PST/1:00 PM EST, Vertex Pharmaceuticals gives a presentation at the J.P. Morgan Healthcare Conference in San Francisco. The presentation is given by Vertex Pharmaceuticals’ President & CEO, Jeff Leiden.

Here are a few highlights from the presentation for orphan drug Kalydeco (Ivacaftor) for the treatment of Cystic Fibrosis (CF) :

“Personalized Medicine” Strategy For Kalydeco Addressing CF Gene Mutations

1) Expand Kalydeco monotherapy :

– Goal of 10 – 15% of CF population

– Handling the R117H mutation

– Children 2 – 5 years of age

– Residual CFTR function

– New FDA Breakthrough Therapy Designation.

2) Combination therapy for F508del CF patients :

– Goal of 50% of CF population

– VX-661 + Kalydeco Phase 2

– VX-809 + Kalydeco Phase 3

– New FDA Breakthrough Therapy Designation.

3) Treat F508del heterozygous and increase benefit for all.

1st Half Of 2013 Expected Milestones For Kalydeco

  1. England reimbursement
  2. New FDA Breakthrough Therapy Designation for Kalydeco
  3. New FDA Breakthrough Therapy Designation for VX-809 + Kalydeco
  4. Initiation of Phase 3 for VX-809 + Kalydeco
  5. Phase 2 Data from VX-661 + Kalydeco.

2nd Half Of 2013 Expected Milestones For Kalydeco

  1. Data from Phase 3 Kalydeco label expansion studies for R117H & other gating
  2. Data from n-of-1 POC study (phenotypic).

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