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Study Status: Fully enrolled and data anticipated mid 2014

Study Status: Fully enrolled and data anticipated mid 2014. A second corrector further enhances in vitro F508del CFTR function. F508del/F508del. F508del/G542X. Kindly provided by Vertex Pharmaceuticals, Inc. Strategic planning for back-up correctors began 4 years go.

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Study Status: Fully enrolled and data anticipated mid 2014

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  1. Study Status: Fully enrolled and data anticipated mid 2014

  2. A second corrector further enhances in vitro F508del CFTR function F508del/F508del F508del/G542X Kindly provided by Vertex Pharmaceuticals, Inc.

  3. Strategic planning for back-up correctors began 4 years go • Reviewed lessons learned from first generation correctors • Created road map for more robust second generation compounds • Strong partners in place • Amazing progress • Novel screens developed • Up to 6 million compounds will be reviewed

  4. How Much CFTR is Enough?The Ivacaftor – G551D Benchmark Study Baseline 150 mg Adapted from Accurso et al New Engl J Med 2010

  5. FEV1 % Predicted Absolute Change from Baseline Treatment effect through Week 24 + 10.6 % P < 0.0001 Treatment effect through Week 48 + 10.5 % P < 0.0001 Ramsey et al., N Engl J Med. 2011 Nov 3;365(18):1663-72

  6. Change from Baseline in Sweat Chloride Treatment effect through Week 24 – 47.9 mmol/L P < 0.0001 Treatment effect through Week 48 – 48.1 mmol/L P < 0.0001 Ramsey et al., N Engl J Med. 2011 Nov 3;365(18):1663-72

  7. The ivacaftor effect persists for many months Open Label Follow-On See: McKone et al. NACFC 2013 Poster #227

  8. Effect of 150 mg BID ivacaftor on hospitalization rate in G551D patients From the GOAL presentation and kindly provided by S. Rowe

  9. Ivacaftor coverage of other gating mutations • In vitro studies have shown that ivacaftor improves chloride transport in CF cells with other CFTR gating mutations1 • G178R, S549N, S549R, G551S, G970R, G1244E, S1251N, G1349D, S1255P • KONNECTION Study: Blinded, placebo-controlled 8 week crossover study of ivacaftor in other CFTR gating mutations with open label extension2 • At 8 weeks, FEV1 change from baseline favored ivacaftor treatment by 10.7% predicted (P < .0001) • Comparable to ivacaftor treatment effect seen at 24 weeks in G551D patients (10.6% predicted, P < .0001) Study Status: Crossover portion complete, supplemental New Drug Application filed 1- Yu et al. J Cyst Fibros. 2012;11(3):237-45. 2- DeBoeck et al. NACFC 2013 Symposium 3.15 and Poster #241 Kindly provided by Vertex Pharmaceuticals

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