Skip to content

Vertex Pharmaceuticals: Cystic Fibrosis Presentation Slides

June 19, 2013

Vertex Pharmaceutials’ Cystic Fibrosis (CF) presentation slides from the 36th European Cystic Fibrosis Society (ECFS) Conference, held June 12 – 15, 2013, in Lisbon, Portugal, are available for FREE download (see below). Abstracts from the meeting are available for download from the Journal of Cystic Fibrosis.

The most common mutation in the CF transmembrane conductance regulator (CFTR) gene, is the F508del mutation. Approximately 70,000 people worldwide have CF, including 30,000 in the United States and 35,000 in Europe. Globally, nearly half of the people with CF have two copies of the F508del mutation and an additional one-third have one copy of the F508del mutation. The two presentation slides below are for two Phase II studies in CF patients who have the two copies of the F508del mutation.

I – VX-661 In Combination With Kalydeco

This oral presentation includes previously announced results from this study as well as additional data on the pattern of FEV1 (Forced Expiratory Volume in one second) response. This is a Phase II Study of VX-661 + Kalydeco for CF patients with 2 copies of the F508del mutation. The conclusions of the data are :

•   “Reductions in sweat chloride were observed with both VX-661 alone and in combination with Ivacaftor
•   Improvements in pulmonary function (FEV1) were observed at all combination doses studied, and were greatest at the VX-661 100mg and 150mg dose levels
•   VX-661 alone and in combination with Ivacaftor was generally well tolerated
•   These data support the concept of a combined corrector and potentiator approach for treatment of patients who are homozygous for the F508del-CFTR mutation.”

The following is the citation and link for a FREE download of the PDF File for the presentation :

•   Lead study investigator Scott Donaldson, M.D., Associate Professor of Medicine, University of North Carolina at Chapel Hill, presented on June 13, 2013, “VX-661, an investigational CFTR corrector, in combination with Ivacaftor, a CFTR potentiator, in patients with CF and homozygous for the F508Del-CFTR mutation: Phase 2 interim analysis”.

II – Lumacaftor (VX-809) In Combination With Kalydeco

This oral presentation includes previously announced results from Cohorts 2 and 3 of the study as well as additional pharmacokinetic data. Also, an additional analysis of pooled lung function data during the combination dosing portions of these cohorts is presented. The purpose of Cohort 2 is to evaluate Lumacaftor’s dose range. The purpose of Cohort 3 is to evaluate the safety and pharmacokinetics of Lumacaftor 400 mg q12h (take every 12 hours) for potential inclusion in Phase 3 studies. The conclusions of the data are :

•   “For F508del homozygotes: Lumacaftor 600 mg once-daily or 400 mg every 12 hours with Ivacaftor 250 mg every 12 hours for 28 days led to statistically and clinically significant improvements in FEV1
•   Lumacaftor in combination with Ivacaftor was generally well tolerated, and the safety profile was similar to placebo
•   Based on these findings, two Phase III confirmatory studies, TRAFFIC and TRANSPORT, have been initiated to evaluate the effects of Lumacaftor in combination with Ivacaftor in patients homozygous for F508del.”

The following is the citation and link for a FREE download of the PDF File for the presentation :

• Lead study investigator Michael P. Boyle, M.D., F.C.C.P., Associate Professor, Director of the John Hopkins Adult Cystic Fibrosis Center, presented on June 13, 2013, “Lumacaftor, an investigational CFTR corrector, in combination with ivacaftor, a CFTR potentiator, in CF patients with the F508Del-CFTR mutation: Phase 2 interim analysis”.

III – Upcoming Events

  1. CF Unite Webinar, “CF Genes & Gene Therapy”, June 29, 2013
  2. CFRI 26th National Cystic Fibrosis Family Education Conference, August 2-4, 2013, Redwood City, California
  3. 10th Australasian Cystic Fibrosis Conference, August 17-20, 2013, Auckland, New Zealand
  4. Worldwide Cystic Fibrosis Day, September 8, 2013
  5. 27th Annual North American Cystic Fibrosis Conference, October 17-19, 2013, Salt Lake City, Utah.

Please Note: “Icon Announcer” by Orion 8 [Public domain] | Wikimedia Commons.

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

Advertisements
Leave a Comment

Leave a Reply

Fill in your details below or click an icon to log in:

WordPress.com Logo

You are commenting using your WordPress.com account. Log Out / Change )

Twitter picture

You are commenting using your Twitter account. Log Out / Change )

Facebook photo

You are commenting using your Facebook account. Log Out / Change )

Google+ photo

You are commenting using your Google+ account. Log Out / Change )

Connecting to %s

%d bloggers like this: