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Vertex Pharmaceuticals: Kalydeco’s Success Continues

July 30, 2013

Vertex Pharmaceuticals has a conference call on July 29, 2013, to review the 2nd Quarter 2013 financial results and recent progress and upcoming milestones in clinical development programs for Cystic Fibrosis (CF) and Hepatitis C (HCV). Investors, CF patients, and CF advocacy groups are watching closely to see what the status and finances are for the commercialization of orphan drug Kalydeco (Ivacaftor) for the treatment of CF.

Vertex Pharmaceuticals’ 2nd Quarter 2013 earnings conference call reports net product revenues of $99 million for Kalydeco, compared to $61.8 million for the 1st Quarter 2013 – an increase of $37.2 million dollars or an increase of about 60% over the 1st Quarter 2013 figures.

Updates to the CF Development Program are presented :

I – Phase III Label-Expansion Study of Kalydeco Monotherapy in Gating Mutations

•   In a second July 29, 2013 Press Release, Vertex Pharmaceuticals announces that the data from the Phase III study, in CF patients who have at least 1 non-G551D CFTR gating mutation, shows statistically significant improvements in lung function

•   Based on this data, Vertex Pharmaceuticals plans to submit a supplemental New Drug Application (sNDA) in the US & a Marketing Authorization Application (MAA) variation in Europe in the 2nd half of 2013

•   Approximately 400 people, ages 6 and older, have a non-G551D gating mutation.

II – Two Additional Phase III Label-Expansion Studies for Kalydeco Monotherapy

  1. CF patients ages 6 and older who have at least 1 copy of R117H mutation :

•   Study data expected in 2nd half of 2013
•   Pending study results, plans to submit an sNDA in early 2014.

2.  CF patients, ages 2-5, who have a gating mutation, including the G551D mutation

•   Pharmacokinetics portion of study is complete
•   24-week dosing period portion of study is underway
•   Study data expected in mid-2014.

III – Phase II Proof-Of-Concept Study For Residual CFTR Function

•   Enrollment is ongoing

•   Study data is expected in 1st half of 2014.

IV – Phase III Registration Program For VX-809 In Combination With Kalydeco

  1. Study to evaluate combination therapy in CF patients with 2 copies of the most common mutation, F508del

•   Two 24-week studies are ongoing in CF patients, ages 12 and older
•   Enrollment expected to be completed in 2nd half of 2013
•   Pending study results, plans to submit a NDA in US in 2014
•   Approximately 50% of CF patients have 2 copies of F508 mutation.

2.  Study to evaluate combination therapy in CF patients with 1 copy (heterozygous) of the F508del mutation

3.  Pharmacokinetics & safety evaluation of combination therapy in children, ages 6-11, with 2 copies of F508del mutation.

V – Advancing Multiple 1st- And 2nd- Generation Correctors

  1. VX-661:

•   Phase II evaluation of a 4-week regimen of VX-661 in combination with Kalydeco in CF patients with 1 copy of the F508del mutation & 1 copy of the G551D mutation

•   Is 1st proof-of-concept study of a combination of a corrector and Kalydeco with G551D mutation

•   Based on in vitro data presented at ECFS, increased chloride transport in human bronchial epithelial cells with 1 copy of F508del mutation & 1 copy of G551D mutation, after treatment with corrector & Kalydeco, as compared to Kalydeco monotherapy

•   Goal is to evaluate multiple 1st-generation correctors (VX-661 & VX-983), in combination with Kalydeco to find regimens  beneficial to CF patients with F508del mutation.

      2.  2nd-generation correctors

•   Goal is to advance 2nd-generation correctors to be used as part of a future dual-corrector regimen in combination with Kalydeco in CF patients with 1 or 2 copies of the F508del mutation.

Vertex Pharmaceuticals updates its financial guidance for total 2013 Kalydeco net revenues. The company expects total 2013 Kalydeco net revenues to be $345-$360 million compared to the prior range (April 2013) of $300-$340 million.

Outside of the positive news on Kalydeco, Vertex Pharmaceuticals did not report positive numbers for the company’s Hepatitis C drug Incivek :

•   Reports 5th straight quarter of losses
•   Slumping sales
•   Revenue declines 52% to $155.8 million.

Please Note: “Application Certification” by the people from the Tango! Desktop Project [Public domain] | Wikimedia Commons.

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

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