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Kalydeco Combo Study Shows Strong Results

April 19, 2013

Vertex Pharmaceuticals announces April 18, 2013, that in a Phase II Study of VX-661 in combination with orphan drug Kalydeco (Ivacaftor), there is a statistically significant improvement in lung function in adults with Cystic Fibrosis (CF). The Study’s CF patients have two copies (homozygous) of the most common mutation in the CFTR gene, F508del.

The Phase II Study is a randomized, double-blind, placebo-controlled, 3-part study to evaluating the safety, efficacy, pharmacokinetics, and pharmacodynamics of :

•    VX-661 monotherapy
•    VX-661/Kalydeco combination therapy

for CF patients who have two copies of the F508del-CFTR mutation. The study evaluates four dose levels of VX-661 (10, 30, 100, & 150 mg) dosed once daily for 28 days, in combination with Kalydeco (150 mg) dosed twice daily. Also, the study evaluates a different group of patients who receive VX-661 (10, 30, 100, & 150 mg) dosed without Kalydeco for 28 days. The results show that :

treatment with a combination of VX-661 and Kalydeco for 28 days in the two highest dose groups (100 & 150 mg), results in mean relative increases in lung function (percent predicted FEV1) of 9.0% (p=0.01) and 7.5% (p=0.02) versus placebo. VX-661 is generally well-tolerated alone and in combination with Kalydeco.”

Thus, the improvement in lung function is dose dependent, with the greatest improvement in groups that receive the highest doses of VX-661 (100 & 150 mg) in combination with Kalydeco. Per, Vertex Pharmaceuticals gains 51% in after-hours trading as a result of the announcement.

Vertex Pharmaceuticals currently has three compounds in development :

1)    VX-809 is being evaluated in combination with Kalydeco as part of two ongoing Phase III studies expected. Approximately 1,000 people, ages 12 and older with two copies of the F508del mutation, are expected to enroll. Vertex Pharmaceuticals expects to obtain 24-week safety and efficacy data from these studies, and to submit a New Drug Application (NDA) to the FDA in 2014, pending study results

2)    VX-661 will be in additional studies to further evaluate its potential for late-stage development, pending regulatory discussions

3)    VX-983 is being evaluated as part of a Phase 1 multiple-ascending-dose study in healthy volunteers. In the second half of 2013, Vertex Pharmaceuticals plans to begin a 28-day study of VX-983 in combination with Kalydeco in patients with two copies of the F508del CFTR mutation.

The initial approval of Kalydeco is for CF patients, ages 6 and older with only the G551D gene. Approximately 4 % of the global CF population have the G551D gene. By seeking regulatory approval for CF patients who have two copies of the F508del mutation, this will increase the CF patients who qualify for treatment with Kalydeco to nearly 50% of about 70,000 global patients.

In January 2013, Kalydeco receives the first two FDA Breakthrough Therapy Designations for :

•   Monotherapy for potential additional indications beyond the current approval for CF patients 6 and older with the G551D mutation
•   Combination therapy with VX-809.

References Phase II Study of VX-661 Alone and in Combination With VX-770 (Kalydeco) in Subjects Homozygous to the F508del-CFTR Mutation

Vertex Pharmaceuticals Pipeline

Vertex Pharmaceuticals April 18, 2013 Webcast for update on Phase II Study of VX-661 + Kalydeco.

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

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

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