Positive Data on Vertex Pharma's Kalydeco - Analyst Blog

Vertex Pharmaceuticals Incorporated VRTX announced positive results from a two-part proof-of-concept study (n=24) on Kalydeco in patients suffering from cystic fibrosis CF who have a residual function mutation.

The first part of the study was a two-week crossover design over two treatment cycles of Kalydeco and placebo. Results from this part revealed that patients under Kalydeco treatment for two weeks showed a significant improvement in mean absolute lung function compared to those under placebo.

The other part of the study was an eight-week open-label design evaluating Kalydeco treatment. Results from the second part of the study revealed an improvement after eight weeks of treatment with Kalydeco. The drug was also well tolerated during the study.

Vertex Pharma intends to start a larger phase III study on Kalydeco in patients with residual function mutations based on results from these studies. The study will commence after the company's discussions with regulatory authorities.

Earlier in the year, Kalydeco was approved in the U.S. for treating patients (age ≥ 6 years) suffering from CF who have one of eight additional mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Following the approval, Kalydeco is now used to treat CF patients with the following nine mutations: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P and G1349D. Kalydeco was earlier approved for treating CF patients (age ≥ 6 years) who have at least one copy of the G551D mutation.

Vertex Pharma has been witnessing encouraging Kalydeco sales since its launch. The company reported Kalydeco sales of $99.5 million in the first quarter of 2014, an increase of 61% from the year-ago period. Kalydeco sales are expected in the range of $470 million to $500 million in 2014.

Kalydeco is being developed for a number of other indications as well. Vertex Pharma plans to get Kalydeco approved for CF patients (aged 12 years and older) with at least one copy of the R117H mutation. Data from two phase III studies (TRAFFIC and TRANSPORT) evaluating Kalydeco plus VX-809 for CF patients with two copies of the F508del mutation is expected in mid 2014. The company also plans to submit regulatory applications in the U.S. and EU for the F508del mutation indication later in 2014, subject to favorable data.

We are encouraged by Vertex Pharma's progress with Kalydeco. Vertex Pharma has a series of Kalydeco related events lined up in the coming quarters. We expect investor focus to remain on the product going forward.

Vertex Pharma carries a Zacks Rank #3 (Hold). Some better-ranked stocks include Allergan Inc. AGN, Gilead Sciences Inc. GILD and Alexion Pharmaceuticals, Inc. ALXN. While Allergan and Gilead carry a Zacks Rank #1 (Strong Buy), Alexion holds a Zacks Rank #2 (Buy).

Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report
 
ALEXION PHARMA ALXN: Free Stock Analysis Report
 
VERTEX PHARM VRTX: Free Stock Analysis Report
 
ALLERGAN INC AGN: Free Stock Analysis Report
 
GILEAD SCIENCES GILD: Free Stock Analysis Report
 
To read this article on Zacks.com click here.
 
Zacks Investment Research