Credit: Shutterstock photoVertex Pharmaceuticals IncorporatedVRTX provided a detailed update on its current and planned studies on investigational treatments for cystic fibrosis. The company continues to make efforts towards the development, approval and launch of new medications for the treatment of patients suffering from the disease.
The company is currently looking to expand Kalydeco's label. The drug is already approved for the treatment of cystic fibrosis in patients aged 2 years and above, who have one of the G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, or R117H mutations in their CF gene. Currently, a supplemental new drug application for the drug is under priority review by the FDA for use in patients aged 2 years and above, who have one of 23 residual function mutations with a response by the agency expected by Feb 6, 2016. Kalydeco is also under review in the EU for cystic fibrosis in children aged 2 years to 5 years, who have one of nine gating mutations in their cystic fibrosis transmembrane conductance regulator (CFTR) gene and in patients aged 18 and above, who have the R117H mutation.
Vertex is also planning to study the effect of Kalydeco on markers of cystic fibrosis in children below 2 years of age. A study is expected to start in the first quarter of 2016.
Meanwhile, Orkambi - the Kalydeco/lumacaftor combination - is in two phase III studies for the treatment of cystic fibrosis in patients aged 6 years - 11 years. Pending data from the first study, Vertex intends to submit a supplemental new drug application to the FDA in the first half of 2016. Orkambi is already approved for the treatment of cystic fibrosis in patients aged 12 years and above, who have two copies of the F508del mutation (F508del/F508del) in their CFTR gene.
Apart from Kalydeco and Orkambi, Vertex is advancing two next-generation correctors, VX-152 and VX-440, to phase I studies, to assess them individually and in combination with VX-661/Kalydeco in healthy volunteers. The studies are expected to commence in Nov 2015. Vertex intends to start phase II studies on VX-152 or VX-440 in combination with VX-661/Kalydeco for the treatment of cystic fibrosis in the second half of 2016.
Vertex also has plans to initiate studies on a triple combination of VX-152/VX-661/ivacaftor and VX-440/VX-661/ivacaftor for the treatment of cystic fibrosis patients, who have two copies of the F508del mutation and those who have one copy of the F508del mutation and a second mutation that results in minimal CFTR function in the second half of 2016.
Additionally, the company is enrolling patients in four phase III studies on the combination of VX-661 (100 mg once daily) and Kalydeco (150 mg every 12 hours (q12h)) in multiple groups of patients suffering from cystic fibrosis, who have at least one copy of the F508del mutation.
The updates were provided in the Annual North American Cystic Fibrosis Conference.
Vertex carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the health care sector are Aegerion Pharmaceuticals, Inc. AEGR , Corcept Therapeutics Incorporated CORT and Anacor Pharmaceuticals, Inc. ANAC . All these stocks carry a Zacks Rank #2 (Buy).
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