New CF Combination Drugs From Vertex Address Various CTFR Mutations

This year at the 28th Annual North American Cystic Fibrosis Conference (NACFC) in Atlanta, Vertex Pharmaceuticals Incorporated announced their ongoing efforts to treat the underlying cause of cystic fibrosis (CF) by focusing on mutations in the CFTR gene (short for cystic fibrosis transmembrane conductance receptor) through a combination of drugs.

Data presented at the conference, held in October, included the results of Phase 3 TRAFFIC and TRANSPORT studies. 48-week treatment with lumacaftor (a drug being developed to treat CF patients with a Cftr specific mutation, the delta-F508) in combination with KALYDECO® (ivacaftor) – approved to treat CF patients with more than six years-old worldwide – was shown to improve lung function in CF patients carrying two copies of delta-F508 mutation.

Additional data included the results from the Phase 2 studies where VX-661, a second CFTR corrector, was combined with ivacaftor, a CFTR potentiator, to treat CF patients with one or two copies of the F508del mutation. A new pivotal Phase 3 development program for VX-661 in combination with ivacaftor was also announced to begin in 2015, aiming to evaluate efficacy and safety in people with different combinations of CFTR mutations — two copies of the F508del mutation; one copy of the F508del mutation and a second mutation leading to a gating defect in the CFTR protein; one copy of the F508del mutation and a second mutation that leads to residual CFTR function; and one copy of the F508del mutation and a second mutation resulting in minimal CFTR function.

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Finally, Vertex announced the submission of a New Drug Application (NDA) for ivacaftor to treat CFTR specific mutations — G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P or G1349D – in children  between 2 and 5 years old.

Jeffrey Chodakewitz, M.D., Senior Vice President and Chief Medical Officer at Vertex, commented, “With KALYDECO, we have shown that treating the underlying cause of CF can have significant and sustained benefits for people with the G551D mutation. Our goal is to develop combinations of medicines to treat many more people with CF and to improve the benefit that these combinations of medicines may provide. Data from the Phase 3 studies of lumacaftor in combination with ivacaftor showed consistent evidence of clinical benefit in lung function and other measures of the disease for people with two copies of the F508del mutation and provided important support to conduct further studies of combination regimens aimed at treating people with one and two copies of the F508del mutation.”