Vertex Reveals Promising Data for Triple Combination Cystic Fibrosis Therapies

Vertex Pharmaceuticals is securing its spot leading the treatment space for cystic fibrosis (CF).

The company unveiled on Tuesday positive data from Phase I and Phase II studies for three different triple combination regimens for CF patients with certain types of gene mutations with one F508del mutation and one minimal function mutation (F508del/Min).

The drugs are labeled VX-659, VX-152, and VX-440, respectively.

VX-659 was tested in a randomized, double-blind, placebo-controlled study where investigators evaluated the safety and tolerability of single and multiple ascending doses of VX-659 alone as well as in triple combination with tezacaftor and ivacaftor in healthy volunteers. Also, the researchers explored the safety and tolerability of VX-659 as a component of a triple combination for two weeks in 12 patients with CF that were ages 18 and older with one F508del mutation and one minimal function mutation (3 in placebo and 9 in VX-659 120 mg), according to the announcement.

Results from this trial indicated the drug was generally well tolerated as part of the triple combination with tezacaftor and ivacaftor. Majority of adverse events were mild or moderate, but the data had shown there was an absolute improvement in ppFEV1 of 9.6 percentage points from the baseline in individuals receiving the triple combination of VX-659 (120mg q12h), tezacaftor, and ivacaftor.

VX-152 and VX-440 were tested in two small phase II studies. Patients who received 200 mg twice a day of VX-152 experienced a 9.7 percentage point improvement in ppFEV1 in volume of air exhaled in one second. Patients who were administered 600 mg of VX-440 twice a day as part of the triple combination experienced an improvement by 12 percentage points, reported Reuters.

“Patients with minimal function mutations have been waiting for a medicine to treat the underlying cause of their disease, which makes these data showing pronounced improvements in lung function particularly important,” said Steven M. Rowe, M.D., M.S.P.H, a professor of medicine leading steering committee comprised of CF experts for Vertex, in a statement. “It’s also encouraging to see that the addition of a next-generation corrector may lead to substantial additional benefits for patients with two copies of the F508del mutation, who were already receiving tezacaftor and ivacaftor.”

Vertex plans on gathering and evaluating more data from these studies and other experiments in order to advance further down the clinical development path by the first half of 2018.