Vertex shifts focus to cystic fibrosis R&D

Vertex Pharmaceuticals is shifting its investment strategy to focus on opportunities in cystic fibrosis, according to CFO Ian Smith, who spoke at the Credit Suisse 2013 Healthcare Conference in mid November.

A few weeks earlier, Vertex Pharmaceuticals announced a 15% workforce reduction associated with support of its hepatitis C drug telaprevir (Incivik) because new medicines for this therapeutic area are nearing approval. The company restructuring was necessary to lower operating expenses, with an expected 2014 savings of $150 million to $250 million, Smith said.

Over the next 3 to 6 months, Vertex expects to review monotherapy data from two phase 3 studies for ivacaftor (Kalydeco), a drug already FDA-approved for cystic fibrosis patients with the G551D mutation in the CFTR gene, which accounts for 4% to 5% of cases of cystic fibrosis. Vertex hopes to expand its ivacaftor label to include cystic fibrosis patients with the R117H-CFTR mutation and pediatric patients ages 2 through 5 years with CFTR gating mutation.

“All this data would potentially increase the patient population to treat from 2,000 to 7,000 patients,” Smith noted.

Vertex also is studying combination therapy of ivacaftor with lumacaftor in patients 12 years and older with cystic fibrosis who are homozygous for the F508del mutation. “We should have the data by the middle of next year,” he said. “This would significantly open up the number of patients that we may be able to treat. This would open up the patient population to another 28,000 patients worldwide.”

Vertex is continuing R&D efforts in areas of oncology, oral therapies for hepatitis C, and other areas as well, Smith said.