How early biomarkers and constant iteration fuel Vertex’s R&D efficiency

As Vertex prepares its fourth NDA for a cystic fibrosis drug in six years, EVP of Global Research and CSO David Altshuler credits the biotech’s R&D efficiency to a focus on agents that show more than incremental benefit on early biomarkers, and constant iteration. Vertex is continuing this strategy in new disease areas and hoping for similar success as it starts to get POC this year in AAT deficiency or hemoglobinopathies.

On May 30, Vertex announced results from its triple combination therapy to treat patients who harbor at least one Δ508 mutation and one minimal function mutation, and said it plans to submit an NDA to FDA next quarter. If approved, the triple therapy would treat up to 90% of CF patients, a massive increase over the 4% of patients who were able to benefit from the initial indication approved for Vertex’s first CF drug Kalydeco ivacaftor. The triplet, which contains Kalydeco, also has a greater benefit on lung function than Kalydeco monotherapy. ...