5:28 PM
Jun 06, 2019
 |  BioCentury  |  Product Development

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

CSO Altshuler on Vertex’s recipe for efficient R&D: set a high bar for early biomarkers and iterate constantly

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.

According to a May report from Leerink analyst Geoffrey Porges, Vertex was able to bring these therapies to market with only a 13% drop out rate for other programs over the last five years, compared with an average drop out rate of 43% among an eight-company peer group.

BioCentury spoke with Altshuler about Vertex’s approach to R&D efficiency.

One key factor is Vertex's disproportionate investment in R&D relative to its market cap peers. From 2016-2018, Vertex invested 46-62% of its revenues in R&D compared to 17-19% for Biogen Inc. and 33-42% for Regeneron Pharmaceuticals Inc.

The money it puts into R&D is not going to SG&A because Vertex focuses on disease areas where the patients have already been diagnosed and are receiving some form of treatment, negating the need to build disease awareness or identify patients and treating physicians.

Altshuler said the biotech uses its investment in R&D to constantly build on what it’s learned, with the goal of each new generation of therapy providing non-incremental benefit over the last. To achieve this, Vertex leverages early biomarkers linked to the underlying disease pathology to predict in Phase I/II whether the molecule will have a big effect size.

"We choose to work in a disease and a...

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