Vertex Pharmaceuticals is looking to acquire “mid- and late-stage assets” to bolster its current research pipeline, CEO Reshma Kewalramani said Monday at the virtual J.P. Morgan Healthcare Conference.
The Boston company also projects more growth for its cystic fibrosis drug business. There are approximately 83,000 cystic fibrosis patients amenable to treatment with Vertex medicines in the US, Europe, Australia, and Canada, based on Vertex’s latest forecasts, an increase of 8,000 patients from its previous estimate.
Vertex medicines, including the recently launched triple-therapy cocktail called Trikafta, can treat 90 percent of cystic fibrosis patients. Vertex did not report financial results on Monday, but analysts are currently expecting sales of $6.2 billion in 2020 and $6.9 billion this year.
Kewalramani’s comments on a more aggressive deal-making approach were the highlight of Monday’s presentation, largely because historically, Vertex has eschewed large M&A deals. Given rising valuations for companies with drugs in the middle and late-stages of clinical development, her comments suggest Vertex may be willing to open the checkbook for multibillion-dollar acquisitions.
With its balance sheet growing with cash from its cystic fibrosis drugs, Vertex now has the “financial firepower” to consider more deals, she said.
Kewalramani made it clear that Vertex was still confident about its current pipeline of experimental medicines, even while acknowledging that not every program would succeed.
Last year, toxicity forced Vertex to shelve an experimental drug for a rare, inherited lung disease called alpha-1 antitrypsin deficiency. But a second-generation drug called VX-684 for the same disease is currently undergoing study in a clinical trial that will read out results in the first half of this year.
Vertex is developing a CRISPR-based, potentially curative treatment called CTX001 for patients with sickle cell disease and beta-thalassemia in partnership with CRISPR Therapeutics. Early, positive results from twin clinical trials were presented in December. On Monday, Kewalramani said “over 20 patients” have now been dosed in those studies and enrollment is expected to be completed in 2021.
Delays in a competing gene therapy program from Bluebird Bio could make it possible for Vertex and CRISPR to catch up or even move ahead with CTX001.
Vertex has also filed the paperwork with the Food and Drug Administration necessary to begin the first human clinical trial of a cell therapy designed to produce insulin-making pancreatic islet cells for patients with Type 1 diabetes. The FDA has not yet cleared the company to start that clinical trial, although that permission is expected this year, Kewalramani said.
Vertex’s research in diabetes comes from its $1 billion acquisition of Semma Therapeutics in 2019. A separate project to develop a combination cell therapy/device for Type 1 diabetes remains in the preclinical stage.