A promising pipeline

Some investors lost interest in biotechnology company Vertex Pharmaceuticals (Nasdaq: VRTX) after it discontinued a clinical trial, but there’s still a lot to like about the company.

It has a big opportunity treating cystic fibrosis (CF) with its newest blockbuster CF drug, Trikafta, which is launching as Kaftrio in parts of Europe and should meet pent-up demand. The company’s other CF drugs may also win approval to treat younger children and patients with additional genetic mutations, giving it a clear growth runway for years to come.

Meanwhile, Vertex has clinical-stage programs in five other disease areas. One of those programs, VX-864, targets the genetic disease alpha-1 antitrypsin deficiency. Vertex’s pipeline also includes promising therapies targeting rare diseases such as sickle cell disease, beta thalassemia and focal segmental glomerulosclerosis (FSGS); several of these programs have important data readouts on the way by the end of next year. The company also hopes to advance its Type 1 diabetes candidate into early-stage testing in 2021. CEO Reshma Kewalramani has noted, “not all of the molecules will succeed,” adding, “not all of them have to.”

Finally, Vertex ended its third quarter with cash, cash equivalents and marketable securities totaling $6.2 billion, giving it a lot of financial flexibility for additional dealmaking. This is a company worth watching — or investing in.