Vertex Picks ‘Impressive’ Cystic Fibrosis Drugs To Aim For A Multi-billion-Dollar Market

Vertex Pharmaceuticals has picked two cystic fibrosis drugs to test in combination with its existing CF medicine, Kalydeco, as part of two different three-drug combinations aimed at helping as many as 90% of patients with cystic fibrosis. (Vertex’s current offerings can treat half.)

Michael Prince for Forbes
Vertex Pharmaceuticals CEO Jeff Leiden is about to advance a pair of potential blockbusters into clinical trials

One new drug, VX-659, increased lung function, measured as forced expiratory volume in one second, of 13.3 percentage points after four weeks of treatment. A second, VX-445, increased expiratory volume by 13.8 percentage points. In both cases, the new drug was used with Kalydeco and tezacaftor, another experimental drug, in patients with CF caused because of one mutation called F508del and one minimal function mutation. The results are in line with earlier results on these drugs that were presented in July.

“The efficacy data is incredibly impressive,” says Bonny Ramsey, Director, Center for Clinical and Translational Research, Seattle Children’s Hospital, “It’s as strong as the original compound they brought out.” She notes that Orkambi, Vertex’s currently approved drug combo, had an FEV1 that wasn’t even 5%. “These are very impressive changes.” She said that the safety of the compounds looked good, as well, although longer studies will be needed. “Obviously, let’s be honest, it’s four weeks,” Ramsey said.

Jennifer Taylor-Cousar, co-director of the adult CF program at National Jewish Health in Colorado, said that picking drugs for phase three trials was a step patients had been awaiting “with bated breath” as those studies would be the next step toward making those treatments available to patients. Both Ramsey and Cousar are members of a committee overseeing Vertex’s CF clinical trials.

Vertex’s first CF drug, Kalydeco, can have dramatic effects on patients’ lung function in cases where the disease is caused by a very specific mutation, and costs more than $300,000 a year. Orkambi, a combination of Kalydeco and another drug, works in a different mutation and costs about $270,000 per patient per year.

Vertex is also announcing earnings for the full year of 2017 that are in-line with analysts’ expectations. Kalydeco generated $845 million in sales in 2017, about 2% more than analysts expected, according to data from FactSet Systems, and a 20% increase from 2016. Orkambi generated $1.32 billion, in line with analyst expectations and a 35% increase from 2016. Vertex earned $1.04 per share using generally accepted accounting principals, 7 cents better than analyst forecasts.

Analysts expect the new Vertex drugs to generate many billions of dollars in annual sales if the next stage of studies are successful. Leerink Partners, an investment bank, forecasts that Vertex sales could triple to $6.3 billion by 2022.

The design of the new trials has not been finalized. The plan is to test VX-659 with tezacaftor and Kalydeco, and VX-445 with a tezacaftor and a new, once-a-day drug that is similar to Kalydeco. Discussions with the Food and Drug Administration about the trials are ongoing.