- Vertex Pharmaceuticals Inc. on Wednesday forecast revenues from its two cystic fibrosis treatments would come in higher this year than the company had originally expected, boosted by strong demand in the U.S. and label expansions that have increased the number of patients the drugs can treat.
- Together, sales of Orkambi and Kalydeco hit $550 million in the third quarter, up 34% from the same period last year. Vertex now expects the drugs to earn between $2.1 billion and $2.15 billion for all of 2017.
- The biotech has inched its revenue guidance up as the year has progressed and the Food and Drug Administration has OK'd Kalydeco for new uses. An approval in August, for example, allowed the drug to be used in roughly 600 cystic fibrosis patients who have one of five mutations that damage the crucial CFTR gene.
With Kalydeco and Orkambi, Vertex is already a leader in the cystic fibrosis market. But mid-stage data disclosed this summer on three triple combinations — pairing Kalydeco (ivacaftor) with the experimental tezacaftor and a next-generation corrector — could give the biotech an even more commanding position, analysts say.
Currently, Vertex's two approved drugs can treat about half of all cystic fibrosis patients. If one of the triple combos succeeds, the company might be able to reach as many as 90% of the market.
Vertex said it plans to initiate pivotal studies of up to two triple regimens in the first half of 2018, pending discussions with regulators and further analysis of which combo looks most promising.
"We're rapidly moving towards enrollment [of triple combo studies], which we believe that up to 90% of patients will be on a single triple regimen and getting maximum effect," said Vertex CEO Jeffrey Leiden. "Ten percent are going to be waiting for genetic therapies, and there will be a few, a small number that remain on Kalydeco monotherapy. That's how we see the world a few years from now."
Doublet decision in February
In the meantime, Vertex could soon win approval for a doublet combo that pairs Kalydeco with tezacaftor. A decision from the FDA is expected by February 28 of next year. Executives believe that it could offer some existing patients a safer option than Orkambi and help grow overall revenues.
"With Orkambi, we've had a number of patients who discontinued Orkambi often due to adverse events," said Stuart Arbuckle, chief commercial officer at Vertex, on a Oct. 25 call with analysts. "Given the benefit/risk profile of tezacaftor/ivacaftor, I think that's a population which is likely to see high demand for tezacaftor/ivacaftor."
Patients with residual function mutations and who are not yet on Kalydeco could also be channeled to the combo as a new option, Arbuckle noted.
Vertex's quarterly update wasn't all positive. The company disclosed that a Phase 3 study of tevacaftor/Kalydeco in CF patients with one copy of a mutation known as F508del and another gating mutation failed to hit its primary endpoint.
But that study compared the combo to patients already on Kalydeco — making it difficult to show benefit.
"The very high bar that Kalydeco has set with those patients made it difficult to identify incremental acute improvements in FEV1 [a measure of simply how much air a person can exhale] because when they ended the study, they were doing so well," explained Vertex CEO Jeffrey Leiden.
Another study of an experimental ENaC inhibitor with Orkambi also failed in patients with two copies of the F508del mutation.
Jefferies analyst Michael Yee, however, dismissed the impact of those two disappointments, noting Kalydeco would continue to serve well for patients. And the ENaC inhibitor, in Yee's analysis, was more of a backup in case data on the triple therapies fell short of expectations.