After last night announcing favorable Phase 1 and 2 results from of three different triple combination regimens in people with cystic fibrosis (CF) who have one F508del mutation and one minimal function mutation (F508del/Min), shares of biotech firm Vertex Pharma (VRTX 162.12, +29.96 +22.7%) trade to all-time highs.
As a bit of background, VRTX initiated its CF research program in 2000 as part of a collaboration with CFFT, the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation. KALYDECO (ivacaftor), ORKAMBI (lumacaftor/ivacaftor), tezacaftor, VX-440, VX-152, VX-659 and VX-445 were discovered by VRTX as part of this collaboration.
VRTX noted that these are the first data to demonstrate the potential to treat the underlying cause of CF in these patients, who have a severe and difficult-to-treat type of the disease.
Data from the Phase 2 studies in these patients showed mean absolute improvements in percent predicted forced expiratory volume in one second (ppFEV1) of 9.7 and 12.0 percentage points from baseline for the triple combination regimens with VX-152 (200mg q12h) or VX-440 (600mg q12h), respectively.
Concurrently, initial data from a Phase 1 study showed a mean absolute improvement in ppFEV1 of 9.6 percentage points from baseline for the triple combination regimen of VX-659, tezacaftor and ivacaftor in people with one F508del mutation and one minimal function mutation. VRTX also announced initial data showing improvements in mean absolute ppFEV1 of 7.3 and 9.5 percentage points when VX-152 or VX-440 was added in people with two copies of the F508del mutation, who were already receiving tezacaftor and ivacaftor.
The triple combination regimens were generally well tolerated across all three studies, and the majority of adverse events were mild to moderate in severity. Across the studies, the discontinuation rate due to adverse events was low.
Next up, VRTX’s VX-445 Phase 2 study is underway and a VX-659 Phase 2 study will begin in early August. VX-445 and VX-659 will be evaluated in triple combination with tezacaftor and ivacaftor in people with one F508del mutation and one minimal function mutation and will be evaluated in people with two copies of the F508del mutation who are already receiving tezacaftor and ivacaftor. Data from both of these Phase 2 studies are expected in early 2018.
Then, pending additional data from these Phase 2 studies and the ongoing studies of VX-152 and VX-440 and discussions with regulatory agencies and the Steering Committee, the company plans to initiate pivotal development of one or more triple combination regimens in the first half of 2018.
In short, the positive data from VRTX’s CF treatment is giving patients and those on the street something to smile about. Some are adding conservative sales for the treatment into their models, but with early indications blowing expectations out of the water, it may be tough to gauge the monetary contribution at this point. Given that VRTX reported 32% growth in ORKAMBI product revenues in Q1 (to $295 million) and 9% growth in KALYDECO product revenues (to $186 million), another CF treatment may fare equally as well on the regimen.