Vertex Pharmaceuticals Incorporated VRTX announced that two phase III studies evaluating its next-generation CFTR corrector, VX-445 in combination with tezacaftor and Kalydeco (ivacaftor) met the primary endpoint of significant improvement in lung function in patients suffering from cystic fibrosis ("CF").
The improvement in lung function was measured as mean absolute improvements in percent predicted forced expiratory volume in one second, or ppFEV1. Cystic fibrosis is a rare genetic disease that causes chronic lung infections and progressive lung damage.
While one study is evaluating the triple combination regimen in patients with one F508del mutation and one minimal function mutation, the other study is evaluating patients with two F508del mutations.
In the study evaluating patients with one F508del mutation and one minimal function mutation, the interim data showed that the triple combination regimen led to a mean absolute improvement in ppFEV1 of 13.8 percentage points from baseline through week four of treatment compared to placebo.
The second study yielded a mean absolute improvement in ppFEV1 of 10.0 percentage points from baseline at week four when VX-445 was added in patients already receiving tezacaftor and ivacaftor compared to placebo plus tezacaftor and ivacaftor. Vertex believes that safety and efficacy profile observed in the study supports a potential NDA submission.
Other than VX-445, Vertex is also evaluating another next-generation CFTR corrector, VX-659 in phase III studies as part of a triple combination with tezacaftor and ivacaftor. Vertex is also evaluating this combination in two separate studies for F508del/Min and F508del homozygous patients. In November, the VX-659 phase III studies also met the primary endpoint showing a significant improvement in lung function. The safety and efficacy profile shown in VX-659 studies also supported a NDA submission.
Given the similarity of the four-week data between the studies of the two regimens, Vertex has decided to wait for the final 24-week data for both regimens, expected to be available in the second quarter of 2019. Accordingly, it will file a regulatory application in the United States for the better of the two regimens in both F508del/Min and F508del homozygous patient populations in the third quarter of 2019. A regulatory application in the EU is expected to be filed in the fourth quarter. The regulatory filing timeline has been slightly delayed from prior timeline of no later than mid-2019 to select the drugs for filing after including the 24-week data in global submissions.
Despite the positive data from the of VX-445 studies, Vertex’s shares were down almost 4% on Wednesday as the company delayed the timeline for the regulatory filing. Vertex’s stock has increased 9.3% this year so far compared with the industry’s increase of 14.5%.
If the triple-combo regimes are successful, Vertex can address a significantly larger CF patient population — almost 90% of patients with CF — in the future. This can expand its reach in the CF market. At present, approximately half of all CF patients are eligible to be treated with a marketed Vertex CF medicine — Kalydeco, Orkambi or Symdeko.
Meanwhile, many other companies like AbbVie ABBV and Proteostasis Therapeutics, Inc. PTI are also developing triple CFTR combinations for CF. Proteostasis Therapeutics is expected to report top-line data from its triple combo CF medicine in 2019.
In 2017, Vertex bought Concert Pharmaceuticals, Inc.’s CNCE CF pipeline candidate, VX-561 (previously CTP-656) and expanded its ongoing triple combination studies to add cohorts to develop VX-561 as a potential once-daily triple combination regimen with its other pipeline drugs (VX-561 + VX-659+ tezacaftor and VX-561 + VX-445+ tezacaftor) to treat the underlying cause of CF.
Positive phase II data were announced in 2018. Phase III studies of the once-daily triple combination regimen are expected to be initiated after data from a dose-ranging phaseII study of VX-561, as a monotherapy in gating mutations, are available.
Vertex has a Zacks Rank #5 (Strong Sell).
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