Vertex Pharmaceuticals Incorporated VRTX announced that two phase III studies evaluating its next-generation CFTR corrector, VX-659 in combination with tezacaftor and Kalydeco (ivacaftor) met the primary endpoint of improvement in lung function in patients suffering from cystic fibrosis ("CF").
Shares of Vertex were up 4.8% in response to the news. In fact, so far this year, Vertex’s shares have gained 17.2% against the industry’s decline of 18.2%.
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 14.0 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-659 was added in patients already receiving tezacaftor and ivacaftor compared to placebo plus tezacaftor and ivacaftor.
Other than VX-659, Vertex is also evaluating another next-generation CFTR corrector, VX-445 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.
Enrollment in the VX-445 phase III studies is now complete with top-line data expected to be presented in the first quarter of 2019. A regulatory submission in the United States for one of the two regimes is planned for mid-2019. Vertex plans to choose the best regimen after evaluating data from the VX-445 and VX-659 triple combination studies in the first quarter of next year. Submissions for regulatory approvals in outside U.S. countries are planned for late 2019.
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’ stock was down more than 9% on Tuesday as Vertex’s strong efficacy data from this pivotal study has raised the competitive bar for later entrants.
In July, 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. Vertex plans to evaluate VX-659 and VX-445 in triple combination with tezacaftor and VX-561 as a once-daily regimen ) to treat the underlying cause of CF. VX-561 will enter a dose-ranging phase II study as a monotherapy in gating mutations in early 2019.
Vertex currently has a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
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