VX-440 phase two study
Vertex Pharmaceuticals (VRTX) conducted a phase two study to evaluate the safety and efficacy of VX-440 200-mg dose and a 600-mg dose in combination with ivacaftor and tezacaftor for the treatment of two groups of individuals with cystic fibrosis aged at least 18 years. One group included individuals with one F508del mutation and one minimal function mutation, and the other group included individuals with two copies of the F508del mutation.
Safety and efficacy results
Vertex Pharmaceuticals found the triple combination to be well tolerated. The study on patients who had two copies of the F508del mutation is ongoing. The phase two study with VX-440 demonstrated significant efficacy of the investigational drug. The positive data from the clinical trials will likely help Vertex Pharmaceuticals commercialize the drug.
Vertex Pharmaceuticals’ peers in the cystic fibrosis drug market include Roche (RHHBY), Novartis (NVS), AbbVie (ABBV), Gilead Sciences, and others. The SPDR S&P 500 ETF (SPY) invests ~0.18% of its total portfolio holding in Vertex Pharmaceuticals.