Vertex's Positive Phase 3 Results for Triplet Combo Reinforce Narrow Moat; No FVE Change
Vertex reported positive results from two Phase 3 studies of its pipeline triplet combination in cystic fibrosis on Nov. 27, but we do not anticipate a change to our $183 fair value estimate as these readouts reinforce our confidence in the narrow-moat firm's cystic fibrosis portfolio. The combination of VX-659, tezacaftor, and ivacaftor met its primary endpoint in both patient populations being tested, heterozygous (one F508del mutation) and homozygous (two mutations), and the combination demon...Vertex Pharmaceuticals is focused on developing and commercializing therapies for the treatment of cystic fibrosis (CF) and developing research and development programs in other indications. The company's marketed products are TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor), SYMDEKO/SYMKEVI (tezacaftor in combination with ivacaftor), ORKAMBI (lumacaftor in combination with ivacaftor) and KALYDECO (ivacaftor), which are approved to treat patients with CF who have specific mutations in their cystic fibrosis transmembrane conductance regulator gene. The company is evaluating CTX001, a genetic therapy as a potential treatment for sickle cell disease and beta-thalassemia.
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