Morningstar | 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 demonstrated a tolerable safety profile overall. In heterozygous patients, the statistically significant improvement from baseline lung function was 14 percentage points, and in homozygous, 10 percentage points. Vertex's Orkambi and Symdeko doublet combination was already treating homozygous patients, but the triplet's encouraging results could lead to an expanded eligible patient population and stronger pricing power, if approved.Vertex also reported that it has completed enrollment of two more Phase 3 studies of a different triplet combination of VX-445, tezacaftor, and ivacaftor, with data expected in the first quarter of 2019. We anticipate Vertex will file for regulatory approval in the U.S. for its best triplet combination by mid-2019.
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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