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Vertex Next-Gen Cystic Fibrosis Drug Passes Key Efficacy, Safety Hurdle

The future of Vertex Pharmaceuticals' (VRTX) cystic fibrosis business looks stronger Tuesday night.

Vertex is announcing successful outcomes from two clinical trials involving tezacaftor, a second-generation "corrector" used in combination therapies to treat the underlying cause of cystic fibrosis. Importantly, the new efficacy and safety data reported from the tezacaftor studies improves upon the performance of Vertex's existing products.

Tezacaftor is a crucial drug for Vertex because it will enable the company to treat more cystic fibrosis patients and potentially blunt competitors racing to catch up.

Vertex shares closed Tuesday at $89.67 in front of today's clinical trial news. The stock is up 22% year to date.

The larger of the two clinical trials announced today -- which Vertex calls EVOLVE -- enrolled just over 500 cystic fibrosis patients with two copies of a genetic mutation known as F508del. These patients represent about 40% of all cystic fibrosis cases.

Over 24 weeks, the combination of tezacaftor with an already approved Vertex drug ivacaftor (sold as Kalydeco) improved lung function by four percentage points compared to a placebo. The outcome was statistically significant, achieving the primary endpoint of the trial, Vertex said Tuesday.

The four percentage point improvement in lung function attributed to "teza/iva" compares well against Orkambi, Vertex's currently approved combination therapy for the...