STAT+: Vertex shelves second drug for inherited lung disease, raising concerns about future growth

Vertex Pharmaceuticals said Thursday that an experimental protein “corrector” drug — the highest-priority medicine in the biotech’s pipeline — failed to achieve the goals of a small clinical trial involving patients with an inherited disorder that causes serious damage to the liver and lungs.

The failure of the oral drug, called VX-864, is the second setback for Vertex and its effort to develop a treatment for patients with a genetic disease called alpha-1 antitrypsin deficiency, or AATD. The company’s first attempt, with a different drug, was shelved due to liver toxicity last year.

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