• Vertex Stops Test of Rare Disease Drug, Dimming Pipeline Prospects

    8 days ago - By Xconomy

    Vertex Pharmaceuticals is halting work on an experimental drug for a rare protein deficiency after safety problems were reported in Phase 2 testing. The study was evaluating the small molecule, VX-814, as a treatment for alpha-1 antitrypsin deficiency, which can lead to liver and lung problems. After the markets closed Wednesday, Vertex disclosed that elevated liver enzymes-eight times greater than normal--were observed in four of the patients who received the drug i. The placebo-controlled study enrolled approximately 50 patients. The liver enzyme levels are resolved or are resolving but...
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