Vertex shares take a hit as liver tox forces the research team to scrap a PhII drug for AAT patients. Will we see new deals now?
Vertex is taking the ax to one of their rare disease drugs after evidence of liver damage emerged in a key Phase II study that blocks any uses of the higher doses that would be needed to treat genetic cases of alpha-1 antitrypsin deficiency. And with that program down and out, one top analyst says that the big biotech has no choice but to start lining up deals. Now.
The big biotech had highlighted a similar approach to mid-stage work that got them through major R&D work in cystic fibrosis, which created the foundation the company is built on. But in the trial of some 50 patients, 4 taking different doses of the drug demonstrated elevated liver enzymes “greater than 8 times the upper limit of normal.”
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