Rare disease specialist actions Vertex Pharmaceuticals (NASDAQ: VRTX) fell nearly 15.7% on the pre-stock market Thursday morning. The biotechnology stock is under pressure today due to a worrying safety signal seen in a mid-term trial of the small molecule drug VX-814 as a potential treatment for alpha-1 antitrypsin deficiency (AATD).
Specifically, development of the drug would be halted after several patients in the study experienced elevated liver enzymes. AATD is a genetic disease associated with an increased risk of lung and liver disease.
If this double-digit drop in pre-market trading continues, Vertex will lose around $ 10 billion in market capitalization today. While VX-814 represented a decent revenue generator for the company, the truth is, this drug would likely have faced stiff competition as an AATD treatment.
Biotechs like Arrowhead Pharmaceuticals, Alnylam Pharmaceuticals, and Digested pharmaceuticals are developing rival AATD therapies, after all. Vertex’s abrupt pullback today, in turn, appears to have more to do with shareholder concerns about the company’s non-cystic fibrosis assets – a market where it has a near-monopoly – than this rare setback. clinical.
Vertex is not throwing in the towel on the AATD, however. The biotech announced in the same press release that its other mid-level candidate, the VX-864, remains on track to produce top-notch data in the first half of 2021. What’s important to understand is this It’s that the VX-864 is structurally distinct from VX-814 – meaning this clinical failure should not be taken as a preview of things to come for the VX-864.