Vertex stumbles after mixed results from rare disease trial

11 June 2021

Shares in Boston, USA-based Vertex Pharmaceuticals (Nasdaq: VRTX) fell 14% in pre-market trading overnight, after the firm said late on Thursday it would pull the plug on development of VX-864.

Vertex has been trialling the candidate for alpha-1 antitrypsin deficiency (AATD), an inherited disease that causes an elevated risk of conditions including chronic obstructive pulmonary disease (COPD) and liver disease.

The announcement follows poor results from a Phase II study, in which the magnitude of treatment effect was “unlikely to translate into substantial clinical benefit,” the firm said.

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