Shares in Alnylam Pharmaceuticals jumped after the company said a drug to treat a rare genetic form of heart disease met its goals in a late-stage trial, opening up the possibility of billions of dollars in additional sales for one of the most valuable US biotechs.
The company said its quarterly Vutrisiran injection cut the risk of mortality and adverse cardiovascular events by at least 28 per cent in a phase-three trial. It also improved capacity for physical exertion among patients with a rare protein disorder that affects heart function.
The disease, called transthyretin-mediated amyloidosis cardiomyopathy, or ATTR-CM, affects around 100,000 patients in the US and up to 500,000 globally, most of whom are men aged 60 and over. However, many of the cases go undiagnosed.