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Shares of Alexion fell Tuesday after the biopharmaceutical company said it missed its goal in a late-stage trial of the drug Solaris.
The stock ended the day down nearly 11 percent after the announcement, and has fallen more than 27 percent this year.
Alexion's drug Solaris is being studied as a treatment for a neuromuscular disease called Myasthenia Gravis, or MG, which causes patients to suffer muscle weakness that makes it difficult or impossible to perform daily activities like walking, talking, swallowing and breathing normally.
The study did not show a "statistical significance" from the baseline after week 26 of the trial, the company said. While it missed the primary goal, the study did hit targets on some secondary endpoints.
"While the REGAIN study missed its primary endpoint, I am encouraged by the clinically meaningful improvements in MG-ADL and QMG measures in patients treated with eculizumab compared with placebo," said Dr. James F. Howard Jr., distinguished professor of neuromuscular disease at the University of North Carolina School of Medicine. "There is an urgent need in the MG community for a therapy with the potential to dramatically improve the lives of patients with refractory MG, who continue to experience profound complement-mediated muscle weakness that makes it difficult or impossible to perform simple daily activities."
Alexion, which makes therapeutic products for patients with severe and ultra-rare disorders, will continue to analyze data from the study and will present results in July, during the International Congress on Neuromuscular Diseases in Toronto, the company said.
Analysts at Leerink Partners, an investment bank specializing in healthcare, reduced their Alexion price target from $210 to a $200 a share but maintained their "outperform" rating. Despite the recent results, they said the overwhelming evidence suggests that the drug is safe and benefits patients.
"The treatment has been endorsed by patients and the qualitative primary endpoint (imposed by regulators) is unlikely to be viewed as the 'be all and end all' in this trial, given the many other measures of benefit," Leerink said in a note published Tuesday. "Alexion's stock is likely to weaken on this announcement, but we would recommend buying especially if there is more than a mid-single digit sell-off."
Shares of Alexion traded below $139 a share Tuesday, down from their 52-week high of $208.88. The stock is down nearly 14 percent year over year.