Sarepta Therapeutics shares plunge as gene therapy trial falls short of primary goal

Sarepta Therapeutics Inc. shares SRPT, +0.77% dropped more than 40% premarket on Tuesday after the company said a trial of its gene therapy for Duchenne muscular dystrophy fell short of its main goal.

The therapy, Elevidys, helped patients’ movement and walking ability but failed to produce a statistically significant improvement versus placebo in an ambulatory assessment after 52 weeks of treatment, Sarepta said in a release Monday.

The trial involved patients age 4 through 7 years with Duchenne muscular dystrophy, a genetic disorder that can cause severe muscle weakness, with symptoms starting in early childhood. Based on the overall trial results, Sarepta said it plans to seek a label expansion so that Elevidys can treat all patients. The U.S. Food and Drug Administration in June approved Elevidys for treatment of patients age 4 through 5 years. The trial results support the conclusion that Elevidys “modifies the trajectory of Duchenne and benefits patients across age groups living with this ferociously degenerative disease,” Doug Ingram, Sarepta’s president and CEO, said in a statement.

The trial data were “extremely disappointing,” Leerink Partners analysts wrote in a note Monday, but “we are not writing off the FDA’s historical flexibility” in Duchenne muscular dystrophy. The analysts maintained an outperform rating on Sarepta shares but cut their price target to $130, from $185.

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