Sarepta micro-dystrophin gene therapy update continues to impress, says Janney Montgomery Scott
Janney Montgomery Scott analyst Yun Zhong believes the three-month muscle biopsy data from the fourth patient and the functional results from all four patients that Sarepta reported at the Annual Congress of the World Muscle Society provide "strong" additional evidence to support a favorable clinical profile of the micro-dystrophin gene therapy program. The analyst notes that management has requested a meeting with the FDA and he continues to expect the agency to be supportive for an expedited regulatory pathway. Upon FDA feedback, patient dosing in the pivotal study could begin in Q4 of 2018/Q1 of 2019, he contends. Additionally, Zhong expects the initial data from the LGMD2E gene therapy program to be positive, based on a readthrough from the micro-dystrophin program. He reiterates a Buy rating and $200 fair value estimate on the shares.