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AveXis, Inc.: Initiation of Research Coverage

Wednesday, August 23, 2017

William Blair & Company initiated research coverage of AveXis, Inc. (AVXS $93.38), a biotechnology company focused on using gene therapies to treat patients with rare and orphan neurological genetic diseases.

Analyst Raju Prasad estimates peak revenue for AVXS-101 in type 1 SMA of $1.5 billion.

“AveXis is a clinical-stage gene therapy company focused on developing and commercializing products to treat patients with rare and life-threatening neurological genetic diseases. We view the company’s lead candidate, AVXS-101 as a potential paradigm shifting gene therapy in spinal muscular atrophy (SMA) type 1,” Prasad said.

“In April 2014, a Phase I clinical trial with AVXS-101 was initiated at Nationwide Children’s Hospital in patients with SMA type 1, a severe form of a neuromuscular disease characterized by the loss of motor neurons; patients frequently die in early childhood from complications related to respiratory failure. As of January 2017, 15 of 15 (100%) patients in the Phase I study reached 13.6 months of age without having an event defined as death or ≥16 hours of ventilation continuously for ≥2 weeks (a natural history study places the event-free rate at 25%). Despite the open-label nature of AveXis’s study, we believe that the endpoint of death or permanent ventilation with genetically confirmed SMA type 1 is difficult to attribute purely to selection bias, particularly if the majority of patients have reached 24 months without an event (versus natural history of 8% event-free survival). In our view, AVXS-101 has a high probability of receiving guidance for a potential regulatory filing ahead of our current estimates following its end-of-Phase I meeting in the fourth quarter,” he added.

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