Jefferies Upgrades AveXis To Buy Following Positive AVXS-101 Data, Doc Feedback

The updated AVXS-101 data released by AveXis Inc AVXS suggests that the FDA may allow for a single-arm pivotal trial in SMA Type 1, Jefferies’ Biren Amin said in a report. He upgraded the rating on the company from Hold to Buy, while maintaining the price target at $42.

Sustained Efficacy

“Data as of July 1st demonstrate that the patients in the therapeutic dose group continue to be event free and their CHOPINTEND scores have improved,” Amin wrote.

Although the company disclosed that one patient in the low dose cohort had experienced an event, this is not a major concern, since the patient was dosed late, was in the low dose cohort, and was on ventilation before enrolling in the study, the analyst stated.

Positive Doc Feedback

Amin mentioned that an expert physician, who had participated in the competitor program, had given positive feedback and stated that AVXS-101 and Nusinersen [Biogen Inc BIIB and Ionis Pharmaceuticals Inc IONS] “should be complementary therapies.”

The physician added that both offered “a compelling benefit,” although additional data is required to conclude if one is superior to the other. So far, AVXS had delivered a better CHOP-INTEND benefit.

“The expert believes that FDA will be flexible in pivotal trial design for AVXS-101 and may allow a single arm study given the NeuroNEXT data is available as a robust historical cohort, and potentially will have the placebo control arm from the BIIB/IONS ENDEAR trial that could be used for comparator purposes,” the Jefferies report noted.

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