AveXis data reinforce effectiveness of Zolgensma for SMA Type 1

AveXis, a part of Swiss pharma giant Novartis (NOVN: VX), late Tuesday announced that interim data from its Phase III STR1VE trial of Zolgensma(onasemnogene abeparvovec-xioi; AVXS-101) in spinal muscular atrophy (SMA) Type 1 showed prolonged event-free survival, an early and rapid increase in CHOP-INTEND scores and significant milestone achievement compared to untreated natural history, consistent with data from the pivotal Phase 1 START trial.

First-in-human biodistribution individual case study data from STR1VE showed Zolgensma successfully transduced intended targets in the central nervous system (CNS) and provided widespread SMN expression comparable to tissue from unaffected individual. Additional data presented showed 95% of patients screened across the Zolgensma clinical development program and managed access program were not excluded from treatment due to elevated AAV9 antibody titers greater than 1:50. These data were presented at the 2019 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference in Orlando, Florida.

"These STR1VE data reinforce what was seen in the pivotal Phase I START trial, including trends toward prolonged survival and milestone achievement never seen in the natural history of the untreated disease," said Dr Olga Santiago, chief medical officer, AveXis. "With a patient population and baseline characteristics closely matched to the START trial, these data build upon the body of evidence supporting the use of Zolgensma for SMA Type 1."