AveXis presented robust data at AAN demonstrating efficacy of Zolgensma in broad spectrum of spinal muscular atrophy (SMA) patients
Novartis International AG / AveXis presented robust data at AAN demonstrating efficacy of Zolgensma in broad spectrum of spinal muscular atrophy (SMA) patients . Processed and transmitted by West Corporation. The issuer is solely responsible for the content of this announcement.
Interim data reported for the first time from STRONG in SMA Type 2 showed rapid motor function gains and milestone achievements with intrathecal Zolgensma (onasemnogene abeparvovec-xioi; AVXS-101)
Basel, May 5, 2019 - AveXis, a Novartis company, today announced interim data from ongoing trials of the investigational product Zolgensma (onasemnogene abeparvovec-xioi; AVXS-101)1 that showed positive results across a broad spectrum of patients with spinal muscular atrophy (SMA). These included the first presentation of data from the Phase 1 STRONG trial, which showed motor function gains and milestone achievements in patients with SMA Type 2 via intrathecal (IT) delivery; new data from the Phase 3 STR1VE trial, which continued to show prolonged event-free survival, increases in motor function and significant milestone achievement consistent with the Phase 1 START trial; and the first presentation of data from the Phase 3 SPR1NT trial, which showed motor milestone achievement consistent with normal development in SMA patients treated pre-symptomatically. These data were presented during the 2019 American Academy of Neurology (AAN) Annual Meeting.
"With just a single, one-time dose, we are seeing Zolgensma provide prolonged survival, rapid motor function improvement and milestone achievements that patients never experience if their disease is left untreated," said David Lennon, President of AveXis. "These robust data presented at AAN represent a growing body of evidence that support the use of Zolgensma as a potential foundational therapy for the treatment of SMA across a variety of populations."