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Novartis Reports Positive Phase III STEER Study Results for OAV101 IT in SMA Type 2 Patients

NextEdge Admin

30 Dec 2024

2 min 50 sec

SMA Type 2 Patients,rare genetic disorder,gene therapy,clinical trials

Novartis announced positive topline results from the Phase III STEER study evaluating intrathecal onasemnogene abeparvovec (OAV101 IT) in treatment-naïve patients with spinal muscular atrophy (SMA) Type 2, aged 2 to <18 years, able to sit but not walk independently. The study met its primary endpoint, demonstrating significant improvements in Hammersmith Functional Motor Scale - Expanded (HFMSE) scores compared to sham controls, indicating better motor function. HFMSE is a gold-standard measure of SMA motor abilities and progression.

The safety profile of OAV101 IT was favorable, with similar adverse events (AEs) and serious AEs between treatment and control arms. The most common AEs were upper respiratory tract infections, pyrexia, and vomiting. Results reinforce OAV101 IT as a safe, effective one-time therapy with the potential to improve motor function in SMA patients.

Dr. Shreeram Aradhye, President of Development and CMO of Novartis, emphasized the therapy’s robust risk-benefit profile and potential to address unmet needs in SMA. Pediatric neurologist Dr. Crystal Proud highlighted its impact on maintaining and enhancing motor function, critical for independence in daily activities.

Building on data from the Phase I/II STRONG study, STEER confirms clinically meaningful motor improvements in SMA Type 2 patients, complementing the broader clinical development program, including the STRENGTH study. Over 100 patients participated in the double-blind, sham-controlled STEER study, with 52-week follow-ups demonstrating sustained benefit.

SMA is a rare genetic disorder caused by SMN1 gene dysfunction, leading to progressive motor neuron loss and muscle impairment. Current therapies require lifelong administration. OAV101 IT represents a paradigm shift as a one-time gene therapy designed to restore SMN1 function.

Novartis plans regulatory submissions in 2025, aiming to make OAV101 IT accessible for SMA patients worldwide. Data will be presented at upcoming medical conferences.