Novartis Reports Updated Positive Data From Phase III SMA Program

Core Insights - Novartis reported positive safety and efficacy results from phase III studies of investigational gene therapy OAV101 IT for spinal muscular atrophy (SMA) in patients aged 2 to under 18 years [1][5] Study Results - In the phase III STEER study, OAV101 IT showed a statistically significant 2.39-point improvement on the Hammersmith Functional Motor Scale Expanded (HFMSE) compared to a 0.51-point increase in the sham control group, indicating better motor function [2][7] - The phase IIIb STRENGTH study demonstrated stabilization of motor function over 52 weeks in patients who had previously discontinued treatment with Spinraza or Evrysdi [3] Market Performance - Year to date, Novartis shares have increased by 14.6%, outperforming the industry growth of 7.2% [4] Regulatory Plans - Based on the success of the studies, Novartis plans to file for regulatory approval of OAV101 IT in multiple regions in the first half of 2025 [9] Additional Studies - OAV101 IT was also evaluated in a phase I/II STRONG study, which included approximately 170 patients with SMA and followed up for up to 6.4 years [8]