Novartis shares updated gene therapy program data for broad SMA population

Novartis announced positive safety and efficacy results from two trials in its phase 3 program for its investigational gene therapy, onasemnogene abeparvovec (OAV101 IT), in children and young adults with spinal muscular atrophy (SMA) Type 2.

The STEER study evaluated the clinical efficacy, safety and tolerability of a one-time dose of intrathecal OAV101 IT in treatment naïve patients with SMA Type 2, aged two to less than 18 years who were able to sit but had never walked independently. Novartis had previously reported that that the study met its primary endpoint showing an increase from baseline across the study population in total Hammersmith Functional Motor Scale - Expanded scores, a gold standard for SMA-specific assessment of motor ability and disease progression. Novartis has now shared details, revealing that the treatment with OAV101 IT led to a statistically significant 2.39-point improvement versus 0.51 points in the sham control.

In the phase 3b STRENGTH study, treatment with OAV101 IT in patients who had discontinued treatment with nusinersen or risdiplam demonstrated stabilization of motor function over 52 weeks of follow-up.

The results of both studies add to the growing body of evidence within the OAV101 IT development program, which has evaluated a broad population of over 170 patients with SMA, spanning a total of over 6.4 years across the STEER, STRENGTH and phase 1/2 STRONG studies.

The new treatment shares the same active ingredient as Novartis’ blockbuster SMA treatment, Zolgensma, approved in 2019. Zolgensma is approved for children less than two years old and is administered intravenously, while OAV101 IT targets older children and is administered intrathecally (through the spinal column).