Novartis has reported positive efficacy and safety outcomes from its Phase III clinical programme for OAV101 IT (onasemnogene abeparvovec), an investigational intrathecal therapy for spinal muscular atrophy (SMA) in individuals aged two to less than 18 years.
In the registrational Phase III STEER trial, the therapy treatment resulted in a 2.39-point improvement on the Hammersmith Functional Motor Scale Expanded (HFMSE) against 0.51 points in the sham control arm, indicating a notable advancement in SMA-specific motor ability assessment.
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In this trial, the efficacy and safety of the therapy were evaluated against a sham control in treatment-naïve SMA Type 2 subjects who could sit but had never walked independently.
After 52 weeks, all eligible subjects had received both the therapy and the sham procedure. The primary endpoint was met, with OAV101 IT showing an improvement in HFMSE scores.
The Phase IIIb STRENGTH trial further demonstrated the motor function stabilisation in subjects switching from other treatments.
This open-label trial assessed the therapy’s efficacy and safety in individuals who had previously been treated with nusinersen or risdiplam. It enrolled 27 subjects with a mean age of 7.4 years.
Over 52 weeks, motor function stabilisation was observed, with a slight increase in HFMSE scores. While all subjects experienced a minimum of one adverse event, none led to death or trial discontinuation.
Novartis Ddevelopment president and chief medical officer Shreeram Aradhye said: “The data presented today from our OAV101 IT programme reinforce our belief in this therapy, which has the potential to have a meaningful impact on a broad range of people with SMA through its continuous benefit via a one-time dose.”
The development programme for the therapy has encompassed over 170 SMA subjects, with a combined follow-up period exceeding 6.4 years across multiple studies such as STEER, STRENGTH, as well as Phase I/II STRONG.
This gene replacement therapy claims to address the genetic root cause of SMA by substituting the nonworking survival motor neuron 1 (SMN1) gene with one dose.
In October 2024, the company reported positive outcomes from the Phase III APPEAR-C3G study of Fabhalta.
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