Novartis AG (NVS), Wednesday, announced the positive results from its Phase III clinical program of investigational gene replacement therapy, intrathecal onasemnogene abeparvovec or OAV101 IT, for the treatment of spinal muscular atrophy.
The phase III program included two studies: the STEER and STRENGTH trials, involving patients aged 2 to less than 18 years.
The studies demonstrated that treatment with OAV101 IT led to a statistically significant improvement in motor function, with a 2.39-point increase in the Hammersmith Functional Motor Scale Expanded or HFMSE compared to the control group, which showed only a 0.51-point improvement.
As per the trial outcomes, OAV101 IT showed consistent safety results in both treatment-naïve and treatment-experienced patients.
The therapy's favorable safety profile was observed across all studied groups, with no severe complications reported.
Novartis plans to move forward with the regulatory filing process in H1 2025, with the aim of offering OAV101 IT as a one-time treatment option to SMA patients.
If regulatory filings be successful, Novartis anticipates the potential for this investigational gene therapy to provide long-term motor function improvement for a broad range of Spinal muscular atrophy patients.
Spinal muscular atrophy (SMA) is a rare genetic disorder caused by the absence of a functional SMN1 gene. This leads to the permanent loss of motor neurons, which affects muscle functions like breathing, swallowing, and basic movements.
OAV101 IT is designed to directly address the genetic root cause of SMA by replacing the nonworking SMN1 gene with a single dose.
Currently, NVS is trading at $111.55 down by 0.60 percent on the Nasdaq.
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