- Novartis AG NVS announced new data from the completed two-copy cohort of Phase 3 SPR1NT trial of Zolgensma (onasemnogene abeparvovec), gene therapy for spinal muscular atrophy (SMA).
- The data were presented at the European Academy for Neurology Virtual Congress 2021.
- In the open-label study, investigators tested the gene therapy in a total of 29 patients six weeks or younger who had a genetic diagnosis of SMA plus two or three copies of the protein-coding gene SMN2 but had not yet developed symptoms.
- In the presentation, investigators reported results specifically for the 14 patients with two copies of SMN2. All of them were alive at 14 months of age, compared with an expected 26% survival at that point in the natural course of the disease.
- All patients could sit independently for 30 seconds, while 11 could stand independently and nine could walk.
- None of the patients required nutritional or respiratory support during the trial.
- While ten patients experienced side effects that were likely treatment-related, there were no severe cases.
- Meanwhile, investigators also wrapped up the STR1VE study, testing the drug in patients six weeks or younger with SMA Type 1 who had biallelic SMN1 gene deletion or point mutations and one or two copies of SMN2.
- In that study, 82% of patients achieved developmental milestones not typically seen in the natural course of the disease, including 16 children who sat without support for more than 30 seconds.
- Price Action: NVS shares closed at $93.01 on Friday.
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