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Novartis AG (NYSE: 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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