Benzinga | Jun 21, 2021 07:24AM EDT

Novartis Touts More Positive Zolgensma Data In Spinal Muscular Atrophy Patients

* 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.