Novartis announces positive data from halted Zolgensma trial
The trial may be on hold, but Novartis says the drug works.
Novartis announced today new data on a delivery route to make the gene therapy Zolgensma applicable and accessible to older kids with spinal muscular atrophy, even as the Phase I/II trial on that route remains on hold. The FDA paused the trial in October after preclinical studies showed loss of neural function in some mice. The hold came after an industry-rocking scandal over how Novartis handled data-tampering in a different set of preclinical Zolgensma studies.
Novartis subsidiary AveXis, which developed and markets Zolgensma, said that in the median dosing arm of their STRONG trial, the 2-year-old to 5-year-old patients met the primary endpoint: They improved an average of 6.0 points on the Hammersmith Functional Motor Scale-Expanded, a metric used to measure muscle function in kids with spinal muscular atrophy. AveXis said that was twice the improvement required to be clinically meaningful and reflected advances in three to six motor skills.
In a press release, AveXis CEO David Lennon praised the results and hinted he hoped they would convince regulators to resume the trial. He used the experimental company name of the drug.
“STRONG data demonstrated potential best-in-category profile with remarkable motor function improvement following a single, one-time intrathecal dose,” Lennon said. “We look forward to sharing these data with regulators to further our discussions toward registration of intrathecal AVXS-101.”
Approved last year, Zolgensma was the second gene therapy to reach the market in the US and just the second-ever treatment for spinal muscular atrophy after the 2016 approval of Ionis’s and Biogen’s Spinraza. The therapy was limited, though, to patients under the age of 2. The swiftness of the genetic disorders reduced the odds of success the older patients got. Most kids with SMA type 1 — the first approved indication — do not live past the age of 2.
People with SMA type 2, however, can live into early adulthood, and Novartis has been trying to test if an intrathecal injection of Zolgensma — as opposed to an intravenous one — can improve their prognosis.
The hold on the trial affected the highest dosing arm of the study. Since October, the Swiss giant has tried to get the FDA to lift the halt, pointing to the fact that the neural damage shown in the preclinical study has never appeared across the 300-plus patients who have received the gene therapy. The company said they expect a decision from the FDA next quarter.