-- Ionis Pharmaceuticals (IONS) said a study shows its zilganersen 50 mg demonstrated statistically significant results in patients aged five and older living with Alexander disease, a rare and often fatal neurodegenerative disorder.
The pivotal study met its primary endpoint with the drug showing clinically meaningful stabilization of gait speed as assessed by the 10-meter walk test in patients, the company said Tuesday in a statement.
New data from the Gross Motor Function Measure-88 showed that younger children aged two to four who received zilganersen improved more in gross motor function than those in the study's control group, the company said.
Zilganersen demonstrated a favorable safety and tolerability profile, with most adverse events mild or moderate in severity, Ionis said.
The drug is under priority review at the US Food and Drug Administration, which is expected to make an approval decision by Sept. 22, Ionis said.
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