AstraZeneca/Ionis present data for rare disease drug meeting trial's main goal
gorodenkoff

AstraZeneca (NASDAQ:AZN) and Ionis Pharmaceuticals (NASDAQ:IONS) presented detailed results from a 35-week interim analysis of a phase 3 trial of eplontersen, which had met its main goal, in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN).
The company presented the data at the International Symposium on Amyloidosis in Germany. Broad results were reported in June.
ATTRv-PN is a rare inherited disorder caused by mutations in the transthyretin (TTR) gene and characterized by abnormal build up of a protein called amyloid in certain organs and tissues leading to their dysfunction. Polyneuropathy is a condition in which a person's peripheral nerves (nerves outside the brain and spinal cord) are damaged.
In the study, eplontersen achieved an 81.2% mean reduction in the co-primary goal of serum transthyretin (TTR) concentration compared to baseline, showing reduced TTR protein production, the company said in a Sept. 7 press release.
The drug also showed a significant treatment effect on the co-primary goal of modified Neuropathy Impairment Score +7 (mNIS+7), a measure of neuropathic disease progression, with a statistically significant difference in mean change from baseline compared to the external placebo group.
The trial also met its key secondary goal, showing that eplontersen significantly improved patient-reported quality of life compared to the placebo group.
Ionis said eplontersen showed a favorable safety profile and the rate of treatment emergent adverse events (TEAEs) in the eplontersen group was either lower or similar compared to placebo.
Ionis reiterated noted that the companieswill seek regulatory approval for eplontersen for ATTRv-PN and plan to file a new drug application with the U.S. Food and Drug Administration this year.