AstraZeneca/Ionis rare disease drug shows positive data in late stage study
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AstraZeneca (NASDAQ:AZN) and Ionis Pharmaceuticals (NASDAQ:IONS) said they would seek regulatory approval for eplontersen in the U.S. after the drug showed clinically meaningful changes on the co-primary goals of a phase 3 trial in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN).
ATTRv-PN is a rare inherited disorder due to mutations in the gene transthyretin 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.
The companies reported data from a 35-week interim analysis of the Phase 3 NEURO-TTRansform study.
The companies said eplontersen showed a statistically significant and clinically meaningful change from baseline for the co-primary goals of percent change in serum transthyretin (TTR) concentration and on a measure of neuropathic disease progression, versus the historical placebo group.
Eplontersen also met its key secondary goals of change from baseline, showing that the drug significantly improved patient-reported quality of life versus the historical placebo group, according to the companies' June 21 press release.
The companies added that the medicine also showed a favorable safety and tolerability profile with no specific concerns.
The companies noted that based on this data, they will seek regulatory approval for eplontersen for ATTRv-PN and plan to file a new drug application with the U.S. Food and Drug Administration in 2022.