Eplontersen, jointly developed by AstraZeneca and Ionis Pharmaceuticals, met all co-primary endpoints and secondary endpoints at 66 weeks versus an external placebo group
Eplontersen paused the progression of neuropathy disease and improved neuropathy impairment and quality of life in the Phase III trial. (Credit: AstraZeneca)
AstraZeneca said that eplontersen has shown consistent and sustained improvement in all measures of disease and quality of life in 66 weeks of the NEURO-TTRansform Phase III trial in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN).
The detailed results have demonstrated that eplontersen, jointly developed by AstraZeneca and Ionis Pharmaceuticals, met all co-primary endpoints and secondary endpoints at 66 weeks compared to an external placebo group.
Eplontersen is a ligand-conjugated antisense (LICA) investigational medicine. It intends to lessen the production of transthyretin (TTR protein) to treat all types of amyloid transthyretin (ATTR).
The positive findings validate the eplontersen’s efficacy, safety and administration profile to provide a new option for ATTR disease.
At the end of 66 weeks, individuals treated with the investigational medicine showed consistent and sustained benefit on the three co-primary endpoints of serum TTR concentration, neuropathy impairment and quality of life (QoL).
Eplontersen also achieved least squares (LS) mean reduction of 82% in TTR serum concentration from baseline, versus an 11% reduction from baseline in the external placebo group.
The medicine paused the disease progression as calculated by a modified Neuropathy Impairment Score +7 (mNIS+7). Overall, it improved neuropathy impairment in 53% of patients at 66 weeks compared to baseline versus 19% in the external placebo group.
AstraZeneca said that overall eplontersen improved the QoL in 58% of treated patients at 66 weeks compared to baseline versus 20% in the external placebo group.
Additionally, the investigational medicine achieved statistically significant improvements in all secondary endpoints versus the placebo. It also continued to show a favourable safety and tolerability profile.
AstraZeneca BioPharmaceuticals R&D EVP Mene Pangalos said: “These results show that eplontersen sustains reduced transthyretin levels and improves neuropathy progression and quality of life consistently across a substantial number of patients.
“We are confident in eplontersen’s potential to be a much-needed and differentiated treatment option for patients living with all types of this devastating disease, which can also lead to heart failure.”
Last month, the US Food and Drug Administration (FDA) accepted a New Drug Application for eplontersen to treat ATTRv-PN. The medicine has also received Orphan Drug Designation in the US.
Currently, eplontersen is being studied in the CARDIO-TTRansform Phase 3 trial for transthyretin-mediated amyloid cardiomyopathy (ATTR-CM).
In December 2021, AstraZeneca and Ionis inked the global development and commercialisation agreement for eplontersen.