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AstraZeneca’s Eplontersen meets endpoints in critical phase 3 trial

Eplontersen significantly improved patient-reported quality of life following successful research.

Positive results from the NEURO-TTRansform phase 3 trial in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) showed AstraZeneca and Ionis’ eplontersen met all its co-primary endpoints.

At 35 weeks, the analysis demonstrated that eplontersen reached a clinically meaningful change while also reaching its primary endpoint of change from baseline in the modified neuropathy impairment score versus an external placebo group.

Results also showed the trial met its secondary endpoint of change showing treatment with eplontersen significantly improved patient-reported quality of life versus the external placebo group. Furthermore, the therapy demonstrated a favourable safety and tolerability profile with no specific concerns.

ATTRv-PN is a debilitating disease that leads to peripheral nerve damage with motor disability within five years of diagnosis and, without treatment, is generally fatal within a decade. Eplontersen –formerly known as IONIS-TTR-LRx – is a ligand-conjugated antisense investigational medicine designed to reduce the production of TTR protein at its source to treat both hereditary and non-hereditary forms of the condition.

Teresa Coelho, a neurophysiologist and investigator for the NEURO-TTRansform trial, enthused: “This encouraging data reinforce the safety profile of eplontersen and demonstrate clear evidence of its potential to provide much-needed therapeutic benefit to patients living with hereditary transthyretin-mediated amyloid polyneuropathy.”

Mene Pangalos, executive vice president, BioPharmaceuticals R&D at AstraZeneca, added: “Amyloid transthyretin polyneuropathy is a rare and fatal disease that can affect up to 40,000 people worldwide. These promising results show eplontersen has the potential to be a new and much-needed treatment where limited options exist and the significant unmet medical need remains.”

As part of the global development and commercialisation agreement with Ionis, eplontersen will be jointly developed and commercialised by both companies in the US and will be developed and commercialised in the rest of the world by AstraZeneca.