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AstraZeneca and Ionis Pharmaceuticals have reported that eplontersen met all co-primary endpoints and secondary endpoints in the Phase III NEURO-TTRansform trial in hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) patients.

A ligand-conjugated antisense (LICA) investigational medicine, eplontersen met the endpoints at 66 weeks against an external placebo group.

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Results from the trial revealed that at 66 weeks, patients receiving eplontersen showed steady and continued benefits regarding the trial’s three co-primary endpoints, namely serum transthyretin (TTR) concentration, neuropathy impairment and quality of life (QoL).

The drug candidate achieved an 82% least squares (LS) mean reduction in TTR serum concentration from baseline as against an 11% reduction from baseline in the external placebo group.

Eplontersen also prevented disease progression as calculated by modified Neuropathy Impairment Score +7 (mNIS+7).

Furthermore, 47% of treated patients demonstrated improvements in neuropathy at 66 weeks in comparison with baseline as opposed to 17% in the external placebo group.

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Among the patients who completed the trial, 53% of treated participants showed improvements in neuropathy at 66 weeks in comparison with baseline versus 19% in the external placebo group.

In addition, eplontersen enhanced the patients’ quality of life (QoL), showing a 5.5 point LS mean decrease (improvement) on the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN), against a 14.2 point increase (worsening) in the external placebo group.

Altogether, 58% of patients treated with eplontersen demonstrated improvements in QoL at 66 weeks in comparison with baseline versus 20% in the external placebo group.

AstraZeneca BioPharmaceuticals R&D executive vice-president Mene Pangalos said: “Without treatment, hereditary transthyretin-mediated amyloid polyneuropathy is a relentlessly progressive disease.

“These results show that eplontersen sustains reduced transthyretin levels and improves neuropathy progression and quality of life consistently across a substantial number of patients.”