AstraZeneca and Ionis’ ATTRv-PN therapy meets trial endpoints
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.
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.
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.”
April 25, 2023
