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Eplontersen Ph III trial met co-primary endpoints
Eplontersen Ph III trial met co-primary endpoints.
About this update from Astrazeneca Plc
[{"type":"text","content":"\n \n \n 21 June 2022 07:00 BST\n \n \n \n \n \n \n \n \n Eplontersen met co-primary and secondary endpoints in interim analysis of the NEURO-TTRansform Phase III trial\n \n \n for hereditary transthyretin-mediated amyloid polyneuropathy (\n \n \n ATTRv-PN)\n \n \n \n \n \n \n \n New Drug Application filing anticipated based on positive data from interim analysis\n \n \n \n \n \n \n \n \n Positive high-level results from the NEURO-TTRansform Phase III trial in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) showed AstraZeneca and Ionis' eplontersen met its co-primary endpoints in a planned interim analysis at 35 weeks. In the trial, eplontersen reached a statistically significant and clinically meaningful change from baseline for its co-primary endpoint of percent change in serum transthyretin (TTR) concentration, reducing TTR protein production. Eplontersen also reached its co-primary endpoint of change from baseline in the modified Neuropathy Impairment Score +7 (mNIS+7), a measure of neuropathic disease progression1, versus external placebo group.\n \n \n \n \n \n High-level results showed the trial also met its secondary endpoint of change from baseline in the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN) showing treatment with eplontersen significantly improved patient-reported quality of life\n versus\n external placebo group\n .\n In the trial, eplontersen demonstrated a favourable safety and tolerability profile with no specific concerns.\n \n \n \n \n \n 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 decade3.\n Eplontersen,\n formerly known as IONIS-TTR-LRx, is a ligand-conjugated antisense (LICA) investigational medicine designed to reduce the production of TTR protein at its source to treat both hereditary and non-hereditary forms of ATTR2,4-6.\n \n \n \n \n \n \n Teresa Coelho, M.D., a neurologist and neurophysiologist at Hospital Santo António, Centro Hospitalar Universitário do Porto, Portugal and an investigator for the NEURO-TTRansform trial, said: \"These encouraging data reinforce the safety profile of eplontersen and demonstrate clear evidence of its potential to provide mu...