Ionis Pharmaceuticals announces ‘positive’ results from Phase 3 NEURO-TTRansform

Ionis Pharmaceuticals (IONS) announced positive topline, 85-week data from the Phase 3 NEURO-TTRansform study in patients with hereditary transthyretin-mediated amyloid polyneuropathy. Ionis and AstraZeneca’s (AZN) eplontersen continued to show sustained improvements in measures of neuropathy disease and a favorable safety and tolerability profile. At 85 weeks, eplontersen continued to show a sustained reduction in serum TTR concentration compared to baseline and continued to halt disease progression as measured by the modified Neuropathy Impairment Score +7. Eplontersen also showed continued improvement on the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy compared to baseline. Results from the NEURO-TTRansform primary analysis at 66 weeks were presented earlier this year.