Ionis Pharmaceuticals Reports Eplontersen Continued to Show Improvement in ATTRv-PN through 85 weeks

Ionis Pharmaceuticals (NASDAQ:IONS) (Nasdaq: IONS) today announced positive topline, 85-week data from the Phase 3 NEURO-TTRansform study in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN). Ionis and AstraZeneca's eplontersen continued to show sustained improvements in measures of neuropathy disease and a favorable safety and tolerability profile.

An overview of key results from the 85-week topline analysis is available here. 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 (mNIS+7). Eplontersen also showed continued improvement on the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN) compared to baseline. Results from the NEURO-TTRansform primary analysis at 66 weeks were presented earlier this year.

"These positive findings further strengthen eplontersen's efficacy and safety profile, underscoring its potential to be an important, differentiated advancement for patients with this progressive, debilitating and fatal disease," said Eugene Schneider, M.D., Ionis' executive vice president and chief clinical development officer. "A substantial number of eplontersen-treated patients showed improvement in neuropathy impairment and quality of life through 19 months of treatment. ATTRv-PN continues to be an underserved patient population and we look forward to working with regulatory authorities to bring this important new, self-administered treatment to patients."

Results from the 85-week exploratory analysis of NEURO-TTRansform will be submitted for presentation at an upcoming medical meeting. As part of a global development and commercialization agreement, Ionis and AstraZeneca are seeking regulatory approval for eplontersen for the treatment of ATTRv-PN in the U.S. and plan to seek regulatory approval in Europe and other parts of the world. The U.S. Food and Drug Administration accepted the New Drug Application for eplontersen for the treatment of ATTRv-PN with a PDUFA action date of Dec. 22, 2023. Eplontersen was granted Orphan Drug Designation in the U.S.

Eplontersen is currently being evaluated in the Phase 3 CARDIO-TTRansform study for transthyretin-mediated amyloid cardiomyopathy (ATTR-CM), a systemic, progressive and fatal condition that typically leads to progressive heart failure and often death within three to five years from disease onset.