Carlsbad:  Ionis Pharmaceuticals, Inc. has announced positive topline results from a 35-week interim analysis of the Phase 3 NEURO-TTRansform study of Ionis and AstraZeneca’s eplontersen in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN). Eplontersen demonstrated a statistically significant and clinically meaningful change from baseline for the co-primary endpoints of percent change in serum transthyretin (TTR) concentration and the modified Neuropathy Impairment Score +7 (mNIS+7), a measure of neuropathic disease progression, versus the historical placebo group. Eplontersen also met its key 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 versus the historical placebo group.

In the study, eplontersen demonstrated a favorable safety and tolerability profile with no specific concerns. The study data are consistent with the clinical profile seen across Ionis’ other LICA programs, further validating how advancements in the company’s LIgand-Conjugated Antisense technology position Ionis to deliver potentially transformative treatments for a range of unmet medical needs.

Based on these study results, the companies will seek regulatory approval for eplontersen for ATTRv-PN and plan to file a new drug application with the U.S. Food and Drug Administration this year.

“These encouraging data reinforce the safety profile of eplontersen and demonstrate clear evidence of its potential to provide much needed therapeutic benefit to patients living with hereditary transthyretin-mediated amyloid polyneuropathy,” said 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 study.

“We are pleased that the data in the NEURO-TTRansform study demonstrate eplontersen had a positive impact on disease progression, including improvement in neuropathy impairment and quality of life in a substantial number of patients. These highly statistically significant and clinically meaningful results put us on the cusp of providing a new therapeutic option for polyneuropathy patients living with this debilitating and fatal disease,” said Eugene Schneider, M.D., Ionis’ executive vice president and chief clinical development officer. “We are grateful to the patients, families and clinicians who are participating in NEURO-TTRansform. Without their commitment the eplontersen program would not have progressed as successfully as it has.”

The data from the 35-week interim analysis will be presented at an upcoming medical meeting later this year.

As part of a global development and commercialization agreement between Ionis and AstraZeneca, eplontersen is being jointly developed and commercialized by both companies in the U.S. and will be developed and commercialized in the rest of the world by AstraZeneca (with the exception of Latin America).

Eplontersen was granted Orphan Drug Designation in the U.S. It is also currently being evaluated in the Phase 3 CARDIO-TTRansform study for amyloid transthyretin cardiomyopathy (ATTR-CM), a systemic, progressive and fatal condition that leads to progressive heart failure and death within four years from diagnosis.

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