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CARLSBAD, Calif., Sept. 28, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that The Journal of the American Medical Association (JAMA) published positive results from the Phase 3 NEURO-TTRansform study of eplontersen, an investigational treatment for hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN).
Results from the week 66 primary analysis showed that eplontersen-treated patients demonstrated improvement across all co-primary and secondary endpoints, including serum transthyretin (TTR) concentration, neuropathy impairment and quality of life, compared to the external placebo group. An end-of-treatment analysis also showed eplontersen continued to demonstrate sustained improvements through 85 weeks.
"The totality of positive, consistent eplontersen data position this therapy, which can be self-administered, to be an important and empowering potential new medicine for treating hereditary transthyretin-mediated amyloid polyneuropathy," said Sami Khella, M.D., chief, department of neurology at Penn Presbyterian Medical Center, professor of clinical neurology at the Perelman School of Medicine at the University of Pennsylvania School of Medicine and a principal investigator on the NEURO-TTRansform trial. "Without treatment, hereditary transthyretin-mediated amyloid polyneuropathy is a debilitating and devasting disease that can ultimately result in death. The JAMA publication reinforces the growing body of evidence showing that eplontersen significantly reduces serum transthyretin concentration, may halt progression of neuropathy impairment, and improves overall patient quality of life, providing hope to this community."
"These data reinforce the ability of eplontersen to halt disease progression and improve quality of life throughout the 19-month treatment period," said Eugene Schneider, M.D., executive vice president and chief clinical development and operations officer for Ionis. "We look forward to the upcoming FDA action date in December and bringing eplontersen to this underserved patient community in the U.S. and around the world."
In the NEURO-TTRansform Phase 3 study, patients treated with eplontersen demonstrated consistent and sustained benefit on the three co-primary endpoints of serum transthyretin (TTR) concentration, neuropathy impairment measured by modified Neuropathy Impairment Score +7 (mNIS+7) and quality of life (QoL) on the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN):
