Today, it was announced that new subgroup analyses from Ionis and AstraZeneca’s Phase 3 NEURO-TTRansform study of WAINUA™ (eplontersen) will be presented at the 2024 International Symposium on Amyloidosis (ISA) in Rochester, Minnesota, taking place from May 26 to May 30. WAINUA was granted approval by the U.S. Food and Drug Administration (FDA) in December 2023 for treating the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults, known as hATTR-PN or ATTRv-PN.
Highlighted presentations include:
- Analysis of Neuropathy Impairment and Nutritional Status in Patients with Hereditary Transthyretin-Mediated Amyloidosis Treated with Eplontersen Abstract ID 174: May 29, 2024, from 10:00-10:30 am ET (poster presentation 1) and 2:45-3:45 pm ET (poster presentation 2) Presenting Author: Jonas Wixner
- Exploratory Analysis of Eplontersen in Patients with Hereditary Transthyretin Amyloidosis with Polyneuropathy, focusing on the V30M TTR Variant and Early-Onset or Late-Onset Disease Abstract ID 230: May 29, 2024, from 10:00-10:30 am ET (poster presentation 1) and 2:45-3:45 pm ET (poster presentation 2) Presenting Author: Julian D. Gillmore
- Exploratory Analysis of Eplontersen in Male and Female Patients with Hereditary Transthyretin Amyloidosis with Polyneuropathy Abstract ID 202: May 29, 2024, from 10:00-10:30 am ET (poster presentation 1) and 2:45-3:45 pm ET (poster presentation 2) Presenting Author: Márcia Waddington Cruz
Under a global development and commercialization agreement, AstraZeneca and Ionis are collaborating to commercialize WAINUA for treating ATTRv-PN in the U.S. and are pursuing regulatory approval in Europe and other regions.
Eplontersen is currently under evaluation in the Phase 3 CARDIO-TTRansform study for adults with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM), a condition that leads to progressive heart failure and often death within three-to-five years from onset. The CARDIO-TTRansform Phase 3 study is the largest study conducted in this patient population to date, with over 1,400 enrolled participants.
About WAINUA™ (eplontersen) WAINUA™ (eplontersen) is a LIgand-Conjugated Antisense (LICA) medicine designed to inhibit the production of transthyretin, or TTR protein. It has been approved in the U.S. for treating the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults (ATTRv-PN).
Indication for WAINUA™ (eplontersen) WAINUA injection for subcutaneous use 45 mg is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.
Important Safety Information for WAINUA™ (eplontersen) Warnings and Precautions include Reduced Serum Vitamin A Levels and Recommended Supplementation. The most common adverse reactions (≥9% in WAINUA-treated patients) were decreased vitamin A levels (15%) and vomiting (9%).
About the NEURO-TTRansform study NEURO-TTRansform is a global, open-label, randomized trial assessing the efficacy and safety of eplontersen in patients with ATTRv-PN at weeks 35, 66, and 85.
About Hereditary Transthyretin-Mediated Amyloid Polyneuropathy (ATTRv-PN) ATTRv-PN is characterized by the accumulation of misfolded mutated TTR protein in the peripheral nerves, leading to debilitating nerve damage and loss of motor functions. It typically results in disability within five years of diagnosis and is often fatal within a decade.
About Ionis Pharmaceuticals, Inc. Ionis Pharmaceuticals, Inc. has a history of developing medicines for serious diseases. With five marketed medicines and a robust pipeline, Ionis is a leader in RNA-targeted medicines and gene editing.
Forward-looking Statements This press release contains forward-looking statements regarding eplontersen, Ionis’ business, and the therapeutic potential of its medicines and technologies. These statements involve certain risks and uncertainties, and Ionis does not undertake to update forward-looking statements. Risks related to Ionis’ programs are detailed in its filings with the SEC.
For more information about Ionis, visit Ionispharma.com and follow them on social media.
