Ionis expands collaboration with AstraZeneca for eplontersen

Ionis Pharmaceuticals (IONS) announced it has expanded its existing collaboration with AstraZeneca (AZN) to include exclusive rights for AstraZeneca to commercialize eplontersen in Latin America. Ionis previously granted AstraZeneca exclusive rights to commercialize eplontersen in all other countries outside the U.S. Ionis and AstraZeneca will continue to jointly develop and commercialize eplontersen in the U.S. The companies are successfully advancing eplontersen by combining Ionis’ industry-leading expertise in RNA-targeted therapeutics and deep knowledge of transthyretin amyloidosis with AstraZeneca’s global cardiovascular commercial capabilities. Eplontersen is an investigational antisense medicine discovered by Ionis that reduces the production of transthyretin protein, or TTR, to treat ATTR, a systemic, progressive, and fatal disease. AstraZeneca paid Ionis $20M to license eplontersen in Latin America. Under the terms of the collaboration agreement, Ionis is eligible to receive up to $3.6B in milestone and other payments.Ionis and AstraZeneca are seeking regulatory approval for eplontersen for the treatment of hereditary transthyretin-mediated amyloid polyneuropathy in the U.S. and plan to seek regulatory approval in the EU and other parts of the world. The FDA accepted the New Drug Application for eplontersen for the treatment of ATTRv-PN with a PDUFA action date of Dec. 22. 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, a systemic, progressive and fatal condition that typically leads to progressive heart failure and often death within three to five years from disease onset.