Ionis Pharmaceuticals, Inc. today announced it has expanded its existing collaboration with AstraZeneca 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 (ATTR) with AstraZeneca's global cardiovascular commercial capabilities.

Eplontersen is an investigational antisense medicine discovered by Ionis that reduces the production of transthyretin protein (TTR) to treat ATTR, a systemic, progressive, and fatal disease.

"The positive clinical results from our Phase 3 NEURO-TTRansform study, combined with eplontersen's self-administration profile, reinforce eplontersen's potential to be an important and differentiated new treatment option for patients with ATTR," said Brett P. Monia Ph.D., chief executive officer of Ionis. "With FDA review of eplontersen for ATTR polyneuropathy already underway and plans to file for regulatory approval in the EU and other countries later this year, today's agreement underscores our shared commitment to ensuring that this much needed treatment is made available to patients around the world."

AstraZeneca paid Ionis $20 million to license eplontersen in Latin America.

Under the terms of the collaboration agreement, Ionis is eligible to receive up to $3.6 billion in milestone and other payments. The collaboration includes territory-specific cost-sharing provisions. Ionis is also eligible to earn royalties in the range of low double-digit to mid-20s percentage depending on region.

Ionis and AstraZeneca are seeking regulatory approval for eplontersen for the treatment of hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) in the U.S. and plan to seek regulatory approval in the EU 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 (NCT04136171) 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.

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