Pharma BD Deal Intelligence
AstraZeneca and Ionis Pharmaceuticals entered a global development and commercialization agreement for eplontersen, Ionis' LIgand-Conjugated Antisense (LICA) medicine that inhibits production of transthyretin (TTR) protein for the treatment of transthyretin amyloidosis (ATTR). Under the terms, Ionis received a $200 million upfront payment, was eligible for up to $485 million in development and approval milestones and up to $2.9 billion in sales-related milestones, plus tiered royalties ranging from low double-digit to mid-20s percent depending on region. The companies jointly develop and commercialize eplontersen in the U.S., while AstraZeneca holds exclusive rights to commercialize in the rest of the world. Hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN) was the first indication: eplontersen was approved by the U.S. FDA on December 21, 2023 under the brand name Wainua, the only ATTRv-PN treatment that can be self-administered via auto-injector. The drug is also being evaluated in the Phase 3 CARDIO-TTRansform study (>1,400 patients) for the much larger ATTR cardiomyopathy (ATTR-CM) indication, with topline data expected in the second half of 2026.