Pharma BD Deal Intelligence
AstraZeneca's $4.2B licensing deal for Ionis' eplontersen (Wainua) delivered a real approval and a first-mover self-administered auto-injector for hereditary ATTR amyloidosis, but the real payoff hinges on the much larger ATTR cardiomyopathy indication still pending Phase 3 data.
AstraZeneca and Ionis signed a deal to develop and commercialize eplontersen, with AstraZeneca paying $200 million upfront and up to $485 million in…
Wainua (eplontersen) granted first-ever regulatory approval in the US for the treatment of adults with polyneuropathy of hereditary transthyretin-mediated…
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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.
10K US cases/yr · $3.5B Global ATTR Market (2024)
Hereditary transthyretin-mediated amyloidosis (hATTR) causes progressive polyneuropathy from misfolded TTR protein deposits on nerves. Wainua (eplontersen) is a self-administered antisense therapy that reduces TTR production by 81%, offering convenience advantages over IV-administered alternatives.
Wainua (eplontersen) was FDA-approved December 2023 with monthly self-injection convenience. Competitors include Onpattro (patisiran, IV), Amvuttra (vutrisiran, quarterly SC), and Tegsedi (inotersen, weekly SC). Wainua targets expansion into ATTR cardiomyopathy, a much larger market.
AstraZeneca licensed eplontersen and other antisense therapies from Ionis for cardiovascular/renal diseases.
FDA approved for polyneuropathy of hereditary ATTR amyloidosis. EU/UK approvals followed 2024-2025.
Wainua approved in US, UK, EU. Global launches underway with additional submissions in progress.
| Deal | Year | Value | Outcome |
|---|---|---|---|
| AstraZeneca PLC / Ionis Pharmaceuticals, Inc. (this deal) | 2021 | $4.2B | 73 |
| AstraZeneca PLC / Daiichi Sankyo Company, Limited | 2019 | $6.9B | 100 |
| AstraZeneca PLC / KuDOS Pharmaceuticals Limited | 2005 | $210M | 98 |
| AstraZeneca PLC / Alexion Pharmaceuticals Inc. | 2020 | $39.0B | 86 |
| AstraZeneca PLC / Acerta Pharma | 2015 | $4.0B | 86 |
| AstraZeneca PLC / Amgen Inc. | 2020 | $2.3B | 84 |
| AstraZeneca PLC / MedImmune Inc. | 2007 | $15.6B | 82 |
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