Pharma BD Deal Intelligence

Akcea Therapeutics, Inc. / Ionis Pharmaceuticals, Inc.

2018 · Licensing/Option · $1.7B · Complete

CALLED IT — OFF BY 14

The Akcea rare-disease commercialization bet collapsed within a year of full buyout — Ionis gutted 70% of the acquired workforce and outsourced the flagship product line it had just paid to bring in-house.

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The coverage arc

Oct 05, 2018 BioPharma Dive Neutral

FDA clears rare disease therapy from Akcea, Ionis; Tegsedi enters market with $450,000 annual price tag and a black-box warning matching its main RNAi…

Sep 09, 2019 BioPharma Dive Bearish

Three Akcea executives including the CEO depart in a 'disconcerting surprise' as Tegsedi launch underperforms expectations and the company struggles against…

Aug 31, 2020 BioPharma Dive Bearish

Ionis reels its spinoff Akcea back in after disappointing commercial execution on Tegsedi and Waylivra, effectively unwinding the 2018 partnership structure.

Source summaries from our enrichment pipeline; follow links for originals.

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Ionis licensed worldwide rights to Akcea for inotersen (Tegsedi) and follow-on AKCEA-TTR-LRx in March 2018. Akcea paid $150M upfront in stock plus Ionis purchased $200M of Akcea common stock; total deal up to $1.7B including $1.3B in commercial milestones. Profit-sharing 60/40 in Ionis's favor for inotersen, transitioning to 50/50 after AKCEA-TTR-LRx launch.

Did it work? Outcome assessment

The Akcea rare-disease commercialization bet collapsed within a year of full buyout — Ionis gutted 70% of the acquired workforce and outsourced the flagship product line it had just paid to bring in-house.

Strategic verdict
Failed to Achieve
Financial impact
Neutral
The 2018 license (up to ~$1.7B in fees/milestones plus profit-share) moved the TTR franchise to majority-owned affiliate Akcea, but Ionis reversed course and bought in the remaining ~24% of Akcea it did not own for ~$500M in October 2020, fully reabsorbing the franchise. Lead product Tegsedi generated only modest revenue (single-digit to low-double-digit $M per quarter in 2019), reflecting commercial underperformance versus competing TTR therapies.
Pipeline outcome
Mixed
Inotersen (Tegsedi) was approved and launched in the US, EU and Canada in 2018 for hATTR polyneuropathy but achieved limited commercial uptake against Alnylam's patisiran/Onpattro. The second licensed asset, AKCEA-TTR-LRx (eplontersen), advanced through development and was later partnered with AstraZeneca (Dec 2021), ultimately approved as Wainua in late 2023, just beyond this 5-year window.

Key facts

Disease & market context

Hereditary transthyretin amyloidosis (hATTR) polyneuropathy

Disease Overview

Hereditary transthyretin amyloidosis is a rare, progressive, and fatal genetic disease caused by misfolded TTR protein depositing as amyloid in nerves and heart, producing polyneuropathy and/or cardiomyopathy. Median onset is age 68 in the US; without treatment median survival is 5-15 years from symptom onset.

Competitive Landscape

The hATTR market in 2018 became a three-horse race. Alnylam's Onpattro (patisiran) launched first in August 2018 as the first approved RNAi drug, an IV every-3-weeks infusion silencing TTR mRNA. Akcea/Ionis's Tegsedi (inotersen) followed in October 2018 as a once-weekly subcutaneous antisense oligonucleotide but carried black-box warnings for thrombocytopenia and glomerulonephritis requiring weekly platelet and renal monitoring. Pfizer's Vyndaqel/Vyndamax (tafamidis), a TTR stabilizer approved May 2019 for cardiomyopathy, dominated the larger ATTR-CM segment. Survey data showed clinicians strongly favored Onpattro over Tegsedi for polyneuropathy due to safety profile, with Onpattro outpacing Tegsedi by ~4x in net revenue ($38M vs $10M in Q2 2019). Tegsedi sales never reached blockbuster expectations; combined Akcea revenue was only ~$70M in 2020. Ionis re-acquired Akcea in 2020 and licensed follow-on AKCEA-TTR-LRx (eplontersen) to AstraZeneca for $200M upfront in 2021, effectively writing off this deal's commercial thesis.

Related deals — scored

DealYearValueOutcome
Akcea Therapeutics, Inc. / Ionis Pharmaceuticals, Inc. (this deal)2018$1.7B27
Pfizer Inc. / FoldRx Pharmaceuticals201099
Abbott Laboratories / Knoll Pharmaceuticals (BASF Pharma)2000$6.9B95
Astra AB / Zeneca Group plc1998$35.0B94
Merck & Co. Inc. / Acceleron Pharma Inc.2021$11.5B90
Johnson & Johnson / Actelion Ltd2017$30.0B90
Eli Lilly and Company / Boehringer Ingelheim GmbH2011$444M89

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