Akcea Therapeutics, Inc.
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Ionis will seek US and EU approvals to treat familial chylomicronemia syndrome in the first half of 2024. Phase III data in the larger severe hypertriglyceridemia indication are due in late 2024 or early 2025.
Plus deals involving ProQR/ Thea, Aeglea/Immedica, Ayala/Biolight, Quince/EryDel, ImmunoBiochem/ImmunoGen and GSK/LimmaTech, as well as deals in brief.
Ionis will launch its first commercial product through a co-commercialization agreement with AstraZeneca early next year, in preparation for its first wholly-owned drug launches soon to follow.
The US firm has sold some of its royalty stream for the gene therapy Spinraza and cardiovascular drug candidate, pelacarsen, to Royalty Pharma for $500m upfront, which should help fund upcoming milestones for Ionis’ late-stage pipeline.
- Antisense, Oligonucleotides
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