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Ionis, AstraZeneca To Use Real-World Data To Accelerate RNA-Targeting Cholesterol Drug

Phase I Shows Promise, But ION449 Must Catch Up With Inclisiran

 
• By Alaric DeArment

Early data presented at the American Heart Association showed strong PCSK9 and LDL cholesterol lowering for ION449; AstraZeneca plans rapid development under an RWE-based FDA pilot program.

Cholesterol plaque in artery
Ionis and AstraZeneca's anti-PCSK9 drug shows promise in Phase I • Source: Shutterstock

Early data from the latest gene silencing-based drug targeting PCSK9 show a medicine with potentially strong efficacy in patients, but one that also faces some significant competitive hurdles, particularly as Alnylam Pharmaceuticals Inc./Novartis AG are poised to have the first-in-class advantage with inclisiran. However, Ionis Pharmaceuticals, Inc.’s ION449 may be able to catch up via the accelerated development plan that partner AstraZeneca PLC has in store for the drug, and it has a potential oral version in preclinical development.

AstraZeneca presented data from the Phase I study of ION449, also known as AZD8233, in a poster session at the...

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