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 American Heart Association’s 2020 conference. The drug is described as a generation 2
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