AZ to drop Ionis RNA therapy for high cholesterol

Sept. 23, 2022

California-based biotech Ionis Pharmaceuticals announced this week that phase 2 trial data for its antisense oligonucleotide therapy ION 449 (also known as AZD8233) failed to impress AstraZeneca, and that the drug candidate won't be advancing into phase 3 development for the treatment of high blood cholesterol.

The decision was made based on results from the SOLANO study, a randomized parallel, double-blind, placebo-controlled study that included 411 patients with high lipid levels and meant to assess the efficacy, safety, and tolerability of the drug against a placebo. While the study met its primary efficacy endpoint, and ION449 showed to reduce low-density lipoprotein cholesterol (LDL-C) levels after 28 weeks compared to placebo, the results did not achieve prespecified efficacy criteria. 

Eugene Schneider, executive vice president, chief clinical development officer at Ionis did not specify what the criteria implicated, only saying that the results “did not meet AstraZeneca's target product profile criteria to invest in a broad Phase 3 development program.”

The drug is given as a monthly injection and uses the company’s investigational LIgand-Conjugated Antisense (LICA) technology platform to reduce plasma levels of a protein that is involved in the regulation of LDL-cholesterol.