Benzinga | Nov 12, 2020 07:12AM EST

Ionis Pharmaceuticals Announces Upcoming Presentations At AHA Scientific Sessions Nov. 13-17

Ionis' novel antisense medicine targeting PCSK9 demonstrates best-in-class potential for the treatment of patients with high cholesterol at risk for cardiovascular disease

CARLSBAD, Calif., Nov. 12, 2020 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ:IONS), announced today the upcoming presentations of clinical and non-clinical data for several of its investigational antisense medicines for cardiovascular disease at the American Heart Association (AHA) Scientific Sessions 2020, to be held virtually November 13-17, 2020.

Two of the abstracts accepted for presentation will provide new data from studies of ION449, also known as AZD8233 for subcutaneous administration and AZD6615 for oral administration. ION449 is an investigational antisense medicine targeting PCSK9, which is integrally involved in the regulation of LDL-cholesterol. ION449 is being developed as part of a collaboration between Ionis and the biopharmaceutical company AstraZeneca. Data presented include a study evaluating the safety, tolerability, pharmacokinetics and pharmacodynamics of single ascending subcutaneous doses of ION449 in patients with elevated LDL-cholesterol and a separate pre-clinical study evaluating this investigational medicine for once-daily oral dosing.

Additional presentations by Ionis scientists will include a review of RNA-targeted treatments designed to inhibit the production of apolipoprotein C-III (apoC-III) and lipoprotein(a) or Lp(a). The apoC-III protein is an independent cardiovascular risk factor that regulates triglyceride metabolism in the blood. Ionis' AKCEA-APOCIII-LRx is an investigational LIgand-Conjugated Antisense (LICA) medicine designed to inhibit the production of apoC-III for patients who are at risk for cardiometabolic disease due to elevated triglyceride levels. AKCEA-APOCIII-LRx is expected to start a Phase 3 study in patients with familial chylomicronemia syndrome (FCS) by year end. Elevated Lp(a) is an independent, genetic risk factor for cardiovascular disease that cannot be well controlled with lifestyle modifications such as diet or exercise or with treatment using existing lipid-lowering therapies. Ionis' pelacarsen is an investigational LICA medicine designed to reduce the production of apolipoprotein(a) in patients who are at risk for cardiometabolic disease due to high Lp(a). Pelacarsen is currently in a Phase 3 cardiovascular outcomes study.

Following are presentations of Ionis programs and collaborations:

Cardiovascular Seminars:

* Friday, November 13, 11:10 a.m. CST 'Experimental Therapy for APOCIII Lowering: ASO, siRNA,' Rosanne Crooke, Ph.D., senior strategic consultant at Ionis

* Friday, November 13, 11:20 a.m. CST 'Experimental Therapy for Lp(a) Lowering,' Sotirios "Sam" Tsimikas, M.D., Ph.D., senior vice president, clinical development and leader, cardiovascular franchise

Poster Presentations in Collaboration with AstraZeneca:

* Friday, November 13, 9:00 a.m. CST 'An Oral Antisense Oligonucleotide for PCSK9 Inhibition in Humans' (Poster #P244)

* Friday, November 13, 9:00 a.m. CST 'Single Dose Safety, Pharmacokinetics, and Pharmacodynamics of a Potent PCSK9 Synthesis Inhibitor, AZD8233, in Subjects With Elevated LDL Cholesterol' (Poster #MP515)

A full list of presentations can be found on the AHA Scientific Sessions website.