Ionis Pharmaceuticals Advances Tryngolza Program with Positive Phase 3 Results in Moderate Hypertriglyceridemia

Ionis Pharmaceuticals has reported promising results from its phase 3 Essence study, evaluating Tryngolza in patients with moderate hypertriglyceridemia who have or are at risk for atherosclerotic cardiovascular disease (ASCVD). The study demonstrated statistically significant reductions in triglyceride (TG) levels, marking an important step in the company's efforts to expand the drug's reach beyond its current approval for familial chylomicronemia syndrome (FCS).

Essence Study Results and Implications

Tryngolza, administered at monthly doses of 80 mg and 50 mg, yielded placebo-adjusted TG reductions of 61% and 58%, respectively, after six months of treatment. The majority of patients in the study achieved normal TG levels, underscoring the drug's potential efficacy in managing hypertriglyceridemia, a condition characterized by abnormally high TG levels that can increase the risk of cardiovascular disease.

Dr. Sam Tsimikas, Senior Vice President of Global Cardiovascular Development at Ionis, emphasized the significance of these results, stating, "This is an important step in bringing forward a potential new treatment for people with severely elevated triglycerides." The positive outcome supports Tryngolza's potential to benefit a broader population beyond the rare disease FCS, for which it is currently approved.

Expanding Tryngolza's Reach

Ionis is currently conducting two additional phase 3 studies, CORE and CORE2, evaluating Tryngolza in severe hypertriglyceridemia (sHTG). Results from these trials are expected in the third quarter of this year, with the company aiming to file for an additional indication by year-end if successful.

The potential approval for sHTG would significantly expand Tryngolza's market reach. While FCS affects only 1 in 1-2 million people worldwide, sHTG affects approximately 3 million individuals in the United States alone. This expansion aligns with Ionis' strategy to evolve into a fully integrated, commercial-stage biotechnology company, as outlined by CEO Brett Monia.

Commercial Performance and Competition

Tryngolza, approved by the FDA in December for FCS treatment, marked Ionis' first independent launch in the United States. In its first full quarter on the market, the drug generated $6 million in U.S. sales. Ionis has partnered with Sobi for commercialization rights outside the U.S., Canada, and China.

As Ionis advances its Tryngolza program, competition looms on the horizon. Arrowhead Pharmaceuticals is developing plozasiran, an RNA interference candidate for FCS, with a potential FDA approval decision expected in November.