Ionis looks to expand use of lipid-lowering drug after trial hits goal

Dive Brief: Ionis on Tuesday said its new lipid-lowering drug Tryngolza met the main goals of two Phase 3 clinical trials that could expand its use. Results showed the drug significantly reduced triglyceride levels in people with severely elevated levels of the fat in their bloods and prevented accompanying pancreatitis episodes. The company will ask the Food and Drug Administration by the end of the year to add severe hypertriglyceridemia to Tryngolza’s label. The drug, Ionis’ first wholly owned product, gained approval in December for a rare inherited condition called familial chylomicronemia syndrome, for which it recorded $25.6 million in sales over the first six months of 2025. Ionis shares rose as much as 34% in morning trading. Leerink Partners analyst Mani Foroohar wrote in a note to clients that the data “easily meet our blue-sky scenario” for the drug because it gives Tryngolza a “bolstered position in a multi-billion dollar” market. Dive Insight: Ionis specializes in drugs that block RNA responsible for production of proteins implicated in certain genetically driven diseases. For example, its drug Spinraza, which Biogen sells, treats spinal muscular atrophy, which can be a fatal condition for many babies and children. Until the launch of Tryngolza, Ionis’ strategy was to license drugs to larger partners like Biogen and AstraZeneca, which have larger commercial arms to reach more physicians and patients. Since Tryngolza, Ionis has also launched a second wholly owned drug called Dawnzera for hereditary angioedema. Tryngolza, which Ionis launched with a list price of $595,000 a year, is approved for a rare condition that affects as many as one in 300,000 people. The potential addition of people with severely elevated triglycerides — a condition that is growing with prevalence of obesity and diabetes — represents a large expansion opportunity for Tryngolza’s market. Called Core and Core2, Ionis’ trials enrolled 617 and 446 people with severely elevated triglycerides and randomized them to take 80 milligrams or 50 milligrams of Tryngolza every four weeks, or placebo. In Core, the 80 or 50 milligram doses of the drug reduced triglycerides by 73% and 63% respectively, and in Core2 the reductions were 68% and 63%, respectively. In Core, people who received placebo experienced triglyceride lowering of less than 1% on average, while in Core2 it was 14%. The reductions seen in people taking Tryngolza were statistically significant compared with the placebo. Treatment with Tryngolza reduced the incidence of acute pancreatitis by 85% compared with placebo, but Ionis didn’t release results from the individual dose arms. “These results reinforce our confidence that olezarsen has the potential to change the [severe hypertriglyceridemia] treatment paradigm,” said Sam Tsimikas, Ionis’ senior vice president for global cardiovascular development, in a statement. Jefferies analyst Akash Tewari raised his bank’s peak sales forecast for Tryngolza from $1.5 billion to $2.5 billion, with the elevated triglyceride indication in the U.S. accounting for about $1.5 billion of the total.