Ionis Pharmaceuticals (NASDAQ:IONS) and its majority-owned spinoff Akcea Therapeutics (AKCA) work together on RNA-based therapies for a wide spectrum of diseases. On January 22, 2020, they announced that the potential therapy AKCEA-APOCIII-LRx for hypertriglyceridemia (high triglycerides in the blood) displayed positive Phase 2 results. While a confirmatory Phase 3 trial will be necessary to obtain commercial approval, if all goes well this could start boosting revenue in 2022 or 2023.
Akcea was spun off from Ionis, which now owns about 75% of it. Because Ionis shareholders in effect own Akcea, but not vice-versa, this article will analyze the new results from the point of view of Ionis. Currently, Ionis has the most lucrative commercial franchise, royalties from Biogen (BIIB) for Spinraza. Akcea uses Ionis's antisense RNA technology to focus on rare diseases and lipid disorders, of which hypertriglyceridemia is an example.
Ionis is well off its 52-week high of $86.58, for no particularly good reason, so I see this as a good opportunity to buy it at a reasonable price. The 52-week low was $53.34. There are many components to the Ionis outlook. Since I want to focus on the hypertriglyceridemia opportunity, I refer you to my prior article, "Ionis: A Long-Term Buy On Earnings, Pipeline," for other aspects of the company's story.
AKCEA-APOCIII-LRx Phase 2 results
Phase 2 AKCEA-APOCIII-LRx top line results were released on January 22. The study showed statistically significant dose-dependent reductions in fasting triglycerides compared to placebo at all APOCIII dose levels. The highest once-monthly dose of 50 mg resulted in more than 90% of patients achieving serum triglycerides of less than 150 mg/dL, compared to less than 5% of patients in the placebo group. There were also significant reductions in other risk factors, notably apo-CIII. Safety was essentially the same in the dosed and placebo groups. The 150 mg/dL triglyceride level is the recognized
