On a mission to expand its recently approved Tryngolza beyond the rare genetic disorder familial chylomicronemia syndrome (FCS), Ionis recently tested its medicine in patients with moderate hypertriglyceridemia while awaiting key results in the severe form of the condition.
The attempt was a success, Ionis reported, as treatment with Tryngolza yielded statistically significant placebo-adjusted reductions in patients' triglyceride (TG) levels. Specifically, treatment with Tryngolza was associated with TG reductions of 61% and 58% after six months with 80 mg and 50 mg monthly doses, respectively.
The company's phase 3 Essence study tested the therapy in patients with moderate hypertriglyceridemia who have or are at risk for atherosclerotic cardiovascular disease (ASCVD).
Hypertriglyceridemia is hallmarked by abnormally high TG levels, which can increase the risk of cardiovascular disease. The "vast majority" of patients in the Essence study achieved a reduction to normal TG levels through Tryngolza treatment, Ionis said in a press release.
Ionis sees the results as an “important step in bringing forward a potential new treatment for people with severely elevated triglycerides,” the company’s senior vice president of global cardiovascular development, Sam Tsimikas, M.D., said in the release.
Following Tryngolza’s “encouraging launch” in FCS, the data support the drug’s potential to “benefit the much broader population of people living with [severe hypertriglyceridemia],” Tsimikas added.
Tryngolza is being evaluated in severe hypertriglyceridemia (sHTG) through the company’s phase 3 CORE and CORE2 studies, which are slated for third-quarter readouts this year. With potential success in those trials, Ionis hopes to file a bid for the additional indication by the end of this year, according to Tsimikas.
That indication is key for Tryngolza, which is currently approved to treat rare disease FCS, which affects 1 out of 1-2 million people worldwide.
A potential second nod sHTG would allow the therapy to reach a much larger patient population of about 3 million in the U.S. alone.
As for the Essence result in moderate hypertriglyceridemia, Ionis' release didn't get into the endpoint of time to TG lowering. Still, this will be an "important metric" likely to be presented at an upcoming medical conference, William Blair analysts highlighted in a note to clients.
The TG-lowering measures reported in the Essence study topped those seen in Ionis' previous study in FCS, the analysts pointed out, which ultimately led to the drug's approval in that disease.
William Blair analysts called the newest results a "positive step forward" for Ionis' Tryngolza program, with the TG lowering data marking a probable indicator of efficacy in the CORE and CORE2 studies.
Tryngolza won its FDA nod in December, marking the company’s first independent launch and the first med cleared to treat FCS in the U.S. While Ionis has since dealt Sobi the drug's commercialization rights outside of the U.S., Canada and China, the company has so far racked up $6 million in U.S. sales for Tryngolza over its first full quarter on the market.
The drug is the first of four launches Ionis has planned through 2027, and its approval marked a “new chapter” for the company as a “fully integrated, commercial-stage biotechnology company,” CEO Brett Monia said in a statement at the time.
Elsewhere in FCS and sHTG, Arrowhead is advancing its RNA interference candidate, plozasiran. That drug is up for a potential FDA approval in FCS in November.