Intellia Therapeutics’ CRISPR-based gene editing therapy significantly suppressed disease attack episodes in a Phase 3 study of hereditary angioedema, according to a June 16 report. Analysts say the results could set up a formidable challenge for Ionis Pharmaceuticals, whose own angioedema drug reached the U.S. market in August.
Intellia began a rolling application to the Food and Drug Administration for lonvo-z in April and expects to complete its submission in the second half of this year, William Blair said. A potential approval and launch are anticipated in the first half of 2027.
In the Phase 3 HAELO study, Intellia enrolled 80 patients with hereditary angioedema who were randomly assigned to receive placebo or a single intravenous infusion of Intellia’s investigational drug, called lonvoguran ziclumeran (lonvo-z). From weeks five through 28 of the trial, "the average number of monthly attacks that required on-demand treatment was 89% lower in those given lonvo-z versus placebo," according to a Saturday release. Over the same period, patients on lonvo-z saw "91% fewer moderate or severe HAE attacks per month." Quality of life improvements were also significantly greater among those treated with lonvo-z.
"Lonvo-z keeps pace with Dawnzera on HAE attack prevention," analysts at William Blair told investors in a note on Monday, referring to Ionis’ antisense oligonucleotide therapy. The product made $16 million in Q1, Ionis reported in April. Dawnzera’s launch "has beaten expectations to date," William Blair said Monday. The analysts added, "We view [the launch] as a positive outlook for the HAE market" more broadly and suggested that Intellia could position lonvo-z as an alternative treatment option dosed less frequently if it enters the HAE space.
“We view lonvo-z’s impressive one-and-done dosing paradigm and good safety profile as a potential different option for patients,” William Blair added.
Aside from overall efficacy data, Intellia touted time plot analysis showing that throughout follow-up when participants continued standard care, monthly HAE attack rates for all patients on lonvo-z remained below pre-screening levels. Analysts at Jefferies agreed with William Blair's assessment: “HAELO’s outcomes compare favorably to existing chronic [treatments] to position lonvo-z as a paradigm-shifting one-time therapy.”
