With a one-time dosing profile, Intellia Therapeutics’ gene editing asset could be “paradigm-shifting” for hereditary angioedema, according to Jefferies. The biotech anticipates market approval next year.
Intellia Therapeutics’ CRISPR-based gene editing therapy significantly suppressed disease attack episodes in a Phase 3 study of hereditary angioedema. The results, analysts say, could set up a formidable challenge for Ionis Pharmaceuticals, whose own angioedema drug hit the U.S. market in August.
Intellia in April kicked off a rolling application to the FDA for lonvo-z, and the biotech 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 (HAE) 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 observation period, patients on lonvo-z saw 91% fewer moderate or severe HAE attacks per month. Quality of life improvements were likewise significantly greater in those treated with the gene editing asset.
“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 on Monday, which could mean good news for lonvo-z. “We view [the launch] as a positive outlook for the HAE market” more broadly, the analysts said. Intellia, if it enters the HAE space, could position lonvo-z as an alternative treatment option dosed less frequently.
“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 on Saturday touted time plot analysis showing that throughout the follow-up period, when participants were still being treated with standard of care, monthly HAE attack rates for all patients on lonvo-z remained below pre-screening levels.
Altogether, these findings paint a “strong profile” for lonvo-z, according to analysts at Jefferies, who agreed with William Blair’s take on the gene editor’s competitive positioning. HAELO’s outcomes “compare favorably to existing chronic [treatments] to position lonvo-z as a paradigm-shifting” one-time therapy, Jefferies said.
