Intellia Therapeutics has dosed the first subject in a randomised, placebo-controlled global Phase III trial of its investigational CRISPR-based therapy, NTLA-2002, to treat hereditary angioedema (HAE).
HAELO is a double-blind trial that will assess the therapy’s safety and efficacy in 60 adult subjects with Type I or Type II HAE.
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Subjects will be randomised to receive either one 50 mg infusion of the therapy or a placebo. Those in the placebo group will have the option to cross over to the therapy treatment at a 28-week time period.
The trial’s key endpoints will measure the count of HAE attacks and the proportion of subjects achieving attack-free status between week five and week 28.
Intellia Therapeutics CEO and president John Leonard said: “We are pleased to have initiated dosing in the HAELO Phase III study as we are in our final lap of clinical development for NTLA-2002. With the promising data we’ve presented thus far, we believe patients could achieve independence from both HAE attacks and medications required to treat this disease.
“We look forward to presenting longer-term data from the ongoing Phase I/II study later this year highlighting the durability of the effect of NTLA-2002.”
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By GlobalDataSubject enrolment is anticipated to be completed by the second half of this year and the company aims to submit a biologics license application (BLA) in 2026.
The therapy launch in the US market is expected in 2027.
Leveraging CRISPR/Cas9 technology, NTLA-2002 aims to target the kallikrein B1 (KLKB1) gene to eliminate attacks.
NTLA-2002 has received several regulatory designations, including the Food and Drug Administration’s (FDA) orphan drug and Regenerative Medicine Advanced Therapy (RMAT) designation, and the UK Medicines and Healthcare Products Regulatory Agency’s (MHRA) Innovation Passport.
It also secured the European Medicines Agency (EMA)’s PRIME designation, and orphan drug status from the European Commission (EC).
In October last year, the company reported positive outcomes from its ongoing Phase I/II trial of NTLA-2002 for HAE.
