Intellia Therapeutics has announced positive Phase 2 data for NTLA-2002, an investigational therapy for hereditary angioedema (HAE). The results, published in The New England Journal of Medicine, indicate significant reductions in monthly attack rates for HAE patients following a single dose of the CRISPR-based gene editing therapy.
The Phase 2 study, part of an ongoing Phase 1/2 trial, involved 27 participants who received either a 25 mg or 50 mg single dose of NTLA-2002 or a placebo via intravenous infusion. The 50 mg dose of NTLA-2002 led to a mean monthly attack rate reduction of 77% during weeks 1-16 and 81% during weeks 5-16, compared to placebo. Notably, eight out of eleven patients in the 50 mg arm were attack-free at the latest follow-up.
HAE is a rare genetic condition characterized by severe, recurring, and unpredictable inflammatory attacks, which can be life-threatening. Current treatments often require chronic administration and may not completely eliminate attacks. NTLA-2002 aims to inactivate the gene responsible for these attacks, offering a potential one-time treatment solution.
Key Findings from the Phase 2 Trial
Patients in the 50 mg dose group experienced the most significant attack rate reductions and a greater reduction in kallikrein protein, a key mediator of HAE attacks. The therapy was well-tolerated across both dose levels, with the most frequent adverse events being mild, such as headache and fatigue. No serious adverse events related to NTLA-2002 were reported.
Implications for HAE Treatment
These results suggest that NTLA-2002 could potentially serve as a functional cure for HAE, offering a significant improvement over existing treatments that require chronic administration. Intellia has selected the 50 mg dose for further evaluation in the global pivotal Phase 3 HAELO study, which is currently recruiting patients. The company aims to redefine the treatment paradigm for HAE, offering hope for a lifetime free from chronic therapy.
Ongoing Clinical Development
Intellia is also advancing other clinical programs, including NTLA-3001 for Alpha-1 Antitrypsin Deficiency (AATD) associated lung disease. The company reported a strong financial position, with $939.9 million in cash reserves, expected to fund operations into late 2026. A pivotal Phase 3 trial for transthyretin amyloid cardiomyopathy (TTR-CM) has also started across 35 global sites.
