January 22, 2025
CAMBRIDGE, Mass., Jan. 22, 2025 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today announced the first patient has been dosed in the global Phase 3 study of NTLA-2002 for the treatment of hereditary angioedema (HAE). NTLA-2002 is a wholly owned investigational in vivo CRISPR-based therapy in development as a single-dose treatment for this potentially life-threatening disease. Intellia expects to complete enrollment in the second half of 2025 and submit a biologics license application (BLA) in 2026 to support the Company’s plans for a U.S. launch in 2027.
“We are pleased to have initiated dosing in the HAELO Phase 3 study as we are in our final lap of clinical development for NTLA-2002,” said Intellia President and Chief Executive Officer John Leonard, M.D. “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 1/2 study later this year highlighting the durability of effect of NTLA-2002.”
"We are excited to have treated the first patient in the U.S. with a new generation of therapy that could potentially provide patients with lifelong relief from the primary symptoms of HAE,” commented Dr. Joshua Jacobs, Medical Director, Allergy and Asthma Clinical Research, Inc.
About the HAELO Study
The pivotal Phase 3 HAELO clinical trial is a randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of NTLA-2002 in 60 adults with Type I or Type II HAE. Patients will be randomized 2:1 to receive a single 50 mg infusion of NTLA-2002 or placebo. Patients randomized to the placebo arm will be eligible for optional crossover to NTLA-2002 at week 28. Key endpoints include the number of HAE attacks and the number of patients who achieve attack-free status from week 5 through week 28. For more information on HAELO (NCT06634420), please visit clinicaltrials.gov.
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