NTLA - Intellia Therapeutics Announces Positive Long-Term Data from Ongoing Phase 1 Study of NTLA-2002 an Investigational In Vivo CRISPR Gene Editing Treatment for Hereditary Angioedema (HAE) | Benzinga

• Extended follow-up data reaching over two years in the earliest patients dosed reinforce the potential of NTLA-2002 to be a functional cure for people living with hereditary angioedema (HAE)
  
• Eight of 10 patients remain completely attack-free following the 16-week primary observation period through the latest follow-up, including patients with the most severe disease
• Single dose of NTLA-2002 led to a 98% mean reduction in monthly HAE attack rate, with an average follow-up of over 20 months across all patients
• 100% of patients who discontinued prophylaxis treatment after NTLA-2002 remain free of chronic prophylaxis treatment
• Favorable safety and tolerability profile observed at all dose levels
• Intellia to host investor webcast on Monday, June 3, at 8 a.m. ET

CAMBRIDGE, Mass., June 02, 2024 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on revolutionizing medicine with CRISPR-based therapies, today announced long-term data from the Phase 1 portion of the ongoing Phase 1/2 study of NTLA-2002. NTLA-2002 is an investigational in vivo CRISPR-based gene editing therapy in development as a single-dose treatment for hereditary angioedema (HAE), a rare genetic condition that leads to potentially life-threatening swelling attacks. The data were shared in an oral presentation at the European Academy of Allergy and Clinical Immunology (EAACI) Congress 2024, being held May 31 – June 3 in Valencia, Spain.

"These unprecedented data strengthen our view that NTLA-2002 could be a groundbreaking treatment for people living with hereditary angioedema," said Intellia President and Chief Executive Officer John Leonard, M.D. "After a single dose of our investigational in vivo CRISPR-based therapy, patients experienced durable elimination of their attacks. We are thrilled to see that the majority of patients have been attack free for over 18 months or longer. These remarkable attack rate reductions have been consistent, even in patients with the most severe symptoms. At the same time, the data from these 10 patients continue to demonstrate a very favorable safety profile. These long-term data provide strong evidence that NTLA-2002 could be a one-time, potential functional cure for this debilitating and life-threatening disease."

In the Phase 1 portion of the study, single doses of 25 mg (N=3), 50 mg (N=4) and 75 mg (N=3) of NTLA-2002 were administered via intravenous infusion, and HAE attacks and plasma kallikrein protein levels were measured for each patient. The first analysis of HAE attack rates occurred at the end of the pre-specified 16-week primary observation period. HAE attacks and plasma kallikrein protein levels will continue to be assessed through the remainder of the two-year follow-up period.

HAE Attack Rate Reduction¹

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