Intellia Therapeutics announced the initiation of HAELO, a global, pivotal Phase 3 study of NTLA-2002 for the treatment of hereditary angioedema (HAE). NTLA-2002 is a wholly owned investigational in vivo CRISPR-based gene editing therapy in development as a single-dose treatment for this potentially life-threatening disease.
Patient screening is active following Intellia’s successful end-of-Phase 2 meeting and submission of an Investigational New Drug Application amendment to the U.S. Food and Drug Administration (FDA).announced that data from the Phase 2 study of NTLA-2002 will be presented at the 2024 American College of Allergy, Asthma & Immunology (ACAAI) Annual Scientific Meeting, taking place 24-28 October 2024 in Boston, Massachusetts.
Intellia President and Chief Executive Officer, John Leonard MD, said: “Initiation of the HAELO Phase 3 trial is a significant milestone for Intellia as we enter the final stage of clinical development for NTLA-2002 for people living with hereditary angioedema. Data from the ongoing Phase 1/2 study showed great promise that a single-dose treatment can lead to a complete response – no more attacks and no further treatment required. We are working urgently to bring forward NTLA-2002 to address the real-world needs of people suffering from this disease and, ultimately, believe it will bring significant value to patients, physicians and payors.”
HAELO is a global, 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. The primary endpoint is the change in number of HAE attacks from week 5 through week 28.
Intellia is initiating the Phase 3 study based on positive safety and efficacy data from the ongoing Phase 1/2 study (NCT05120830) of NTLA-2002. Interim Phase 1 clinical data showed dramatic reductions in attack rate, as well as consistent, deep and durable reductions in kallikrein levels. Intellia previously announced positive toplines results from the Phase 2 portion of the study. The Company plans to present the detailed results at the 2024 American College of Allergy, Asthma & Immunology (ACAAI) Annual Scientific Meeting, taking place October 24 – 28 in Boston, Massachusetts.
(Source: Intellia)