Het grote nieuws bij concurrenten voor Pharming blijft binnen stromen.
Zoals ik al eens eerder zei is 2024 het jaar dat er meer duidelijkheid komt bij concurrentie.
Intellia Therapeutics Announces Initiation of HAELO Phase 3 Study of NTLA-2002,
an Investigational In Vivo CRISPR Gene Editing Treatment for Hereditary Angioedema (HAE)
October 7, 2024
NTLA-2002 is a single-dose treatment designed to prevent potentially life-threatening swelling attacks in people with hereditary angioedema (HAE)
NTLA-2002 is Intellia’s second in vivo candidate to enter late-stage clinical development from its modular gene editing platform
CAMBRIDGE, Mass., Oct. 07, 2024 (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 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).
“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,” said Intellia President and Chief Executive Officer John Leonard, M.D. “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.