Global Phase 3 Trial of Intellia Therapeutics' Lonvoguran Ziclomeran completed for Hereditary Angioedema Treatment
Intellia Therapeutics, Inc. (NASDAQ: NTLA), a leading clinical-stage gene editing company, is making significant strides in the development of lonvoguran ziclumeran, a potential one-time treatment for hereditary angioedema (HAE). Lonvoguran ziclumeran, based on Nobel Prize-winning CRISPR/Cas9 technology, is designed to prevent HAE attacks by inactivating the kallikrein B1 (KLKB1) gene. The company is on track to submit a biologics license application (BLA) in the second half of 2026 for this groundbreaking therapy.
Intellia Therapeutics' people are helping set the standard for a new class of medicine. They have a deep scientific, technical, and clinical development experience, and they continue to expand the capabilities of their CRISPR-based platform.
The Phase 3 HAELO study of lonvoguran ziclumeran (lonvo-z) for the treatment of HAE has recently completed enrollment. The study is a randomized, double-blind, placebo-controlled trial designed to evaluate the efficacy and safety of lonvo-z in at least 60 adults and adolescents aged 16 years and older with Type I or Type II HAE. Key endpoints of the HAELO trial include the number of HAE attacks and the number of patients who achieve attack-free status from week 5 through week 28.
Collaborating institutions with Intellia Therapeutics, Inc. on the Phase 3 study of Lonvo-z for HAE treatment include the U.S. Food and Drug Administration (FDA) and multiple clinical research centers specializing in hereditary angioedema across the United States.
Lonvoguran ziclumeran has received five notable regulatory designations, including Orphan Drug and RMAT Designation by the U.S. Food and Drug Administration (FDA). The anticipated U.S. launch of lonvoguran ziclumeran is planned for the first half of 2027.
Intellia Therapeutics is focused on revolutionizing medicine with CRISPR-based therapies. For more information, visit intelliatx.com or follow them on Twitter @intelliatx. The company's contact information for investors and media is also provided.
However, it's important to note that Intellia Therapeutics' forward-looking statements are subject to numerous risks and uncertainties. The success of lonvoguran ziclumeran and its timeline for approval and launch are dependent on various factors, including regulatory approvals, clinical trial results, and market conditions.
The Phase 1/2 study of lonvoguran ziclumeran showed dramatic reductions in attack rate and consistent, deep, and durable reductions in kallikrein levels. The topline data from the Phase 3 HAELO study are expected in the first half of 2026, which will provide a clearer picture of the therapy's potential efficacy and safety.
With its advancements in CRISPR-based gene editing therapies, Intellia Therapeutics is poised to make a significant impact in the treatment of hereditary angioedema and potentially revolutionize the field of medicine.
