Intellia to Present Phase 2 NTLA-2002 Data for HAE at 2024 ACAAI Meeting

Intellia Therapeutics, Inc. recently announced that detailed results from the Phase 2 study of their investigational therapy, NTLA-2002, will be presented at the 2024 American College of Allergy, Asthma & Immunology (ACAAI) Annual Scientific Meeting in Boston, scheduled for October 24-28. NTLA-2002 is a CRISPR-based gene editing therapy designed for the treatment of hereditary angioedema (HAE), a rare genetic disorder known for causing severe and potentially fatal swelling attacks.

Hereditary angioedema affects approximately one in 50,000 individuals and is characterized by spontaneous, painful, and often debilitating swelling in various parts of the body. Current treatments focus on both acute symptom management and long-term prophylaxis to prevent attacks, but they typically require frequent administration, such as bi-weekly intravenous (IV) or subcutaneous (SC) injections, or daily oral medications. Despite these ongoing treatments, patients may still experience breakthrough attacks, which underscores the need for more effective solutions.

NTLA-2002 aims to offer a one-time treatment option by leveraging Nobel-prize-winning CRISPR/Cas9 technology to inactivate the kallikrein B1 (KLKB1) gene responsible for producing prekallikrein, a precursor protein that plays a significant role in swelling episodes. Previous interim Phase 1 data showcased the therapy's potential, revealing significant reductions in attack rates and sustained decreases in kallikrein levels.

Intellia Therapeutics will share comprehensive Phase 2 trial results during a session titled "Results From a Phase 2, Randomized, Placebo-Controlled Trial of CRISPR-Based Therapy NTLA-2002 for Hereditary Angioedema." This presentation is scheduled for Saturday, October 26, 2024, from 4:30 to 6:30 p.m. ET, with Danny Cohn, M.D., Ph.D., from the Amsterdam University Medical Center, as the presenter.

Regulatory bodies have taken note of NTLA-2002's promise. The U.S. Food and Drug Administration (FDA) has granted it five significant designations, including Orphan Drug and RMAT (Regenerative Medicine Advanced Therapy) status. The therapy has also received the Innovation Passport from the U.K. Medicines and Healthcare products Regulatory Agency (MHRA), Priority Medicines (PRIME) Designation from the European Medicines Agency, and Orphan Drug Designation from the European Commission.

Intellia Therapeutics, headquartered in Cambridge, Massachusetts, is at the forefront of clinical-stage gene editing, focusing on transformative CRISPR-based therapies. Their in vivo programs facilitate precise gene editing within the human body, targeting disease-causing genes, while their ex vivo programs engineer human cells outside the body for cancer and autoimmune disease treatments. The company's extensive scientific, technical, and clinical expertise is setting new standards in the field of gene editing, aiming to unlock the full potential of this groundbreaking technology through continuous advancement and innovation.

In conjunction with the presentation, Intellia will host an investor webcast on Monday, October 28, 2024, at 8:00 a.m. ET to discuss the new data in detail. This live webcast will be accessible via Intellia's website, with a replay available for at least 30 days following the event.

The upcoming detailed results from the Phase 2 trial of NTLA-2002 mark a significant milestone for Intellia Therapeutics and the broader field of gene editing. Should the therapy prove successful, it could represent a monumental leap forward in the treatment of hereditary angioedema, potentially transforming it from a chronic, life-altering condition to one that can be effectively managed with a single treatment.