Intellia presents positive results from the Phase 2 Study of NTLA-2002, an investigational in vivo crispr gene editing treatment for hereditary angioedema (HAE)

Intellia Therapeutics announced positive Phase 2 data from the ongoing Phase 1/2 study of NTLA-2002 in patients with hereditary angioedema (HAE), with results continuing to indicate that NTLA-2002 has the potential to eliminate HAE attacks following a one-time infusion.

Results were published in The New England Journal of Medicine and will be presented on Saturday 26 October at the 2024 American College of Allergy, Asthma & Immunology (ACAAI) Scientific Meeting in Boston, Massachusetts.

Intellia President and Chief Executive Officer John Leonard MD, said: “These positive NTLA-2002 Phase 2 results underscore the tremendous potential of our in vivo CRISPR gene editing therapy to be a functional cure and redefine the treatment paradigm for HAE. The Phase 2 data demonstrated that a majority of patients in the 50mg arm experienced a complete response — no attacks at all and no further treatment needed — after a one-time infusion of NTLA-2002 through the latest follow-up, consistent with the long-term Phase 1 data. We are highly encouraged by these results, which we believe sets NTLA-2002 apart from other prophylaxis treatments. What was previously an unimaginable potential to be free of chronic therapy is one step closer to becoming a reality for the HAE community.”

Danny Cohn MD PhD, Internist, Department of Vascular Medicine, Amsterdam University Medical Center and the Phase 2 study’s lead principal investigator, said: “Approved HAE therapies can reduce but frequently do not eliminate all angioedema attacks and require chronic administration, resulting in a significant treatment burden and a major impact on the quality of life for people living with HAE. These NTLA-2002 Phase 2 data are remarkable, showing this investigational therapy could permanently stop swelling attacks with a single infusion. I am optimistic that NTLA-2002 will change the way we treat HAE and put an end to the need for a lifetime of chronic treatment.”

(Source: Intellia)