Intellia to seek FDA approval of its HAE gene-editing therapy in 2026
HAELO trial of NTLA-2002 now recruiting 60 adults with HAE type 1 or 2
Intellia Therapeutics is planning to submit an application requesting the approval of NTLA-2002, its gene-editing therapy for hereditary angioedema (HAE), to U.S. regulators in late 2026.
The company recently announced it would be discontinuing programs for other diseases, as well as reducing its workforce, to focus efforts on advancing the development of NTLA-2002 and nexiguran ziclumeran (nex-z), its experimental treatment for transthyretin amyloidosis. Both of these experimental therapies are currently in the late stages of clinical development.
“We understand the significant potential of our late-stage programs, and within a challenging market environment, have made a difficult decision to focus our resources predominantly on NTLA-2002 and nex-z where we have the greatest opportunity to create significant, near-term value,” John Leonard, MD, president and CEO of Intellia, said in a company press release.
HAE is caused by mutations that lead to the overproduction of bradykinin, a signaling molecule that can trigger swelling. NTLA-2002 is a one-time treatment designed to inactivate the gene that provides instructions to make a precursor of kallikrein, the enzyme responsible for making bradykinin. By inactivating that gene, the therapy is expected to reduce kallikrein production and activity, thereby lowering bradykinin levels and ultimately preventing swelling attacks.
HAELO to test NTLA-2002’s effect on number of swelling attacks over 6 months
Intellia is currently sponsoring a Phase 3 clinical trial called HAELO (NCT06634420) to evaluate the efficacy of NTLA-2002 in reducing the number of HAE swelling attacks. The study is expected to enroll about 60 adults with HAE types 1 or 2. Participants will be randomly assigned to receive either a single into-the-vein infusion of NTLA-2002 or a placebo with the main goal of evaluating the treatment’s effect on the number of swelling attacks occurring over the course of about six months.
HAELO is currently recruiting participants at sites in California, Ohio, and Colorado. The first participant is expected to be dosed in the coming months, and patient enrollment is to be completed in the second half of the year, Intellia said.
If all goes well, the company is then planning to use data from HAELO and previous trials as a basis to ask the U.S. Food and Drug Administration to approve NTLA-2002. The company said it anticipates submitting a biologics license application seeking approval in the second half of 2026.
Data from a previous Phase 1/2 clinical trial (NCT05120830) showed most patients treated with NTLA-2002 were free from swelling attacks in the months after receiving the gene-editing therapy, with some experiencing no swelling attacks for more than a year. The company also announced it plans to present longer-term data from this study.
About the Author
