Intellia ups the ante in HAE dosing battle, showcasing durability of one-and-done option

The future of hereditary angiodema treatment seems to be coming down to how many shots it takes to reduce attacks. Gene editing biotech Intellia Therapeutics is the latest to reveal updated data for its candidate, showing that a single dose of NTLA-2002 provided a durable reduction in the swelling attacks that define the disease.

Intellia showcased a 98% mean reduction in monthly attack rate and a 99% mean reduction in moderate to severe attacks after just one dose of NTLA-2002. The results of the in vivo CRISPR-based gene editing therapy were presented at the 2024 European Academy of Allergy and Clinical Immunology Annual Congress in Valencia, Spain, this weekend.

From week one to 16, patients experienced a 90% reduction in attacks, a number that rose to 92% between weeks five and 16 and then to 98% during the on-study period, Intellia said.

The results compare to a similar cut from the 10 patients presented at the same conference last year, which showed a 95% mean reduction in monthly attack rate.

Now, Intellia has found that eight out of the 10 patients remain attack-free after that 16-week observation period through the latest follow-up, which had a cut-off date in February of this year. The average follow-up for the patients is 20 months. All of the patients who discontinued prophylaxis treatment after receiving NTLA-2002 have not had to resume.

“We are thrilled to see that the majority of patients have been attack-free for over 18 months or longer. These remarkable attack rate reductions have been consistent, even in patients with the most severe symptoms,” Intellia CEO John Leonard said in a statement.

As for safety, NTLA-2002 was well tolerated with mild adverse events mostly including infusion-related reactions and fatigue. There have been no dose-limiting toxicities, serious adverse events or grade 3 or higher adverse events.

Intellia’s presentation came a few days after Ionis debuted data showing the RNA-targeting prophylactic therapy donidalorsen spurred an 81% lower monthly rate of swelling attacks in patients who received the drug every four weeks.

Analysts from Willian Blair heralded donidalorsen’s “highly competitive” profile and said it could have an edge over CSL Behring’s garadacimab, which is already under review but requires more frequent injections and an initial in-office loading dose.

With Intellia showing the potential to provide a one-and-done option, the game is officially on in HAE.