Intellia Therapeutics has revealed interim data from the first portion of its Phase I/II study (NCT05120830) showing its investigational drug, NTLA-2002, could become a functional cure for people living with hereditary angioedema (HAE).

The results, unveiled at the European Academy of Allergy and Clinical Immunology (EAACI) Hybrid Congress 2023 (being held June 9 – 11), demonstrated that a single dose of NTLA-2002 led to a 95% mean reduction in HAE attack rate. The median follow-up was 9 months. In the ten patients investigated, the elimination of HAE attacks was long-lasting. The longest attack-free duration is 11.5 months and counting.

The drug was also found to consistently decrease plasma kallikrein. Kallikrein is an accepted pharmaceutical target to prevent and treat HAE attacks. In the 75mg group, a 95% reduction in plasma kallikrein was observed.

Patients were divided into three groups of doses 25mg, 50mg, and 75mg. The treatment was administered intravenously. The single-ascending dose global study involves adults with Type I or Type II HAE. Intellia says that screening and dosing in the Phase II portion of the HAE study is ongoing.

NTLA-2002 is based on CRISPR/Cas9 technology. According to the Massachusetts, US-based company, it is the first single-dose treatment being investigated in clinical trials with a goal of continuously reducing kallikrein activity and preventing attacks in people living with hereditary angioedema (HAE).

“After a single dose of our investigational CRISPR-based therapy, patients living with hereditary angioedema experienced durable elimination of their attacks. We are thrilled to see that the earliest-dosed patients are attack free for approximately a year or longer, with NTLA-2002 demonstrating a very favourable safety profile,” said Intellia’s president and CEO John Leonard, M.D.

Other companies with ongoing trials for HAE treatment include Astria Therapeutics and BioCryst Pharmaceuticals.