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.
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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.
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