Intellia touts further early data for single-dose HAE therapy 

03 Jun 2024
firstwordpharma.com
Clinical ResultPhase 3Immunotherapy
Data from an early-stage study showed that a single dose of Intellia Therapeutics’ in vivo CRISPR-based gene editing therapy NTLA-2002 led to a 98% mean reduction in monthly attack rate in patients with hereditary angioedema (HAE). The median duration of follow-up in the first part of the Phase I/II trial was 20.1 months.
CEO John Leonard said the “unprecedented data” demonstrated that after a single dose of NTLA-2002, patients experienced durable elimination of their attacks with eight of the 10 study participants having been attack free for over 18 months or longer.
One-time, functional cure
The findings, which were presented at the European Academy of Allergy and Clinical Immunology (EAACI) congress, also showed that a single dose of NTLA-2002 was also associated with a 99% mean reduction in moderate-to-severe attacks. Leonard indicated that the “attack rate reductions have been consistent, even in patients with the most severe symptoms.”
“These long-term data provide strong evidence that NTLA-2002 could be a one-time, potential functional cure” for HAE, remarked Leonard. The company expects to begin a pivotal, Phase III trial of NTLA-2002 in the second half.
More to come.
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