Intellia Therapeutics announces publication in the New England Journal of Medicine of positive interim phase 1 data for NTLA-2002 in patients with hereditary angioedema

Intellia Therapeutics announced that interim results from the Phase 1 portion of the Phase 1/2 study of NTLA-2002 were published online in the New England Journal of Medicine (NEJM).

The reported data showed that a single dose of NTLA-2002 led to a 95% mean reduction in monthly HAE attack rate across all 10 patients in the Phase 1 portion. Nine out of 10 patients remained completely attack-free following the 16-week primary observation period through the latest follow-up. Further, all patients who discontinued concomitant long-term HAE prophylaxis treatment after NTLA-2002 administration (n=6) have reported no HAE attacks since discontinuation. NTLA-2002 has been well tolerated at all dose levels. The most frequent adverse events reported were mild, transient infusion-related reactions and fatigue.

Intellia anticipates initiating the global pivotal Phase 3 trial of NTLA-2002 in HAE in the second half of 2024, subject to regulatory feedback. The company also expects to present updated data from Phase 1 and new data from Phase 2 portion in 2024.

NTLA-2002 is an investigational in vivo CRISPR-based gene editing therapy in development as a single-dose treatment for hereditary angioedema (HAE), a rare genetic condition that leads to potentially life-threatening swelling attacks.

John Leonard MD, Intellia President and Chief Executive Officer, said: “The interim NTLA-2002 clinical data published suggest that a single dose of NTLA-2002 may eliminate angioedema attacks for people suffering from hereditary angioedema. We are highly encouraged by these data and look forward to presenting extended follow-up from the Phase 1 and results from the Phase 2 portion later this year. Additionally, we remain on track to initiate a global pivotal study for NTLA-2002 in the second half of 2024, subject to regulatory feedback.”

(Source: Intellia)