Hereditary Angioedema Update Solidifies Intellia's Cell Therapy's Competitive Efficacy Profile: Analyst

Sunday, Intellia Therapeutics Inc NTLA announced updated interim results from the Phase 1 portion of the ongoing Phase 1/2 study of NTLA-2002.

NTLA-2002 is an in vivo, systemically administered CRISPR candidate developed as a single-dose treatment for hereditary angioedema (HAE). 

Across all patients (n=10), a single dose of NTLA-2002 led to a 95% mean reduction in monthly HAE attack rate through the latest follow-up.

All patients who achieved greater than 60% plasma kallikrein reduction (n=9) remain completely attack free following the 16-week primary observation period through the latest follow-up; the longest attack-free interval is 13.0 months and ongoing.

NTLA-2002 has been well tolerated at all dose levels.

William Blair says that the attack rate reductions achieved and sustained across all dose cohorts as encouraging, and the data continues to support a competitive profile relative to peers (on par with Ionis Pharmaceuticals Inc's IONS donidalorsen), with a tolerable safety profile and convenience as a one-time therapy. 

These data bode well for continued follow-up and Phase 2 data(potentially in 2024), the analyst writes and reiterates its Outperform rating.

Price Action: NTLA shares are up 0.21% at $43.33 on the last check Monday.