From Intellia Therapeutics, Inc.’s presentation of the financial results for the second quarter of 2020:
NTLA-2002 is a wholly owned, in vivo development candidate for the treatment of HAE. Building on Intellia’s modular lipid nanoparticle (LNP) delivery system, NTLA-2002 is designed to knock out the prekallikrein B1 (KLKB1) gene in the liver after a single course of treatment, which is expected to prevent improperly regulated bradykinin production and therefore, reduce HAE attacks.
In a non-human primate (NHP) study of its lead LNP formulation for NTLA-2002, the knockout of KLKB1 resulted in a therapeutically relevant reduction of serum kallikrein levels and activity following a single dose. Consistent with the durability achieved in earlier NHP studies for its lead in vivo program, Intellia has now demonstrated sustained kallikrein activity reduction for 10 months in an ongoing study. Based on these results, Intellia believes NTLA-2002 could be efficacious and durable in preventing HAE attacks following a single course of treatment.
Intellia continues to progress IND-enabling (Investigational New Drug) activities and is on track to submit an IND or IND-equivalent for NTLA-2002 in the second half of 2021.