Intellia Therapeutics(NTLA) - 2024 Q2 - Earnings Call Presentation

Corporate Strategy & Pipeline - Intellia plans to initiate two additional in vivo Phase 3 studies in 2024[6] - Intellia expects to submit a BLA for NTLA-2002 for HAE in 2026[16] - Strategic priorities for 2024-2026 include demonstrating human proof-of-concept for targeted in vivo gene insertion and initiating clinical development for its allogeneic ex vivo program[3] NTLA-2001 (nexiguran ziclumeran) for ATTR Amyloidosis - NTLA-2001 led to a -91% change from baseline in serum TTR at day 28[34] - A Phase 3 trial is underway for ATTR-CM patients with NT-proBNP baseline ≥ 1000 pg/mL[38] - GlobalData projects an $11B+ global market size for ATTR amyloidosis by 2029[28] NTLA-2002 for Hereditary Angioedema (HAE) - NTLA-2002 demonstrated a 98% reduction in HAE attack rate, with 8 of 10 patients attack-free in the post-primary observation period[48] - NTLA-2002 continues to show dose-dependent and durable reductions in plasma kallikrein protein over time[46] - GlobalData projects a $6B+ global market size for HAE by 2029[41] NTLA-3001 for Alpha-1 Antitrypsin Deficiency (AATD) - NTLA-3001 aims to achieve normal human levels of AAT protein and halt progression of lung disease[50] - In preclinical studies, NTLA-3001 achieved durable production of physiologic levels of hAAT through one year in NHPs[51]