Intellia Therapeutics (NTLA) Earnings Call Presentation

NTLA-2002 (Hereditary Angioedema - HAE) - NTLA-2002 aims to be the first to offer lifelong freedom from attacks and prophylaxis after a single dose for HAE, with a BLA submission planned in 2026[8] - Phase 2 study results showed 73% of patients were attack-free and off chronic prophylaxis after a single dose of NTLA-2002 at 16 weeks[37, 41] - The global market for HAE is projected to reach approximately $5 billion by 2028[8, 44] Nex-z (Transthyretin Amyloidosis - ATTR) - Nex-z aims to be the first to stabilize or reverse disease progression with a single dose for ATTR, with a BLA submission planned in 2028 for polyneuropathy[8] - Phase 1 data showed deep, rapid, and durable reductions in serum TTR, with an 89% reduction in ATTR-CM and a 90% reduction in ATTRv-PN at day 28[63] - The global market for ATTR is projected to reach approximately $12 billion by 2028[8, 86] Clinical Development and Milestones - Intellia expects three commercial launches starting in 2027[8] - The company plans to complete enrollment in the Phase 3 HAELO trial for NTLA-2002 in the second half of 2025 and the MAGNITUDE-2 trial for ATTRv-PN in 2026[2] - Intellia plans to enroll at least 550 patients in the Phase 3 MAGNITUDE trial for ATTR-CM in 2025 and complete enrollment in early 2027[2]