Intellia Therapeutics(NTLA) - 2024 Q3 - Earnings Call Transcript

Financial Data and Key Metrics - Cash, cash equivalents, and marketable securities were approximately $944.7 million as of September 30, 2024, compared to$ 1 billion as of December 31, 2023, driven by $335 million used to fund operations [24] - Collaboration revenue was$ 9.1 million in Q3 2024, down from $12 million in Q3 2023, primarily due to reduced revenue from the AvenCell license and collaboration agreement [25] - R&D expenses increased to$ 123.4 million in Q3 2024 from $113.7 million in Q3 2023, driven by the advancement of lead programs [26] - G&A expenses rose to$ 30.5 million in Q3 2024 from $29.4 million in Q3 2023, primarily due to stock-based compensation [26] Business Line Updates - NTLA-2002 for Hereditary Angioedema (HAE): Phase II results showed 8 out of 11 patients in the 50 mg arm were attack-free for 16 weeks post-treatment, with 80% of patients in the Phase I/II study appearing functionally cured [11][12] - NEX-Z (NTLA-2001) for ATTR Amyloidosis: FDA cleared the IND for the Phase III MAGNITUDE-2 trial, with enrollment expected to begin soon [9][15] - NTLA-3001 for Alpha-1 Antitrypsin Deficiency: First patient dosing in the Phase I/II study is expected by year-end, with non-human primate data showing durable alpha-1 protein levels for 2 years [21] Market and Strategic Direction - The company is leading the field of in vivo CRISPR-based medicines with three active Phase III studies expected by year-end [9] - The emerging product profile of NTLA-2002 aligns with patient and physician needs, driving rapid enrollment in the Phase III HAELO study [13] - The company is advancing gene editing programs in five different tissues, expanding its platform and pipeline [22] Management Commentary - The company is encouraged by the Phase II results for NTLA-2002 and expects to submit a BLA in 2026 [8] - Management highlighted the potential of NEX-Z to significantly reduce TTR levels, which could improve clinical outcomes for ATTR Amyloidosis patients [17] - The company is focused on advancing its in vivo gene insertion programs, with NTLA-3001 representing a major step forward for alpha-1 patients [21] Q&A Session Enrollment in MAGNITUDE-1 - Enrollment is ahead of projections, with 765 patients targeted globally [28] Capital Allocation Priorities - The company has$ 945 million in cash and is focused on three Phase III studies, with cash expected to fund operations until late 2026 [32] MAGNITUDE-2 Trial Design - The trial is placebo-controlled with 50 patients, designed to provide a solid readout on drug performance and safety [34][35] HAELO Study Randomization - The 2:1 randomization in the HAELO study is designed to accelerate enrollment and provide more data on the active arm [39] AATD Program - The Phase I/II study for NTLA-3001 aims to normalize alpha-1 protein levels, with initial data expected when meaningful [64] Ex-U.S. Strategy for NTLA-2002 - The company plans to launch NTLA-2002 in the U.S. and expand to Europe, with potential partnerships to extend reach [70] ATTR Therapy Excitement - Investigators are excited about the deep and rapid TTR reduction achieved with NEX-Z, which could outperform silencers [80] MAGNITUDE-2 U.S. Initiation - The trial will be conducted ex-U.S. to facilitate a placebo-controlled study, with FDA agreement on the design [74] AHD Program Expansion - Additional sites beyond New Zealand are planned, with data potentially available in 2025 [76]