Core Insights - Intellia Therapeutics is advancing two late-stage in vivo pipeline candidates: nex-z (NTLA-2001) for ATTR amyloidosis and lonvo-z (NTLA-2002) for hereditary angioedema (HAE) [1] Group 1: Nex-z Development - Intellia is collaborating with Regeneron Pharmaceuticals on nex-z, which is being evaluated in two late-stage studies: MAGNITUDE for ATTR amyloidosis with cardiomyopathy and MAGNITUDE-2 for ATTR amyloidosis with polyneuropathy [2] - Regeneron shares 25% of the development costs and commercial profits for nex-z, providing Intellia with additional resources for development [3] - The FDA placed a clinical hold on the MAGNITUDE studies due to observed Grade 4 liver enzyme elevations in under 1% of patients in the MAGNITUDE study, with none in MAGNITUDE-2 [4] - Intellia is working with investigators and regulators to address the clinical hold and has suspended its milestone guidance for nex-z until a regulatory path is established [5] Group 2: Lonvo-z Development - Intellia is also developing lonvo-z for HAE, with patient enrollment in the pivotal phase III HAELO study completed in September 2025, and top-line data expected by mid-2026 [6] - The regulatory setback for nex-z has raised concerns about the outlook for lonvo-z, making the HAE study outcome critical for Intellia's near-term success [7] Group 3: Competitive Landscape - Intellia's CRISPR-based therapies face competition from other companies utilizing CRISPR/Cas9 technology, which complicates the development process [8] - CRISPR Therapeutics is the first company to market a CRISPR/Cas9-based therapy, Casgevy, approved for sickle cell disease and transfusion-dependent beta-thalassemia [9] - Beam Therapeutics is also developing genome-editing candidates and in vivo therapies targeting various diseases [10][11] Group 4: Financial Performance - Year to date, Intellia's shares have declined by 25.6%, underperforming the industry, sector, and S&P 500 [13] - Intellia's shares are trading at a price/book ratio of 1.24, lower than the industry average of 3.65, and below its five-year mean of 3.01 [14] - The Zacks Consensus Estimate for Intellia's loss per share for 2025 has narrowed from $4.14 to $4.00, and for 2026 from $4.10 to $3.61 [15]
Can NTLA Stock Bounce Back in 2026 After Recent Pipeline Setbacks?