Operational Highlights
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Transthyretin (ATTR) Amyloidosis: Intellia is making significant progress with NTLA-2001, an investigational in vivo CRISPR-based therapy for ATTR amyloidosis. The Phase 3 MAGNITUDE trial is enrolling patients ahead of projections, with over 30 patients dosed and more than 40 in screening. A new Phase 3 trial for hereditary ATTR amyloidosis with polyneuropathy is planned to start by year-end, following FDA alignment on trial design.
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Hereditary Angioedema (HAE): The company is advancing NTLA-2002, a CRISPR-based therapy for HAE, with plans to initiate a global Phase 3 study in the second half of 2024. Updated Phase 1 data will be presented at the EAACI Congress 2024, with topline Phase 2 results expected mid-2024.
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Alpha-1 Antitrypsin Deficiency (AATD): Intellia expects to dose the first patient in a Phase 1 study of NTLA-3001, a gene insertion candidate for AATD-associated lung disease, in 2024.
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In Vivo Platform Expansion: The company is expanding its CRISPR-based technologies to target diseases in tissues outside the liver, including bone marrow, brain, muscle, lung, and eye.
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Ex Vivo Program Updates: Intellia is advancing allogeneic cell engineering platforms for immuno-oncology and autoimmune diseases, aiming to overcome T cell- and NK cell-mediated rejection.
Financial Results
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Cash Position: Intellia ended the first quarter with $953.4 million in cash, cash equivalents, and marketable securities, expected to fund operations into late 2026.
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Collaboration Revenue: Increased to $28.9 million, primarily due to a non-cash revenue recognition adjustment related to the AvenCell collaboration.
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R&D Expenses: Rose to $111.8 million, driven by the advancement of lead programs.
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Net Loss: Reported at $107.4 million for the quarter.
Intellia Therapeutics continues to lead in the development of CRISPR-based therapies, with a strong pipeline and financial position supporting its ambitious clinical and operational goals.
