Intellia Therapeutics (NTLA) investor relations material

Key clinical updates and results

• Presented top-line phase III HAELO study results in HAE, showing 87% attack rate reduction and 62% of patients attack and therapy free after 28 weeks, exceeding competitor benchmarks.

• Crossover and long-term follow-up data suggest further improvement in attack-free rates, with phase I/II data showing 31 of 32 patients attack and therapy free.

• Preparing for commercial launch of lonvo-z in the first half of next year, with BLA submission to be completed before year-end.

• TTR program (MAGNITUDE-2 and MAGNITUDE) cleared clinical holds, with patient screening and enrollment ongoing and operational updates expected later this year.

• Durability data for both HAE and TTR programs show no waning of effect, with up to four years of follow-up in HAE and five years in TTR.

Commercialization and market strategy

• Engaged in constructive payer discussions, highlighting the value of a one-time therapy versus high annual costs of current treatments ($750,000/year).

• Pricing strategy considers a multiple of annual treatment costs, aiming for a balance between value capture and payer acceptance.

• U.S. commercialization to be handled internally, with foundational leadership and field force being built; ex-U.S. access to be pursued via partnerships.

• Broad addressable market includes both LTP and on-demand therapy patients, with 70% of phase III patients previously on LTPs.

• Adoption curve expected to be strong, with 75-80% of the market considered switchable over the first few years.

Operational and regulatory milestones

• Rolling BLA submission for lonvo-z underway, with completion expected before year-end and approval targeted for first half of next year.

• Commercial infrastructure build-out is ongoing, with pricing and contracting strategy to be finalized ahead of launch.

• Ex-U.S. commercialization to rely on partnerships with established companies or distributors.

• Long-term follow-up commitments include 15 years for cell and gene therapies, with ongoing extension studies.

• Cash position of $700 million is sufficient to fund operations into 2028, even without lonvo-z revenue.