Intellia Therapeutics (NTLA) investor relations material

Key data milestones and clinical progress

• Anticipates completion of pivotal Phase III HAELO study for HAE with top-line data expected mid-year and potential first in vivo gene editing product launch next year.

• Data will focus on attack rate reductions, attack-free intervals, and safety, aiming to be competitive or superior to current market leaders.

• TTR polyneuropathy and cardiomyopathy studies resumed after clinical hold, with over 650 patients enrolled; operational steps underway for full restart.

• Protocol modifications include additional monitoring and minor inclusion/exclusion changes, expected to have minimal impact on patient mix.

• Real-world evidence and long-term follow-up are expected to further validate efficacy and safety profiles.

Commercial strategy and market opportunity

• HAE market in the US is large, growing, and filled with premium products; trend toward long-term prophylaxis and patient willingness to switch therapies.

• Early adopters likely to be underserved patients with breakthrough attacks; market research suggests 20-25% early adoption, with up to 50% considering switching after real-world data.

• US market represents 65-70% of global value; international expansion may involve partnerships or licensing.

• HAE community is tight-knit and highly informed, with advocacy groups supporting education and adoption.

• Market durability expected to decrease as more patients achieve attack-free status, but total addressable market remains significant at $14-15 billion.

Product profile, pricing, and reimbursement

• Manufacturing process is straightforward with high gross margins expected (>95%), leveraging simple chemistry and established CDMOs.

• Pricing will be based on multiples of current standard of care ($650,000-$700,000 annually), aiming for a highly profitable one-time therapy.

• Engagement with payers ongoing; value proposition centers on durable, attack-free outcomes and significant healthcare cost savings.

• Payers show little resistance to reimbursement for a one-time, durable therapy; value-based agreements not anticipated to be necessary.

• Commercial payers dominate the US market, facilitating quicker reimbursement and patient retention.