Intellia Therapeutics (NTLA) investor relations material

Platform and pipeline overview

• Pioneered in vivo gene editing, focusing on delivery challenges and leveraging lipid nanoparticle technology for liver-targeted therapies.

• Advanced to late-stage trials for HAE and TTR cardiomyopathy/polyneuropathy, with ongoing efforts to expand delivery beyond the liver to organs like the brain and lung.

• Emphasis on commercializing the first CRISPR-based therapy and validating the platform's potential.

HAE program and commercialization plans

• BLA for HAE therapy accepted by FDA, with priority review and a PDUFA date set for March 10; no advisory committee planned.

• Phase III data show strong efficacy (87% attack rate reduction, 62% attack-free at 28 weeks) and a clean safety profile.

• Broad patient inclusion in trials, with expectations for a wide label excluding only those under 16.

• Market access work ongoing, with payer discussions focused on pricing multiples and the therapy's durability; patient advocacy and education are key.

• Commercial footprint will be targeted, with 250 prescribers covering half of U.S. patients; field force and infrastructure are being built out in anticipation of approval.

Launch dynamics and financial outlook

• Launch expected to be gradual, with revenue ramping over several quarters; early metrics will focus on treatment center penetration and patient starts.

• Even a moderate launch could cover operating costs, enabling reinvestment in pipeline programs.

• Strong cash position ($630M as of Q2) and a new $300M debt facility provide flexibility; runway extends into 2028 without HAE revenue.