Intellia Therapeutics (NTLA) TD Cowen 46th Annual Health Care Conference summary

Key program updates and clinical milestones

• lonvo-z for HAE is completing Phase III, with top-line results expected mid-2026, BLA filing in the second half, and a planned U.S. launch in 2027.

• MAGNITUDE study for TTR cardiomyopathy has resumed enrollment after a clinical hold, with operational changes implemented.

• Phase III HAELO study for lonvo-z enrolled quickly, reflecting strong patient and physician interest.

• Polyneuropathy (PN) study for TTR is expected to fully enroll by year-end, with an 18-month observation period.

• Cardiomyopathy study for TTR exceeded enrollment targets before the hold and is expected to regain momentum.

Clinical data and efficacy insights

• Pooled analysis of lonvo-z at 50mg shows excellent efficacy and safety, with attack rate reductions expected in the 80% range.

• Attack-free rates are anticipated to be at or above 60%, surpassing current market leaders.

• Patients in TTR studies show consistent, deep reductions in TTR protein, with many achieving levels below 20 micrograms/mL.

• Extended follow-up in TTR studies indicates patients do not progress and some even improve beyond baseline.

• Early efficacy in cardiomyopathy and polyneuropathy studies is driving strong interest from both patients and physicians.

Safety profile and regulatory engagement

• lonvo-z has demonstrated an exceptionally clean safety profile, with no severe transaminase elevations or major adverse events.

• TTR program experienced a safety event leading to a clinical hold, but enhanced monitoring and steroid protocols have been implemented.

• FDA interactions have been positive and responsive, with regulatory designations like RMAT facilitating ongoing dialogue.

• Multinational studies are designed to meet ex-U.S. regulatory requirements, though initial launch focus is on the U.S.