Intellia Therapeutics (NTLA) investor relations material

Program highlights and clinical data

• Achieved 87% average attack rate reduction in HAE patients over 28 weeks, with 62% experiencing no attacks and all patients off long-term prophylaxis after a single outpatient IV infusion.

• Phase III studies for HAE completed; TTR amyloidosis program in phase III for cardiomyopathy and polyneuropathy, with ongoing patient-level data updates and long-term follow-up extending to four years.

• Upcoming EAACI meeting to present detailed patient-level data, subgroup analyses, and real-world baseline comparisons.

• Consistent efficacy observed across diverse patient subgroups, regardless of prior therapy or disease severity.

• Safety profile remains favorable, with only mild to moderate infusion reactions, headaches, and fatigue reported.

Regulatory and commercialization plans

• Rolling BLA submission for HAE therapy began in April, aiming for completion in the second half of the year and a U.S. launch in the first half of 2027.

• RMAT designation expected to facilitate priority and expeditious FDA review; CMC and manufacturing processes are complete and commercial-ready.

• U.S. commercialization is the primary focus, with ex-U.S. strategies under evaluation, including potential partnerships or out-licensing.

• Commercial team and reimbursement infrastructure are in place, with ongoing payer engagement and pricing strategy development.

• Education initiatives target both physicians and patient advocacy groups to drive adoption and awareness.

Market outlook and financials

• Market research indicates strong patient and physician interest, with 64% of patients and 54% of physicians likely to adopt the therapy.

• Initial adoption expected to be patient-driven, with advocacy groups playing a key role in information dissemination.

• Synthetic, non-viral manufacturing expected to yield substantial margins at scale.

• Recent capital raise extends cash runway into 2028, with future cash needs dependent on the success of the HAE launch.