Intellia Therapeutics, Inc. Q4 FY2024 earnings call

John Leonard highlighted the strong start in 2025 with focus on operational execution, progress in late-stage studies like NTLA-2002 for HAE (expecting complete enrollment in Phase III HAELO in 2025 second half) and TTR amyloidosis Phase III studies, discontinued NTLA-3001, and welcomed new CSO Birgit Schultes. David Lebwohl provided updates on clinical programs: NTLA-2002 in HAE Phase III study with first patient dosed, positive data from nex-z Phase I study for ATTR amyloidosis, enrollment progress in MAGNITUDE study, and RMAT designation for nex-z. Ed Dulac discussed financials: cash balance ~$861.7M as of Dec 31, 2024, collaboration revenue $12.9M in Q4 2024, R&D expenses $116.9M, G&A expenses $32.4M, and restructuring impact on GAAP operating expenses with year-over-year declines expected in 2025.

GAAP operating expenses expected to have year-over-year declines of approximately 5% to 10% in 2025 due to restructuring. Cash balance expected to fund operating plans into the first half of 2027. Anticipate data from NTLA-2002 and nex-z later in 2025.

Q: Can you help give us a little bit of an update on the tempo of OpEx decline over the course of this year and as you execute on the restructuring?

A: Edward Dulac mentioned first quarter will be noisy with wind down costs but benefit will accrue throughout 2025, peak operating expenses behind us with costs naturally sunsetting for studies post-enrollment.

Q: For nex-z or 2001, what’s the latest regarding your assumptions on Phase III events accrual rate based on longer term data?

A: David Lebwohl said events accrual rate expected to be similar to recent studies like acoramidis and vutrisiran, with favorable results from Phase I suggesting low event rate and potential for favorable interim analysis.

Q: We saw Alnylam moving forward with a next-generation TTR silencer, any reflection on MAGNITUDE study?

A: John Leonard said they look at data and competition, see differences in their own drug's results, and are pleased with their study design.

Q: What makes you feel confident to pick 550 patient numbers by year end for the enrollment regarding the ATTR cardiomyopathy trial?

A: John Leonard said they under promise and over deliver, enrollment is rolling robustly and highly likely to reach 550 by year end.

Q: How should we think through the gating factor as you get ready for the BLA filing for HAE?

A: John Leonard and David Lebwohl said it involves clinical package, commercial form of product used in Phase III, RMAT designation, and favorable interactions with FDA.

Q: Could you comment on how big of a sales force you anticipate needing to launch 2002 and other preparations?

A: Edward Dulac said investment started in 2024, will hire senior leadership, and significant spend in 2027 for launch.

Q: On HAE, how should we think about the attack free rate and long-term follow-up?

A: John Leonard and David Lebwohl discussed attack rates related to TTR reduction, longer follow-up in Phase III, and data expected later in 2025.

Q: On the Regeneron collaboration, any milestones?

A: John Leonard and Edward Dulac mentioned Regeneron's potential opt-in for co-commercialization partnership in U.S. related to PN indication timeline.

Q: You briefly mentioned discontinuing NTLA-3001, any commentary?

A: John Leonard said no clinical issues, stopped due to prioritization and restructuring, and will talk about second generation approach later.