Intellia Therapeutics, Inc. Q3 FY2025 earnings call

• For nex-z: Enrollment in MAGNITUDE (ATTR amyloidosis with cardiomyopathy) and MAGNITUDE-2 (hereditary ATTR amyloidosis with polyneuropathy) trials; patient experienced liver issues leading to clinical hold, patient death, and actions taken for safety. - For lonvo-z: Enrollment completed in HAELO Phase III trial for HAE, on track to share top-line data mid-2026, submit BLA in H2 2026, and launch in US H1 2027. - Financials: Solid balance sheet, cash runway extended into mid-2027, expected year-over-year decline in GAAP operating expenses of at least 10%.

Intellia's cash, cash equivalents and marketable securities were $669.9 million as of September 30, 2025, down from $861.7 million as of December 31, 2024. Collaboration revenue was $13.8 million in Q3 2025 compared to $9.1 million in the prior year quarter. R&D expenses were $94.7 million in Q3 2025 vs. $123.4 million prior year. G&A expenses were $30.5 million in Q3 2025, same as prior year. Net loss for Q3 2025 was $101.3 million, down from $135.7 million prior year.

• Expect year-over-year decline in GAAP operating expenses of at least 10%. - Cash runway expected to fund operations into mid-2027. - Lonvo-z timelines for data sharing, BLA submission, and launch remain unabated. - Unable to maintain nex-z milestone guidance pending regulator plan.

• Clinical hold on nex-z trials due to liver enzyme elevations and patient death. - Uncertainties around FDA clinical hold letter and evaluating the patient's case. - Need to identify risk mitigation strategies for nex-z trials.

Q: Any initial hypothesis on liver enzyme elevation for nex-z?

A: Can't speculate yet, but this case is distinct with other comorbidities.

Q: Comorbidities of the patient?

A: Can't disclose specifics, but patient had complicated medical course.

Q: Risk mitigation strategies for nex-z?

A: Ongoing comprehensive analysis to identify at-risk patients or interventions.

Q: Number of ATTR patients in 3-5 week post-dose window?

A: Vast majority have passed through, smaller set remaining.

Q: Impact of clinical hold on OpEx?

A: Near term may spend less, but will reassess once clinical hold is resolved.

Q: Liver-related comorbidities in patient?

A: Can't disclose.

Q: Steps from here with FDA?

A: Await FDA letter, then work with them to mitigate risks.

Q: Differences between nex-z and lonvo-z?

A: Guide RNA differs, leading to different patient outcomes.

Q: Additional liver monitoring in lonvo-z trial?

A: Monitoring not as intense as nex-z, but experience is distinct.

Q: Depth of comprehensive analysis?

A: Comprehensive, looking at all patient data, but lonvo-z experience is distinct.

Q: Lab monitoring frequency previously?

A: Previously monitored in window, now increased to weekly for first few weeks post-dose.

Q: Impact of TTR patient death on HAE commercial opportunity?

A: A lot of time and info to accumulate before discussing commercial opportunity.

Q: Cause of death?

A: Will share info once all final material is in hand.