Intellia Therapeutics, Inc. Q2 FY2024 earnings call

• John Leonard discussed NTLA-2002 Phase II results for HAE, where a single 25 or 50-mg dose led to deep attack reductions, selected 50-mg dose for Phase III, completed successful end-of-Phase II meeting with FDA, plans to start Phase III in second half of 2024 and submit BLA in 2026. Also discussed NTLA-2001 (NEX-Z) progress in ATTR amyloidosis, rapid enrollment in MAGNITUDE trial, and strategic appointments. • Laura Sepp-Lorenzino talked about NTLA-3001 receiving UK regulatory approval for first-in-human study, Phase 1/2 plan for NTLA-3001, and R&D updates on gene editing technologies, including the ability to redose patients with NTLA-2001 being well-tolerated.

No specific product segments with revenue contribution percentages discussed; focus is on clinical programs like NTLA-2002 for HAE, NTLA-2001 for ATTR amyloidosis, and NTLA-3001 for alpha-1 antitrypsin deficiency associated lung disease.

• Plan to begin Phase III trial for NTLA-2002 in second half of 2024. • Intend to submit BLA for NTLA-2002 in 2026. • NTLA-3001 on track to dose first patients in second half of 2024. • Cash balance expected to fund operating plans into late 2026.

• Risks associated with clinical trial outcomes and regulatory approvals. • Competition in the gene editing therapy space. • Uncertainties related to patient enrollment and trial design.

Q: Regarding the HAE Phase 2 data, can you talk about how competitive it is against INS drug?

A: John Leonard stated they're resetting the bar for HAE treatment, aiming for a single dose that leads to complete elimination of attacks and patients no longer needing medication, which they believe will be without equal.

Q: Looking at the AATD program, could you comment on how impactful the wholly owned insertional program could be for the current market?

A: John Leonard said current AATD therapies are almost non-existent, and their program aims to reset the bar with a single dose to produce fully normal alpha-1 antitrypsin protein.

Q: As we think about the 3001-PN proposed study being ex-US, do you think the FDA would like to see dosing of any US patients prior to approval?

A: John Leonard said they've reviewed with FDA and have alignment, with no requirement for US dosing in that indication as they'll see extensive exposure from the cardiomyopathy study.

Q: On the Intellia-3001 program, how do you think about the competition for patient enrollment?

A: John Leonard said they're confident in recruiting expeditiously in Phase I settings across various countries without seeing a competitive situation.

Q: For 2001, can you talk about the number of study sites for the magnitude study?

A: John Leonard said they anticipate around 100 sites globally, with over 35 active already and enrollment ahead of expectations.

Q: On the back of recently reported re-dosing data, is there any plan to build in re-dosing for certain studies?

A: John Leonard said re-dosing doesn't apply to current programs as nearly all patients treated have achieved desired reductions, and it's a one-and-done case for most.