Intellia Therapeutics, Inc. Q3 FY2024 earnings call

John Leonard:

• Progress in CRISPR-based gene editing treatments, including Phase II results of NTLA-2002 for HAE showing potential functional cure, FDA clearance for NEX-Z Phase III trial, and upcoming AHA presentation for NEX-Z.

David Lebwohl:

• NTLA-2002 Phase II results show 8 of 11 patients in 50 mg arm had no attacks post-single dose, with long-term follow-up showing continued improvement. NEX-Z Phase III trial details, including FDA clearance and ongoing enrollment in MAGNITUDE study.

Laura Sepp-Lorenzino:

• Advancing NTLA-3001 program for alpha-1 antitrypsin deficiency and R&D efforts on gene editing technologies.

Ed Dulac:

• Financials: Cash, cash equivalents, and marketable securities at $944.7M as of Sep 30, 2024; collaboration revenue down, R&D and G&A expenses up; cash expected to fund operations until late 2026.

No detailed breakdown of product segments with absolute revenue and contribution % provided. Collaboration revenue was $9.1 million in Q3 2024 vs. $12 million in Q3 2023. R&D expenses were $123.4 million in Q3 2024 vs. $113.7 million in Q3 2023. G&A expenses were $30.5 million in Q3 2024 vs. $29.4 million in Q3 2023.

Forward-looking statements:

• Expect to submit BLA for NTLA-2002 Phase III HAELO study in 2026.
• Anticipate initiating NEX-Z Phase III MAGNITUDE-2 study in ex-U.S. sites.
• Cash balance expected to fund operations until late 2026.

Potential risks:

• Uncertainties related to clinical trial outcomes, regulatory approvals for gene editing therapies, and market acceptance of CRISPR-based treatments as discussed in SEC filings.

Q: Rick Bienkowski from Cantor Fitzgerald asked about enrollment into MAGNITUDE-1.

A: John Leonard said HAELO study is a good proxy, enrolling ahead of projections with global sites activated.

Q: Matthew from Bank of America asked about capital allocation for in vivo vs ex vivo.

A: Ed Dulac mentioned focus on 3 Phase III studies, cash balance of ~$945M, and cash expected to last until late 2026.

Q: Yanan Zhu from Wells Fargo Securities asked about MAGNITUDE-2 trial design.

A: John Leonard and David Lebwohl explained placebo-controlled trial design, large TTR reduction effect size, and no silencers used in trial sites.

Q: CJ from Leerink asked about randomization in HAELO study.

A: David Lebwohl said 2-to-1 randomization is attractive to patients with 2/3 chance to start on active drug, and allows rapid enrollment.

Q: Unidentified Analyst from BMO capital Markets asked about AATP program and AAT protein during infections.

A: John Leonard and David Lebwohl discussed basing excitement on pre-clinical data showing normal protein levels, and insertion approach for alpha-1 disease.

Q: Unidentified Analyst from Leerink asked about Phase III HAELO study and randomization.

A: David Lebwohl explained 2-to-1 randomization benefits patients with 2/3 chance to start on active drug and ability to crossover.

Q: Unidentified Analyst from Gena Wang asked about silencer drop-in rate in ATTR.

A: David Lebwohl said they factored silencer drop-in into trial, expecting less drop-in than before Helios data, and patients unlikely to switch to silencers due to payer considerations.

Q: Unidentified Analyst from Jefferies asked about AHA update on biomarkers and functional capacity.

A: John Leonard and David Lebwohl mentioned sharing biomarker, functional, and clinical data at AHA, excited about insights into cardiac therapies.

Q: Unidentified Analyst from RBC asked about 18-month endpoint in TTR polyneuropathy study.

A: David Lebwohl said 18 months was their own decision as optimal time to see full benefit of drug, not required by regulators.

Q: Unidentified Analyst from Goldman Sachs asked about NTLA-2002 Phase II data and HAELO study.

A: John Leonard said Phase II data showed 12 of 15 patients achieving functional cure, expecting similar results in HAELO study with longer follow-up.

Q: Unidentified Analyst from William Blair asked about 3001 trial patient dosing and AAT levels.

A: John Leonard said they report data when meaningful, consistent, and interpretable, targeting normalization of AAT protein levels.

Q: Unidentified Analyst from Truist Securities asked about gene writing technology use.

A: John Leonard said they use gene writing in both in vivo and ex vivo settings, applying tools based on program needs, starting with alpha-1 program.

Q: Jay Olson from Oppenheimer asked about ex-U.S. strategy for NTLA-2002.

A: John Leonard said current plan is to launch drug themselves, evaluating partnerships to extend reach.

Q: Unidentified Analyst from JPMorgan asked about 3001 differentiation vs peers.

A: John Leonard said they are the only ones aware of presenting normal protein levels in non-human primates for alpha-1 program.

Q: Unidentified Analyst from Citizens JMP asked about NEX-Z Phase III trial initiation in U.S.

A: John Leonard said study will be done outside U.S. due to placebo-controlled trial feasibility and similar standards of care globally.

Q: Unidentified Analyst from Bernstein asked about expanding AHD program sites.

A: David Lebwohl said they will have other sites, and reasonable to expect data in 2025.

Q: Unidentified Analyst from Wolf Research asked about 18-month endpoint in TTR polyneuropathy study.

A: David Lebwohl said 18 months was their own decision as optimal time to see full benefit of drug, not required by regulators.

Q: Unidentified Analyst from Goldman Sachs asked about NTLA-2002 Phase II data and HAELO study.

A: John Leonard said Phase II data showed 12 of 15 patients achieving functional cure, expecting similar results in HAELO study with longer follow-up.

Q: Unidentified Analyst from William Blair asked about 3001 trial patient dosing and AAT levels.

A: John Leonard said they report data when meaningful, consistent, and interpretable, targeting normalization of AAT protein levels.

Q: Unidentified Analyst from Truist Securities asked about gene writing technology use.

A: John Leonard said they use gene writing in both in vivo and ex vivo settings, applying tools based on program needs, starting with alpha-1 program.

Q: Jay Olson from Oppenheimer asked about ex-U.S. strategy for NTLA-2002.

A: John Leonard said current plan is to launch drug themselves, evaluating partnerships to extend reach.

Q: Unidentified Analyst from JPMorgan asked about 3001 differentiation vs peers.

A: John Leonard said they are the only ones aware of presenting normal protein levels in non-human primates for alpha-1 program.

Q: Unidentified Analyst from Citizens JMP asked about NEX-Z Phase III trial initiation in U.S.

A: John Leonard said study will be done outside U.S. due to placebo-controlled trial feasibility and similar standards of care globally.

Q: Unidentified Analyst from Bernstein asked about expanding AHD program sites.

A: David Lebwohl said they will have other sites, and reasonable to expect data in 2025.

Q: Unidentified Analyst from Wolf Research asked about silencer use and safety in elderly for ATTR.

A: David Lebwohl said drug is more rapid and deeper in TTR reduction than silencers, well-tolerated in elderly patients, and no safety concerns raised.