Intellia Therapeutics — November 12, 2026 Earnings Summary

Financial Performance:

• The press release contains no specific financial performance data, including revenue, earnings, profit margins, cash flow, debt, or deferred revenue figures.
• No specific financial metrics or industry-relevant financial data are disclosed in the document.

Guidance and Future Outlook:

• The Biologics License Application (BLA) for lonvo-z (lonvoguran ziclumeran) for the treatment of hereditary angioedema (HAE) was accepted by the FDA for priority review.
• The Prescription Drug User Fee Act (PDUFA) goal date is set for March 10, 2027.
• The FDA has indicated it is not currently planning to hold an advisory committee meeting to discuss the application.
• Intellia anticipates a commercial launch of lonvo-z following potential approval in March 2027.
• The company is preparing for a one-time therapy launch, having built a dedicated team and network of authorized treatment centers (ATCs) in anticipation of approval.

Business Segments and Product Lines:

• Lonvo-z (Lonvoguran Ziclumeran): An investigational in vivo CRISPR-based therapy designed to permanently inactivate the KLKB1 gene to treat HAE.
• Clinical Trial Results (HAELO Phase 3):
  • Achieved primary and all key secondary endpoints.
  • Demonstrated a 62% mean attack-rate reduction (Weeks 5–28) compared to placebo (p<0.0001).
  • Showed an 87% reduction in attack-free weeks (Weeks 5–28) compared to placebo (p<0.0001).
  • Patients treated with lonvo-z were approximately 3 times more likely to achieve complete control (attack-free) compared to their prior long-term prophylaxis (LTP).
  • 100% of patients were LTP-free as of the data cutoff (February 10, 2026).
  • Kallikrein reduction remained deep and durable with up to 4 years of follow-up, showing no waning of effect.
• Safety Profile:
  • Based on 117 patients treated (93 received 50 mg dose) with up to 4 years of follow-up.
  • Most common Treatment Emergent Adverse Events (TEAEs) were infusion-related reactions (55%), headache (19%), fatigue (18%), nasopharyngitis (16%), and upper respiratory tract infection (14%).
  • Grade 3 TEAEs occurred in less than 1% of patients (pulmonary embolism and perforated gangrenous appendicitis, neither considered treatment-related).
  • Serious Adverse Events (SAE) occurred in less than 1% of patients.
• Pipeline:
  • Two additional Phase 3 trials are ongoing for transthyretin amyloidosis (ATTR-CM and ATTRv-PN).
  • Over 600 patients have been dosed with Intellia's investigational in vivo CRISPR-based therapies.
  • 6+ publications in 6 August 2021 regarding CRISPR-Cas9 in vivo gene editing.

Market and Competitive Landscape:

• Market Size: Approximately 7,500 U.S. adults are treated for HAE.
• Current Treatment Costs: A single patient on long-term prophylaxis (LTP) can cost the U.S. healthcare system over $6 million per decade (approx. $641,000 per year).
• Patient Demand: 79% of patients are very worried about needing lifelong therapy; 78% are very interested in no longer taking LTP.
• Physician Interest: 88% of surveyed HCPs would prescribe lonvo-z within one year of approval; 65% would prescribe to at least one patient, and 54% (core prescribers) would prescribe to ≥50% of their patients.
• Competitive Advantage: Lonvo-z is the clear preference leader, with 2.8x the patient preference of the highest-ranked clinical-stage LTP (Deucrictibant).
• Market Burden: 90% of HAE management time is not reimbursable, and HCPs report significant staff time strains relative to patients managed.

Risks and Challenges:

• Regulatory Risk: The presentation contains forward-looking statements subject to risks regarding the ability to obtain approval for the BLA and successfully launch lonvo-z.
• Commercialization Risk: Risks include the ability to successfully commercialize lonvo-z, protect intellectual property, and maintain relationships with third parties (contract manufacturers, licensors).
• Clinical Risk: Uncertainties related to the authorization, initiation, and conduct of preclinical and clinical studies; results of studies may not be predictive of future results.
• Financial Risk: Risks related to Intellia's future financial condition and ability to fund operations.
• Collaboration Risk: Risks related to collaborations with Regeneron Pharmaceuticals, Inc. or other partners not continuing or being successful.
• Launch Execution Risk: Lessons from prior one-time therapy launches indicate potential hurdles in coverage/reimbursement timelines, authorized treatment center (ATC) network preparation, and hiring.

Management Commentary and Tone:

• Dr. John Leonard (CEO): Emphasized that Intellia is leading a new era of medicine with a strong strategy and full team in place. Stated the company is "advancing toward launch" with proactive work underway.
• Dr. Jing L. Marantz (CMO): Highlighted the robust clinical data, including deep and durable kallikrein reduction and favorable safety profiles up to 4 years.
• Dr. Marc Riedl & Dr. Danny Cohn: Described the patient experience as "life-changing," with patients reporting freedom from HAE burdens and anxiety.
• Maria Natale (SVP, Commercial): Outlined a comprehensive launch execution plan, including payer outreach since early 2025, ATC network building, and ongoing education for HCPs and patients.
• Tone: The tone is highly confident, optimistic, and focused on the transformative potential of a one-time therapy to redefine HAE treatment.

Analyst Questions and Answers:

• No analyst questions and answers are included in the provided press release text.

Other Key Points:

• Patient Perspectives: Qualitative interviews with 23 patients showed 100% reported HAE as well-controlled after lonvo-z, compared to 48% before. Patients described feeling "free" and "like they haven't got it anymore."
• Launch Readiness: Intellia has assembled a dedicated team with an average of 19 years in the industry and experience in 15 CGT launches.
• Payer Engagement: Outreach began in early 2025, engaging payers covering 280M+ U.S. lives. Payers value the one-time dosing, durable control, and potential cost offsets compared to chronic LTPs.
• Network Strategy: The goal is to have most patients within 100 miles of an Authorized Treatment Center (ATC), utilizing a "buy-and-bill" model via specialty pharmacies to eliminate upfront funding exposure.
• Timeline: The presentation outlines a projected Year One momentum plan, with first revenue generation expected between months 3-6 post-approval.
• Historical Milestones: Intellia achieved the world's first patient dosed (2020), first in vivo gene editing clinical data (2021), first Phase 3 initiated (2024), and first fully enrolled Phase 3 (2025).