Intellia Therapeutics, Inc. · NASDAQ:NTLA
Intellia Therapeutics — Investor Presentation (January 2026)
37 slides from Intellia Therapeutics's investor presentation, Jan 9, 2026
Slide 1: Intellia Therapeutics Corporate Overview January 2026
INTELLIA THERAPEUTICS Corporate Overview January 2026 Exhibit 99.1
Slide 2: Legal Disclaimer
Intellia Therapeutics’ Legal Disclaimer This presentation contains “forward-looking statements” of Intellia Therapeutics, Inc. (“Intellia”, “we” or “our”) within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, exp
Slide 3: Our Vision: Transform the Lives of Patients Leveraging Gene Editing Technology
Our Vision: Transform the Lives of Patients Leveraging Gene Editing Technology Treat patients at the root cause of their disease 1x Single dose treatment with potential lifelong benefit Reduce burden for the patient and the healthcare system Best outcomes for patients
Slide 4: Intellia is Leading a New Era of Medicine
Intellia is Leading a New Era of Medicine 3 Phase 3 Trials in hereditary angioedema (HAE) and transthyretin amyloidosis (ATTR-CM and ATTRv-PN) Patients Dosed with Intellia’s investigational in vivo CRISPR-based therapies 600+ Working diligently to bring the first-ever in vivo CRISPR-based therapies
Slide 5: Pipeline With Significant, Near-Term Opportunities
Pipeline With Significant, Near-Term Opportunities PROGRAM APPROACH Research and Preclinical Early-Stage Clinical Late-Stage Clinical PARTNERS In Vivo: CRISPR is the therapy Lonvo-z*: Hereditary Angioedema Knockout Nex-z**: Transthyretin Amyloidosis Knockout Hemophilia B**** Insertion Research Progr
Slide 6: Two Late-Stage Assets: Breakthrough Profiles and Blockbuster Potential
Two Late-Stage Assets: Breakthrough Profiles and Blockbuster Potential Hereditary Angioedema (HAE) Potential to be the first to offer lifelong freedom from attacks and prophylaxis with a single dose Phase 3 trial fully enrolled with 80 patients │RMAT, ODD, PRIME designation Expect to present Phase 3
Slide 7: Key 2025 Accomplishments and Upcoming Objectives
Key 2025 Accomplishments and Upcoming Objectives Lonvo-z HAE Presented longer-term data from Phase 1/2 clinical trial Completed enrollment of 80 patients in global Phase 3 HAELO trial Present topline Phase 3 HAELO data by mid-2026 Planned BLA submission in second half of 2026 Planned U.S. commercial
Slide 8: Lonvoguran Ziclumeran (lonvo-z) for Hereditary Angioedema
Lonvoguran Ziclumeran (lonvo-z) for Hereditary Angioedema
Slide 9: Hereditary Angioedema: Life-Long Genetic Condition with Significant Burden
Hereditary Angioedema (HAE): Currently a Life-Long Genetic Condition with Significant Burden Rare, genetic and life-threatening disease Patients have unpredictable, recurrent, painful and potentially life-threatening swelling attacks1,2 Average age of diagnosis is 20 years old3 Symptoms often begin
Slide 10: HAE: Significant Market Opportunity – Ripe for Disruptive Innovation
HAE: Significant Market Opportunity – Ripe for Disruptive Innovation 1. Evaluate Pharma Consensus Analyst forecasts (October 2025). 2. Castaldo et al. Allergy Asthma Immunol, 2025. 3. Mendivil et al. Allergy, Asthma & Clinical Immunology, 2023. 4. Lumry et al. ACAAI poster, 2022. B: billion; HAE: he
Slide 11: Lonvo-z Has the Potential to be a One-Time Treatment for HAE
Lonvo-z Has the Potential to be a One-Time Treatment for Hereditary Angioedema First and only investigational therapy that targets the KLKB1 gene, to reduce production of kallikrein protein at its source Observations from Phase 1/2 trial: Consistently rapid, deep and durable kallikrein reductions Fr
Slide 12: Pooled Analysis of Phase 1/2: Deep Kallikrein Reduction Remained Stable
Pooled Analysis of Phase 1/2 Clinical Data: Deep Kallikrein Reduction Remained Stable After a One-Time Treatment with Lonvo-z 50 mg Data cutoff: August 29, 2025. One month = 30.4375 days. This presentation includes data for an investigational product not yet approved by regulatory authorities. BL: b
Slide 13: Most Patients Attack-Free After One-Time Lonvo-z 50 mg
Most Patients were Attack-Free After a One-Time Treatment with Lonvo-z 50 mg; Attack Rate was Low and Stable with Up to 3 Years of Follow-Up Pooled Phase 1/2 Analysis Data cutoff date: August 29, 2025. The monthly attack rate is calculated as number of attacks that occurred during each 28-day interv
Slide 14: After Becoming Attack-Free and LTP-Free for ≥6 Months, All Maintained Response
Pooled Analysis of Phase 1/2 Clinical Data: After Becoming Attack-Free and LTP-Free for ≥6 Months, All Patients Maintained Their Response Data cutoff date: August 29, 2025. Phase 1 eligibility was determined by historical attack period. a. Patient IDs align with prior Phase 1 and Phase 2 publication
Slide 15: Lonvo-z Well Tolerated with No Long-Term Risks Observed
A One-Time Treatment of Lonvo-z 50 mg was Well Tolerated with No Long-Term Risks Observed with Up to 3 Years of Follow-Up Pooled Phase 1/2 Analysis Data cutoff date: August 29, 2025. a. AEs that occurred after each patient received lonvo-z 50 mg are reported. b. The patient had multiple risk factors
Slide 16: Comparison of HAE Patient Survey vs. Lonvo-z Clinical Data
Comparison of HAE Patient Survey vs. Lonvo-z Clinical Data Most Surveyed Patients Reported They Continue to Have HAE Attacks1 Data cutoff date: August 29, 2025. 1. Busse, P et al. ACAAI poster, 2025. N=100 2. Cohn, DM, et al. Phase 1/2 pooled analysis of patients receiving a 50 mg dose of lonvo-z pr
Slide 17: Market Research: Most Surveyed Patients Very Likely to Use Lonvo-z
Intellia Market Research: Most Surveyed Patients Were Very Likely to Use “Product X” (Lonvo-z), Including Those on Leading LTPs 1. Intellia Commissioned Market Research Study Conducted with 104 US Patients and Caregivers in November and December 2025. LTP: long term prophylaxis. Unlikely (1 or 2) So
Slide 18: Physicians Reported High Enthusiasm for Lonvo-z Target Profile
Physicians Reported a High Degree of Enthusiasm for Lonvo-z’s Target Profile1 Physicians Reported a Willingness to Prescribe Lonvo-z to Most Patients1 Intellia Market Research: Vast Majority of 151 Surveyed Physicians Were Willing to Prescribe “Product X” (Lonvo-z) 1. Intellia Commissioned Market Re
Slide 19: Phase 3 Clinical Trial of Lonvo-z in HAE Fully Enrolled With 80 Patients
Clinicaltrials.gov ID: NCT06634420. * Patients on long-term prophylaxis are required to wash out of therapy prior to the run-in period of screening. 1. Week 28 data expected to support BLA filing in second half of 2026. After week 28, participants will be eligible for an optional blinded crossover,
Slide 20: Preparing for a Successful Planned Commercial Launch in 1H 2027
Preparing for a Successful Planned Commercial Launch in 1H 2027 Core commercialization team in place Scale field sales and reimbursement teams Finalize distribution model Identify treatment centers Finalize pricing Contracting strategy Priorities in 2026… Overall launch strategy in place Field medic
Slide 21: Lonvo-z Has Unique Potential to Eliminate Attacks and Ongoing Therapy
Lonvo-z Has the Unique Potential to Eliminate Attacks and Burdensome Ongoing Therapy * This graphic includes data from the blinded time periods of distinct clinical trials with their own enrollment criteria and methodologies. Cross-trial comparisons have inherent limitations and should be interprete
Slide 22: Nexiguran Ziclumeran (nex-z) for ATTR Cardiomyopathy and Polyneuropathy
Nexiguran Ziclumeran (nex-z) for ATTR Cardiomyopathy and ATTR Polyneuropathy
Slide 23: ATTR: Large and Growing Market with Significant Unmet Need
Transthyretin Amyloidosis (ATTR): Large and Growing Market with Significant Unmet Need Severe, fatal, progressive disease with shortened life expectancy CM patients have debilitating shortness of breath, arrythmias, reduced mobility and quality of life, as well as a high rate of hospitalization Wild
Slide 24: Seeking to Meet Patient and Provider Needs in a Large and Growing ATTR Market
Seeking to Meet Patient and Provider Needs in a Large and Growing ATTR Market If approved, nex-z would present an opportunity for significant revenues and healthcare system savings 1. Consensus analyst forecasts per FactSet (Dec. 2025). 2. Intellia commissioned ATTR patient and caregiver interviews
Slide 25: Nex-z Has the Potential to Transform the Standard of Care for ATTR
Nex-z Has the Potential to Transform the Standard of Care for ATTR Amyloidosis First and only investigational therapy that targets the TTR gene, to reduce production of TTR protein at its source Potential to provide patients with: Consistently rapid, deep and durable TTR reduction Stability or impro
Slide 26: Nex-z Consistent Rapid, Deep, Durable TTR Reductions: Cross-Trial Comparison to Vutrisiran
Nex-z Led to Consistent Rapid, Deep, and Durable Reductions in Serum TTR: Cross-Trial Comparison to Vutrisiran TTR Profile from HELIOS-B Vutrisiran2 190 180 182 178 160 175 Nex-z 36 35 36 36 36 36 Nex-z Vutrisiran Mean % Change (95% CI) * This graphic includes data from distinct clinical trials with
Slide 27: Phase 1 Data: One-time Nex-z Dose Led to Durable Serum TTR Reduction
Phase 1 Clinical Data: One-time Nex-z Dose Led to Consistent, Rapid, Deep and Durable Serum TTR Reduction Data cutoff date: August 23, 2025. Data at Month 36 collected from patients who entered the long-term follow-up safety monitoring study (data-cutoff date: August 21, 2025). Changes in N from tot
Slide 28: Phase 1 ATTR-CM Data: Improvement/Stabilization Maintained Regardless of NYHA Class
Phase 1 ATTR-CM Clinical Data: Improvement/Stabilization of Disease Progression Maintained Regardless of NYHA Class NT-proBNP hs-Troponin T 6MWT Distance (meters) Data cutoff date: August 23, 2025. Changes in N from total population (N=36) reflect patient death (n=2), withdrawal (n=1), or missing as
Slide 29: Evaluation of Mortality in Nex-z Patients Compared with Matched Cohort
Evaluation of Mortality in Patients Treated with Nex-z Compared with a Matched Cohort of Patients a. Numbers for patients treated with nex-z represent values at trial baseline. b. Nex-z patient population includes 2 homozygous patients. ATTR-CM: ATTR amyloidosis cardiomyopathy; CKD-EPI: chronic kidn
Slide 30: ATTR-CM: All-Cause Mortality ~3-Fold Lower than Matched Cohort
Phase 1 ATTR-CM Clinical Data: All-Cause Mortality Rate was ~3-Fold Lower than that Observed in a Matched Cohort of Patients a. Tafamidis was initiated in patients in both cohorts shortly after it became available in the UK. Follow up truncated to the last follow up time available for nex-z patients
Slide 31: Phase 1 ATTR-CM Safety Summary
Phase 1 ATTR-CM Safety Summary Data cutoff date: August 23, 2025. a. In addition, 1 patient in the long-term follow-up had unrelated serious adverse events. b. One death was due to ischemic heart disease on Day 506, one due to decompensated heart failure on Day 675, one due to decompensated congesti
Slide 32: At Month 24, Majority of ATTRv-PN Patients Experienced Improvements in mNIS+7
At Month 24, Majority of Patients With ATTRv-PN Experienced Improvements in mNIS+7 mNIS+7 Change From Baseline No recent patisiran use Patients who experienced disease progression during prior patisiran treatment mNIS+7 Change From Baseline at Month 24 (Part 2) Improvement 66 4 2 8 79 45 7 22 27 79
Slide 33: Multiple ATTRv-PN Clinical Measures Show Stability or Improvement Through Month 24
NfL (Mean, 95% CI) 35 (N) 36 36 34 Change From Baseline (pg/mL) Improvement Multiple ATTRv-PN Disease-Related Clinical Measures Show Stability or Improvement Through Month 24 Norfolk QoL-DN (Mean, 95% CI) Change From Baseline 35 (N) 36 36 34 Improvement Change From Baseline (kg/m2 × g/L) mBMI (Mean,
Slide 34: Safety Summary in Patients with ATTRv-PN Treated with Nex-z
Safety Summary in Patients with ATTRv-PN Treated with Nex-z Safety Events All Patients (N=36) n (%) At least 1 AE 36 (100) AEs occurring in ≥15% of patients IRR 21 (58) Headache 10 (28) Diarrhea 8 (22) Thyroxine decreased 8 (22)a AST increased 6 (17)b Any serious AE 11 (31) Treatment-related SAEs 3
Slide 35: Working to Resolve Clinical Holds on MAGNITUDE and MAGNITUDE-2
Working to Resolve Clinical Holds on MAGNITUDE and MAGNITUDE-2 Phase 3 Clinical Trials Initiated enrollment: March 2024 Accruing data from enrolled patients; expect to provide an update after we have finalized a plan with regulators on the path forward Patients enrolled: >650 Enrollment target: ~1,2
Slide 36: Established CRISPR Leader Working Diligently to Deliver First In Vivo Gene Therapies
Established CRISPR Leader Working Diligently to Deliver the First Ever In Vivo Gene Therapies to Patients Nex-z: A potential one-time treatment for ATTR-CM and ATTRv-PN Aiming to stabilize or reversing course of disease for most patients Rapidly growing multi-billion-dollar market Majority of patien
Slide 37: Intellia Therapeutics
THERAPEUTICS®
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