Intellia Therapeutics, Inc. · NASDAQ:NTLA
Intellia Therapeutics — Investor Presentation (August 2026)
37 slides from Intellia Therapeutics's investor presentation, August 2026
Slide 1: Intellia Therapeutics Corporate Overview
Intellia Therapeutics Corporate Overview August 2026
Slide 2: Intellia Therapeutics' 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: Leveraging CRISPR Gene Editing and Other Core Technologies
Our Vision: Leveraging CRISPR Gene Editing and Other Core Technologies to Transform the Lives of Patients 1x Treat patients Single dose treatment Reduce burden Best outcomes at the root cause with potential for the patient and the for patients of their disease lifelong benefit healthcare system 3 CR
Slide 4: Intellia is Leading a New Era of Medicine
Intellia is Leading a New Era of Medicine Working diligently to bring the first-ever in vivo CRISPR-based therapies to patients living with severe diseases Positive Phase 3 Readout 6 Publications in 1st in hereditary angioedema (HAE) AUG 5 CRISPR-Cas9 In Vivo Gene Editing 2021 for Transthyretin Amyl
Slide 5: Intellia's Pipeline Advancing Toward Significant Near-Term Milestones
Intellia’s Pipeline Advancing Toward Significant Near-Term Milestones Research/ Early-Stage Late-Stage BLA Program Indication Preclinical Clinical Clinical Submission Lonvo-z1 Hereditary Angioedema (HAE) Transthyretin Amyloidosis with LEAD Polyneuropathy (ATTRv-PN) Nex-z2 Transthyretin Amyloidosis w
Slide 6: Key Upcoming Objectives
Key Upcoming Objectives Lonvo-z Present topline Phase 3 HAELO data by mid-2026 HAE Present additional Phase 3 HAELO data at EAACI in June 2026 Announce BLA acceptance by FDA in second half of 2026 U.S. approval and commercial launch in first half of 2027 Nex-z Resume patient enrollment in Phase 3 MA
Slide 7: Lonvoguran Ziclumeran (lonvo-z) for Hereditary Angioedema
Lonvoguran Ziclumeran (lonvo-z) for Hereditary Angioedema
Slide 8: Hereditary Angioedema (HAE): Currently a Lifelong Condition with Significant Burden
Hereditary Angioedema (HAE): Currently a Lifelong Condition with Significant Burden Rare, genetic and life-threatening disease • Caused by a hereditary deficiency or dysfunction of the C1 inhibitor protein that leads to an imbalance in the kallikrein-kinin system and an overproduction of bradykinin
Slide 9: Despite Available Treatments, Many Patients are Unable to Break the Chronic Cycle
Despite Available Treatments, Many Patients are Unable to Break the Chronic Cycle of Managing Their HAE Patient-Reported Burdens Lifelong Significant Incomplete Treatment for Financial Impact Control for Most All Life-Threatening Burden to Attacks Maintain Access 80% reported having 68% highly 45% h
Slide 10: The Cumulative Costs for Chronic HAE Treatments are Sizable
The Cumulative Costs for Chronic HAE Treatments are Sizable U.S. Treated Patients Average Age of HAE Cumulative U.S. Healthcare System Costs for with Type 1 & 2 HAE1 Diagnosis in U.S.2 Chronic and On-Demand HAE Therapies3 ~7,000 $19.1B On Demand LTP ~2,600 On Demand ~20 $11.5B ~4,400 $15.0B LTP $3.8
Slide 11: Lonvo-z Has the Potential to be a One-Time Treatment for Hereditary Angioedema
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 reducti
Slide 12: Pooled Analysis of Phase 1/2 Clinical Data
Pooled Analysis of Phase 1/2 Clinical Data: After Becoming Attack-Free and LTP-Free for ≥6 Months, All Patients Maintained Their Response Patient ID* Baseline Monthly Phase-Initial Dose-Pt # Attack Rate† LTP initiation LTP withdrawal Attack-free and LTP-free: ≥6 months <6 months Attack: Mild Moderat
Slide 13: A Placebo-Controlled, Double-Blind, Randomized Phase 3 Trial of Lonvo-z
A Placebo-Controlled, Double-Blind, Randomized Phase 3 Trial of Lonvo-z as a One-Time HAE Treatment End of Study Day 1 Week 28 Week 104 Lonvo-z 50 mg 2:1 randomization Placebo (n=52) All patients Efficacy invited to Screening 1 Screening 2 One-time infusion evaluation Optional, Follow-up enroll in t
Slide 14: HAELO Phase 3 Demographics and Baseline Characteristics
HAELO Phase 3 Demographics and Baseline Characteristics Demographic Characteristics Lonvo-z (n=52) Placebo (n=28) Age, median years (range) 42 (23 – 71) 40 (19 – 76) Female, n (%) 35 (67%) 20 (71%) Enrolled in United States, n (%) 26 (50%) 13 (46%) Hereditary angioedema type, n (%) Type 1 49 (94%) 2
Slide 15: HAELO Trial Achieved its Primary and All Key Secondary Endpoints
HAELO Trial Achieved its Primary and All Key Secondary Endpoints Mean Attack-Rate Reduction Weeks 5 - 28 Attack Free Weeks 5 - 28 (Primary Endpoint) 1,2 (Key Secondary Endpoint) 2 2.5 70% 2.10 62% Mean HAE attacks/month 60% Percent of patients 2 50% 1.5 87% reduction 40% Odds Ratio 12.8 (95% CI: 78%
Slide 16: 100% of Patients in Lonvo-z Arm Experienced Attack-Rate Reduction
100% of Patients in Lonvo-z Arm Experienced Attack-Rate Reduction from Baseline During Weeks 5-28 Clinically relevant Attack-free and 62% 38% 72% mean attack-rate therapy-free reduction* All patients who received lonvo-z at baseline or in crossover were LTP-free * Patients with ≥1 attack during week
Slide 17: Attack-Rate Reduction Observed Quickly Following Lonvo-z Dosing
Attack-Rate Reduction Observed Quickly Following Lonvo-z Dosing; Further Reduction Observed in Early Crossover Data Following Week 28 Mean Monthly Investigator-Confirmed HAE Attack Rate Over Time 5 Lonvo-z Placebo Placebo→Lonvo-z (post crossover) Number of HAE Attacks per Start of Crossover Period 4
Slide 18: Favorable Safety and Tolerability Data
Favorable Safety and Tolerability Data Primary Observation Period • No SAEs or Grade ≥3 TEAEs Lonvo-z (N=52) Placebo (N=28) (Weeks 1 – 28) reported in lonvo-z arm TEAEs in ≥10% of patients, n (%) • All IRRs were mild or moderate and Infusion-related reaction 32 (62%) 5 (18%) were transient Headache
Slide 19: Additional HAELO Perspectives
Additional HAELO Perspectives Significant Diverse Mix Extensive Patient Enthusiasm of Patients (and Still Expanding) Phase 3 Database 80 patients enrolled Multi-national trial with (original target: ≥60) ~50% of enrolled patients Longest Phase 3 trial in U.S.; broad age range undertaken in HAE to da
Slide 20: Lonvo-z: Unique Potential to Eliminate Attacks and Ongoing Therapy with One Treatment
Lonvo-z: Unique Potential to Eliminate Attacks and Ongoing Therapy with One Treatment P H AS E 3 C R O S S - T RI AL C O M P AR I S O N * % of Patients Attack % Attack Reduction % of Patients Product & Therapy Free* vs. Placebo* Attack Free* Lifetime Dosing Burden lonvo-z 1x (investigational) 62% 87
Slide 21: Nearing Completion of Readiness Efforts for a Successful Planned Launch in 1H 2027
Nearing Completion of Readiness Efforts for a Successful Planned Launch in 1H 2027 Established core commercialization team Deployed field medical team Priorities ahead… Finalized overall launch strategy BLA acceptance Commenced payer engagement Scale field sales and reimbursement teams Continued pat
Slide 22: Nexiguran Ziclumeran (nex-z) for ATTR-CM and ATTR-PN
Nexiguran Ziclumeran (nex-z) for ATTR Cardiomyopathy (CM) and ATTR Polyneuropathy (PN)
Slide 23: Transthyretin Amyloidosis (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 •
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 Global ATTR market projected to Patients want a highly effective therapy reach ~$16.8B dollars by 20301 and freedom from chronic treatment2 $18 “It would be incredible to have a one-time therapy. This would get rid of the
Slide 25: Nex-z Has the Potential to Transform the Standard of Care for ATTR Amyloidosis
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
Slide 26: Nex-z Led to Consistent Rapid, Deep and Durable Reductions in Serum TTR
Nex-z Led to Consistent Rapid, Deep and Durable Reductions in Serum TTR: Cross-Trial Comparison to Vutrisiran TTR Profile from HELIOS-B 0 TTR Percent Change -10 From Baseline* -20 Mean % Change (95% CI) -30 -40 Research has shown a correlation -50 Vutrisiran between deeper reductions in TTR and -60
Slide 27: Phase 1 Clinical 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 275 Absolute Serum TTR 275 Absolute Serum TTR (patients with ATTR-CM) (patients with ATTRv-PN)1,* 250 250 225 225 200 200 Serum TTR (μg/mL) Serum TTR (μg/mL) Mean (95% CI) Mean (95% CI) 175 175
Slide 28: Phase 1 ATTR-CM Clinical Data: Improvement/Stabilization of Disease Progression
Phase 1 ATTR-CM Clinical Data: Improvement/Stabilization of Disease Progression Maintained Regardless of NYHA Class NT-proBNP hs-Troponin T 6MWT Distance (meters) 2 2 100 Fold Change From Baseline, Fold Change From Baseline, Change From Baseline, Geometric Mean (95% CI) Geometric Mean (95% CI) 60 Me
Slide 29: Evaluation of Mortality in Patients Treated with Nex-z Compared with a Matched Cohort
Evaluation of Mortality in Patients Treated with Nex-z Compared with a Matched Cohort of Patients Patients With ATTR-CM Matched Treated with • A cohort of ATTR-CM patients from the Characteristic* cohort nex-z P value National Amyloidosis Centre (NAC) who were (N=1751) (N=36) not receiving stabilize
Slide 30: Phase 1 ATTR-CM Clinical Data: All-Cause Mortality Rate 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 Death From Any Cause Nex-z mortality rate: 3.9 (1.5, 10.5) per 100py Survival probability Matched cohort mortality rate: 12.7 (11.4, 14.2) per 100py HR: 0.27 (0.10, 0.73), P=0
Slide 31: Phase 1 ATTR-CM Safety Summary
Phase 1 ATTR-CM Safety Summary Event n (%) At least one AE 36 (100) AEs occurring in ≥15% of patients Cardiac failure 13 (36) In this Phase 1 Trial COVID-19 8 (22) • Nex-z was generally well tolerated with IRRs being the Upper respiratory tract infection 7 (19) most frequent related AE Atrial fibril
Slide 32: Phase 3 Trial of Nex-z in Patients with ATTR-CM
A Phase 3, Randomized, Double-Blind, Placebo-Controlled Trial to Evaluate Nexiguran Ziclumeran (nex-z)* in Patients with ATTR Amyloidosis with Cardiomyopathy (ATTR-CM) Primary Endpoint Nexiguran ziclumeran* • Composite endpoint of CV-related Randomization Single 55 mg IV infusion mortality and CV-re
Slide 33: At Month 24, Majority of Patients With ATTRv-PN Experienced Improvements in mNIS+7
At Month 24, Majority of Patients With ATTRv-PN Experienced Improvements in mNIS+7 15 mNIS+7 Change From Baseline at Month 24 (Part 2) mNIS+7 Change From Baseline 10 5 0 -5 Improvement -10 -15 -20 -25 No recent patisiran use Patients who experienced disease progression during prior patisiran treatme
Slide 34: Multiple ATTRv-PN Disease-Related Clinical Measures Show Stability or Improvement
Multiple ATTRv-PN Disease-Related Clinical Measures Show Stability or Improvement Through Month 24 NfL Norfolk QoL-DN (Mean, 95% CI) (Mean, 95% CI) Change From Baseline Change From Baseline 4 4 Improvement 0 Improvemen 0 (pg/mL) -4 -4 t -8 -8 -12 -16 -12 0 2 4 6 8 10 12 14 16 18 20 22 24 0 2 4 6 8 1
Slide 35: Safety Summary in Patients with ATTRv-PN Treated with Nex-z
Safety Summary in Patients with ATTRv-PN Treated with Nex-z All Patients (N=36) • All patients received the intended dose of nex-z Safety Events n (%) At least 1 AE 36 (100) • All IRRs were Grade ≤2 and resolved AEs occurring in ≥15% of patients • Three patients had ALT and/or AST elevations >5× ULN
Slide 36: Phase 3 Trial of Nex-z in Patients with Hereditary ATTR Amyloidosis with Polyneuropathy
A Phase 3, Randomized, Double-Blind, Placebo-Controlled Trial to Evaluate Nexiguran Ziclumeran (nex-z)* in Patients with Hereditary ATTR Amyloidosis with Polyneuropathy (ATTRv-PN) Nexiguran ziclumeran* Primary Endpoints Randomization Single 55 mg IV infusion • Change from baseline in mNIS+7 at 1:1 m
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