Intellia Therapeutics, Inc. · NASDAQ:NTLA
Intellia Therapeutics — Q2 2025 Earnings Presentation
33 slides from Intellia Therapeutics's earnings presentation, Aug 7, 2025
Slide 1: Corporate Overview
August 2025 INTELLIA THERAPEUTICS Corporate Overview
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: Leveraging Gene Editing Technology to Develop Differentiated Medicines for Superior Patient Outcomes
Leveraging Gene Editing Technology to Develop Differentiated Medicines for Superior Patient Outcomes 1x Treat patients Single dose treatment Reduce burden Best-in-class at the root cause with potential to the patient and outcomes for patients of their disease lifelong benefit healthcare system 3
Slide 4: A Decade-long Mission to Bring Novel Therapies to Patients
A Decade-long Mission to Bring Novel Therapies to Patients Deep Clinical Experience Scientific Validation Nearly 200 patient-years of January 31, 2024 August 5, 2021 experience 100+ patients – significant clinical experience and data presented to date October 24, 2024 November 16, 2024 4+ years of f
Slide 5: Two Late-stage Assets with Breakthrough Profiles and Blockbuster Potentials
Two Late-stage Assets with Breakthrough Profiles and Blockbuster Potentials T H R E E C O M M E R C I AL L AU N C H E S E X P E C TE D S TA RT I N G I N 2 0 2 7 Lonvo-z* Nex-z* Target Hereditary Angioedema (HAE) Transthyretin Amyloidosis (ATTR) Indication Unique Potential to be the first to offer li
Slide 6: Bringing Innovative Solutions to Patients
Bringing Innovative Solutions to Patients THREE ACTIVE PHASE 3 STUDIES Completed Recruiting Actively Recruiting Actively Recruiting Hereditary Angioedema (HAE) Transthyretin (ATTR) Amyloidosis Transthyretin (ATTR) Amyloidosis with Cardiomyopathy (CM) with Polyneuropathy (PN) 6
Slide 7: Treatment is Designed for Patient and Provider-Friendly Experience
Treatment is Designed for Patient and Provider-Friendly Experience* DAY PRIOR DAY OF One orally- Two orally- Single outpatient Return home administered pill administered pills infusion before infusion 7 * Does not involve apheresis, myeloablative conditioning therapy, or post administration steroid
Slide 8: Focused on Completing Clinical Programs and Capitalizing on Multi-billion Dollar Commercial Opportunities
Focused on Completing Clinical Programs and Capitalizing on Multi-billion Dollar Commercial Opportunities Report Complete Launch MAGNITUDE-2 MAGNITUDE Lonvo-z* for HAE study results in enrollment in as first commercial ATTRv-PN to ATTR-CM product support BLA submission Delivering on three Phase 3 St
Slide 9: 2025 Key Milestones for Lonvo-z and Nex-z
2025 Key Milestones for Lonvo-z and Nex-z Lonvo-z Dose first patient in pivotal Phase 3 HAELO trial for HAE in 1Q25 HAE Complete enrollment in the HAELO study Present longer-term data from the Phase 1/2 study Nex-z Dose first patient in pivotal Phase 3 MAGNITUDE-2 trial for PN in 1Q25 ATTR Enroll >5
Slide 10: Pipeline Focus on Near-Term and Significant Opportunities
Pipeline Focus on Near-Term and Significant Opportunities Research and Early-Stage Late-Stage PROGRAM APPROACH PARTNERS Preclinical Clinical Clinical In Vivo: CRISPR is the therapy Lonvo-z*: Hereditary Angioedema Knockout Nex-z*: Transthyretin Amyloidosis Knockout LEAD Hemophilia B*** Insertion LEAD
Slide 11: Lonvoguran Ziclumeran (lonvo-z)
Lonvoguran Ziclumeran (lonvo-z)* For the treatment of hereditary angioedema 11 * Formerly referred to as NTLA-2002
Slide 12: Hereditary Angioedema (HAE): Currently a 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 attacks.1,2 • Average age of diagnosis is 20 years old.3 • Symptoms oft
Slide 13: Intellia is Committed to Ending the Burden of Disease by Eliminating Attacks and Chronic Treatment of HAE
Intellia is Committed to Ending the Burden of Disease by Eliminating Attacks and Chronic Treatment of HAE H AE T R E AT M E N T E V O L U T I O N Goal: SURVIVAL PREVENTION CURE Disease Attacks Treated with Attack Frequency Reduced with Lifelong Freedom Burden: On-Demand Treatment Chronic Prophylaxis
Slide 14: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Lonvo-z in Patients with HAE
A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Lonvoguran Ziclumeran (lonvo-z)* in Patients with Hereditary Angioedema (HAE) PRIMARY OBSERVATION WEEK 28 Lonvo-z (N = 40) Single 50 mg IV infusion Endpoints1 (of interest) Screening* R HAE Attack Rate; 2:1 Attack-Free Rate Placebo (N
Slide 15: Phase 2 Study: Dose-Dependent and Durable Reductions in Plasma Kallikrein Protein
Phase 2 Study: A Single Dose of Lonvo-z Showed Dose-Dependent and Durable Reductions in Plasma Kallikrein Protein Over Time 40 Lonvo-z 25 mg (n=10) 30 Lonvo-z 50 mg (n=11) Kallikrein Protein Percentage Change from Baseline 20 Placebo (n=6) 10 2% 0 -10 (Mean ± SD) -20 -30 -40 -55% -50 -60 -70 -86% -8
Slide 16: Lonvo-z Has the Potential to Free Patients from HAE Attacks and Chronic Therapy
Lonvo-z Has the Potential to Free Patients from HAE Attacks and Chronic Therapy P H AS E 1 AN D 2 R E S U LT S L O N V O - Z Phase 2 data show the potential of a single 50 mg dose to eliminate attacks and chronic prophylaxis1, 2 100% of patients had − All 11 patients had a reduction in attacks red
Slide 17: Phase 2 Study: Lonvo-z Continues to Be Well Tolerated Across All Dose Levels
Phase 2 Study: Lonvo-z Continues to Be Well Tolerated Across All Dose Levels TEAEs in ≥2 Patients After Lonvo-z Lonvo-z • All TEAEs were Grade 1 or 2* NTLA-2002 Administration 25 mg 50 mg Placebo (pooled), n (%) (n=10) (n=11) (n=6) • No SAEs in patients treated with Any TEAE 10 (100) 11 (100) 6 (100
Slide 18: Lonvo-z Has the Potential to Eliminate Attacks and Chronic Prophylaxis
Lonvo-z Has the Potential to Eliminate Attacks and Chronic Prophylaxis C R O S S T R I AL C O M PAR I S O N * % of Patients Attack-Free Product Study Phase % of Patients Attack-Free* Dosing Regimen w/o chronic prophylaxis Lonvo-z Phase 21 73% 73% 1x (investigational) at 16 weeks at 16 weeks infusion
Slide 19: Uniquely Positioned for Product Leadership in a Growing HAE Market
Uniquely Positioned for Product Leadership in a Growing HAE Market Lonvo-z demonstrated unprecedented efficacy with a single dose in an ongoing phase 1/2 study H AE M A R K E T O P P O RT U NI T Y Global market projected to Physicians seeking simpler, more effective HCPs willing to offer lonvo-z rea
Slide 20: Nexiguran Ziclumeran (nex-z)
Nexiguran Ziclumeran (nex-z)* For the treatment of ATTR amyloidosis 20 * Formerly referred to as NTLA-2001
Slide 21: 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 22: Intellia is Committed to Developing the Best Treatment for ATTR Amyloidosis
Intellia is Committed to Developing the Best Treatment for ATTR Amyloidosis AT T R T R E AT M E N T E V O L U TI O N DISEASE STASIS Goal: PALLIATION DISEASE SLOWING OR REVERSAL Disease Progressive Disease Treated with Therapies Slow but Do Not Stop or Life-long Reduction Burden: Palliative Therapy R
Slide 23: Phase 3 Study of Nex-z in Patients with ATTR Amyloidosis with Cardiomyopathy
A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Nexiguran Ziclumeran (nex-z)* in Patients with ATTR Amyloidosis with Cardiomyopathy (ATTR-CM) Primary Endpoint Nexiguran ziclumeran* Randomization • Composite endpoint of CV-related Single 55 mg IV infusion mortality and CV-re
Slide 24: Phase 3 Study of Nex-z in Patients with Hereditary ATTR Amyloidosis with Polyneuropathy
A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study 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
Slide 25: Nex-z Led to Deep, Rapid and Durable Reductions in Absolute Serum TTR
Nex-z Led to Deep, Rapid and Durable Reductions in Absolute Serum TTR in Every Patient Following a Single Dose 260 260 ATTR-CM ATTRv-PN 240 240 220 220 Serum TTR (ug/mL) Serum TTR (μg/mL) 200 200 Mean (95% CI) Mean (95% CI) 180 180 160 160 140 140 120 120 100 100 80 80 60 60 40 40 20 20 0 0 0 2 4 6
Slide 26: Nex-z Treatment Led to Stability of NT-proBNP, hs-Troponin and 6MWT Over 12 Months
Nex-z Treatment Led to Stability of NT-proBNP, hs-Troponin and 6MWT Over 12 Months Markers of Disease Progression1 NT-proBNP hs-Troponin T 6MWT Distance (meters) 2 2 100 Median (IQR) Change from Baseline Fold Change from Baseline, Fold Change from Baseline, Geometric Mean (95% CI) Geometric mean (95
Slide 27: Phase 1 Results for ATTR-CM Show Potential to be Best TTR-Directed Drug
Phase 1 Results for ATTR-CM Show Potential to be Best TTR-Directed Drug P H AS E 1 R E S U LT S N E X - Z 1 0 TTR Percent Change • Deep, rapid, consistent and durable Change in NYHA Class From Baseline* reductions in serum TTR -10 at 12 months Mean % Change (95% CI) -20 100% • Stability or improveme
Slide 28: Phase 1 Study: Nex-z Was Well Tolerated Across All Patients
Phase 1 Study: Nex-z Was Well Tolerated Across All Patients Event n (%) Event n (%) At least one AE 34 (94) Any SAE 14 (39) AEs occurring in ≥15% of patients SAEs occurring in ≥5% of patients Cardiac failure 13 (36) Cardiac failure 5 (14) COVID-19 7 (19) Acute myocardial infarction 3 (8) Upper respi
Slide 29: Preliminary Nex-z Phase 1 Data Demonstrates Potential to Improve Cardiovascular Outcomes
Preliminary Nex-z Phase 1 Data Demonstrates Potential to Improve Cardiovascular Outcomes *This graphic includes data from distinct clinical trials with their own enrollment criteria and methodologies. Cross-trial comparisons have inherent limitations and should be interpreted with caution. Nex-z res
Slide 30: Nex-z's Potential to Improve Cardiovascular Outcomes: NTLA-2001 and HELIOS-B Time to First Event
Nex-z’s Potential to Improve Cardiovascular Outcomes: NTLA-2001 and HELIOS-B Time to First Event with 95% CIs *This graphic includes data from distinct clinical trials with their own enrollment criteria and methodologies. Cross-trial comparisons have inherent limitations and should be interpreted wi
Slide 31: Positioned to Meet Patient and Provider Needs in a Large and Growing ATTR Market
Positioned to Meet Patient and Provider Needs in a Large and Growing ATTR Market AT T R M A R K E T O P P O RT U NI T Y Global market projected to Patients want a highly effective therapy High willingness of cardiologists reach ~12B dollars by 2028 and freedom from chronic treatment2 to prescribe ne
Slide 32: We are committed to making best-in-class therapies a reality
We are committed to making best-in-class therapies a reality for people suffering from difficult-to-treat diseases. OREN, SHANNA, DAMIAN Living with hereditary angioedema 32
Slide 33: Intellia Therapeutics
THERAPEUTIC S®
Intellia Therapeutics — Q2 2025 Earnings Presentation on 6ix