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Intellia Therapeutics 43rd Annual J.P. Morgan Healthcare Conference John Leonard, M.D. President and Chief Executive Officer January 13, 2025
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2 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, express or implied statements about Intellia’s beliefs and expectations regarding: our ability to successfully develop and commercialize nexiguran ziclumeran (“nex-z”), formerly known as NTLA-2001, for the treatment of transthyretin (“ATTR”) amyloidosis and NTLA-2002 for the treatment of hereditary angioedema (“HAE”) to address the significant unmet needs of patients and prescribers in HAE and ATTR amyloidosis; our ability to achieve near-term clinical milestones, including dosing the first patient in the Phase 3 HAELO trial for NTLA-2002 in the first quarter of 2025, completing enrollment in the Phase 3 HAELO trial in the second half of 2025, dosing the first patient in the Phase 3 MAGNITUDE-2 trial for hereditary ATTR amyloidosis with polyneuropathy (“ATTRv-PN”) in the first quarter of 2025 and completing enrollment in 2026, enrolling at least 550 patients across the Phase 3 MAGNITUDE trial for ATTR amyloidosis with cardiomyopathy (“ATTR-CM”) in 2025, substantially completing enrollment of the MAGNITUDE trial for ATTR-CM in 2026, and completing enrollment of the MAGNITUDE trial for ATTR-CM in 2027; the expected timing of data releases from our clinical trials of nex-z and NTLA-2002, including presenting longer-term data from the Phase 1/2 study of NTLA-2002, longer-term data from the Phase 1 study of nex-z in 2025, results from the HAELO trial for NTLA-2002 in 2026, and results from the MAGNITUDE-2 trial for ATTRv-PN in 2027; our ability to prepare for commercial launch, including having all commercial capabilities in place by end of 2026; our interactions with regulatory authorities, including the potential submission of a biologics license application for NTLA-2002 for the treatment of HAE in 2026; our ability to launch NTLA-2002 as our first commercial product in 2027; our ability to optimize the impact of our collaborations on our development programs, including our collaboration with Regeneron Pharmaceuticals, Inc. and their co- development program for ATTR amyloidosis, and to advance additional development candidates; our expectations regarding our uses of capital, expenses, and ability to fund operations through first commercial launch in the first half of 2027; and the potential commercial opportunities, including the value and market potential for our product candidates, including the potential of nex-z and NTLA-2002 to be single-dose treatments, the potential of nex-z to halt and reverse disease, result in lifelong, stable TTR reduction, be best TTR-directed drug, and represent a meaningful revenue opportunity starting in 2029, and the potential of NTLA-2002 to be a functional cure, eliminate significant treatment burden, eliminate HAE attacks and chronic prophylaxis, and represent a meaningful revenue opportunity starting in 2027. Any forward-looking statements in this presentation are based on management’s current expectations and beliefs of future events, and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: risks related to Intellia’s ability to protect and maintain its intellectual property position; risks related to Intellia’s relationship with third parties, including our contract manufacturers, licensors and licensees; risks related to the ability of our licensors to protect and maintain their intellectual property position; uncertainties related to the authorization, initiation and conduct of preclinical and clinical studies and other development requirements for our product candidates, including uncertainties related to regulatory approvals to conduct clinical trials; risks related to the ability to develop and commercialize any one or more of Intellia’s product candidates successfully; risks related to the results of preclinical studies or clinical studies not being predictive of future results in connection with future studies; the risk that clinical study results will not be positive; risks related to the development and advancement of novel platform capabilities, such as DNA writing technology and gene editing in tissues outside the liver; risks related to Intellia’s future financial condition and our ability to fund our operations; risks related to Intellia’s collaborations with Regeneron Pharmaceuticals, Inc. or our other collaborations not continuing or not being successful; and risks related to our Intellia's ability to recruit and retain a management team and other key personnel to execute its strategic plans, including completing pivotal clinical trials and commercial lauch of its product candidates, such as nex-z and NTLA- 2002. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Intellia’s actual results to differ from those contained in the forward- looking statements, see the section entitled “Risk Factors” in Intellia’s most recent Quarterly Report on Form 10-Q as well as discussions of potential risks, uncertainties, and other important factors in Intellia’s other filings with the Securities and Exchange Commission. All information in this presentation is as of the date on its cover page, and Intellia undertakes no duty to update this information unless required by law.
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3 Leveraging Gene Editing Technology to Develop Differentiated Medicines for Superior Patient Outcomes Treat patients at the root cause of their disease Reduce burden to the patient and healthcare system 1x Single dose treatment with potential lifelong benefit Best-in-class outcomes for patients
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4 Regulatory and Scientific Expertise Deep Clinical Experience Bringing Innovative Solutions to Patients A Decade-long Mission to Bring Novel Therapies to Patients 3 actively enrolling Phase 3 studies 12+ health authority approvals for clinical studies Multiple regulatory designations 4 New England Journal of Medicine publications Nearly 200 patient-years of experience 100+ patients significant clinical experience and data presented to date 4+ years of follow-up in earliest dosed patients FDA FTD FDA ODD FDA RMAT MHRA IP Abbreviations: FTD – Fast Track Designation; ODD – Orphan Drug Designation; RMAT – Regenerative Medicine Advanced Therapy; IP – Innovation Passport; PRIME – Priority Medicines EMA PRIME
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5 Hereditary Angioedema (HAE) Potentially first to offer lifelong freedom from attacks and prophylaxis after a single dose Phase 3 initiated │RMAT, ODD, PRIME BLA submission planned in 2026 WW Prevalence ~150,0001 Projected to reach global sales of $5B2 by 2028 Transthyretin amyloidosis (ATTR) Potential to be the first to stabilize or reverse disease progression with a single dose Phase 3 enrolling │RMAT & ODD designation PN BLA submission planned in 2028 CM enrollment completion by early 2027 WW Prevalence 250,000 to 500,0003-6 Projected to reach global sales of $12B2 by 2028 Target Indication Unique Proposition Program Status Total Market NTLA-2002 Nex-z* Two Late-stage Assets with Breakthrough Profiles and Blockbuster Potentials * Nex-z (nexiguran ziclumeran), formerly referred to as NTLA-2001. 1. Zuraw et al 2008; 2. Evaluate Pharma Consensus Analyst forecasts October-December 2024; 3. Hawkins et al, 2015; 4. Maurer et al, 2019; 5. Nativi-Nicolau et al, 2021; 6. Gillmore et al, 2022 Abbreviations: RMAT – Regenerative Medicine Advanced Therapy; ODD - Orphan Drug Designation; PRIME – Priority Medicine; BLA – Biologics License Application; WW – worldwide; PN – Polyneuropathy; CM - Cardiomyopathy Data for 108 patients presented across programs; robust treatment effect continues at longest follow up through 2 years THREE COMMERCIAL LAUNCHES EXPECTED STARTING IN 2027
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6 Treatment is Designed for Patient and Provider-Friendly Experience* DAY OFDAY PRIOR One orally- administered pill Two orally- administered pills before infusion Single outpatient infusion Return home * does not involve apheresis, myeloablative conditioning therapy, or post administration steroid therapy
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7 Phase 3 HAELO results and first planned BLA submission in 2026 Complete enrollment for MAGNITUDE-2 in ATTR-PN Substantially complete enrollment for MAGNITUDE in ATTR-CM Continue to build for commercial success Building on Recent Accomplishments NTLA- 2002 Nex-z 2024 Key Achievements 2025 Operational Excellence 2026 Readying for Commercialization Complete Target Enrollment by YE Cumulative Enrollment >550 Patients by YE • Clinical data suggest nex-z may halt and potentially reverse disease progression for ATTR amyloidosis • Phase 1/2 data indicate NTLA-2002 may be a functional HAE cure for most patients • Nex-z RMAT designation for ATTR PN • Initiated 3 pivotal Phase 3 studies Abbreviations: RMAT – Regenerative Medicine Advanced Therapy; ATTR – Transthyretin amyloidosis; HAE – Hereditary Angioedema; PN - Polyneuropathy
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8 • Clinical data suggest nex-z may halt and potentially reverse disease progression for ATTR amyloidosis • Phase 1/2 data indicate NTLA-2002 may be a functional HAE cure for most patients • Nex-z RMAT designation for ATTR PN1 • Initiated 3 pivotal Phase 3 studies Phase 3 HAELO results and first planned BLA submission in 2026 Complete enrollment for MAGNITUDE-2 in ATTR-PN Substantially complete enrollment for MAGNITUDE in ATTR-CM Continue to build for commercial success Accelerating Clinical Development NTLA- 2002 Nex-z 2024 Key Achievements 2025 Operational Excellence 2026 Readying for Commercialization Complete Target Enrollment by YE Cumulative Enrollment >550 CM Patients by YE Abbreviations: CM – Cardiomyopathy; YE – yearend
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9 • Clinical data suggest nex-z may halt and potentially reverse disease progression for ATTR amyloidosis • Phase 1/2 data indicate NTLA-2002 may be a functional HAE cure for most patients • Nex-z RMAT designation for ATTR PN1 • Initiated 3 pivotal Phase 3 studies Phase 3 HAELO results and first planned BLA submission in 2026 Complete enrollment for MAGNITUDE-2 in ATTR-PN Substantially complete enrollment for MAGNITUDE in ATTR-CM Continue to build for commercial success Preparing the Market for Launch NTLA- 2002 Nex-z 2024 Key Achievements 2025 Operational Excellence 2026 Readying for Commercialization Complete Target Enrollment by YE Cumulative Enrollment >550 Patients by YE Abbreviations: BLA – Biologics License Application; ATTR – Transthyretin amyloidosis; PN – Polyneuropathy; CM - Cardiomyopathy
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10 Phase 3 HAELO results and first planned BLA submission in 2026 Complete enrollment for MAGNITUDE-2 in ATTR-PN Substantially complete enrollment for MAGNITUDE in ATTR-CM Continue to build for commercial success Maturing as a Fully-integrated, Commercial-stage Company • Clinical data suggest nex-z may halt and potentially reverse disease progression for ATTR amyloidosis • Phase 1/2 data indicate NTLA-2002 may be a functional HAE cure for most patients • Nex-z RMAT designation for ATTR PN1 • Initiated 3 pivotal Phase 3 studies NTLA- 2002 Nex-z 2024 Key Achievements 2025 Operational Excellence 2026 Readying for Commercialization Complete Target Enrollment by YE Cumulative Enrollment >550 CM Patients by YE Abbreviations: RMAT – Regenerative Medicine Advanced Therapy; ATTR – Transthyretin amyloidosis; HAE – Hereditary Angioedema; PN – Polyneuropathy; CM – Cardiomyopathy; YE – yearend; BLA – Biologics License Application
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11 Focused on Completing Clinical Programs and Capitalizing on Multi-billion Dollar Commercial Opportunities Launch NTLA-2002 for HAE as first commercial product Report MAGNITUDE-2 study results in ATTR-PN to support BLA submission Complete MAGNITUDE enrollment in ATTR-CM Delivering on 3 Phase 3 Studies by 2027
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NTLA-2002
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13 DAMIAN Living with HAE Hereditary Angioedema: Currently a Life-long Genetic Condition with Significant Burden ▪ Patients have unpredictable, recurrent, painful and potentially life-threatening swelling attacks.1,2 ▪ Symptoms often begin in the first decade of life and typically worsen in puberty.3,4 ▪ Attacks can be triggered by stress, trauma, infection, fatigue, and hormones.2 ▪ Approximately 6K patients in the US.5 Rare, genetic and life-threatening disease Despite available treatments, significant unmet need persists ▪ Many patients only achieve partial clinical control.6,7,8 ▪ Patients make lifestyle modifications to manage fear and anxiety.9 ▪ Treatment burden negatively affects patients, especially those taking injectable medications.10 ▪ Insurance delays and denials associated with maintaining access have significant impacts on individuals with HAE.11 1 Zuraw, NEJM (2008), 2 Busse and Christiansen, NEJM (2020), 3 Norris et al., Allergy Asthma Proc. (2022), 4 Pancholy et al., Curr Opin. Pediatr. (2019), 5 Busse et al., JACI In Practice (2021), 6 Banerji et al., JAMA (2018) 7 Zuraw et al., Allergy Clin. Immunol. (2021), 8 Longhurst et al., NEJM (2017), 9 Bork et al., Allergy Asthma Clin. Immunol. (2021), 10 Radojicic et al., Allergy Asthma Proc. (2021), 11 Arora et al., JACI In Practice (2023)
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14 HAE TREATMENT EVOLUTION Intellia is Committed to Ending the Disease and Treatment Burden of HAE PREVENTION Treatment Burden: Disease Burden: Multiple Injections / Hundreds of Pills Annually Attack Frequency Reduced with Chronic Prophylaxis Dozens of Injections Annually Attacks Treated with On-Demand Treatment CUREGoal: “I would love to not have to ever take another injection or another pill. It would be amazing” “I am hesitant to switch jobs because I know these are expensive treatments and I may not always have access” “Treatment has improved but I am still experiencing a high number of attacks” Patients report significant disease and treatment burden with available therapies1 Life-long Freedom from Chronic Therapy Life-long Freedom from Attacks Source: 1. Intellia commissioned HAE patient and caregiver interviews (n=58) SURVIVAL
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15 NTLA-2002 Has the Potential to Be a Functional Cure for Patients With HAE *Safety findings: Most common AEs (> 25%) were headache (36%), fatigue (27%), nasopharyngitis (27%), and infusion-related reaction (27%). All TEAEs were grade 1 and 2. 1 Cohn et al., NEJM (2024) 2 Cohn et al., ACAAI (2024) https://www.intelliatx.com/wp-content/uploads/Intellia_NTLA-2002-Phase-2-Data-Investor-Presentation_10.24.24_vF.pdf 3 Longhurst, EAACI (2024) https://www.intelliatx.com/wp-content/uploads/EAACI-NTLA-2002-Phase-1-Update_2June24.pdf 100% of patients had reduction in attacks 73% of patients attack free and off chronic prophylaxis ▪ Phase 2 data show the potential of a single 50mg dose to eliminate attacks and chronic prophylaxis1, 2 – All 11 patients had a reduction in attacks – All but 1 patient remained free from chronic prophylaxis – Most patients achieved complete elimination of attacks – Safety profile continued to be highly encouraging* ▪ Demonstrated durability through 2 years in phase 13 PHASE 1 & 2 RESULTS NTLA -2002
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16 *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 interpreted with caution.". Sources: 1. Cohn et al., NEJM (2024), 2. Riedl et al, N. Engl. J. Med (2024), 3. Craig et al, Lancet (2023), 4. Banjeri, et al, JAMA (2018), 5. Zuraw et al, J. All. Clin. Imm. (2021), 6. Wolfe Research (2024), 7. Longhurst et al, N. Engl. J Med (2017), 8. CINRYZE FDA Package Insert; Abbreviations: HAE – Hereditary Angioedema; TEAEs – Treatment Emergent Adverse Events; Wks – Weeks PRODUCT Study Phase % of Patients Attack-Free* % of Patients Attack-Free w/o chronic prophylaxis DOSING REGIMEN NTLA-2002 (investigational) Phase 21 infusion / lifetime Donidalorsen (investigational) Phase 32 0% 6–12 injections / year Garadacimab (investigational) Phase 33 0% 12 injections / year Phase 34 0% 13–26 injections / year Phase 35,6 No statistical difference 0% Daily oral tablets Phase 37 Not measured 0% 104 injections / year 73% at 16 wks 62% at 26 wks 35-43% at 24 wks 31-44% at 26 wks CROSS TRIAL COMPARISON* 1x x365 x104 For illustrative purposes only. NTLA-2002 has the Potential to Eliminate Attacks and Chronic Prophylaxis 73% at 16 wks
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17 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 2026. Optional Blinded Crossover @ week 28. Patients will be observed in extended follow-up. 2:1 Placebo (N = 20) NTLA-2002 (N = 40) Single 50 mg IV infusion Screening* PRIMARY OBSERVATION R Endpoints1 (of interest) HAE Attack Rate; Attack-Free Rate NTLA-2002 Upcoming 2025 Milestones WEEK 28 Dose first patient in pivotal Phase 3 HAELO trial for HAE in 1Q25 Complete enrollment in the HAELO study Present longer-term data from the Phase 1/2 study A Phase 3, Randomized, Double-blind, Placebo-Controlled Study of NTLA-2002 in Patients with HAE
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18 3.0 3.5 4.9 2024 2026 2028 Uniquely Positioned for Product Leadership in a Growing HAE Market NTLA-2002 demonstrated unprecedented efficacy with a single dose NTLA-2002 represents a meaningful revenue opportunity starting in 2027 HCPs willing to offer NTLA-2002 to all patients regardless of severity in the first 3 years3 Physicians seeking simpler, more effective solutions and easier access to therapy for patients2 HAE MARKET OPPORTUNITY “If you're telling me that there's a medicine that doesn't require repeat dosing, that's like nothing else we have available.” U.S. HAE HCP “What it's offering very simply is the potential for having a onetime therapy that over time, if all goes well, allows patients to not require any more treatment and potentially not even any more disease management…” U.S. HAE HCP "I have to go through the paperwork every year with these patients and every time they change insurances...We actually have a whole section in our clinic with staff dedicated to getting these medicines approved. That's how much of a burden it is." U.S. HAE HCP 1 Source: 1. Evaluate Pharma Consensus Analyst forecasts as of October 2024; 2. Intellia commissioned HAE HCP qualitative study post Phase 2 data readout; 3. Intellia commissioned Physician quantitative survey (n=192) based on NTLA-2002 target product profile Global market projected to reach ~5B dollars by 2028 75%
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Nex-z Nex-z (nexiguran ziclumeran) formerly referred to as NTLA-2001
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20 NANCY Living with ATTR amyloidosis with polyneuropathy Transthyretin Amyloidosis (ATTR): Large and Growing Market with Significant Unmet Need ▪ CM patients have debilitating shortness of breath, arrythmias, reduced mobility and quality of life, as well as a high rate of hospitalization ▪ Wild-type disease, the most common form, occurs with aging, and manifests as heart failure; inherited TTR mutations lead to rapidly progressive heart failure and/or polyneuropathy ▪ 20K incident US CM patients; increasing rates due to an aging population and improved disease awareness ▪ PN presents as motor and sensory dysfunction, muscle wasting, weight loss, as well as autonomic neuropathy with severe GI symptoms Severe, fatal, progressive disease with a shortened life expectancy Despite available treatments, significant unmet need persists ▪ Inconsistent and slow TTR lowering response observed with silencers1 ▪ In phase 3 studies of silencer or stabilizer therapies for CM, the annual rate of CV events or death is high at ~15% of enrolled patients in the first year1,2 ▪ Even on existing therapies, CM patients have a marked decline in quality of life, and functional capacity as measured by 6MWT1,2 ▪ Treatment adherence due to frequent administration/polypharmacy remains an issue Sources: 1. Fontana et al, N. Engl. J. Med (2024), 2. Gillmore et al, N. Engl. J. Med (2024)
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21 ATTR TREATMENT EVOLUTION Intellia is Committed to Developing the Best Treatment for ATTR Amyloidosis DISEASE SLOWING Treatment Burden: Disease Burden: Multiple Injections / Hundreds of Pills Annually with Inconsistent Response Therapies Slow but Do Not Stop or Reverse Progression Ineffective Progressive Disease Treated with Palliative Therapy DISEASE STASIS OR REVERSALGoal: “Even with tafamidis, progression is a question of when, not if.” – US Amyloidosis KOL “Traveling for regular infusions is time away from my family, but I just couldn’t bring myself to do injections at home.” – ATTR Patient “Every year, there is a fight because insurance tries to deny my lifesaving medication.” – ATTR Patient What US HCPs and Patients have to say about current therapies in ATTR1 Life-long Freedom from Chronic Therapy Life-long Reduction in Disease Burden Sources:1. Intellia commissioned HCP and patient quantitative and qualitative market research PALLIATION
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22 Nex-z Phase 1 Results forATTR-CM Show Potential to be Best TTR-Directed Drug Deep, rapid, consistent and durable reductions in serum TTR Stability or improvement of disease markers in a population with advanced disease which is expected to progress rapidly 66% of patients had no worsening in any marker (NT-proBNP,Troponin, 6MWT) at 12 months Encouraging safety and tolerability 1 Updated Dec. 2024: Low rate of hospitalization for cardiac disease, (genotype-weighted) 0.11 events/pt/yr, is favorable based on reference studies3 with higher rates PHASE 1 RESULTS NEX -Z 1 Phase 3 MAGNITUDE (CM) and MAGNITUDE 2 (PN) global studies are actively recruiting patients -100 -90 -80 -70 -60 -50 -40 -30 -20 -10 0 0 2 4 6 8 10 12 Month TTR Percent Change From Baseline* Vutrisiran2 190 180 182 178 160175 Nex-z 36 35 36 36 3636 0% 10% 20% 30% 40% 50% 60% 70% 80% 90% 100% Change in NYHA Class at 12 months *This graphic includes data from distinct clinical trials with their own enrollment criteria and methodologies. Cross -trial comparisons have inherent limitations and shou ld be interpreted with caution Sources: 1. Fontana et al, N. Engl. J. Med (2024), 2. Fontana et al, N. Engl. J. Med (2024), 3. Gillmore et al, N. Engl. J. Med (2024) Abbreviations: 6MWT - 6-minute walk test; NT-proBNP – N-terminal pro-B-type natriuretic peptide; NYHA - New York Heart Association Nex-z Vutrisiran 8% Worsened 44% No Change 47% Improved Mean % Change (95% CI)
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23 Phase 3 Studies in Patients with ATTR-CM and ATTR-PN RN Placebo + SOC* 2:1765 A T T R - CM Primary Endpoint • CV-related mortality and CV-related events Key Secondary Endpoints • TTR and KCCQ-OS score 1:150 A T T R v - PN Primary Endpoints • mNIS+7 and serum TTR Key Secondary Endpoints • Norfolk QOL-DN, mBMI and TTR Nex-z (Single 55 mg IV infusion) + SOC* Nex-z Upcoming 2025 Milestones *SOC = Standard of Care, which often includes tafamidis, may vary across countries within our MAGNITUDE study in ATTR-CM P R O G R A M Dose first patient in pivotal Phase 3 MAGNITUDE-2 trial for PN in 1Q25 Enroll >550 CM patients cumulatively in MAGNITUDE trial Present longer-term data from the Phase 1 study in CM and PN
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24 Positioned to Meet Patient and Provider Needs in a Large and Growing ATTR Market Nex-z represents significant revenue opportunities starting by 2029 High willingness of cardiologists to prescribe nex-z across US and other major developed markets3 Patients want a highly effective therapy and freedom from chronic treatment2 ATTR MARKET OPPORTUNITY “My number one wish would be a cure” U.S. ATTR-CM Patient “This treatment could help me get my life back. I would feel more comfortable going back to work knowing there is a permanent treatment.” UK ATTR-CM Patient “It would be incredible to have a one-time therapy. This would get rid of the mental energy and anxiety I get from going to infusion centers.” U.S. ATTR-PN Patient 1 6.5 9.4 11.8 2024 2026 2028 89% Sources: 1. Evaluate Pharma Consensus Analyst forecasts as of December 2024. 2. Intellia commissioned ATTR patient and caregiver interviews (n=46); 3. Intellia commissioned Physician quantitative survey (n=232) based on nex-z target product profile Global market projected to reach ~12B dollars by 2028 Extremely or Very Likely to Prescribe
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Pipeline
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26 PROGRAM APPROACH Research and Preclinical Early-Stage Clinical Late-Stage Clinical PARTNERS In Vivo: CRISPR is the therapy NTLA-2002: Hereditary Angioedema Knockout Nex-z*: Transthyretin Amyloidosis Knockout Hemophilia A / B*** Insertion Research Programs for Extra-hepatic Targets Various Ex Vivo: CRISPR creates the therapy Research Programs Allogeneic and other Broad Development Pipeline Fueled by Robust Research Engine ** LEAD LEAD ** Lead refers to lead development and commercial party. * Nex-z (nexiguran ziclumeran), formerly referred to as NTLA-2001 ** Intellia is advancing both wholly owned and partnered programs. *** Hemophilia A program is in the research stage; Hemophilia B is being advanced by Regeneron – Intellia is eligible for milestones and royalties.
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27 NTLA-2002 HAE Dose first patient in pivotal Phase 3 HAELO trial for HAE in 1Q25 Complete enrollment in the HAELO study Present longer-term data from the Phase 1/2 study Nex-z ATTR Dose first patient in pivotal Phase 3 MAGNITUDE-2 trial for PN in 1Q25 Enroll >550 CM patients cumulatively in MAGNITUDE trial Present longer-term data from the Phase 1 study in CM and PN Upcoming 2025 Key Milestones for NTLA-2002 and Nex-z Sufficient Cash to Fund Operations through First Commercial Launch (1H 2027)
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28 Intellia is Well-positioned for Near-term Value Creation Focus to successfully launch NTLA-2002 in 2027 Phase 3 programs actively recruiting 3 2 1 Potential blockbusters: NTLA-2002 and nex-z Management team’s prior track record in development and commercialization of best-in-class medicines, including one-time therapies Feedback from market research confirms strong receptivity and willingness to prescribe/use NTLA-2002 and nex-z emerging product profiles Commercial and medical affairs teams’ prior track record of launching multiple blockbusters, including in HAE and heart failure COMPANY OUTLOOK Planning to have all commercial capabilities in place by end of 2026
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29 Developing Best in Class Therapies by Realizing the Promise of Gene Editing At Intellia, we innovate every day to make CRISPR-based medicines a reality for patients. This is just the beginning of a revolution.