Intellia Therapeutics, Inc. · NASDAQ:NTLA
Intellia Therapeutics — Technical Presentation (October 2024)
29 slides from Intellia Therapeutics's technical presentation, Oct 24, 2024
Slide 1: Results from Phase 2 Study of NTLA-2002 for Hereditary Angioedema
Results from Phase 2 Study of NTLA-2002 for Hereditary Angioedema October 24, 2024
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: Agenda
Agenda Welcome Introduction Dr. John Leonard Chief Executive Officer, Intellia Therapeutics Review of NTLA-2002 Phase 2 Data Dr. Danny Cohn Internist, Department of Vascular Medicine, Amsterdam University Medical Center NTLA-2002 Clinical Development Update Dr. David Lebwohl Chief Medical Officer, I
Slide 4: Agenda
Agenda Welcome Introduction Dr. John Leonard Chief Executive Officer, Intellia Therapeutics Review of NTLA-2002 Phase 2 Data Dr. Danny Cohn Internist, Department of Vascular Medicine, Amsterdam University Medical Center NTLA-2002 Clinical Development Update Dr. David Lebwohl Chief Medical Officer, I
Slide 5: NTLA-2002 Has the Potential to Transform the HAE Treatment Paradigm
NTLA-2002 Has the Potential to Transform the Hereditary Angioedema Treatment Paradigm Goal with NTLA-2002 = Functional Cure Freedom from attacks 1x = + Freedom from chronic One-time treatment prophylaxis treatment of NTLA-2002 SHANNA Living with hereditary 5 angioedema
Slide 6: Key Learnings from the NTLA-2002 Phase 2 Study
Key Learnings from the NTLA-2002 Phase 2 Study Deep reductions in hereditary angioedema (HAE) attacks at both dose levels tested Safety profile continued to be highly encouraging Single 50 mg dose led to majority of patients (8/11) achieving complete elimination of attacks 50 mg dose led to greater
Slide 7: Agenda
Agenda Welcome Introduction Dr. John Leonard Chief Executive Officer, Intellia Therapeutics Review of NTLA-2002 Phase 2 Data Dr. Danny Cohn Internist, Department of Vascular Medicine, Amsterdam University Medical Center NTLA-2002 Clinical Development Update Dr. David Lebwohl Chief Medical Officer, I
Slide 8: Results From a Phase 2, Randomized, Placebo-Controlled Trial of NTLA-2002
Results From a Phase 2, Randomized, Placebo-Controlled Trial of CRISPR-Based Therapy NTLA-2002 for Hereditary Angioedema Danny M. Cohn,1* Padmalal Gurugama,2 Markus Magerl,3,4 Constance H. Katelaris,5 Adele Golden,6 Mrinal Y. Shah,6 David Maag,6 Hilary J. Longhurst7 1Amsterdam University Medical Cen
Slide 9: Disclosures
Disclosures • Dr. Cohn has acted as a consultant or speaker for or participated in research with Astria, BioCryst, CSL Behring, Intellia Therapeutics, Ionis Pharmaceuticals, KalVista, Pharming, Pharvaris, and Takeda 9
Slide 10: NTLA-2002 Phase 1/2 Trial Design
NTLA-2002 Phase 1/2 Trial Design International, multicenter study to assess safety, tolerability, PK, PD and effect of NTLA-2002 on attacks in adults with Type I or Type II HAE PHASE 1 PHASE 2 Open-Label, Expansion study to Single-Ascending Dose confirm recommended dose 75 mg (n=3) 50 mg (n=11) Rand
Slide 11: Clinical Development Supporting Phase 3 Study Initiation
Clinical Development Supporting Phase 3 Study Initiation PHASE 1 PHASE 2 Trial Dynamics • N = 10 patients • N = 27 patients • Open-label study, no placebo-control • Double-blind study, randomized, placebo-control • No washout of chronic prophylaxis therapy • Required washout of chronic prophylaxis t
Slide 12: Patient Demographics and Characteristics
Patient Demographics and Characteristics NTLA-2002 25 mg NTLA-2002 50 mg Placebo Characteristic (n=10) (n=11) (n=6) Median age (range), years 48.5 (34-62) 44.0 (18-61) 47.0 (31-76) Sex, n (%) Male 7 (70.0) 5 (45.5) 2 (33.3) Median weight (range), kg 89 (58-133) 78 (56-107) 79 (50-92) HAE-C1INH-Type1
Slide 13: NTLA-2002 Continues to Be Well Tolerated Across All Dose Levels
NTLA-2002 Continues to Be Well Tolerated Across All Dose Levels TEAEs in ≥2 Patients After NTLA-2002 NTLA-2002 NTLA-2002 Administration 25 mg 50 mg Placebo (pooled), n (%) (n=10) (n=11) (n=6) Any TEAE 10 (100) 11 (100) 6 (100) • All TEAEs were Grade 1 or 2* Headache 4 (40) 4 (36) 1 (17) Fatigue 3 (3
Slide 14: Dose-Dependent and Durable Reductions in Plasma Kallikrein Protein
A Single Dose of NTLA-2002 Showed Dose-Dependent and Durable Reductions in Plasma Kallikrein Protein Over Time 40 NTLA-2002 25 mg (n=10) Kallikrein Protein Percentage Change from Baseline 30 NTLA-2002 50 mg (n=11) 20 Placebo (n=6) 10 2% 0 -10 (Mean ± SD) -20 -30 -40 -55% -50 -60 -70 -86% -80 -90 -10
Slide 15: Reduction in Monthly Angioedema Attack Rate Compared With Placebo
A Single Dose of NTLA-2002 Led to a Reduction in Monthly Angioedema Attack Rate Compared With Placebo NTLA-2002 25 mg NTLA-2002 50 mg Placebo VARIABLE (n=10) (n=11) (n=6) Median follow-up (range), month 8.2 (4.4-11.8) 5.6 (2.9-11.5) 6.9 (1.9-12.5) Weeks 1-16 Mean no. of angioedema attacks per month
Slide 16: Deep Reductions in Monthly HAE Attack Rate at Both Dose Levels
Deep Reductions in Monthly HAE Attack Rate Compared to Placebo at Both Dose Levels Weeks 1-16 Weeks 5-16 Placebo Placebo 25 mg -75% (-95%, 27%)* 25 mg -80% (-96%, 14%)* 50 mg -77% (-95%, 15%)* 50 mg -81% (-97%, 3%)* *95% CI of the mean attack rate percentage difference relative to placebo. Following
Slide 17: Eight of 11 Patients Completely Attack Free Following a Single 50 mg Dose
Eight of 11 Patients Were Completely Attack Free Following a Single 50 mg Dose The length of the gray bars indicates the interval from the start of the screening period to the last assessment as of the data cutoff date. Arrows indicate the patient is still on study. 17 The width of the colored bars
Slide 18: Eight of 11 Patients Completely Attack Free Following a Single 50 mg Dose
Eight of 11 Patients Were Completely Attack Free Following a Single 50 mg Dose Change from Baseline in Change from Baseline in Administration of Plasma Kallikrein Level Attack rate from NTLA-2002 Attack Severity: Mild Moderate Severe Start of Prophylaxis: at Latest Assessment Pt Weeks 1-16 1 29% -91
Slide 19: Complete Response: Attack-Free and No Subsequent Treatment Required
Eight of 11 Patients Receiving a Single 50 mg Dose Experienced a Complete Response – Attack-Free and No Subsequent Treatment Required 100% % of Patients Attack Free During Primary Observation Period (Weeks 1-16) Patients with Complete Response (%) 50% 73% (8 of 11) 40% (4 of 10) 0% (0 of 6) 0% Place
Slide 20: NTLA-2002 Phase 2 Clinical Data Results Published in NEJM
NTLA-2002 Phase 2 Clinical Data Results Published in NEJM October 24, 2024 20 N Engl J Med 2024; DOI: 10.1056/NEJMoa2405734
Slide 21: Acknowledgements
Acknowledgements This study is funded by Intellia Therapeutics. We wish to extend our gratitude to: • The patients, their caregivers, and their families • Study site coordinators and staff • The staff of Simbec-Orion for assistance with study management and operations support • Medical writing and e
Slide 22: Agenda
Agenda Welcome Introduction Dr. John Leonard Chief Executive Officer, Intellia Therapeutics Review of NTLA-2002 Phase 2 Data Dr. Danny Cohn Internist, Department of Vascular Medicine, Amsterdam University Medical Center NTLA-2002 Clinical Development Update Dr. David Lebwohl Chief Medical Officer, I
Slide 23: Phase 3 Randomized, Double-blind, Placebo-Controlled Study of NTLA-2002 in HAE
A Phase 3, Randomized, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of NTLA-2002 in Patients with Hereditary Angioedema (HAE) WEEK 28 Primary Analysis DAY 1 Data to support WEEK 104 planned BLA PRIMARY LONG-TERM submission OBSERVATION in 2026 OBSERVATION NTLA-2002 (N =
Slide 24: Patients and Physicians Highly Receptive to Target Profile of NTLA-2002
Despite Available Treatments, Patients and Physicians are Highly Receptive to Target Profile of NTLA-2002 Patients want better treatment options… 75% of U.S. prescribers surveyed Treatment has definitely improved but I am still experiencing a high number of attacks. - HAE Patient on lanadelumab, 24
Slide 25: NTLA-2002 is a Potential Paradigm-Shift Compared to Chronic Prophylaxis Therapies
NTLA-2002 is a Potential Paradigm-Shift in Treating HAE Compared to Chronic, Long-Term Prophylaxis Therapies THERAPY F R E Q U E N C Y O F T R E AT M E N T O V E R 1 Y E A R NTLA-2002 1x One-time treatment Once or twice monthly Up to 24 treatments per year Twice weekly Up to 104 treatments per year
Slide 26: NTLA-2002 could be a functional cure for people living with HAE
WHERE WE ARE TODAY…. WHERE WE THINK WE’RE HEADED… HAE patients are seeking improved efficacy and convenience to be free from disease and chronic treatment Phase 2 data supports the potential of NTLA-2002 to eliminate NTLA-2002 could attacks after a one-time treatment be a functional cure for people
Slide 27: Agenda
Agenda Welcome Introduction Dr. John Leonard Chief Executive Officer, Intellia Therapeutics Review of NTLA-2002 Phase 2 Data Dr. Danny Cohn Internist, Department of Vascular Medicine, Amsterdam University Medical Center NTLA-2002 Clinical Development Update Dr. David Lebwohl Chief Medical Officer, I
Slide 28: NTLA-2002 Phase 2 Clinical Data Update
NTLA-2002 Phase 2 Clinical Data Update
Slide 29: Intellia Therapeutics
Inteiia THERAPEUTICS
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