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Biological NTLA-2002

Phase 1

Hereditary Angioedema | Monoclonal antibody | Other |Intellia Therapeutics, Inc.|Last Updated: Mar 24, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials1
Total Enrollment37
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05120830NTLA-2002 in Adults With Hereditary Angioedema (HAE)PHASE1 ACTIVE NOT_RECRUITING 37 — — Dec 10, 2021Jul 1, 2026Mar 24, 20269 Australia, France +4
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Study Endpoints
Primary Endpoints
Safety and tolerability of NTLA-2002 as determined by adverse events (AEs) and dose limiting toxicities (DLTs)
From NTLA-2002 infusion up to week 104 post-infusion

(Phase 1 only)

Number of HAE attacks per month (Weeks 1-16)
From study drug infusion up to week 16 post-infusion

(Phase 2 only)

Secondary Endpoints
Change from baseline in total plasma kallikrein protein level
From NTLA-2002 infusion up to week 104 post-infusion
Plasma and urine concentrations for DMG-PEG2k, LP000001, Cas9 mRNA, and sgRNA
From NTLA-2002 infusion up to week 104 post-infusion
Safety and tolerability of NTLA-2002 as determined by AEs
From study drug infusion up to week 104 post-infusion
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Study Design & Arms
AllocationRANDOMIZED
MaskingDOUBLE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Phase 1 Study ArmEXPERIMENTALParticipants assigned to 1 of 3 dose-escalation cohorts will receive a single dose of NTLA-2002 on Day 1 and will then be followed for 104 weeks. Primary observation period is 16 weeks.
Phase 2 Experimental Study ArmEXPERIMENTALParticipants randomized to NTLA-2002 (2 dose levels), will receive a single dose of NTLA-2002 on Day 1 and will then be followed for 104 weeks. Primary observation period is 16 weeks.
Phase 2 Placebo Comparator Study ArmPLACEBO_COMPARATORParticipants randomized to placebo will receive IV normal saline on Day 1 and will then be followed for up to 104 weeks. Primary observation period is 16 weeks.
Placebo Crossover and Follow-On Dosing Substudy ArmEXPERIMENTALParticipants assigned to this Substudy Arm (participants who previously received either 25mg or placebo only) will have the opportunity to receive a single dose of NTLA-2002 (50mg) and will then be followed for 52 weeks.
Interventions
NameTypeDescription
Biological NTLA-2002BIOLOGICALCRISPR/Cas9 gene editing system delivered by LNP for IV administration
Normal Saline IV AdministrationOTHERThe administration of IV normal saline
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites9

Inclusion Criteria: 1. Age \>18 years 2. Diagnosis of HAE Types I or II 3. Ability to provide evidence of HAE attacks to meet the screening requirement 4. Subjects must have access to, and the ability to use, ≥ 1 acute medication(s) to treat angioedema attacks. 5. Adequate chemistry and hematology ...

Countries:AustraliaFranceGermanyNetherlandsNew ZealandUnited Kingdom
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Competitive Landscape -Hereditary Angioedema 10 trials
CompanyTickerTrialsLead PhaseDrugs
Intellia Therapeutics, Inc.NTLA3PHASE3NTLA-2002, Normal Saline Administration, Biological NTLA-2002
BioCryst Pharmaceuticals, Inc.BCRX2PHASE3Berotralstat, berotralstat
Ionis Pharmaceuticals, Inc.IONS1PHASE3Donidalorsen
KalVista Pharmaceuticals, Inc.KALV1PHASE3KVD900, Drug: KVD900
Pharvaris N.V.PHVS1PHASE2deucrictibant
BioMarin Pharmaceutical Inc.BMRN1PHASE1Dose 1 of BMN 331
Astria Therapeutics, Inc.ATXS1PHASE2STAR-0215
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Recent Changes (Last 90 Days)
MEDIUMMay 26, 2026NCT05120830primaryCompletionDate: changed
LOWMay 24, 2026NCT05120830studyFirstPostDate: changed