Recent Updates
Recently added Catalysts

NTLA-2001

Phase 3

Transthyretin Amyloidosis (ATTR) With Cardiomyopathy | Monoclonal antibody | Cardiovascular |Intellia Therapeutics, Inc.|Last Updated: Mar 27, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
Premium
Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
Premium
Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment1,200
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06128629MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)PHASE3 RECRUITING 1,200Dec 13, 2023Apr 1, 2028Mar 27, 2026132 United States, Argentina +24
Unlock Drug Trial Details
Study Endpoints
Primary Endpoints
Composite outcome of cardiovascular (CV) mortality and CV events
Maximum study duration is dependent on event rates and is estimated to be at least 18 months and up to approximately 5 years
Secondary Endpoints
Change in baseline to month 18 in serum TTR
Baseline, Month 18
Change from baseline to month 18 in KCCQ-OS score
Baseline, Month 18
Unlock Study Endpoints
Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
NTLA-2001EXPERIMENTALSingle intravenous (IV) infusion of NTLA-2001
PlaceboPLACEBO_COMPARATORSingle IV infusion of normal saline
Interventions
NameTypeDescription
NTLA-2001BIOLOGICALNTLA-2001 (55mg) by IV infusion
PlaceboDRUGNormal saline (0.9% NaCl) by IV infusion
Unlock Study Design Details
Eligibility Criteria
Age Range18 Years — 90 Years
SexALL
Healthy VolunteersNo
Study Sites132

Inclusion Criteria: * Documented diagnosis of ATTR amyloidosis with cardiomyopathy * Medical history of heart failure (HF) * Symptoms of HF are optimally managed and clinically stable within 28 days prior to administration of study intervention * Screening NT-proBNP, a blood marker of HF severity, ...

Countries:United StatesArgentinaAustraliaAustriaBelgiumBrazilCanadaCzechiaDenmarkFranceGermanyHungaryIsraelItalyJapanMexicoNetherlandsNew ZealandNorwayPortugalSingaporeSouth KoreaSpainSwedenTaiwanUnited Kingdom
Unlock Eligibility Criteria
Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT06128629Status: ACTIVE_NOT_RECRUITING → RECRUITING
LOWMay 24, 2026NCT06128629studyFirstPostDate: changed