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nexiguran ziclumeran

Phase 3

Neuromuscular Disease | Monoclonal antibody | Rare Disease |Intellia Therapeutics, Inc.|Last Updated: Apr 16, 2026

Success Probability

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials1
Total Enrollment60

FDA Designations

RMATORPHAN_DRUG

Clinical trial landscape

nexiguran ziclumeran · 1 trial · 20 indications

Phase 3 1
NCT06672237A Phase 3 Study of NTLA-2001 in ATTRv-PNNeuromuscular Disease
RECRUITING60 Analytics
PHASE3RECRUITING
A Phase 3 Study of NTLA-2001 in ATTRv-PN
Neuromuscular DiseaseUnlock trial analytics

Study Endpoints

Primary Endpoints

Modified Neuropathy Impairment Score +7 (mNIS+7)
18 Months
Serum TTR
29 Days

Secondary Endpoints

Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QoL-DN) Questionnaire
18 Months
Modified Body Mass Index (mBMI)
18 Months
Serum TTR
18 Months
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
nexiguran ziclumeranEXPERIMENTALnexiguran ziclumeran 55 mg by single IV infusion
Normal SalinePLACEBO_COMPARATORPlacebo; Normal saline (0.9% NaCl) by single IV infusion

Interventions

NameTypeDescription
nexiguran ziclumeranBIOLOGICALnexiguran ziclumeran 55 mg by single IV infusion
Normal Saline as PlaceboDRUGNormal saline (0.9% NaCl) by single IV infusion
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Eligibility Criteria

Age Range18 Years to 85 Years
SexALL
Healthy VolunteersNo
Study Sites14

Inclusion Criteria: * Diagnosis of ATTRv-PN * Karnofsky Performance Status (KPS) ≥ 60 Exclusion Criteria: * Other causes of amyloidosis (amyloidosis caused by non-TTR protein) * Other known causes of sensorimotor or autonomic neuropathy * Diabetes mellitus * New York Heart Association Class III o...

Countries:ArgentinaBrazilMexicoSingaporeTaiwanThailand
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Frequently asked questions about nexiguran ziclumeran

What is nexiguran ziclumeran used for?

Nexiguran ziclumeran is an investigational therapy being developed for the treatment of neuromuscular disease, specifically hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN). It is currently in Phase 3 clinical development and has received FDA designations including Regenerative Medicine Advanced Therapy (RMAT) and Orphan Drug designation.

What does nexiguran ziclumeran target?

Nexiguran ziclumeran is a monoclonal antibody being developed by Intellia Therapeutics. While the specific molecular target is not disclosed in the available information, the drug is being studied in a Phase 3 trial for hereditary transthyretin amyloidosis with polyneuropathy, a condition caused by the accumulation of abnormal transthyretin protein.

Who makes nexiguran ziclumeran?

Nexiguran ziclumeran is being developed by Intellia Therapeutics, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol NTLA. The company is conducting a Phase 3 clinical trial of the drug for the treatment of hereditary transthyretin amyloidosis with polyneuropathy.

What phase is nexiguran ziclumeran in?

Nexiguran ziclumeran is currently in Phase 3 clinical development. It is an investigational drug and has not been approved by the FDA. The drug has received Regenerative Medicine Advanced Therapy (RMAT) and Orphan Drug designations from the FDA for the treatment of hereditary transthyretin amyloidosis with polyneuropathy.

What clinical trials is nexiguran ziclumeran in?

Nexiguran ziclumeran is being evaluated in a Phase 3 clinical trial registered as NCT06672237, titled 'A Phase 3 Study of NTLA-2001 in ATTRv-PN'. This randomized, double-blind, placebo-controlled study is recruiting 60 participants aged 18 years and older across Argentina, Brazil, Mexico, Singapore, Taiwan, and Thailand.

Is nexiguran ziclumeran the same as NTLA-2001?

Yes, nexiguran ziclumeran is also known as NTLA-2001. The Phase 3 clinical trial NCT06672237, which is evaluating the drug for hereditary transthyretin amyloidosis with polyneuropathy, is titled 'A Phase 3 Study of NTLA-2001 in ATTRv-PN'.