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Also known as NTLA-2001
Nexiguran ziclumeran · 3 trials · 24 indications
| Arm | Type | Description |
|---|---|---|
| nexiguran ziclumeran | EXPERIMENTAL | nexiguran ziclumeran 55 mg by single IV infusion |
| Normal Saline | PLACEBO_COMPARATOR | Placebo; Normal saline (0.9% NaCl) by single IV infusion |
| NTLA-2001 | EXPERIMENTAL | Single intravenous (IV) infusion of NTLA-2001 |
| Placebo | PLACEBO_COMPARATOR | Single IV infusion of normal saline |
| Polyneuropathy Part 1: NTLA-2001 | EXPERIMENTAL | Participants, assigned to one of 4 dose-escalation cohorts, will receive a single dose of NTLA-2001. |
| Polyneuropathy Part 2: NTLA-2001 | EXPERIMENTAL | Participants, assigned to the dose-expansion cohort, will receive a single dose of NTLA-2001. |
| Cardiomyopathy Part 1 (UK only): NTLA-2001 | EXPERIMENTAL | Participants, assigned to one of 2 dose-escalation cohorts, will receive a single dose of NTLA-2001. |
| Cardiomyopathy Part 2 (UK only): NTLA-2001 | EXPERIMENTAL | Participants, assigned to the dose-expansion cohort, will receive a single dose of NTLA-2001. |
| Polyneuropathy Follow-on Dosing (PN Part 1 Dose Level 1 Subjects only): NTLA-2001 | EXPERIMENTAL | Participants assigned to the follow-on dosing cohort will receive a subsequent dose of NTLA-2001. |
| Name | Type | Description |
|---|---|---|
| nexiguran ziclumeran | BIOLOGICAL | nexiguran ziclumeran 55 mg by single IV infusion |
| Normal Saline as Placebo | DRUG | Normal saline (0.9% NaCl) by single IV infusion |
| NTLA-2001 | BIOLOGICAL | NTLA-2001 (55mg) by IV infusion |
| Placebo | DRUG | Normal saline (0.9% NaCl) by IV infusion |
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...
Nexiguran ziclumeran is an investigational therapy being developed for the treatment of hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN), a form of neuromuscular disease. It is currently in Phase 3 clinical development and has not been approved by regulatory authorities.
Nexiguran ziclumeran targets the transthyretin (TTR) protein. By targeting TTR, the therapy is designed to address the underlying cause of hereditary transthyretin amyloidosis with polyneuropathy, a condition in which abnormal TTR protein accumulates and damages nerves.
Nexiguran ziclumeran is being developed by Intellia Therapeutics, Inc., a biotechnology company traded on NASDAQ under the ticker symbol NTLA. The company is conducting a Phase 3 clinical trial of the therapy in patients with hereditary transthyretin amyloidosis with polyneuropathy.
Nexiguran ziclumeran is in Phase 3 clinical development. It is an investigational therapy and has not been approved by the FDA. The drug has received Regenerative Medicine Advanced Therapy (RMAT) designation and Orphan Drug designation from the FDA.
Nexiguran ziclumeran is being studied in a Phase 3 clinical trial registered as NCT06672237. This randomized, double-blind, placebo-controlled study is recruiting 60 participants aged 18 years and older with hereditary transthyretin amyloidosis with polyneuropathy. The trial is being conducted in Argentina, Brazil, Mexico, Singapore, Taiwan, and Thailand.
Yes, nexiguran ziclumeran is also known as NTLA-2001. The Phase 3 clinical trial NCT06672237 is titled 'A Phase 3 Study of NTLA-2001 in ATTRv-PN,' confirming that the drug is being evaluated under this alternative name in clinical research.