Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
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 RNA therapy being developed for transthyretin amyloidosis, including ATTR with cardiomyopathy and ATTR familial amyloid polyneuropathy. It is also being studied in neuromuscular disease associated with ATTR. The drug is designed to reduce production of the transthyretin protein that forms amyloid deposits in these conditions.
Nexiguran ziclumeran targets transthyretin (TTR), the protein that misfolds and accumulates as amyloid in ATTR amyloidosis. By targeting TTR, the therapy aims to lower circulating TTR levels and reduce amyloid deposition in nerves and the heart. It is an RNA-based therapy given as a single intravenous infusion.
Nexiguran ziclumeran is being developed by Intellia Therapeutics, Inc., which trades on the Nasdaq under the ticker NTLA. Intellia is the sponsor of the clinical trials evaluating the drug in transthyretin amyloidosis with cardiomyopathy and in ATTR familial amyloid polyneuropathy.
Nexiguran ziclumeran is in Phase 3 clinical development. Two Phase 3 trials are currently recruiting: one in ATTR with cardiomyopathy and one in ATTRv-PN. A completed Phase 1 study evaluated its safety, tolerability, pharmacokinetics, and pharmacodynamics. The drug has received RMAT and orphan drug designations from the FDA.
Nexiguran ziclumeran is being studied in three clinical trials. The Phase 3 MAGNITUDE trial (NCT06128629) is evaluating it in transthyretin amyloidosis with cardiomyopathy and has an enrollment of 1,200 participants. A separate Phase 3 trial (NCT06672237) is testing it in ATTRv-PN with 60 participants. The completed Phase 1 trial (NCT04601051) enrolled 72 patients.
Yes, Nexiguran ziclumeran is also known as NTLA-2001. NTLA-2001 was the original investigational code used by Intellia Therapeutics during early development, and the drug has since been given the international nonproprietary name nexiguran ziclumeran. Both names refer to the same RNA therapy targeting transthyretin.