Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
NTLA-2001 · 2 trials · 4 indications
| Arm | Type | Description |
|---|---|---|
| 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 |
|---|---|---|
| NTLA-2001 | BIOLOGICAL | NTLA-2001 (55mg) by IV infusion |
| Placebo | DRUG | Normal saline (0.9% NaCl) by IV infusion |
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, ...
NTLA-2001 is an investigational therapy being developed for transthyretin amyloidosis (ATTR) with cardiomyopathy and transthyretin-related (ATTR) familial amyloid polyneuropathy. It is currently in Phase 3 clinical development for these indications.
NTLA-2001 targets TTR, a protein involved in transthyretin amyloidosis. By targeting TTR, the therapy aims to address the underlying protein accumulation associated with the disease.
NTLA-2001 is being developed by Intellia Therapeutics, Inc., a biopharmaceutical company. Intellia's stock is traded under the ticker symbol NTLA on the stock market.
NTLA-2001 is currently in Phase 3 clinical development. A Phase 3 trial, known as MAGNITUDE, is recruiting participants with transthyretin amyloidosis with cardiomyopathy. The therapy is investigational and not yet approved.
NTLA-2001 has been studied in two clinical trials. NCT04601051 was a Phase 1 study completed in patients with hereditary transthyretin amyloidosis with polyneuropathy and cardiomyopathy. NCT06128629 is the Phase 3 MAGNITUDE trial, currently recruiting for transthyretin amyloidosis with cardiomyopathy.
NTLA-2001 is the primary name for this investigational therapy. No alternative names have been reported for this drug in clinical trial registrations.