Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Biological NTLA-2002 · 1 trial · 1 indication
(Phase 1 only)
(Phase 2 only)
| Arm | Type | Description |
|---|---|---|
| Phase 1 Study Arm | EXPERIMENTAL | Participants assigned to 1 of 3 dose-escalation cohorts will receive a single dose of NTLA-2002 on Day 1 and will then be followed for 104 weeks. Primary observation period is 16 weeks. |
| Phase 2 Experimental Study Arm | EXPERIMENTAL | Participants randomized to NTLA-2002 (2 dose levels), will receive a single dose of NTLA-2002 on Day 1 and will then be followed for 104 weeks. Primary observation period is 16 weeks. |
| Phase 2 Placebo Comparator Study Arm | PLACEBO_COMPARATOR | Participants randomized to placebo will receive IV normal saline on Day 1 and will then be followed for up to 104 weeks. Primary observation period is 16 weeks. |
| Placebo Crossover and Follow-On Dosing Substudy Arm | EXPERIMENTAL | Participants assigned to this Substudy Arm (participants who previously received either 25mg or placebo only) will have the opportunity to receive a single dose of NTLA-2002 (50mg) and will then be followed for 52 weeks. |
| Name | Type | Description |
|---|---|---|
| Biological NTLA-2002 | BIOLOGICAL | CRISPR/Cas9 gene editing system delivered by LNP for IV administration |
| Normal Saline IV Administration | OTHER | The administration of IV normal saline |
Inclusion Criteria: 1. Age \>18 years 2. Diagnosis of HAE Types I or II 3. Ability to provide evidence of HAE attacks to meet the screening requirement 4. Subjects must have access to, and the ability to use, ≥ 1 acute medication(s) to treat angioedema attacks. 5. Adequate chemistry and hematology ...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Intellia Therapeutics, Inc. | NTLA | 3 | PHASE3 | NTLA-2002, Normal Saline Administration |
| BioCryst Pharmaceuticals, Inc. | BCRX | 2 | PHASE3 | Berotralstat |
| Ionis Pharmaceuticals, Inc. | IONS | 1 | PHASE3 | Donidalorsen |
| Pharvaris N.V. | PHVS | 1 | PHASE2 | deucrictibant |
| BioMarin Pharmaceutical Inc. | BMRN | 1 | PHASE1 | Dose 1 of BMN 331 |
| Astria Therapeutics, Inc. | ATXS | 1 | PHASE2 | STAR-0215 |
NTLA-2002 is an investigational therapy being developed for the treatment of hereditary angioedema, a genetic condition characterized by recurrent episodes of severe swelling. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities.
NTLA-2002 is a monoclonal antibody designed to target a specific molecular pathway involved in hereditary angioedema. By modulating this target, the therapy aims to reduce the frequency and severity of angioedema attacks. The exact molecular target is not disclosed in the available information.
NTLA-2002 is being developed by Intellia Therapeutics, Inc., a biotechnology company publicly traded under the ticker symbol NTLA. The company is conducting clinical trials to evaluate the safety and efficacy of this investigational therapy for hereditary angioedema.
NTLA-2002 is currently in Phase 1 clinical development. It is an investigational drug, meaning it has not been approved by the FDA or other regulatory agencies. The therapy has received Regenerative Medicine Advanced Therapy (RMAT) and Orphan Drug designations from the FDA.
NTLA-2002 is being studied in a Phase 1 clinical trial with the identifier NCT05120830, titled 'NTLA-2002 in Adults With Hereditary Angioedema (HAE)'. This randomized, double-blind, placebo-controlled trial is actively recruiting participants across multiple countries, including Australia, France, Germany, the Netherlands, New Zealand, and the United Kingdom.
NTLA-2002 is the primary name for this investigational therapy. No alternative names have been reported for this drug in the available information. It is distinct from other therapies and is being developed specifically for hereditary angioedema.