Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT06634420 | HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE) | PHASE3 | ACTIVE NOT_RECRUITING | 80 | — | — | Jan 15, 2025 | Sep 1, 2027 | May 15, 2026 | 29 | United States, Australia +7 |
| Arm | Type | Description |
|---|---|---|
| Arm A: NTLA-2002 | ACTIVE_COMPARATOR | Arm A: NTLA-2002 (50 mg; single IV infusion) |
| Arm B: Placebo | PLACEBO_COMPARATOR | Arm B: Placebo (saline; single IV infusion) |
| Name | Type | Description |
|---|---|---|
| NTLA-2002 | BIOLOGICAL | CRISPR/Cas9 gene editing system delivered by lipid nanoparticle (LNP) for intravenous (IV) administration |
| Normal Saline IV Administration | BIOLOGICAL | The administration of intravenous (IV) normal saline |
Inclusion Criteria: 1. Age ≥16 years 2. Clinical history consistent with HAE-C1INH-Type 1 or -Type 2 3. Ability to provide evidence of HAE attacks (confirmed by the Investigator) to meet the screening requirement 4. Must agree to refrain from the use of long-term prophylactic therapies from the sta...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Intellia Therapeutics, Inc. | NTLA | 3 | PHASE3 | NTLA-2002, Normal Saline Administration, Biological NTLA-2002 |
| BioCryst Pharmaceuticals, Inc. | BCRX | 2 | PHASE3 | Berotralstat, berotralstat |
| Ionis Pharmaceuticals, Inc. | IONS | 1 | PHASE3 | Donidalorsen |
| KalVista Pharmaceuticals, Inc. | KALV | 1 | PHASE3 | KVD900, Drug: KVD900 |
| 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 |