Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as Biological NTLA-2002
NTLA-2002 · 2 trials · 1 indication
(Phase 1 only)
(Phase 2 only)
| 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) |
| 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 |
|---|---|---|
| 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 |
| Biological NTLA-2002 | BIOLOGICAL | CRISPR/Cas9 gene editing system delivered by LNP for IV administration |
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 |
| 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 (HAE), a genetic condition characterized by recurrent episodes of severe swelling. It is currently being evaluated in a Phase 3 clinical trial for this indication.
NTLA-2002 targets the KLKB1 gene, which encodes plasma kallikrein, a protein involved in the inflammatory pathway that leads to swelling attacks in Hereditary Angioedema. By targeting KLKB1, the therapy aims to reduce the production of kallikrein and thereby prevent angioedema attacks.
NTLA-2002 is being developed by Intellia Therapeutics, Inc., a biopharmaceutical company. Intellia is publicly traded on the Nasdaq stock exchange under the ticker symbol NTLA.
NTLA-2002 is currently in Phase 3 clinical development. It is an investigational therapy and has not yet been approved by regulatory authorities. The drug has received FDA designations including Regenerative Medicine Advanced Therapy (RMAT) and Orphan Drug designation.
NTLA-2002 is being studied in the HAELO trial (NCT06634420), a Phase 3, randomized, double-blind, placebo-controlled study in participants with Hereditary Angioedema. The trial is active but not recruiting, with an enrollment of 80 participants across multiple countries including the United States, Australia, and several European nations.
NTLA-2002 is the primary name for this investigational therapy. No alternative names have been reported for this drug candidate in the available information.