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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 |
| 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 | BMN 331 |
| Astria Therapeutics, Inc. | ATXS | 1 | PHASE2 | STAR-0215 |
NTLA-2002 is an investigational therapy being developed for hereditary angioedema (HAE), a rare genetic disorder characterized by recurrent episodes of severe swelling. It is not yet approved for any indication and remains in clinical development.
NTLA-2002 targets KLKB1, the gene that encodes plasma prekallikrein. By targeting KLKB1, the therapy aims to reduce the production of kallikrein, a key enzyme in the contact activation pathway that drives bradykinin generation and angioedema attacks in hereditary angioedema.
NTLA-2002 is being developed by Intellia Therapeutics, Inc., which trades on the Nasdaq under the ticker NTLA. Intellia is a clinical-stage biotechnology company focused on genome editing therapies.
NTLA-2002 is in late-stage clinical development, with a Phase 3 trial ongoing and an NDA/BLA-stage designation. It has not been approved by the FDA. The program has received RMAT and Orphan Drug designations from the FDA for hereditary angioedema.
NTLA-2002 is being evaluated in two clinical trials: the Phase 3 HAELO study (NCT06634420) in participants with hereditary angioedema, and a Phase 1 study (NCT05120830) in adults with HAE. Both trials are active but not recruiting, with a combined enrollment of 117 participants.
Yes, Biological NTLA-2002 is an alternative name for NTLA-2002. Both refer to the same investigational therapy developed by Intellia Therapeutics for hereditary angioedema.