Intellia’s CRISPR Therapy NTLA-2002 Shows 81% Reduction in Hereditary Angioedema Attacks in Phase 2 Study

Efficacy:
Intellia Therapeutics' CRISPR therapy NTLA-2002 reduced hereditary angioedema (HAE) attacks by 81% in a Phase 2 study, specifically in the 50 mg dose group during weeks 5-16.

Dosing:
The therapy involves a one-time infusion, with the 50 mg dose showing the most significant reduction in attacks.

Complete Response:
Eight of 11 patients in the 50 mg arm experienced a complete response, with no attacks during the 16-week observation period and continued to be attack-free through the latest follow-up (median of eight months).

Safety:
NTLA-2002 demonstrated an encouraging safety and tolerability profile, with common adverse events including headache, fatigue, and nasopharyngitis, and no serious adverse events attributed to the treatment.

Future Development:
Intellia is moving forward with the 50 mg dose in the global, pivotal Phase 3 HAELO study, which is actively screening patients.

Market Reaction:
Despite positive results, Intellia's shares were down 9% in premarket trading, possibly due to investor expectations based on Phase 1 data and competitive pressures from other HAE treatments.