MedPath

NTLA-2002

Generic Name
NTLA-2002
Drug Type
Biotech
Background

NTLA-2002 is a lipid nanoparticle encapsulating single guide RNA (G012267) targeting the human KLKB1 gene and messenger RNA (mRNA000042) encoding Cas9. It is an investigational therapy designed to knock out the target gene kallikrein B1 (KLKB1) to reduce plasma kallikrein activity, thereby preventing hereditary angioedema attacks.

Clinical Trials
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Intellia Therapeutics Announces Strategic Reorganization and 2025 Milestones

Intellia Therapeutics, Inc. (NTLA) has announced a strategic reorganization focusing on its late-stage programs, NTLA-2002 and nex-z, targeting hereditary angioedema (HAE) and transthyretin (ATTR) amyloidosis, respectively. The company also disclosed a 27% workforce reduction and an $8 million restructuring charge. Additionally, Intellia's price target was adjusted by BMO Capital and Wells Fargo, reflecting changes in its strategic direction and clinical trial progress.

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NTLA-2002 Gene Editing Shows Promise in Hereditary Angioedema

• A phase II study showed that NTLA-2002, a CRISPR-based gene-editing therapy, significantly reduced angioedema attacks in patients with hereditary angioedema compared to placebo. • Patients receiving NTLA-2002 experienced a 75% to 77% reduction in mean monthly attack rate during the primary observation period (weeks 1-16) compared to placebo. • A significant portion of patients on NTLA-2002 achieved a complete response, remaining attack-free post-dosing, with 73% in the 50mg dose group showing complete response. • NTLA-2002 was well-tolerated, supporting the selection of the 50-mg dose for the ongoing phase III trial in hereditary angioedema patients.

CRISPR Therapy NTLA-2002 Shows Promise in Hereditary Angioedema Treatment

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NTLA-2002 Demonstrates Significant Reduction in Angioedema Attacks via CRISPR-Cas9 Gene Editing

• A single dose of NTLA-2002, an investigational CRISPR-Cas9 gene-editing therapy, significantly reduced angioedema attacks in patients with hereditary angioedema (HAE). • The Phase 2 trial data showed a dose-related reduction in plasma kallikrein levels, with the 50-mg dose reducing kallikrein by 86% from baseline. • A substantial portion of participants, particularly in the 50-mg group (73%), remained attack-free throughout the 16-week observation period without needing additional intervention. • NTLA-2002 was generally well-tolerated, with mostly mild-to-moderate adverse events, supporting its further investigation in a Phase 3 trial.

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Phase 3 Trial of NTLA-2002 for Hereditary Angioedema Now Recruiting

• A Phase 3 clinical trial (NCT06634420) is actively recruiting adult participants with Hereditary Angioedema (HAE) to evaluate the efficacy and safety of NTLA-2002. • The study is a multinational, multicenter, double-blind, placebo-controlled trial randomizing approximately 60 participants in a 2:1 ratio to receive NTLA-2002 or placebo. • Participants will receive a single IV infusion of NTLA-2002 or placebo, followed by a Primary Observation Period (Week 1-28) and a Long-Term Observation Period (76 weeks), totaling 104 weeks. • The trial aims to assess the impact of NTLA-2002 on HAE, with a total study duration of approximately 28 months, including screening and run-in periods.

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NTLA-2002 Shows Promise as Functional Cure for Hereditary Angioedema in Phase 2 Trial

• Phase 2 trial results show NTLA-2002 significantly reduces swelling attacks in hereditary angioedema (HAE) patients, with up to 80% attack reduction at the 50 mg dose. • A substantial portion of patients receiving NTLA-2002 experienced complete attack-free periods, with some remaining attack-free through the latest assessments. • NTLA-2002 was generally well-tolerated, with no serious side effects reported, suggesting a favorable safety profile for this gene-editing therapy. • Intellia Therapeutics has initiated a Phase 3 trial based on these positive results, potentially redefining the treatment paradigm for HAE.

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Intellia's NTLA-2002 Shows Promise in Phase II Trial for Hereditary Angioedema

• Intellia Therapeutics' NTLA-2002, a CRISPR-based therapy, significantly reduced monthly attack rates in hereditary angioedema (HAE) patients compared to placebo. • The 50mg dose of NTLA-2002 led to an 86% mean reduction in kallikrein protein levels at week 16, with eight out of eleven patients experiencing complete response. • NTLA-2002 was well-tolerated, and the 50mg dose has been selected for the pivotal Phase III HAELO study, which is currently screening patients. • The Phase II trial demonstrated the potential of NTLA-2002 as a one-time treatment, offering a functional cure for HAE by inactivating the KLKB1 gene.
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