Zinger Key Points
- Intellia selected 50 mg for evaluation in the global, pivotal Phase 3 HAELO study, and is actively screening patients.
- Four of the ten patients in the 25 mg arm experienced a complete response, and zero patients in the placebo arm.
- Get New Picks of the Market's Top Stocks
On Thursday, Intellia Therapeutics, Inc. NTLA released Phase 2 data from the ongoing Phase 1/2 study of NTLA-2002 in patients with hereditary angioedema (HAE).
NTLA-2002 is an investigational in vivo CRISPR-based gene editing therapy in development as a one-time treatment for HAE, a rare genetic condition that results in recurrent attacks of severe swelling.
The data cut-off date for the analysis was April 4, when the 25th patient completed the 16-week primary observation period.
A single dose of NTLA-2002 showed mean monthly attack rates relative to placebo were reduced by 75% and 77% for the 25 mg and 50 mg arms during weeks 1–16 and by 80% and 81% during weeks 5–16, respectively.
In the 50 mg arm, eight of 11 patients experienced a complete response after a single dose of NTLA-2002, with no attacks at all during the 16-week primary observation period; these eight patients continued to be attack-free through the latest follow-up (median of eight months), and no subsequent treatment has been required.
Four of the ten patients in the 25 mg arm experienced a complete response, and zero patients in the placebo arm.
Patients who received the 50 mg dose achieved a greater kallikrein protein reduction, with an 86% mean reduction from baseline compared to 55% in the 25 mg arm at week 16.
At both dose levels, NTLA-2002 was well tolerated.
Based on these results, Intellia selected 50 mg for evaluation in the global, pivotal Phase 3 HAELO study, actively screening patients.
The Phase 3 study is expected to conclude by September 2027.
When in June, Intellia Therapeutics released data from the Phase 1 portion of the ongoing Phase 1/2 study, a single dose of NTLA-2002 led to a 98% mean reduction in monthly HAE attack rate, with an average follow-up of over 20 months across all patients.
The trial, which administered a single dose of NTLA-2002 to 10 patients, tracked a 90% reduction in attacks from weeks 1 to 16, which increased to 92% for weeks 5-16.
Price Action: NTLA stock is down 18.3% at $16.28 at last check Thursday.
Read Next:
Image created using Dall-E.
© 2024 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.
Trade confidently with insights and alerts from analyst ratings, free reports and breaking news that affects the stocks you care about.
