Intellia Therapeutics, Inc. (NTLA) has delivered encouraging news for patients suffering from Hereditary Angioedema (HAE) with the release of Phase 2 data from its ongoing Phase 1/2 study of NTLA-2002. This investigational in vivo CRISPR-based gene editing therapy holds the potential to be a revolutionary one-time treatment for HAE, a rare genetic condition causing severe, recurring swelling attacks.
The Phase 2 data, which analyzed results from the first 25 patients who completed the 16-week primary observation period, revealed significant reductions in attack rates. Patients receiving a single dose of NTLA-2002 experienced a mean monthly attack rate reduction of 75% and 77% for the 25 mg and 50 mg arms, respectively, during weeks 1-16. This reduction rose to 80% and 81% during weeks 5-16.
The results are even more promising for patients receiving the 50 mg dose. Eight out of eleven patients in this arm experienced a complete response, with no HAE attacks during the 16-week observation period. These eight patients continued to remain attack-free even after the latest follow-up, with a median of eight months passed, indicating a long-lasting effect. Notably, no subsequent treatment was required for these individuals. In comparison, only four out of ten patients in the 25 mg arm experienced a complete response, while none in the placebo arm did.
Furthermore, patients receiving the 50 mg dose achieved a greater reduction in kallikrein protein, a crucial factor in HAE attacks. This group saw an 86% mean reduction from baseline compared to 55% in the 25 mg arm at week 16.
The safety profile of NTLA-2002 remained positive at both dose levels, demonstrating excellent tolerability.
Based on these impressive results, Intellia has chosen the 50 mg dose for its global, pivotal Phase 3 HAELO study, actively screening patients for participation. This Phase 3 study is expected to conclude by September 2027, marking a significant step towards bringing this potential life-changing treatment to market.
The news builds on the positive data released in June from the Phase 1 portion of the ongoing Phase 1/2 study. In that data, a single dose of NTLA-2002 resulted in a remarkable 98% mean reduction in monthly HAE attack rates with an average follow-up exceeding 20 months across all patients. This study, involving 10 patients, documented a 90% reduction in attacks from weeks 1 to 16, rising to 92% for weeks 5-16.
These encouraging findings have fueled excitement for the potential of NTLA-2002 as a transformative treatment for HAE. However, it’s important to note that this is still an ongoing clinical trial, and further research and regulatory approvals are necessary before NTLA-2002 can become a commercially available therapy. Nevertheless, the recent data provides strong evidence for its potential impact on the lives of HAE patients.