Intellia Therapeutics, a US-based biotech firm, has announced positive Phase 3 results for its gene therapy lonvo-z in patients with hereditary angioedema (HAE). The treatment has shown significant efficacy in reducing symptoms of the condition, which affects approximately 1 in 50,000 people in the UK.
The Phase 3 trial, known as the 'NOMAD' study, enrolled 75 patients with HAE who received either a single dose of lonvo-z or a placebo. The results showed that patients who received the treatment experienced a significant reduction in HAE symptoms, including swelling, pain, and difficulty breathing.
According to Dr John Leonard, President and Chief Executive Officer of Intellia Therapeutics, 'These Phase 3 results demonstrate the potential of lonvo-z to transform the lives of patients with HAE.' The company plans to submit a Biologics License Application (BLA) to the US FDA in the second half of 2024.
While the results are promising, further clinical trials are planned to confirm the long-term efficacy and safety of lonvo-z. This includes a Phase 3 trial in patients with HAE who have previously received treatment with other therapies.
The gene therapy works by targeting the responsible gene for HAE and reducing the production of the faulty protein that causes symptoms. The treatment has shown minimal side effects and a high response rate in clinical trials.