Zinger Key Points
- The company announced the single dose treatment led to a 98% mean reduction in monthly HAE attacks.
- NTLA-2002 was well-tolerated across all patients with no severe side effects.
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Intellia Therapeutics, Inc. NTLA stock is moving higher Monday after the company announced long-term data from its Phase 1 Study of NTLA-2002.
The Details: Intellia’s NTLA-2002 is an experimental CRISPR based gene-editing therapy being developed as a one-time treatment for hereditary angioedema (HAE). The company announced the single dose treatment led to a 98% mean reduction in monthly HAE attacks.
In Phase 1 of the study, patients received single doses of NTLA-2002 between 25 mg and 75 mg via intravenous infusion. HAE attack rates and other data was measured at the start of the study and will continue to be measured over the course of a two-year follow-up period.
The study included a total of 10 patients, eight of whom experienced no attacks during the initial 16-week observation period. The treatment was well-tolerated across all patients with no severe side effects.
“These unprecedented data strengthen our view that NTLA-2002 could be a groundbreaking treatment for people living with hereditary angioedema,” said Intellia president and CEO John Leonard, M.D.
“After a single dose of our investigational in vivo CRISPR-based therapy, patients experienced durable elimination of their attacks. We are thrilled to see that the majority of patients have been attack free for over 18 months or longer. These remarkable attack rate reductions have been consistent, even in patients with the most severe symptoms. At the same time, the data from these 10 patients continue to demonstrate a very favorable safety profile. These long-term data provide strong evidence that NTLA-2002 could be a one-time, potential functional cure for this debilitating and life-threatening disease.”
NTLA Price Action: At the time of writing, Intellia Therapeutics shares are up 11.2% and trading at $23.78 per data from Benzinga Pro.
Image: Courtesy of Intellia Therapeutics, Inc.
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