Intellia Up on Upbeat Long-Term Angioedema Study Data
Intellia Therapeutics‘ (NASDAQ: NTLA) shares gained 10% on Jun 3 after the company reported encouraging long-term data from the phase I portion of its early to mid-stage study evaluating NTLA-2002 for treating hereditary angioedema. Shares of NTLA continued to gain another 1.1% in the after-market hours.
NTLA-2002 is Intellia’s investigational single-dose CRISPR/Cas9 gene-editing therapy, which is being developed as a one-time cure for HAE. It is a rare genetic condition that is marked by severe inflammatory attacks in various body organs and tissues, which can be painful and even life-threatening. Despite the current treatment options that require chronic administration for disease control, HAE attacks still occur.
NTLA-2002 aims to prevent HAE attacks by suppressing the plasma kallikrein activity. Per the management, the long-term data presented at a recent medical conference has bolstered the candidate’s potential as a groundbreaking treatment for HAE as patients experienced durable elimination of their attacks in the ongoing phase I/II study.
Year to date, shares of Intellia have plunged 22.9% compared with the industry’s 6.6% decline.
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A total of 10 patients are being treated in the phase I portion of the study with either of the three one-time doses (25 mg, 50 mg, or 75 mg) of NTLA-2002,administered via intravenous infusion. Eight of these patients experienced no attacks during the initial 16-week observation period. These patients have experienced ongoing attack-free durations of greater than 18 months.
After the last assessment of HAE attacks as of Feb 12, 2024, a 98% mean reduction in monthly attack rate …
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