Phase II Intellia data fail to impress investors

25 October 2024

Intellia Therapeutics (Nasdaq: NTLA) has announced Phase II results for its CRISPR-based therapy, NTLA-2002, aimed at providing a one-time treatment for hereditary angioedema (HAE).

This would mark an important shift from chronic HAE treatments which require frequent administration. The market is currently led by BioCryst’s (Nasdaq: BCRX) Orladeyo (berotralstat) and Takeda’s (TYO: 4502) Takhzyro (lanadelumab). Orladeyo is taken orally once daily, while Takhzyro is administered via injection every two to four weeks​.

Intellia’s data show sustained reductions in attack rates, building on earlier Phase I data that demonstrated a 98% reduction in monthly HAE attacks, with most participants remaining attack-free for 18 months or longer.

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