Intellia gains after data for gene editing candidate in hereditary angioedema
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Intellia Therapeutics (NASDAQ:NTLA) added ~5% pre-market Monday after announcing new data from its Phase 1/2 trial for its gene editing candidate NTLA-2002 in hereditary angioedema (HAE), a rare genetic disorder characterized by inflammatory attacks.
Citing a data cut on Feb. 17 based on a 10-patient sample in the Phase 1 portion of the trial, Intellia (NTLA) said that a single dose of NTLA-2002 led to a 95% mean reduction in monthly HAE attack rates across all trial subjects.
“While early, these unprecedented interim data from the Phase 1 study continue to reinforce our belief that NTLA-2002 could be a potential functional cure for people with hereditary angioedema,” CEO John Leonard remarked.
Other highlights of the readout include about one year or longer attack-free durations seen in those who first received the therapy, therefore with the longest follow-up.
Six of the ten patients undergoing long-term HAE prophylaxis before receiving NTLA-2002 remained free of HAE attacks even after they discontinued the prophylactic therapy.
In terms of safety, the CRISPR candidate was well tolerated with no dose-limiting toxicities across all three doses investigated, Intellia (NTLA) said.
The Cambridge, Massachusetts-based biotech has started dosing patients for the Phase 2 portion of the trial, and the company expects to complete its enrollment in H2 2023.
Related: Makers of HAE prophylaxis therapies include Takeda (TAK) and BioCryst Pharmaceuticals (BCRX)