FDA Clears Intellia To Start US Tests Of ‘in Vivo Gene Editing Drug
The Food and Drug Administration has cleared Intellia Therapeutics to begin human trials in the United States of an experimental CRISPR drug to treat a rare tumor, suggesting the agency may be used to drugs that alter genes in the body.
Intelia announced Thursday that the FDA has approved the drug NTLA-2002, which the biotech company is developing to treat hereditary angioedema, for US patients. Intellia has tested the drug in preliminary studies so far in the Netherlands, the UK and New Zealand. With FDA approval, Intellia can enroll US patients in a Phase 2 study.
The decision is noteworthy because it marks one of the few times the FDA has allowed a gene-altering treatment to be tested "in vivo," or inside the body, in the United States.
Other companies, such as Sangamo Biosciences and, more recently, startup Excision Biotherapeutics, have also come here. But the FDA has been cautious lately, with biotech companies Verve Therapeutics and Beam Therapeutics slow to test gene-editing techniques for heart disease and cancer, respectively. The agency delayed Beam's nomination for four months. Verve has been banned since November. (In Thursday's earnings call, Verve said it is reviewing the FDA's requests and will respond "as soon as possible.")
These conditions have increased uncertainty about what the FDA expects from drugmakers before testing gene-editing therapies in the human body, wrote SVB Securities analyst Mani Foruhar. Complying with Intellia's request for the agency is "a big win for gene editing in general" and "especially for Vivo Systems' programs," analyst Stifel de Gong Ha wrote.
The decision "opens the door to programs seeking a green light from the FDA, albeit a small one," Ha added in a note to clients.
Another of these programs is Intellia, a treatment for a rare genetic disease, transthyretin amyloidosis, which is undergoing clinical trials abroad. Intellia announced last week that it plans to apply to the U.S. and begin studies as late as late 2023. This app will be different, as Intellia aims to go straight to testing after initial trials in France, Sweden. New Zealand and the United Kingdom.
Baird analyst Jack Allen wrote in a note to clients that the "positive opinion" on NTLA-2002, along with the FDA's opinion on Intel's other request, "must be interpreted accordingly." Intellia shares rose 10% in early Thursday trading.
NTLA-2002 is currently intended as a one-time solution for a condition requiring chronic treatment. The drug activates the gene that codes for kallikrein, which is involved in blood clotting and other processes, and aims to reduce the painful and life-threatening inflammatory events associated with the disease.
Intellia released preliminary results last year and plans to release more data in 2023. A second phase of the study recently began enrolling patients outside the United States.
