Intellia Therapeutics on Wednesday said it has received a green light from the Food and Drug Administration to start a Phase 3 trial of a gene editing treatment it’s developing for the rare disease transthyretin amyloidosis.
According to Intellia, the trial will be the first late-stage test in the U.S. of a CRISPR-based therapy that’s works in vivo, or by editing genes inside the human body rather than in cells extracted from patients. Intellia expects to begin the study by the end of the year.
Dubbed NTLA-2001, Intellia’s therapy is designed to be a one-time fix for transthyretin amyloidosis. Using CRISPR/Cas9, it knocks out a gene responsible for producing proteins that, in people with the condition, are damaged and build up in toxic accumulations in the heart and nerves.
Intellia developed NTLA-2001 together with Regeneron, with which it’s worked since 2016. The companies recently expanded their collaboration to develop in vivo medicines for neurological and muscular diseases.
A few medicines are approved in the U.S. to treat nerve damage caused by transthyretin amyloidosis. One, from Pfizer, is cleared for the cardiomyopathy form of the disease, which is the target of Intellia’s Phase 3 study. (Intellia is also planning another study in the polyneuropathy disease type.)
The go-ahead for Intellia to start its Phase 3 trial signals increasing confidence from the FDA in CRISPR gene editing treatments, analysts said.
“Today's [Investigational New Drug application] clearance signals that [the] FDA is comfortable with the potential risks of off-target editing related to in vivo gene editing therapies and provides further regulatory derisking for [Intellia’s] platform,” wrote Kostas Biliouris, an analyst at BMO Capital Markets.
Emerging clinical data may have also bolstered Intellia’s case, Biliouris said.
Other in vivo gene editing treatments have been cleared for U.S. trials, including ones from Sangamo Biosciences, Excision BioTherapeutics and another from Intellia. NTLA-2001 is the furthest along in testing, however.
“Successful execution here affirms [Intellia] as the front-runner among companies … working to commercialize in vivo editing,” Mani Foroohar, an analyst at Leerink Partners, wrote in a note to clients.
The green light from the FDA comes as Vertex Pharmaceuticals and CRISPR Therapeutics await the FDA’s verdict on approval of an ex vivo treatment for sickle cell disease. If approved by the agency’s Dec. 8 target, it would be the first CRISPR-based medicine to arrive on market in the U.S.
Shares in Intellia dropped by about 4% in Wednesday morning trading.