The Food and Drug Administration has cleared Intellia Therapeutics to start human testing in the U.S. of an experimental CRISPR medicine for a rare swelling disorder, a sign the agency could be getting more comfortable with medicines that edit genes inside the body.
Intellia on Thursday said the FDA will allow it to begin dosing U.S. patients with a drug called NTLA-2002 that the biotechnology company is developing for hereditary angioedema. Intellia has been studying the medicine in an early-stage study that it has so far conducted in the Netherlands, U.K., and New Zealand. With the FDA clearance, Intellia can enroll U.S. patients in a Phase 2 portion of the trial.
The decision is notable, as it represents one of only a few times the FDA has cleared testing in the U.S. of a treatment that edits genes “in vivo,” or inside the body.
Others, such as Sangamo Biosciences and, more recently, startup Excision BioTherapeutics, have reached that point as well. But the FDA has recently appeared cautious, delaying plans by biotechs Verve Therapeutics and Beam Therapeutics to test in vivo gene editing therapies for heart disease and cancer, respectively. The agency held up Beam’s application for four months. Verve’s has been suspended since November. (In an earnings announcement Thursday, Verve said it’s addressing the FDA’s requests and will submit a response “as expeditiously as possible.”)
Those situations have fueled uncertainty about what the FDA wants from drugmakers before they’re allowed to test inside-the-body gene editing therapies in humans, wrote Mani Foroohar, an analyst at SVB Securities. That the agency cleared Intellia’s request is a “a big win for gene editing more broadly” and “particularly systemic in vivo programs,” wrote Stifel analyst Dae Gon Ha.
The decision “opens the door, however marginal, for subsequent programs seeking FDA green light,” Ha added in a note to clients.
Among those programs are another Intellia prospect for the rare genetic disorder transthyretin amyloidosis, which is in clinical testing overseas. Intellia said last week it expects to submit a U.S. application by the middle of the year and begin a late-stage study by the end of 2023. That request will be different, as Intellia is aiming to jump straight into pivotal testing after having conducted early tests in France, Sweden, New Zealand and the U.K.
Baird analyst Jack Allen wrote in a client note that the “positive sentiment” surrounding the FDA’s view of NTLA-2002 should “read through positively” to Intellia’s other request. Intellia shares climbed 10% in early trading Thursday.
NTLA-2002 is designed to be a one-time fix for a condition that currently requires chronic therapy. The drug inactivates a gene encoding for kallikrein, a protein involved in blood clotting and other processes, and is meant to reduce the rates of the painful and potentially life-threatening swelling attacks associated with the disease.
Intellia disclosed early results last year, and expects to release additional data in 2023. It recently began enrolling patients outside the U.S. in the study’s Phase 2 portion.