Editas Clears Australian Hurdle To Dose First Patients With Cholesterol Gene Editor EDIT-401
An 8-K says the Strive trial can start in Australia; first safety data are targeted for the first quarter of 2027.
Editas Medicine said in an 8-K filed Thursday that it has approval to start the Phase 1/2 Strive trial of EDIT-401, its gene-editing treatment for high cholesterol, in Australia. It is the first human trial for the drug, which so far has been tested only in animals.
What the 8-K says
The trial won sign-off from a Human Research Ethics Committee and finished the Clinical Trial Notification process with Australia's Therapeutic Goods Administration. It enrolls patients with heterozygous familial hypercholesterolemia, an inherited form of high cholesterol.
The application has also been submitted for review in New Zealand. If New Zealand approves, patients in both countries can enroll. Editas has picked five sites across the two countries.
Strive tests a single dose of EDIT-401 for safety, tolerability and efficacy. Part 1 is an open-label, dose-finding study in which the dose is raised step by step. Part 2 will be a randomized expansion study compared against a placebo group.
The press release attached to the filing says Editas remains on track to begin dosing this year. It expects initial safety and tolerability data in the first quarter of 2027, and plans to complete Part 1 enrollment, with topline safety and efficacy results later in 2027.
The primate data
Editas describes EDIT-401 as designed to edit the LDLR gene to raise production of the LDL receptor protein and lower LDL cholesterol. In earlier primate studies, it cut LDL-C, lipoprotein(a) and ApoB by about 90 percent or more on average.
New data from an ongoing primate study show the roughly 90 percent LDL-C reduction held for 10 months. Chief Medical Officer Dan Ory will present them in an oral session at the American Heart Association Scientific Sessions in Chicago on Sunday, November 8, from 3:30 to 4:45 p.m. CT.
Ory called Australian approval "a significant step" in the drug's development. He said the new data "further support its potential to provide durable, lifelong benefits to patients."
What to watch
New Zealand's decision determines whether the trial can enroll in both countries. The next dated event is the November 8 presentation of the durability data.
Editas's first human data, on safety and tolerability, are targeted for the first quarter of 2027.
