NIH awards $4 million to initiate large clinical trial of stroke drug invented at Scripps Research
Experimental drug 3K3A-APC showed promising effects in earlier clinical trial.
May 24, 2022
LA JOLLA, CA—The National Institute of Neurological Disorders and Stroke (NINDS), part of the National Institutes of Health (NIH), has awarded the first $4 million tranche of funding for a large, “phase three” clinical trial of an experimental stroke drug invented at Scripps Research.
The award, totaling approximately $20 million in direct funds over 6 years, will fund an international, multi-center clinical trial of the experimental treatment 3K3A-APC in about 1,400 patients led by the Keck School of Medicine of the University of Southern California (USC). The trial will focus on patients who experience the most common form of stroke (“ischemic” stroke), which is caused by the blockage of blood flow—usually from a blood clot—in vessels serving the brain. The trial will determine whether 3K3A-APC can hasten recovery and improve outcomes for these patients when added to standard clot-busting treatment.
“I’m very encouraged to see that NINDS is supporting the testing of 3K3A-APC at this scale—ischemic stroke is a major disorder, and any new and effective treatment has the potential to preserve life and otherwise improve outcomes for a very large patient population,” says John Griffin, PhD, a professor in the Department of Molecular Medicine at Scripps Research whose lab invented 3K3A-APC.
Americans suffer ischemic strokes at the rate of roughly 700,000 per year, and although the clot-busting drug tPA has improved outcomes since its introduction in 1996, these vascular events remain a common cause of death and severe disability, particularly among the elderly.
3K3A-APC has demonstrated strong brain-protecting effects in animal models of stroke. In a recent phase 2 clinical trial, 3K3A-APC, in conjunction with tPA or surgical clot removal, showed promising signs of tolerability and improved imaging markers in patients.
3K3A-APC is a modified version of a natural human protein called Activated Protein C (APC). While the latter has powerful brain cell-protecting properties, it also has a strong anticoagulant effect. Griffin and his colleagues slightly modified APC to 3K3A-APC to remove virtually all the anticoagulant effect and reduce the risk of brain bleeding—a risk that is already elevated by the use of tPA.
Griffin collaborated in many groundbreaking studies of 3K3A-APC with Berislav Zlokovic, MD, PhD, director of Zilkha Neurogenetic Institute at the Keck School of Medicine. Zlokovic co-founded ZZ Biotech, the Houston-based company that now sponsors its clinical development. The FDA last summer gave 3K3A-APC development a Fast Track designation, speeding up its application review process.
The new trial, due to begin next year, will be conducted under the supervision of stroke clinician Patrick Lyden, MD, professor of physiology, neuroscience and neurology at the medical school
3K3A-APC is also currently being studied in a phase 2 trial, as a potential treatment for patients with ALS (amyotrophic lateral sclerosis), based on earlier studies suggesting that its brain cell-protecting properties may enable it to slow or stop ALS progression.
For more information, contact press@scripps.edu