Scripps Research Stroke Drug Shows Effectiveness, Safety

A stroke drug invented at The Scripps Research Institute has shown signs of safety and efficacy in a mid-stage clinical trial. If it succeeds, it would not only help stroke patients, but bring more money to TSRI’s top-rated biomedical research.

The drug is designed to improve the usefulness of tPA, a popular stroke drug. Called 3K3A-APC, the drug helps protect brain cells from stroke damage and the side effects of tPA. It allows tPA to be used later than the usual 4-5-hour “window” after a stroke caused by blood clots occurs.

TSRI licensed the drug to Houston’s ZZ Biotech, which is conducting the clinical trials and recently announced the interim Phase 2 results. If the drug reaches the market, TSRI stands to benefit financially.

While the institute has an endowment of about $300 million, it has run deficits in recent years, so bringing in more money is critical to the institute’s long-term financial stability and scientific excellence. TSRI was ranked first worldwide in the quality and impact of its science, according to a 2017 report by the top-tier scientific journal Nature.

The stroke drug was invented by a team led by TSRI professor John Griffin. The scientists modified an existing blood protein called protein C, which inhibits coagulation and protects cells.

Griffin’s team collaborated with the lab of Berislav Zlokovic, MD, director of the Zilkha Neurogenetic Institute at the University of Southern California. Together, they showed the drug’s benefits in preclinical research, which led to launching clinical trials.

Zlokovic is the scientific founder of ZZ Biotech, which licensed the drug.

Stroke is the fifth leading cause of death in the United States, according to the Centers for Disease Control and Prevention. It’s also a major cause of disability, heightening the need for improved therapies. Most strokes are ischemic, caused by blood clots. The drug tPA dissolves the clots, restoring blood flow to the brain.

The National Institutes of Health funded the Phase 2 study. It provided a grant to Patrick Lyden, MD, chair of the Department of Neurology at Cedars-Sinai in Los Angeles. In addition, the NeuroNEXT Infrastructure Resource Access provided a grant to ZZ Biotech. The study was co-led by Lyden and ZZ Biotech’s Kent Pryor.

The placebo-controlled study’s aim was to find the maximum dose patients could tolerate, and secondarily, to evaluate cerebral hemorrhage incidence and volume. It found both were substantially reduced in the treated patients.

The next step is to confirm the results in further clinical trials. Success could lead to approval to sell the drug.

Accelerating the pace of commercializing such drugs has become a top priority under Peter G. Schultz, CEO of The Scripps Research Institute. Calibr, a biomedical institute Schultz helped found, has become closely allied with Scripps Research toward that end.

By more quickly translating basic research to clinical development, Scripps Research hopes to get new revenue streams that can be used to protect and expand the institute’s commitment to biomedical scientific research and education.