Groton biotech firm working on much-needed drug to treat stroke, brain injuries
Groton — A small drug development company that moved its headquarters from Cambridge, Mass., to the BioCT Innovations Commons on Shennecossett Road last year is aiming to have a huge impact on the treatment of strokes and brain injuries.
Co-founded by William Korinek of Stonington, who spent nearly a decade at Pfizer, Astrocyte Pharmaceuticals expects to launch a phase one clinical trial of its leading drug candidate in the first half of 2022. Last week, Astrocyte put out word that Stroke, a peer-reviewed journal, had published the results of preclinical studies of the drug, known for now as AST-004.
“Its potential is enormous,” said Korinek, the company’s chief executive officer.
Given the statistics, it’s easy to understand why.
According to the U.S. Centers for Disease Control and Prevention, nearly 800,000 people a year suffer strokes in the United States, about one every 40 seconds. Every four minutes, someone dies of a stroke. In addition, some 1.7 million people sustain a traumatic brain injury, about 75% of which are concussions.
The lack of available treatments for stroke is a major unmet medical need, one that could qualify Astrocyte’s drug development for fast-tracking if the results of upcoming human trials are sufficiently promising, according to Ted Liston, Astrocyte’s vice president of research.
More than 80% of stroke patients receive no significant treatment, either with enzymes that must be administered within hours of a stroke or with surgery to remove blood clots, said Liston, a Stonington resident who spent nearly 25 years at Pfizer. Instead, he said, many patients are getting “palliative care — bedrest, physical therapy,” and often end up in nursing homes and rehabilitation centers.
Astrocyte Pharmaceuticals, whose other co-founder and current adviser, James Lechleiter, began researching the role of “neuroprotectors” at the University of Texas Health Science Center at San Antonio more than a decade ago, has focused its efforts on astrocytes — so-called “caretaker” cells that help the brain's neurons, or nerve cells, recover from injury.
“When you get a concussion, they tell you to rest, the brain will heal itself,” Korinek said. “Our drug helps the caretaker cells do their job better. ... One analogy is that the neuron is like a toddler, the astrocyte is the parent or the babysitter taking care of the neuron.”
Liston said Astrocyte is one of only a few companies working on stroke treatments to target astrocytes instead of neurons.
In preclinical studies, Astrocyte has tested the drug on rodents and more recently monkeys. In the Stroke-published study, researchers sought to reproduce in "non-human primates" what typically happens when human stroke patients are treated in the emergency room. The results published in the article show “highly significant improvements across a number of metrics, with benefits beyond the current standard of care — reperfusion by thrombectomy surgery,” which means restoring blood flow to brain tissue through removal of a blood clot.
“Administration of AST-004 resulted in rapid and statistically significant decreases in lesion growth rate and total lesion volume,” Astrocyte reported.
“Neurologists are desperate to save brain tissue after a stroke,” Liston said. “If you can increase the amount you’re saving by 5%, it would be remarkable. We demonstrated we’re saving up to 45%, a very striking effect of our drug. And it happens as soon as you administer it.”
Astrocyte envisions two forms of its drug, one of which could be administered intravenously to patients in an emergency room or in an ambulance following a stroke or an accident, Korinek said. In tablet form, the drug could help injured soldiers and football players equipped to take it right away.
Signaling its interest, the U.S. Department of Defense recently provided Astrocyte with $3.5 million in grants to support the company’s research.
Korinek said it could take several years to complete clinical trials of the drug, a process that would include testing on healthy individuals and then on those with the conditions the drug is designed to treat. If the drug progresses through the trials, the U.S. Food and Drug Administration would have to approve it before it could be placed on the market.
The FDA could accelerate approval by deeming AST-004 a breakthrough drug.