Groton ― A local company is about to begin testing a drug therapy designed to protect the brain after it is injured.

Astrocyte Pharmaceuticals, a small drug development company based at BioCT Innovation Commons in the City of Groton, has been working for years to advance a novel treatment to protect the brain after injuries, such as a concussion, traumatic brain injury or stroke, said CEO Bill Korinek.

Korinek said pre-clinical studies of the neuroprotective drug on small and large animals have shown promising results. The company plans to start next year clinical trials with collaborators to test the drug and see if those results translate to humans.

The hope is that the drug, known as AST-004, will someday be a treatment for a wide range of brain injuries, he said.

The company, which last month marked 10 years since its founding, also earned new recognition when Sen. Chris Murphy, D-Conn, named the company “Innovator of the Month.”

“Astrocyte is breaking new ground with their innovative approach to treating strokes and TBIs ― two of the most common causes of death and disability,” Murphy said in a statement. “Their work has the potential to transform the lives of countless patients, and I look forward to seeing their continued success and growth in our state.”

The company is named after the cell the treatment is targeting in the brain: the astrocyte, said Ted Liston, vice president of research.

“We’re helping an important cell in the brain maintain its function and that contributes to saving brain tissue,” Liston said.

“The astrocytes are the caretaker cells in the brain so if you help the caretaker cells, they help the rest of the brain,” added Rusty Poe, head of Chemistry and Manufacturing Controls (CMC).

The 10-year-old company’s origins trace to a conversation Korinek had with James Lechleiter, a professor at the University of Texas-San Antonio, about Lechleiter’s new data and a patent regarding molecules that help protect the brain.

Lechleiter’s molecules were targeting astrocytes, the most common cell in the brain, and their energy metabolism, which represented a novel approach, Korinek said.

He said they knew they needed to pursue the potential treatment to see if it could work in larger animals ― and then if it could work in human brains.

They ended up writing a business plan and forming Astrocyte Pharmaceuticals, he said.

“It’s just very exciting to be tackling this big opportunity, this big unmet need, through some really novel thinking and an innovative approach,” Korinek said.

Korinek, Liston and Poe, who spoke about the research in an interview at BioCT Innovation Commons earlier this month, all had careers at nearby Pfizer, where Korinek was vice president in charge of worldwide research and development business operations, Liston was vice president of research and development, heading up a group of 200 scientists and coordinating global research at multiple sites, and Poe was a pharmaceutical scientist team leader.

Liston said they chose to have their headquarters in Groton because they all live in southeastern Connecticut due to their connections to Pfizer and its central location between New York and Cambridge, Mass. and Boston. Southeastern Connecticut has a strong scientific community, not only because of Pfizer, but also the offshoots of Pfizer, consultants and small companies.

“It’s a very rich environment to do our work, and it’s often overlooked just how extensive the scientific network is in this part of Connecticut,” Liston said.

The company also has an office in Cambridge and works with collaborators across the world, they said. The company has five employees and five half- or full-time consultants.

Liston said they collaborate with investigators around the world, but much of the planning, interpretation of the results, and determining next steps happens here.

Korinek said even though it’s a small company, there’s a high level of rigor, as they have experience from working at Pfizer, and are doing additional testing that isn’t typical of a company at its stage and size.

A military benefit

Korinek said Astrocyte has raised $25 million over the past decade to advance AST-004 from the initial studies on mice through the Phase 1 clinical trials. Most of the funding has been from private investors, while a third has been from grants from groups like the National Institutes of Health or the U.S. Department of Defense.

Korinek said the company has a strong connection with the military and has received several grants from the Department of Defense and the U.S. Army. He said the number-one injury among military personnel is traumatic brain injury, so advancing new treatments is a priority.

The military also places a high priority on being able to treat more injuries on the battlefield and has given the company a $3 million grant, partly to fund the development of oral disintegrating tablets of the treatment. The company currently has prototypes that look fantastic, he said.

Korinek said the idea is that someone suffering a concussion while playing sports or an injury on the battlefield would take a tablet as soon as possible after injury. The drug would help reduce the amount of injury and brain swelling and help the person become symptom-free and recover faster. It could potentially even reduce the long-term consequences, such as the chance of neuro-inflammation or chronic traumatic encephalopathy.

In addition to tablets that could be in doctor’s offices, trainer’s bags or soldiers’ backpacks, the company also has an intravenous formulation that could be used for more severe injuries, he said.

Korinek said a study treating pigs after they had a traumatic brain injury showed a 28% shrinkage in brain swelling. Liston said the pre-clinical studies in non-human primates showed up to a 45% savings of brain tissue.

Liston said millions of people around the world suffer strokes, concussions or traumatic brain injuries, so a potential treatment would be life-changing on a global scale.

Stroke is among the leading causes of death worldwide, according to the World Health Organization.

A revolutionary drug

Drugs typically go through three phases of clinical trials: first, a set of safety trials, second, efficacy studies, and third, typically a larger study to validate that the drug is both safe and effective, Korinek explained.

Liston said the human trials to date show that the drug does get into the human brain at the levels associated with significant efficacy in preclinical models, so that favors a positive outcome in phase 2, though nothing is guaranteed.

The company completed the first phase and conducted two clinical trials in healthy human volunteers, which showed that the drug is safe, well-tolerated and without significant side effects, he said.

The company is now cleared to move into Phase 2 clinical studies and is planning a concussion study in football players, potentially in Australia, and a study in large vessel occlusion stroke patients.

Korinek said if the results are even half as good as they are in animals, it would be very exciting.

“It would be revolutionary,” Liston added.

If the trials show the benefit, the drug could potentially receive a breakthrough designation from the U.S. Food and Drug Administration and potentially the drug could be approved three years from now, Korinek said.

More likely though, the results would mean the company would do another phase 3 clinical study, and if that is successful, could be on track to have a therapy in five to six years.

Korinek said the company anticipates having the data results from phase 2 in 2026. If it’s successful and the treatment does help the human brain heal and reduce stress, there is the potential to expand the treatment. It would offer a lot of promise for companies to explore how the mechanism can be beneficial to a range of conditions.

“The ultimate goal of a pharmaceutical scientist is to have an impact on human health, and it’s very exciting to be involved in an area that’s been largely ignored or given up on by large Pharma,” Liston said. “So we’re taking a risk by going into these areas, but it’s very exciting to be seeing a potential impact.”

k.drelich@theday.com