Sectors

The air is crisp, and the sounds of whistles fill the air. The excitement is tangible as football, soccer, and other fall sports go into full swing. Yet behind the scenes of every game is the harsh reality of what happens when an athlete takes a hit—a momentary blackout, disorientation, and the fear of lasting damage. For too long, the best available treatments for concussions and brain injuries have been limited to rest and observation. Astrocyte Pharma, however, is disrupting the playing field with its game-changing neuroprotective drug; they are advancing to treat people with concussions, traumatic brain injuries (TBI), strokes, or neurodegenerative diseases.

"We don't merely treat symptoms. We help protect the brain when it's most vulnerable," share co-founders William Korinek, Ph.D. and Jim Lechleiter, Ph.D. "Brain injuries are a widespread medical issue and rank among the top causes of death and disability globally. Neurons have a limited ability to regenerate and heal, so they are particularly susceptible to damage. We are leading the field of neuroprotection solutions by targeting the very cells that monitor and oversee brain health: astrocytes."

Lechleiter and Korinek bring an impactful blend of expertise and vision to the mission of Astrocyte Pharma. Lechleiter is a prominent Professor of Cell Systems and Anatomy at the University of Texas Health Science Center at San Antonio, where his research focuses on the molecular and cellular mechanisms of protection during brain injury and aging, particularly through enhancing astrocyte mitochondrial ATP production. With extensive experience in imaging technology and as the director of the Optical Imaging facility, he has made seminal contributions to Ca2+ signaling and mitochondrial metabolism. 

Korinek's background is notable as well, including nearly a decade at Pfizer in strategic and operational research and development roles, where he managed Worldwide R&D business operations, drove multiple R&D strategic initiatives, and secured significant funding for enterprise productivity programs. Korinek also brings experience from L.E.K. Consulting and holds a Ph.D. from Harvard University. Together, their combined expertise and passion drive Astrocyte's mission to advance neuroprotection through innovative approaches for enhancing brain health and recovery.

Heal Better, Recover Faster

Unlike previous approaches directly focused on limiting neuronal damage and cell death, Astrocyte Pharma's drug enhances the brain's energy metabolism—providing the cells with the fuel they need to heal faster, restore balance and homeostasis, and mitigate long-term damage. As Korinek explains, "We aim to treat people soon after that concussion or brain injury so they are able to heal better, experience less symptoms, recover faster, and have less long-term complications."

Astrocytes, which support neurons, play a crucial role in keeping the brain functional and resilient. When the brain sustains an injury, these star-shaped cells are the first responders, helping stabilize damaged areas. However, Lechleiter's research revealed that the intrinsic healing mechanisms of astrocytes are short-lived. "The brain has some natural, intrinsic ways that it tries to heal itself from damage, and what we do is actually enhance that signal and prolong those healing processes," Lechleiter explains. 

While competitors are evaluating anti-inflammatory treatments or steroids, Astrocyte's solution steps in and up by stimulating the brain's own defense systems and extends the astrocytes' ability to protect and repair neurons. "With AST-004, we are really helping the caretaker brain cells, the astrocytes, generate more energy and perform better and longer," asserts Lechleiter.

Astrocyte's solution has already made waves in preclinical studies, notably in non-human primates (large animals), where it reduced brain damage by up to 45% after strokes. The implications for human treatment are monumental. Imagine a dissolving tablet available for a soccer player immediately after a head injury, a small intervention that would not only speed up recovery but potentially reduce the long-term risk of conditions like Alzheimer's and chronic traumatic encephalopathy (CTE). "It's such an exciting opportunity," Korinek reflects. "Being able to treat someone with a convenient oral tablet that helps them limit the injury, feel better faster, and reduce their long-term chances of neuroinflammation—it's a chance to change the game."

Astrocyte Pharmaceuticals is navigating a challenging but promising path. While conducting clinical trials in the neuroprotection space has historically been difficult, advances in brain imaging and blood biomarkers are paving the way for more precise and effective studies. By grouping patients with similar injuries and using tools like CT scans and MRIs and similar levels of trauma biomarkers in the blood, it's now possible to have more homogenous trial populations and see the treatment effect of a novel therapeutic. 

With AST-004 set to start Phase 2 clinical trials in 2025, the groundwork is being laid for a significant leap forward in treating brain injuries. Astrocyte's Vice President of Research, Ted Liston, PhD, brings 35 years of drug development experience to the team. Liston highlights that the company's success in preclinical trials in the use of cutting-edge pharmaceutical development techniques, such as the careful characterization of drug concentrations in the brain, reduces the development risk that has long plagued the neuroprotection market. 

"It is kind of rare not only to have a drug that helps protect the brain in small laboratory animals, rodents, but also to demonstrate this protection in large animals with brains much more like humans. We believe this significantly increases the likelihood that what we saw in animal models will translate to human patients. Very few companies have shown this previously, so we're very proud of that research," Liston emphasizes.

Named "Innovator of the Month" in the state of Connecticut by Senator Chris Murphy and as one of the top three publications in 2022 in the scientific journal Stroke, Astrocyte's solution is garnering serious attention for its vast potential beyond brain injury—if it works to help heal human brains after injury, it could work in broader conditions where the brain is stressed or not functioning well. Likewise, Astrocyte's research has shown that chronic treatment with AST-004 substantially reduced beta-amyloid in mouse models of Alzheimer's disease, leading to a grant from the NIH National Institute of Aging and an investment from the Alzheimer's Drug Discovery Foundation. As Korinek notes, "We're engaging a very central mechanism for neuroprotection, and there's a lot of potential to explore its role in multiple areas of brain health."

As cooler weather arrives and Friday night lights glow, Astrocyte Pharmaceuticals' solution becomes more crucial than ever. With over 3.8 million concussions diagnosed annually in the U.S. alone, the need for a new therapy is more urgent than ever. "The brain has natural mechanisms to heal itself from damage, but they're short-lived and can't handle severe injuries. Our drug mimics and extends these intrinsic healing processes to protect the brain after concussions, strokes, and other injuries," notes Korinek. "Our treatment is designed to enhance these recovery pathways, offering a more effective approach to neuroprotection, to transform recovery for millions at risk of traumatic brain injuries, strokes, and neurodegenerative diseases."

Find out more about Astrocyte Pharmaceuticals today.