Autobahn Therapeutics — a biotechnology company first established out of a lab at Oregon Health & Science University — has generated a $100 million round of private investment to develop new treatments for people affected by neuropsychiatric disorders, such as depression, and eventually for other central nervous system disorders.
The funding raised by San Diego-based Autobahn Therapeutics will be used to advance the company's pipeline of brain-penetrant small molecules, meaning molecules capable of crossing the blood-brain barrier. Their research is led by the OHSU-discovered compound known as ABX-002, a first-in-class brain-targeting thyromimetic agent — that acts by mimicking the effects of thyroid hormones — to treat patients with major depressive disorder and patients with bipolar depression disorder. Together, the disorders affect more than 27 million people in the United States alone.
Tom Scanlan, Ph.D., professor of chemical physiology and biochemistry in the OHSU School of Medicine, worked for decades toward the initial discovery of the compound, and to develop the technology that now underpins the company's ABX-002 program. OHSU licensed the compound to Autobahn in 2018.
"This is huge," Scanlan said. "I've always believed these drugs would be useful in the clinic. I've always believed that very strongly. It's exciting to see us getting closer and closer."
The funding round is especially notable at a time when biotechnology funding worldwide has become much more conservatively managed.
"This investment dramatically validates the research enterprise at OHSU, which is ultimately about creating opportunities for patients to receive better treatment," said Travis Cook, senior director of OHSU Technology Transfer. "It's a testament to the management team at Autobahn Therapeutics and the drug discovery efforts by the Scanlan lab to improve the health and wellbeing of Oregonians and people beyond Oregon."
Winding path of discovery
Scanlan and his OHSU collaborators developed a brain-penetrating version of a molecule originally designed to lower cholesterol. After arriving at OHSU from the University of California, San Francisco in 2006, Scanlan found that he was able to further develop similar molecules that target diseases of the central nervous system.
Next, Scanlan and collaborators found that these new molecules may be useful in repairing the protective sheath around nerve fibers in the central nervous system, known as myelin. Working in a mouse model, they engineered the compound to cross the blood-brain barrier so that it could be useful in treating conditions like multiple sclerosis, and would also have the brain-penetration needed to treat other central nervous system disorders.
In 2022, Autobahn announced research that supported its plans to advance ABX-002, the OHSU-discovered compound, into clinical testing for major depressive disorder. After announcing successful results in 2023, Autobahn now plans to advance ABX-002 in two trials: a Phase 2 clinical trial for patients with major depressive disorder and a Phase 2 trial for patients with bipolar depression.
Scanlan's hope is that the compound is ultimately approved for clinical use to treat different forms of depression, and eventually other central nervous system conditions, as well.
"Depression is a very large and growing clinical need that must be addressed," he said. "I'm confident that this compound and the innovative research at Autobahn will lead to other important clinical uses, as well."
Scanlan continues to work with OHSU scientific investigators looking to advance treatment of central nervous system disorders, starting in animal models. This week's massive infusion of investment capital into Autobahn Therapeutics affirms his confidence.
