Autobahn Therapeutics Completes Dosing in its Phase 1 Multiple-ascending Dose Study of ABX-002 for Major Depressive Disorder and Reports Company Progress

SAN DIEGO--(BUSINESS WIRE)-- Autobahn Therapeutics, a biotechnology company leveraging its brain-targeting chemistry platform, validated biology and biomarker-driven strategies to develop restorative treatments for people affected by central nervous system (CNS) disorders, today announced progress with its lead clinical candidate, ABX-002, and early pipeline advancements. In addition, the company further strengthened its senior leadership team with the promotion of Gudarz Davar, M.D., to Executive Vice President, Head of Research and Development. Dr. Davar is an accomplished neurologist and neuroscientist with three decades of corporate and academic leadership in neuroscience research.

ABX-002 Advancing into Phase 2 Development
Autobahn has completed dosing in its double-blind, randomized, placebo-controlled, single- and multiple ascending dose and food effect Phase 1 study designed to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of its lead candidate, ABX-002, in healthy volunteers. ABX-002 is a novel, orally administered, potent, and selective thyroid hormone beta (TRβ) agonist. In both the single-ascending dose (SAD) and multiple-ascending dose (MAD) cohorts, ABX-002 administration has been generally well-tolerated, and the blinded data support a favorable exposure profile suitable for development in neurological diseases. The company plans to assess and report topline data from the Phase 1 study in the fourth quarter of 2023. Based on the Phase 1 experience, Autobahn plans to submit an investigational new drug (IND) application to the US. Food and Drug Administration and advance into a Phase 2 clinical trial in their target patient population of patients with major depressive disorder (MDD) in the first half of 2024. The Phase 2 trial is designed as a double-blind, placebo-controlled study to explore the safety and efficacy of ABX-002 in patients diagnosed with MDD who have had an inadequate response to their antidepressant.

“Millions of people are living with MDD, and for more than half of them, today’s antidepressants do not provide adequate responses. There remains a significant need for a therapy like ABX-002, for which there is clear clinical precedent showing that thyroid hormone agonism can be effective in both boosting and maintaining the response to antidepressant treatment,” said Dr. Davar. “Completion of the Phase 1 SAD/MAD program represents a significant milestone for Autobahn, and we are encouraged to move directly into Phase 2 development to impact patients’ lives as quickly as we can. We believe ABX-002 could offer a significant benefit to patients with this highly prevalent condition, and we look forward to reporting topline results from the Phase 1 trial later this year.”

ABX-101 Progress
Additionally, Autobahn has declared its third development candidate, ABX-101, a next generation, brain-penetrant, oral sphingosine 1-phosphate (S1P) receptor modulator for the treatment of a range of neuroinflammatory disorders. S1P receptor modulators are a well-studied class of drugs that are ideally suited for Autobahn’s FAAH-mediated brain-targeting prodrug platform. In preclinical models of neuroinflammation disease, ABX-101 has demonstrated robust CNS exposure and effectiveness. The company plans to progress ABX-101 through IND-enabling studies in 2024 to support advancement into Phase 1 clinical development.

“This is an incredibly exciting time for Autobahn as we’ve made significant progress toward our mission of bringing restorative treatments to people affected by CNS disorders,” said Kevin Finney, President and Chief Executive Officer at Autobahn. “The expedited advancement of our lead program and the naming of our third development candidate for the treatment of neuroinflammatory disorders is a direct reflection of the dedication and execution by our team. Leading that charge is Dr. Gudarz Davar who, together with the Autobahn team, has advanced our brain-targeting chemistry platform with two key assets, one that is Phase 2 ready and a second that is advancing toward IND-enabling studies. With an expert team and strong science behind us, I am confident in our ability to achieve our near- and mid-term goals.”

About Major Depressive Disorder (MDD)
MDD is the third most common cause of disability worldwide, and leaves patients to suffer from an exasperated state of helplessness, grief and increased suicidality. The cause of MDD is not fully understood but has centered around disruption of the monoaminergic system (e.g., serotonin and norepinephrine activity). Approved drugs that enhance monoaminergic signaling in the brain have shown beneficial effects in MDD, but many patients continue to suffer from an inadequate response to treatment that sustains their depressive symptoms and disability.

About ABX-002
ABX-002 is an orally administered, potent, selective thyroid hormone beta (TRβ) agonist that is brain enhanced, demonstrates target engagement in brain regions associated with depression, and has reduced peripheral liabilities when compared with synthetic thyroid hormone, T3, used at its therapeutic dose. ABX-002, like thyroid hormone, is expected to augment and boost antidepressant treatments by potentiating the beneficial effects of these drugs on monoaminergic signaling in the brain.

About Autobahn Therapeutics
Autobahn Therapeutics is a biotechnology company developing a portfolio of neuropsychiatric and neurodegenerative clinical candidates leveraging its brain-targeting chemistry platform. Autobahn aims to unlock new therapeutic opportunities through precision tuning of CNS exposure, pursuing validated clinical and biologic targets, and guiding development with biomarkers. The company’s pipeline is led by ABX-002, a thyroid hormone receptor beta agonist, being developed as a potential adjunctive treatment for people with MDD, including those who have had an inadequate response to their antidepressant. Autobahn Therapeutics is based in San Diego. For more information, visit www.autobahntx.com.

View source version on businesswire.com: https://www.businesswire.com/news/home/20230911332427/en/