Autobahn Therapeutics Announces Initiation of Phase 2 Trial of ABX-002 as an Adjunctive Treatment for Bipolar Depression

Phase 2 trial designed to provide biological and clinical proof-of-concept for ABX-002 in bipolar depression, utilizing neuroimaging and clinical endpoints; topline data expected in the second half of 2025

ABX-002 also in Phase 2 development for the adjunctive treatment of major depressive disorder, with topline data from the AMPLIFY study also expected in the second half of 2025

Autobahn Therapeutics, a biotechnology company developing restorative treatments for people affected by neuropsychiatric and neuroimmunologic disorders, today announced the initiation of a Phase 2 clinical trial evaluating ABX-002, its highly potent, oral, thyroid hormone beta receptor (TRβ) selective agonist, as an adjunctive treatment in adults with bipolar depression. The Phase 2 trial aims to establish biological and clinical proof-of-concept for ABX-002 in bipolar depression and will inform Autobahn’s further clinical development strategy.

“Bipolar depression is one of the most prevalent and difficult-to-treat psychiatric conditions due to a host of burdensome symptoms,” said Roger McIntyre, MD, Professor of Psychiatry and Pharmacology at University of Toronto. “People living with bipolar disorder often experience prolonged periods of depression, many of whom are not adequately benefiting from, or have had difficulties tolerating, existing treatments. For decades, preclinical and clinical evidence has supported the effectiveness of thyroid hormone as an adjunctive treatment in adults living with bipolar depression, providing the basis for rigorously evaluating an optimized, brain-targeted, potent and selective TRβ agonist which could serve as a highly promising therapeutic option in the future.”

The Phase 2 open-label trial will study ABX-002 as an adjunctive treatment in 30 adult patients with depressive episodes associated with bipolar I and bipolar II disorder and will utilize neuroimaging assessments and validated clinical scales to evaluate changes in metabolites associated with brain energy metabolism and improvements in depressive symptoms. The primary endpoints will be to assess the correlation of change from baseline of nucleoside triphosphate (NTP) and phosphocreatine (PCr) in the brain, as determined by phosphorus magnetic resonance spectroscopy imaging (³¹P-MRS), with percent change in the Hamilton Depression Rating Scale-17 (HAMD-17) over six weeks of dosing, respectively. Key secondary and exploratory endpoints will evaluate changes from baseline in the HAMD-17, HAMD-29, as well as other neuroimaging assessments, including brain activity as measured by resting state functional magnetic resonance imaging (rs-fMRI). Autobahn anticipates reporting topline data from this study in the second half of 2025.

“We are thrilled to initiate this Phase 2 trial of ABX-002, which we believe has the potential to reshape the treatment paradigm for bipolar depression, particularly as an adjunctive therapy for bipolar patients who do not experience adequate relief with their current antidepressant,” said Gudarz Davar, M.D., Executive Vice President, Head of Research and Development for Autobahn Therapeutics. “The clinical evidence generated to date for ABX-002 supports its promise as a centrally-active, differentiated thyromimetic, with a potentially favorable safety profile. This trial initiation expands our clinical depression program, and with our AMPLIFY trial in MDD also underway, sets up 2025 to be a transformational year with two Phase 2 trial readouts, bringing us markedly closer to our goal of addressing the unmet needs of patients living with depression worldwide.”

ABX-002 is also being evaluated in the company’s ongoing AMPLIFY Phase 2 trial as an adjunctive treatment for patients with major depressive disorder (MDD), with topline data expected in the second half of 2025.

About Autobahn Therapeutics

Autobahn Therapeutics is a biotechnology company developing a portfolio of neuropsychiatric and neuroimmunologic 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 (TRβ) agonist being developed as a potential adjunctive treatment for people with major depressive disorder and bipolar disorder depression. Autobahn Therapeutics is based in San Diego. For more information, visit www.autobahntx.com.

About ABX-002

ABX-002 is an orally administered, potent and selective thyroid hormone beta receptor (TRβ) agonist designed to enhance the CNS benefits of thyroid hormone biology while also reducing the peripheral liabilities of synthetic thyroid hormone (e.g., triiodothyronine, T3), a treatment which has shown efficacy in numerous placebo-controlled human studies across MDD and bipolar disorder depression. Thyroid hormone agonism has demonstrated activity on cellular energy metabolism pathways, which play an important role on the regulation of brain bioenergetics and may be uniquely suited to address symptoms of atypical depression, a highly prevalent and underserved sub-population of MDD. In nonclinical and clinical studies, ABX-002 has demonstrated optimized PK properties, target engagement in brain regions associated with depression, and an attractive safety and tolerability profile.

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