Autobahn Therapeutics announced today that it will present clinical results from its completed Phase 1 trial of ABX-002 at the upcoming 2025 American Society of Clinical Psychopharmacology (ASCP) Annual Meeting in Scottsdale, Arizona. The presentation will highlight findings that support the advancement of ABX-002 into Phase 2 development as an adjunctive treatment for major depressive disorder (MDD) and bipolar disorder depression.
ABX-002 is a highly potent, oral, thyroid hormone beta receptor (TRβ) selective agonist specifically designed to enhance the central nervous system benefits of thyroid hormone biology for patients with affective disorders. The drug represents a novel approach to addressing the significant unmet needs in depression treatment.
Phase 1 Trial Results Show Favorable Safety Profile
The completed Phase 1 trial evaluated ABX-002 in healthy volunteers through a double-blind, randomized, placebo-controlled, single and multiple ascending dose study. Results demonstrated that ABX-002 was safe and well tolerated, with no serious adverse events observed throughout the trial.
"We are excited to share the Phase 1 findings at ASCP, which demonstrated a favorable safety and tolerability profile and enhanced CNS target engagement with ABX-002, and support its evaluation in the ongoing Phase 2 trials in major depressive disorder and bipolar depression," said Gudarz Davar, M.D., Executive Vice President, Head of Research and Development for Autobahn.
The study also revealed dose-proportional pharmacokinetics and clinical evidence of CNS target engagement consistent with brain-activating thyroid effects. These findings have helped inform dose selection for the company's ongoing Phase 2 clinical trials.
Addressing Significant Unmet Needs in Depression Treatment
Despite the availability of numerous therapies for depression, many patients continue to struggle with achieving adequate symptom relief. According to Dr. Davar, "Despite availability of existing therapies, many individuals with MDD and bipolar depression struggle to achieve adequate relief. Our team remains deeply committed to developing novel, CNS-targeted treatments that we believe have the potential to meaningfully improve the lives of those affected by these debilitating conditions."
Major depressive disorder affects approximately 280 million people worldwide, with bipolar disorder impacting over 45 million individuals globally. Both conditions can significantly impair quality of life and functioning, highlighting the need for more effective treatment options.
Mechanism of Action and Potential Advantages
ABX-002 works by selectively targeting the thyroid hormone beta receptor, which plays a crucial role in brain function. This selective approach is designed to enhance the CNS benefits of thyroid hormone biology while reducing the peripheral liabilities associated with synthetic thyroid hormone treatments like triiodothyronine (T3).
Thyroid hormone agonism has demonstrated activity on cellular energy metabolism pathways, which are important for the regulation of brain bioenergetics. This mechanism may be particularly well-suited to address symptoms of atypical depression, a highly prevalent and underserved sub-population of MDD patients.
In both nonclinical and clinical studies, ABX-002 has demonstrated optimized pharmacokinetic properties, target engagement in brain regions associated with depression, and an attractive safety and tolerability profile.
Ongoing Clinical Development
Autobahn Therapeutics is currently evaluating ABX-002 in two separate Phase 2 clinical trials:
- The AMPLIFY Phase 2 trial, which is assessing ABX-002 as a potential adjunctive treatment for people with major depressive disorder
- A separate Phase 2 trial evaluating ABX-002 as a potential adjunctive treatment for bipolar depression
The poster presentation at the ASCP Annual Meeting will be delivered by Bridgette Franey, M.D., Senior Medical Director and Global Medical Lead for Clinical Development at Autobahn Therapeutics. The presentation is scheduled for Thursday, May 29, 2025, from 11:30 a.m. to 1:15 p.m. MT at the Fairmont Scottsdale Princess in Scottsdale, Arizona.
About Autobahn Therapeutics
Autobahn Therapeutics is a San Diego-based biotechnology company developing a portfolio of neuropsychiatric and neuroimmunologic clinical candidates. The company leverages its brain-targeting chemistry platform 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, which is being developed as a potential adjunctive treatment for people with major depressive disorder and bipolar disorder depression. Autobahn aims to address significant unmet needs in neuropsychiatric medicine through its innovative approach to drug development.
