ATX-101 (Autobahn Therapeutics)

ATX-101 is an investigational, brain-penetrant, oral small molecule prodrug developed by Autobahn Therapeutics for the treatment of bipolar depression. It utilizes a proprietary brain-targeting chemistry platform where the prodrug is selectively cleaved by the enzyme fatty acid amide hydrolase (FAAH)—which is highly expressed in the central nervous system—to release a potent thyroid hormone receptor beta (TRβ) agonist specifically within the brain. By targeting TRβ in the CNS, ATX-101 aims to modulate mood-regulating pathways and potentially promote remyelination while minimizing peripheral side effects, such as cardiac or bone toxicity, typically associated with systemic thyroid hormone exposure. The drug is currently being evaluated in the Phase 2 AMPLIFY-BD clinical trial for patients with Bipolar I or Bipolar II disorder experiencing a major depressive episode.