Liothyronine Sodium

Company to initiate Phase 2 trial of ABX-002 for bipolar depression by yearend 2024 ABX-002 also being evaluated as an adjunctive treatment for major depressive disorder in the ongoing AMPLIFY Phase 2 trial SAN DIEGO--(BUSINESS WIRE)--Autobahn Therapeutics, a biotechnology company developing restorative treatments for people affected by neuropsychiatric and neuroimmunologic disorders, today announced the clearance of an investigational new drug (IND) application by the U.S. Food and Drug Administration (FDA) to support initiation of a Phase 2 trial of ABX-002 as an adjunctive treatment for patients with bipolar depression. ABX-002 is a highly potent, oral, thyroid hormone beta receptor (TRβ) selective agonist designed to enhance the CNS benefits of thyroid hormone biology and drug concentrations directly in the brain. “We are thrilled to expand our depression treatment program with ABX-002 and evaluate its potential to safely address the debilitating symptoms of bipolar depression, a highly prevalent and potentially life-threatening disorder,” said Kevin Finney, Autobahn’s President and CEO. “In the U.S. alone, seven million patients suffer from bipolar disorder and there are limited safe and effective therapies that focus specifically on the depressive symptoms that patients face. Our planned Phase 2 trial is designed to provide proof-of-concept for ABX-002 and inform further clinical development in bipolar depression. With this regulatory clearance, we are excited to advance ABX-002 into a second clinical development program later this year alongside our recently initiated Phase 2 study in major depressive disorder.” The Phase 2 trial will evaluate ABX-002 as adjunctive therapy in adult patients with depressive episodes associated with bipolar I or bipolar II disorder. Autobahn will evaluate the biological and clinical proof-of-concept for ABX-002 across multiple endpoints in the trial, including changes in energy metabolism in the brain as determined by phosphorus magnetic resonance spectroscopy ( 31 P-MRS) and changes in patients’ depressive symptoms as measured by the Hamilton Depression Rating Scale (HAMD). 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 . 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 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. Contacts Investors: Alex Straus THRUST Strategic Communications alex@thrustsc.com Media: Ryan Flinn In Like Flinn Communications ryan@inlikeflinncommunications.com

AMPLIFY 230-patient trial aims to improve on efficacy and safety established with thyroid hormone as an adjunctive treatment for MDD; topline data expected in the second half of 2025 ABX-002 deemed safe and well-tolerated in Phase 1 and showed clinical evidence of enhanced target engagement consistent with brain-activating thyroid hormone biology Company on-track to expand ABX-002 depression program with Phase 2 trial for bipolar disorder depression to begin late 2024 SAN DIEGO, CA, USA I September 16, 2024 I Autobahn Therapeutics, a biotechnology company developing restorative treatments for people affected by neuropsychiatric and neuroimmunologic disorders, today announced initiation of AMPLIFY, a Phase 2 clinical trial evaluating ABX-002 as an adjunctive treatment for adults with major depressive disorder (MDD). ABX-002 is a highly potent, oral, thyroid hormone beta receptor (TRβ) selective agonist designed to enhance the CNS benefits of thyroid hormone biology and drug concentrations directly in the brain, for greater potency, efficacy and safety. In a completed Phase 1 trial of ABX-002 , ABX-002 demonstrated clinical evidence of CNS target engagement consistent with brain-activating thyroid effects and was well-tolerated, with no serious adverse events observed. “Among the millions of people in the U.S. suffering from the debilitating effects of depression, more than half experience an inadequate response to their current antidepressant, underscoring the urgent need for treatment options that provide relief for patients,” said Dan Iosifescu, M.D., Professor of Psychiatry at NYU Grossman School of Medicine. “Synthetic thyroid hormone has long been known to provide benefit as an adjunctive treatment for MDD, and a highly potent and selective TRβ agonist may offer the therapeutic benefits of synthetic thyroid hormone, with a safety profile that enables chronic use for optimal patient management.” AMPLIFY is a placebo-controlled, double-blind Phase 2 trial in 230 adults with moderate-to-severe depression. The trial will evaluate the safety, tolerability, efficacy and pharmacodynamic (PD) effects of ABX-002 as an adjunctive treatment to patients’ current selective serotonin reuptake inhibitor (SSRI) or serotonin-noradrenaline reuptake inhibitor (SNRI). The primary endpoint is the change from baseline on the Hamilton Depression Rating Scale-17 (HAMD–17) after six weeks of treatment. Key secondary endpoints include changes from baseline on the HAMD-29 to assess improvements on atypical depression symptoms, the Montgomery–Åsberg Depression Rating Scale (MADRS), and other measures of clinical improvement. “We are thrilled to initiate this Phase 2 trial of ABX-002, marking a substantially evolved profile for Autobahn as a mid-stage development company progressing multiple clinical development candidates,” said Gudarz Davar, M.D., Executive Vice President, Head of Research and Development for Autobahn. “The clinical data we’ve generated with ABX-002 support its promise as a brain-penetrating, centrally active thyromimetic with favorable safety, and give us greater confidence in its potential as a game-changing treatment for depression, especially for patients who remain underserved by today’s treatment options. Our team is dedicated to advancing innovative therapies that have the potential to transform lives, and this milestone with ABX-002 is a significant step toward achieving our vision.” Autobahn anticipates reporting topline data from the AMPLIFY Phase 2 trial of ABX-002 in patients with MDD in the second half of 2025. In addition, by yearend 2024, Autobahn plans to initiate a second Phase 2 trial with ABX-002 in patients with bipolar disorder depression, a chronic and difficult mental health condition. 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. SOURCE: Autobahn Therapeutics