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SAN DIEGO – Mar. 3, 2022 – Autobahn Therapeutics, a biotechnology company focused on restoring hope for people affected by CNS disorders, today announced that the company will present preclinical data highlighting the potential of its lead small molecule product candidate, ABX-002, as a treatment for neurodegenerative diseases at the American Academy of Neurology (AAN) Annual meeting. ABX-002 is a potent and selective, CNS-directed thyroid hormone receptor beta (TRb) agonist that the company is developing as a treatment for a range of CNS disorders, including treatment-resistant depression, multiple sclerosis and adrenomyeloneuropathy (AMN), the adult form of adrenoleukodystrophy (ALD). In addition, the company will present findings from research conducted around the AMN patient journey. The findings will be detailed during poster sessions at AAN, being held in Seattle from April 2-7, 2022.

“We have specifically designed ABX-002 with the unique combination of potency, specificity and CNS penetration that supports its potential to address a range of neurodegenerative disorders,” said Brian Stearns, Ph.D., chief scientific officer of Autobahn. “Part of our strategy is a commitment to developing reliable and predictive biomarkers that allow us to optimize and de-risk clinical development for our programs. These new preclinical data demonstrate on-target effects with ABX-002, evidenced by its ability to impact specific biomarkers, which gives us even greater confidence in its therapeutic potential and supports our plans for clinical development, beginning with assessment as a treatment for treatment-resistant depression, where there is a significant need for effective options.”

In preclinical models of ALD and multiple sclerosis, Autobahn evaluated several potential disease biomarkers of CNS-delivered thyromimetics, including

  • Disease-causing brain and plasma very long chain fatty acids (VLCFA), mRNA and microRNA expression profiles in ALD, and
  • Synthesis of 24-S-hydroxycholesterol (24-OHC, the brain metabolite of cholesterol and an indirect measure of myelin turnover) and mRNA expression profiles in MS.

The findings showed that administration of ABX-002:

  • Decreased brain and plasma VLCFA in an ALD animal model
  • Increased 24-OHC synthesis in brain and plasma, suggesting an increased rate of myelin synthesis in an MS model, and
  • Increased expression of known T3-responsive genes and identified novel T3-responsive microRNAs that respond to thyromimetic treatment in both disease models and normal animals, suggesting that microRNAs could be utilized as target engagement biomarkers.


Presentation Details:

Title: Thyromimetics Improve Disease Endpoints and Modulate Potential Target Engagement Biomarkers in Rodent Models of ALD and MS

Session: P3: MS Immunology and Basic Science 1

Date & Time: Saturday, April 2, 2022, from 5:30 PM – 6:30p.m. PT

Title: Understanding the Adrenomyeloneuropathy (AMN) Patient Journey

Session: P12: Neuromuscular Disease: Peripheral Neuropathy 4

Date & Time: Tuesday, April 5, 2022, from 5:30 – 6:30p.m. PT


About Autobahn Therapeutics

Autobahn Therapeutics is focused on restoring hope for people affected by CNS disorders. Autobahn is leveraging its brain-targeting chemistry platform to unlock new therapeutic opportunities through precision tuning of the central exposure of its molecules. The company’s pipeline is led by ABX-002, a thyroid hormone receptor beta agonist, being developed for the treatment of several CNS diseases, including treatment resistant depression, multiple sclerosis and adrenomyeloneuropathy (AMN), a rare genetic disorder. Autobahn Therapeutics is based in San Diego. For more information, visit


Alicia Davis, THRUST Strategic Communications