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Company Plans to Initiate Clinical Development for ABX-002 for the Treatment of Adrenomyeloneuropathy in 2021

SAN DIEGO – Dec. 1, 2020 – Autobahn Therapeutics, a biotechnology company focused on restoring hope for people affected by CNS disorders, today announced it has successfully completed a pre-Investigational New Drug (IND) meeting with the U.S. Food and Drug Administration (FDA) regarding the development plan for its lead candidate, ABX-002, a potent and selective thyroid hormone receptor beta agonist. Autobahn has gained alignment with FDA on the initial Phase 1a/b clinical trial protocol for ABX-002 as a monotherapy treatment for a rare neurological disorder, adrenomyeloneuropathy (AMN).

AMN is a progressive and debilitating form of X-linked adrenoleukodystrophy (X-ALD) with no effective treatment options. AMN is characterized by a buildup of toxic very long chain fatty acids (VLCFAs) in the central nervous system and periphery, accompanied by demyelination. This buildup is driven by mutations in the ABCD1 gene which encodes a transporter that shuttles cytosolic VLCFAs into peroxisomes for degradation. ABX-002 acts by increasing expression of ABCD2, a compensatory transporter which functionally compliments defective ABCD1. ABX-002 also stimulates remyelination by promoting the differentiation of oligodendrocyte precursor cells, which may be of additional benefit to AMN patients.

As part of the meeting, FDA aligned with Autobahn’s dose selection strategy and patient inclusion criteria for the Phase 1a/b trial, as well as the company’s proposal to conduct a single Phase 2b/3 adaptive trial to support a potential future registration of ABX-002. In addition, Autobahn may apply for Fast Track Designation and Orphan Drug Designation for the broader indication of X-ALD, potentially expanding ABX-002’s reach to a larger group of patients.

“We are very pleased to have gained alignment with FDA on our clinical strategy for ABX-002 for AMN, which we believe demonstrates the Agency’s view of the unmet needs for patients with this devastating disease,” said Kevin Finney, chairman and chief executive officer of Autobahn. “ABX-002 uses Autobahn’s proprietary brain targeting chemistry platform to achieve balanced distribution in the central nervous system and the periphery, and has demonstrated the ability to induce ABCD2 expression in preclinical models, which could be therapeutically relevant in patients with AMN. Based on our recent regulatory interaction, we have multiple opportunities for accelerated development timelines and are working diligently to finalize our protocol design to initiate our first-in-human study of ABX-002 in 2021.”

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 by tuning the central exposure of its molecules. The company’s pipeline is led by ABX-002, a thyroid hormone receptor beta agonist for the treatment of adrenomyeloneuropathy (AMN), a rare genetic disorder. Autobahn Therapeutics is based in San Diego. For more information, visit


Alicia Davis, THRUST Strategic Communications