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First-of-its-kind Brain Penetrant Therapy on Track to Enter Clinical Studies in Q4 2021

SAN DIEGO – August 2, 2021 – Autobahn Therapeutics, a biotechnology company focused on restoring hope for people affected by CNS disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for the company’s investigational product candidate, ABX-002, for the treatment of patients with X-linked adrenoleukodystrophy (X-ALD). ABX-002 is a CNS-penetrating prodrug that provides a balanced delivery of LL-340001, a potent and selective thyroid hormone receptor beta (TRβ) agonist, for the potential treatment of the adult-onset form of X-ALD, adrenomyeloneuropathy (AMN), a progressive and debilitating rare disease.

“AMN is a highly debilitating neurodegenerative disease that leads to motor disabilities, fatigue, ataxia, sensory loss, adrenal insufficiency, incontinence and impotence, with no approved treatments today,” said Kevin Finney, chief executive officer of Autobahn. “We are pleased to have received this designation from the FDA for ABX-002, which underscores both the need for treatments for patients with AMN, as well as ABX-002’s potential to positively impact disease progression in this patient population. Based on the preclinical data observed to date, demonstrating potent and on-target activity of ABX-002, we look forward to continuing to advance this novel program and are on-track to initiate clinical development in the fourth quarter of this year.”

AMN is characterized by a toxic buildup of very long chain fatty acids (VLCFAs) in the CNS and periphery, often 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 and is a direct target gene of thyroid hormone. By increasing thyroid hormone signaling, ABCD2 expression is increased, leading to a reduction of VLCFAs. ABX-002 also stimulates remyelination by promoting the differentiation of oligodendrocyte precursor cells, which may be of additional benefit to AMN patients.

Orphan Drug Designation is granted by the FDA to drugs or biologics intended to treat a rare disease or condition, defined as one that affects fewer than 200,000 people in the United States. Programs with Orphan Drug status receive partial tax credit for clinical trial expenditures, waived user filing fees and potential eligibility for seven years of marketing exclusivity post-approval.

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 prodrug 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