Autobahn Therapeutics 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 therapy ABX-002, a thyroid hormone receptor beta agonist for the treatment of adrenomyeloneuropathy (AMN).
Following the FDA’s alignment on the therapy’s initial trial protocols, the company plans to initiate clinical development of ABX-002 in 2021.
“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,” Kevin Finney, chairman and CEO of Autobahn, said in a press release.
AMN is the adult-onset form of adrenoleukodystrophy, an inherited condition caused by mutations in the ABCD1 gene.
A selective thyroid hormone receptor beta agonist, ABX-002 works by increasing the expression of ABCD2, which may partially compensate for the ABCD1 gene mutations.
It also can bind to thyroid hormone receptors in the brain, mimicking the action of the thyroid hormone, which stimulates the production of mature oligodendrocytes — the cells that produce myelin. Myelin is the protective coat that insulates nerve cells and that gets damaged in AMN.
“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,” Finney said.
Autobahn now is aligned with the FDA on the initial Phase 1a/b clinical trial protocol to evaluate ABX-002 as a monotherapy for AMN, including its strategy for dose selection and the criteria for patient inclusion.
The FDA also aligned with the company’s proposal to conduct a single Phase 2b/3 adaptive trial to support a potential future registration of ABX-002.
“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,” Finney said.
In addition, Autobahn Therapeutics may apply for fast track and orphan drug designations for the broader indication of adrenoleukodystrophy, which could potentially expand ABX-002’s reach to a larger patient community.
