1 December 2020 - - US-based biotechnology company Autobahn Therapeutics has successfully completed a pre-Investigational New Drug meeting with the US Food and Drug Administration regarding the development plan for its lead candidate, ABX-002, a potent and selective thyroid hormone receptor beta agonist, the company said.

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 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.

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, a rare genetic disorder. Autobahn Therapeutics is based in San Diego.