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Our Science

Harnessing the regenerative power of the human body

Through a deep understanding of human biology, we are developing a pipeline of transformative small molecule therapies for the treatment of CNS disorders

  • Our Approach

  • Brain Targeting Chemistry Platform

  • Pipeline

  • AMN Program

  • MS Program

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Our Approach

Our unique approach to CNS drug development combines three distinct elements:

  • Brain-Targeting Chemistry Platform

    Our brain-targeting chemistry platform creates orally administered small molecule prodrugs with tunable ratios of central vs. peripheral exposure. This approach enables us to develop molecules with bespoke distribution profiles tailored to match disease pathophysiology.

  • Validated Human Biology

    Our portfolio is anchored by high-confidence programs that leverage established human genetics and biology, de-risking our pipeline and accelerating early development.

  • Biomarker-Driven Development

    Our programs utilize biomarker-driven development strategies to demonstrate on-target / on-tissue activity and proof-of-mechanism early in the clinic.

Collectively, these elements enable us to de-risk and accelerate the development of our transformative small molecule therapies.

Brain Targeting Chemistry Platform

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The blood-brain barrier is composed of tightly linked endothelial cells that limit the passage of pathogens and specific types of small and large molecules from the blood into the brain. This critical protective function also restricts the diffusion of therapeutics into the brain representing a major challenge to the development of new medicines for CNS diseases.

By leveraging the highly specialized enzymatic activity and brain-focused tissue distribution profile of fatty-acid amide hydrolase (FAAH), our prodrug technology enables us to precisely tailor CNS and peripheral exposure of the active molecule to match disease pathophysiology. This precision tuning provides Autobahn with a range of platform opportunities.

Our Pipeline

We have a pipeline of transformative small molecule therapies that are in development for the treatment of both rare and prevalent CNS disorders. Our lead molecule, ABX-002, is a novel thyroid hormone receptor beta (TRβ) agonist in development to treat adrenomyeloneuropathy (AMN), a rare genetic disorder.  ABX-003 is our next generation TRβ agonist in development to promote remyelination in multiple sclerosis (MS).

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ABX-002 for AMN

Adrenomyeloneuropathy (AMN) is the adult form of X-linked adrenoleukodystrophy (X-ALD), a progressive and debilitating rare disease 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 is a potent and selective TRβ agonist with balanced central and peripheral distribution.  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.

About AMN

ALD Connect
  • Prevalence

    Rare genetic disorder

  • Patient Impact

    Debilitating motor disabilities, fatigue, ataxia, sensory loss, adrenal insufficiency, incontinence and impotence

  • Current Treatment

    No approved treatments available today

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Science graphic

ABX-003 for Multiple Sclerosis

Multiple sclerosis (MS) is a progressive autoimmune disease characterized by immune attack on myelin, the protective covering on neurons. Damage to myelin disrupts the effective transmission of nerve signals and can lead to permanent damage to neurons.

ABX-003 is a potent and selective TRβ agonist with enhanced central versus peripheral distribution.  Human genetics unambiguously links thyroid hormone signaling to myelination, and the ability of thyroid hormone to promote remyelination is well established.  ABX-003 promotes the differentiation of oligodendrocyte precursor cells (OPCs) into mature oligodendrocytes. These cells regenerate myelin to protect demyelinated neurons and restore neural conduction velocity to halt and potentially reverse the progressive course of MS.  We are developing ABX-003 for use in combination with standard-of-care immunosuppressive therapies.

  • Prevalence

    ~ 1 million people affected in the U.S.

  • Patient Impact

    Debilitating motor disabilities, fatigue, loss of vision and cognitive decline

  • Current Treatment

    Immunosuppressants reduce the frequency of relapse but none have shown the ability to halt or reverse disease

About Autobahn

About Us