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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

  • Our Science

  • Brain Delivery

  • Pipeline

  • MS Program

  • AMN Program

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Our Approach

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

  • Validated Human Biology

    Our scientific foundation and therapeutic programs leverage established human biology, de-risking our programs and accelerating early development. Our lead program, ABX-002, leverages the unambiguous link between thyroid hormone and myelin biology.

  • Brain-Targeting Chemistry Platform

    Our brain-targeting chemistry platform creates prodrugs that can be administered orally, are relatively inactive in the periphery, demonstrate enhanced brain penetrance and are selectively liberated to their active form in the CNS. This approach maximizes brain activity and minimizes undesired peripheral effects.

  • Biomarker-Driven Development

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

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

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Our Science

Science graphic

We are restoring hope for people affected by CNS disorders by harnessing the regenerative power of the human body. Validated human biology unambiguously links thyroid hormone to the production of myelin, the protective covering on neurons. Our lead program, ABX-002, engages thyroid hormone receptors selectively in the brain to restore myelin to demyelinated axons to halt and potentially reverse the progressive course of multiple sclerosis and other demyelinating diseases.

The ABX-002 program leverages our brain-targeting chemistry platform to maximize on-target exposure in the CNS and minimize peripheral exposure and the potential for unwanted side effects. We are actively pursuing a similar chemical strategy with other target classes where a dramatic shift in relative brain exposure can deliver future regenerative medicines for CNS disorders.

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 maximizes the on-target / on-tissue effects of the active molecule in the brain while minimizing systemic activity and the potential for unwanted side effects.

Photo chart of a brain

Our Pipeline

Our pipeline of transformative small molecule therapies 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 agonist in development for multiple sclerosis and adrenomyeloneuropathy (AMN), a rare genetic disorder.

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Autobahn pipeline diagram

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ABX-002 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-002 engages thyroid hormone receptors selectively in the brain to drive 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-002 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

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ABX diagram

ABX-002 for Rare AMN

Adrenomyeloneuropathy (AMN) is the adult form of X-linked adrenoleukodystrophy (X-ALD), a rare genetic disorder, where mutations in the ABCD1 gene prevent the degradation of very long-chain fatty acids (VLCFAs).  The accumulation of VLCFAs is toxic and causes progressive degeneration and demyelination of the long neurons of the spinal cord.

ABX-002 engages thyroid hormone receptors selectively in the spinal cord and brain to upregulate the ABCD2 gene, an alternative VLCFA transporter, that can compensate for the loss of ABCD1 function, lowering VLCFA levels and the resulting toxicity in the CNS. It also drives the differentiation of oligodendrocyte precursor cells into mature, myelinating oligodendrocytes that restore myelin and protect neurons.

About AMN

ALD Connect
  • Prevalence

    Rare genetic disorder affecting ~15,000 people in the U.S.

  • Patient Impact

    Severe gait disturbance with spasticity and ataxia, sensory loss, adrenal insufficiency, incontinence and impotence

  • Current Treatment

    No approved treatments available today

About Autobahn

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