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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

  • MDD

  • Multiple Sclerosis

  • Publications

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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 combining the highly specialized enzymatic activity and brain-focused tissue distribution profile of fatty-acid amide hydrolase (FAAH), amplified with novel peripherally restricted FAAH inhibitors, our prodrug and peripheral restriction technology enables us to precisely tailor CNS and peripheral drug exposure.

Optimizing central versus peripheral exposure of active molecules enables us to match biodistribution with disease pathophysiology, providing Autobahn with a range of opportunities to leverage our platform to develop new therapeutics addressing unmet human health needs.

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 potent and selective, CNS-directed thyroid hormone receptor beta (TRβ) agonist that we are developing as a treatment for multiple CNS disorders.

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Treatment Resistant Depression

MDD is the third most common cause of disability worldwide, and leaves patients to suffer from an exasperated state of helplessness, grief, and increased suicidality. The cause of MDD is not fully understood but has centered around disruption of the monoaminergic system (e.g., serotonin and norepinephrine activity). Approved drugs that enhance monoaminergic signaling in the brain have shown beneficial effects in MDD, but many patients continue to suffer from an inadequate response to treatment that sustains their depressive symptoms and disability. ABX-002, is a potent and selective, brain-boosting TRβ agonist that is anticipated to enhance serotonin activity in the brain in TRD patients. ABX-002 is expected to augment antidepressant treatments by potentiating the beneficial effects of these drugs on monoaminergic signaling.

About MDD

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  • Prevalence

    ~21M patients in the U.S. suffer from MDD

  • Patient impact

    MDD patients suffer from an exasperated state of helplessness, grief, and increased suicidality

  • Current treatment

    Limited adjunctive therapies, limited hope for patients with inadequate response to their antidepressant

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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 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-002 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-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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About Autobahn