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
Brain Targeting Chemistry Platform
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 clinical precedence, human genetics and biology, de-risking our pipeline and accelerating early 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
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.
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 potential treatment for multiple CNS disorders, initially focusing on the adjunctive treatment for people with major depressive disorder (MDD).
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 MDD patients, and may have additional benefits on cellular energy metabolism and neurogenesis and increased brain connectivity. Synthetic thyroid hormone (T3/T4) has been used for over 50 years as an adjunctive treatment for people suffering from MDD and extensive literature suggests that a brain-targeted thyromimetic, such as ABX-002, may, in a more selective fashion, elicit relevant brain pharmacology while also diminishing peripherally-driven side effects of synthetic thyroid hormone. ABX-002 has completed dosing in a Phase 1 single and multiple ascending dose trial evaluating the safety, tolerability, and pharmacokinetic profile and is currently being prepared for Phase 2.
~21M patients in the U.S. suffer from MDD
MDD patients suffer from an exasperated state of helplessness, grief, and increased suicidality
Limited safe and effective adjunctive therapies leaving patients desperate for new treatment options
We are advancing the development of ABX-003, a next generation TRβ-selective thyromimetic that utilizes combination with a peripheral FAAH inhibitor to further enhance brain distribution of free drug. ABX-003 offers lifecycle management and franchise opportunity to maximize the value of selective and potent, CNS-active thyromimetics. ABX-003 is currently in IND enabling studies.
Sphingosine-1-Phosphate (S1P) receptor modulators are a well-studied and clinically validated class of drugs that are ideally suited for Autobahn’s FAAH-mediated brain-targeting prodrug platform. Direct anti-inflammatory and neuroprotective actions of S1P in the CNS are known but limited evidence exists to support CNS-specific benefit of other programs, given their inability to achieve meaningful free drug concentrations in the brain.
Autobahn is applying a rationale chemistry, PK, and pharmacology approach to demonstrate improved delivery of free drug to the brain and an ability to unlock novel central pharmacology that would be applicable to both broad neuroimmune/neurodegenerative disorders and rare indications. Autobahn has demonstrated robust neuroinflammatory and neuroprotective benefits with ABX-101 in multiple disease-relevant nonclinical models. We are currently conducting drug candidate designation studies.
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