Bringing a new approach to precision medicine neuroscience
We’re creating innovative therapies for diseases of the central nervous system
Brain-penetrating therapies to address neurodegenerative and neurological diseases and brain cancers are often limited by serious peripheral toxicities, such as irreversible liver, lung, and cardiovascular damage, gastrointestinal issues, anemia, and infection.
As a result, these diseases remain among the most significant unmet medical needs, with many important targets still considered undruggable.
Montara’s BrainOnly™ platform holds the potential to pursue numerous previously undruggable targets across a broad spectrum of brain-related diseases and usher in a new generation of brain-targeting drugs.
Two-drug combination therapy to enable next-generation brain-targeting drugs
The BrainOnly platform enables the design of two-drug combination therapies: a brain-penetrant, target-specific drug + a non–brain-penetrant “universal” peripheral blocker. Montara’s novel approach blocks the target-specific drug’s activity in the periphery while simultaneously activating and concentrating its activity in the brain.
Abbreviation: BBB, blood-brain barrier.
How the BrainOnly technology works
Both drugs in our two-drug combination therapies are designed to bind to a druggable and broadly expressed presenter protein in the brain and the periphery.
We block peripheral activity to prevent adverse events and enhance safety.
The non–brain-penetrant universal blocker is a non-immunosuppressive small molecule that saturates immunophilin presenter proteins in the periphery. Because of this saturation, the active chimera (ie, the brain-penetrant, target-specific drug engineered with a competing immunophilin binder) cannot bind to the immunophilin presenter in the periphery. It can only be activated when it binds to open presenter protein binding sites in the brain.
This prevents peripheral activity of the target-specific drug and thus peripheral adverse events, enhancing safety.
We push the dose of the target-specific drug in the brain to enhance specificity and efficacy.
The active chimera crosses the blood-brain barrier. It binds with the target protein as well as the immunophilin presenter protein, allowing activity. The high intracellular concentration of the active drug may enhance efficacy against the target.
Based on pioneering research published in Nature
The BrainOnly platform is based on pioneering research conducted in the lab of Professor Kevan Shokat, PhD, at the University of California, San Francisco (UCSF). The research was published in Nature in 2022. Montara holds an exclusive license from UCSF for the development and commercialization of the BrainOnly platform.
Learn more about our foundersEnabling broad therapeutic discovery across brain indications
The BrainOnly platform can be leveraged to discover novel small molecules and enhance the safety and efficacy of existing small molecules. This affords Montara vast opportunities to address unmet medical needs through internal pipeline development and partnering.
Brain-penetrant, target-specific drug engineered with a competing immunophilin binder
Potential Indications
Neurodegeneration
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Alzheimer’s disease |
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ALS/FTD |
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Parkinson’s disease |
Neurological disorders
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Epilepsy and seizure disorders |
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Psychiatric indications |
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Multiple sclerosis |
Neuro-oncology
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Brain tumors |
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Brain metastases |
Abbreviations: ALS, amyotrophic lateral sclerosis; FTD, frontotemporal degeneration.
