Praxis Precision Medicines, Inc.

We are a fully integrated, leading central nervous system (CNS) precision neuroscience biopharmaceutical company. We translate insights from genetic epilepsies to develop therapies for CNS disorders marked by an imbalance in neuronal excitation and inhibition. Normal brain function relies on a delicate balance of excitation and inhibition in neuronal circuits, and dysregulation can lead to abnormal function and neurological disorders. We apply genetic insights to discover and develop therapies for neurological disorders using two proprietary platforms, based on our understanding of shared biological targets and circuits in the brain. Each platform currently has multiple programs with potential for expansion. Our Cerebrum™ platform, for small molecules, uses a deep understanding of neuronal excitability and networks, applying computational and experimental tools to develop orally available precision therapies. Our Solidus™ platform, for antisense oligonucleotides (ASO), is an efficient, targeted precision medicine discovery and development engine built on a proprietary computational methodology. Both platforms use a patient-guided approach, leveraging translational tools like novel transgenic and predictive translational animal models and electrophysiology markers, to achieve proof-of-concept in patients efficiently. This approach has resulted in a diversified, multimodal CNS portfolio with four clinical-stage product candidates for movement disorders and epilepsy. Cerebrum™ uses computational and experimental tools to discover and develop CNS small molecule therapies. Our ion channel discovery science team and collaborators create and execute novel screening cascades for selected genetic targets. Our multiscale disease models link genetic causes to network function to identify disease drivers. We use translational biomarkers like quantitative electroencephalography (qEEG) to guide dose selection. Genomics and informatics guide the precision application of our candidates to identify, stratify, and activate patients for trial recruitment and execution.