The Biology of Brain Function
The central nervous system (CNS) relies on precise electrical signals to control how we move, think, learn, and interact with the world. These signals are generated by neurons and regulated by ion channels (sodium, potassium, etc) that help control neuronal excitability. When this delicate balance is disrupted, neurons can become either overactive or underactive, contributing to a wide range of neurological disorders.
Our Approach to Precision Neuroscience
We use precision neuroscience to identify and target defined mechanisms of CNS disorders, integrating insights from neuronal signaling biology, genetics, translational science, and patient experience to guide therapeutic development. Informed by the genetics of disease and broader advances in CNS research, our work supports a diversified portfolio across movement disorders and epilepsy, spanning our small molecule and antisense oligonucleotide platforms, CerebrumTM and SolidusTM.
1
Genetics
Focus on therapeutic targets identified through human genetics
2
Translational Tools
Utilize translational tools to validate the potential of our targets and product candidates and potentially provide early proof of biology
3
Patient-guided
Apply patient-guided development strategies to deliver on what patients actually need
4
Efficient & Rigorous
Pursue efficient, rigorous clinical development paths to proof-of-concept in humans
Multiple Platforms, Multiple Possibilities
Different diseases present different challenges, from the underlying biological mechanisms to how treatments fit into patients’ lives. Our two discovery platforms give us the flexibility to pursue the therapeutic strategy best suited to each opportunity.
Cerebrum™
Small Molecule Platform
Cerebrum™ utilizes deep understanding of neuronal excitability and neuronal networks and applies a series of computational and experimental tools to develop orally available precision therapies.
- Designed with everyday treatment experience in mind
- Optimized to reach CNS targets
- Built on a well-established, scalable therapeutic modality
| MOLECULE | INDICATION | MECHANISM |
|---|---|---|
| ulixacaltamide1 | Essential Tremor | Selective T-type calcium channel modulator |
| vormatrigine | Focal Onset Seizures & Generalized Epilepsy | Sodium channel functional state modulator for broad use |
| relutrigine2 | SCN2A- & SCN8A-DEE Broad DEEs | Sodium channel functional state modulator for phenotypic DEEs |
| PRAX-020 | KCNT1 | KCNT1 specific inhibitor |
| PRAX-050 | Movement Disorders | Not disclosed |
Solidus™
Antisense Oligonucleotide (ASO) Platform
Solidus™ is an efficient, targeted precision medicine discovery and development engine for ASOs anchored on a proprietary, computational methodology.
- Addresses disease biology at the genetic level
- Designed to target disease-causing RNA with high specificity
- Potential to reach targets not accessible with traditional medicines
| MOLECULE | INDICATION | MECHANISM |
|---|---|---|
| elsunersen3 | Early onset SCN2A | Gapmer ASO |
| PRAX-080 | PCDH19 | Gapmer ASO |
| PRAX-090 | SYNGAP1 | Splice switching ASO |
| PRAX-100 | SCN2A Autism | Undisclosed mechanism ASO |
- Ulixacaltamide has received Breakthrough Therapy Designation (BTD)
- Relutrigine has received BTD, Orphan Drug Designation (ODD) and Rare Pediatric Disease (RPD) designation from the FDA, and ODD from the European Medicines Agency (EMA) for the treatment of SCN2A and SCN8A-DEE and RPD designation for Dravet Syndrome
- Elsunersen has received ODD and RPD designation from the FDA, and ODD and Priority Medicines (PRIME) designations from the EMA for the treatment of early onset SCN2A DEE
DEE: developmental & epileptic encephalopathy, FOS: focal onset seizures
A Differentiated CNS Pipeline
Our portfolio applies precision neuroscience to address mechanisms of disease across multiple CNS conditions, with multiple late-stage programs and a growing pipeline focused on areas of urgent need.
Disease Focus
Link to: Essential Tremor
Essential Tremor (ET)
ET is one of the most common movement disorders, causing involuntary shaking that can make everyday activities more difficult. An estimated 7 million people in the U.S. are affected by ET.
Link to: Developmental and Epileptic Encephalopathies
Developmental & Epileptic Encephalopathies (DEEs)
DEEs are rare, severe epilepsies that often begin in childhood and can affect development, cognition, movement, communication, and daily life. DEEs impact more than 200,000 people in the U.S.
Link to: Epilepsy
Epilepsy
Epilepsy is a chronic neurological disorder marked by recurrent seizures that can vary in frequency, severity, and presentation. An estimated 3 million people in the U.S. are living with epilepsy.