Our pipeline is built on deep insights into the biology of central nervous system (CNS) disorders, advancing investigational therapies, including several recognized with FDA Breakthrough Therapy Designation, designed to address the underlying causes of disease and improve outcomes for patients.
Small Molecule Platform Selective T-type calcium channel modulator Sodium channel functional state modulator for phenotypic DEEs Sodium channel functional state modulator for broad use KCNT1 specific inhibitor
Cerebrum™
Program
Indication
Pre-Clinical
Phase 1
Phase 2
Phase 3
FILING
ulixacaltamide1
Essential Tremor
Pre-clinical
Phase 1
Phase 2
Phase 3
FILING
relutrigine2
SCN2A- and SCN8A-DEE
Pre-clinical
Phase 1
Phase 2
Phase 3
FILING
Broad DEEs
Pre-clinical
Phase 1
Phase 2
Phase 3
FILING
vormatrigine
Adjunctive FOS
Pre-clinical
Phase 1
Phase 2
Phase 3
FILING
Monotherapy FOS
Pre-clinical
Phase 1
Phase 2
Phase 3
FILING
PRAX-020
KCNT1
Pre-clinical
Phase 1
Phase 2
Phase 3
FILING
Antisense Oligonucleotide (ASO) Platform Gapmer ASO Gapmer ASO Splice switching ASO Undisclosed mechanism ASO
Solidus™
Program
Indication
Pre-Clinical
Phase 1
Phase 2
Phase 3
FILING
elsunersen3
Early onset SCN2A
Pre-clinical
Phase 1
Phase 2
Phase 3
FILING
PRAX-080
PCDH19
Pre-clinical
Phase 1
Phase 2
Phase 3
FILING
PRAX-090
SYNGAP1
Pre-clinical
Phase 1
Phase 2
Phase 3
FILING
PRAX-100
SCN2A Autism
Pre-clinical
Phase 1
Phase 2
Phase 3
FILING
- 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 BTD, ODD and RPD designation from the FDA, and ODD and Priority Medicines (PRIME) designations from the EMA for the treatment of early SCN2A DEE
Note: DEE: developmental & epileptic encephalopathy, FOS: focal onset seizures
About Our Platforms
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Cerebrum™ – Small Molecule Platform
CerebrumTM 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
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Solidus™ – Antisense Oligonucleotide (ASO) Platform
SolidusTM 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
Explore More
Link to: Publications
Publications
Browse the scientific publications that showcase our research, discoveries, and commitment to advancing neuroscience.
Link to: Science
Our Science
Learn how insights from genetics, neuronal excitability, and disease biology inform our approach to developing potential therapies for neurologic disorders.
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Investor Hub
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