DARE FOR MORE ®
We dare for more in every aspect of what we do – advancing our portfolio of precision therapies for central nervous system disorders – to make the impossible possible.
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About Us
Praxis Precision Medicines is a fully integrated, leading central nervous system (CNS) precision neuroscience biopharmaceutical company, translating insights from genetic epilepsies into the development of therapies for CNS disorders characterized by neuronal excitation-inhibition imbalance.
Praxis is the process of using a theory or learning in a practical way. It is the mindset that allows us to simplify the complex and independently define a solution without being restricted by predefined processes.
Our Approach to Precision Neuroscience
We use precision neuroscience to identify and target biologically defined mechanisms of disease, integrating insights from human biology, genetics, translational science, and patient experience to guide therapeutic development. Informed by genetic epilepsies and broader CNS biology, our work supports a diversified portfolio across movement disorders and epilepsy, spanning both small molecule and antisense platforms.
01
Genetics
Focus on therapeutic targets identified through human genetics.
02
Translational Tools
Utilize translational tools to validate the potential of our targets and product candidates and potentially provide early proof of biology.
03
Patient-Guided
Apply patient-guided development strategies to deliver on what patients actually need.
04
Efficient & Rigorous
Pursue efficient, rigorous clinical development paths to proof-of-concept in humans.
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A Differentiated CNS Pipeline
Our portfolio spans movement disorders and epilepsy, targeting shared biological drivers of disease across CNS conditions, with multiple late-stage programs.
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
| Molecule | Indication | Mechanism |
|---|---|---|
| ulixacaltamide | Essential Tremor | T-type calcium channel modulator |
| vormatrigine | Focal Onset Seizures & Generalized Epilepsy | Sodium channel functional state modulator for broad use |
| relutrigine* | DEE | Sodium channel functional state modulator for pediatric use |
| PRAX-020 | KCNT1 Epilepsy | 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
| Molecule | Indication | Mechanism |
|---|---|---|
| elsunersen** | SCN2A DEE | Gapmer ASO |
| PRAX-080 | PCDH19 DEE | Gapmer ASO |
| PRAX-090 | SYNGAP1 DEE | Splice switching ASO |
| PRAX-100 | SCN2A Autism | Undisclosed mechanism ASO |
* Relutrigine has received Breakthrough Therapy Designation (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 PRIME designations from the EMA for the treatment of SCN2A GoFDEE=developmental & epileptic encephalopathy, GoF=gain-of-function, LoF=loss-of-function, PRIME=Priority Medicines
| Program | Pre Clinical | Phase One | Phase Two | Phase Three | NDA | |
|---|---|---|---|---|---|---|
|
Cerebrum™
Small Molecule
Platform |
Ulixacaltamide | |||||
| Essential Tremor1 | ||||||
| Relutrigine | ||||||
| SCN2A- and SCN8A-DEE2 | ||||||
| Broad DEEs | ||||||
| Vormatrigine | ||||||
| Adjunctive focal epilepsy | ||||||
| Monotherapy focal epilepsy | ||||||
| PRAX-020 KCNT13 | ||||||
| Pre-Clinical | ||||||
|
Solidus™
ASO Platform
|
Elsunersen | |||||
| Early Onset SCN2A4 | ||||||
| PRAX-080 PCDH19 | ||||||
| PRAX-090 SYNGAP1 | ||||||
| PRAX-100 SCN2A LoF | ||||||
- Ulixacaltamide has received Breakthrough Therapy Designation
- Relutrigine has received Breakthrough Therapy Designation (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 PRIME designations from the EMA for the treatment of SCN2A GoF
DEE=developmental & epileptic encephalopathy, GoF=gain-of-function, LoF=loss-of-function, PRIME=Priority Medicines
Careers
Join Us in Transforming Neuroscience
As we transition toward commercialization, we’re building a team to support the delivery of our first therapies and continued pipeline growth.
- Mission-driven work with real patient impact
- Collaborative, high-accountability culture
- Opportunity to help build a commercial-stage CNS company
Leadership Team
We have attracted a talented team of scientists and researchers in genetics and biology, chemistry and translational medicine as well as business leaders with established track records of successfully executing innovative drug discovery and development programs.
We share a common vision of reshaping the human condition into a more freeing and fulfilled existence by developing high impact medicines for patients and families affected by and living with complex brain disorders.