Praxis Precision Medicines, Inc. is a biopharmaceutical firm operating at the clinical stage, dedicated to creating innovative treatments for central nervous system ...
Ulixacaltamide is a differentiated, highly selective small molecule inhibitor of T-type calcium channels designed to block abnormal neuronal burst firing in the cerebello-thalamo-cortical (CTC) circuit correlated with tremor activity. It is the most advanced program within Praxis's Cerebrum small molecule platform and has received Breakthrough Therapy Designation from the FDA. The Essential3 program includes two Phase 3 studies: a parallel-design placebo-controlled study (Study 1) and a randomized withdrawal study (Study 2). Topline results from both studies are expected in Q3 2025. Despite a pre-planned interim futility analysis for Study 1, the company continues the studies to completion.
Price Range: Not yet commercialized (pricing to be determined post-approval)
Pain Points: Essential tremor is inadequately managed with existing therapies and has high unmet need. Current treatments have limited efficacy or tolerability issues. Ulixacaltamide aims to provide a well-tolerated, effective therapy specifically targeting the underlying tremor mechanism.
Solutions: It provides a potential first-line or adjunctive therapy for essential tremor by selectively modulating T-type calcium channels to reduce tremor intensity and improve quality of life.
Target Users: Patients with essential tremor who are inadequately controlled on current therapies, primarily adults, and their prescribing neurologists.
Supply Chain
API synthesis via contract manufacturing organizations (CMOs)Formulation and fill/finish at CMOsQuality control and release testingDistribution through specialty pharmacy networks (if approved)
Vormatrigine (formerly PRAX-628) is a potent, orally available sodium channel functional state modulator designed to target hyperexcitable sodium channels in the brain. It is being developed for adult focal onset seizures and generalized epilepsy. Preclinical data show it is highly selective for the disease-state sodium channel, and Phase 1 data demonstrated exposures over 20 times the predicted human equivalent of the rodent MES EC50, with a tolerable safety profile and no food effect. The ENERGY program includes the RADIANT Phase 2 study (topline expected mid-2025), the POWER1 Phase 2/3 registrational study (topline expected H2 2025), and the POWER2 study (initiation H2 2025). It has a best-in-class potential due to its potency, selectivity, and safety profile.
Price Range: Not yet commercialized
Pain Points: Current sodium channel blocking antiepileptic drugs have significant safety and efficacy limitations, including side effects and insufficient seizure control. Vormatrigine aims to provide a more potent, selective, and better-tolerated option.
Solutions: It offers a potential best-in-class therapy for common epilepsies by precisely targeting hyperexcitable sodium channels, achieving high efficacy while minimizing off-target effects.
Target Users: Adults with focal onset seizures or generalized epilepsy, especially those with treatment-resistant forms. Prescribers include neurologists and epileptologists.
Supply Chain
API supply from CMOsFinished dosage form manufacturingAnalytical testing and releaseDistribution to clinical trial sites and eventual commercial pharmacies
Relutrigine (formerly PRAX-562) is a small molecule designed to preferentially block persistent sodium current, a key driver of seizures in early-onset SCN2A-DEE and SCN8A-DEE. It has shown dose-dependent seizure inhibition up to complete control in mouse models and has been generally well-tolerated in Phase 1 studies. It has received FDA Breakthrough Therapy Designation, Orphan Drug Designation, and Rare Pediatric Disease Designation for multiple DEE indications. The registrational EMBOLD study for SCN2A/SCN8A-DEE is ongoing, with topline results expected no later than H1 2026. The company will also initiate the EMERALD study for broad DEEs in mid-2025.
Price Range: Not yet commercialized
Pain Points: DEEs are severe, often treatment-resistant epilepsies with profound developmental impact and limited therapeutic options. Relutrigine targets the underlying pathophysiology of persistent sodium current to reduce seizures and potentially improve neurodevelopmental outcomes.
Solutions: It provides a disease-modifying therapy for DEEs by addressing a core mechanism, offering hope for seizure control and developmental improvement in affected children.
Target Users: Pediatric patients with SCN2A- or SCN8A-DEE (and eventually broader DEEs), their caregivers, and pediatric neurologists specializing in epilepsy.
Supply Chain
API sourced from CMOsOral suspension or tablet manufacturingAnalytical testing and stability studiesDistribution to clinical trial centers
Elsunersen (formerly PRAX-222) is a gapmer ASO that selectively decreases SCN2A gene expression, directly targeting the underlying cause of gain-of-function SCN2A developmental and epileptic encephalopathy. In vitro studies show reduction in SCN2A expression and protein levels; in vivo, it has demonstrated dose-dependent reduction in seizures, improved behavior, and increased survival in mouse models. It has received Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA, and PRIME designation from the EMA. The program is in collaboration with Ionis Pharmaceuticals and RogCon. The EMBRAVE Part A Phase 1/2 study is enrolling, with topline results expected H1 2026, and the Phase 3 EMBRAVE3 study will initiate mid-2025. An emergency-use patient experience was published in Nature Medicine.
Price Range: Not yet commercialized
Pain Points: SCN2A DEE is a severe, often refractory epilepsy with limited treatment options and devastating developmental consequences. Elsunersen targets the genetic cause directly.
Solutions: It provides a potential disease-modifying therapy by reducing expression of the mutant SCN2A gene, reducing seizure burden and improving neurodevelopmental outcomes.
Target Users: Infants and children with early-onset SCN2A gain-of-function DEE, their families, and pediatric neurologists with expertise in genetic epilepsies.
Supply Chain
Oligonucleotide synthesis at CMOs with GMP capabilityFormulation and filling into vials for intrathecal useCold chain distribution to clinical sitesQuality control and release testing
PRAX-020 is a small molecule development candidate that specifically inhibits KCNT1 potassium channels. KCNT1 gain-of-function mutations cause severe epilepsy. The program is preclinical and was part of a collaboration with UCB (which exercised option to in-license). Praxis continues development internally or with partner. No clinical trials yet announced.
Price Range: Preclinical (not yet priced)
Pain Points: KCNT1 epilepsy is a rare, severe condition with no targeted therapies. Addresses underlying channelopathy.
Solutions: Precision inhibitor of KCNT1, reducing neuronal hyperexcitability due to gain-of-function mutations.
Target Users: Patients with KCNT1 mutation-positive epilepsy (pediatric).
Supply Chain
Research-stage supply from CROsFuture clinical supply through CMOs if advanced
PRAX-080 is a gapmer ASO from the Solidus platform designed to address PCDH19 loss-of-function epilepsy by targeting mosaic expression. It is currently in preclinical development, with a clinical candidate expected to be declared in the first half of 2026.
Price Range: Preclinical (not yet priced)
Pain Points: No approved therapies for PCDH19 epilepsy; ASO approach can modulate gene expression to restore function.
Solutions: Targets the underlying genetic defect, potentially reducing seizure frequency and improving development.
Target Users: Female patients with PCDH19 mutations (pediatric and adult).
Supply Chain
Oligonucleotide synthesis at CMOsFormulation developmentPreclinical testing
PRAX-090 is a splice-switching ASO designed to correct SYNGAP1 loss-of-function, a leading cause of severe intellectual disability and epilepsy in DEEs. Preclinical program aiming to declare a clinical candidate in the first half of 2026.
Price Range: Preclinical (not yet priced)
Pain Points: SYNGAP1 mutations cause severe epilepsy, intellectual disability, autism, and no targeted treatments exist.
Target Users: Patients with SYNGAP1 deficiency (primarily children) and their caregivers.
Supply Chain
ASO synthesis by CMOsFormulation and delivery optimization
PRAX-100 is an ASO (mechanism undisclosed) designed to address SCN2A loss-of-function mutations, the predominant genetic link to de novo autism spectrum disorders. Preclinical program expecting to declare a clinical candidate in the first half of 2026.
Price Range: Preclinical (not yet priced)
Pain Points: Autism due to SCN2A mutations has no targeted therapies; current treatments are symptomatic.
Solutions: Directly addresses the genetic cause by modulating SCN2A expression, potentially improving core autism symptoms.
Target Users: Individuals with SCN2A loss-of-function mutation-related autism, likely diagnosed in childhood.
Supply Chain
ASO manufacturing at CMOsFormulation for intrathecal or systemic delivery
