The FDA has accepted Praxis Precision Medicines' NDA for relutrigine with priority review and set a PDUFA target date of September 27, 2026. The therapy targets SCN2A and SCN8A developmental and epileptic encephalopathies and could become the first approved treatment for these rare conditions. The company has bolstered its cash position to support commercial launch preparations.
Research from Germany and Australia shows zebrafish functional testing can identify false-positive SMA newborn screening results, preventing unnecessary treatment in rare cases where infants carry functional SMN1 gene variants.
Multiple analysts have raised price targets on Praxis Precision Medicines, with Wolfe Research initiating coverage at $500 and Guggenheim increasing its target to $800, citing strong market potential for ulixacaltamide in essential tremor.
Praxis Precision Medicines submitted NDAs for ulixacaltamide and relutrigine to the FDA, reported cash and investments of $926 million as of December 31, 2025, and disclosed a net loss of $303.3 million for full-year 2025.
Praxis Precision Medicines is preparing to submit two New Drug Applications by mid-February 2026 for ulixacaltamide and relutrigine, both holding FDA Breakthrough Therapy Designation, targeting essential tremor and epilepsy markets.
Praxis Precision Medicines has submitted new drug applications for ulixacaltamide in essential tremor and relutrigine in rare epilepsies, both granted Breakthrough Therapy Designation. The company reported $926 million in cash as of December 31, 2025.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07595666 |
Comparison of NeuroEXPLORER PET/CT Versus Standard-of-care PET/CT of the Head and Neck Region. |
ENROLLING_BY_INVITATION | NA |
| NCT07384442 |
Effects of Targeted Temporal Interference Stimulation of Cerebellar Nuclei on Tremor and Gait Disturbance in Parkinson's Disease Patients |
RECRUITING | NA |
| NCT07236892 |
Towards Restoring Complex Movement After Paralysis: Algorithm Development With Healthy Participants |
RECRUITING | NA |
| NCT07232147 |
Clinical Research on Stem Cell Therapy for Parkinson's Disease |
NOT_YET_RECRUITING | PHASE1 |
| NCT07131865 |
Comparison of the Functional Status, Psychosocial Aspects and Discharge Locations of Geriatric Patients Before and After Inpatient Geriatric Rehabilitation |
COMPLETED | |
| NCT06999096 |
Long-read Genome Sequencing for the Molecular Diagnosis of Dystonia |
RECRUITING | NA |
| NCT06967727 |
Registry and Natural History of Epilepsy-Dyskinesia Syndromes |
RECRUITING | |
| NCT06958341 |
Neuroimmunology Registry and Biobank |
RECRUITING | |
| NCT06948019 |
Safety and Efficacy of AAV9/AP4B1 (BFB-101) For Patients With AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47) |
NOT_YET_RECRUITING | PHASE1/PHASE2 |
| NCT06912841 |
Deep Brain Stimulation (DBS) MatchMaker |
ENROLLING_BY_INVITATION |
