PRAX | Market Cap: $8.8B (07/13/26)
Industry:
Pharma & Biotech

DESCRIPTION

Praxis Precision Medicine is a clinical-stage CNS drug developer focused on epilepsy and movement disorders, built around the insight that genetics of epilepsy illuminate shared biological targets across neurological conditions characterized by excessive neuronal excitability. Praxis has four clinical-stage drug candidates: ulixacaltamide for essential tremor, relutrigine for developmental and epileptic encephalopathies (DEEs), vormatrigine for focal onset seizures, and elsunersen, an antisense oligonucleotide (ASO) for a rare genetic epilepsy. Praxis is pre-revenue but has filed NDAs for both ulixacaltamide and relutrigine, with approval decisions pending and commercial launches being prepared simultaneously. Vormatrigine and elsunersen are in Phase 2/3 trials, with NDA submissions targeted for 2027. The company discovers drugs through two platforms: Cerebrum, which produces oral small molecules targeting ion channels, and Solidus, which produces ASOs that modulate gene expression. Praxis's business model is standard specialty pharma — invest in R&D to develop patented drugs, then sell at premium prices into unmet medical needs. Relutrigine and elsunersen carry Rare Pediatric Disease Designation, entitling Praxis to Priority Review Vouchers upon approval, which typically sell for hundreds of millions of dollars. Praxis outsources all manufacturing to CDMOs, keeping the asset base relatively light. The company ended 2025 with roughly $926M in cash, boosted by a January 2026 follow-on offering to a pro forma ~$1.5B, which management says funds operations into 2028.

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