Praxis Precision Medicines is a clinical-stage biopharmaceutical company focused on developing new treatments for disorders affecting the central nervous system. The company translates insights gained from studying genetic epilepsies into therapies designed to address conditions characterized by an imbalance in neuronal excitation and inhibition. Their work aims to improve the lives of individuals living with complex brain disorders.
Decoding the Drug Development Pipeline
The journey of a new medicine from discovery to patient use involves a structured process known as the drug development pipeline. This pipeline begins with foundational research and discovery, identifying potential drug candidates and understanding their biological targets. After this initial stage, promising candidates move into preclinical testing, which involves laboratory and animal studies to assess safety and efficacy before human trials.
Following successful preclinical results, a drug candidate progresses into human clinical trials. Phase 1 studies involve a small group of healthy volunteers or patients to evaluate safety, dosage, and how the drug is processed by the body. Phase 2 trials then enroll a larger patient population to further assess effectiveness and monitor for side effects. Finally, Phase 3 trials are extensive, involving hundreds to thousands of patients, to confirm the drug’s effectiveness, track adverse reactions, and compare it to existing treatments.
Leading Drug Candidates and Their Focus
Praxis Precision Medicines is advancing several drug candidates, each designed to address specific neurological conditions.
Relutrigine, also known as PRAX-114, is being developed for various epilepsies, including severe forms like developmental and epileptic encephalopathies (DEEs) such as SCN2A and SCN8A DEEs, and Dravet Syndrome.
Another candidate, elsunersen (PRAX-222), targets early-seizure-onset SCN2A gain-of-function developmental epilepsies. This investigational antisense oligonucleotide (ASO) is designed to selectively reduce the expression of the SCN2A gene. Laboratory studies have shown elsunersen can decrease SCN2A gene expression and protein levels, and animal models demonstrated reductions in seizures, improved activity, and increased survival. In an initial analysis of the EMBRAVE study, four patients receiving elsunersen experienced a 43% reduction in seizure burden over a four-month period.
Vormatrigine (PRAX-628) is a small molecule for focal onset seizures and generalized epilepsy. This drug is a next-generation, functionally selective compound that targets hyperexcitable sodium channels in the brain. It is being developed as a once-daily oral medication. Ulixacaltamide is undergoing late-stage development for essential tremor.
Proprietary Technology Platforms
Praxis Precision Medicines utilizes two distinct proprietary technology platforms to discover and develop its drug candidates: the Cerebrum™ small molecule platform and the Solidus™ ASO platform.
The Cerebrum™ platform focuses on developing small molecule drugs, which are chemical compounds with a low molecular weight. These molecules are able to enter cells and interact with specific targets within the body.
The Solidus™ ASO platform is dedicated to developing Antisense Oligonucleotides, or ASOs. ASOs are short, synthetic strands of nucleic acids designed to bind selectively to messenger RNA (mRNA), which carries genetic instructions from DNA for protein production. By targeting specific mRNA molecules, ASOs can either increase or decrease the production of particular proteins.
Milestones in Clinical Development
Praxis Precision Medicines has several key milestones anticipated for its pipeline candidates. The registrational EMERALD study for relutrigine is expected to begin in the first half of 2025. The EMBOLD study, which is enrolling patients with SCN2A and SCN8A DEEs, anticipates topline results from its second registrational cohort in the first half of 2026, with a New Drug Application filing projected for later that year.
For elsunersen, Praxis has completed regulatory discussions. The second cohort of the EMBRAVE study is continuing to enroll patients in Brazil. Vormatrigine’s development is progressing, with topline results from the RADIANT Phase 2 study for focal onset seizures and generalized epilepsy expected by mid-2025.
The Essential3 program for ulixacaltamide, targeting essential tremor, had its Study 1 recommended for discontinuation. Praxis plans to complete both Study 1 and 2, with topline results anticipated in the third quarter of 2025. In December 2024, UCB exercised its option to license a KCNT1 small molecule development candidate.