PRAX-628 is used for the treatment of focal epilepsy. Credit: Gerd Altmann / Pixabay.

Praxis Precision Medicines has reported positive topline results from a Phase I clinical trial of PRAX-628 for the treatment of focal epilepsy.

The study has enrolled 40 healthy subjects to evaluate the tolerability, safety, and pharmacokinetics (PK) of single ascending doses (SAD) and multiple ascending doses (MAD) of PRAX-628.

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Out of 40, 30 subjects were divided into SAD cohorts and MAD cohorts, and the remaining as placebo.

SAD cohorts received 5mg to 45mg dosage of PRAX-628 while MAD cohorts were administered with 20mg and 30mg doses.

PRAX-628 was well-tolerated at all tested doses with common treatment-related adverse events such as nausea, dizziness, somnolence, fatigue, headache, and disturbance in attention observed.

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PK data showed dose-dependent exposure and supported dosing once a day without titration for achieving potentially therapeutically effective drug concentration levels.

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No vital signs or neurological examination findings, clinically significant ECG findings, or serious adverse events were observed during the study.

Praxis Precision Medicines president and CEO Marcio Souza said: “The ultimate goal of an anti-seizure medication is seizure freedom for all affected patients, and a wide therapeutic window is critical to reaching that goal.

“Today’s results support previously announced preclinical data to demonstrate that the unique functional selectivity of PRAX-628 may lead to a wider therapeutic range than current standard-of-care and other potential treatments in development.”

The company is also planning to initiate a Phase II study of PRAX-628 in the fourth quarter of this year for focal epilepsy.

A next-generation, functionally selective small molecule PRAX-628 targets the hyperexcitable state of sodium channels in the brain.