Praxis Precision Medicines Says PRAX-628 Can Potentially Be The Best-In-Class Treatment For Focal Epilepsy

Praxis Precision Medicines Inc PRAX announced topline results from the PRAX-628 Phase 1 healthy volunteer study evaluating single ascending doses (SAD) and multiple ascending doses (MAD) of PRAX-628.

PRAX-628 is a next-generation, functionally selective small molecule targeting the hyperexcitable state of sodium channels in the brain. 

PRAX-628 is being developed as a once-daily oral treatment for adult focal onset epilepsy.

Related: Despite Trial Failure, Praxis Still Seeks Advancing Essential Tremor Program Into Phase 3 Study.

In the study, PRAX-628 or placebo was administered to 40 healthy participants (n=30, placebo=10)

SAD cohorts evaluated PRAX-628 doses ranging from 5 mg to 45 mg, and MAD cohorts evaluated PRAX-628 doses of 20 mg and 30 mg, resulting in concentrations of more than 15-fold the mouse Maximal Electroshock Seizure model (MES) EC50.

PRAX-628 was generally well-tolerated at all tested doses.

PK data demonstrated dose-dependent exposure supporting once-daily dosing without titration to achieve potentially therapeutically effective drug concentration levels.

The most common treatment-related adverse events (AEs) across all cohorts were fatigue, dizziness, somnolence, headache, disturbance in attention, and nausea.

Praxis intends to advance PRAX-628 into a Phase 2 study in focal epilepsy in Q4 of 2023.

Price Action: PRAX shares are down 0.93% at $1.07 on the last check Thursday.