Praxis’ Mid-Stage Triumph Propels Advancement of Novel Epilepsy Drug

1. Praxis Precision Medicines, Inc.'s stock (PRAX) experienced a 23.1% surge due to positive results from their mid-stage PRAX-628 trial.
2. PRAX-628 targets the hyperexcitable state of sodium channels in the brain, aiming to treat adult focal onset epilepsy as a once-daily oral treatment.
3. The Phase IIa proof of concept study included two doses (15 mg and 45 mg) with ten patients demonstrating photo paroxysmal response (PPR).
4. In the 45 mg cohort, 100% of patients achieved complete responses, while in the 15 mg cohort, 80% had complete responses, and 20% partial responses.
5. Safety profile was consistent with prior dose escalation studies, and pharmacokinetic analysis confirmed therapeutic exposures.
6. Praxis plans to initiate an efficacy study for PRAX-628 in focal onset epilepsy during the second half of 2024.
7. Praxis has established a diversified, multimodal central nervous system (CNS) portfolio with four clinical-stage product candidates across movement disorders and epilepsy.

These positive results from the mid-stage trial have cleared the path for PRAX-628's next phase of testing, which will be an efficacy study planned for later this year, 2024.