Mid-stage win for Praxis clears path for epilepsy drug's next test

2024-03-26

临床结果临床2期

Praxis Precision Medicines revealed Phase IIa findings for its experimental PRAX-628 therapy, demonstrating a 100% complete response in the high-dose group of patients with photosensitive epilepsy. The positive readout Tuesday paves the way for Praxis to advance the programme later this year.

"The strength and consistency of response across both study arms, combined with a continued positive tolerability and safety profile, build on our earlier conviction that PRAX-628 has the potential to be the first precision sodium channel modulator for focal epilepsy patients," said CEO Marcio Souza.

The study included 10 epilepsy patients who demonstrated photo paroxysmal response (PPR) during screening, with eight eligible for assessment. PPR studies can reveal abnormal patterns of brain activity in response to flashing or flickering lights. Analysing EEG signatures after intermittent photic stimulation can serve as a gauge for the effectiveness of potential anti-epileptic treatments, according to the company.

The study is evaluating two dose levels. Earlier this year, Praxis said a preliminary analysis of the lower 15mg cohort had exceeded its expectations, driving up shares by 23% at the time. On Tuesday, the company said four of the five patients (80%) in that group achieved a complete response, which was defined as the complete elimination of generalised PPR events at any assessment period versus baseline.

In the higher 45mg cohort, all three patients (100%) achieved a complete response. The combined response rate was 88% across the two cohorts. In a note prior to the readout, Jefferies analysts said they viewed ≥50% complete/partial suppression as "an important threshold to meet/exceed" in to order to demonstrate proof-of-concept.

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