Praxis(US:PRAX)2022-08-11 17:55Pipeline Overview - Praxis is developing new classes of treatments inspired by the genetics of epilepsy, targeting common and rare diseases connected by neuronal imbalance[3, 6, 11] - Praxis has a broad CNS portfolio with uncorrelated program risk and a deep early-stage pipeline enabling continuous advancement of new programs[16] - Praxis anticipates a milestone-rich second half of 2022, including Phase 2a Part B topline results for PRAX-944 in Essential Tremor (ET), initiating a Phase 2 trial for PRAX-944 in Parkinson's Disease (PD), and initiating a Phase 2 study for PRAX-222 in SCN2A-DEE[17, 22, 27] PRAX-944 for Movement Disorders - The US ET market represents a $4B+ opportunity[37] - Approximately 50% of patients discontinue medication due to limited efficacy and poor tolerability[33] - PRAX-944 is a differentiated, selective T-type calcium channel blocker with potential for effectiveness across a range of neuronal activity levels[42] - In a Phase 2a study, patients treated with PRAX-944 showed a 42% mean improvement in modified ADLs from baseline, while withdrawal of PRAX-944 resulted in regression to baseline severity with a 139% worsening[48] Epilepsy Portfolio - Praxis is delivering first and best-in-class precision medicines for 25,000+ rare epilepsy patients[75] - The US diagnosed prevalence for rare epilepsy programs represents a $742 million market in 2021 sales, estimated to grow to $1.5 billion in 2026[76] - Preclinical and emerging clinical data demonstrate PRAX-562 will be a first- and best-in-class NaV blocker for DEEs[78] - In Part A of Phase 1 study, dosing with 90 mg of PRAX-562 over 28 days resulted in concentrations that exceeded the EC50 in the MES model by 13-fold[94]