Praxis Precision Medicines, Inc. · NASDAQ:PRAX
Praxis Precision Medicines — 2022 Epilepsy Foundation Pipeline Conference
8 slides from Praxis Precision Medicines's conference presentation, 2022
Slide 1: PRAX-562 and Praxis pipeline updates
PRAX-562 and Praxis pipeline updates Kris Kahlig, PhD Senior Director, Biology Praxis Precision Medicines 2022 Epilepsy Foundation Pipeline Conference 1 ©2020 Epilepsy Foundation of America, Inc.
Slide 2: Imperatives which guide our epilepsy portfolio build
Imperatives which guide our epilepsy portfolio build Focus directly on Focus on implicated Focus on nodes of underlying genetic defects genes in common pathophysiological convergence in rare epilepsy diseases informed by genetics PRAX-222* PRAX-114 PRAX-562 ASO SMALL MOLECULE SMALL MOLECULE PRAX-020
Slide 3: PRAX-562 preclinical data
PRAX-562 preclinical data MES EFFICACY sLMA TOLERABILITY Total DIstance Travelled (mm) 70000 ** 60 60000 ED50: 2 mg/kg TD50: 44 mg/kg ** MES Latency (sec) 50000 ** 40 40000 ** 30000 20 20000 10000 0 0 Veh 0.3 1 3 10 Veh 10 20 40 CD-1 mice; (n=20/group) CD-1 mice; (n=12/group) **p<0.01 vs. Veh PRAX-5
Slide 4: PRAX-562 preclinical data
PRAX-562 preclinical data IN VIVO POC IN SCN2A SPONTAEOUS SEIZURES1 IN VIVO POC IN SCN8A AUDIOGENIC EVOKED SEIZURES2 +50% 50 100% % Change in Seizures Proportion with Seizures 0 50% -50% -50 * -100% -100 ** ** 0% ** Veh 0.3 1 3 10 Veh Sidack’s post hoc comparison test *p<0.05 vs. Veh PRAX-562 (mg/kg
Slide 5: PRAX-562 emerging clinical data
PRAX-562 emerging clinical data • Superior selectivity for disease-state NaV channel hyperexcitability • Unprecedented therapeutic window with potential for superior safety and efficacy • Convenient auto-titration regimen with stable PK 2022 Epilepsy Foundation Pipeline Conference 5 ©2020 Epilepsy F
Slide 6: PRAX-562 well tolerated in Phase 1 study in healthy adults
PRAX-562 well tolerated in Phase 1 study in healthy adults MAD Cohorts Only** Myoclonus • All TEAEs mild to Lethargy Hypoaesthesia moderate in severity Facial spasm Percentage of Dysgeusia • No drug-related SAEs or Disturbance in attention treatment emergent Vertigo severe AEs • No significant aberr
Slide 7: PRAX-562 in healthy volunteers safely exceeds projected therapeutic exposure
PRAX-562 in healthy volunteers safely exceeds projected therapeutic exposure EXPOSURES AT 28 DAYS, PRAX-562 EXPOSURES ON DAY 1 WELL ABOVE PREDICTED THERAPEUTIC EXPOSURE* Mean PRAX-562 120mg Mean PRAX-562 Concentration (ng/mL) 90mg Concentration (ng/mL) 10,000 45mg 90mg Cmax 30mg 10,000 90mg Ctrough
Slide 8: Three epilepsy drugs in clinic by end of 2022
Three epilepsy drugs in clinic by end of 2022 PRAX-562 PRAX-628 PRAX-222 (SCN2A, SCN8A, TSC) (FOCAL EPILEPSY) (SCN2A) Initiate Phase 2 Study: Initiate Phase 1 Study: Initiate Seamless Study: 2H22 4Q22 2H22* PRAX-222 and PRAX-562 received Orphan Drug Designations for severe pediatric epilepsy indicat
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