Positive results from preliminary analysis of PRAX-222 Part 1 showed 44% median reduction in seizures after three doses for SCN2A-gain-of-function pediatric patients
Phase 3 (Essential3) program for ulixacaltamide initiating this quarter with mADL11 as primary endpoint; nearly 600 patients have already expressed interest in participating
Ulixacaltamide
- Additional analysis from the Essential1 data set continues to support the design of the Phase 3 program for ulixacaltamide (Essential3)
- Data from Essential1 showed a meaningful difference in a minimum 3-point improvement in the modified Activities of Daily Living 11-point scale (mADL11) between patients on ulixacaltamide v. placebo (55% v. 31%, p=0.023)
- In the extension period of Essential1, which continued after Week 8 through Week 14, 64% of patients in the ulixacaltamide arm showed at least a 3-point improvement in mADL11 versus baseline. For patients transitioning from placebo at Week 8 onto ulixacaltamide, 69% achieved at least a 3-point improvement to baseline at Week 14
- Ulixacaltamide also demonstrated incremental benefit to patients on propranolol. 48% of patients on propranolol and ulixacaltamide in the Essential1 study achieved at least a 3-point improvement in mADL11 compared to 25% for patients on propranolol and placebo
- mADL11 confirmed to serve as primary endpoint in both Phase 3 studies of Essential3 based on protocol feedback received from the U.S. Food and Drug Administration (FDA)
- In a pre-recruitment observational study launched in September 2023, nearly 600 essential tremor patients have already expressed interest in joining a Phase 3 trial sponsored by Praxis
- Praxis expects to submit a New Drug Application (NDA) for ulixacaltamide in 2025
PRAX-222
- Dosing for Part 1 of the EMBRAVE study is nearing completion, with patients receiving 1 mg doses once a month for four months at Le Bonheur Children’s Hospital in Memphis, Tennessee
- No treatment related adverse events (AEs) or serious adverse events (SAEs) were observed in preliminary safety analysis as of the cutoff date of September 26, 2023
- As of the cutoff date, data was evaluable for three of four dosing periods showing:
- Patients achieved a 44% median reduction in seizures versus baseline, on top of best available standard of care
- Patients observed an increased number of days without seizures, achieving a median of 35% seizure free days over the dosing period compared to a baseline of 21% seizure free days
- All patients achieved significant seizure reduction after one dose
- Praxis intends to request a meeting in the fourth quarter of 2023 with the FDA to align on next steps for the program
PRAX-628
- Additional data from the Phase 1 single ascending dose (SAD) and multiple ascending dose (MAD) study continue to reinforce the potential of PRAX-628 as a next generation precision anti-seizure medication (ASM)
- Electroencephalogram (EEG) data showed rapid and sustained activity in the brain when dosing PRAX-628 as compared to placebo
- PRAX-628 achieved and sustained target therapeutic concentrations in excess of the equivalent Maximal Electroshock Seizure Model (MES) EC50 after first administration
- Praxis expects to read out the results from the Phase 2 Photoparoxysmal Response (PPR) study in the fourth quarter of 2023
PRAX-562
- Praxis is utilizing a decentralized recruiting and enrollment approach for its Phase 2 EMBOLD study for the treatment of pediatric patients with DEEs, with sites in both the U.S. and Europe
- Praxis expects topline results from the EMBOLD study in the first half of 2024
