ZTALMY® Clinical and Commercial Overview - ZTALMY® is FDA-approved for CDKL5 Deficiency Disorder (CDD) and has a differentiated mechanism of action addressing an unmet need[12, 13] - In a Phase 3 CDD trial, patients taking ganaxolone experienced a median reduction of 30.7% in monthly major motor seizures compared to 6.9% for placebo[15] - The most frequent adverse events reported during the double-blind phase were somnolence (36% vs 16%), pyrexia (18% vs 8%), and upper respiratory tract infection (10% vs 6%)[18] - Net product revenue for ZTALMY® in Q3 2024 was $8.5 million, a 56% increase from Q3 2023, with full year 2024 expected net product revenue between $33 million and $34 million[20] Ganaxolone Prodrug Program - The company is developing a second-generation ganaxolone prodrug with the goal of increasing efficacy, improving tolerability, and reducing dosing frequency[40] - Early PK work supports once-daily (QD) or twice-daily (BID) dosing and the ability to achieve higher steady-state serum concentrations with the prodrug[50] RAISE Trial in Refractory Status Epilepticus - In the RAISE trial, 13% of patients in the IV Ganaxolone arm experienced SE cessation within 30 minutes compared to 0% in the placebo arm (p<0.0001)[63] - 80% of patients in the IV Ganaxolone arm had no progression to IV anesthesia for 36 hours compared to 63% in the placebo arm (p=0.1619)[63] - The median percent reduction in EEG seizure burden from hours 1-36 was 93% for Ganaxolone compared to 36% for placebo (p=0.003)[66] Financial Status - As of September 30, 2024, the company had $42.2 million in cash and cash equivalents, providing a cash runway into Q2 2025[74] - Full year 2024 SG&A and R&D expenses are guided to be between $135 million and $138 million, including approximately $20 million in stock-based compensation[69]