Risk Factors Update Summary

• Several post-marketing authorization measures were added, including submitting various study reports and conducting toxicity studies.
• Monetized Rare Pediatric Disease Priority Review Voucher for $110.0 million in cash.
• Failure to comply with Medicaid Drug Rebate Program reporting could result in civil monetary penalties.
• Received a second patent for SE on June 20, 2023, strengthening intellectual property portfolio.
• The company may face substantial monetary awards and loss of revenue if regulatory authorities approve competing products.
• Identified substantial doubt about ability to continue as a going concern due to limited revenue.
• Weight of evidence assessments requested by EMA for carcinogenicity and juvenile toxicity studies.
• The company had an accumulated deficit of $571.9 million as of December 31, 2023.
• Implementing additional measures to ensure personal data protection equivalent to the EU GDPR.
• Filed petition for post-grant review of Ovid '817 Patent, with PTAB granting institution.
• Added commercialization in the EU for ZTALMY, with FDA and EMA regulatory requirements.
• Increased full-time employees from 151 to 165, a significant 14 employee rise.
• Planned initiation of RAISE II trial in the second half of 2023, targeting enrollment of approximately 70 patients.
• FDA approval for ZTALMY in the EU for CDD, with marketing authorization granted by the EC.
• Reduced part-time employees from two to one, streamlining workforce efficiency.
• Expanded commercial launch plans for ZTALMY in Europe with Orion Corporation collaboration.
• Expanded RAISE trial eligibility criteria to include patients previously treated with high-dose IV anesthesia.
• The European Commission adopted a draft adequacy decision for the EU - U.S. Data Privacy Framework.
• Addition of a 26-week Oral Gavage Carcinogenicity Study for ganaxolone and M2.
• The company plans to seek regulatory approval for ganaxolone in several other geographic regions.
• Adjusted titration schedule for Phase 3 TSC trial to optimize tolerability.
• Potential increase in ownership concentration from 37% to 55%, impacting stock price.
• Expanded investment in ZTALMY for rare epilepsies, planning Phase 2 trial in LGS.
• The company has identified conditions raising substantial doubt about its ability to continue as a going concern.
• Entered exclusive distribution agreement with Biologix, received upfront payment of $0.5 million.
• Agreed to pay Purdue $4 million in respect of claim, accrued liability recorded in 2023.
• Renewed SME designation in the EU, anticipating further state and federal healthcare reform measures.