Risk Factors Update Summary

• FDA may mandate Phase 4 clinical trials as a condition of NDA approval.
• Added the possibility of paying treble damages and attorneys' fees in intellectual property litigation.
• Challenges in enrolling and retaining patients in XEN1101 Phase 3 epilepsy clinical trials.
• Expanded pipeline to include XEN496 for KCNQ2 developmental and epileptic encephalopathy, with topline results reported.
• Payers may limit coverage for drug products, affecting reimbursement rates, potentially impacting revenue generation.
• Uncertainty in intellectual property litigation outcomes, which could limit research and development activities and commercialization efforts.
• Risks related to intellectual property rights, including challenges in obtaining and maintaining adequate patent protection.
• New chemical entities receive 8 years of data exclusivity and an additional 2 years of marketing exclusivity.
• Initiated Phase 3 clinical trial X-ACKT for primary generalized tonic-clonic seizures, evaluating 25mg XEN1101.
• Changes in healthcare laws, like the Inflation Reduction Act of 2022, could impact drug pricing.
• Phase 2 clinical trial results for XEN1101 in patients with MDD were positive.
• Mentioned the potential loss of exclusivity of licenses, which could significantly harm business operations.
• Competing for clinical trial sites with limited patient populations, especially in ultra-orphan and orphan indications.
• Potential costly litigation and diversion of resources in defending against claims of patent infringement or violations of intellectual property rights.
• Highlighted the importance of maintaining trademarks to build name recognition in potential markets.
• Legislative challenges, like ongoing health care reform efforts, may affect drug pricing and reimbursement.
• Positive Phase 2 results for XEN496 in KCNQ2-DEE, showing a dose response with a 3.04 difference.
• Orphan products can obtain an additional 2 years of market exclusivity for completing pediatric studies.
• Collaboration with Neurocrine Biosciences for NBI-921352, with Phase 2 trial results in adult focal epilepsy.
• Discussed the impact of intellectual property litigation on reputation and market price of common shares.
• Potential delays in completing clinical trials due to challenges in identifying suitable patients.
• Potential impact from state-level initiatives, such as drug price transparency laws, on product commercialization.
• Pediatric clinical trials conducted in accordance with a Pediatric Investigation Plan are eligible for a 6-month supplementary protection certificate extension.
• Increased focus on privacy and security laws, including data protection regulations, may affect operations.
• Noted the uncertainty and potential adverse effects of conflicts in Ukraine, Russia, and the Middle East.
• Drugs designated as breakthrough therapies are eligible for accelerated approval and priority review.
• Intellectual property expansion with new patents issued in 2021 and 2023, covering XEN1101 and related compounds.
• Risks associated with international operations, including regulatory differences and geopolitical factors, could impact business.
• Addressed the risk of market-wide liquidity problems due to adverse developments affecting financial institutions.
• Fast Track designation expedites the FDA review process but does not guarantee approval.
• Ongoing Phase 2 study of XEN1101 for MDD, showing early efficacy and statistically significant reductions in MADRS and SHAPS.
• Compliance with evolving laws and regulations, such as anti-kickback statutes and transparency laws, is crucial.
• Mentioned ongoing rulemaking efforts by regulators, including those related to environmental, social, and governance matters.
• The Clinical Trials Regulation harmonizes procedures for clinical drug trials in the EU.
• Potential competition in epilepsy treatment from various companies developing product candidates for epilepsy and MDD.