证 券 研 究 报 告 赛道百花齐放，潜力靶点或迎新突破 ——癫痫药物行业深度报告 证券分析师： 张静含 A0230522080004 凌静怡 A0230522060001 2025.6.17 投资案件 www.swsresearch.com 证券研究报告 2 ◼ 癫痫是神经系统常见疾病，患者人数众多。根据《柳叶刀-公共卫生》数据统计：2021年，全球有 5170万人患有癫痫，年龄标准化患病率为658/10万人。据中国抗癫痫协会数据，中国约有900万左 右的癫痫患者，其中500-600万是活动性癫痫患者，同时每年新增加癫痫患者约40万，在中国癫痫 已经成为神经科仅次于头痛的第二大常见病。 ◼ 癫痫的发病机制复杂多样，存在较大治疗缺口。癫痫发病机制复杂多样，包括离子通道异常、中枢 神经系统兴奋性/抑制性神经递质分泌不平衡、神经环路异常，以及神经炎症和神经胶质细胞异常等， 抗癫痫发作药物（ASMs）是目前最重要和最基本的治疗手段，但疗效有限、副作用明显等问题依然 影响着癫痫患者的治疗效果。我国癫痫病人经单药序贯或多药联合治疗后，仍有30%的癫痫患者发 作无法得到有效控制，治疗存在着较大缺口。 ◼ 抗癫痫药物在研管 ...

Summary of Xenon Pharmaceuticals Conference Call Company Overview - **Company**: Xenon Pharmaceuticals - **CEO**: Ian Mortimer Industry Context - **Industry**: Epilepsy treatment - **Market Size**: Approximately 3 million Americans have epilepsy, with 60% experiencing focal onset seizures, translating to about 2 million patients. 30-50% of these patients do not achieve good seizure control, indicating a significant market opportunity for new treatments [30][31]. Key Points and Arguments Clinical Trials and Drug Development - **Phase III Studies**: Xenon is conducting two Phase III clinical trials named XTOL-2 and XTOL-3, designed to mirror the successful Phase II study in terms of size, inclusion criteria, and dosing [6][7][9]. - **Efficacy Data**: The Phase II data for Ezetucalner showed robust efficacy, with a placebo-adjusted efficacy that is the best seen in focal onset seizures. The drug has a high power of over 99% at the primary endpoint in Phase III [4][9]. - **Enrollment Challenges**: There has been a slight delay in enrollment for XTOL-2, but the company is confident in completing enrollment in the coming months, with data expected in early 2026 [15][17]. Safety and Efficacy Profile - **Long-term Data**: Over 150 patients have been on the drug for more than three years, with a one in three chance of being seizure-free for 12 months or more. This is significant given the baseline characteristics of patients who had previously failed multiple treatments [22][23]. - **Adverse Events**: The safety profile is consistent with other anti-seizure medications, with common CNS adverse events like dizziness and fatigue. No significant issues related to pigmentation or urinary retention have been observed, addressing concerns from previous drugs in the same class [25][27][28]. Market Opportunity and Competitive Landscape - **Polypharmacy**: The treatment landscape for epilepsy often involves polypharmacy, with many patients not achieving adequate seizure control. Ezetucalner is positioned as a second or third-line treatment option [32][33]. - **Comparison with Competitors**: Ezetucalner offers advantages over competitors like XCOPRI, including no titration required and early onset of efficacy. XCOPRI is projected to generate $400-$450 million in sales this year, with expectations of reaching $1 billion by the end of the decade [34][35][36]. Future Directions - **Major Depressive Disorder (MDD)**: Xenon is also exploring Ezetucalner for MDD, with a Phase III program initiated. Initial data from a small investigator-sponsored study showed some treatment effects, but the company is focusing on its larger Phase III studies for more definitive results [37][41]. - **Pipeline Development**: The company is excited about its drug development pipeline, including new targets for pain and other syndromes, with plans for investor webinars to discuss these developments [52][53]. Additional Important Insights - **Regulatory Interactions**: Ongoing interactions with the FDA have not indicated any changes in the regulatory landscape, and the company expects more engagement as it approaches the NDA submission [19]. - **Patient Feedback**: Positive anecdotal feedback from physicians indicates that patients are experiencing improved quality of life, including increased independence and social interactions due to better seizure control [24]. This summary encapsulates the key points discussed during the conference call, highlighting the company's strategic focus, clinical trial progress, market positioning, and future opportunities.