Pipeline • Top-line Phase 1 data demonstrate extended-release PHVS719 suitable for once-daily dosing. The Phase 1 pharmacokinetics (PK) study of PHVS719 included 8 healthy volunteers dosed in an open-label • Phase 1 pharmacokinetics study demonstrates PHVS719 well tolerated with extended-release profile supporting once-daily dosing • Orphan Drug Designation for PHA121 granted by FDA • RAPIDe-1, Phase 2 on-demand study of PHVS416 for the treatment of HAE attacks ongoing; top-line data anticipated in 4Q22 • C ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 20-F (Mark One) ☐ REGISTRATION STATEMENT PURSUANT TO SECTION 12(b) OR (g) OF THE SECURITIES EXCHANGE ACT OF 1934 OR ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2021 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to . OR ☐ SHELL COMPANY REPORT PURSUANT TO SE ...

公司概况 - 公司专注遗传性血管性水肿（HAE）及其他缓激肽介导疾病治疗，全球HAE市场规模超20亿美元，预计5年复合年增长率13%[6][7] - 公司财务状况良好，截至2021年12月31日现金达2.094亿欧元，可支撑至2024年第一季度初[7] 疾病特征 - HAE是罕见终身疾病，全球每1万至5万人中有1人患病，美国至少6600例，欧洲至少8900例，且全球存在诊断和治疗不足情况[25] - HAE发作不可预测、使人虚弱且可能致命，患者每年发作中位数为14次，女性19次，男性9次[28][29] 市场情况 - HAE市场有价值且在增长，2018年市场规模超20亿美元，年增长率约13%，多数获批产品为注射剂，患者对口服疗法有需求[40] 产品策略 - 公司开发两款口服产品PHVS416和PHVS719，均采用PHA121活性成分，分别用于按需治疗和预防HAE发作[43][45] 产品优势 - PHA121对缓激肽B2受体的抑制效力比依卡替班强25倍，口服PHA121在临床前体内研究中抑制缓激肽挑战的持续时间更长、起效更快[56][77] - PHA121在1期试验中耐受性良好，药代动力学呈剂量比例关系，半衰期约3.4 - 5.6小时，比依卡替班长约3倍[58][63] 临床研究 - PHVS416按需治疗HAE的RAPIDe - 1研究和预防HAE的CHAPTER - 1研究预计2022年第四季度公布2期顶线数据[49][51][96][112] - PHVS719的1期药代动力学研究预计2022年第一季度公布顶线结果，旨在评估每日一次给药的潜力[117]

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 20-F (Mark One) ☐ REGISTRATION STATEMENT PURSUANT TO SECTION 12(b) OR (g) OF THE SECURITIES EXCHANGE ACT OF 1934 OR ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2020 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to . OR ☐ SHELL COMPANY REPORT PURSUANT TO SE ...