Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 10-Q (Mark One) ⌧ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2020 OR ◻ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ________ to ________ Commission File Number: 001-39231 PASSAGE BIO, INC. (Exact Name of Registrant as Specified in its Charter) Delaware 82-2 ...

业绩总结 - 截至2020年3月31日，公司现金余额为3.668亿美元，预计可支持运营至2023年[60] - 2020年3月，公司通过首次公开募股（IPO）筹集了净收益2.275亿美元[60] 用户数据 - PBGM01针对婴儿型GM1神经节苷脂症，预计全球发病率为每10万活产中0.5至1例[16] - PBFT02针对前额叶痴呆，预计影响美国约3000至6000名患者[36] 新产品和新技术研发 - Passage Bio计划在2020年第二季度提交PBGM01的IND，并在2020年第四季度启动临床试验[10] - 预计在2021年上半年启动针对前额叶痴呆（FTD）和克拉贝病的PBFT02和PBKR03的临床试验[62] - PBGM01在小鼠模型中显示出剂量依赖性的生存率改善，最高两剂量达到100%生存率[25] - PBGM01在GLB1基因敲除小鼠模型中显示出脑、脑脊液和外周组织中β-半乳糖苷酶活性的显著增加[67] - PBKR03在自然发生的克拉贝犬模型中显示出完全的表型恢复，体重和神经传导速度与野生型犬相当[57] - PBKR03治疗在自然发生的Twitcher小鼠和犬模型中显示出脑和外周器官GALC活性增加[90] 临床试验和市场扩张 - 预计PBGM01在2020年第四季度启动针对婴儿GM1的1/2期试验[61] - PBKR03的临床试验设计为多中心、开放标签、单臂、剂量递增研究，预计持续5年[58] - PBFT02在临床试验中将使CSF中的PGRN浓度提高超过正常人类CSF浓度的50倍[35] 合作与战略 - Passage Bio与宾夕法尼亚大学的基因治疗项目和孤儿疾病中心建立了合作关系，以提高技术和监管成功的概率[4] - 公司在罕见疾病和基因治疗领域拥有深厚的领导经验[6] 负面信息 - AAV-GRN治疗的KO小鼠在所有脑区的脂褐素沉积显著减少，p值小于0.0001[79] - AAV1对脑室上皮细胞的转导率为48%，显著高于AAVhu68的1-2%[86]

Passage Bio, Inc. (NASDAQ:PASG) Q1 2020 Earnings Conference Call May 11, 2020 8:30 AM ET Company Participants Zoe Mita - IR, Stern Investor Relations, Inc. Jill Quigley - COO Bruce Goldsmith - President and CEO Rich Morris - CFO Gary Romano - Chief Medical Officer Conference Call Participants Andrea Tan - Goldman Sachs Brendan Smith - Cowen and Company Anupam Rana - JPMorgan Gbola Amusa - Chardan Capital Markets Operator Ladies and gentlemen, thank you for holding. Good morning and welcome to the Passage Bi ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 10-Q (Mark One) ⌧ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 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 ________ Commission File Number: 377-02943 PASSAGE BIO, INC. (Exact Name of Registrant as Specified in its Charter) Delaware 82- ...