PBGM-01

Enrolled first FTD-GRN patient in Cohort 2 in upliFT-D trial Plan to present updated safety and biomarker data from Cohort 1 FTD-GRN patients treated with PBFT02 at 14th International Conference on Frontotemporal Dementias (ISFTD2024) in September 2024 Achieved alignment with Food and Drug Administration (FDA) on the company's proposed clinical development plans to expand the upliFT-D trial to assess PBFT02 in FTD patients with C9orf72 gene mutations Completed out-licensing of pediatric lysosomal storage disease programs to GEMMA Biotherapeutics Strong balance sheet to support continued execution, with cash runway to fund operations extended to the end of Q2 2026 PHILADELPHIA, Aug. 08, 2024 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (Nasdaq: PASG), a clinical stage genetic medicines company focused on improving the lives of patients with neurodegenerative diseases, today reported financial results for the second quarter ended June 30, 2024 and provided recent business highlights. “This has been a pivotal quarter for our company, marked by significant momentum in our upliFT-D trial, increased clarity around our strategy to expand PBFT02 into additional adult neurodegenerative indications, and out-licensing of our pediatric lysosomal storage disorder programs,” said Will Chou, M.D., president and chief executive officer of Passage Bio. “The positive recommendation of the IDMC to proceed to Cohort 2 dosing in our upliFT-D trial for FTD-GRN reinforces the well-tolerated safety profile of PBFT02 when combined with our revised immunosuppression regimen, and we are excited to present updated safety and biomarker data from Cohort 1 at the ISFTD2024 conference in Amsterdam this September. We are also pleased the FDA is aligned with our proposed expansion of our upliFT-D trial to include FTD-C9orf72 patients and expect to initiate dosing of these patients in the first half of 2025. The company remains on track to gain regulatory feedback on the pathway to treating ALS patients with PBFT02 in the second half of 2024.” Dr. Chou continued, "Furthermore, we completed the out-licensing of our pediatric lysosomal storage disease programs to GEMMA Biotherapeutics. Dr. Wilson and the entire GEMMABio team are committed to developing these promising therapies for pediatric patients with life-threatening conditions. We look forward to our continued research and collaboration efforts with their team, driving advancements and hope for those affected by neurodegenerative diseases.” Recent Highlights Enrolled first patient in Cohort 2 in upliFT-D trial for FTD-GRN: Following IDMC review of all available Cohort 1 safety data, the first patient in Cohort 2 has been enrolled in the trial; Cohort 2 will consist of three to five FTD-GRN patients to be treated with Dose 1. Dose 1 of PBFT02 continues to be well-tolerated in Cohort 1 patients who received the revised immunosuppression protocol (n=4). Recruitment efforts for subsequent Cohort 2 patients are well underway across seven clinical trial sites in Brazil, Canada, the United States and Europe. Plan to present updated safety and biomarker data from Cohort 1 patients in upliFT-D trial at ISFTD2024: Juan Chavez, M.D., vice president of clinical development at Passage Bio, will deliver updated data from Cohort 1 during an oral presentation, Interim Safety and Biomarker Data From upliFT-D Trial of PBFT02 in FTD with GRN Mutations, on Friday, September 20, 2024 at the 14th International Conference on Frontotemporal Dementias (ISFTD2024). Received FDA agreement on the company’s proposed expansion of the ongoing upliFT-D trial to treat FTD-C9orf72 patients with PBFT02: Substantial preclinical evidence, coupled with available safety and robust progranulin (PGRN) expression data from the first cohort of FTD-GRN patients treated with PBFT02, supported FDA alignment on the proposed trial expansion. The company intends to amend the protocol for the ongoing upliFT-D study to introduce a new population of FTD-C9orf72 patients and expects to initiate dosing of FTD-C9orf72 patients in 1H 2025. Completed out-licensing of pediatric lysosomal storage disease programs to GEMMA Biotherapeutics: Under the terms of the transaction, the company will receive initial payments of $10 million for the purchase of clinical product supply and up to an additional $10 million contingent on completion of certain GEMMABio business milestones. The company is also eligible to receive up to an additional $114 million in development and commercial milestones, as well as future royalties. GEMMA Biotherapeutics, a newly formed company co-founded by James M. Wilson, M.D., Ph.D., received an exclusive, worldwide license to PBGM01 for the treatment of GM1 gangliosidosis, PBKR03 for the treatment of Krabbe disease, and PBML04 for the treatment of metachromatic leukodystrophy. GEMMA Biotherapeutics is responsible for all future development of, and any remaining financial obligations owed to the University of Pennsylvania for the licensed programs. Entered into research, collaboration, and license agreement with GEMMA Biotherapeutics: Following a restructuring of the company’s original agreement with the University of Pennsylvania’s Gene Therapy Program, a research, collaboration, and license agreement with GEMMA Biotherapeutics was finalized. The partnership will focus on continued execution of preclinical studies in Huntington's disease, and the company will retain rights to future options in the CNS field, ensuring continued strategic growth and innovation in these critical areas. Anticipated Upcoming Milestones: FTD-GRN Report updated safety and biomarker data from Cohort 1 patients at ISFTD2024 in September 2024Report 12-month Cohort 1 and interim Cohort 2 data in 1H 2025Seek regulatory feedback on pivotal trial design in 2H 2025 FTD-C9orf72 and ALS Obtain regulatory feedback on the pathway to treating amyotrophic lateral sclerosis (ALS) patients with PBFT02 in 2H 2024Initiate dosing of FTD-C9orf72 patients in 1H 2025 Second Quarter 2024 Financial Results Cash Position: Cash, cash equivalents and marketable securities were $91.8 million as of June 30, 2024, as compared to $151.5 million as of June 30, 2023. The company expects current cash, cash equivalents and marketable securities, together with the initial payments from our out-licensing agreements with Gemma Biotherapeutics, to fund operations to the end of Q2 2026.Research and Development (R&D) Expenses: R&D expenses were $10.4 million for the quarter ended June 30, 2024, as compared to $17.3 million for the quarter ended June 30, 2023.General and Administrative (G&A) Expenses: G&A expenses were $6.5 million for the quarter ended June 30, 2024, as compared to $8.1 million for the quarter ended June 30, 2023.Net Loss: Net loss was $16.0 million, or $0.26 per basic and diluted share, for the quarter ended June 30, 2024, as compared to a net loss of $23.9 million, or $0.44 per basic and diluted share, for the quarter ended June 30, 2023. About Passage Bio Passage Bio (Nasdaq: PASG) is a clinical stage genetic medicines company on a mission to improve the lives of patients with neurodegenerative diseases. Our primary focus is the development and advancement of cutting-edge, one-time therapies designed to target the underlying pathology of these conditions. Passage Bio’s lead product candidate, PBFT02, seeks to treat neurodegenerative conditions, including frontotemporal dementia, by elevating progranulin levels to restore lysosomal function and slow disease progression. To learn more about Passage Bio and our steadfast commitment to protecting patients and families against loss in neurodegenerative conditions, please visit: www.passagebio.com. Forward-Looking Statements This press release contains “forward-looking statements” within the meaning of, and made pursuant to the safe harbor provisions of, the Private Securities Litigation Reform Act of 1995, including, but not limited to: our expectations about timing and execution of anticipated milestones, including the initiation of dosing of FTD-C9orf72 patients, feedback from regulatory authorities, the progress of clinical studies and the availability of clinical data from such trials; our expectations about our collaborators’ and partners’ ability to execute key initiatives; our ability to receive milestone payments from our partners; our expectations about cash runway; and the ability of our product candidates to treat their respective target CNS disorders. These forward-looking statements may be accompanied by such words as “aim,” “anticipate,” “believe,” “could,” “estimate,” “expect,” “forecast,” “goal,” “intend,” “may,” “might,” “plan,” “potential,” “possible,” “will,” “would,” and other words and terms of similar meaning. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including: our ability to develop and obtain regulatory approval for our product candidates; the timing and results of preclinical studies and clinical trials; risks associated with clinical trials, including our ability to adequately manage clinical activities, unexpected concerns that may arise from additional data or analysis obtained during clinical trials, regulatory authorities may require additional information or further studies, or may fail to approve or may delay approval of our drug candidates; the occurrence of adverse safety events; the risk that positive results in a preclinical study or clinical trial may not be replicated in subsequent trials or success in early stage clinical trials may not be predictive of results in later stage clinical trials; failure to protect and enforce our intellectual property, and other proprietary rights; our dependence on collaborators and other third parties for the development and manufacture of product candidates and other aspects of our business, which are outside of our full control; risks associated with current and potential delays, work stoppages, or supply chain disruptions; and the other risks and uncertainties that are described in the Risk Factors section in documents the company files from time to time with the Securities and Exchange Commission (SEC), and other reports as filed with the SEC. Passage Bio undertakes no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise. Passage Bio, Inc. Balance Sheets (Unaudited) (in thousands, except share and per share data) June 30, 2024 December 31, 2023Assets Current assets: Cash and cash equivalents $24,770 $21,709 Marketable securities 67,003 92,585 Prepaid expenses and other current assets 1,397 923 Prepaid research and development 1,830 2,742 Total current assets 95,000 117,959 Property and equipment, net 13,054 15,295 Right of use assets - operating leases 16,822 16,858 Other assets 516 433 Total assets $125,392 $150,545 Liabilities and stockholders’ equity Current liabilities: Accounts payable $621 $1,298 Accrued expenses and other current liabilities 8,063 11,670 Operating lease liabilities 3,701 3,373 Total current liabilities 12,385 16,341 Operating lease liabilities - noncurrent 22,450 22,921 Total liabilities 34,835 39,262 Stockholders’ equity: Preferred stock, $0.0001 par value: 10,000,000 shares authorized; no shares issued and outstanding at both June 30, 2024 and December 31, 2023 — — Common stock, $0.0001 par value: 300,000,000 shares authorized; 61,754,786 shares issued and outstanding at June 30, 2024 and 54,944,130 shares issued and outstanding at December 31, 2023 6 5 Additional paid‑in capital 717,788 705,789 Accumulated other comprehensive income (loss) (67) (43)Accumulated deficit (627,170) (594,468)Total stockholders’ equity 90,557 111,283 Total liabilities and stockholders’ equity $125,392 $150,545 Passage Bio, Inc. Statements of Operations and Comprehensive Loss (Unaudited) Three Months Ended June 30, Six Months Ended June 30, (in thousands, except share and per share data) 2024 2023 2024 2023Operating expenses: Research and development $10,430 $17,324 $21,965 $34,160 General and administrative 6,510 8,064 13,025 27,111 Impairment of long-lived assets 438 — 438 — Loss from operations (17,378) (25,388) (35,428) (61,271)Other income (expense), net 1,387 1,532 2,726 3,077 Net loss $(15,991) $(23,856) $(32,702) $(58,194)Per share information: Net loss per share of common stock, basic and diluted $(0.26) $(0.44) $(0.55) $(1.06)Weighted average common shares outstanding, basic and diluted 61,682,475 54,683,817 58,989,007 54,651,488 Comprehensive loss: Net loss $(15,991) $(23,856) $(32,702) $(58,194)Unrealized gain (loss) on marketable securities 2 87 (24) 626 Comprehensive loss $(15,989) $(23,769) $(32,726) $(57,568) For further information, please contact: Investors: Stuart Henderson Passage Bio 267.866.0114 shenderson@passagebio.com Media: Mike Beyer Sam Brown Inc. Healthcare Communications 312.961.2502 MikeBeyer@sambrown.com

▎药明康德内容团队编辑 基因疗法先驱James Wilson博士创建新锐公司，引进多款儿科基因疗法 日前，基因疗法先驱，宾夕法尼亚大学基因治疗项目（GTP）主任James Wilson博士宣布将成立新锐公司，致力将宾夕法尼亚大学GTP过去30多年内取得的突破性发现转化为有效的治疗方法，并能够为全球患者使用。Wilson博士将辞去基因治疗项目主任的职务，专注于这些新公司。其中，GEMMA Biotherapeutics是一家新的基因疗法开发公司，专注于研发针对罕见疾病患者的先进疗法。Wilson博士将担任该公司的首席执行官。 今日，Passage Bio宣布已与GEMMA Biotherapeutics签署一系列协议，将其三个处于临床阶段的儿科溶酶体贮积症项目授权给GEMMA Biotherapeutics，包括用于治疗GM1神经节苷脂病的PBGM01，用于治疗Krabbe病的PBKR03，以及用于治疗异染性脑白质营养不良的PBML04。 根据授权协议的条款，Passage Bio公司将收到1000万美元的初始付款。此外，该公司还有资格获得高达1.14亿美元的开发和商业里程碑付款。 囊获潜在“first-in-class”口服疗法，大冢制药8亿美元收购Jnana 大冢制药（Otsuka Pharmaceutical）和Jnana Therapeutics今日宣布，双方已签署一份最终的合并协议，大冢将斥资8亿美元收购Jnana，并可能支付高达3.25亿美元的额外开发和监管里程碑付款。 Jnana的药物发现方法是基于其下一代化学蛋白质组学平台RAPID。该平台设计用于针对难以成药靶点的药物。RAPID利用高通量、基于结合的筛选方法，灵活地在靶蛋白表面发现结合位点，并识别出能引发多种药理作用的小分子。基于这一平台开发的JNT-517是一种SLC6A19别构小分子抑制剂。SLC6A19是一个调节肾脏中氨基酸再吸收的溶质载体。JNT-517有望成为一种潜在“first-in-class”苯丙酮尿症口服疗法，它在1b/2期临床研究中表现出疗效，并且耐受性和安全性良好。 1.44亿美元助力新一代细胞疗法开发 Outpace Bio今天宣布完成超额认购的B轮融资，筹集1.44亿美元。获得的资金将用于推进多种T细胞候选疗法，以期在实体肿瘤治疗中获得早期临床概念验证。 该公司的主打项目OPB-101是一种针对间皮素的嵌合抗原受体（CAR）T细胞，利用Outpace的专有技术OUTSMART、OUTLAST、OUTSPACER和OUTSAFE进行增强，计划于2025年获得IND许可并首次在晚期铂耐药性上皮性卵巢癌、输卵管癌或原发性腹膜癌患者进行给药。此外，这些资金还将支持Outpace利用其模块化技术平台扩展未来的产品线。 ▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放 参考资料： [1] Outpace Bio Announces $144M Oversubscribed Series B Financing to Advance AI-powered Cell Therapies into Clinical Development. Retrieved August 1, 2024, from https://www.outpacebio.com/news/outpace-bio-announces-144m-oversubscribed-series-b-financing-to-advance-ai-powered-cell-therapies-into-clinical-development [2] To Further Advance Gene Therapy Research, Industry Pioneer Jim Wilson is Forming Two Independent Companies Dedicated to Genetic Medicine. Retrieved August 1, 2024, from https://www.prnewswire.com/news-releases/to-further-advance-gene-therapy-research-industry-pioneer-jim-wilson-is-forming-two-independent-companies-dedicated-to-genetic-medicine-302211669.html [3] Passage Bio Out-licenses Three Pediatric Gene Therapy Programs to GEMMA Biotherapeutics and Enters New Research Collaboration. Retrieved August 1, 2024, from https://finance.yahoo.com/news/passage-bio-licenses-three-pediatric-110000481.html [4] Otsuka Pharmaceutical to Acquire Jnana Therapeutics Inc. Retrieved August 1, 2024, from https://www.jnanatx.com/otsuka-pharmaceutical-to-acquire-jnana-therapeutics-inc/?pag=1 免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。 版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。 分享，点赞，在看，聚焦全球生物医药健康创新