 Submitted IND for PBGM01 Phase 12 trial; currently on FDA clinical hold pending additional review of proposed ICM delivery device Anticipate dosing first patient in PBGM01 Phase 12 trial in late 4Q2020 or early 1Q2021 Initial PBGM01 clinical safety and biomarker data remains on track for 1H2021 read out Strong cash balance of 353M expected to fund operations into 2023 Management to host conference call today at 8:30 a.m. ET PHILADELPHIA, Aug. 13, 2020 GLOBE NEWSWIRE -- Passage Bio, Inc. Nasdaq: PASG, a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system disorders, today reported financial results for the second quarter ended June 30, 2020 and provided recent business highlights.We have made substantial progress over the past quarter, including the submission of our first IND application to the FDA for a Phase 12 clinical trial in infantile GM1 patients with PBGM01, demonstrating our teams ability to work collaboratively with our partners at the Gene Therapy Program led by Dr. James Wilson, said Gary Romano, M.D., Ph.D, chief medical officer of Passage Bio. We are confident that we can efficiently and successfully address the FDA clinical hold questions related to biocompatibility of our proposed ICM delivery device so that we can begin to dose patients before the end of this year or early next year. Importantly, we continue to believe that the initial clinical safety and biomarker data from this trial will be available late in the first half of 2021.Bruce Goldsmith, Ph.D., president and chief executive officer of Passage Bio, said: I am proud of the world-class team we are building at Passage Bio, including substantial expansion of the clinical, manufacturing, and corporate operation teams. We have also been effective at navigating the unpredictable environment caused by COVID-19 both internally and with our key external partners to maintain our pipeline advancement. This has enabled us to make significant progress toward delivering on the promise of PBGM01 for patients suffering from GM1. We look forward to treating our first patient as well as demonstrating the potential of all our investigational product candidates as safe and effective treatment options for devastating rare diseases like GM1. With our operational progress and the continued strength of our balance sheet, together with our robust pipeline, we are well positioned to achieve these goals.Recent Business Highlights Received feedback from U.S. Food and Drug Administration FDA on Investigational New Drug IND application for PBGM01 In June 2020, Passage Bio submitted its first ever IND for PBGM01 for the treatment of GM1 gangliosidosis to the FDA in collaboration with the University of Pennsylvanias Gene Therapy Program. Following this submission, the Company was notified that the IND was placed on clinical hold pending additional risk assessments of the biocompatibility of the proposed ICM delivery device. The ICM route of administration delivers PBGM01 directly to the brain into the cisterna magna, a space within the lower portion of the brain, with techniques and delivery devices commonly used both in current medical practice and other clinical trials, including those for gene therapy. The Company is evaluating options for conducting additional risk assessments while it awaits official written feedback from the FDA. During the IND review, the Company addressed specific clinical and protocol questions raised by the FDA, and the agency confirmed that there are no further clinical information requests. As a result of the clinical hold, the Company now expects to initiate dosing of its Phase 12 trial late in the fourth quarter of 2020 or early in the first quarter of 2021 and remains on track to report initial 30 day safety and biomarker data late in the first half of 2021. Added to Russell 2000 Index In June 2020, Passage Bio was added to the Russell 2000 Index, a subset of the Russell 3000 Index, which measures the performance of the small-cap segment of the U.S. equity market. Expanded gene therapy collaboration with the University of Pennsylvania UPenn In May 2020, Passage Bio expanded its research and development collaboration and licensing agreement with the University of Pennsylvania. The amendment increased the number of remaining available licensing options for programs to treat rare monogenic CNS disorders from six to eleven and extended the window for the exercise of options by three years. Accordingly, the window to exercise all eleven remaining options extends to May 2025. The Company also received exclusive rights and licenses, subject to limitations, to certain technologies resulting from discovery research at Gene Therapy Program GTP for Passage Bio products developed with GTP, such as novel capsids, toxicity reduction technologies and delivery and formulation improvements. Announced presentation of preclinical data models of Krabbe Disease at American Society of Gene Cell Therapy ASGCT 23rd Annual Meeting In May 2020, the University of Pennsylvanias Gene Therapy Program, Passage Bios preclinical development partner, presented encouraging preclinical data from a Krabbe dog and Twitcher mouse model at the ASGCT Annual Meeting demonstrating the potential of an AAVhu68 capsid carrying a functional GALC gene to normalize GALC enzyme activity and nerve conduction, leading to dose-dependent phenotypic correction and increased survival. Passage Bio is currently developing an AAVhu68 GALC therapeutic, PBKR03, for the treatment of infantile Krabbe disease and expects to initiate a Phase 12 trial in the first half of 2021. Granted Orphan Drug and Rare Pediatric Disease designation by FDA for PBGM01 In April and May 2020, respectively, the FDA granted Orphan Drug and Rare Pediatric Disease designation to PBGM01 for the treatment of infantile GM1. Orphan Drug designation is intended to promote the development of safe and effective treatments for rare diseases through certain financial and market exclusivity incentives, and Rare Pediatric Disease designation is intended to encourage treatments for life-threatening disease affecting children of 18 years of age or younger by qualifying the sponsor for a priority review voucher upon approval that may be sold or transferred.Anticipated Upcoming Milestones Initiate a Phase 12 trial for the lead program, PBGM01, for the treatment of patients with infantile GM1 late in the fourth quarter of 2020 or early in the first quarter of 2021. Report initial 30-day safety and biomarker data late in the first half of 2021. Continue to advance lead programs PBFT02 for the treatment of frontotemporal dementia FTD and PBKR03 for the treatment of Krabbe disease toward clinical trial initiations in the first half of 2021. Continue to advance PBML04, PBLA05 and PBCM06 toward IND-enabling studies.Second Quarter 2020 Financial Results Cash Position: Cash and cash equivalents were 353.4 million as of June 30, 2020 as compared to 158.9 million as of December 31, 2019. Research and Development RD Expenses: RD expenses were 19.9 million for the quarter ended June 30, 2020, compared to 6.3 million for the same quarter in 2019. The increase was primarily due to an increase of 4.5 million in costs incurred with the University of Pennsylvania in preparation for several IND filings, an increase of 4.0 million in clinical manufacturing costs, a 1.4 million increase in clinical development costs and a 0.5 million increase in consulting expense as we prepare for our clinical trials to begin in the second half of 2020 and early 2021. The Company also had a 3.1 million increase in personnelrelated costs and a 0.1 million increase in facility and other costs due to increases in employee headcount in the RD function. General and Administrative GA Expenses: GA expenses were 7.4 million for the quarter ended June 30, 2020, compared to 1.0 million for the same quarter in 2019. The increase was primarily due to a 4.7 million increase in personnel-related and sharebased compensation expense due to increases in employee headcount. The Companys professional fees and facility costs also increased by 0.7 million and 1.0 million, respectively, as Passage Bio expanded its operations to support its RD efforts. Net Loss: Net loss was 27.2 million, or a net loss of 0.60 per basic and diluted share, for the quarter ended June 30, 2020, compared to 13.4 million, or a net loss of 3.19 per basic and diluted share, for the quarter ended June 30, 2019.Conference Call Details Passage Bio will host a conference call and webcast today at 8:30 a.m. ET. To access the live conference call, please dial 833-528-0605 domestic or 830-221-9711 international and reference conference ID number 5679946. A live audio webcast of the event will be available on the Investors Media section of Passage Bios website at investors.passagebio.com. The archived webcast will be available on Passage Bios website approximately two hours after the completion of the event and for 30 days following the call.About Passage Bio Passage Bio is a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system disorders with limited or no approved treatment options. The company is based in Philadelphia, PA and has a research, collaboration and license agreement with the University of Pennsylvania and its Gene Therapy Program GTP. The GTP conducts discovery and IND-enabling preclinical work and Passage Bio conducts all clinical development, regulatory strategy and commercialization activities under the agreement. 