Passage Bio to Showcase GM1 Gangliosidosis and Krabbe Clinical Programs at 2022 WORLDSymposium, February 7 – 11

• Late-breaker presentation to include new clinical data from Cohort 1 in the Imagine-1 clinical trial for GM1 gangliosidosis, Friday, February 11
• Podium presentations on clinical trial designs for Imagine-1 and GALax-C, the clinical trial for Krabbe disease, Wednesday, February 9
• Global Genes poster presentation on Passage Bio-supported patient identification and engagement initiative for rare central nervous system disorders, Tuesday, February 8
• Management to host conference call and webcast following late-breaker GM1 gangliosidosis presentation on Friday, February 11 at 1 p.m. ET

PHILADELPHIA, Jan. 31, 2022 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (Nasdaq: PASG), a clinical-stage genetic medicines company focused on developing transformative therapies for central nervous system (CNS) disorders, today announced its participation at the 18th Annual WORLDSymposium, a research conference dedicated to lysosomal diseases, taking place virtually and in person in San Diego, Calif., February 7 – 11, 2022.

Passage Bio is a lead sponsor of the patient advocacy organization Global Genes’ patient identification and engagement initiative for rare CNS disorders, which will be featured in a poster presentation on Tuesday, February 8. Passage Bio will also deliver two platform presentations on the designs of its clinical trials for Imagine-1 for GM1 gangliosidosis and GALax-C for Krabbe disease on Wednesday, February 9. On Friday, February 11, as part of the conference’s late-breaking science session, the company will present safety and clinical development milestone data for the two patients in Cohort 1 of the Imagine-1 clinical trial.

“As part of our Imagine-1 late-breaker presentation, we will be excited to share new clinical data from Cohort 1 in the ongoing Imagine-1 trial,” said Bruce Goldsmith, Ph.D., president and chief executive officer, Passage Bio. “We are also looking forward to attending WORLD to learn about new scientific research and engage with other researchers, physicians and patient advocacy groups, who share our commitment to continuing to advance the field with the development of transformative medicines for patients with rare CNS disorders.”

Poster Presentation

Tuesday, February 8, 3 to 5 p.m., Pacific Time, Translational Research

Poster 191, Improving patient identification, inclusion and engagement in research for LDs and other rare CNS conditions (PIE4CNS). Presenter: Craig Martin, CEO, Global Genes, Boston

Platform Presentations

Wednesday, February 9, Clinical Applications

• A phase 1/2 open-label, multicenter, dose ranging and confirmatory study to assess the safety, tolerability and efficacy of PBKR03 administered to pediatric subjects with early infantile Krabbe disease (globoid cell leukodystrophy; GALax-C). Podium presentation, 10:30 a.m., Pacific Time. Presenter: Eric Mallack, M.D., MBE, Weill Cornell Medicine, New York
• Phase 1/2 open-label, multi-center study to assess the safety, tolerability and efficacy of a single dose of PBGM01 delivered into the cisterna magna of subjects with type 1 (early onset) and type 2a (late onset) infantile GM1 gangliosidosis. Podium presentation, 1 p.m., Pacific Time. Presenter: Jeanine Jarnes, Pharm D., University of Minnesota, Minneapolis, MN

Friday, February 11, Late-breaking Science 

• Safety, biomarker and preliminary efficacy results following ICM administration of PBGM01 in children with late onset infantile GM1-gangliosidosis. Podium presentation, 8 a.m., Pacific Time. Presenter: David Weinstein, M.D., M.M.Sc., Passage Bio, Philadelphia, PA
  

Passage Bio is planning to host a virtual investor webinar on Friday, February 11 at 1 p.m. ET to review the new clinical data from Cohort 1 of the Imagine-1 trial presented at the WORLDSymposium. A live webcast will be available under “Events and Presentations” in the Investors & News section of the company’s website at www.passagebio.com.The archived webcast will be available on Passage Bio's website approximately two hours after the completion of the event and for 30 days following the call.

About Passage Bio

At Passage Bio (Nasdaq: PASG), we are on a mission to provide life-transforming genetic medicines for patients with CNS diseases that replace their suffering with boundless possibility, all while building lasting relationships with the communities we serve. Based in Philadelphia, PA, our company has established a strategic collaboration and licensing agreement with the renowned University of Pennsylvania’s Gene Therapy Program to conduct our discovery and IND-enabling preclinical work. This provides our team with enhanced access to a broad portfolio of gene therapy candidates and future gene therapy innovations that we then pair with our deep clinical, regulatory, manufacturing and commercial expertise to rapidly advance our robust pipeline of optimized gene therapies into clinical testing. As we work with speed and tenacity, we are always mindful of patients who may be able to benefit from our therapies. More information is available at 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; initiation of clinical trials and the availability of clinical data from such trials; our expectations about our collaborators’ and partners’ ability to execute key initiatives; our expectations about manufacturing plans and strategies; our expectations about cash runway; and the ability of our lead 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 caused by the coronavirus pandemic; 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.

For further information, please contact:

Passage Bio Investors:

Stuart Henderson
Passage Bio
267-866-0114
shenderson@passagebio.com

Passage Bio Media:

Gwen Fisher
Passage Bio
215-407-1548
gfisher@passagebio.com