Passage Bio (NasdaqGS:PASG) FY Conference November 12, 2025 09:30 AM ET Speaker0Joining Guggenheim's twenty twenty five Healthcare Innovations Conference. I am Debjit, one of the therapeutic analysts. And my privilege to welcome our next presenting company, Passage Bio. And joining from Passage is President and CEO, William Cho. Good morning.Thanks for having me. Thanks for making time for us, and, you know, as is customary, let's do a quick intro before we get into the Q and A.Speaker1Sure. Will Chew. I'm ...

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 September 30, 2025 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 ...

PASSAGE BIO REPORTS THIRD QUARTER 2025 FINANCIAL RESULTS AND PROVIDES RECENT BUSINESS HIGHLIGHTS Actively enrolling Cohort 3 (FTD-GRN) and Cohort 4 (FTD-C9orf72) patients in ongoing upliFT-D study Aligned with the U.S. Food and Drug Administration (FDA) on an analytical approach to establish comparability of a high-productivity, suspension-based PBFT02 manufacturing process On track to obtain regulatory feedback on FTD-GRN registrational trial design in 1H 2026 Cash runway into 1Q 2027 PHILADELPHIA – Novemb ...

Actively enrolling Cohort 3 (FTD-GRN) and Cohort 4 (FTD-C9orf72) patients in ongoing upliFT-D study Aligned with the U.S. Food and Drug Administration (FDA) on an analytical approach to establish comparability of a high-productivity, suspension-based PBFT02 manufacturing process On track to obtain regulatory feedback on FTD-GRN registrational trial design in 1H 2026 Cash runway into 1Q 2027 PHILADELPHIA, Nov. 10, 2025 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (Nasdaq: PASG), a clinical stage genetic medicines c ...

PHILADELPHIA, Nov. 05, 2025 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (NASDAQ: PASG), a clinical stage genetic medicines company focused on improving the lives of patients with neurodegenerative diseases, today announced that Will Chou, M.D., president and chief executive officer, will participate in a fireside chat during the Guggenheim Securities 2nd Annual Healthcare Innovation Conference on Wednesday, November 12, 2025 at 9:30 a.m. ET in Boston, MA. A live webcast of the event will be available on the Inves ...

Summary of Conference Call on Gene Therapy for Neurodegenerative Diseases Industry Overview - The conference focused on gene therapy applications for neurodegenerative diseases, featuring speakers from three companies: Mira, uniQure, and Passage Bio [1][2][6]. Key Points by Company Mira - **Company Overview**: Mira specializes in genetic medicines, focusing on local delivery of small doses to treat severe indications, which enhances safety and reduces costs [2]. - **Recent Milestones**: Mira has two pivotal studies and two awaiting Biologics License Application (BLA) filings, including treatments for rare and common eye diseases and a Parkinson's treatment that has shown three positive studies [3][4]. - **Innovative Approach**: The company utilizes AI technology to analyze data from the largest neurohospital in Europe, demonstrating physiological changes in the brain related to their Parkinson's treatment [4][24]. - **Potential Applications**: Mira's gene therapy can convert glutamate to GABA, potentially treating various neurodegenerative diseases by calming hyperactive neurons [22][24]. uniQure - **Company Overview**: uniQure is a pioneer in genomic medicine, known for developing the first approved AAV gene therapies, including Glybera and Hemgenix [6][9]. - **Lead Program**: The company is focused on Huntington's disease, utilizing a platform called MyCure to suppress the aberrant Huntingtin protein [8]. - **Clinical Findings**: In a study involving 45 patients, uniQure reported a 75% statistically significant slowing of disease progression based on the UHDRS [9]. - **Regulatory Plans**: A pre-BLA meeting is expected in Q4, with a BLA submission planned for Q1 of the following year [9]. Passage Bio - **Company Overview**: Passage Bio is based on technology from the University of Pennsylvania, focusing on gene therapy for frontotemporal dementia and Huntington's disease [14]. - **Clinical Approach**: The company delivers AAV1 via a minimally invasive procedure, showing high levels of target engagement and stabilization of neurofilaments [14][15]. - **Future Directions**: Passage Bio is also developing a preclinical program targeting the DNA damage response pathway in Huntington's disease [15]. Challenges in Gene Therapy - **Delivery Issues**: The primary challenge remains delivering therapies to the correct brain regions, especially for diseases like Huntington's [18][19]. - **Slow Disease Progression**: Designing clinical studies for slowly progressing neurodegenerative diseases is difficult, as changes in function are hard to detect [19][20]. - **Heterogeneity**: Variability among patients complicates the assessment of treatment efficacy, necessitating innovative statistical methods to account for differences [31][32]. Regulatory Considerations - **FDA Flexibility**: The FDA is showing increased willingness to consider alternative trial designs and endpoints for rare diseases with high unmet needs [44][46]. - **Natural History Data**: Utilizing natural history datasets can help in understanding disease progression and support regulatory submissions [30][31]. Safety Concerns - **Patient Safety**: The panel acknowledged the importance of safety in gene therapy, especially following recent patient deaths in trials [35][37]. - **Local Delivery Advantages**: Localized delivery methods may reduce systemic exposure and associated risks, as demonstrated by lower doses used in certain therapies [38][41]. Access and Implementation - **Training and Infrastructure**: Successful implementation of gene therapies will depend on training healthcare providers and establishing treatment centers [52][54]. - **Data-Driven Adoption**: Strong clinical data will be crucial for gaining acceptance among medical professionals and ensuring broad access to therapies [55][56]. Conclusion - The conference highlighted the potential of gene therapy in treating neurodegenerative diseases, the challenges faced in delivery and study design, and the evolving regulatory landscape that may facilitate the development of these innovative treatments [1][17][42].

Core Insights - Passage Bio, Inc. is a clinical stage genetic medicines company focused on neurodegenerative diseases [3] - The company will participate in the Chardan 9th Annual Genetic Medicines Conference on October 21, 2025 [1] - The lead product candidate, PBFT02, aims to treat frontotemporal dementia by elevating progranulin levels [3] Company Overview - Passage Bio is dedicated to developing one-time therapies targeting the underlying pathology of neurodegenerative conditions [3] - The company emphasizes its commitment to improving the lives of patients and families affected by these diseases [4] Event Details - Will Chou, M.D., the CEO, will be part of a panel discussion at the conference [1] - A live webcast of the event will be available on the company's website, with a replay accessible for 90 days [2]

Summary of Passage Bio (PASG) FY Conference Call - September 05, 2025 Company Overview - Passage Bio is a clinical stage genetic medicines company focused on neurodegenerative diseases, particularly frontotemporal dementia (FTD) [2][3] Core Program: PBFT02 - PBFT02 is the lead clinical program targeting frontotemporal dementia with the GRN mutation, addressing a significant unmet need as there are no approved disease-modifying therapies for this condition [2][4] - The target population includes approximately 18,000 patients with GRN mutation FTD and 21,000 with C9orf72 mutation FTD across the U.S. and Europe [3] Clinical Development and Milestones - The ongoing phase 1/2 study of PBFT02 is a multicenter international open-label dose exploration study, currently active in the U.S., Canada, Portugal, and Brazil [7] - Key upcoming milestones include seeking regulatory feedback on manufacturing comparability in Q4 2025 and reporting dose two data in the first half of 2026 [3][21] Mechanism of Action - PBFT02 utilizes AAV gene therapy to increase levels of progranulin, a protein crucial for CNS cell homeostasis, which is deficient in patients with GRN mutation FTD [4][5] - The administration route is intracisterna magna, allowing for broad CNS biodistribution and lower doses compared to systemic delivery [9][10] Clinical Data and Safety Profile - Initial cohorts have shown promising results, with dose one achieving an average of over 25 nanograms per ml of progranulin levels at 12 months, indicating robust and durable responses [15][18] - The safety profile includes three serious adverse events (SAEs) among eight treated patients, with a decision to implement prophylactic anticoagulation for future patients due to observed venous thromboembolic events [12][20] Biomarker Analysis - Plasma neurofilament levels, a marker of neurodegeneration, showed a significant reduction in treated patients compared to the expected annual increase of 29% in untreated patients, suggesting a potential slowing of disease progression [17][20] - The study is also tracking CSF progranulin levels and clinical dementia rating scales to assess treatment efficacy [9][11] Competitive Landscape - PBFT02 is positioned as a potentially best-in-class therapy, with higher and more durable progranulin levels compared to other therapies in development for FTD [18][19] - Market research indicates a preference for a one-time therapy over monthly treatments, particularly for patients with behavioral dementia [19] Future Directions - Plans to expand the clinical study to include FTD C9 patients, as raising progranulin may also benefit TDP-43 pathology seen in multiple neurodegenerative diseases [20][21] - The company has a cash balance of $58 million, providing a runway into Q1 2027 to continue patient treatment and data collection [22] Conclusion - Passage Bio is advancing its clinical program for PBFT02 with promising early data and a clear regulatory pathway, aiming to address significant unmet needs in neurodegenerative diseases [22]

Core Viewpoint - Passage Bio, Inc. (PASG) shows significant potential for upside, with a mean price target of $59.25 indicating a 731% increase from its current trading price of $7.13 [1] Price Targets and Estimates - The average price target for PASG ranges from a low of $10.00 to a high of $120.00, with a standard deviation of $46.71, suggesting a high variability in analyst estimates [2] - The lowest estimate indicates a potential increase of 40.3%, while the most optimistic estimate suggests a 1583% upside [2] - Analysts' consensus on price targets is often questioned, as they may not accurately reflect future stock prices [3][7] Earnings Estimates and Analyst Agreement - Strong agreement among analysts regarding PASG's ability to report better earnings than previously predicted supports the view of potential upside [4] - The Zacks Consensus Estimate for the current year has increased by 30.3% over the past month, with two estimates revised higher and no negative revisions [12] - PASG holds a Zacks Rank 2 (Buy), placing it in the top 20% of over 4,000 ranked stocks based on earnings estimates [13] Implications of Price Movement - While the consensus price target may not be a reliable indicator of potential gains, the direction implied by these targets appears to be a useful guide for further research [14]

Company Overview - Passage Bio, Inc. is a clinical stage genetic medicines company focused on improving the lives of patients with neurodegenerative diseases [3] - The company's lead product candidate, PBFT02, aims to treat neurodegenerative conditions such as frontotemporal dementia by elevating progranulin levels to restore lysosomal function and slow disease progression [3] Upcoming Events - Management will participate in the H.C. Wainwright 27th Annual Global Investment Conference from September 8-10, 2025, in New York City [1] - Will Chou, M.D., president and CEO, will present an overview of the company and engage in one-on-one meetings with investors [2] - A video webcast of the presentation will be available to registered attendees starting September 5, 2025, at 7:00 a.m. ET, and a replay will be accessible for 30 days post-event [2]