Passage BIO, Inc. Q3 FY2021 earnings call

Strategic Pillars - Strong partnership with Dr. Jim Wilson and his team at the University of Pennsylvania’s Gene Therapy Program (GTP). - Robust and differentiated pipeline. - Integrated manufacturing supply chain. - Strong cash balance. ### Clinical Progress - Three global Phase I/II programs with site activations despite COVID-19 challenges. - Plan to dose first patients in upliFT-D study for frontal temporal dementia and GALax-C study for Krabbe disease by year-end. - Expect to report first-in-human data from Imagine-1 trial for GM1 gangliosidosis later this quarter. - Aim to report initial safety and 30-day biomarker data from upliFT-D and GALax-C trials in the first half of 2022. ### Patient Identification - Collaboration with disease experts, community leaders, and partners offering free genetic testing and counseling. - Efforts to support early diagnosis of Krabbe disease through partnerships with advocacy organizations for newborn screening. ### Financials - Current cash, cash equivalents, and marketable securities are expected to fund operations for at least the next 24 months.

Simona King reported that research and development (R&D) expenses for the third quarter ended September 30, 2021, were $26.6 million, compared to $20.8 million in the same quarter of 2020. General and administrative (G&A) expenses were $15 million for the third quarter of 2021, versus $7.8 million in the same quarter of 2020. Net loss was $46.9 million in the third quarter of 2021, compared to $28.5 million in 2020. The company ended the quarter with approximately $354 million in cash, cash equivalents, and marketable securities.

• Expect to report first-in-human data from the initial cohort of patients in the Imagine-1 trial for GM1 gangliosidosis later this quarter. - Plan to dose the first patients in the upliFT-D study for frontal temporal dementia and GALax-C study for Krabbe disease by year-end. - Anticipate reporting initial safety and 30-day biomarker data from the upliFT-D and GALax-C trials in the first half of 2022. - Initiating research programs in Alzheimer’s disease and temporal lobe epilepsy.

• Forward-looking statements are subject to risks and uncertainties, including timing and execution of clinical milestones, collaborators’ ability to execute, manufacturing plans, financial performance, COVID-19 impact, and ability to fund R&D. - Risks of actual results differing significantly from forward-looking statements, as detailed in the company’s SEC filings.

Q: About the FTD-GRN and Krabbe studies, can you lay out the process in terms of site openings, screening, enrollment, or dosing?

A: Bruce and Eliseo discussed site activations, with progress made despite COVID-19, and mentioned a vast network of physicians, experts, and advocacy organizations for patient identification.

Q: Timing for the FTD-GRN data?

A: Bruce explained there is a 60-day interval between dosing, expecting late first half of 2022 for initial safety and 30-day biomarker data.

Q: What are the constraints on site activation?

A: Bruce and Eliseo noted operational challenges like IRB reviews, budget negotiations, and interactions with multiple hospital departments.

Q: Concerns about screening failures in Imagine-1?

A: Bruce discussed inclusion/exclusion criteria related to patient age and developmental stage, which can affect enrollment.

Q: About the FTD program patient screening and timeline updates?

A: Bruce explained active patient identification through partnerships, and updates will be provided when the first patient is dosed.

Q: Timeline for earlier pipeline data?

A: Bruce stated they will share preclinical data in 2022 in partnership with the University of Pennsylvania’s Gene Therapy Program as they advance and feel comfortable with the data.