Passage BIO, Inc. Q2 FY2021 earnings call

• Strategic pillars include strong partnership with Penn's Gene Therapy Program, broad and robust pipeline, integrated manufacturing supply chain, and solid corporate foundation. - Amended strategic collaboration and license agreement with GTP to broaden scope to large CNS diseases, initiating exploratory research on Alzheimer's disease and Temporal Lobe Epilepsy while maintaining focus on rare monogenic disorders. - Opened new Gene Therapy Research and Development Lab in Hopewell, New Jersey. - New executive appointments made, including Chief Commercial Officer, Chief Financial Officer, and Chief Medical Officer, and strong Board of Directors support. - Clinical programs progress: Imagine-1 trial for GM1 gangliosidosis progressing as planned; GALC trial for early infantile Krabbe disease expected to dose first patient in third quarter; upliFT-D trial for FTD-GRN expected to dose first patient in third quarter with PBFT02 receiving orphan designation from the European Commission.

Cash, cash equivalents and marketable securities ended the quarter at approximately $408 million compared to $305 million as of December 31, 2020. R&D expenses were $33.1 million for the quarter ended June 30, 2021, compared to $19.9 million in the same quarter of 2020. The increase was due to various factors including higher clinical manufacturing costs, clinical development costs, personnel related and share based compensation expense. G&A expenses were $15.4 million for the quarter ended June 30, 2021, compared to $7.4 million in the same quarter of 2020, driven by increases in personnel related and share based compensation expense and expanded operations. Net loss was $48.4 million in the second quarter of 2021 compared to $27.2 million in the same quarter of 2020. Net loss per basic and diluted shares was $0.90 in the second quarter of 2021 compared to $0.60 in the same quarter of 2020.

• Dose first patients in upliFT-D and GALC trials in the third quarter of 2021. - Report initial safety and 30 day biomarker data for Cohort One of the Imagine-1 study in the fourth quarter of 2021. - Report initial safety and 30 day biomarker data from upliFT-D and GALC trials in the first half of 2022.

• Forward-looking statements subject to risks and uncertainties such as COVID-19 impact on operations, cost management, and funding of R&D programs. - Challenges in site initiation and patient identification for clinical programs, including IRB timing and study initiation backlogs.

Q: Regarding the first patient dosed in the infantile GM1 study in April, have you dosed the second patient and on site activation?

A: First patient dosed, site initiation activities ongoing with sequential activation strategy.

Q: Regarding the FTD study first patient dosing time?

A: Due to idiosyncratic site differences and environmental factors, there was a slight delay but site initiation activities are progressing.

Q: Regarding future programs like MLD, ALS and MLD program advantages?

A: Exploratory research of new agreement will proceed first, MLD program has its own advantages to be further observed.

Q: Regarding expectations for GM1 study in the fourth quarter and biomarker measurement?

A: Report safety updates and 30 day biomarker data in the fourth quarter, biomarker measurement work progressing.

Q: Regarding the expanded UPenn collaboration reason and balance strategy?

A: Natural extension of existing partnership and work, aiming to balance and diversify pipeline while continuing clinical programs.

Q: Regarding the definition of early symptomatic FTD in the upliFT-D study and AAV Vector meeting impact?

A: Early symptomatic FTD defined by CDR 0.5 - 1 and high serum NfL level, focusing on AAV vector meeting potential impacts and related toxicity risks.