Passage Bio, Inc.

Good morning and welcome to the Passage Bio Board Quarter and Full Year 2022 Financial and Operating Results Conference Call. At this time, all participants are in listen-only mode. Following the formal remarks, we will open the call-out for your questions. Please be advised that this call is being recorded at the company's request. Now, I'd like to turn it over to Stuart Henderson, Vice President of Corporate Development and Investor Relations. Stuart, please proceed.

Thank you, Operator. This morning, we issued a press release that outlines the topics we plan to discuss today. This release is available on the Passage Bio website under the Press Releases and Statements section of Investors & News. On today's call, Chief Executive Officer Will Chu will review our fourth quarter 2022 and recent business highlights. Mark Foreman, our Chief Medical Officer, will review our clinical programs, and Simona King, our Chief Financial Officer, will review our fourth quarter and full year 2022 financial results. Before we begin, please note that today's call may include a number of forward-looking statements. These forward-looking statements are based on assumptions that are subject to risks and uncertainties that could cause the company's actual results to differ significantly from those suggested by these statements. Given these risks and uncertainties, you should not place undue reliance on these forward-looking statements. Please refer to the company's filings with the SEC for information concerning risk factors that could cause its actual results to differ materially from expectations including any forward-looking statements made on this call. Except as required by law, the company disclaims any obligation to publicly update or revise any forward-looking statements to account for or reflect events or circumstances that occur after this call. It is now my pleasure to pass the call over to Will.

Thanks, Stuart, and thank you all for joining us this morning. I want to begin today's call by briefly reflecting on this past year. By honing our strategy and operational focus, we ended 2022 in a strong position, and I'm proud of the work the team did to lay the groundwork and build the momentum needed to ensure we can deliver meaningful clinical data from our two lead programs in 2023. I'm excited by what 2023 has in store for Passage Bio, and I'm pleased to report that the year is already off to a strong start with the presentation of additional data from cohorts one through three of our IMAGINE 1 clinical trial for GM1 gangliosidosis at the World Symposium last month. The data shared at World continue to demonstrate that PBGMO1 administration is well tolerated, has a positive safety profile, and exerts a biological effect across all treated infantile GM1 patients. Mark will review these results in more detail shortly, but this data builds upon the information we presented in December and adds to our confidence of the potential benefit that PB-GMO1 may offer the GM1 patient community. To date, we've completed dosing of the first four cohorts in our IMAGINE ONE study for a total of eight patients. and look forward to reporting initial data from cohort four, which is early infantile patients treated with a high dose, by mid-year. Based on the encouraging data we have generated thus far and our supporting preclinical data, we are moving forward with plans to treat additional patients with a higher dose than doses administered in cohorts one through four. We also plan to revise our study inclusion criteria to maximize the benefit-risk profile of PB-GMO1. By treating additional patients at a higher dose, we will generate important clinical data to further inform dose and patient selection, as well as discussions with regulatory authorities on the design of a confirmatory study. For our uplifted trial for frontotemporal dementia, or FTD, we remain focused on driving patient identification and recruitment initiatives and expanding our global footprint of clinical trial sites. Based on the momentum we are experiencing, we expect to report initial safety and biomarker data from cohort one in the second half of the year. While our primary focus is on generating meaningful clinical data for our GM1 and FTD programs, We also continue to advance our promising preclinical programs in amyotrophic lateral sclerosis, Huntington's disease, and temporal lobe epilepsy through our ongoing innovative research partnership with Penn's gene therapy program. Moreover, following the prioritization of our research and development efforts announced last November, we remain committed to exploring strategic alternatives to advance our two clinical stage programs for Krabbe disease and metachromatic leukodystrophy. In addition, we continue to leverage our strong in-house analytical capabilities at our CMC lab at Princeton West, as these capabilities provide a competitive advantage and are critical to support ongoing and future clinical development of our programs. Lastly, we are supported by a strong balance sheet, providing us with the necessary runway to generate meaningful clinical data for our GM1 and FTD programs. Simona will describe our financials in more detail, but we expect existing cash resources to fund operations into the first half of 2025. With that, I will now turn it over to Mark to review our two lead clinical programs and the recent GM1 data.