Passage Bio, Inc.

Cash equivalents and marketable securities were $189.6 million at December 31, 2022, versus $315.8 million at December 31, 2021. R&D expense was $17.7 million for Q4 2022 and $86.1 million for full-year 2022, down from $33.0 million and $117.7 million a year earlier. G&A expense was $10.6 million for Q4 2022 and $49.3 million for full-year 2022, versus $17.2 million and $60.1 million in the prior-year periods. Net loss was $27.1 million for Q4 2022 and $136.1 million for full-year 2022, compared with $51.2 million and $185.4 million in 2021. Management said existing cash resources are expected to fund operations into the first half of 2025. For 2023, the company expects initial data from Cohort 4 in mid-2023, higher-dose GM1 dosing in the second half of 2023, and initial safety/biomarker data from PBFT02 Cohort 1 in the second half of 2023.

Will Chou framed 2022 as a year of strategy refinement and operational focus, saying Passage Bio ended the year in a strong position to deliver meaningful clinical data in 2023. He emphasized momentum in GM1 after additional WORLD data, the plan to treat more patients at a higher dose, and the intention to revise eligibility criteria to improve the benefit-risk profile. His tone was constructive and execution-focused, with repeated emphasis on 2023 milestones and the company’s ability to fund its programs.

Simona King focused on balance sheet runway and expense discipline. She said cash, cash equivalents, and marketable securities were $189.6 million at December 31, 2022, and that the company expects these resources to fund operations into the first half of 2025. She also cited lower spending versus 2021, with R&D at $17.7 million in Q4 and $86.1 million for the year, G&A at $10.6 million in Q4 and $49.3 million for the year, and net loss of $27.1 million in Q4 and $136.1 million for the year.

Analysts focused heavily on the GM1 dose-escalation plan, asking whether new patients had already been identified, what biomarker or functional thresholds would define success, and how long follow-up would be needed for a registrational study. Management said dozens of families had expressed interest, the revised criteria should still allow recruitment, and they are using CSF GM1 ganglioside reduction as the main pharmacodynamic marker, with a minimum of six months of follow-up needed to see developmental change. On FTD, management said Cohort 1 data will center on safety and CSF progranulin at baseline and day 30, with expectations for physiologic CSF progranulin levels and data from multiple patients in the second half of 2023.