PASG

Will Chou reflected on 2022, noting the strong position and plans for 2023 to deliver clinical data from lead programs. Mark Forman reviewed PBGM01 for GM1 Gangliosidosis, highlighting positive interim data from Imagine-1 trial with well-tolerated PBGM01, dose-dependent biomarker changes, and clinical stabilization. He also discussed PBFT02 for FTD, focusing on patient recruitment and expected safety/biomarker data in H2 2023. Simona King provided financials, noting cash equivalents, R&D and G&A expenses, and net loss figures.

Existing cash resources are expected to fund operations into the first half of 2025. Plan to report initial data from Cohort 4 of Imagine-1 in mid-2023, dose higher dose GM1 patients in H2 2023, and initial safety/biomarker data from PBFT02 Cohort 1 in H2 2023.

Forward-looking statements subject to risks and uncertainties. Regulatory review and potential delays in clinical trials. Supply chain issues affecting preclinical programs could impact progress.

Q: In terms of the dose escalation plan, confirm if additional patients are identified and details on dose escalation and pivotal study timelines.

A: Mark Forman stated they've received interest from many families, confident in recruiting with revised criteria. Biomarker response and clinical improvement are key, with at least 6 months follow-up needed for GM1 approval.

Q: Disclose higher dose for PBGM01 and status of PBFT02 patient dosing.

A: Higher dose for PBGM01 is subject to regulatory review. For PBFT02, patients are queued at sites, with no specific details on individual patient dosing provided.

Q: On FTD biomarkers and cohort data, details on biomarkers and cohort enrollment.

A: Initial data from PBFT02 Cohort 1 will focus on safety and CSF progranulin levels, with plans to provide data on multiple patients from the first cohort later in the year