Passage BIO(US:PASG)2022-08-11 17:50Pipeline and Programs - Passage Bio is developing transformative therapies for CNS disorders, targeting both rare and large patient populations[5] - The company's pipeline includes programs for GM1 gangliosidosis (PBGM01), Krabbe disease (PBKR03), metachromatic leukodystrophy (PBML04), and frontotemporal dementia (PBFT02)[5] - PBGM01 targets GM1 gangliosidosis, a rare pediatric disease with an incidence of approximately 1 per 100,000 live births worldwide[11] - PBKR03 addresses Krabbe disease, another devastating pediatric condition with an incidence of about 26 per 1 million live births worldwide[33] - PBFT02 is focused on frontotemporal dementia (FTD-GRN), a rare adult disease with an estimated U S prevalence of approximately 3,000 to 6,000 patients[40] - PBML04 targets metachromatic leukodystrophy (MLD), a rare pediatric disease with an incidence of approximately 1 per 100,000 live births worldwide[47] Clinical Trial Updates - Initial safety and biomarker data from Cohorts 2 & 3 of the PBGM01 trial were expected in the second half of 2022[16] - Initial safety and biomarker data from a subset of Cohort 1 in the PBKR03 (GALax-C) trial were expected by the end of 2022[38] - First patient dosing in the Phase 1/2 trial for PBFT02 was expected in mid-2022[42] - An IND clearance was obtained from the FDA in Q2 2022 for PBML04[50, 59] Financial Status - The company reported a cash balance of $239 million as of June 30, 2022, which is expected to fund operations into Q2 2024[59]