Passage BIO(US:PASG)2022-05-16 16:40Pipeline 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), frontotemporal dementia (PBFT02), amyotrophic lateral sclerosis (PBAL05), Huntington's disease, Alzheimer's disease, and temporal lobe epilepsy[5] - PBGM01 is in Phase 1/2 clinical trials for GM1 gangliosidosis, a rare pediatric disease with an incidence of approximately 1 per 100,000 live births worldwide[5, 11] - PBKR03 is in Phase 1/2 clinical trials for Krabbe disease, another rare pediatric disease with an incidence of approximately 26 per million live births worldwide[5, 39] - PBFT02 is in Phase 1/2 clinical trials for frontotemporal dementia (FTD-GRN), a rare adult disease with an estimated U S prevalence of approximately 3,000 to 6,000 patients[5, 46] Clinical Data and Milestones - Interim data from the PBGM01 Phase 1/2 trial showed a positive safety profile, demonstration of functional transgene expression, and meaningful gains in developmental milestones in Cohort 1[15, 18] - In PBGM01 Cohort 1, post-treatment CSF and serum β-gal activity for both patients were above natural history study patient values[18] - Patient 1 in the PBGM01 trial demonstrated meaningful gains in developmental milestones, advancing from baseline to 9 months developmental age[31] - Patient 2 in the PBGM01 trial regained previously lost developmental milestones at month 4, including the ability to walk and use specific words[36] - The company anticipates initial safety and biomarker data from PBKR03's GALax-C trial by the end of 2022[44] Financials - Passage Bio had a cash balance of $267 million as of March 31, 2022, which is expected to fund operations into Q2 2024[62]