Passage Bio Inc
NASDAQ:PASG
Passage Bio Inc
Passage Bio, Inc. operates as a genetic medicines company. The company is headquartered in Philadelphia, Pennsylvania and currently employs 133 full-time employees. The company went IPO on 2020-02-28. The company is focused on developing transformative therapies for central nervous system (CNS) disorders. Its first product candidate, PBGM01, utilizes AAVhu68 capsid to deliver to the brain and peripheral tissues a functional GLB1 gene encoding lysosomal beta-galactosidase (β-gal) for gangliosidosis (GM1). Its second product candidate, PBFT02, utilizes AAV1 capsid to deliver to the brain a functional GRN gene encoding progranulin (PGRN) for frontotemporal dementia (FTD) caused by progranulin deficiency (FTD-GRN). Its third product candidate, PBKR03, utilizes AAVhu68 capsid to deliver to the brain and peripheral tissues a functional gene encoding the hydrolytic enzyme galactosylceramidase (GALC) for Krabbe disease. The company also has four programs in the research stage: PBML04 for metachromatic leukodystrophy (MLD); PBAL05 for amyotrophic lateral sclerosis (ALS); PBCM06 for Charcot-Marie-Tooth Type 2A (CMT2A), and other.
Passage Bio, Inc. operates as a genetic medicines company. The company is headquartered in Philadelphia, Pennsylvania and currently employs 133 full-time employees. The company went IPO on 2020-02-28. The company is focused on developing transformative therapies for central nervous system (CNS) disorders. Its first product candidate, PBGM01, utilizes AAVhu68 capsid to deliver to the brain and peripheral tissues a functional GLB1 gene encoding lysosomal beta-galactosidase (β-gal) for gangliosidosis (GM1). Its second product candidate, PBFT02, utilizes AAV1 capsid to deliver to the brain a functional GRN gene encoding progranulin (PGRN) for frontotemporal dementia (FTD) caused by progranulin deficiency (FTD-GRN). Its third product candidate, PBKR03, utilizes AAVhu68 capsid to deliver to the brain and peripheral tissues a functional gene encoding the hydrolytic enzyme galactosylceramidase (GALC) for Krabbe disease. The company also has four programs in the research stage: PBML04 for metachromatic leukodystrophy (MLD); PBAL05 for amyotrophic lateral sclerosis (ALS); PBCM06 for Charcot-Marie-Tooth Type 2A (CMT2A), and other.
Strong Cash Runway: Passage Bio ended 2022 with $189.6 million in cash, expected to fund operations into the first half of 2025.
Clinical Progress: The company advanced its two lead gene therapy programs, with new positive interim data in GM1 gangliosidosis and plans to dose additional patients at higher levels.
Key 2023 Milestones: Initial data readouts for high-dose infantile GM1 and the first cohort of the FTD trial are targeted for mid and late 2023, respectively.
Study Design Adjustments: Passage Bio is revising inclusion criteria to focus on earlier-stage GM1 patients and increasing dosing to optimize outcomes based on biomarker responses.
Cost Controls: Streamlined operations led to a significant reduction in both R&D and G&A expenses year-over-year.
Regulatory Strategy: The company will discuss registrational study design with regulators after generating additional high-dose patient data, delaying such conversations to 2024.
