Passage Bio, Inc. Announces Update on Significant Pipeline and Corporate Advancement
January 11, 2021 at 07:00 am
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Passage Bio, Inc. announced that the company’s lead program PBGM01 is entering the clinic to study its safety and efficacy in addressing infantile GM1, a rare and often life-threatening CNS disorder with no approved disease-modifying therapies. The global Phase 1/2 study, Imagine-1, is expected to enroll the first patient in the first quarter of 2021 and is expected to report initial 30-day safety and biomarker data mid-year 2021. PBGM01 has received regulatory agency clearance from FDA and MHRA. The investigational therapy has also received orphan drug and rare pediatric disease designations from FDA, as well as orphan drug designation from the European Medicines Agency for the treatment of GM1. The company has submitted investigational new drug (IND) applications to FDA for Phase 1/2 clinical studies of PBFT02 in FTD-GRN, a devastating form of early onset dementia; and PBKR03 in Krabbe disease, a pediatric disease with rapid progression, typically resulting in death by 2 years old. The company recently received orphan drug designation from FDA for PBFT02 for FTD-GRN. PBKRO3 has orphan drug and rare pediatric disease designations from FDA for treatment of Krabbe disease. Passage Bio anticipates the start of both clinical programs in the first half of 2021. Initial data from these trials are anticipated to potentially readout in late 2021 or early 2022, depending on the timing of first patient treated in each study. In parallel with pipeline advancement, the company has focused on establishing manufacturing and global distribution from clinical development through initial commercialization. Clinical supply for PBGM01 for phase 1 /2 global trial, manufactured through partnership with Catalent, is already in place. The company have also manufactured clinical supplies to initiate clinical trials for next two most advanced programs – PBKRO3 for Krabbe disease and PBFT02 for FTD-GRN. In December, Passage Bio announced that it had completed construction and started production at its dedicated Current Good Manufacturing Practices (CGMP) manufacturing suite at Catalent Cell & Gene Therapy’s facility in Maryland. The company also announced the planned opening in the second quarter of 2021 of a gene therapy manufacturing research and development site at the Princeton West Innovation Campus in Hopewell, New Jersey, for Chemistry, Manufacturing and Controls (CMC) laboratory operations to support analytics, assay development, and product testing for the company’s gene therapy programs.
Passage Bio, Inc. is a clinical-stage genetic medicines company focused on improving the lives of patients with neurodegenerative diseases. Its clinical product candidate, PBFT02, seeks to elevate progranulin levels to restore lysosomal function and slow disease progression across a variety of neurodegenerative diseases. PBFT02 utilizes an adeno-associated virus capsid to deliver a functional granulin gene, or GRN, encoding progranulin to the brain via intra cisterna magna administration. The lead indication for PBFT02 is frontotemporal dementia, or FTD, caused by progranulin deficiency, or FTD-GRN. Its development programs consist of PBFT02 for the treatment of FTD-GRN, PBFT02 for the treatment of FTD-C9orf72 and amyotrophic lateral sclerosis, and PBFT02 for the treatment of Alzheimerâs disease. Its clinical product candidates include PBGM01, PBKR03 and PBML04. It has one unnamed preclinical research program that explores multiple potential treatment targets for Huntington's disease.