PHILADELPHIA, PA — Passage Bio, Inc. has unveiled promising interim data from its ongoing global Phase 1/2 upliFT-D clinical trial at the 14th International Conference on Frontotemporal Dementias (ISFTD2024). The study examines PBFT02, a gene therapy utilizing adeno-associated virus (AAV) delivery, aimed at treating frontotemporal dementia (FTD) with granulin (GRN) mutations.
The trial’s first cohort, consisting of five patients, showed that Dose 1 of PBFT02 resulted in significant increases in cerebrospinal fluid (CSF) progranulin levels, sustained up to 12 months after administration. The therapy was well-tolerated, with patients receiving a revised immunosuppression regimen to prevent adverse immune responses.
Dr. Juan Chavez, vice president of clinical development, presented the data during a poster session at the conference. This session highlighted the safety and biomarker outcomes of the trial, emphasizing the potential of PBFT02 as a groundbreaking treatment for neurodegenerative diseases.
The interim results revealed that all four patients who received the enhanced immunosuppression protocol experienced no serious adverse events. Any adverse events that did emerge were mild to moderate. Notably, there was no evidence of a significant immune response or hepatotoxicity, critical factors in assessing the treatment’s safety profile. Patient follow-ups also showed no dorsal root ganglion toxicity, confirmed through nerve conduction studies.
Biomarker analysis demonstrated robust increases in CSF progranulin levels across all patients, with levels rising up to six-fold at one month and ten-fold at six months post-treatment. These levels significantly exceeded those found in healthy adult controls, affirming the treatment’s effectiveness in elevating progranulin levels.
Dr. Will Chou, president and CEO of Passage Bio, expressed optimism about these findings, stating, “We are very encouraged by the positive Cohort 1 data from our upliFT-D trial demonstrating that intra-cisterna magna delivery of Dose 1 of PBFT02 resulted in robust and durable increases in CSF PRGN expression, with elevated levels maintained for up to one year after treatment.” He further emphasized the therapy’s potential, noting its promising safety profile and significant biomarker results.
The data presented reinforce Passage Bio’s strategy to advance PBFT02 as a one-time treatment for FTD and other neurodegenerative diseases. The company remains committed to furthering this research, with continued support from trial participants, their caregivers, and the clinical team. The detailed outcomes of this study highlight the promising horizon in genetic therapies for complex neurological disorders.
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