PHILADELPHIA, PA — Passage Bio, Inc. (NASDAQ: PASG), a gene therapy company, recently unveiled initial safety and biomarker data from the first phase of its upliFT-D clinical trial. The trial is testing PBFT02, an adeno-associated virus (AAV)-delivery gene therapy, designed to treat frontotemporal dementia (FTD) with granulin mutations. This type of dementia presents early onset symptoms and currently has no approved disease-modifying therapies.
The therapy involves a single dose administered via intra-cisterna magna injection. The PBFT02 uses an AAV1 viral vector to deliver a functional copy of the GRN gene encoding PGRN to a patient’s cells, potentially remedying the genetic deficiency that results in FTD.
Positive Results from Phase 1/2 upliFT-D Clinical Trial
The preliminary data from the first three patients in the trial shows promising results. The first dose of PBFT02 was generally well-tolerated in patients 2 and 3 who received an enhanced steroid regimen following an amendment to the protocol.
No serious adverse events or clinically significant immune responses were reported, and the treatment did not show any signs of hepatotoxicity or safety-related imaging abnormalities. Patient 1, who received a lower level of immunosuppression, experienced two asymptomatic serious adverse events, both consistent with an immune response.
Following these findings, the protocol was subsequently amended to increase the steroid regimen for subsequent patients. No toxicity or complications related to the administration method were observed across any of the three patients.
Biomarkers Indicate Potential Efficacy
The therapy also resulted in a substantial increase in CSF PGRN, a key biomarker associated with FTD. The levels observed at day 30 were 3.6 to 6.6 times higher than baseline and remained at supraphysiologic levels at six months. This increase in PGRN levels could potentially slow or halt the progress of FTD.
Strategic Priorities and Future Plans
Passage Bio is now focusing on several strategic priorities, including continuing the clinical development of PBFT02 for FTD-GRN and extending its use to treat other adult neurodegenerative diseases such as FTD-C9orf72, ALS, and Alzheimer’s disease.
The company is also prioritizing its preclinical program for Huntington’s disease, leveraging its existing partnership with the Penn Gene Therapy Program. It is actively exploring potential partnerships for clinical-stage pediatric programs in GM1 gangliosidosis, Krabbe disease, and metachromatic leukodystrophy.
Passage Bio expects to initiate dosing for the second cohort of FTD-GRN patients in the first half of 2024 and report full six-month safety and biomarker data from the first cohort in the latter half of the same year.
Given these promising early results, investors may want to keep a close eye on this innovative biotech firm.
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