(RTTNews) - Passage Bio (PASG), a clinical-stage genetic medicines company, on Friday announced positive interim data from its ongoing Phase 1/2 upliFT-D clinical trial evaluating PBFT02 for the treatment of frontotemporal dementia or FTD with granulin or GRN mutations.
upliFT-D is a Phase 1/2 global, multi-center, open-label clinical trial of PBFT02 administered by single injection into the cisterna magna in patients aged 35 to 75 years with FTD-GRN or FTD-C9orf72.
The clinical trial will sequentially enroll three FTD-GRN cohorts and two FTD-C9orf72 cohorts.
The upliFT-D study has shown that Dose 1 of PBFT02 significantly increased cerebrospinal fluid or CSF progranulin or PGRN levels, with durable results observed up to 18 months.
This increase in PGRN levels correlated with early signs of improvement in plasma neurofilament light chain or NfL levels, a biomarker of disease progression.
In contrast, untreated symptomatic FTD-GRN patients typically experience increasing NfL levels, underscoring the potential of PBFT02 in slowing disease progression.
As of December 2024, Passage Bio is progressing to evaluate a 50 percent lower Dose 2 in additional cohorts of FTD-GRN and FTD-C9orf72 patients to support further dose exploration and refine its regulatory strategy.
The company anticipates sharing 12-month data and interim safety and biomarker results from Dose 2 in the second half of 2025.
Passage Bio also highlighted the completion of a new, more efficient manufacturing process for PBFT02 and an operational restructure aimed at reducing expenses.
These efforts have extended the company's cash runway into the first quarter of 2027.
The company remains on track to initiate dosing of FTD-C9orf72 patients in the first half of 2025 and plans to seek regulatory feedback on the trial design for a potential pivotal study in 1H 2026.
Passage Bio's focus on advancing PBFT02 in the treatment of FTD is supported by the preclinical evidence and promising clinical data, positioning the company to potentially address the unmet needs of patients with neurodegenerative diseases.
The company had a cash, cash equivalents and marketable securities were $84.8 million as of September 30, 2024.
Currently, PASG is trading at $0.61 down by 7.43%
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