Announcement • Apr 21
Passage Bio Reports Updated Interim Data from Uplift-D Trial Passage Bio, Inc. reported updated data from the ongoing Phase 1/2 upliFT-D clinical trial evaluating PBFT02 for the treatment of frontotemporal dementia (FTD) with granulin (GRN) mutations. PBFT02 administration resulted in improvements in two disease progression biomarkers, as compared to natural history, reducing brain atrophy and stabilizing plasma NfL levels. Updated interim upliFT-D data from FTD-GRN patients treated with PBFT02: Brain Atrophy as Measured by Volumetric Magnetic Resonance Imaging (vMRI) Patients earlier in their disease progression (global score of 1 at baseline on the Clinical Dementia Rating scale, or CDR) who received PBFT02 exhibited reduced rates of whole brain atrophy and frontotemporal cortex atrophy compared to natural history data from patients at the same stage of disease progression. In contrast, patients with more advanced disease progression (global CDR score of 2 at baseline) showed no improvements on either atrophy measure versus natural history data from a comparable global CDR 2 population. Patients with global CDR scores of 0.5 and 1 at baseline are the intended target population for PBFT02, and patients with global CDR scores of 2 or greater have been excluded from future enrollment in the ongoing upliFT-D study. Whole Brain Atrophy PBFT02-treated patients with a global CDR score of 1 at baseline experienced a 64% reduction in whole brain atrophy at 12 months, on average, as compared to vMRI analysis of untreated global CDR 1 patients from the ALLFTD natural history data. PBFT02-treated global CDR 1 patients (n=2): 3.1% atrophy at 12 months ALLFTD natural history global CDR 1 sample (n=7): 8.7% atrophy at 12 months Frontotemporal Cortex Atrophy PBFT02-treated patients with a global CDR score of 1 at baseline experienced a 54% reduction in frontotemporal cortex atrophy at 12 months, on average, as compared to vMRI analysis of untreated global CDR 1 patients from the ALLFTD natural history data. PBFT02-treated global CDR 1 patients (n=2): 4.6% atrophy at 12 months ALLFTD natural history global CDR 1 sample (n=7): 9.9% atrophy at 12 months Plasma Neurofilament (NfL) Patients who received PBFT02 showed stabilization of plasma NfL levels at 12 months posttreatment, comparing favorably to natural history. Plasma NfL levels among PBFT02 treated patients showed an average reduction of 1.0 pg/mL (n=6) at 12 months compared to baseline. In contrast, analysis of untreated symptomatic FTD-GRN patients from the ALLFTD natural history data showed an average increase of 13.5 pg/mL (n=7) at 12 months compared to baseline. Cerebrospinal Fluid (CSF) Progranulin (PGRN) Dose 1 PBFT02 (4.5e13 total genome copies) resulted in a robust and durable increase in CSF PGRN expression through 18 months post-treatment. Dose 1 PBFT02 increased CSF PGRN in all patients from below 3 ng/mL at baseline to a mean of 22.8 ng/mL (n=6) at 12 months and 24.2 ng/mL (n=3) at 18 months. Dose 2 PBFT02 (2.2e13 total genome copies) achieved comparable CSF PGRN levels as Dose 1 at six months post-treatment. Dose 2 PBFT02 increased CSF PGRN to a mean of 8.6 ng/mL (n=2) at one month, above the upper limit of a healthy adult reference range, and 22.6 ng/mL (n=1) at 6 months. Safety (as of March 23, 2026; n=10 FTD-GRN patients and n=1 FTD-C9orf72 patient) PBFT02 continued to be generally well-tolerated, with no new treatment-related serious adverse events (SAEs) reported. As previously disclosed, two patients who received Dose 1 PBFT02 experienced a total of three asymptomatic SAEs related to PBFT02: venous sinus thrombosis (n=2) and hepatotoxicity (n=1). No evidence of dorsal root ganglion (DRG) toxicity and no complications from intracisterna magna (ICM) administration have been reported. A presentation summarizing the interim data update can be accessed on the Events and Presentations page of the Investors and News section of the Company’s website. Regulatory & Program Update The Company has completed a Type C meeting with the FDA to seek guidance on key elements of a future registrational trial of PBFT02 for FTD-GRN patients. Based on the feedback received, the FDA has indicated that a randomized controlled registrational study design is required for PBFT02 in this indication. A randomized controlled registrational trial poses substantial ethical concerns for patients and their families as well as logistical and financial challenges. As such, the Company is evaluating potential next steps in the clinical development of PBFT02 in FTD-GRN and FTD-C9orf72 in the upliFT-D trial. Corporate Update The Company has initiated a review of strategic alternatives to maximize shareholder value. These strategic alternatives may include merger or acquisition transactions, a reverse merger, a sale of assets of the Company, strategic partnerships, licensing opportunities, or other potential paths. The strategic review is underway, and the Company does not intend to provide updates until the Board approves a specific action or otherwise determines that disclosure is appropriate or required. There can be no assurance that the process will result in any such transaction. The Company has engaged Wedbush PacGrow as a financial advisor to assist in the strategic review process. Announcement • Apr 08
Passage Bio, Inc., Annual General Meeting, May 19, 2026 Passage Bio, Inc., Annual General Meeting, May 19, 2026. Announcement • Aug 12
Passage Bio, Inc. Provides Recent Business Highlights Passage Bio, Inc. reported financial results for the second quarter ended June 30, 2025, and provided recent business highlights. Completed dosing of FTD-GRN Cohort 2 in upliFT-D study: Patient 9 was treated with Dose 2 PBFT02 in July, thereby completing dosing of Cohort 2. Cohort 2 consists of a total of four FTD-GRN patients split equally between Dose 1 and Dose 2 PBFT02. Reported updated interim data showing that PBFT02 demonstrated robust, durable elevation in cerebrospinal fluid (CSF) progranulin (PGRN) levels and improvement in a disease progression biomarker: Dose 1 PBFT02 treatment resulted in a robust and durable increase in CSF PGRN expression through 18 months post-treatment, increasing expression in all patients. CSF PGRN levels for the first patient treated with Dose 2 PBFT02 (50% of Dose 1) increased substantially at one-month post-treatment, approaching the upper limit of a healthy adult reference range. In addition, patients who received Dose 1 PBFT02 experienced a reduced annual rate of change in plasma neurofilament light chain (NfL) levels compared to rates observed in natural history studies. These data were also shared in a poster presentation at the Alzheimer’s Association International Conference in July in Toronto, Canada.
Submitted amended upliFT-D study protocol to global trial sites and health authorities: The company amended the upliFT-D clinical trial protocol to introduce a short course of low dose prophylactic anticoagulation and to revise study inclusion criteria to allow for enrollment of patients who are prodromal or have mild cognitive impairment and to exclude patients who are more severely progressed. The amended protocol has been submitted to all active global trial sites and associated health authorities. Following review and acceptance of the amended protocol at trial sites, the company plans to begin enrollment of Cohort 3 (FTD-GRN) and Cohort 4 (FTD-C9orf72), which will continue to evaluate Dose 2 PBFT02. 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-GRNcohorts and two FTD-C9orf72cohorts. Enrollment is currently ongoing. The primary endpoint of the clinical trial is to evaluate the safety and tolerability of PBFT02. Secondary endpoints include disease biomarkers and clinical outcome measures. upliFT-D is a two-year clinical trial with a three-year safety extension.
Passage Bio is pursuing several initiatives to support clinical trial recruitment and enrollment, including a collaborative partnership with InformedDNA to provide no-cost genetic counseling and testing for adults who have been diagnosed by their physicians with FTD. 
