PASG

Enrolled first three FTD-GRN patients in Cohort 3 of ongoing upliFT-D study Treated first FTD-C9orf72 patient with Dose 2 PBFT02 in Cohort 4 of upliFT-D study On track to report updated interim safety and biomarker data from upliFT-D and obtain regulatory feedback on FTD-GRN registrational trial design in 1H 2026 Advancing differentiated preclinical program for Huntington’s with clinical candidate selection expected in 2H 2026 Cash runway through 1Q 2027 PHILADELPHIA, March 03, 2026 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (Nasdaq: PASG), a clinical stage genetic medicines company focused on improving the lives of patients with neurodegenerative diseases, today reported financial results for the fourth quarter and year ended December 31, 2025, and provided recent business highlights. “We are proud of the progress made in 2025 as we meaningfully advanced our PBFT02 clinical program for the treatment of genetic forms of frontotemporal dementia,” said Will Chou, M.D., president and chief executive officer of Passage Bio. “As we enter 2026, we are excited by the strong enrollment momentum in our upliFT-D clinical study and look forward to sharing important data and regulatory updates in the first half of the year. FTD is a devastating disease, and the clinical unmet need remains substantial. We remain committed to advancing our program in the hope that we might one day offer patients and their families a therapy to redefine the course of their disease.” Recent Highlights Enrolled first three FTD-GRN patients in Cohort 3 of ongoing upliFT-D study: The first three patients in Cohort 3 of the upliFT-D trial have been enrolled and patient dosings are anticipated over the coming weeks. Cohort 3 will evaluate Dose 2 PBFT02 and is expected to consist of a total of 10 patients, with patient enrollment occurring in parallel across current active trial sites in Australia, Brazil, Canada, Portugal, and the United States. The cohort will be enrolled under the recently amended protocol, which focuses on patients earlier in their disease progression and includes a short course of low-dose prophylactic anticoagulation. The company plans to report updated interim safety and biomarker data from upliFT-D and obtain regulatory feedback on registrational trial design for FTD-GRN in the first half of 2026. Treated first FTD-C9orf72 patient with Dose 2 PBFT02 in Cohort 4 of upliFT-D stu...

Actively enrolling Cohort 3 (FTD-GRN) and Cohort 4 (FTD-C9orf72) patients in ongoing upliFT-D study Aligned with the U.S. Food and Drug Administration (FDA) on an analytical approach to establish comparability of a high-productivity, suspension-based PBFT02 manufacturing process On track to obtain regulatory feedback on FTD-GRN registrational trial design in 1H 2026 Cash runway into 1Q 2027 PHILADELPHIA, Nov. 10, 2025 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (Nasdaq: PASG), a clinical stage genetic medicines company focused on improving the lives of patients with neurodegenerative diseases, today reported financial results for the third quarter ended September 30, 2025, and provided recent business highlights. “We are pleased to report that we have opened enrollment in our third FTD-GRN and first FTD-C9orf72 patient cohorts in our ongoing upliFT-D clinical trial of PBFT02. We recognize the urgent need for the development of disease-modifying therapies for the FTD patient community, and we remain focused on advancing our study expeditiously,” said Will Chou, M.D., president and chief executive officer of Passage Bio. “In addition, we completed a successful meeting with the FDA where we aligned on key elements of an analytical comparability plan to support the future use of our high-productivity, suspension-based manufacturing process for PBFT02 in a registrational study. As we look towards the first half of 2026, we are excited to share additional data to further inform our understanding of the potential of PBFT02 and initiate discussions with the FDA on a registrational study design in FTD-GRN.” Recent Highlights Actively enrolling Cohort 3 (FTD-GRN) and Cohort 4 (FTD-C9orf72) patients in upliFT-D study: Cohort 3 is expected to consist of five to 10 FTD-GRN patients, with patient enrollment occurring in parallel; Cohort 4 is expected to consist of three to five FTD-C9orf72 patients. Both cohorts will evaluate Dose 2 PBFT02. The company recently amended the upliFT-D trial protocol to introduce a short course of low-dose prophylactic anticoagulation and 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 was submitted to all global trial sites and relevant health authorities and has been implemented at initial trial si...

Completed dosing of FTD-GRN Cohort 2 in upliFT-D study Reported updated interim data showing that PBFT02 continued to demonstrate robust, durable elevation in CSF PGRN levels and improvement in plasma NfL, a disease progression biomarker, compared to natural history Submitted amended upliFT-D study protocol to global trial sites and health authorities Cash runway into 1Q 2027 PHILADELPHIA, Aug. 12, 2025 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (Nasdaq: PASG), a clinical stage genetic medicines company focused on improving the lives of patients with neurodegenerative diseases, today reported financial results for the second quarter ended June 30, 2025, and provided recent business highlights. “We continue to be encouraged by the emerging data from our upliFT-D clinical trial, which underscore the potential of PBFT02 to offer differentiated therapy for the FTD-GRN patient community. As we remain focused on execution, we are pleased to have completed dosing of Cohort 2 and submitted the amended protocol to global trial sites and health authorities as planned,” said Will Chou, M.D., president and chief executive officer of Passage Bio. “We look forward to initiating enrollment of our third FTD-GRN and first FTD-C9orf72 patient cohorts once the protocol amendment is approved at trial sites and remain on track to engage with health authorities in the first half of 2026 to seek guidance on the registrational pathway for the program in FTD-GRN.” Recent 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 lig...

Treated first FTD-GRN patient with Dose 2 PBFT02 and enrolled second patient; several additional patients being evaluated for trial eligibility Announced presentation of process development data of a high productivity, suspension-based manufacturing process for PBFT02 at American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting Cash runway into 1Q 2027 PHILADELPHIA, May 13, 2025 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (Nasdaq: PASG), a clinical stage genetic medicines company focused on improving the lives of patients with neurodegenerative diseases, today reported financial results for the first quarter ended March 31, 2025, and provided recent business highlights. “We made steady progress in executing against our core operational objectives this quarter as we focus on the enrollment of FTD-GRN and FTD-C9orf72 patients in our ongoing global Phase 1/2 upliFT-D trial of PBFT02,” said Will Chou, M.D., president and chief executive officer of Passage Bio. “As we turn towards the second half of the year, we look forward to delivering additional data from upliFT-D to add to our understanding of PBFT02’s safety profile, durability of progranulin expression, dose-response, and impact on plasma neurofilament levels, a disease progression biomarker that is well characterized in FTD natural history literature. We expect these data to foster meaningful engagement with health authorities in the first half of 2026 as we seek guidance on the registrational pathway for the program in FTD-GRN.” Recent Highlights Treated first FTD-GRN patient with Dose 2 PBFT02 and enrolled second patient. with several additional patients being evaluated for trial eligibility: Dose 2, which is fifty percent lower than Dose 1, was introduced given the robust CSF progranulin (PGRN) expression achieved at Dose 1 and to aid future discussions with healthy authorities regarding a registrational study design. A total of three patients in Cohort 2 will receive Dose 2. Following completion of Cohort 2, the Independent Data Monitoring Committee will review available safety data prior to the commencement of Cohort 3 (n=3-5). The company plans to report interim safety and biomarker data from Dose 2 and 12-month data from Dose 1 patients in the second half of 2025. Opened enrollment for FTD-C9orf72 patients in upliFT-D study: The company is currently enrolling up to five symptomatic FTD-C9orf7...