PHILADELPHIA, PA — Passage Bio, Inc. (Nasdaq: PASG) reported third-quarter financial results and outlined steady progress across its upliFT-D gene therapy program, positioning the company for critical regulatory and clinical milestones in the first half of 2026. The company is developing PBFT02, an AAV-delivered therapy for frontotemporal dementia (FTD) caused by GRN or C9orf72 mutations — conditions with no approved disease-modifying treatments.
CEO Will Chou said enrollment is now open for Cohort 3 of FTD-GRN patients and Cohort 4 of FTD-C9orf72 patients in the ongoing Phase 1/2 study. Both groups will receive Dose 2 of PBFT02. Recent protocol amendments allow enrollment of patients with prodromal or mild cognitive impairment, while excluding more advanced cases, and introduce short-course, low-dose prophylactic anticoagulation to support patient safety. Updated interim safety and biomarker data are expected in the first half of next year.
“We recognize the urgent need for disease-modifying therapies for the FTD community, and we remain focused on advancing our study expeditiously,” Chou said.
Manufacturing Advances Clear Path Toward Registrational Planning
Passage Bio also announced alignment with the FDA on a comparability plan for its high-productivity, suspension-based manufacturing process. The updated approach, now executed at 200-liter scale, delivers substantially higher purity and yields than the adherent-based process—producing more than 1,000 PBFT02 doses from a single batch with over 90% purity and more than 70% full capsids.
The company expects to use this process in future registrational development and plans to seek FDA feedback on a potential FTD-GRN registrational study design in the first half of 2026, including the possibility of a single-arm trial compared against natural history controls.
Preparing for a Data-Rich 2026
Key upcoming milestones include:
- Updated interim Dose 2 safety and biomarker data in early 2026
- FDA feedback on registrational trial design in FTD-GRN in the first half of 2026
To support trial recruitment, Passage Bio is expanding its collaboration with InformedDNA to offer no-cost genetic counseling and testing for adults diagnosed with FTD.
Third-Quarter Financials Show Lower Spend, Solid Runway
The company ended the quarter with $52.8 million in cash, cash equivalents, and marketable securities, providing runway into the first quarter of 2027. R&D expenses fell to $4.3 million from $8.7 million a year ago, reflecting reduced clinical manufacturing and development costs as earlier cohorts concluded. G&A expenses also declined, dropping to $4.3 million from $7.3 million.
Net loss narrowed significantly to $7.7 million from $19.3 million in the prior-year quarter, aided by tighter operating expenses following the July 2025 reverse stock split.
With multiple cohorts enrolling, a maturing manufacturing platform, and upcoming regulatory discussions, Passage Bio is entering 2026 with a clearer path toward late-stage development of PBFT02 — and rising anticipation within a field where therapeutic options remain scarce.
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