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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
Anticipated Upcoming Milestones:
Third Quarter 2025 Financial Results
About upliFT-D (NCT04747431)
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. 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. More information about upliFT-D can be found here.
About Passage Bio
Passage Bio (Nasdaq: PASG) is a clinical stage genetic medicines company on a mission to improve the lives of patients with neurodegenerative diseases. Our primary focus is the development and advancement of cutting-edge, one-time therapies designed to target the underlying pathology of these conditions. Passage Bio’s lead product candidate, PBFT02, seeks to treat neurodegenerative conditions, including frontotemporal dementia, by elevating progranulin levels to restore lysosomal function and slow disease progression.
To learn more about Passage Bio and our steadfast commitment to protecting patients and families against loss in neurodegenerative conditions, please visit: passagebio.com.
Forward-Looking Statements
This press release contains “forward-looking statements” within the meaning of, and made pursuant to the safe harbor provisions of, the Private Securities Litigation Reform Act of 1995, including, but not limited to: our expectations about timing and execution of anticipated milestones, including the progress of clinical studies and the availability of clinical data from such trials; the initiation of dosing of FTD-C9orf72 patients; timing of feedback from regulatory authorities; our expectations about our collaborators’ and partners’ ability to execute key initiatives; our expectations about cash runway; and the ability of our product candidates to treat their respective target CNS disorders. These forward-looking statements may be accompanied by such words as “aim,” “anticipate,” “believe,” “continue,” “could,” “should,” “target,” “estimate,” “expect,” “forecast,” “goal,” “intend,” “may,” “might,” “plan,” “potential,” “possible,” “will,” “would,” and other words and terms of similar meaning. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including: our ability to develop and obtain regulatory approval for our product candidates; the timing and results of preclinical studies and clinical trials; risks associated with clinical trials, including our ability to adequately manage clinical activities, unexpected concerns that may arise from additional data or analysis obtained during clinical trials, regulatory authorities may require additional information or further studies, or may fail to approve or may delay approval of our drug candidates; the occurrence of adverse safety events; the risk that positive results in a preclinical study or clinical trial may not be replicated in subsequent trials or success in early stage clinical trials may not be predictive of results in later stage clinical trials; failure to protect and enforce our intellectual property, and other proprietary rights; our dependence on collaborators and other third parties for the development and manufacture of product candidates and other aspects of our business, which are outside of our full control; risks associated with current and potential delays, work stoppages, or supply chain disruptions; and the other risks and uncertainties that are described in the Risk Factors section in documents the company files from time to time with the Securities and Exchange Commission (SEC), and other reports as filed with the SEC. Passage Bio undertakes no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise.
| Passage Bio, Inc. Balance Sheets | ||||||||
| (Unaudited) | ||||||||
| (in thousands, except share and per share data) | September 30, 2025 | December 31, 2024 | ||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 52,773 | $ | 37,573 | ||||
| Marketable securities | — | 39,183 | ||||||
| Prepaid expenses and other current assets | 1,637 | 838 | ||||||
| Prepaid research and development | 1,320 | 1,221 | ||||||
| Total current assets | 55,730 | 78,815 | ||||||
| Property and equipment, net | 5,159 | 9,331 | ||||||
| Right of use assets - operating leases | 13,001 | 13,803 | ||||||
| Other assets | 270 | 463 | ||||||
| Total assets | $ | 74,160 | $ | 102,412 | ||||
| Liabilities and stockholders’ equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 952 | $ | 742 | ||||
| Accrued expenses and other current liabilities | 4,001 | 6,707 | ||||||
| Non-refundable sublicense and transition services payments | 13,750 | 8,226 | ||||||
| Operating lease liabilities | 3,542 | 3,688 | ||||||
| Total current liabilities | 22,245 | 19,363 | ||||||
| Operating lease liabilities - noncurrent | 20,795 | 21,788 | ||||||
| Total liabilities | 43,040 | 41,151 | ||||||
| Stockholders’ equity: | ||||||||
| Preferred stock, $0.0001 par value: 10,000,000 shares authorized; no shares issued and outstanding at both September 30, 2025 and December 31, 2024 | — | — | ||||||
| Common stock, $0.0001 par value: 300,000,000 shares authorized; 3,178,710 shares issued and outstanding at September 30, 2025 and 3,161,503 shares issued and outstanding at December 31, 2024 | — | — | ||||||
| Additional paid‑in capital | 722,894 | 720,488 | ||||||
| Accumulated other comprehensive income (loss) | — | 8 | ||||||
| Accumulated deficit | (691,774 | ) | (659,235 | ) | ||||
| Total stockholders’ equity | 31,120 | 61,261 | ||||||
| Total liabilities and stockholders’ equity | $ | 74,160 | $ | 102,412 | ||||
| Passage Bio, Inc. Statements of Operations and Comprehensive Loss (Unaudited) | ||||||||||||||||
| Three Months Ended September 30, | Nine Months Ended September 30, | |||||||||||||||
| (in thousands, except share and per share data) | 2025 | 2024 | 2025 | 2024 | ||||||||||||
| Operating expenses: | ||||||||||||||||
| Research and development | $ | 4,307 | $ | 8,656 | $ | 17,858 | $ | 30,621 | ||||||||
| General and administrative | 4,348 | 7,251 | 14,953 | 20,276 | ||||||||||||
| Impairment of long-lived assets | — | 4,795 | 2,637 | 5,233 | ||||||||||||
| Loss from operations | (8,655 | ) | (20,702 | ) | (35,448 | ) | (56,130 | ) | ||||||||
| Other income (expense), net | 906 | 1,362 | 2,909 | 4,088 | ||||||||||||
| Net loss | $ | (7,749 | ) | $ | (19,340 | ) | $ | (32,539 | ) | $ | (52,042 | ) | ||||
| Per share information: | ||||||||||||||||
| Net loss per share of common stock, basic and diluted | $ | (2.44 | ) | $ | (6.15 | ) | $ | (10.26 | ) | $ | (17.04 | ) | ||||
| Weighted average common shares outstanding, basic and diluted | 3,178,710 | 3,146,582 | 3,170,573 | 3,054,440 | ||||||||||||
| Comprehensive loss: | ||||||||||||||||
| Net loss | $ | (7,749 | ) | $ | (19,340 | ) | $ | (32,539 | ) | $ | (52,042 | ) | ||||
| Unrealized gain (loss) on marketable securities | — | 99 | (8 | ) | 75 | |||||||||||
| Comprehensive loss | $ | (7,749 | ) | $ | (19,241 | ) | $ | (32,547 | ) | $ | (51,967 | ) | ||||
For further information, please contact:
Investors:
Stuart Henderson
Passage Bio
[email protected]
Media:
Mike Beyer
Sam Brown Inc. Healthcare Communications
312.961.2502
[email protected]
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