Passage Bio Reports First Quarter 2025 Financial Results and Provides Recent Business Highlights
- Development of new suspension-based manufacturing process yields over 1,000 doses at Dose 2 with 90% purity
- Cash runway extended into Q1 2027
- Reduced net loss to $15.4M from $16.7M year-over-year
- R&D expenses decreased by 33% to $7.7M from $11.5M
- Cash position declined to $63.4M from $104.5M year-over-year
- Still operating at a significant net loss of $15.4M for Q1 2025
Insights
Passage Bio shows promising progress in FTD trials with optimized dosing strategy and manufacturing improvements despite posting quarterly loss.
Passage Bio's Q1 update presents a measured clinical advancement of their lead gene therapy candidate PBFT02 for frontotemporal dementia. The company has successfully treated their first FTD-GRN patient with a 50% reduced dose (Dose 2) and enrolled a second patient, which represents a strategically important development. This dose reduction wasn't implemented due to safety concerns but rather reflects the robust CSF progranulin expression achieved with the higher dose—suggesting potential efficacy at lower concentrations.
The expansion to include FTD-C9orf72 patients is particularly noteworthy. These patients share TDP-43 pathology with FTD-GRN patients, and preclinical evidence suggests progranulin overexpression could benefit both populations. This represents a pipeline-in-a-product strategy that could significantly expand PBFT02's market potential without developing an entirely new therapeutic.
The company's manufacturing improvements are substantial from a development perspective. Their new suspension-based process at 200-liter scale reportedly yields over 1,000 doses per batch with >90% purity and >70% full capsids—metrics that substantially exceed industry standards for AAV-based gene therapies. These improvements address one of the fundamental challenges in gene therapy commercialization: manufacturing scale and consistency.
Their cash runway extension into Q1 2027 provides approximately 22 months of operational capability, sufficient to reach their stated milestone of seeking regulatory feedback on a registrational trial design in H1 2026. The 36% decrease in R&D expenses (from $11.5M to $7.7M year-over-year) likely reflects the company's transition from broader pipeline development to focused execution on the lead PBFT02 program.
Passage Bio demonstrates operational efficiency with reduced cash burn and extended runway, though clinical progress remains early-stage.
The financial picture for Passage Bio shows a company making deliberate efforts to extend its cash runway while advancing its clinical programs. With $63.4 million in cash and equivalents as of March 31, 2025, the company has experienced a 39% year-over-year reduction in cash reserves from the $104.5 million reported in Q1 2024. However, management has successfully stretched this capital, projecting runway into Q1 2027—a conservative but sufficient timeframe to reach critical clinical and regulatory milestones.
Quarterly net losses improved slightly to $15.4 million ($0.25 per share), compared to $16.7 million ($0.30 per share) in the same period last year. This 7.8% reduction in net loss reflects effective cost management, with R&D expenses decreasing by 33% to $7.7 million and G&A expenses declining by 6.2% to $6.1 million. The substantial reduction in R&D spending indicates a shift from broader pipeline investment to focused development of their lead FTD programs.
From a manufacturing economics perspective, the new suspension-based process represents a potential significant cost advantage. The reported capability to produce over 1,000 doses at Dose 2 from a single batch could dramatically reduce cost-of-goods-sold (COGS), a critical factor for commercial viability of gene therapies. This improved manufacturing efficiency could translate to improved gross margins if PBFT02 reaches commercialization.
While the clinical progress is encouraging, investors should note that Phase 1/2 data remains preliminary, with pivotal regulatory discussions not expected until H1 2026. The company has prioritized capital efficiency and core program advancement, which appears to be the appropriate strategy given current biotech funding challenges. The expansion to include FTD-C9orf72 patients represents potential market expansion without significant additional development costs, potentially improving the overall commercial outlook for PBFT02.
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-C9orf72 patients to receive Dose 2 PBFT02 following amendment of the upliFT-D trial protocol to include this patient population. Preclinical studies have demonstrated that overexpression of PGRN can slow neurodegeneration and reduce TDP-43 pathology, which underlies this disease.
- Announced presentation of process development data of a high productivity, suspension-based manufacturing process for PBFT02 at ASGCT: The internally developed suspension-based, GMP-ready manufacturing process for PBFT02 at 200-liter scale substantially improved productivity, capsid purity and the percentage of full capsids compared to the current adherent-based manufacturing process. A single batch of the suspension process is estimated to yield more than 1,000 doses at Dose 2 with over
90% purity and over70% full capsids. Additional details will be shared during a poster session from 6:00 – 7:30 p.m. CT today at the ASGCT 28th Annual Meeting, being held in New Orleans, LA and virtually. The company plans to engage with health authorities later this year to obtain feedback on comparability of the suspension process to the original process.
Anticipated Upcoming Milestones:
FTD-GRN
- Report 12-month data from Dose 1 and interim safety and biomarker data from Dose 2 in 2H 2025
- Seek regulatory feedback on registrational trial design in 1H 2026
FTD-C9orf72
- Initiate dosing of FTD-C9orf72 patients in 1H 2025
First Quarter 2025 Financial Results
- Cash Position: Cash, cash equivalents and marketable securities were
$63.4 million as of March 31, 2025, as compared to$104.5 million as of March 31, 2024. The company expects current cash, cash equivalents and marketable securities to fund operations into 1Q 2027. - Research and Development (R&D) Expenses: R&D expenses were
$7.7 million for the quarter ended March 31, 2025, as compared to$11.5 million for the quarter ended March 31, 2024. - General and Administrative (G&A) Expenses: G&A expenses were
$6.1 million for the quarter ended March 31, 2025, as compared to$6.5 million for the quarter ended March 31, 2024. - Net Loss: Net loss was
$15.4 million , or$0.25 per basic and diluted share, for the quarter ended March 31, 2025, as compared to a net loss of$16.7 million , or$0.30 per basic and diluted share, for the quarter ended March 31, 2024.
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,” “could,” “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) | March 31, 2025 | December 31, 2024 | ||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 63,357 | $ | 37,573 | ||||
| Marketable securities | — | 39,183 | ||||||
| Prepaid expenses and other current assets | 2,121 | 838 | ||||||
| Prepaid research and development | 1,191 | 1,221 | ||||||
| Total current assets | 66,669 | 78,815 | ||||||
| Property and equipment, net | 5,523 | 9,331 | ||||||
| Right of use assets - operating leases | 13,540 | 13,803 | ||||||
| Other assets | 274 | 463 | ||||||
| Total assets | $ | 86,006 | $ | 102,412 | ||||
| Liabilities and stockholders’ equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 1,458 | $ | 742 | ||||
| Accrued expenses and other current liabilities | 3,994 | 6,707 | ||||||
| Non-refundable sublicense and transition services payments received | 8,732 | 8,226 | ||||||
| Operating lease liabilities | 3,641 | 3,688 | ||||||
| Total current liabilities | 17,825 | 19,363 | ||||||
| Operating lease liabilities - noncurrent | 21,475 | 21,788 | ||||||
| Total liabilities | 39,300 | 41,151 | ||||||
| Stockholders’ equity: | ||||||||
| Preferred stock, | — | — | ||||||
| Common stock, | 6 | 6 | ||||||
| Additional paid‑in capital | 721,340 | 720,482 | ||||||
| Accumulated other comprehensive income (loss) | — | 8 | ||||||
| Accumulated deficit | (674,640) | (659,235) | ||||||
| Total stockholders’ equity | 46,706 | 61,261 | ||||||
| Total liabilities and stockholders’ equity | $ | 86,006 | $ | 102,412 | ||||
| Passage Bio, Inc. | ||||||||
| Statements of Operations and Comprehensive Loss | ||||||||
| (Unaudited) | ||||||||
| Three Months Ended March 31, | ||||||||
| (in thousands, except share and per share data) | 2025 | 2024 | ||||||
| Operating expenses: | ||||||||
| Research and development | $ | 7,737 | $ | 11,535 | ||||
| General and administrative | 6,085 | 6,515 | ||||||
| Impairment of long-lived assets | 2,637 | — | ||||||
| Loss from operations | (16,459 | ) | (18,050 | ) | ||||
| Other income (expense), net | 1,054 | 1,339 | ||||||
| Net loss | $ | (15,405 | ) | $ | (16,711 | ) | ||
| Per share information: | ||||||||
| Net loss per share of common stock, basic and diluted | $ | (0.25 | ) | $ | (0.30 | ) | ||
| Weighted average common shares outstanding, basic and diluted | 62,109,991 | 56,295,540 | ||||||
| Comprehensive loss: | ||||||||
| Net loss | $ | (15,405 | ) | $ | (16,711 | ) | ||
| Unrealized gain (loss) on marketable securities | (8 | ) | (26 | ) | ||||
| Comprehensive loss | $ | (15,413 | ) | $ | (16,737 | ) | ||
For further information, please contact:
Investors:
Stuart Henderson
Passage Bio
shenderson@passagebio.com
Media:
Mike Beyer
Sam Brown Inc. Healthcare Communications
312.961.2502
MikeBeyer@sambrown.com
FAQ
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