Passage Bio Reports Fourth Quarter and Full Year 2024 Financial Results and Provides Recent Business Highlights
Passage Bio (NASDAQ: PASG) reported its Q4 and full-year 2024 financial results, highlighting significant progress in its PBFT02 program for FTD-GRN patients. Interim data showed promising results, with Dose 1 PBFT02 increasing CSF progranulin levels from below 3 ng/mL to 13-27 ng/mL at six months and 22-34 ng/mL at 12 months.
The company has enrolled its first patient for Dose 2 PBFT02, which is 50% lower than Dose 1. Financial results show cash position of $76.8 million as of December 31, 2024, with runway extended into Q1 2027. Net loss for 2024 was $64.8 million ($1.07 per share), improved from $102.1 million in 2023. R&D expenses decreased to $40.2 million from $61.4 million, while G&A expenses reduced to $25.0 million from $41.6 million year-over-year.
Passage Bio (NASDAQ: PASG) ha riportato i risultati finanziari del quarto trimestre e dell'intero anno 2024, evidenziando progressi significativi nel suo programma PBFT02 per pazienti con FTD-GRN. I dati preliminari hanno mostrato risultati promettenti, con il Dose 1 di PBFT02 che aumenta i livelli di progranulina nel liquido cerebrospinale da sotto 3 ng/mL a 13-27 ng/mL a sei mesi e 22-34 ng/mL a dodici mesi.
L'azienda ha arruolato il suo primo paziente per il Dose 2 di PBFT02, che è inferiore del 50% rispetto al Dose 1. I risultati finanziari mostrano una posizione di cassa di 76,8 milioni di dollari al 31 dicembre 2024, con un'estensione della liquidità fino al primo trimestre del 2027. La perdita netta per il 2024 è stata di 64,8 milioni di dollari (1,07 dollari per azione), migliorata rispetto ai 102,1 milioni di dollari del 2023. Le spese per R&S sono diminuite a 40,2 milioni di dollari da 61,4 milioni, mentre le spese generali e amministrative sono scese a 25,0 milioni di dollari da 41,6 milioni anno su anno.
Passage Bio (NASDAQ: PASG) informó sus resultados financieros del cuarto trimestre y del año completo 2024, destacando avances significativos en su programa PBFT02 para pacientes con FTD-GRN. Los datos interinos mostraron resultados prometedores, con la Dosis 1 de PBFT02 aumentando los niveles de progranulina en el líquido cefalorraquídeo de menos de 3 ng/mL a 13-27 ng/mL a los seis meses y 22-34 ng/mL a los doce meses.
La compañía ha inscrito a su primer paciente para la Dosis 2 de PBFT02, que es un 50% menor que la Dosis 1. Los resultados financieros muestran una posición de efectivo de 76,8 millones de dólares al 31 de diciembre de 2024, con un plazo extendido hasta el primer trimestre de 2027. La pérdida neta para 2024 fue de 64,8 millones de dólares (1,07 dólares por acción), mejorando desde 102,1 millones de dólares en 2023. Los gastos de I+D disminuyeron a 40,2 millones de dólares desde 61,4 millones, mientras que los gastos generales y administrativos se redujeron a 25,0 millones de dólares desde 41,6 millones año tras año.
패시지 바이오 (NASDAQ: PASG)는 2024년 4분기 및 전체 연도 재무 결과를 보고하며, FTD-GRN 환자를 위한 PBFT02 프로그램에서 중요한 진전을 강조했습니다. 중간 데이터는 유망한 결과를 보여주었습니다, 1차 투여에서 PBFT02가 뇌척수액의 프로그라눌린 수치를 3 ng/mL 미만에서 6개월 후 13-27 ng/mL, 12개월 후 22-34 ng/mL로 증가시켰습니다.
회사는 1차 투여량보다 50% 낮은 2차 투여량 PBFT02의 첫 환자를 등록했습니다. 재무 결과는 2024년 12월 31일 기준 7,680만 달러의 현금 보유액을 보여주며, 2027년 1분기까지의 자금 여유가 있습니다. 2024년 순손실은 6,480만 달러 (주당 1.07 달러)로, 2023년의 1억 2,210만 달러에서 개선되었습니다. 연구 및 개발 비용은 6,140만 달러에서 4,020만 달러로 감소했으며, 일반 관리 비용은 4,160만 달러에서 2,500만 달러로 감소했습니다.
Passage Bio (NASDAQ: PASG) a publié ses résultats financiers pour le quatrième trimestre et l'année entière 2024, soulignant des progrès significatifs dans son programme PBFT02 pour les patients atteints de FTD-GRN. Les données préliminaires ont montré des résultats prometteurs, avec la Dose 1 de PBFT02 augmentant les niveaux de progranuline dans le liquide céphalorachidien de moins de 3 ng/mL à 13-27 ng/mL après six mois et à 22-34 ng/mL après douze mois.
L'entreprise a recruté son premier patient pour la Dose 2 de PBFT02, qui est inférieure de 50 % à la Dose 1. Les résultats financiers montrent une position de trésorerie de 76,8 millions de dollars au 31 décembre 2024, avec une prolongation de la liquidité jusqu'au premier trimestre 2027. La perte nette pour 2024 s'élevait à 64,8 millions de dollars (1,07 dollar par action), en amélioration par rapport à 102,1 millions de dollars en 2023. Les dépenses de R&D ont diminué à 40,2 millions de dollars contre 61,4 millions de dollars, tandis que les dépenses générales et administratives ont été réduites à 25,0 millions de dollars contre 41,6 millions de dollars d'une année sur l'autre.
Passage Bio (NASDAQ: PASG) hat seine Finanzzahlen für das vierte Quartal und das gesamte Jahr 2024 veröffentlicht und dabei bedeutende Fortschritte in seinem PBFT02-Programm für FTD-GRN-Patienten hervorgehoben. Vorläufige Daten zeigten vielversprechende Ergebnisse, wobei die Dosis 1 von PBFT02 die Progranulin-Spiegel im Liquor cerebrospinalis von unter 3 ng/mL auf 13-27 ng/mL nach sechs Monaten und auf 22-34 ng/mL nach zwölf Monaten erhöhte.
Das Unternehmen hat seinen ersten Patienten für die Dosis 2 von PBFT02 rekrutiert, die 50 % niedriger ist als die Dosis 1. Die Finanzzahlen zeigen eine Barreserve von 76,8 Millionen Dollar zum 31. Dezember 2024, mit einer Ausdehnung des finanziellen Spielraums bis ins erste Quartal 2027. Der Nettoverlust für 2024 betrug 64,8 Millionen Dollar (1,07 Dollar pro Aktie), eine Verbesserung gegenüber 102,1 Millionen Dollar im Jahr 2023. Die F&E-Ausgaben sanken auf 40,2 Millionen Dollar von 61,4 Millionen Dollar, während die allgemeinen und Verwaltungskosten auf 25,0 Millionen Dollar von 41,6 Millionen Dollar im Jahresvergleich gesenkt wurden.
- PBFT02 showed strong efficacy with increased CSF progranulin levels
- 13% reduction in disease progression biomarker (plasma NfL)
- Extended cash runway into Q1 2027
- 37% reduction in net loss year-over-year
- Significant decrease in operating expenses
- Net loss of $64.8M in 2024
- Cash position decreased from $114.3M to $76.8M year-over-year
Insights
Passage Bio's Q4 and full-year 2024 results showcase promising developments for their lead candidate PBFT02 in frontotemporal dementia with progranulin mutations (FTD-GRN). The interim data reveals consistent, durable increases in CSF progranulin levels from below 3 ng/mL at baseline to 13-27 ng/mL at six months and 22-34 ng/mL at twelve months. More significantly, the 13% reduction in plasma neurofilament light chain (NfL) levels contrasts sharply with the expected 29% annual increase in untreated patients, suggesting potential disease-modifying activity.
The company has strategically introduced a lower dose cohort (50% of the original dose) based on the robust progranulin expression seen at the higher dose. This dose-ranging approach strengthens their position for future regulatory discussions about pivotal trial design, expected in 1H 2026.
Financially, Passage has improved operational efficiency, reducing both R&D expenses ($40.2M vs $61.4M in 2023) and G&A costs ($25.0M vs $41.6M). Their net loss narrowed to $64.8M from $102.1M year-over-year, while extending cash runway into Q1 2027. With $76.8M in cash as of December 2024, they appear adequately funded to reach critical upcoming milestones in both FTD-GRN and their planned expansion into FTD-C9orf72.
The biomarker data from Passage Bio's PBFT02 gene therapy trial represents a significant development in addressing the underlying pathology of FTD-GRN. The substantial increase in CSF progranulin levels (achieving 13-34 ng/mL) is noteworthy as progranulin deficiency is directly implicated in the neurodegeneration observed in these patients.
The reported 13% reduction in plasma NfL at 12 months post-treatment is particularly encouraging. NfL is an established biomarker of axonal damage, and its levels typically rise as neurodegeneration progresses. The observed reduction, contrasted with the expected 29% annual increase in untreated patients, suggests PBFT02 may be slowing the neurodegenerative process.
The approach of testing a lower dose (Dose 2) represents sound clinical development strategy. Given the robust progranulin expression at Dose 1, establishing a dose-response relationship will be important for regulatory discussions. Expansion to include C9orf72 mutation carriers is also scientifically justified, as both mutations result in similar clinical syndromes despite different underlying mechanisms.
While these biomarker results are promising, it remains essential to correlate them with clinical outcomes in larger patient cohorts. The upcoming 12-month data in 2H 2025 will be critical in determining whether these biochemical changes translate to meaningful clinical benefits for FTD patients who currently have no disease-modifying treatment options.
Interim data from Dose 1 PBFT02 demonstrated durable, elevated CSF PGRN and early evidence of reduction in plasma NfL levels, a disease progression biomarker, as compared to published natural history
Enrolled first FTD-GRN patient to be treated with Dose 2 PBFT02,
Expect to report 12-month data from Dose 1 and interim safety and biomarker data from Dose 2 in 2H 2025; plan to seek regulatory feedback on FTD-GRN pivotal trial design in 1H 2026
Extended cash runway into 1Q 2027
PHILADELPHIA, March 04, 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 fourth quarter and year ended December 31, 2024, and provided recent business highlights.
“We are pleased to report strong performance in 2024 as we meaningfully advanced our PBFT02 program, delivering promising data in FTD-GRN patients showing robust, durable progranulin expression and early evidence of improvement in a disease progression biomarker. Furthermore, we completed the process development and scale-up of a high-productivity, suspension-based manufacturing process for PBFT02 and are well-positioned for late-stage development,” said Will Chou, M.D., president and chief executive officer of Passage Bio. “As we enter 2025, we remain focused on execution of our ongoing upliFT-D trial in FTD-GRN and are excited to expand enrollment to include FTD patients with C9orf72 gene mutations. We look forward to building upon the encouraging data generated to date and engaging with health authorities to explore the registrational pathway for this promising, one-time therapy to address a significant unmet patient need.”
Recent Highlights:
- Interim data from FTD-GRN patients in upliFT-D clinical trial demonstrated that Dose 1 PBFT02 consistently increased cerebrospinal fluid (CSF) progranulin (PGRN) and showed early evidence of improvement in a disease progression biomarker: Dose 1 PBFT02 consistently increased CSF PGRN levels in all patients from below 3 ng/mL at baseline to 13 – 27 ng/mL at six months (n=4) and 22 – 34 ng/mL at 12 months (n=2). In addition, plasma neurofilament light chain (NfL) levels, which are associated with disease progression, were
13% lower than baseline on average at 12 months (n=2) post-treatment compared to an expected increase of29% per year based on published natural history data from untreated symptomatic FTD-GRN patients. - Enrolled first FTD-GRN patient to receive Dose 2 PBFT02: Given the robust CSF PGRN levels achieved at Dose 1 and to aid future discussions with health authorities regarding a registrational study design, the company introduced a lower dose level, Dose 2, which is fifty percent of Dose 1. Dose 2 is expected to be administered to the three remaining FTD-GRN patients in Cohort 2 of the upliFT-D study. The first of these three patients has been enrolled, and multiple patients are being evaluated for eligibility across seven active trial sites in Brazil, Canada, Portugal, and the United States.
- Extended cash runway into 1Q 2027 by moving to outsourced analytical testing model and reducing operating expenses: In January, the company implemented a series of measures in connection with moving to an outsourced analytical testing model and to reduce operating expenses. As a result, the company’s cash runway was extended into the first quarter of 2027 as it continues to focus on the execution of the ongoing upliFT-D clinical trial in FTD-GRN and FTD-C9orf72 and the advancement of its preclinical program in Huntington’s disease.
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
Fourth Quarter and Full-Year 2024 Financial Results
- Cash Position: Cash, cash equivalents and marketable securities were
$76.8 million as of December 31, 2024, as compared to$114.3 million as of December 31, 2023. 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
$9.6 million for the quarter ended December 31, 2024, and$40.2 million for the year ended December 31, 2024, compared to$12.1 million and$61.4 million for the same quarter and year ended in 2023, respectively. - General and Administrative (G&A) Expenses: G&A expenses were
$4.7 million for the quarter ended December 31, 2024, and$25.0 million for the year ended December 31, 2024, compared to$6.3 million and$41.6 million for the same quarter and year ended in 2023, respectively. - Net Loss: Net loss was
$12.7 million , or$0.20 per basic and diluted share, for the quarter ended December 31, 2024 and$64.8 million , or$1.07 per basic and diluted share, for the year ended December 31, 2024, compared to a net loss of$16.8 million , or$0.30 per basic and diluted share, for the quarter ended December 31, 2023 and$102.1 million , or$1.86 per basic and diluted share, for the year ended December 31, 2023.
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 PBFT02
PBFT02 is a gene therapy that utilizes an AAV1 viral vector to deliver, through ICM administration, a functional GRN gene that encodes PGRN. This vector construct and delivery approach aim to elevate PGRN levels in the central nervous system to alter the course of neurodegenerative diseases. Interim clinical data from the Phase 1/2 upliFT-D study in FTD-GRN participants showed that gene replacement by ICM administration of PBFT02 resulted in robust PGRN elevations in the CSF.
The potential clinical benefit of PBFT02 is supported by extensive preclinical studies. In non-human primates, a single ICM administration of PBFT02 led to broad vector distribution throughout the CNS, and robust, dose-dependent elevations in PGRN levels in CSF. An NHP study also demonstrated that AAV1 was particularly proficient at transducing ependymal cells. In a murine FTD model, PBFT02 administration improved lysosomal function and reduced neuroinflammation.
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 initiation of dosing of FTD-C9orf72 patients, timing of feedback from regulatory authorities, the progress of clinical studies and the availability of clinical data from such trials; our expectations about our collaborators’ and partners’ ability to execute key initiatives; the financial impact of the restructuring and reduction in workforce and 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 | ||||||||
| December 31, | ||||||||
| (in thousands, except share and per share data) | 2024 | 2023 | ||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 37,573 | $ | 21,709 | ||||
| Marketable securities | 39,183 | 92,585 | ||||||
| Prepaid expenses and other current assets | 838 | 923 | ||||||
| Prepaid research and development | 1,221 | 2,742 | ||||||
| Total current assets | 78,815 | 117,959 | ||||||
| Property and equipment, net | 9,331 | 15,295 | ||||||
| Right of use assets - operating leases | 13,803 | 16,858 | ||||||
| Other assets | 463 | 433 | ||||||
| Total assets | $ | 102,412 | $ | 150,545 | ||||
| Liabilities and stockholders’ equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 742 | $ | 1,298 | ||||
| Accrued expenses and other current liabilities | 6,707 | 11,670 | ||||||
| Non-refundable sublicense and transition services payments received | 8,226 | — | ||||||
| Operating lease liabilities | 3,688 | 3,373 | ||||||
| Total current liabilities | 19,363 | 16,341 | ||||||
| Operating lease liabilities - noncurrent | 21,788 | 22,921 | ||||||
| Total liabilities | 41,151 | 39,262 | ||||||
| Stockholders’ equity: | ||||||||
| Preferred stock, | — | — | ||||||
| Common stock, | 6 | 5 | ||||||
| Additional paid‑in capital | 720,482 | 705,789 | ||||||
| Accumulated other comprehensive income (loss) | 8 | (43 | ) | |||||
| Accumulated deficit | (659,235 | ) | (594,468 | ) | ||||
| Total stockholders’ equity | 61,261 | 111,283 | ||||||
| Total liabilities and stockholders’ equity | $ | 102,412 | $ | 150,545 | ||||
| Passage Bio, Inc. Statements of Operations and Comprehensive Loss | ||||||||
| Year Ended December 31, | ||||||||
| (in thousands, except share and per share data) | 2024 | 2023 | ||||||
| Operating expenses: | ||||||||
| Research and development | $ | 40,179 | $ | 61,419 | ||||
| General and administrative | 24,988 | 41,580 | ||||||
| Impairment of long-lived assets | 5,233 | 5,390 | ||||||
| Loss from operations | (70,400 | ) | (108,389 | ) | ||||
| Other income (expense), net | 5,633 | 6,327 | ||||||
| Net loss | $ | (64,767 | ) | $ | (102,062 | ) | ||
| Per share information: | ||||||||
| Net loss per share of common stock, basic and diluted | $ | (1.07 | ) | $ | (1.86 | ) | ||
| Weighted average common shares outstanding, basic and diluted | 60,405,036 | 54,743,490 | ||||||
| Comprehensive loss: | ||||||||
| Net loss | $ | (64,767 | ) | $ | (102,062 | ) | ||
| Unrealized gain (loss) on marketable securities | 51 | 923 | ||||||
| Comprehensive loss | $ | (64,716 | ) | $ | (101,139 | ) | ||
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
What were the key findings from Passage Bio's PBFT02 trial for FTD-GRN patients?
When will Passage Bio (PASG) report the next PBFT02 trial data?
How much did Passage Bio's operating expenses decrease in 2024?
What is Passage Bio's (PASG) current cash runway?
How much did Passage Bio reduce its net loss in 2024?
