Core Viewpoint - Passage Bio, Inc. (PASG) has seen a significant stock price increase of 31.5% over the past four weeks, closing at $7.59, with analysts suggesting a potential upside of 1415.2% based on a mean price target of $115 [1][2]. Price Targets and Analyst Estimates - The mean estimate of $115 is derived from four short-term price targets, with the lowest estimate at $40.00 indicating a 427% increase, while the highest target suggests a surge of 3325.6% to $260.00 [2]. - The standard deviation of $103.76 indicates variability among analysts' estimates, with a smaller standard deviation suggesting greater agreement [2][9]. Earnings Estimates and Analyst Agreement - Analysts show strong agreement in revising earnings estimates higher for PASG, which is a positive indicator for potential stock upside [4][11]. - The Zacks Consensus Estimate for the current year has increased by 8.7% over the past month, with no negative revisions, further supporting the optimistic outlook [12]. Zacks Rank and Investment Potential - PASG holds a Zacks Rank 2 (Buy), placing it in the top 20% of over 4,000 ranked stocks based on earnings estimate factors, indicating strong potential for near-term upside [13]. - While consensus price targets may not be entirely reliable, the direction they imply appears to be a useful guide for potential price movement [14].

PART I. FINANCIAL INFORMATION This section presents the unaudited interim financial statements and management's analysis for the period ended June 30, 2025 [Item 1. Interim Financial Statements (Unaudited)](index=5&type=section&id=Item%201.%20Interim%20Financial%20Statements%20(Unaudited)) The unaudited interim financial statements for June 30, 2025, report a net loss of $24.8 million and total assets of $79.2 million [Balance Sheets](index=5&type=section&id=Balance%20Sheets) Total assets decreased to $79.2 million by June 30, 2025, with cash increasing to $57.6 million and equity declining Balance Sheet Highlights (in thousands) | Account | June 30, 2025 | December 31, 2024 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $57,626 | $37,573 | | Marketable securities | $0 | $39,183 | | Total current assets | $60,315 | $78,815 | | Total assets | $79,198 | $102,412 | | **Liabilities & Equity** | | | | Total liabilities | $40,940 | $41,151 | | Total stockholders' equity | $38,258 | $61,261 | | Accumulated deficit | $(684,025) | $(659,235) | [Statements of Operations and Comprehensive Loss](index=6&type=section&id=Statements%20of%20Operations%20and%20Comprehensive%20Loss) Net loss improved to $9.4 million for Q2 2025 and $24.8 million for six months, due to lower operating expenses Operating Results (in thousands, except per share data) | Metric | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :--- | :--- | :--- | :--- | :--- | | Research and development | $5,814 | $10,430 | $13,551 | $21,965 | | General and administrative | $4,520 | $6,510 | $10,605 | $13,025 | | Impairment of long-lived assets | $0 | $438 | $2,637 | $438 | | Loss from operations | $(10,334) | $(17,378) | $(26,793) | $(35,428) | | Net loss | $(9,385) | $(15,991) | $(24,790) | $(32,702) | | Net loss per share | $(2.96) | $(5.09) | $(7.83) | $(10.87) | [Statements of Stockholders' Equity](index=7&type=section&id=Statements%20of%20Stockholders'%20Equity) Stockholders' equity decreased to $38.3 million by June 30, 2025, primarily due to a $24.8 million net loss - Total stockholders' equity decreased by **$22.9 million** in the first six months of 2025, mainly due to the net loss of **$24.8 million**[19](index=19&type=chunk) [Statements of Cash Flows](index=9&type=section&id=Statements%20of%20Cash%20Flows) Net cash used in operations was $20.2 million, with investing activities providing $40.2 million, increasing cash to $57.6 million Cash Flow Summary (in thousands) | Activity | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :--- | :--- | :--- | | Net cash used in operating activities | $(20,177) | $(32,065) | | Net cash provided by investing activities | $40,216 | $26,299 | | Net cash provided by financing activities | $14 | $8,827 | | **Net increase in cash** | **$20,053** | **$3,061** | | **Cash at end of period** | **$57,626** | **$24,770** | [Notes to Unaudited Interim Financial Statements](index=10&type=section&id=Notes%20to%20Unaudited%20Interim%20Financial%20Statements) Notes detail the company's focus on neurodegenerative therapies, recurring losses, $684.0 million accumulated deficit, and a 12-month cash runway - The company is a clinical-stage genetic medicines company focused on neurodegenerative diseases, with its lead candidate being **PBFT02** for frontotemporal dementia (FTD-GRN)[25](index=25&type=chunk) - As of June 30, 2025, the company had an accumulated deficit of **$684.0 million** but expects its current cash and cash equivalents to be sufficient to fund operations for at least the next 12 months[26](index=26&type=chunk)[30](index=30&type=chunk)[31](index=31&type=chunk) - A **1-for-20 reverse stock split** was effected on July 14, 2025, to regain Nasdaq compliance, with all share and per-share data retroactively adjusted[34](index=34&type=chunk)[118](index=118&type=chunk) - In January 2025, the company reduced its workforce by **55%** and ceased lab operations, incurring **$1.7 million** in severance and a **$2.6 million** impairment charge[47](index=47&type=chunk)[85](index=85&type=chunk)[86](index=86&type=chunk) - The company out-licensed three programs to Gemma Biotherapeutics, receiving **$9.7 million** in payments recorded as a liability pending revenue recognition[81](index=81&type=chunk)[84](index=84&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=43&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses PBFT02, reduced operating losses, a $57.6 million cash runway into Q1 2027, and strategic business developments [Overview and Pipeline](index=43&type=section&id=Overview%20and%20Pipeline) The company focuses on neurodegenerative genetic medicines, with lead candidate PBFT02 for FTD-GRN, and out-licensed three pediatric programs - The lead clinical product candidate is **PBFT02**, a gene therapy for FTD-GRN, showing robust increases in CSF progranulin (PGRN) levels in the upliFT-D trial[140](index=140&type=chunk)[149](index=149&type=chunk) - Development of **PBFT02** is expanding to FTD-C9orf72 and ALS, with positive regulatory feedback on the clinical pathway[141](index=141&type=chunk)[153](index=153&type=chunk)[159](index=159&type=chunk) - Three pediatric programs (for GM1, Krabbe disease, and MLD) have been out-licensed to Gemma Biotherapeutics[142](index=142&type=chunk) [Results of Operations](index=57&type=section&id=Results%20of%20Operations) Operating results improved, with net loss decreasing to $9.4 million for Q2 2025 and $24.8 million for H1 2025, due to reduced R&D and G&A Comparison of Operating Results (in thousands) | Period | Metric | 2025 | 2024 | Change | | :--- | :--- | :--- | :--- | :--- | | **Three Months Ended June 30** | R&D Expenses | $5,814 | $10,430 | $(4,616) | | | G&A Expenses | $4,520 | $6,510 | $(1,990) | | | **Net Loss** | **$(9,385)** | **$(15,991)** | **$6,606** | | **Six Months Ended June 30** | R&D Expenses | $13,551 | $21,965 | $(8,414) | | | G&A Expenses | $10,605 | $13,025 | $(2,420) | | | **Net Loss** | **$(24,790)** | **$(32,702)** | **$7,912** | - The decrease in R&D expenses was primarily due to lower preclinical research costs, reduced wages and benefits from lower headcount, and decreased facility and manufacturing expenses following the cessation of lab operations in Hopewell, New Jersey[193](index=193&type=chunk)[197](index=197&type=chunk)[201](index=201&type=chunk) - An impairment charge of **$2.6 million** was recorded in the first six months of 2025 related to laboratory equipment that was subsequently sold[204](index=204&type=chunk) [Liquidity and Capital Resources](index=63&type=section&id=Liquidity%20and%20Capital%20Resources) The company holds $57.6 million in cash, with a runway into Q1 2027, and reduced net cash used in operations to $20.2 million - The company's cash and cash equivalents of **$57.6 million** as of June 30, 2025, are expected to provide a cash runway into the **first quarter of 2027**[168](index=168&type=chunk)[206](index=206&type=chunk) - Net cash used in operating activities decreased to **$20.2 million** for the first half of 2025 from **$32.1 million** in the first half of 2024, reflecting lower operating expenses[213](index=213&type=chunk)[214](index=214&type=chunk)[215](index=215&type=chunk) - The company has an ATM facility with **$15.8 million** of capacity remaining, though its use is limited by 'baby shelf' rules due to its public float[211](index=211&type=chunk)[257](index=257&type=chunk) [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=70&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company has indicated that quantitative and qualitative disclosures about market risk are not applicable - The company has indicated that quantitative and qualitative disclosures about market risk are not applicable[234](index=234&type=chunk) [Item 4. Controls and Procedures](index=70&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded disclosure controls were effective as of June 30, 2025, with no material changes to internal controls - Management concluded that as of June 30, 2025, the company's disclosure controls and procedures were effective[235](index=235&type=chunk) - No material changes were made to the internal control over financial reporting during the quarter ended June 30, 2025[236](index=236&type=chunk) PART II. OTHER INFORMATION This section covers legal proceedings, risk factors, equity sales, defaults, and other required disclosures [Item 1. Legal Proceedings](index=71&type=section&id=Item%201.%20Legal%20Proceedings) The company is involved in ongoing litigation with a former employee regarding a disputed settlement, with a favorable judgment under appeal - The company is involved in ongoing litigation with a former employee regarding a disputed settlement agreement from 2020[239](index=239&type=chunk) - A trial judge delivered a judgment in the company's favor in December 2024, finding no binding agreement was reached, with the plaintiff's appeal pending[115](index=115&type=chunk)[239](index=239&type=chunk) [Item 1A. Risk Factors](index=71&type=section&id=Item%201A.%20Risk%20Factors) The company faces significant financial, development, third-party reliance, commercialization, and regulatory risks, including losses and dependence on PBFT02 - **Financial Risks:** The company has a history of operating losses (**$684.0 million** accumulated deficit) and will need to raise additional capital, which may not be available on acceptable terms[242](index=242&type=chunk)[245](index=245&type=chunk)[253](index=253&type=chunk) - **Development Risks:** The business is dependent on its sole clinical candidate, **PBFT02**, with gene therapy development being lengthy, expensive, and uncertain[244](index=244&type=chunk)[261](index=261&type=chunk)[270](index=270&type=chunk) - **Third-Party Reliance:** The company relies on Gemma for preclinical research and Catalent for manufacturing, creating risks related to performance and disruptions[317](index=317&type=chunk)[322](index=322&type=chunk)[348](index=348&type=chunk) - **Commercialization & Regulatory Risks:** Significant competition, market acceptance, pricing, reimbursement, and a complex, evolving regulatory landscape pose substantial challenges[369](index=369&type=chunk)[374](index=374&type=chunk)[433](index=433&type=chunk) [Item 2. Unregistered Sales of Equity Securities, Use of Proceeds, and Issuer Purchases of Equity Securities](index=175&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities,%20Use%20of%20Proceeds,%20and%20Issuer%20Purchases%20of%20Equity%20Securities) The company reported no unregistered sales of equity securities during the reporting period - The company reported no unregistered sales of equity securities for the period[534](index=534&type=chunk) [Item 3. Defaults Upon Senior Securities](index=175&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities) The company reported no defaults upon senior securities during the period - There were no defaults upon senior securities[536](index=536&type=chunk) [Item 4. Mine Safety Disclosures](index=175&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) Mine safety disclosures are not applicable to the company - Mine safety disclosures are not applicable[537](index=537&type=chunk) [Item 5. Other Information](index=175&type=section&id=Item%205.%20Other%20Information) No directors or officers adopted, modified, or terminated a Rule 10b5-1 trading plan during the quarter - No directors or officers adopted, modified, or terminated a Rule 10b5-1 trading plan during the quarter[538](index=538&type=chunk) [Item 6. Exhibits](index=176&type=section&id=Item%206.%20Exhibits) This section lists exhibits filed with the Form 10-Q, including license agreement amendments and SOX certifications - Filed exhibits include amendments to license agreements with Gemma Biotherapeutics, Inc. for the GM1, Krabbe, and MLD programs[541](index=541&type=chunk) - Certifications by the CEO and CFO as required by Sections 302 and 906 of the Sarbanes-Oxley Act are included as exhibits[541](index=541&type=chunk)

[Passage Bio Q2 2025 Financial Results and Business Highlights](index=1&type=section&id=Passage%20Bio%20Q2%202025%20Financial%20Results%20and%20Business%20Highlights) [Business Highlights and Outlook](index=1&type=section&id=Business%20Highlights%20and%20Outlook) Passage Bio advanced its upliFT-D trial for PBFT02, completing dosing for the second cohort and reporting positive interim data, with a cash runway into Q1 2027 - **Completed dosing** of the second FTD-GRN patient cohort (Cohort 2) in the upliFT-D study in July[1](index=1&type=chunk)[6](index=6&type=chunk) - Reported updated interim data showing **PBFT02 robustly increases CSF PGRN levels** and **reduces plasma NfL**, a disease progression biomarker[1](index=1&type=chunk)[6](index=6&type=chunk) - Submitted an amended clinical trial protocol to global authorities, introducing prophylactic anticoagulation and revising inclusion criteria for earlier-stage patients[1](index=1&type=chunk)[3](index=3&type=chunk)[6](index=6&type=chunk) - Anticipates key milestones including seeking regulatory feedback on manufacturing in 2H 2025, reporting updated interim safety and biomarker data from Dose 2 in 1H 2026, and seeking regulatory feedback on registrational trial design in 1H 2026[5](index=5&type=chunk)[6](index=6&type=chunk) [Clinical Program Overview](index=3&type=section&id=Clinical%20Program%20Overview) The company's lead program, PBFT02, is an AAV1 gene therapy for FTD, evaluated in the upliFT-D trial, aiming to elevate PGRN levels - **upliFT-D (NCT04747431)** is a Phase 1/2 global, multi-center, open-label clinical trial of PBFT02 for FTD-GRN or FTD-C9orf72 patients aged 35 to 75[8](index=8&type=chunk) - The upliFT-D trial's primary endpoint is **safety and tolerability of PBFT02**, with secondary endpoints including disease biomarkers and clinical outcome measures[8](index=8&type=chunk) - **PBFT02** is an AAV1 gene replacement therapy designed to deliver a functional GRN gene, elevating PGRN levels in the CNS to alter neurodegenerative disease progression[10](index=10&type=chunk) - Collaboration with InformedDNA provides **no-cost genetic counseling and testing** for FTD patients, supporting clinical trial recruitment[9](index=9&type=chunk) [Second Quarter 2025 Financial Results](index=3&type=section&id=Second%20Quarter%202025%20Financial%20Results) Passage Bio reported a reduced net loss of $9.4 million in Q2 2025, driven by lower operating expenses, with a cash runway into Q1 2027 Q2 2025 Financial Highlights (vs. Q2 2024) | Financial Metric | Q2 2025 (Million USD) | Q2 2024 (Million USD) | Change (%) | | :--- | :--- | :--- | :--- | | Cash, Cash Equivalents & Marketable Securities | $57.6 | $91.8 | -37.2 | | R&D Expenses | $5.8 | $10.4 | -44.1 | | G&A Expenses | $4.5 | $6.5 | -30.8 | | Net Loss | ($9.4) | ($16.0) | +41.3 | | Net Loss Per Share | ($2.96) | ($5.09) | +41.8 | - Current **cash, cash equivalents, and marketable securities of $57.6 million** are expected to fund operations into the first quarter of 2027[1](index=1&type=chunk)[12](index=12&type=chunk) - **Total assets decreased to $79.2 million** as of June 30, 2025, from $102.4 million as of December 31, 2024[17](index=17&type=chunk) - All share and per-share data reflect a **1-for-20 reverse stock split** effected on July 14, 2025[12](index=12&type=chunk)[17](index=17&type=chunk)[18](index=18&type=chunk) [Corporate Information](index=5&type=section&id=Corporate%20Information) Passage Bio is a clinical-stage genetic medicines company focused on developing PBFT02 for neurodegenerative diseases, with forward-looking statements subject to inherent risks - Passage Bio's mission is to develop **one-time therapies for neurodegenerative diseases**, targeting underlying pathology to improve patient lives[13](index=13&type=chunk) - **PBFT02**, the lead product candidate, is designed to treat neurodegenerative conditions like frontotemporal dementia by elevating progranulin levels[13](index=13&type=chunk) - The press release includes **forward-looking statements** concerning clinical trial progress, regulatory feedback, and cash runway, subject to risks detailed in SEC filings[15](index=15&type=chunk)

Core Insights - Passage Bio, Inc. reported updated interim data from the upliFT-D study, indicating that PBFT02 shows robust and durable elevation in cerebrospinal fluid (CSF) progranulin (PGRN) levels and improvement in plasma neurofilament light chain (NfL), a biomarker for disease progression [1][5] - The company has completed dosing of Cohort 2 in the upliFT-D study and submitted an amended protocol to global trial sites and health authorities [2][5] - As of June 30, 2025, the company has a cash position of $57.6 million, which is expected to fund operations into the first quarter of 2027 [4] Recent Highlights - The dosing of Cohort 2 in the upliFT-D study was completed, with a total of four FTD-GRN patients treated [5] - Interim data showed that Dose 1 PBFT02 treatment resulted in a significant increase in CSF PGRN levels, with the first patient treated with Dose 2 showing substantial increases at one month post-treatment [5] - The company plans to initiate enrollment of Cohort 3 (FTD-GRN) and Cohort 4 (FTD-C9orf72) once the amended protocol is approved [2][5] Financial Results - Research and Development (R&D) expenses for Q2 2025 were $5.8 million, down from $10.4 million in Q2 2024 [13] - General and Administrative (G&A) expenses for Q2 2025 were $4.5 million, compared to $6.5 million in Q2 2024 [13] - The net loss for Q2 2025 was $9.4 million, or $2.96 per share, a decrease from a net loss of $16.0 million, or $5.09 per share, in Q2 2024 [13][19] Anticipated Upcoming Milestones - The company seeks regulatory feedback on the suspension-based manufacturing process comparability in the second half of 2025 [5] - Updated interim safety and biomarker data from Dose 2 is expected to be reported in the first half of 2026 [5] - Regulatory feedback on the registrational trial design in FTD-GRN is anticipated in the first half of 2026 [5]

Core Viewpoint - Passage Bio, Inc. has announced a reverse stock split at a ratio of 1-for-20 to comply with Nasdaq's minimum bid price requirement of $1.00 per share, effective July 14, 2025 [1][2]. Group 1: Reverse Stock Split Details - The reverse stock split will reduce the number of outstanding shares from approximately 62,405,898 to about 3,120,295 [2]. - Proportionate adjustments will be made to the exercise prices and number of shares underlying the Company's equity awards and incentive plans [2]. - The reverse stock split will not change the number of authorized shares or the par value per share [2]. Group 2: Stockholder Information - Registered stockholders holding shares in book-entry form will not need to take action to receive post-split shares [3]. - Stockholders with shares in brokerage accounts will have their positions automatically adjusted, with no action required on their part [3]. - No fractional shares will be issued; stockholders entitled to fractional shares will receive the nearest whole share [3]. Group 3: Company Overview - Passage Bio is a clinical-stage genetic medicines company focused on developing therapies for neurodegenerative diseases [4]. - The lead product candidate, PBFT02, aims to treat conditions like frontotemporal dementia by elevating progranulin levels [4].

PBFT02 Development and Preclinical Results - PBFT02 is an AAV gene therapy designed to deliver functional PGRN to the brain for the treatment of FTD-GRN [13] - In Grn-/- mice, AAV.hGRN vector ICV administration improved lysosomal function, reduced lipofuscin fluorescence in the thalamus, and reduced brain hexosaminidase activity [21, 23] - AAV1 was selected as the vector serotype due to superior hPGRN levels in CSF compared to AAV5 and AAVhu68 in NHPs [28, 29] - In Grn-/- mice, PBFT02 reduced lipofuscin deposition and neuroinflammation in the brain after intra-CSF delivery [34, 37] - ICM administration of PBFT02 enables PGRN delivery throughout the CNS [40] - In NHPs, PBFT02 dose-dependently increased PGRN in CSF up to day 14 [46, 48] - In NHPs, PBFT02 at Dose 1 resulted in approximately 10e4 GC/ug DNA throughout the brain [43] Clinical Trial (upliFT-D) and Safety - The upliFT-D trial is a global Phase 1/2 multi-center, open-label, dose-escalation study with PBFT02 [52, 55] - FTD-GRN Cohort 1 (n = 5) dosing is complete [56] - All four Cohort 1 participants who received a revised immunosuppression regimen had no SAEs or significant immune responses [57] - Cohort 1 interim data shows PBFT02 administration leads to robust and sustained increases in CSF PGRN [58]

Core Insights - Passage Bio, Inc. reported updated data from the Phase 1/2 upliFT-D clinical trial for PBFT02, showing robust and durable increases in cerebrospinal fluid (CSF) progranulin (PGRN) levels and improvements in plasma neurofilament (NfL) levels, indicating potential efficacy in treating frontotemporal dementia (FTD) with granulin mutations [1][2][10] Clinical Trial Data - Dose 1 of PBFT02 increased CSF PGRN levels from below 3 ng/mL at baseline to a mean of 12.4 ng/mL at one month, 19.4 ng/mL at six months, 25.9 ng/mL at 12 months, and 23.8 ng/mL at 18 months [5] - The first patient treated with Dose 2 (50% of Dose 1) saw CSF PGRN levels rise from 1.5 ng/mL at baseline to 7.6 ng/mL at one month, nearing the upper limit of a healthy adult reference range [5] - Plasma NfL levels increased by an average of 4% at 12 months post-treatment, compared to an expected increase of 28% to 29% per year in untreated symptomatic FTD-GRN patients [5] Safety Profile - Treatment emergent adverse events were mostly mild to moderate, with three serious adverse events reported among eight patients, including venous sinus thrombosis and pulmonary embolism [5][6] - No evidence of dorsal root ganglion toxicity was observed, and no complications occurred during the administration of PBFT02 [5] Study Protocol Amendments - The company plans to amend the upliFT-D protocol to include a prophylactic course of low-dose anticoagulation and modify inclusion criteria to allow earlier-stage patients [6][7] - The amended protocol is expected to be submitted to health authorities in early July 2025, with plans to begin enrollment in additional cohorts thereafter [7] Anticipated Milestones - The company aims to seek regulatory feedback on the pivotal trial design for FTD-GRN in the first half of 2026 and report updated interim safety and biomarker data from Dose 2 in the same timeframe [14]

Financial Performance - As of March 31, 2025, Passage Bio reported total assets of $86.0 million, down from $102.4 million as of December 31, 2024, representing a decrease of approximately 16%[13] - The company incurred a net loss of $15.4 million for the three months ended March 31, 2025, an improvement from a net loss of $16.7 million for the same period in 2024, reflecting a reduction of about 7.8%[14] - The accumulated deficit as of March 31, 2025, was $674.6 million, up from $659.2 million at the end of 2024, indicating an increase of approximately 2.2%[13] - The company reported a comprehensive loss of $15.4 million for Q1 2025, compared to a comprehensive loss of $16.7 million in Q1 2024, showing an improvement of about 7.8%[14] - The Company recorded a net loss per share for the three months ended March 31, 2025, with basic and diluted loss per share being the same due to the anti-dilutive effect of potential securities[62] - The Company recorded a loss from operations of $16.5 million for the three months ended March 31, 2025, compared to a loss of $18.1 million for the same period in 2024[190] - The company used $13.8 million in net cash for operating activities in Q1 2025, reflecting a net loss of $15.4 million[206] - Cash used in operating activities in Q1 2024 was $18.9 million, reflecting a net loss of $16.7 million[207] Cash and Liquidity - Cash and cash equivalents increased to $63.4 million as of March 31, 2025, compared to $37.6 million at the end of 2024, marking a growth of about 68.6%[13] - As of March 31, 2025, the company's total cash and cash equivalents amounted to $63.357 million, an increase from $37.573 million as of December 31, 2024, reflecting a significant growth of 68.6%[66] - The company had cash and cash equivalents of $63.4 million as of March 31, 2025, expected to fund operations into Q1 2027[166] - The company has $15.8 million remaining capacity to offer and sell shares under the ATM Facility as of March 31, 2025[203] - The company currently has no credit facility or committed sources of capital, indicating a need for additional funds for operational needs and clinical trials[201] Research and Development - Research and development expenses decreased to $7.7 million in Q1 2025 from $11.5 million in Q1 2024, indicating a reduction of approximately 33%[14] - Total research and development expenses for the three months ended March 31, 2025, were $7.737 million, down from $11.535 million in the same period of 2024, indicating a reduction of about 33.5%[133] - The Company’s research and development costs primarily consist of expenses related to contract research organizations and internal activities, with estimates made based on progress to completion[48] - The company expects research and development expenses to decrease in the near future due to a 55% reduction in workforce and cessation of lab operations in January 2025[183] - The lead clinical product candidate, PBFT02, aims to elevate progranulin levels to enhance lysosomal function and slow disease progression in neurodegenerative diseases, with a focus on frontotemporal dementia (FTD) caused by progranulin deficiency[137] - PBFT02 is currently in clinical development for FTD-GRN, with plans to expand its use to other adult neurodegenerative diseases, including FTD-C9orf72 and ALS, based on positive regulatory feedback[138] Impairment and Expenses - The Company recognized impairment expenses of $2.6 million for laboratory equipment and certain other assets for the three months ended March 31, 2025, compared to no impairment expenses for the same period in 2024[43] - The company recorded an impairment of $2.6 million related to the remeasurement of long-lived assets, which was included in the statement of operations for the three months ended March 31, 2025[70] - The Company recorded severance and termination-related costs of $1.7 million for the three months ended March 31, 2025, compared to no such costs in the same period of 2024[79] - The Company recorded share-based compensation expense of $858,000 for the three months ended March 31, 2025, compared to $1.595 million for the same period in 2024, reflecting a decrease of approximately 46.3%[120] - General and administrative expenses decreased by $0.4 million to $6.1 million for the three months ended March 31, 2025, from $6.5 million in the same period of 2024[192] Funding and Future Plans - The company plans to seek additional funding through public or private equity offerings, debt financings, and strategic alliances, with $15.8 million remaining available under its ATM Facility as of March 31, 2025[29][30] - Future funding requirements will depend on various factors, including the progress of clinical trials and collaborations with third parties[200] - The ongoing clinical trials and research programs are expected to incur significant expenses, with the need for additional capital to support operations and growth strategies[165] - The company anticipates an increase in general and administrative expenses if product candidates progress into later-stage clinical trials and require commercialization efforts[187] Lease and Sublease Agreements - The Company has classified all leases with terms greater than one year as operating leases as of March 31, 2025[45] - The Company has a total undiscounted lease liability of $25.116 million, with future minimum lease payments totaling $40.205 million[93] - The Company entered into a sublease agreement for approximately 29,000 square feet with a base rent of $0.9 million per year, effective from March 1, 2024, through August 2026[84] - The Company subleased approximately 16,000 square feet with a base rent of $0.3 million per year, starting March 26, 2024, and expiring September 30, 2025[86] - The company has a lease obligation of approximately $11.8 million for office space in Philadelphia, expiring in December 2031, with potential cash inflows from sublease agreements[211] Clinical Trials and Product Development - The estimated prevalence of FTD-GRN in the US and Europe is approximately 18,000, while FTD-C9orf72 is estimated at 21,000[145][155] - In the upliFT-D trial, Dose 1 of PBFT02 resulted in robust increases in cerebrospinal fluid (CSF) PGRN levels, from below 3.0 ng/mL at baseline to 22.3 to 34.0 ng/mL at 12 months post-treatment[146] - Plasma neurofilament light chain (NfL) levels decreased by an average of 13% at 12 months post-treatment with Dose 1 of PBFT02, contrasting with an expected 29% annual increase in untreated patients[148] - The company plans to report 12-month follow-up data from Dose 1 and interim safety and biomarker data from Dose 2 in the second half of 2025[153] Collaboration Agreements - The Company has entered into sublicense agreements with Gemma Biotherapeutics for multiple gene therapy programs, including treatments for GM1 gangliosidosis, Krabbe disease, and metachromatic leukodystrophy[139] - The Gemma Collaboration Agreement requires payments of up to $16.5 million per product candidate for Huntington's disease and $39.0 million for Temporal Lobe Epilepsy[102] - Under the Gemma Sublicenses, the company is entitled to receive initial payments of $10 million and up to an additional $114 million in development and commercial milestone payments[176] - The Amended Catalent Agreements establish a limited exclusive relationship for the manufacture of drug products for adeno-associated virus delivery therapeutic candidates until November 6, 2030[180]

Financial Performance - Passage Bio reported a net loss of $15.4 million, or $0.25 per share, for Q1 2025, compared to a net loss of $16.7 million, or $0.30 per share, for Q1 2024[9][13]. - Cash, cash equivalents, and marketable securities totaled $63.4 million as of March 31, 2025, down from $104.5 million a year earlier, with an expected cash runway into Q1 2027[9][12]. - Total assets decreased to $86.0 million as of March 31, 2025, from $102.4 million as of December 31, 2024[12][9]. Research and Development - Research and Development (R&D) expenses decreased to $7.7 million in Q1 2025 from $11.5 million in Q1 2024, reflecting a reduction of approximately 33%[9][13]. - The company treated its first FTD-GRN patient with Dose 2 PBFT02 and enrolled a second patient, with several additional patients being evaluated for trial eligibility[4][3]. - A high productivity, suspension-based manufacturing process for PBFT02 was presented, which is expected to yield over 1,000 doses at Dose 2 with over 90% purity and over 70% full capsids[4][3]. - 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[5][4]. - Enrollment for FTD-C9orf72 patients in the upliFT-D study has commenced, with plans to initiate dosing in the first half of 2025[5][4]. - The Independent Data Monitoring Committee will review safety data from Cohort 2 before proceeding to Cohort 3, which will include 3-5 patients[4][3]. - The company anticipates seeking regulatory feedback on the registrational trial design in the first half of 2026[5][4].

Core Viewpoint - Passage Bio, Inc. is making progress in its clinical trials for PBFT02, a treatment for neurodegenerative diseases, with a focus on frontotemporal dementia (FTD) and plans for future regulatory engagement [2][6]. Recent Highlights - The company treated its first FTD-GRN patient with Dose 2 of PBFT02 and enrolled a second patient, with additional patients being evaluated for trial eligibility [6]. - A high productivity, suspension-based manufacturing process for PBFT02 was presented at the ASGCT 28th Annual Meeting, showing significant improvements in productivity and purity [6]. - The company has a cash runway expected to last into the first quarter of 2027, with cash and cash equivalents totaling $63.4 million as of March 31, 2025 [12]. Anticipated Upcoming Milestones - The company plans to initiate dosing of FTD-C9orf72 patients in the first half of 2025 [4]. - Interim safety and biomarker data from Dose 2 and 12-month data from Dose 1 patients are expected to be reported in the second half of 2025 [6]. Financial Results - For the first quarter of 2025, research and development expenses were $7.7 million, down from $11.5 million in the same quarter of 2024 [12]. - General and administrative expenses decreased to $6.1 million from $6.5 million year-over-year [12]. - The net loss for the quarter was $15.4 million, or $0.25 per share, compared to a net loss of $16.7 million, or $0.30 per share, in the prior year [12][15].