Passage Bio (PASG) FY Conference September 05, 2025 07:00 AM ET Speaker1The Research Associate at HC Wainwright. Please introduce Dr. William (Will) Cho, who is the President and Chief Executive Officer of Passage Bio.Speaker0Great, thanks for the introduction, Joseph, and for having us here today. Will Cho, CEO of Passage Bio. We are a clinical stage genetic medicines company focused on neurodegenerative disease. I'm going to be talking today primarily about our lead clinical program in frontotemporal deme ...

Passage Bio, Inc. (PASG) closed the last trading session at $7.13, gaining 24.2% over the past four weeks, but there could be plenty of upside left in the stock if short-term price targets set by Wall Street analysts are any guide. The mean price target of $59.25 indicates a 731% upside potential.The average comprises four short-term price targets ranging from a low of $10.00 to a high of $120.00, with a standard deviation of $46.71. While the lowest estimate indicates an increase of 40.3% from the current ...

Company Overview - Passage Bio, Inc. is a clinical stage genetic medicines company focused on improving the lives of patients with neurodegenerative diseases [3] - The company's lead product candidate, PBFT02, aims to treat neurodegenerative conditions such as frontotemporal dementia by elevating progranulin levels to restore lysosomal function and slow disease progression [3] Upcoming Events - Management will participate in the H.C. Wainwright 27th Annual Global Investment Conference from September 8-10, 2025, in New York City [1] - Will Chou, M.D., president and CEO, will present an overview of the company and engage in one-on-one meetings with investors [2] - A video webcast of the presentation will be available to registered attendees starting September 5, 2025, at 7:00 a.m. ET, and a replay will be accessible for 30 days post-event [2]

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)

Completed dosing of FTD-GRN Cohort 2 in upliFT-D study Reported updated interim data showing that PBFT02 continued to demonstrate robust, durable elevation in CSF PGRN levels and improvement in plasma NfL, a disease progression biomarker, compared to natural history Submitted amended upliFT-D study protocol to global trial sites and health authorities Cash runway into 1Q 2027 PHILADELPHIA, Aug. 12, 2025 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (Nasdaq: PASG), a clinical stage genetic medicines company focused ...

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]