Summary of Passage Bio FY Conference Call Company Overview - **Company**: Passage Bio (NasdaqGS:PASG) - **Focus**: Development of gene therapies for neurodegenerative diseases, specifically targeting Frontotemporal Dementia (FTD) with granulin mutation and Huntington's disease Key Points Clinical Programs - **FTD-GRN Program**: - Targets Frontotemporal Dementia (FTD) with granulin mutation affecting approximately 18,000 patients in the U.S. and Europe [2] - FTD is characterized by early-onset behavioral dementia with no current treatment options, leading to an average lifespan of eight years post-diagnosis [2] - The program aims to address low levels of progranulin, a protein critical for lysosomal function, which is deficient in these patients due to granulin gene mutation [2][3] - The AAV1 gene therapy product aims to replace the missing progranulin to improve neurodegeneration outcomes [3] - **Study Progress**: - Nine patients have been treated in the ongoing Phase 1/2 study, with modifications to inclusion criteria to focus on milder cases of FTD [4] - The study is assessing safety, tolerability, and biomarkers such as CSF progranulin levels, plasma neurofilaments, and volumetric MRI [5][6] - Initial data shows that higher doses of the therapy significantly elevate CSF progranulin levels, with expectations for lower doses to also show positive trends [8][9] - **Safety Profile**: - Three serious adverse events (SAEs) were reported at the higher dose, leading to a shift to a lower dose and the introduction of prophylactic anticoagulation [10][12] - The safety profile remains a critical focus as the company prepares for regulatory discussions [39] Competitive Landscape - **Comparison with Other Therapies**: - Passage Bio's approach is differentiated from other therapies, such as the anti-sortilin antibody therapy, which did not meet endpoints in clinical trials [23] - The company emphasizes the importance of patient selection and the mechanistic differences in their therapy compared to competitors [24][28] Future Outlook - **Regulatory Strategy**: - The company is seeking early regulatory feedback on the registrational path for FTD-GRN, considering both single-arm and randomized controlled study designs [38] - Plans to declare a clinical candidate for Huntington's disease in the second half of the year, with ongoing discussions about the development path [16][52] - **Enrollment and Interest**: - Enrollment for the FTD-GRN cohort has been brisk, with increased interest following the discontinuation of other studies in the space [44][50] - The company is optimistic about the momentum in patient recruitment due to the unmet need in the orphan indication [50] Manufacturing and Partnerships - **Manufacturing Process**: - The company has established an efficient manufacturing process for its gene therapies, which is crucial for scaling production [59] - Current manufacturing capabilities are focused on the FTD program, with plans to expand as the Huntington's program develops [60] Additional Insights - The company is committed to addressing neurodegenerative diseases through innovative gene therapy approaches, with a focus on safety and efficacy as they navigate regulatory pathways and clinical development [59][60]

Clinical Development and Product Candidates - The lead clinical product candidate, PBFT02, aims to elevate progranulin levels to enhance lysosomal function and slow disease progression in neurodegenerative diseases, specifically targeting frontotemporal dementia (FTD) caused by progranulin deficiency (FTD-GRN) [23]. - PBFT02 has shown robust and durable increases in cerebrospinal fluid (CSF) progranulin levels, with concentrations rising from below 3.0 ng/mL at baseline to a mean of 25.9 ng/mL at 12 months in the upliFT-D trial [34]. - PBFT02 is also being evaluated for additional indications, including FTD-C9orf72 and amyotrophic lateral sclerosis (ALS), with an estimated prevalence of FTD-C9orf72 at approximately 21,000 [41]. - The company is actively developing a genetic medicine for Huntington's disease (HD) through a collaboration with Gemma Biotherapeutics, with an estimated prevalence of HD in the U.S. and Europe at approximately 70,000 [25]. - The company expects to declare a clinical candidate for the HD program in the second half of 2026, focusing on reducing somatic instability to slow neurodegeneration [48]. - The company has initiated preclinical studies to explore the potential benefits of increased progranulin levels in diseases with TDP-43 pathology, which is associated with multiple neurodegenerative conditions [39]. - The company has manufactured the PBFT02 clinical supply to support the ongoing Phase 1/2 clinical trial in FTD-GRN and FTD-C9orf72, as well as the initiation of a registrational trial in FTD-GRN [45]. - The company is advancing PBFT02 for the treatment of FTD-GRN, prioritizing the ongoing upliFT-D trial with the goal of moving to the registrational phase [52]. - Initial clinical data for PBFT02 shows robust and durable elevations in CSF PGRN levels and early evidence of reductions in plasma NfL, a disease progression biomarker [52]. - PBFT02 aims to provide higher levels of PGRN to the CNS, addressing the deficiency in GRN mutation carriers, with CSF levels ranging from 30% to 50% of those in normal individuals [69]. - The clinical trial upliFT-D for PBFT02 includes three cohorts with a total of 3 to 10 subjects per cohort, focusing on safety and tolerability over 60 months [95]. - Interim biomarker data showed that Dose 1 of PBFT02 increased CSF PGRN levels from below 3.0 ng/mL at baseline to a mean of 12.4 ng/mL at one month [96]. - PBFT02 was well-tolerated with no significant adverse effects on body weight or neurological signs observed in the 90-day toxicology study [86]. - The company is exploring the therapeutic potential of PBFT02 in additional neurodegenerative indications, including ALS and AD, leveraging shared research and development costs [52]. Regulatory and Compliance - The FDA granted Orphan Drug Designation and Fast Track Designation to PBFT02 for the treatment of FTD-GRN [106]. - The FDA review process for BLAs typically takes ten months for standard reviews and six months for priority reviews, with potential extensions for additional information [153]. - The company must comply with extensive FDA regulations, including preclinical tests and clinical trials, which can take many years to complete [146]. - A BLA submission incurs substantial costs, including application user fees and annual program fees that typically increase annually [153]. - The FDA may require a Risk Evaluation and Mitigation Strategy (REMS) as a condition of BLA approval, which can affect market potential and profitability [156]. - The FDA has specific guidance for gene therapy products, including long-term tracking of subjects for potential delayed adverse effects [158]. - The FDA grants Orphan Drug Designation to products for rare diseases affecting fewer than 200,000 individuals in the U.S., providing a seven-year exclusive marketing period for the first approved product [159]. - Orphan Drug Designation includes benefits such as tax credits for research and a waiver of the BLA user fee [161]. - Fast Track Designation is available for drugs addressing serious conditions with no effective treatment, expediting development and review processes [162]. - Priority Review may be granted for products that significantly improve safety and effectiveness compared to existing therapies, directing additional resources to their evaluation [163]. - Post-approval requirements include adverse event reporting and compliance with cGMPs, with the FDA conducting periodic inspections [171]. Financial Performance and Projections - The company incurred net losses of $45.5 million and $64.8 million for the years ended December 31, 2025 and 2024, respectively, with an accumulated deficit of $704.8 million as of December 31, 2025 [211]. - The company has never generated revenue from product sales and does not expect to achieve profitability for many years, if ever [215]. - As of December 31, 2025, the company's cash and cash equivalents were $46.3 million, expected to fund operations through the end of Q1 2027 [218]. - The company anticipates significant increases in expenses as it advances product candidates through clinical development and seeks regulatory approvals [212]. - Future capital requirements will depend on the progress of clinical trials and the need to maintain and expand the intellectual property portfolio [219]. - The company expects to incur substantial future capital needs to complete planned preclinical and clinical development for its product candidates [217]. - The company has funded operations primarily through proceeds from sales of convertible preferred stock and public offerings [210]. - The ability to raise additional funding is critical for the company to continue its research and development efforts [217]. Intellectual Property and Collaborations - The company has a patent portfolio that includes claims directed to rAAV for treating FTD, with expected expiration dates in 2040 and 2041 [134]. - The company has a patent portfolio that includes seven patent families with pending or issued claims for rAAV treatments for GM1, Krabbe, and MLD [135]. - The patent family for purifying rAAV includes applications pending in 12 jurisdictions, with expected expiration on October 6, 2043 [137]. - The patent family for rAAV treatment of FTD and other neurodegenerative diseases includes a PCT application, with expected expiration on March 3, 2045 [138]. - The company has options under existing agreements to add additional intellectual property to its license portfolio [136]. - The Gemma Collaboration Agreement includes payments of up to $16.5 million per product candidate for Huntington's disease and future CNS indications [125]. - The company is required to pay $0.9 million to Catalent due to the outlicense and transition of its GM1 gangliosidosis program to Gemma [110]. - The company has a collaboration agreement with Catalent for cGMP manufacturing capacity, which is crucial for current and future clinical trials [109]. Workforce and Corporate Culture - As of December 31, 2025, the company had 24 full-time employees, including 7 with advanced degrees and 13 engaged in research and development [191]. - The company faces intense competition for skilled individuals in genetic medicine, leading to high turnover rates and a shortage of experienced professionals [195]. - The company offers a comprehensive total rewards package, including competitive pay, healthcare benefits, and a 5% employer match for retirement plans [196]. - The company emphasizes a commitment to diversity and inclusion as essential to its success and aims to create a thriving workplace [194]. - The company’s mission focuses on improving the lives of patients with neurodegenerative diseases while fostering strong community relationships [192]. Legal Matters - The company is involved in litigation with a former employee regarding a breach of contract claim, with a jury ruling in favor of the company [203]. - The company is not currently a party to any other legal proceedings that would materially affect its business, aside from the ongoing litigation mentioned [204].

Core Insights - Passage Bio, Inc. has made significant progress in its clinical programs for genetic forms of frontotemporal dementia (FTD) and is on track to report updated interim safety and biomarker data in the first half of 2026 [1][2][5]. Recent Highlights - The company has enrolled the first three patients with FTD-GRN in Cohort 3 of the ongoing upliFT-D study, which will evaluate Dose 2 PBFT02 [5]. - The first patient with FTD-C9orf72 has been treated with Dose 2 PBFT02 in Cohort 4 of the upliFT-D study, which is expected to include up to five patients [5]. - A differentiated preclinical program for Huntington's disease is advancing, with a clinical candidate selection anticipated in the second half of 2026 [5]. Anticipated Upcoming Milestones - Updated interim safety and biomarker data from the upliFT-D study is expected to be reported in the first half of 2026 [5]. - The company plans to seek regulatory feedback on the registrational trial design for FTD-GRN in the first half of 2026 [5]. - A clinical candidate for Huntington's disease is expected to be declared in the second half of 2026 [5]. Financial Results - As of December 31, 2025, the company reported cash and cash equivalents of $46.3 million, down from $76.8 million in 2024, with an expected cash runway through the first quarter of 2027 [12]. - Research and development expenses for Q4 2025 were $5.4 million, and for the full year, they totaled $23.3 million, compared to $9.6 million and $40.2 million in the same periods of 2024, respectively [12]. - The net loss for Q4 2025 was $13.0 million, or $4.09 per share, while the net loss for the full year was $45.5 million, or $14.35 per share, compared to a net loss of $64.8 million in 2024 [12][15].

Summary of Passage Bio FY Conference Call Company Overview - **Company**: Passage Bio (NasdaqGS:PASG) - **Focus**: Development of gene therapies for neurodegenerative diseases, specifically frontotemporal dementia (FTD) and Huntington's disease Key Points Clinical Programs - **Lead Program**: PBFT02 for treating frontotemporal dementia (FTD) with the granulin mutation - **Preclinical Program**: Huntington's disease targeting CAG repeat expansion - **Cash Runway**: Expected to last until Q1 2027 with a cash balance of $46 million and a burn rate of $30 million to $35 million per year [2][25] Frontotemporal Dementia (FTD) - **Target Population**: Patients with granulin mutation; estimated 18,000 patients in the U.S. and Europe [3] - **Mechanism**: AAV1 gene therapy delivered via nonsurgical injection to cerebrospinal fluid (CSF) to increase progranulin levels, addressing lysosomal dysfunction and neurodegeneration [3][4] - **Clinical Trial Status**: Ongoing Phase I/II study with nine patients treated to date; two dose levels being evaluated [5][6] - **Safety and Efficacy**: Preliminary data shows robust increases in CSF progranulin levels, with Dose 1 achieving levels in the mid-20s, significantly above normal [12][14] - **Biomarkers**: Plasma neurofilaments show a 4% increase in treated patients compared to a 28%-29% annual increase in untreated patients, indicating slowed neurodegeneration [15][16] Safety Profile - **Adverse Events**: Generally well-tolerated; some serious adverse events (SAEs) reported, including venous sinus thrombus and liver function test (LFT) increases, managed with adjusted immunosuppression [16][17] - **Prophylactic Measures**: Future cohorts will receive anticoagulation therapy to mitigate thrombotic risks [17] Competitive Landscape - **Comparison with Competitors**: Other programs, such as Elektor's, faced challenges due to mechanisms that may not effectively deliver progranulin to lysosomes; Passage Bio's approach focuses on direct delivery to CSF [28][30] - **Differentiation**: Passage Bio's one-time therapy shows durability and higher target engagement compared to competitors, with a focus on treating patients earlier in the disease progression [18][19] Huntington's Disease Program - **Target**: MSH3 DNA repair protein to reduce CAG repeat expansion, with plans for an optimized intraparenchymal delivery approach [21][22] - **Clinical Candidate Timeline**: Expected to declare a clinical candidate in the second half of the year, with ongoing preclinical studies [23][46] Future Outlook - **Upcoming Data Releases**: Anticipated updates on safety and biomarker data for both Dose 1 and Dose 2 in the first half of the year [24][39] - **Regulatory Engagement**: Seeking feedback on registrational trial design for FTD-GRN to clarify approval requirements [25] Additional Insights - **Patient Selection**: Moving forward, the company will focus on patients with lower Clinical Dementia Rating (CDR) scores (0.5 and 1) to maximize treatment efficacy [11][31] - **Long-term Goals**: Aiming to establish a strong clinical profile for both FTD and Huntington's programs, with a focus on early intervention and effective delivery methods [44][45]

Financial Position - Cash runway expected to extend into Q1 2027 based on cash, cash equivalents, and marketable securities as of December 31, 2025[3] - As of December 31, 2025, the company reported a cash balance of approximately $46 million, providing a runway into the first quarter of 2027[31] Clinical Development - PBFT02 shows promising data from initial clinical study in FTD-GRN, with a one-time gene replacement therapy approach[4] - The ongoing upliFT-D trial is a global Phase 1/2 study currently enrolling patients, focusing on safety and biomarkers[13] - PBFT02's differentiated gene therapy approach is expected to address the urgent patient need in FTD-GRN with no approved disease-modifying therapies[4] - PBFT02 aims to provide a one-time therapy capable of achieving the highest progranulin levels, addressing underlying pathologies in frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS)[20] - The company plans to initiate discussions with the FDA regarding a registrational study design for FTD-GRN in the first half of 2026[30] - The company expects to declare a clinical candidate for Huntington's disease in the second half of 2026[27] Efficacy and Safety - PBFT02 administration in preclinical studies resulted in CSF hPGRN levels 5x higher than AAVhu68, indicating superior response[10] - PBFT02 demonstrated robust, durable increases in CSF PGRN levels, with a patient increasing from 1.5 ng/mL at baseline to 7.6 ng/mL at month 1[16] - Plasma NfL in PBFT02-treated patients showed a reduced annual rate of change of 3.7% compared to published natural history data of ~29%[17] - PBFT02 demonstrated a generally well-tolerated safety profile with no serious adverse events (SAEs) related to Dose 2, while two SAEs occurred at Dose 1[18] - The mean cerebrospinal fluid (CSF) PGRN level at 12 months was 26 ng/mL, with a durable elevation observed at 18 months in some patients[19] Manufacturing and Production - The company has completed a high-productivity manufacturing process, with a single production lot estimated to yield over 1,000 doses with over 70% full capsids[29] - The company has aligned with the FDA on an analytical approach to establish comparability of the suspension-based manufacturing process for PBFT02[29] Market Context - Estimated prevalence of neurodegenerative diseases includes ~70K for FTD-GRN, ~21K for FTD-C9orf72, and ~18K for Huntington's disease in the US and EU[5] - Key baseline demographics for FTD-GRN participants indicate a mean age of 64.4 years and a mean PGRN level of 36.6 ng/mL in CSF[15] - PBFT02 is positioned as a potentially transformative therapy for FTD-GRN patients, with no approved disease-modifying therapies currently available[32] - The company is pursuing a differentiated gene therapy approach in Huntington's disease, focusing on reducing MSH3 expression to decrease somatic instability in the HTT gene[27]

Core Insights - Passage Bio and PepGen are small-cap medical companies being compared based on various financial and operational metrics to determine which stock is more favorable [1][3][4]. Institutional Ownership - Passage Bio has 53.5% of its shares owned by institutional investors, while PepGen has 58.0% [1]. - Insider ownership stands at 5.0% for Passage Bio and 5.2% for PepGen, indicating a similar level of insider investment [1]. Analyst Ratings - Passage Bio has a consensus target price of $42.67, suggesting a potential upside of 363.26% [3][4]. - PepGen's consensus target price is $10.00, indicating a potential upside of 83.82% [3][4]. - Passage Bio has a stronger consensus rating with a score of 2.50 compared to PepGen's score of 2.33 [3]. Earnings and Valuation - Passage Bio reported a net income of -$64.77 million with an earnings per share (EPS) of -$14.40 [5]. - PepGen's net income is -$89.98 million with an EPS of -$2.82 [5]. - Passage Bio has a higher price-to-earnings ratio compared to PepGen, which is currently trading at a lower valuation [5]. Profitability - Passage Bio has a net margin of -102.09% and a return on equity of -52.97% [6]. - PepGen's net margin is -84.15% with a return on equity of -65.59% [6]. Risk & Volatility - Passage Bio has a beta of 1.86, indicating it is 86% more volatile than the S&P 500 [7]. - PepGen has a beta of 1.91, meaning it is 91% more volatile than the S&P 500 [7]. Summary - PepGen outperforms Passage Bio in 6 out of 11 factors compared [8].

Summary of Passage Bio FY Conference Call Company Overview - **Company**: Passage Bio (NasdaqGS:PASG) - **Industry**: Gene Therapy - **Focus**: Lead clinical program in frontotemporal dementia (FTD) with a granular mutation and a preclinical program in Huntington's disease [2][3] Key Points and Arguments Clinical Program Insights - **FTD Program**: The primary focus of the discussion was on the FTD program, particularly following a disappointing phase three study from a competitor [3][11] - **Progranulin Levels**: The company is investigating the significance of progranulin levels in the context of FTD GRN patients, noting that the average CSF progranulin level in a competitor's study was 4-5 ng/ml, while the normal range is 3-8 ng/ml [4][22] - **Mechanism of Action**: Passage Bio's approach involves using AAV (adeno-associated virus) to increase intracellular progranulin levels, contrasting with competitors that may inhibit natural cellular processes [8][25] Study Design and Patient Population - **Patient Selection**: The company plans to focus on earlier-stage patients (CDR 0.5 and 1) to enhance the likelihood of observing clinical responses, as opposed to including more severe patients [14][26] - **Epidemiology**: There are approximately 3,000 to 6,000 patients in the U.S. with FTD GRN, and genetic testing is crucial for early diagnosis [16][18] Regulatory Considerations - **FDA Guidance**: Recent FDA guidance indicates a potential openness to single-arm studies for rare diseases, which could benefit Passage Bio's registration strategy [21][29] - **Statistical Analysis Plan**: The company emphasizes the importance of prespecifying the statistical analysis plan and intends to engage with the FDA early in the study design process [28][29] Manufacturing and Financials - **Manufacturing**: The company collaborates with Catalent for manufacturing, utilizing a high productivity suspension process that can treat over 1,000 patients per batch [43][44] - **Cash Position**: Passage Bio has a cash balance sufficient to sustain operations into 2027, with an annual expenditure of approximately $30 million [45] Additional Important Insights - **Natural History Studies**: The company can leverage data from large natural history studies (All FTD and GenFi) to inform its clinical program [32] - **Neurofilament Biomarker**: The increase in plasma neurofilament levels observed in the study is consistent with age-related changes, suggesting the potential efficacy of the therapy [34][35] - **Future Data Release**: The company plans to refresh data in the first half of next year and will seek FDA guidance on the registration path, which is seen as a critical catalyst for investor confidence [39][40] This summary encapsulates the key aspects of Passage Bio's conference call, highlighting the company's strategic focus, clinical insights, regulatory considerations, and financial health.

Financial Performance - As of September 30, 2025, Passage Bio reported total assets of $74,160,000, a decrease of 27.6% from $102,412,000 on December 31, 2024[13] - For the three months ended September 30, 2025, net loss was $7,749,000, a reduction of 60% compared to a net loss of $19,340,000 for the same period in 2024[14] - The net loss per share for the three months ended September 30, 2025, was $2.44, compared to $6.15 for the same period in 2024[14] - The company reported a comprehensive loss of $7,749,000 for the three months ended September 30, 2025, compared to $19,241,000 for the same period in 2024[14] - For the nine months ended September 30, 2025, the net loss was $32.5 million, a decrease from a net loss of $52.0 million in the same period of 2024, representing a 37.4% improvement[23] - The company reported net losses of $7.7 million and $19.3 million for the three months ended September 30, 2025 and 2024, respectively[168] Cash and Liquidity - The company’s cash and cash equivalents increased to $52,773,000 from $37,573,000, representing a growth of 40.3%[13] - The company reported cash and cash equivalents of $52.8 million at the end of the period, up from $32.3 million at the end of September 2024, reflecting a 63.7% increase[23] - As of September 30, 2025, the company had cash and cash equivalents of $52.8 million, expected to fund operations into Q1 2027[171] - Cash flows used in operating activities amounted to $25.0 million for the nine months ended September 30, 2025, compared to $39.5 million for the same period in 2024, indicating a 36.5% reduction in cash outflows[23] - The company anticipates needing additional funds for operational needs and capital requirements for clinical trials and research and development expenditures[207] Research and Development - Research and development expenses decreased to $4,307,000 from $8,656,000, reflecting a decline of 50.3% year-over-year[14] - Total research and development expenses for the nine months ended September 30, 2025, were $17.9 million, down 41.6% from $30.6 million in the same period of 2024[141] - The company expects research and development expenses to remain consistent in the near future, with potential increases if product candidates progress to later-stage clinical trials[186] - The lead clinical product candidate, PBFT02, is focused on treating frontotemporal dementia caused by progranulin deficiency, aiming to restore lysosomal function[25] - The company has a gene therapy pipeline addressing multiple neurodegenerative diseases, including Alzheimer's disease, with a potential prevalence of 3.9 million in the US/EU[149] Equity and Stock - Total stockholders' equity decreased to $31,120,000 from $61,261,000, a decline of 49.1%[13] - The total number of shares authorized under the Incentive Plan as of September 30, 2025, was 947,598, with 423,039 shares available for future grants[125] - The number of shares reserved for issuance under the Incentive Plan increased by 155,155 shares in January 2025[125] - The company issued 6,000,000 shares under the ATM Facility, resulting in net proceeds of $8.7 million after deducting offering costs of $0.3 million[210] Impairment and Expenses - The company recognized impairment expenses of $2.6 million for long-lived assets during the nine months ended September 30, 2025, compared to $2.7 million for the same period in 2024[45] - The company recorded $2.6 million of impairment expense related to laboratory equipment during the nine months ended September 30, 2025[200] - General and administrative expenses decreased by $3.0 million to $4.3 million for the three months ended September 30, 2025, from $7.3 million in the same period in 2024[195] Workforce and Operational Changes - The company reduced its workforce by approximately 55% in January 2025 to extend its cash runway[85] - Severance and termination-related costs recorded for the nine months ended September 30, 2025, amounted to $1.7 million[86] - A decrease of $3.3 million in wages and benefits due to a lower headcount from restructuring in January 2025[203] Clinical Trials and Product Development - PBFT02, a gene replacement therapy for FTD-GRN, is currently in a Phase 1/2 clinical trial with an active IND application from the FDA[152] - The company has opened enrollment in the upliFT-D study for PBFT02 in FTD-C9orf72 patients, following positive regulatory feedback on the clinical pathway[146] - The company plans to report updated interim safety and biomarker data from Dose 2 in the first half of 2026[164] Lease and Sublease Agreements - The company has classified all leases with terms greater than one year as operating leases as of September 30, 2025[49] - The company is pursuing additional opportunities to sublease space to offset financial obligations under the Laboratory Lease Agreement[101] - Total undiscounted lease payments amount to $38,217,000, with total lease liabilities at $24,337,000 after deducting imputed interest of $13,880,000[102]

Financial Performance - Cash, cash equivalents, and marketable securities were $52.8 million as of September 30, 2025, down from $84.8 million as of September 30, 2024[12] - Net loss for Q3 2025 was $7.7 million, or $2.44 per share, compared to a net loss of $19.3 million, or $6.15 per share, in Q3 2024[12] - Total current assets decreased to $55.7 million as of September 30, 2025, from $78.8 million as of December 31, 2024[15] - Total liabilities increased to $43.0 million as of September 30, 2025, compared to $41.2 million as of December 31, 2024[15] Expenses - Research and Development (R&D) expenses for Q3 2025 were $4.3 million, a decrease of 50.4% compared to $8.7 million in Q3 2024[12] - General and Administrative (G&A) expenses for Q3 2025 were $4.3 million, down 40.4% from $7.3 million in Q3 2024[12] Clinical Trials and Regulatory - Actively enrolling Cohort 3 (FTD-GRN) with 5 to 10 patients and Cohort 4 (FTD-C9orf72) with 3 to 5 patients in the ongoing upliFT-D study[6] - The company is on track to obtain regulatory feedback on the FTD-GRN registrational trial design in the first half of 2026[6] - The company plans to report updated interim safety and biomarker data from Dose 2 in the first half of 2026[6] Manufacturing - A single batch of the new suspension-based PBFT02 manufacturing process is estimated to yield over 1,000 doses with over 90% purity and over 70% full capsids[6]

Core Insights - Passage Bio, Inc. is actively enrolling patients in Cohort 3 (FTD-GRN) and Cohort 4 (FTD-C9orf72) for the ongoing upliFT-D clinical trial of PBFT02, recognizing the urgent need for disease-modifying therapies for frontotemporal dementia (FTD) [1][2] - The company has aligned with the FDA on an analytical approach to establish comparability of a high-productivity, suspension-based manufacturing process for PBFT02, which is expected to yield over 1,000 doses per batch with over 90% purity [5][2] - Passage Bio is on track to obtain regulatory feedback on the registrational trial design for FTD-GRN in the first half of 2026, with plans to report updated interim safety and biomarker data from Dose 2 during the same period [5][4] Recent Highlights - The upliFT-D trial is a Phase 1/2 global, multi-center, open-label clinical trial aimed at evaluating the safety and tolerability of PBFT02, with secondary endpoints including disease biomarkers and clinical outcome measures [6] - The trial protocol has been amended to allow for the enrollment of patients who are prodromal or have mild cognitive impairment, while excluding those with more severe progression [5] Financial Overview - As of September 30, 2025, the company reported cash, cash equivalents, and marketable securities totaling $52.8 million, down from $84.8 million a year prior, with an expected cash runway into the first quarter of 2027 [11] - Research and Development (R&D) expenses for the third quarter of 2025 were $4.3 million, a decrease from $8.7 million in the same quarter of 2024 [11] - General and Administrative (G&A) expenses also decreased to $4.3 million from $7.3 million year-over-year [11] - The net loss for the third quarter of 2025 was $7.7 million, or $2.44 per share, compared to a net loss of $19.3 million, or $6.15 per share, for the same period in 2024 [11][16]