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]

PBFT02 continued to demonstrate robust, durable elevation in CSF PGRN levels and improvement in plasma NfL, a disease progression biomarker, compared to natural history Dose 2, 50% lower than Dose 1, substantially increased CSF PGRN levels at 30-days, reaching the upper limit of a healthy adult reference range Plan to amend upliFT-D protocol to include a prophylactic course of low dose anticoagulation and modify inclusion criteria to study patients earlier in disease progression Remain on track to seek regu ...

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. (PASG) has received an upgrade to a Zacks Rank 2 (Buy), indicating a positive outlook based on rising earnings estimates, which significantly influence stock prices [1][3]. Earnings Estimates and Stock Price Impact - The Zacks rating system tracks the Zacks Consensus Estimate, which reflects EPS estimates from sell-side analysts, and is crucial for understanding a company's earnings outlook [1][4]. - Changes in earnings estimates are strongly correlated with near-term stock price movements, as institutional investors adjust their valuations based on these estimates [4][6]. Recent Performance and Projections - For the fiscal year ending December 2025, Passage Bio is expected to earn -$0.69 per share, representing a 35.5% change from the previous year's reported number [8]. - Over the past three months, the Zacks Consensus Estimate for Passage Bio has increased by 6.1%, indicating a positive trend in earnings expectations [8]. Zacks Rank System - The Zacks Rank system classifies stocks into five groups based on earnings estimates, with Zacks Rank 1 (Strong Buy) stocks historically generating an average annual return of +25% since 1988 [7]. - The upgrade of Passage Bio to Zacks Rank 2 places it in the top 20% of Zacks-covered stocks, suggesting potential for market-beating returns in the near term [10].

Financial Performance - The net loss for the year ended December 31, 2024, was $64.767 million, compared to a net loss of $102.062 million in 2023, indicating an improvement in financial performance [650]. - The company reported a comprehensive loss of $64.716 million for 2024, compared to $101.139 million in 2023, reflecting a reduction in overall losses [650]. - The company reported a net loss of $64,767,000 for the year ended December 31, 2024, compared to a net loss of $102,062,000 for the previous year, representing a 36.5% improvement in losses [657]. - The loss from operations improved from $108.389 million in 2023 to $70.400 million in 2024, suggesting better operational efficiency [650]. - The company's accumulated deficit increased from $594.468 million in 2023 to $659.235 million in 2024, highlighting ongoing financial challenges [649]. Cash and Securities - As of December 31, 2024, the company held $76.8 million in cash, cash equivalents, and marketable securities, a decrease from $117.959 million in 2023 [636][649]. - Cash and cash equivalents at the end of 2024 were $37,573,000, an increase from $21,709,000 at the beginning of the year, indicating a net increase of $15,864,000 [657]. - The company's marketable securities totaled $39,183,000 as of December 31, 2024, compared to $92,585,000 as of December 31, 2023, indicating a decrease of about 58% [714]. - The fair value of the company's total financial assets was $73,229,000 as of December 31, 2024, down from $96,935,000 in the previous year, reflecting a decline of approximately 24% [721]. Research and Development - Research and development expenses decreased from $61.419 million in 2023 to $40.179 million in 2024, showing a reduction in operational costs [650]. - Total research and development expenses for the year ended December 31, 2024, were $40.2 million, down from $61.4 million in 2023, reflecting a decrease of approximately 34.5% [814]. - The Company plans to reduce its workforce by approximately 55% as part of a restructuring plan aimed at aligning its workforce with strategic research and development needs [817]. Funding and Future Outlook - The company anticipates continued operating losses for the foreseeable future and will need to raise additional funding to achieve profitability [17]. - The company plans to seek additional funding through public or private equity offerings, debt financings, and strategic alliances to support its operations and product development [663]. - The company anticipates incurring additional losses until it can generate significant sales from its product candidates currently in development [660]. Shareholder Impact - The weighted average common shares outstanding increased from 54,743,490 in 2023 to 60,405,036 in 2024, indicating potential dilution for existing shareholders [650]. - The total number of shares authorized under the Incentive Plan as of December 31, 2024, was 15,848,867, with 8,604,096 shares available for future grants [785]. - The weighted average number of stock options excluded from diluted shares increased from 9,290,308 in 2023 to 11,554,173 in 2024, indicating a rise of approximately 24.4% [707]. Impairment and Expenses - The Company recognized impairment expenses for property and equipment of $2.7 million in 2024 and $3.2 million in 2023, indicating a decrease of approximately 15.6% year-over-year [679]. - The Company recorded impairment losses of $5,233,000 for the year ended December 31, 2024, compared to $5,390,000 in 2023, showing a slight decrease of about 3% [724]. - The Company recognized an impairment expense of $4.8 million in 2024, including $2.5 million for ROU assets and $2.3 million for property and equipment, due to the carrying values exceeding estimated undiscounted cash flows [751]. Lease and Sublease Agreements - The Company subleased approximately 8,000 square feet under Sublease Agreement A for an annual base rent of $0.1 million, increasing by 2.75% annually, with the sublease term running from November 1, 2023, to March 31, 2029 [735]. - The Company subleased approximately 29,000 square feet under Sublease Agreement B for an annual base rent of $0.9 million, with the sublease term from March 1, 2024, to August 2026 [738]. - The total undiscounted lease payments for the Company's operating leases are projected to be $41.182 million, with total lease liabilities amounting to $25.476 million after imputed interest [752]. Tax and Deferred Assets - The Company had a net operating loss carryforward of $339.1 million for federal tax purposes as of December 31, 2024, an increase from $265.5 million in 2023 [804]. - The valuation allowance for deferred tax assets increased by $19.1 million during the year ended December 31, 2024 [802]. - The Company had no accrued interest or penalties related to uncertain tax positions as of December 31, 2024 [809].

Financial Position - As of December 31, 2024, the company's cash, cash equivalents, and marketable securities position is approximately $76.8 million, enabling funding of operating expenses and capital expenditures into Q1 2027[5]. Restructuring Plan - The company is implementing a restructuring plan that includes ceasing lab operations in New Jersey and reducing its workforce by approximately 55%, expected to decrease annual operating costs by $9.0 million to $11.0 million[7][9]. - The company anticipates incurring approximately $2.0 million in severance and exit costs primarily in Q1 2025 due to the restructuring plan[9]. - The restructuring plan is expected to be substantially complete by the end of Q1 2025[8]. Impairment and Costs - The company expects to recognize impairment expenses for laboratory equipment of approximately $1.0 million to $3.0 million in the three-month period ending March 31, 2025[10]. Clinical Trial Updates - Updated data from the ongoing Phase 1/2 upliFT-D clinical trial for PBFT02 shows a robust increase in PGRN expression, with cerebrospinal fluid levels rising from below 3 ng/mL at baseline to 13-27 ng/mL at six months[17]. - Plasma neurofilament light chain levels were 13% lower than baseline on average at 12 months post-treatment, contrasting with untreated patients whose levels are expected to increase by 29% per year[17]. - The company plans to initiate dosing of FTD-C9orf72 patients in the first half of 2025 and expects to report 12-month data from Dose 1 in the second half of 2025[21]. Manufacturing Process - The company is transitioning to an outsourced analytical testing model as part of its manufacturing process improvements for PBFT02[14]. Forward-Looking Statements - The company cautions that forward-looking statements regarding financial impacts and clinical trial progress involve risks and uncertainties that could materially affect actual results[20].

Core Insights - Passage Bio, Inc. reported strong performance in 2024, advancing its PBFT02 program with promising data in FTD-GRN patients, showing robust and durable progranulin expression and early evidence of improvement in a disease progression biomarker [3][6] - The company has extended its cash runway into Q1 2027 by implementing cost-reduction measures and moving to an outsourced analytical testing model [6][15] - Upcoming milestones include reporting 12-month data from Dose 1 and interim safety and biomarker data from Dose 2 in 2H 2025, and seeking regulatory feedback on the registrational trial design in 1H 2026 [6][5] Clinical Trial Updates - Interim data from the upliFT-D clinical trial demonstrated that Dose 1 PBFT02 increased cerebrospinal fluid (CSF) progranulin (PGRN) levels significantly, from below 3 ng/mL at baseline to 13-27 ng/mL at six months and 22-34 ng/mL at 12 months [6] - Plasma neurofilament light chain (NfL) levels were found to be 13% lower than baseline on average at 12 months post-treatment, contrasting with an expected increase of 29% per year based on natural history data [6] - The first FTD-GRN patient has been enrolled to receive Dose 2 PBFT02, which is 50% lower than Dose 1, to facilitate discussions with health authorities regarding a registrational study design [6][3] Financial Performance - As of December 31, 2024, the company reported cash, cash equivalents, and marketable securities totaling $76.8 million, down from $114.3 million as of December 31, 2023 [15] - Research and Development (R&D) expenses for Q4 2024 were $9.6 million, and for the full year, they totaled $40.2 million, compared to $12.1 million and $61.4 million for the same periods in 2023 [15] - The net loss for Q4 2024 was $12.7 million, or $0.20 per share, and for the full year, it was $64.8 million, or $1.07 per share, showing a decrease in losses compared to the previous year [15][21]

Core Insights - Passage Bio, Inc. reported updated data from the ongoing Phase 1/2 upliFT-D clinical trial for PBFT02, a gene replacement therapy targeting frontotemporal dementia (FTD) with granulin (GRN) mutations, showing promising results in increasing CSF PGRN levels and early signs of reduced plasma NfL levels, a biomarker for disease progression [1][2][4] Clinical Trial Updates - The company is evaluating Dose 2 of PBFT02, which is 50% lower than Dose 1, in FTD-GRN and FTD-C9orf72 patients to explore dosing options and support regulatory strategy [1][4] - Interim data from FTD-GRN patients treated with Dose 1 showed CSF PGRN levels increased from below 3 ng/mL at baseline to 13-27 ng/mL at six months and 22-34 ng/mL at 12 months, with plasma NfL levels averaging 13% lower than baseline at 12 months [4][8] - The company plans to report 12-month data from Dose 1 and interim safety and biomarker data from Dose 2 in the second half of 2025 and seek regulatory feedback on the pivotal trial design in the first half of 2026 [1][6] Manufacturing and Financial Strategy - Passage Bio completed the development and scale-up of a high-productivity, suspension-based manufacturing process for PBFT02, which is expected to improve yield and reduce costs [1][4] - The company extended its cash runway into the first quarter of 2027 by transitioning to an outsourced analytical testing model and reducing operating expenses, including a workforce reduction of approximately 55% [1][4][8] Future Plans - The company is on track to initiate dosing of FTD-C9orf72 patients in the first half of 2025, with an estimated 21,000 patients affected in the U.S. and Europe, and no approved disease-modifying therapies currently available [6][8] - The upliFT-D trial is a global, multi-center, open-label study evaluating the safety and efficacy of PBFT02, with a primary endpoint focused on safety and tolerability [9][10]