Form 8-K - Current report

UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington, D.C. 20549

FORM 8-K

CURRENT REPORT

Pursuant to Section 13 or 15(d)

of the Securities Exchange Act of 1934

Date of Report (Date of earliest event reported): January 8, 2025

PASSAGE BIO, INC.

(Exact name of registrant as specified in its charter)

Delaware	001-39231	82-2729751
(State or other jurisdiction of incorporation)	(Commission File Number)	(IRS Employer Identification No.)

One Commerce Square 2005 Market Street, 39^th Floor Philadelphia, PA	19103
(Address of principal executive offices)	(Zip Code)

(267) 866-0311

(Registrant’s telephone number, including area code)

N/A

(Former name or former address, if changed since last report)

Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:

¨	Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)

¨	Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)

¨	Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))

¨	Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))

Securities registered pursuant to Section 12(b) of the Act:

Title of each class	Trading symbol(s)	Name of each exchange on which registered
Common Stock, $0.0001 Par Value Per Share	PASG	The Nasdaq Stock Market LLC (Nasdaq Global Select Market)

Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§240.12b-2 of this chapter).

Emerging growth company x

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ¨

Item 2.02 Results of Operations and Financial Condition.

On January 10, 2025, the Company disclosed in a corporate presentation, among other things, that the preliminary, unaudited amount of the Company’s cash, cash equivalents and marketable securities position as of December 31, 2024 is approximately $76.8 million, which, including the activities noted below, will enable it to fund its operating expenses and capital expenditure requirements into the first quarter of 2027. This amount is preliminary, unaudited and may change, was prepared by management and is based on the most current information available to management. Further, this amount is subject to completion by management of the financial statements as of and for the year ended December 31, 2024, including completion of the review procedures, final adjustments and other developments that may arise between now and the time the financial results for this period are finalized, and completion of the audit of such financial statements.

The information in this Item 2.02 shall not be deemed to be “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that section or Sections 11 and 12(a)(2) of the Securities Act of 1933, as amended (the “Securities Act”). The information contained in this Item 2.02 shall not be incorporated by reference into any other filing under the Exchange Act or under the Securities Act, except as shall be expressly set forth by specific reference in such filing.

Item 2.05 Costs Associated with Exit or Disposal Activities.

On January 8, 2025, the Board of Directors of Passage Bio, Inc. (the “Company”) approved, and management began implementing, a restructuring plan (the “Restructuring Plan”) to (i) cease its lab operations at its approximately 62,000 feet of leased laboratory space in Hopewell, New Jersey (the “Laboratory Lease”) and (ii) reduce operating costs and better align its workforce with the needs of its strategic research and development strategy. The implementation of the Restructuring Plan should be substantially complete by the end of the first quarter 2025.

Under the Restructuring Plan, the Company is reducing its overall workforce by approximately 55%. Impacted employees are eligible to receive severance benefits. These severance benefits are contingent upon an impacted employee’s execution (and non-revocation) of a severance agreement, which includes a general release of claims against the Company.

The Company expects that the Restructuring Plan will decrease its annual operating costs by approximately $9.0 million to $11.0 million, excluding the Company’s estimates of aggregate severance and exit costs that it will incur of approximately $2.0 million, which will be recorded primarily in the first quarter of 2025. The cost that the Company expects to incur in connection with the Restructuring Plan is subject to a number of assumptions, and actual results may differ materially. The Company may also incur additional costs not currently contemplated due to events that may occur as a result of, or that are associated with, the Restructuring Plan.

Item 2.06 Material Impairments.

In connection with the Restructuring Plan, the Company is pursuing opportunities to sublease the space at the Laboratory Lease to offset portions of its financial obligations and is pursuing opportunities to sell laboratory equipment. As a result, the Company reassessed asset groups and evaluated such asset groups for impairment under Accounting Standards Codification 360, Long-lived assets: Impairment or disposal of long-lived assets, for all long-lived assets at the Laboratory Lease, which comprises primarily of right-of-use assets, leasehold improvements and laboratory equipment. As a result, the Company expects to recognize impairment expenses for its laboratory equipment of approximately $1.0 million to $3.0 million in the three-month period ending March 31, 2025. This range is preliminary, unaudited and may change, subject to management’s completion of the reassessment of asset groups and finalization of assumptions used in impairment testing. Additionally, this range was prepared by management based on the most current information available to management, and is subject to the completion by management of the financial statements as of and for the three-month period ending March 31, 2025, including completion of the review procedures, final adjustments and other developments that may arise between now and the time the financial results for this period are finalized, and completion of the audit of such financial statements. Additionally, if the Company is successful in entering sublease agreements or agreements for the sale of laboratory equipment in the future, additional impairments of right-of-use assets, leasehold improvements and laboratory equipment could occur based on the economic terms included in such agreements.

The non-cash impairment expenses do not impact the Company’s cash runway and the Company expects that its existing cash, cash equivalents, and marketable securities, including the impacts of the Restructuring Plan, will enable it to fund its operating expenses and capital expenditure requirements into the first quarter of 2027.

Item 7.01 Regulation FD Disclosure.

On January 10, 2025, the Company issued a press release announcing the Restructuring Plan and other business updates related to its PBFT02 program. A copy of the press release is attached as Exhibit 99.1 to this Current Report on Form 8-K.

The information in this Item 7.01, including Exhibit 99.1 to this Current Report on Form 8-K, shall not be deemed to be “filed” for purposes of Section 18 of the Exchange Act, or otherwise subject to the liabilities of that section or Sections 11 and 12(a)(2) of the Securities Act. The information contained in this Item 7.01 and in the accompanying Exhibit 99.1 shall not be incorporated by reference into any other filing under the Exchange Act or under the Securities Act, except as shall be expressly set forth by specific reference in such filing.

Item 8.01 Other Events.

PBFT02 Program Updates

On January 10, 2025, the Company announced updated data from the ongoing Phase 1/2 upliFT-D clinical trial evaluating PBFT02 for the treatment of frontotemporal dementia with mutations in the GRN (“FTD-GRN”) and anticipated upcoming milestones. The Company also announced the successful completion of process development and scale-up of an improved-productivity, suspension-based manufacturing process for PBFT02 and its plans to transition to an outsourced analytical testing model.

Updated interim data from FTD-GRN patients treated with Dose 1 PBFT02

Biomarkers

Dose 1 of PBFT02 treatment resulted in a robust and durable increase in progranulin (“PGRN”) expression.

PBFT02 consistently increased cerebrospinal fluid (“CSF”) PGRN expression in all patients from below 3 ng/mL at baseline to 13 – 27 ng/mL at six months (n=4) and 22 – 34 ng/mL at 12 months (n=2).

CSF PGRN levels generally plateaued by month 6 and have remained durable through the longest available follow-up of 18 months (n=1).

Plasma neurofilament light chain (“NfL”) levels were 13% lower than baseline on average at 12 months (n=2) post-treatment.

In contrast, in untreated symptomatic FTD-GRN patients, plasma NfL levels are expected to increase by 29% per year, according to published natural history data.

Plasma PGRN expression remained below healthy reference levels across all patients.

Safety (patient follow-up up to 18 months as of December 9, 2024, data cutoff)

In five of seven patients, all treatment emergent adverse events were mild to moderate in severity.

Two of seven patients experienced a total of three serious adverse events (“SAEs”). As previously disclosed, the first treated patient experienced the asymptomatic SAEs of venous sinus thrombosis (VST) and hepatotoxicity, leading to a revised immunosuppression regimen in all subsequent patients (1,000 mg IV methylprednisolone on days 1-3 followed by 60 mg oral prednisone through day 60). Patient 7 also experienced the SAE of VST, which was asymptomatic and completely resolved prior to day 30 following treatment with anticoagulants. This patient had no evidence of hepatotoxicity, immune response or other laboratory abnormalities and remains enrolled in the clinical study.

No evidence of clinically significant immune responses in any patient who received the revised immunosuppression regimen

No evidence of dorsal root ganglion toxicity, as measured by nerve conduction studies, and no complications during intra cisterna magna administration were observed across any of the seven treated patients.

Anticipated Upcoming Milestones

Evaluating Dose 2, 50% lower than Dose 1, in subsequent FTD-GRN and FTD-C9orf72 patients to allow for dose exploration and support program regulatory strategy.

Expect to report 12-month data from Dose 1 and interim safety and biomarker data from Dose 2 in the second half of 2025.

Initiate dosing of FTD-C9orf72 patients in the first half 2025.

Plan to seek regulatory feedback on FTD-GRN registrational trial design in the first half of 2026.

Forward-Looking Statements.

This Current Report on Form 8-K contains forward-looking statements within the meaning of the U.S. Private Securities Litigation Reform Act of 1995 and other federal securities laws. Any statements contained herein that do not describe historical facts, including, but not limited to, statements related to the expected costs associated with termination benefits and the financial impact of the restructuring and the reduction in workforce; the Company’s expectations about cash runway; the Company’s expectations about the timing and execution of anticipated milestones, including the outsource of its analytical testing model, the initiation of dosing of FTD-C9orf72, the timing of feedback from regulatory authorities, the progress of clinical trials and the availability of clinical data from such trials; and the ability of our lead product candidates to treat their respective target CNS disorders, are forward-looking statements that involve risks and uncertainties that could cause actual results to differ materially from those discussed in such forward-looking statements. Such risks and uncertainties include, among others, the risks identified in the Company’s filings with the SEC, including its Quarterly Report on Form 10-Q for the three months ended September 30, 2024, filed with the SEC on November 13, 2024, and subsequent filings with the SEC. Any of these risks and uncertainties could materially and adversely affect the Company’s results of operations, which would, in turn, have a significant and adverse impact on the Company’s stock price. The Company cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. The Company undertakes no obligation to update publicly any forward-looking statements to reflect new information, events or circumstances after the date they were made or to reflect the occurrence of unanticipated events.

Item 9.01 Financial Statements and Exhibits.

(d) Exhibits

Exhibit No.		Description
99.1		Passage Bio, Inc. press release dated January 10, 2025.
104		Cover Page Interactive Data File (formatted as Inline XBRL).

SIGNATURE

Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.

	PASSAGE BIO, INC.

Date: January 10, 2025	By:	/s/ Kathleen Borthwick
		Kathleen Borthwick
		Chief Financial Officer

Exhibit 99.1

PASSAGE BIO ANNOUNCES INTERIM DATA FROM UPLIFT-D STUDY IN FTD-GRN AND PROVIDES BUSINESS UPDATES

PBFT02 demonstrated durable, elevated CSF PGRN levels and early evidence of reduction in plasma NfL levels, a disease progression biomarker, compared to published natural history data

Evaluating Dose 2, 50% lower than Dose 1, in subsequent FTD-GRN and FTD-C9orf72 patients to allow for dose exploration and support regulatory strategy

Expect to report 12-month data from Dose 1 and interim safety and biomarker data from Dose 2 in 2H 2025; plan to seek regulatory feedback on FTD-GRN pivotal trial design in 1H 2026

Completed process development and scale-up of a high-productivity, suspension-based manufacturing process for PBFT02

Extended cash runway into 1Q 2027 by moving to outsourced analytical testing model and reducing operating expenses, allowing for the achievement of meaningful program milestones

PHILADELPHIA – January 10, 2025 – Passage Bio, Inc. (Nasdaq: PASG), a clinical stage genetic medicines company focused on improving the lives of patients with neurodegenerative diseases, today reported updated data from the ongoing Phase 1/2 upliFT-D clinical trial evaluating PBFT02 for the treatment of frontotemporal dementia (FTD) with granulin (GRN) mutations and anticipated upcoming milestones. The company also announced the successful completion of process development and scale-up of a suspension-based manufacturing process for PBFT02. As the PBFT02 program continues to advance, the company reviewed its operating needs and will transition to an outsourced analytical testing model. This action, coupled with an associated workforce reorganization and reductions in operating expenses, extends cash runway through meaningful program milestones into the first quarter of 2027.

“We are pleased to report updated interim data from our ongoing upliFT-D clinical trial in FTD-GRN patients showing that Dose 1 PBFT02 consistently increased CSF PGRN expression and that this elevation translated to early signals of improvement in a disease progression biomarker when compared to published natural history data,” said Will Chou, M.D., president and chief executive officer of Passage Bio. “Given the robust levels of CSF PGRN achieved with Dose 1 and to support future discussions with health authorities regarding the registrational pathway, we look forward to introducing Dose 2, which is fifty percent lower than Dose 1, for subsequent FTD-GRN and FTD-C9orf72 patients. We remain focused on advancing the upliFT-D study in each of these patient populations and look forward to sharing additional data in the second half of 2025.”

“As our PBFT02 program advances, we continue to assess our operating needs to ensure that we can deliver on meaningful program milestones as we endeavor to bring this promising therapy to the FTD patient community,” Dr. Chou continued. “After careful consideration, we will transition to an outsourced analytical testing model and have restructured our organization and reduced operating expenses accordingly. Following the implementation of these actions, we expect existing cash resources will be sufficient to fund operations into the first quarter of 2027, which will allow us to further validate the potential of PBFT02 and determine the registrational pathway for the program. We want to thank our talented team for their commitment and important contributions as we continue to pursue our mission of improving the lives of patients with neurodegenerative diseases.”

Updated interim data from FTD-GRN patients treated with Dose 1 PBFT02:

Biomarkers

Dose 1 of PBFT02 treatment resulted in a robust and durable increase in PGRN expression.

	·	PBFT02 consistently increased CSF PGRN expression in all patients from below 3 ng/mL at baseline to 13 – 27 ng/mL at six months (n=4) and 22 – 34 ng/mL at 12 months (n=2).
	·	CSF PGRN levels generally plateaued by month 6 and have remained durable through the longest available follow-up of 18 months (n=1).

Plasma NfL levels were 13% lower than baseline on average at 12 months (n=2) post-treatment.

In contrast, in untreated symptomatic FTD-GRN patients, plasma NfL levels are expected to increase by 29% per year, according to published natural history dataⁱ.

Plasma PGRN expression remained below healthy reference levels across all patients.

Safety (patient follow-up up to 18 months as of December 9, 2024, data cutoff)

	·	In 5 of 7 patients, all treatment emergent adverse events (AEs) were mild to moderate in severity.
	·	2 of 7 patients experienced a total of 3 serious adverse events (SAEs). As previously disclosed, the first treated patient experienced the asymptomatic SAEs of venous sinus thrombosis (VST) and hepatotoxicity, leading to a revised immunosuppression regimen in all subsequent patients (1,000 mg IV methylprednisolone on days 1-3 followed by 60 mg oral prednisone through day 60). Patient 7 also experienced the SAE of VST, which was asymptomatic and completely resolved prior to day 30 following treatment with anticoagulants. This patient had no evidence of hepatotoxicity, immune response or other laboratory abnormalities and remains enrolled in the clinical study.
	·	No evidence of clinically significant immune responses in any patient who received the revised immunosuppression regimen.
	·	No evidence of dorsal root ganglion (DRG) toxicity, as measured by nerve conduction studies, and no complications during intra cisterna magna (ICM) administration were observed across any of the seven treated patients.

Recent Highlights:

Plan to evaluate Dose 2, a 50% lower dose than Dose 1, to allow for dose exploration and support program regulatory strategy: Given robust CSF PGRN expression achieved at Dose 1 and to aid future discussions with health authorities regarding the registrational study design, the company has introduced a lower dose level, Dose 2, which is fifty percent of Dose 1. Cohort 2, which consists of five FTD-GRN patients, will be split between the dose levels; two patients have received Dose 1 and the remaining three patients in the cohort will receive Dose 2. The study is actively enrolling for treatment at Dose 2, and following completion of Cohort 2, the company plans to continue enrollment in the optional Cohort 3 (n=5).

Completed process development and scale-up of a high-productivity, suspension-based manufacturing process for PBFT02: The company completed internal development of a suspension-based, GMP-ready manufacturing process for PBFT02 at 200-liter scale. This process is substantially more efficient than the current adherent-based process, with improved yield and the promise of a lower cost of goods. In addition, the company developed and aligned with FDA on the suitability of a potency assay for the release of PBFT02 for late-stage clinical studies and commercialization. These two achievements position the PBFT02 program well for late-stage development.

Extended cash runway into 1Q 2027 by moving to outsourced analytical testing model and reducing operating expenses: After assessing its operating needs to support the continued advancement of the PBFT02 program, the company will transition to an outsourced analytical testing model. This transition, coupled with an associated reduction in workforce of approximately 55% and reductions in operating expenses, is expected to extend cash runway into the first quarter of 2027. The company will continue to focus on execution of the ongoing upliFT-D clinical trial in FTD-GRN and FTD-C9orf72 and the advancement of its preclinical program in Huntington’s disease.

On track to initiate dosing of FTD-C9orf72 patients in 1H 2025: The company has amended the upliFT-D clinical trial protocol to include two cohorts of FTD-C9orf72 patients to be enrolled sequentially. Each cohort will consist of up to five symptomatic FTD patients with C9orf72 gene mutations; initially, patients will receive Dose 2 PBFT02. The protocol amendment is under review at clinical trial sites and the company remains on track to dose the first FTD-C9orf72 patient in the first half of 2025. FTD-C9orf72 is estimated to affect approximately 21,000 patients between the United States and Europe and there are currently no disease modifying therapies approved. Preclinical studies have demonstrated that increasing PGRN levels can slow neurodegeneration and reduce TDP-43 pathology, which underlies the disease.

Anticipated Upcoming Milestones:

FTD-GRN

	·	Report 12-month data from Dose 1 and interim safety and biomarker data from Dose 2 in 2H 2025
	·	Seek regulatory feedback on registrational trial design in 1H 2026

FTD-C9orf72

Initiate dosing of FTD-C9orf72 patients in 1H 2025

About upliFT-D (NCT04747431)

upliFT-D is a Phase 1/2 global, multi-center, open-label clinical trial of PBFT02 administered by single injection into the cisterna magna in patients aged 35 to 75 years with FTD-GRN or FTD-C9orf72. The clinical trial will sequentially enroll three FTD-GRN cohorts and two FTD-C9orf72 cohorts. Enrollment is currently ongoing. The primary endpoint of the clinical trial is to evaluate the safety and tolerability of PBFT02. Secondary endpoints include disease biomarkers and clinical outcome measures. upliFT-D is a two-year clinical trial with a three-year safety extension.

Passage Bio is pursuing several initiatives to support clinical trial recruitment and enrollment, including a collaborative partnership with InformedDNA to provide no-cost genetic counseling and testing for adults who have been diagnosed by their physicians with FTD. More information about upliFT-D can be found here.

About PBFT02

PBFT02 is a gene replacement therapy that utilizes an AAV1 viral vector to deliver, through ICM administration, a functional GRN gene that encodes PGRN. This vector construct and delivery approach aim to elevate PGRN levels in the central nervous system to alter the course of neurodegenerative diseases. Interim clinical data from the upliFT-D Phase 1/2 study in FTD-GRN participants shows that ICM administration of PBFT02 resulted in robust PGRN elevations in the CSF.

The potential clinical benefit of PBFT02 is supported by extensive preclinical studies. In non-human primates, a single ICM administration of PBFT02 led to broad vector distribution throughout the CNS, and robust, dose-dependent elevations in PGRN levels in CSF. An NHP study also demonstrated that AAV1 was particularly proficient at transducing ependymal cells. In a murine FTD model, PBFT02 administration improved lysosomal function and reduced neuroinflammation.

About Passage Bio

Passage Bio (Nasdaq: PASG) is a clinical stage genetic medicines company on a mission to improve the lives of patients with neurodegenerative diseases. Our primary focus is the development and advancement of cutting-edge, one-time therapies designed to target the underlying pathology of these conditions. Passage Bio’s lead product candidate, PBFT02, seeks to treat neurodegenerative conditions, including frontotemporal dementia, by elevating progranulin levels to restore lysosomal function and slow disease progression.

To learn more about Passage Bio and our steadfast commitment to protecting patients and families against loss in neurodegenerative conditions, please visit: passagebio.com.

Forward-Looking Statements

This press release contains “forward-looking statements” within the meaning of, and made pursuant to the safe harbor provisions of, the Private Securities Litigation Reform Act of 1995, including, but not limited to: our expectations about timing and execution of anticipated milestones, including the outsource of our analytical testing model, the initiation of dosing of FTD-C9orf72 patients, timing of feedback from regulatory authorities, the progress of clinical studies and the availability of clinical data from such trials; our expectations about our collaborators’ and partners’ ability to execute key initiatives; the financial impact of the restructuring and reduction in workforce and our expectations about cash runway; and the ability of our product candidates to treat their respective target CNS disorders. These forward-looking statements may be accompanied by such words as “aim,” “anticipate,” “believe,” “could,” “estimate,” “expect,” “forecast,” “goal,” “intend,” “may,” “might,” “plan,” “potential,” “possible,” “will,” “would,” and other words and terms of similar meaning. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including: our ability to develop and obtain regulatory approval for our product candidates; the timing and results of preclinical studies and clinical trials; risks associated with clinical trials, including our ability to adequately manage clinical activities, unexpected concerns that may arise from additional data or analysis obtained during clinical trials, regulatory authorities may require additional information or further studies, or may fail to approve or may delay approval of our drug candidates; the occurrence of adverse safety events; the risk that positive results in a preclinical study or clinical trial may not be replicated in subsequent trials or success in early stage clinical trials may not be predictive of results in later stage clinical trials; failure to protect and enforce our intellectual property, and other proprietary rights; our dependence on collaborators and other third parties for the development and manufacture of product candidates and other aspects of our business, which are outside of our full control; risks associated with current and potential delays, work stoppages, or supply chain disruptions; and the other risks and uncertainties that are described in the Risk Factors section in documents the company files from time to time with the Securities and Exchange Commission (SEC), and other reports as filed with the SEC. Passage Bio undertakes no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise.

For further information, please contact:

Investors:

Stuart Henderson

Passage Bio

267.866.0114

shenderson@passagebio.com

Media:

Mike Beyer
Sam Brown Inc. Healthcare Communications
312.961.2502
MikeBeyer@sambrown.com

ⁱ Saracino et al, J Neurol Neurosurg Psych 2021; 92:1278-1288.

Cover	Jan. 08, 2025
Cover [Abstract]
Document Type	8-K
Amendment Flag	false
Document Period End Date	Jan. 08, 2025
Entity File Number	001-39231
Entity Registrant Name	PASSAGE BIO, INC.
Entity Central Index Key	0001787297
Entity Tax Identification Number	82-2729751
Entity Incorporation, State or Country Code	DE
Entity Address, Address Line One	One Commerce Square
Entity Address, Address Line Two	2005 Market Street, 39th Floor
Entity Address, City or Town	Philadelphia
Entity Address, State or Province	PA
Entity Address, Postal Zip Code	19103
City Area Code	267
Local Phone Number	866-0311
Written Communications	false
Soliciting Material	false
Pre-commencement Tender Offer	false
Pre-commencement Issuer Tender Offer	false
Title of 12(b) Security	Common Stock, $0.0001 Par Value Per Share
Trading Symbol	PASG
Security Exchange Name	NASDAQ
Entity Emerging Growth Company	true
Elected Not To Use the Extended Transition Period	false