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Passage Bio Reports First Quarter 2024 Financial Results and Provides Recent Business Highlights

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Passage Bio (NASDAQ: PASG) reported its Q1 2024 financial results and provided business updates, including advances in its global upliFT-D clinical trial for FTD-GRN. The trial's Dose 1 PBFT02 consistently elevated CSF progranulin at six months in two patients. All five patients in Cohort 1 have been dosed, with safety and biomarker data expected in 2H 2024. Cohort 2 dosing is planned to start by the end of 1H 2024. The company initiated discussions with the FDA for using PBFT02 to treat FTD-C9orf72, with feedback expected in 2H 2024. Financially, the company ended Q1 with $104.5 million in cash, projecting a cash runway into Q4 2025. Net loss decreased to $16.7 million from $34.3 million year-over-year.

Positive
  • Dose 1 PBFT02 consistently elevated CSF progranulin at six months in patients in the upliFT-D trial.
  • Completed dosing of all five patients in Cohort 1 of the upliFT-D trial.
  • On track to deliver 6-month safety and biomarker data from Cohort 1 in 2H 2024.
  • Cohort 2 dosing expected to start by end of 1H 2024.
  • Initiated regulatory process with FDA for PBFT02 in treating FTD-C9orf72.
  • Strong cash position with $104.5 million, sufficient to fund operations into Q4 2025.
  • Net loss reduced to $16.7 million from $34.3 million year-over-year.
  • R&D expenses decreased to $11.5 million from $16.8 million year-over-year.
  • G&A expenses dropped to $6.5 million from $19.0 million year-over-year.
Negative
  • Cash position decreased significantly from $167.8 million in the previous year.
  • Patient 3 plasma progranulin levels remained below healthy adult controls six months post-treatment.
  • Dependence on continued trial success and regulatory approvals for future growth.
  • Impact of non-recurring G&A charges in the previous period challenges year-over-year comparisons.

Insights

The financial results reported by Passage Bio reveal some notable trends. Firstly, the company's cash position has declined from $167.8 million to $104.5 million over the year, although they have managed to raise an additional $8.7 million through their ATM Facility. The company expects these funds to sustain operations until Q4 2025, providing a stable financial runway for the upcoming research milestones.

Research and Development (R&D) expenses have decreased to $11.5 million from $16.8 million in the same quarter the previous year. This reduction suggests a more efficient allocation of resources or a shift in project phases. Similarly, General and Administrative (G&A) expenses have significantly dropped from $19.0 million to $6.5 million, primarily due to the absence of non-recurring charges related to the Amended Catalent Agreements. As a result, the company has effectively halved its net loss, from $34.3 million to $16.7 million.

For investors, this data indicates Passage Bio is strategically managing its resources while advancing its clinical trials. However, the company's continued negative net income warrants cautious optimism. Monitoring future financial reports for sustained cost management and successful trial outcomes will be essential.

The updated interim data from the upliFT-D trial is promising. The consistent elevation of cerebrospinal fluid (CSF) progranulin levels in two patients at six months post-treatment with Dose 1 PBFT02 is a important indicator of the drug's potential efficacy. Progranulin is a protein that plays a significant role in neurodegenerative diseases and its deficiency is linked to frontotemporal dementia (FTD-GRN).

The trial's continued focus on Dose 1 in Cohort 2, with plans to initiate dosing by the end of the first half of 2024, highlights the preliminary success of the clinical protocol. This steady progress in clinical trials is critical for patients with FTD, a condition with limited therapeutic options.

However, it's essential to note that while the consistent elevation in CSF progranulin is encouraging, the patient sample size is small (n=2) and more extensive data is needed to confirm these findings. The upcoming 6-month safety and biomarker data from Cohort 1 will be pivotal in determining the drug's future.

  • Updated interim data from the upliFT-D trial in FTD-GRN demonstrated that Dose 1 PBFT02 achieved consistent elevation of CSF progranulin at six months post-treatment in the two patients with longest follow-up
  • Completed dosing of Cohort 1 (n=5) in upliFT-D trial; on track to deliver 6-month safety and biomarker data from Cohort 1 in 2H 2024
  • Plan to continue studying Dose 1 in Cohort 2 of upliFT-D trial; expect to initiate dosing by the end of 1H 2024, as planned
  • Initiated regulatory process with FDA to receive feedback on pathway to treating FTD-C9orf72 patients with PBFT02
  • Robust balance sheet sufficient to deliver meaningful clinical data, with cash runway into Q4 2025

PHILADELPHIA, May 14, 2024 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (Nasdaq: PASG), a clinical stage genetic medicines company focused on improving the lives of patients with neurodegenerative diseases, today reported financial results for the first quarter ended March 31, 2024 and provided recent business highlights.

"The beginning of 2024 has been marked by strong execution as we continue to advance our global upliFT-D clinical trial in FTD-GRN and plan to deliver meaningful data over the next twelve months,” said Will Chou, M.D., president and chief executive officer of Passage Bio. “We are excited to share updated interim data demonstrating continued elevation in CSF progranulin levels at six months after treatment in two patients. This consistent progranulin response underscores the differentiated profile of PBFT02 and gives us confidence in the impact our therapy may have on patients at the current dose. Momentum in our program has been strong since we shared encouraging initial data last year, and we are pleased to have completed dosing of all five patients in Cohort 1.”

Recent Highlights

  • Dose 1 PBFT02 achieved consistent elevation of cerebrospinal fluid (CSF) progranulin (PGRN) at six months post-treatment in two patients with the longest follow-up in the global Phase 1/2 upliFT-D clinical trial evaluating PBFT02 for frontotemporal dementia (FTD) with granulin mutations (GRN): Building on interim data shared in December 2023, Dose 1 PBFT02 has now been shown to elevate CSF PGRN levels at six months post-treatment in Patients 2 and 3, with a concentration of 21.7 to 27.3 ng/mL. Consistent with prior findings, Patient 3 plasma PGRN levels remained below levels found in healthy adult controls at up to six months post-treatment. PBFT02 continues to be well-tolerated in participants who received the enhanced immunosuppression regimen at up to eleven months post-treatment.

  • Completed dosing of Cohort 1 patients in upliFT-D clinical trial: Completed dosing of Cohort 1, with five patients now having received Dose1 of PBFT02. The company remains on track to deliver 6-month safety and biomarker data from Cohort 1 in the second half of 2024.

  • Plan to continue studying Dose 1 in Cohort 2 patients in upliFT-D trial: Based on initial clinical evidence that Dose 1 PBFT02 achieved robust CSF progranulin levels, sustained for up to six months post-treatment (n=2), the company plans to continue studying Dose 1 in Cohort 2, which will consist of three to five patients. Recruitment momentum remains strong, with multiple GRN+ patients identified and being evaluated for study eligibility across seven clinical trial sites in Brazil, Canada, the United States and Europe. The company continues to expect to initiate dosing of Cohort 2 later in the first half of 2024.

  • Initiated regulatory process with FDA to receive feedback on the company’s plans to treat FTD-C9orf72 patients with PBFT02: The company expects to receive feedback from the U.S. Food and Drug Administration (FDA) on the clinical development pathway for treating FTD patients with mutations in the C9orf72 gene with PBFT02 in the second half of 2024. FTD-C9orf72, like FTD-GRN, is characterized by TDP-43 pathology, in which the TDP-43 protein abnormally accumulates in the cytoplasm of neurons, contributing to neuronal dysfunction and degeneration. Published, third-party preclinical studies have shown that elevating progranulin levels can reduce TDP-43 pathology and slow neurodegeneration. There are currently no approved disease-modifying therapies for FTD-C9or72, which is estimated to affect approximately 21,000 patients in the U.S. and Europe.

Anticipated Upcoming Milestones:

FTD-GRN

  • Initiate dosing for Cohort 2 FTD-GRN patients in 1H 2024
  • Report 6-month safety and biomarker data from Cohort 1 patients in 2H 2024
  • Announce 12-month follow-up data from Cohort 1 patients in 1H 2025
  • Report initial safety and biomarker data from Cohort 2 patients in 1H 2025

FTD-C9orf72 and ALS

  • Obtain regulatory feedback on the pathway to treating FTD-C9orf72 and amyotrophic lateral sclerosis (ALS) patients with PBFT02 in 2H 2024

First Quarter 2024 Financial Results

  • Cash Position: Cash, cash equivalents and marketable securities were $104.5 million as of March 31, 2024, as compared to $167.8 million as of March 31, 2023. During the quarter ended March 31, 2024, the company raised $8.7 million, net of offering costs, under its ATM Facility. The company expects current cash, cash equivalents and marketable securities to fund operations into Q4 2025.
  • Research and Development (R&D) Expenses: R&D expenses were $11.5 million for the quarter ended March 31, 2024, as compared to $16.8 million for the quarter ended March 31, 2023.
  • General and Administrative (G&A) Expenses: G&A expenses were $6.5 million for the quarter ended March 31, 2024, as compared to $19.0 million for the quarter ended March 31, 2023. G&A Expenses in the period in 2023 included $11.3 million in non-recurring charges related to the Amended Catalent Agreements.
  • Net Loss: Net loss was $16.7 million, or $0.30 per basic and diluted share, for the quarter ended March 31, 2024, as compared to a net loss of $34.3 million, or $0.63 per basic and diluted share, for the quarter ended March 31, 2023.

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: www.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 progress of clinical trials and the availability of clinical data from such trials; our expectations about our collaborators’ and partners’ ability to execute key initiatives; our expectations about manufacturing plans and strategies; our expectations about cash runway; our expectations about potential out-licensing opportunities related to PBGM01, PBKR03 and PBML04; 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.

 
Passage Bio, Inc.
Balance Sheets
 
  (Unaudited)   
(in thousands, except share and per share data)    March 31, 2024    December 31, 2023
Assets      
Current assets:      
Cash and cash equivalents $36,773  $21,709 
Marketable securities  67,752   92,585 
Prepaid expenses and other current assets  1,784   923 
Prepaid research and development  1,984   2,742 
Total current assets  108,293   117,959 
Property and equipment, net  14,489   15,295 
Right of use assets - operating leases  17,087   16,858 
Other assets  662   433 
Total assets $140,531  $150,545 
Liabilities and stockholders’ equity      
Current liabilities:      
Accounts payable $1,687  $1,298 
Accrued expenses and other current liabilities  7,483   11,670 
Operating lease liabilities  3,671   3,373 
Total current liabilities  12,841   16,341 
Operating lease liabilities - noncurrent  22,807   22,921 
Total liabilities  35,648   39,262 
       
Stockholders’ equity:      
Preferred stock, $0.0001 par value: 10,000,000 shares authorized; no shares issued and outstanding at both March 31, 2024 and December 31, 2023      
Common stock, $0.0001 par value: 300,000,000 shares authorized; 61,611,796 shares issued and outstanding at March 31, 2024 and 54,944,130 shares issued and outstanding at December 31, 2023  6   5 
Additional paid‑in capital  716,125   705,789 
Accumulated other comprehensive income (loss)  (69)  (43)
Accumulated deficit  (611,179)  (594,468)
Total stockholders’ equity  104,883   111,283 
Total liabilities and stockholders’ equity $140,531  $150,545 


 
Passage Bio, Inc.
Statements of Operations and Comprehensive Loss
(Unaudited)
 
  Three Months Ended March 31, 
(in thousands, except share and per share data) 2024     2023 
Operating expenses:      
Research and development $11,535  $16,836 
General and administrative  6,515   19,047 
Loss from operations  (18,050)  (35,883)
Other income (expense), net  1,339   1,545 
Net loss $(16,711) $(34,338)
Per share information:      
Net loss per share of common stock, basic and diluted $(0.30) $(0.63)
Weighted average common shares outstanding, basic and diluted  56,295,540   54,618,799 
Comprehensive loss:      
Net loss $(16,711) $(34,338)
Unrealized gain (loss) on marketable securities  (26)  539 
Comprehensive loss $(16,737) $(33,799)


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


FAQ

What were Passage Bio's Q1 2024 financial results?

Passage Bio reported a net loss of $16.7 million, or $0.30 per share, with $104.5 million in cash, cash equivalents, and marketable securities.

What progress has Passage Bio made in the upliFT-D trial?

The upliFT-D trial's Dose 1 PBFT02 elevated CSF progranulin at six months in patients. All five patients in Cohort 1 have been dosed, with Cohort 2 dosing expected by end of 1H 2024.

When will Passage Bio start dosing Cohort 2 in the upliFT-D trial?

Passage Bio plans to start dosing Cohort 2 later in the first half of 2024.

How is Passage Bio advancing its FTD-C9orf72 program?

Passage Bio initiated the regulatory process with the FDA and expects feedback on its clinical development pathway for FTD-C9orf72 in 2H 2024.

What is Passage Bio's cash runway based on its current financials?

Passage Bio expects its current cash, cash equivalents, and marketable securities to fund operations into Q4 2025.

Passage Bio, Inc.

NASDAQ:PASG

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Biotechnology
Biological Products, (no Disgnostic Substances)
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United States of America
PHILADELPHIA