PHILADELPHIA - Passage Bio, Inc. (NASDAQ:PASG), a genetic medicines company, presented new preclinical and interim clinical data at the European Society of Gene & Cell Therapy (ESGCT) Annual Congress in Rome, Italy. The data suggest that their lead product candidate, PBFT02, shows promise as a gene therapy for frontotemporal dementia with GRN mutations (FTD-GRN).
The company's presentation on Thursday highlighted preclinical studies that informed the selection of the AAV1 vector for delivering PBFT02, which aims to increase progranulin levels in the central nervous system (CNS). Studies in non-human primates demonstrated that the AAV1 vector achieved higher human progranulin levels in the cerebrospinal fluid (CSF) compared to other vectors following intra-cisterna magna administration.
Further preclinical results in Grn knockout mice showed that PBFT02 improved lysosomal histopathology and reduced neuroinflammation, suggesting that higher levels of progranulin may offer additional benefits. The data also indicated that PBFT02 achieved widespread distribution throughout the nervous system in non-human primates.
Interim data from the upliFT-D clinical study indicated that the first dose of PBFT02 was generally well-tolerated and led to consistent and durable increases in CSF progranulin levels in patients, with effects sustained up to 12 months post-administration.
Dr. Sue Browne, Chief Scientific Officer, presented the findings, which support the ongoing clinical development strategy for PBFT02. Will Chou, M.D., president and CEO of Passage Bio, expressed confidence in the AAV1 vector and intracerebral magna (ICM) administration as a differentiated approach for their gene therapy.
The potential clinical benefit of PBFT02 is underpinned by extensive preclinical studies, which have shown positive effects on lysosomal function and neuroinflammation reduction in a murine FTD model. Passage Bio remains committed to advancing PBFT02 in FTD and exploring its therapeutic potential in additional neurodegenerative diseases that could benefit from elevated progranulin levels.
This article is based on a press release statement from Passage Bio.
In other recent news, Passage Bio, Inc. has made significant strides in its business operations. The company has reported an estimated impairment cost between $3.5 million and $5.5 million, aligning with its recent sublease agreement and corporate restructuring efforts. Furthermore, Passage Bio has out-licensed treatments for GM1 gangliosidosis, Krabbe disease, and metachromatic leukodystrophy to GEMMA Biotherapeutics, Inc. This deal includes an upfront payment of $10 million and potential additional payments tied to business milestones.
Additionally, Canaccord Genuity maintained a Buy rating for Passage Bio, based on positive interim data from the upliFT-D Phase 1/2 study. The firm highlighted an increase in the biomarker CSF PGRN over time, suggesting potential for the stock's value to rise. However, the company is currently evaluating options to regain compliance with Nasdaq's listing requirements, following a potential delisting notice due to its share price falling below the minimum bid price requirement.
In a strategic move, Passage Bio has appointed Thomas Kassberg as a Class I director and member of the Audit Committee. Kassberg, with an extensive background in biotechnology, was granted non-incentive stock options as part of his compensation package. Lastly, Passage Bio has received approval from the U.S. Food and Drug Administration to evaluate its gene therapy treatment, PBFT02, for frontotemporal dementia patients with C9orf72 gene mutations. These are the recent developments in Passage Bio's ongoing efforts.
InvestingPro Insights
As Passage Bio (NASDAQ:PASG) continues to advance its promising gene therapy candidate PBFT02, investors should consider some key financial metrics and insights from InvestingPro.
According to InvestingPro data, Passage Bio's market capitalization stands at $35.76 million, reflecting the company's current valuation in the biotechnology sector. This relatively small market cap is not uncommon for early-stage biotech companies focused on developing novel therapies.
InvestingPro Tips highlight that Passage Bio holds more cash than debt on its balance sheet, which is crucial for a company in the research and development phase. This financial position provides some runway for the company to continue its clinical programs, including the development of PBFT02.
However, it's important to note that Passage Bio is quickly burning through cash, as indicated by another InvestingPro Tip. This is typical for biotech companies investing heavily in R&D, but it underscores the importance of successful clinical outcomes and potential future financing needs.
The company's stock is currently trading near its 52-week low, which may present an opportunity for investors who believe in the long-term potential of PBFT02 and Passage Bio's pipeline. However, this should be weighed against the fact that analysts do not anticipate the company to be profitable this year, as noted in the InvestingPro Tips.
For investors seeking a more comprehensive analysis, InvestingPro offers additional tips and insights beyond those mentioned here. In fact, there are 10 more InvestingPro Tips available for Passage Bio, which could provide valuable context for understanding the company's financial health and market position as it progresses with its gene therapy developments.
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