PHILADELPHIA - Passage Bio, Inc. (NASDAQ:PASG), a genetic medicines company, presented promising preclinical and interim clinical data for its gene therapy product, PBFT02, at the European Society of Gene & Cell Therapy (ESGCT) Annual Congress on Thursday. The therapy is designed to treat frontotemporal dementia with GRN mutations (FTD-GRN).
The preclinical studies demonstrated that the adeno-associated virus (AAV1) vector used in PBFT02 achieved higher levels of human progranulin in the cerebrospinal fluid (CSF) compared to other vectors. In animal models, the therapy improved lysosomal histopathology and lessened neuroinflammation, key factors in FTD pathology. Additionally, the data indicated that PBFT02 had a broad distribution throughout the nervous system, including critical brain regions affected by FTD, when administered via intra-cisterna magna (ICM).
Interim clinical data from the upliFT-D Phase 1/2 study suggested that PBFT02 was generally well-tolerated and led to consistent, durable increases in CSF progranulin levels, sustained up to 12 months post-administration.
Will Chou, M.D., CEO of Passage Bio, expressed confidence in PBFT02's potential as a best-in-class therapy for raising progranulin levels, citing the preclinical results' influence on their clinical strategy. The company aims to explore the therapy's potential in additional neurodegenerative diseases that could benefit from elevated progranulin levels.
The therapy utilizes an AAV1 viral vector for ICM administration of a functional GRN gene, which is expected to increase progranulin levels and alter the course of neurodegenerative diseases. The preclinical data supporting PBFT02's development was detailed in the oral presentation at the ESGCT congress.
Passage Bio's focus remains on the development of one-time therapies targeting the underlying pathology of neurodegenerative conditions. The company's approach and progress reflect its commitment to addressing the unmet needs of patients with these diseases.
The information in this article is based on a press release statement from Passage Bio.
In other recent news, Passage Bio, Inc. revealed advances in gene therapy for dementia, with promising preclinical and interim clinical data for their lead product candidate, PBFT02. The company also reported an estimated impairment cost between $3.5 million and $5.5 million, in line with its recent sublease agreement and corporate restructuring efforts. In a strategic move, 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.
Analysts at Canaccord Genuity maintained a Buy rating for Passage Bio, based on positive interim data from the upliFT-D Phase 1/2 study. 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.
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. Finally, 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
While Passage Bio's preclinical and interim clinical data for PBFT02 show promise, the company's financial health presents a more complex picture. According to InvestingPro data, Passage Bio's market capitalization stands at $35.76 million, reflecting its current position as a small-cap biotech firm.
InvestingPro Tips highlight that Passage Bio is "quickly burning through cash" and "suffers from weak gross profit margins." These factors are not uncommon for early-stage biotech companies investing heavily in research and development. The company's operating income for the last twelve months as of Q2 2024 was -$88.02 million, underscoring the significant costs associated with developing novel gene therapies.
Despite these challenges, it's worth noting that Passage Bio "holds more cash than debt on its balance sheet," which could provide some financial flexibility as it continues to advance its pipeline. Additionally, the stock is "trading near its 52-week low," which may interest investors looking for potential entry points in the biotech sector.
For those seeking a more comprehensive analysis, InvestingPro offers 10 additional tips for Passage Bio, providing a deeper understanding of the company's financial position and market performance.
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