PHILADELPHIA - Passage Bio, Inc. (NASDAQ:PASG), a genetic medicines company, has received a favorable response from the U.S. Food and Drug Administration (FDA) regarding its plans to evaluate a gene therapy treatment, PBFT02, for frontotemporal dementia (FTD) patients with C9orf72 gene mutations.
This decision follows a Type C meeting with the agency, where the company presented preclinical and early clinical data to support the expansion of its ongoing upliFT-D trial.
The proposed expansion of the upliFT-D Phase 1/2 global study aims to include a new cohort of patients, specifically those with FTD-C9orf72 mutations, which is a subset of FTD characterized by certain pathological markers and estimated to affect around 21,000 individuals in the United States and Europe.
The company plans to submit the revised trial protocol to health authorities and ethics committees soon, with the goal of starting patient dosing in the first half of 2025.
PBFT02, which has been granted Fast Track and Orphan Drug designations by the FDA, and an Orphan designation from the European Commission, uses an adeno-associated virus (AAV1) vector to deliver a functional GRN gene encoding for progranulin (PGRN).
The treatment is designed to increase PGRN levels in the central nervous system, potentially altering the course of neurodegenerative diseases. Interim clinical data has shown robust increases in cerebrospinal fluid PGRN concentrations.
Passage Bio's CEO, Will Chou, M.D., commented on the significance of the FDA's support for the trial's amendment, highlighting the potential for PBFT02 to address the unmet needs of the FTD-C9orf72 patient community. The company remains focused on advancing the clinical development of PBFT02.
This news comes as a significant development for Passage Bio as it works to expand its treatment options for neurodegenerative diseases. The company is dedicated to developing one-time therapies targeting the underlying pathology of these conditions. The information in this article is based on a press release statement from Passage Bio.
InvestingPro Insights
As Passage Bio, Inc. (NASDAQ:PASG) makes strides with its gene therapy treatment, PBFT02, for frontotemporal dementia, investors and stakeholders are closely monitoring the company's financial health and market performance. Key metrics from InvestingPro provide a snapshot of the company's current financial status:
- Market Capitalization: Passage Bio's market cap stands at a modest $56.79 million, reflecting its status as a smaller player in the biotech industry.
- Price to Earnings (P/E) Ratio: With a negative P/E ratio of -0.6 and an adjusted P/E ratio for the last twelve months as of Q1 2024 at -0.63, the company is not currently profitable.
- Price to Book (P/B) Ratio: The P/B ratio as of the last twelve months of Q1 2024 is 0.54, which may suggest that the stock is undervalued relative to its book value.
InvestingPro Tips highlight several critical aspects for investors considering Passage Bio:
- The company holds more cash than debt on its balance sheet, which could provide financial flexibility in its operations and development efforts.
- Despite a significant return over the last week, analysts are not optimistic about the company's profitability in the near term. They point out weak gross profit margins and a valuation that implies a poor free cash flow yield.
For those interested in a deeper analysis, there are additional InvestingPro Tips available for Passage Bio, including insights on cash burn rate, liquid assets, and dividend policies. To explore these further, visit InvestingPro and consider using the coupon code PRONEWS24 to get up to 10% off a yearly Pro and a yearly or biyearly Pro+ subscription.
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