PHILADELPHIA - Passage Bio Inc. (NASDAQ:PASG) reported Monday updated data from its Phase 1/2 upliFT-D clinical trial evaluating PBFT02 for frontotemporal dementia with granulin mutations, showing improvements in two disease progression biomarkers compared to natural history data.
According to a press release statement, patients earlier in disease progression with a global Clinical Dementia Rating scale score of 1 who received PBFT02 exhibited a 64% reduction in whole brain atrophy at 12 months compared to untreated patients from the ALLFTD natural history data. PBFT02-treated patients showed 3.1% atrophy versus 8.7% in the natural history sample.
The same patient group experienced a 54% reduction in frontotemporal cortex atrophy, with 4.6% atrophy compared to 9.9% in untreated patients. Patients with more advanced disease showed no improvements on either measure.
PBFT02-treated patients demonstrated stabilization of plasma neurofilament levels at 12 months, with an average reduction of 1.0 pg/mL, while untreated patients showed an average increase of 13.5 pg/mL.
The treatment resulted in durable increases in cerebrospinal fluid progranulin expression through 18 months. The higher dose increased levels from below 3 ng/mL at baseline to a mean of 22.8 ng/mL at 12 months. The lower dose achieved comparable levels at six months.
The company reported PBFT02 was generally well-tolerated with no new treatment-related serious adverse events. Two patients previously experienced asymptomatic serious adverse events including venous sinus thrombosis and hepatotoxicity.The clinical progress comes as Passage Bio navigates challenging financial conditions. With a market capitalization of just $37.4 million, the company holds more cash than debt on its balance sheet, though InvestingPro data shows it is quickly burning through cash with levered free cash flow of negative $31.5 million over the last twelve months. Despite these headwinds, investors have shown enthusiasm, with the stock delivering a 76.8% return over the past year and a 36.7% gain over the last six months.
Passage Bio disclosed that the FDA indicated in a recent Type C meeting that a randomized controlled registrational trial will be required for PBFT02 in FTD-GRN, rather than a single-arm trial design.
The company has initiated a strategic review process and engaged Wedbush PacGrow as financial advisor to evaluate alternatives including potential mergers, acquisitions, or partnerships.According to InvestingPro analysis, the stock currently trades below its Fair Value, suggesting potential upside for investors. Analysts maintain a bullish outlook with price targets ranging from $10 to $68. For investors seeking deeper insights, InvestingPro offers 10 additional exclusive tips on PASG, along with comprehensive financial health scores and advanced metrics to help navigate this volatile biotech stock.
In other recent news, Passage Bio Inc. has caught the attention of analysts with two notable firms initiating coverage on the company. Both Baird and Oppenheimer have given Passage Bio an outperform rating, setting a price target of $30.00. These developments come amid the company's stock experiencing a 39% decline year-to-date. Analysts from both firms suggest that this decline may be an overreaction to setbacks experienced by competitors in the progranulin class, such as PR006 and latozinemab. Baird and Oppenheimer's ratings reflect a positive outlook on Passage Bio's potential despite the broader challenges in the biotech sector. These recent assessments provide investors with renewed perspectives on the company's future prospects.