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PHILADELPHIA - Passage Bio, Inc. (NASDAQ:PASG), a clinical-stage genetic medicines company with a market capitalization of $24.3 million, reported updated interim data from its Phase 1/2 upliFT-D clinical trial evaluating PBFT02, a one-time gene therapy for frontotemporal dementia (FTD) with granulin mutations. According to InvestingPro analysis, the company appears undervalued based on its Fair Value metrics, though investors should note its volatile stock performance, with shares down over 57% in the past year.
The company’s data showed that patients receiving the higher dose (Dose 1) of PBFT02 maintained elevated cerebrospinal fluid (CSF) progranulin levels through 18 months post-treatment. The first patient treated with the lower dose (Dose 2, 50% of Dose 1) showed substantial increases in CSF progranulin at one month. While advancing its clinical programs, InvestingPro data reveals the company maintains a healthy current ratio of 3.74, with more cash than debt on its balance sheet, though it’s currently experiencing rapid cash burn.
Patients treated with Dose 1 demonstrated a 4% average increase in plasma neurofilament light chain (NfL) levels at 12 months post-treatment, compared to expected increases of 28-29% per year in untreated patients according to natural history data.
On the safety front, three of eight patients experienced serious adverse events. The company plans to amend the trial protocol to include prophylactic low-dose anticoagulation and modify inclusion criteria to study patients earlier in disease progression.
"These data continue to demonstrate the ability of our investigational, one-time gene therapy to elevate progranulin, the deficient protein in FTD-GRN, in a robust and durable manner," said Will Chou, M.D., president and CEO of Passage Bio.
The company intends to submit the amended protocol to health authorities in July 2025 and begin enrollment in additional cohorts following approval. Passage Bio remains on track to seek regulatory feedback on a registrational trial design for FTD-GRN in the first half of 2026. Analyst expectations remain mixed, with price targets ranging from $2 to $13 per share. For deeper insights into Passage Bio’s financial health and growth prospects, including 8 additional exclusive ProTips, visit InvestingPro.
The upliFT-D trial is evaluating PBFT02 administered by single injection into the cisterna magna in patients aged 35 to 75 years with FTD-GRN or FTD-C9orf72.
This information is based on a company press release statement.
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