Passage Bio, Inc. reported updated interim data from the Phase 1/2 upliFT-D clinical trial for PBFT02 in FTD-GRN patients. The data showed continued robust, durable elevation in CSF progranulin (PGRN) levels and improvement in plasma neurofilament light chain (NfL) compared to natural history.
Dose 2, which is 50% lower than Dose 1, substantially increased CSF PGRN levels at 30 days, reaching the upper limit of a healthy adult reference range. This indicates the potential for efficacy at a lower dose.
However, the company plans to amend the upliFT-D protocol to include a short course of low-dose prophylactic anticoagulation and modify inclusion criteria to study patients earlier in disease progression. This decision was supported by study investigators and the Independent Data Monitoring Committee (IDMC) following serious adverse events (SAEs) in three patients, which included venous sinus thrombosis, hepatotoxicity, and pulmonary embolism.
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