By Katherine Hamilton
Passage Bio initiated a strategic review process after regulators didn't support a single-arm trial design for its dementia drug.
The Food and Drug Administration indicated a randomized controlled registrational study design--rather than a trial without a placebo or control group--is required to evaluate PBFT02 for the treatment of frontemporal dementia with granulin gene mutations, Passage Bio said Monday.
The clinical-stage genetic medicines company said that type of trial poses "substantial ethical concerns for patients and their families," along with financial and logistical barriers.
Shares fell 20% to $9.10 in pre-market trading Monday. Through Friday's close, the stock was roughly flat this year.
As a result of the FDA's recommendation, Passage has engaged Wedbush PacGrow as a financial advisor to help review strategic alternatives. These could include a merger, acquisition, reverse merger, sale of assets, strategic partnership, licensing opportunities or other potential paths, it said.
Passage said it doesn't plan to provide updates on the review until the board approves a specific action.
The Philadelphia company also shared data from a continuing phase 1/2 clinical trial for PBFt02, which showed the treatment may slow neurodegeneration in patients with frontotemporal dementia--a type of dementia that affects the frontal and temporal lobes of the brain.
Patients earlier in their disease progression who received the treatment showed reduced rates of whole-brain atrophy and frontotemporal cortex atrophy compared with natural history data from patients at the same stage of disease progression.
Cortex atrophy refers to a degeneration of the brain as part of dementia.
Patients with more advanced disease progression showed no improvements in either atrophy measure versus natural history data
Write to Katherine Hamilton at katherine.hamilton@wsj.com
(END) Dow Jones Newswires
April 20, 2026 07:59 ET (11:59 GMT)
Copyright (c) 2026 Dow Jones & Company, Inc.