Biopharmaceutical company Passage Bio highlighted promising progress in its phase 1/2 clinical trial for a form of genetic FTD in a recent quarterly update.

Passage Bio’s upliFT-D trial is evaluating PBFT02, an experimental gene therapy for FTD caused by mutated variants of the GRN gene that can create an inability to produce the protein progranulin, which plays a crucial role in various biological processes. PBFT02 uses an engineered virus to deliver a healthy copy of GRN. In late 2023, the company announced promising preliminary results from the trial.

“The beginning of 2024 has been marked by strong execution as we continue to advance our global upliFT-D clinical trial in FTD-GRN and plan to deliver meaningful data over the next twelve months,” said Passage Bio President and CEO Will Chou, MD. “We are excited to share updated interim data demonstrating continued elevation in [cerebrospinal fluid] progranulin levels at six months after treatment in two patients. This consistent progranulin response underscores the differentiated profile of PBFT02 and gives us confidence in the impact our therapy may have on patients at the current dose. Momentum in our program has been strong since we shared encouraging initial data last year, and we are pleased to have completed dosing of all five patients in Cohort 1.”

In addition to dosing all participants in the first cohort, Passage Bio announced that it plans to begin dosing a second group later in the first half of 2024. Passage Bio noted that multiple people with a GRN mutation are being evaluated for study eligibility at trial sites in Brazil, Canada, the United States, and Europe. Additional safety and biomarker data from the first cohort is expected in the latter half of 2024, while 12-month follow-up data is expected in the first half of 2025, along with initial biomarker and safety data from the second cohort.

Passage Bio also announced that it had begun a process with the U.S. Food and Drug Administration to receive feedback on its plans to treat FTD caused by C9orf72 genetic mutations. Similarly to FTD-GRN, FTD-C9orf72 is associated with abnormal accumulations of the protein TDP-43, with existing research showing that elevating progranulin levels can reduce TDP-43-related pathology and slow the process of neurodegeneration. The company expects feedback from the FDA later this year.

Approximately 40% of people diagnosed with FTD have a family history of one or more relatives diagnosed with FTD or a similar condition in what is known as “familial FTD.” In a subset of those with familial FTD, a known genetic mutation like GRN or c9orf72 is the cause. Visit FTD and Genetics to learn more.

Are you interested in participating in a trial like upliFT-D? Sign up for the FTD Disorders Registry to keep up to date on research opportunities and to share your experiences with FTD to guide researchers. You can also visit the studies seeking participants page to learn about trial participation opportunities for which you may be eligible.