Passage Bio Shares Promising Updates on Gene Therapy Trial

Promising Interim Results from Passage Bio's upliFT-D Study

Passage Bio, Inc. (Nasdaq: PASG), a clinical-stage genetic therapies company focused on advancing treatments for neurodegenerative diseases, recently provided an update on the ongoing Phase 1/2 upliFT-D clinical trial for PBFT02, aiming to treat frontotemporal dementia (FTD) linked to granulin (GRN) mutations. The new data released highlights significant progress and potential for the gene therapy.

Key Findings on CSF and Plasma Levels

The interim results reveal that PBFT02 successfully demonstrated robust and lasting increases in cerebrospinal fluid (CSF) progranulin (PGRN) levels. Specifically, the treatment led to consistent elevation from baseline measurements, while improvements in plasma neurofilament (NfL), a critical marker of disease progression, were also observed.

Details of Progranulin Elevation

After administration of the first dose of PBFT02, patients exhibited a remarkable increase in CSF PGRN levels over 18 months, which were sustained well above the baseline. After just one month post-treatment, the average CSF PGRN concentrations climbed impressively to 12.4 ng/mL, with further escalation noted at six and 12 months.

The second dose, being 50% smaller than the first, also yielded significant results. The initial patient to receive this lower dose experienced a jump in CSF PGRN levels from 1.5 ng/mL at baseline to 7.6 ng/mL just a month later, reaching nearly the upper limit of what is deemed normal for healthy adults.

Impact on Plasma Neurofilament Levels

The results concerning plasma neurofilament levels were equally promising. Patients receiving PBFT02 after the first dose had a notably reduced annual increase in plasma NfL levels. While untreated patients typically saw an increase of around 28% per year, those on the therapy only experienced an average increase of 4% at 12 months post-administration.

Elevated Safety Profile Observed

Regarding the safety profile monitored till mid-2025, mild to moderate adverse events were reported across several patients. Notably, the investigation team has not detected any significant toxicity related to dorsal root ganglion or complications during the intrathecal administration.

Next Steps For upliFT-D Protocol

In alignment with ongoing assessments from investigators and the Independent Data Monitoring Committee (IDMC), Passage Bio plans to amend the protocol governing the upliFT-D study. This includes the implementation of a prophylactic regimen involving low-dose anticoagulation and revisions to the inclusion criteria, allowing earlier intervention for patients at less advanced stages of the disease.

The company is also slated to submit these changes for regulatory review. Once approved, they can enhance the enrollment speed for the upcoming cohorts, with serious efforts in reaching out to patients experiencing prodromal symptoms or mild cognitive impairment.

Looking Ahead: Key Milestones

Attention is now turning towards critical milestones, including planned submissions to health authorities concerning the amended protocol and feedback sessions regarding the processes required for manufacturing compliance. Additionally, interim safety and biomarker data from the next dosing stage will be presented in the first half of the coming year.

The Bigger Picture: About Passage Bio

Passage Bio embodies a focused ambition to alter the trajectory of neurodegenerative diseases by developing one-time gene therapies, specifically for conditions like FTD, through elevating progranulin levels. By unlocking the potential for enhanced lysosomal functions, they aim to improve quality of life for patients affected by such debilitating diseases.

As they pursue innovative channels for trial recruitment—including collaborations for genetic counseling—Passage Bio continues to advocate for the needs of the FTD community. Interested parties can learn more about their efforts and discoveries through their official website: passagebio.com.

Frequently Asked Questions

What is the upliFT-D study?

The upliFT-D study is an ongoing Phase 1/2 clinical trial assessing the efficacy and safety of PBFT02 for treating frontotemporal dementia with granulin mutations.

What are the primary findings from the updated data?

The updated data shows robust and durable increases in CSF PGRN levels and a reduced annual rate of increase in plasma NfL, compared to untreated patients.

What safety measures are in place for participants?

Data monitoring and assessments ensure participant safety, including planned adjustments to the therapeutic protocol based on emerging safety reports.

When will regulatory feedback be sought?

Passage Bio plans to engage with regulatory bodies regarding the registrational trial design in the first half of 2026.

How can patients participate in the upliFT-D study?

Patients interested in participating should consult with their healthcare providers to determine eligibility and explore enrollment opportunities in upcoming cohorts.

About The Author

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