Passage Bio Advances PBFT02 Gene Therapy Program Amidst Positive Q3 2024 Results

Passage Bio

PHILADELPHIA, PAPassage Bio, Inc. (Nasdaq: PASG) this week reported its third quarter financial results for 2024, highlighting substantial progress in its PBFT02 gene therapy program for frontotemporal dementia with progranulin mutations (FTD-GRN). The company continues to make significant strides in its clinical trials and strategic initiatives.

CEO Will Chou expressed optimism about the advancements, stating, “This quarter, we’ve made important strides in advancing our PBFT02 gene therapy program. Execution of our ongoing upliFT-D trial for FTD-GRN remains our core priority, and we are pleased to share that 4 patients in Cohort 2 have been enrolled, with patient dosing progressing as planned.”

The upliFT-D trial, which is in its global Phase 1/2 stage, has shown promising results. Updated interim data from Cohort 1 indicated that PBFT02 was generally well-tolerated and successfully elevated cerebrospinal fluid (CSF) progranulin levels for up to 12 months after treatment. The trial’s progress was underscored by presentations at key scientific conferences, showcasing the potential of PBFT02 to emerge as a leading progranulin-raising therapy.

In addition to clinical advancements, Passage Bio has strengthened its leadership with the appointment of Tom Kassberg to its board of directors. Kassberg brings a wealth of experience in corporate development, enhancing the company’s strategic capabilities.

Financially, Passage Bio reported cash, cash equivalents, and marketable securities totaling $84.8 million as of September 30, 2024, ensuring a cash runway to the end of the second quarter of 2026. The company also noted a reduction in research and development expenses to $8.7 million from $15.1 million the previous year, reflecting efficient resource management.

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Looking ahead, Passage Bio plans to report 12-month Cohort 1 and interim Cohort 2 data in the first half of 2025, while seeking regulatory feedback on pivotal trial design later that year. These efforts, coupled with a strong financial position, strategically position the company to achieve several key milestones in the coming quarters.

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