Passage Bio Q2 2024 Financial Results and Business Highlights

Passage Bio, Inc., a clinical-stage genetic medicines company, recently announced significant developments and financial results for the second quarter ending June 30, 2024. The company, focused on developing therapies for neurodegenerative diseases, has made meaningful strides in its upliFT-D trial and expanded its program portfolio.

A pivotal achievement for Passage Bio was the enrollment of the first patient in Cohort 2 in their upliFT-D trial for frontotemporal dementia patients with GRN mutations (FTD-GRN). This followed a review by the Independent Data Monitoring Committee (IDMC), which supported the continuation to Cohort 2, reinforcing the safety profile of PBFT02 when combined with a revised immunosuppression regimen. The company plans to present updated safety and biomarker data from Cohort 1 at the 14th International Conference on Frontotemporal Dementias (ISFTD2024) in Amsterdam.

In another major development, Passage Bio reached an agreement with the FDA to expand the upliFT-D trial to include patients with C9orf72 gene mutations associated with frontotemporal dementia (FTD-C9orf72). This decision was backed by robust progranulin expression data and available safety data from the first cohort of FTD-GRN patients treated with PBFT02. The company aims to initiate dosing for FTD-C9orf72 patients in the first half of 2025.

Additionally, Passage Bio completed the out-licensing of its pediatric lysosomal storage disease programs to GEMMA Biotherapeutics. This includes the transfer of rights to PBGM01, PBKR03, and PBML04, aimed at treating GM1 gangliosidosis, Krabbe disease, and metachromatic leukodystrophy, respectively. Under the terms of this agreement, Passage Bio will receive initial and milestone payments, with potential additional payments totaling up to $114 million, as well as future royalties.

Financially, Passage Bio reported a solid balance sheet, with cash, cash equivalents, and marketable securities totaling $91.8 million as of June 30, 2024. This is expected to fund operations until the end of the second quarter of 2026. Despite a net loss of $16.0 million for the quarter, the company's financial health is supported by the initial payments from the GEMMA Biotherapeutics out-licensing agreements.

Research and development expenses for the quarter were $10.4 million, a decrease from $17.3 million in the same period last year, reflecting a strategic focus on key programs. General and administrative expenses also saw a reduction, coming in at $6.5 million compared to $8.1 million in the previous year.

Will Chou, M.D., President and CEO of Passage Bio, emphasized the company's strategic clarity and momentum, particularly in expanding PBFT02 into additional adult neurodegenerative indications. He highlighted the positive recommendations from the IDMC, the alignment with the FDA, and the out-licensing agreement with GEMMA Biotherapeutics as key drivers of the company's progress.

Looking ahead, Passage Bio has several anticipated milestones. These include presenting updated data from Cohort 1 patients at ISFTD2024, reporting 12-month Cohort 1 and interim Cohort 2 data in the first half of 2025, and seeking regulatory feedback on the pivotal trial design in the second half of 2025. The company also plans to obtain regulatory feedback on the pathway to treating ALS patients with PBFT02 and initiate dosing for FTD-C9orf72 patients in the first half of 2025.

Passage Bio remains committed to its mission of developing one-time therapies for neurodegenerative diseases, aiming to restore lysosomal function and slow disease progression. With a strong strategic direction and financial stability, the company is poised to make significant advancements in its clinical programs and continue its innovative work in genetic medicines.