Passage Bio Surges Ahead with Promising Gene Therapy Trial for Neurodegenerative Diseases

Passage Bio

PHILADELPHIA, PA — In the competitive landscape of genetic medicine, one company is carving out a substantial niche. Passage Bio, Inc. (Nasdaq: PASG), a clinical-stage genetic medicine company with a focus on neurodegenerative diseases, has recently reported its financial results for the fourth quarter and the year ending December 31, 2023. More importantly, it has taken significant strides in its FTD-GRN program, marking a significant move towards treating a host of neurological diseases.

In 2023, Passage Bio caused a stir in the industry with encouraging data from three patients in Cohort 1 of the FTD-GRN program. The groundbreaking data demonstrated that even at its lowest dosage, PBFT02 can augment CSF progranulin to super-physiological levels. This positions PBFT02 as a potentially best-in-class treatment for FTD-GRN, Frontotemporal Dementia with granulin mutations – a rare form of dementia that affects the front and sides of the brain.

In a statement, Will Chou, M.D., President and CEO of Passage Bio, announced the activation of their first clinical trial site in Europe. This move complements the strong momentum seen in their global upliFT-D study and is set to bolster recruitment efforts. The company is on track to complete dosing for Cohort 1 in the first half of this year and share additional safety and biomarker data in the second half of 2024.

But the company’s ambitions extend further. In light of the promising initial data and evidence supporting progranulin’s role in neurodegeneration, the company is optimistic about expanding the application of PBFT02 beyond FTD-GRN and into areas with significant unmet needs. Indications include FTD-C9orf72, ALS (Amyotrophic Lateral Sclerosis), and Alzheimer’s disease.

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The potential for PBFT02 to treat these more common neurodegenerative diseases marks a major step forward. Passage Bio’s plans for expansion are backed by a robust balance sheet, as the company steers its focus on the exploration of PBFT02 in additional indications alongside potential out licensing options for clinical-stage pediatric programs.

To help drive their financial strategy further, Passage Bio has appointed Kathleen Borthwick as Chief Financial Officer; an industry veteran who previously served as the company’s Senior Vice President, Finance and Interim CFO and brings with her 25 years of pharmaceutical and biotech strategic financial leadership experience.

Financially, Passage Bio remains strong, declaring a cash position of $114.3 million as of December 31, 2023. The company’s R&D expenses were reported at $12.1 million for the quarter and $61.4 million for the year, showing an efficient spend compared to the same time in 2022. The company’s net loss was $16.8 million for the quarter ending on December 31, 2023, and $102.1 million for the year, both figures reflecting better outcomes compared to the previous year.

As Passage Bio braces for a catalyst-rich 2024, industry watchers await with bated breath. The company’s relentless pursuit of transformative genetic medicine is a welcome beacon in the fight against neurodegenerative diseases. As more milestones stand to be achieved, 2024 could be the year Passage Bio establishes itself as a prime contender in genetic medicine.

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