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Passage Bio, Inc. (NASDAQ: PASG) is a pioneering genetic medicines company dedicated to the development of transformative therapies for rare monogenic central nervous system (CNS) diseases. Leveraging cutting-edge gene therapy techniques, Passage Bio is advancing a robust pipeline aimed at addressing unmet medical needs in both pediatric and adult CNS disorders.
The company's key initiatives include the development of PBGM01 for infantile GM1 gangliosidosis, PBFT02 for frontotemporal dementia (FTD) caused by progranulin deficiency, and PBKR03 for infantile Krabbe disease. These therapies utilize proprietary adeno-associated virus (AAV) capsids to deliver functional genes directly to the brain and peripheral tissues, promising significant therapeutic benefits.
Passage Bio collaborates with leading institutions to bolster its research and development efforts. This includes a strategic research partnership with the University of Pennsylvania's Gene Therapy Program and a collaboration agreement with Catalent Maryland, Inc. Such alliances enhance Passage Bio's capacity to innovate and expedite the clinical development of its promising therapies.
Financially, Passage Bio is in a strong position, with a cash runway extending into the fourth quarter of 2025, allowing the company to diligently progress its clinical programs. Recent achievements include positive interim data from the Imagine-1 study, a Phase 1/2 clinical trial of PBGM01, demonstrating a favorable safety profile and initial evidence of efficacy in early and late infantile GM1 gangliosidosis.
Additionally, the company is making strides with PBFT02. Initial data from the upliFT-D clinical trial indicate promising safety and biomarker outcomes, showing elevated cerebrospinal fluid (CSF) progranulin levels. Such results underscore the potential of PBFT02 to address the underlying pathology of FTD and other neurodegenerative conditions.
Passage Bio is also committed to fostering the next generation of scientists through initiatives like the Tachi Yamada Scholarship program, which supports students in life sciences disciplines.
Passage Bio (NASDAQ: PASG) has received positive feedback from the FDA on expanding its upliFT-D trial of PBFT02 to include FTD-C9orf72 patients. The company plans to amend the ongoing Phase 1/2 global study protocol to introduce this new patient population, with dosing expected to begin in 1H 2025. This expansion is supported by compelling preclinical evidence and safety data from the first cohort of FTD-GRN patients treated with PBFT02.
FTD-C9orf72 affects approximately 21,000 individuals in the US and Europe. The FDA has granted PBFT02 Fast Track and Orphan Drug designations, while the European Commission has also given it Orphan designation. This development could bring new hope to an underserved patient community with clinical trial options.
Passage Bio (NASDAQ: PASG), a clinical-stage genetic medicines company, will present at the Goldman Sachs 45th Annual Global Healthcare Conference. President and CEO William Chou, M.D., will deliver the presentation on June 12, 2024, at 4:00 p.m. ET. The live webcast and a 30-day replay will be accessible on Passage Bio's website.
Passage Bio (Nasdaq: PASG), a clinical stage genetic medicines company, announced Carrie Gordon, a sophomore at Drexel University, as the third recipient of its Tachi Yamada Scholarship. This award honors Dr. Tachi Yamada, the co-founder and former Chairman, and supports life sciences students in Pennsylvania. Carrie, pursuing a degree in Biological Science with a minor in Neuroscience, has a passion for neurology and aims to provide healthcare to underserved communities. The scholarship provides financial assistance and a mentor from Passage Bio for professional growth.
Passage Bio (NASDAQ: PASG) reported its Q1 2024 financial results and provided business updates, including advances in its global upliFT-D clinical trial for FTD-GRN. The trial's Dose 1 PBFT02 consistently elevated CSF progranulin at six months in two patients. All five patients in Cohort 1 have been dosed, with safety and biomarker data expected in 2H 2024. Cohort 2 dosing is planned to start by the end of 1H 2024. The company initiated discussions with the FDA for using PBFT02 to treat FTD-C9orf72, with feedback expected in 2H 2024. Financially, the company ended Q1 with $104.5 million in cash, projecting a cash runway into Q4 2025. Net loss decreased to $16.7 million from $34.3 million year-over-year.
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