PHILADELPHIA—Passage Bio, Inc., a company focused on rare central nervous system (CNS) disorders, has reported that the FDA has granted Fast Track designation to the company’s three lead investigational gene therapies. These therapies are PBGM01, for the treatment of GM1 gangliosidosis (GM1); PBFT02, for treating frontotemporal dementia with granulin mutations (FTD-GRN); and PBKR03, for the treatment of Krabbe disease. Passage Bio aims to initiate clinical trials for all three therapies in the first half of 2021.
“At Passage Bio, we are working to address some of the world’s rarest and most devastating neurological diseases that affect infants and adults,” stated Dr. Bruce Goldsmith, president and CEO of Passage Bio. “The FDA’s decision to grant Fast Track designation to each of our lead gene therapy candidates highlights the urgent need for new treatments in these diseases, and represents an important step towards achieving our objective of getting potentially transformative therapies to patients as quickly as possible.”
GM1 gangliosidosis progressively destroys neurons in the brain and spinal cord. With PBGM01, Passage Bio is targeting the infantile form of the disease, which has no current treatment options. The therapy has also received Orphan Drug and Rare Pediatric Disease designations from the FDA, and Orphan Drug designation from the European Medicines Agency (EMA). Passage Bio has activated its first US clinical trial site for the global Imagine-1 study evaluating PBGM01, and is currently recruiting patients. The company says that it will dose the first patient in the trial in the first quarter of 2021, and report initial 30-day safety and biomarker data in mid-2021.
Passage Bio’s PBFT02 is intended for the treatment of patients with frontotemporal dementia with granulin mutations, a form of early-onset dementia. In addition to the new Fast Track designation, the FDA previously granted PBFT02 Orphan Drug designation for treatment of frontotemporal dementia. The PBFT02 clinical program is scheduled to begin in the first half of 2021, with initial data readouts planned in late 2021 or early 2022.
Passage Bio plans to study PBKR03 as a monotherapy for the treatment of early infantile Krabbe disease, a disorder involving nerve cell demyelination. The therapy was previously granted both Orphan Drug and Rare Pediatric Disease designations for PBKR03 by the FDA. The Committee for Orphan Medicinal Products at the EMA recently issued a positive opinion for an Orphan medicinal product designation for PBKR03, and final endorsement by the European Commission is expected later in the first quarter. Passage Bio will begin a Phase 1/2 trial for PBKR03 in the first half of 2021, with plans to report initial 30-day safety and biomarker data in late 2021 or early 2022.
“In the last year, we have laid the groundwork for Passage Bio to boldly execute on our strategy. These regulatory designations are intended to accelerate the timelines in our efforts to make a meaningful difference in patients’ lives,” Goldsmith added.