PHILADELPHIA, May 21, 2020 (GLOBE NEWSWIRE) — Passage Bio, Inc. (NASDAQ: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system disorders, today announced the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation to PBGM01 broadly for the treatment of GM1 gangliosidosis. PBGM01 is an AAV-delivery gene therapy currently being developed for the treatment of infantile GM1 gangliosidosis (GM1) and has previously been granted Orphan Drug designation.
“This is the second regulatory designation we have received from the FDA for our lead program in GM1 and reflects the high unmet need in this patient population,” said Bruce Goldsmith, Ph.D, president and chief executive officer of Passage Bio. “As a company we are committed to developing therapies that transform the lives of patients suffering from serious life-threatening CNS disorders. We believe that PBGM01 has the potential to restore developmental progression, enabling patients to achieve additional milestones and improve quality of life. We look forward to advancing PBGM01 into clinical testing later this year.”
RPD designation is granted by the FDA to encourage treatments for serious or life-threatening diseases primarily affecting children 18 years of age and younger and fewer than 200,000 people in the United States. Under the RPD program, a sponsor who receives approval for a drug or biologic for a “rare pediatric disease” may qualify for a priority review voucher that may be sold or transferred.
Passage anticipates starting a Phase 1/2 trial for PBGM01 for the treatment infantile GM1 in the fourth quarter of 2020. The trial will be an open-label, dose escalation study of PBGM01 administered by a single injection into the intra cisterna magna, or ICM, in pediatric subjects with infantile GM1.
GM1 gangliosidosis (GM1) is a rare and often life-threatening monogenic recessive lysosomal storage disease caused by mutations in the GLB1 gene, which encodes lysosomal acid beta-galactosidase (β-gal). Reduced β-gal activity results in the accumulation of toxic levels of GM1 ganglioside in neurons throughout the brain, causing rapidly progressing neurodegeneration. GM1 manifests as a continuum of disease and is most severe in the Infantile form, which is characterized by onset in the first 6 months of life with hypotonia (reduced muscle tone), progressive CNS dysfunction, and rapid developmental regression. Life expectancy for infants with GM1 is two to four years, and infantile GM1 represents approximately 62.5% of the incidence of 0.5 to 1 in 100,000 live births. Currently, there are no approved disease-modifying therapies available.
PBGM01 is an AAV-delivery gene therapy currently being developed for the treatment of infantile GM1, in which patients have mutations in the GLB1 gene causing little or no residual β-gal enzyme activity and subsequent neurodegeneration. PBGM01 utilizes a next-generation AAVhu68 capsid administered through intra-cisterna magna (ICM) to deliver a functional GLB1 gene encoding β-gal to the brain and peripheral tissues. By reducing the accumulation of GM1 gangliosides, PBGM01 has the potential to reverse neuronal toxicity, thereby restoring developmental potential. In preclinical models, PBGM01 has demonstrated broad brain distribution and wide uptake of the β-gal enzyme in both the central nervous system (CNS) and critical peripheral organs, suggesting potential treatment for both the CNS and peripheral manifestations of GM1. The Company has received Orphan Drug and Rare Pediatric Disease designation for PBGM01 for patients with GM1 and plans to initiate a Phase 1/2 trial in the fourth quarter of 2020 with initial 30 day safety and biomarker data expected in the late first half of 2021.
About Passage Bio
Passage Bio is a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system disorders with limited or no approved treatment options. The company is based in Philadelphia, PA and has a research, collaboration and license agreement with the University of Pennsylvania and its Gene Therapy Program (GTP). The GTP conducts discovery and IND-enabling preclinical work and Passage Bio conducts all clinical development, regulatory strategy and commercialization activities under the agreement. The company has a development portfolio of six product candidates, with the option to license eleven more, with lead programs in GM1 gangliosidosis, frontotemporal dementia and Krabbe disease.