PBML04 represents the company's fourth program to receive IND clearance

PHILADELPHIA, June 08, 2022 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (NASDAQ:PASG), a clinical-stage genetic medicines company focused on developing transformative therapies for central nervous system disorders, today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for PBML04, an adeno-associated virus (AAV)-delivery gene therapy that is being studied for the treatment of Metachromatic Leukodystrophy (MLD). MLD is a rare, fatal, pediatric, lysosomal storage disease that currently has limited available treatment options.

"We are thrilled to have received IND clearance for our program in metachromatic leukodystrophy, marking our fourth IND clearance as a company and our third pediatric lysosomal storage disorder program to reach clinical development," said Edgar (Chip) Cale, interim chief executive officer of Passage Bio. "Clearance of this IND is a testament to the quality preclinical data supporting PBML04 through our partnership with the University of Pennsylvania's Gene Therapy Program and the strong CMC and analytics capabilities we have developed internally. MLD, GM1 gangliosidosis and Krabbe disease are similar diseases each caused by a single gene mutation leading to deficiency of a critical enzyme. Our approach and clinical development plans across these three programs also share similarities, including use of our next-generation, proprietary AAVhu68 capsid and ICM delivery. We are excited by the promise of PBML04 to offer a potentially transformative treatment for this devastating disease."

MLD is a fatal inherited disease that is caused by mutations in the arylsulfatase-A (ARSA) gene which reduces enzyme activity, leading to progressive build-up of toxic sulfatides in the central and peripheral nervous system. We are targeting infantile-onset MLD, which is characterized by progressive muscle weakness, rigidity, gait disorder, developmental delays and is typically fatal by five years of age. The estimated worldwide incidence of MLD is approximately 1 in 100,000 live births.

The Phase 1 clinical trial will utilize intra-cisterna magna (ICM) administration to deliver an AAVhu68 capsid to express ARSA and potentially address both central nervous system and peripheral manifestations of this devastating disease.