Business
Passage Bio Receives MHRA Clinical Trial Authorization for PBGM01 for Treatment of GM1 Gangliosidosis
- MHRA approval represents first clinical trial authorization for the global PBGM01 clinical trial program - - Patient enrollment in UK clinical study site
About this update from Passage Bio, Inc.
[{"type":"text","content":"- MHRA approval represents first clinical trial authorization for the global PBGM01 clinical trial program - - Patient enrollment in UK clinical study site expected to start in the second quarter of 2021 - PHILADELPHIA, Dec. 10, 2020 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (Nasdaq: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system (CNS) disorders, today announced that the United Kingdom’s (UK) Medicines Healthcare Products Regulatory Agency (MHRA) has approved the Clinical Trial Authorization (CTA) for PBGM01, the company’s lead gene therapy candidate being studied for the treatment of GM1 gangliosidosis (GM1). This is the first regulatory authorization for the global PBGM01 clinical trial program, the Imagine-1 study, for the treatment of infantile GM1, a rare and often life-threatening CNS disorder with no approved disease-modifying therapies available. The company expects dosing of our first patient to begin in the global phase 1 / 2 clinical trial program for PBGM01, an adeno-associated virus (AAV)-delivery gene therapy, in the first quarter of 2021. Patient enrollment for the UK clinical study site, which is part of the global program, is expected to start in the second quarter of 2021. “Patients with GM1 urgently need treatment options, and we are pleased to have the support of the MHRA as we continue plans to initiate our global clinical trial program for PBGM01,” said Bruce Goldsmith, Ph.D., president and chief executive officer of Passage Bio. “This CTA approval is the first of several regulatory authorizations we are expecting for our global Imagine-1 study and brings us one step closer to starting studies to treat these children.\" GM1, a rare monogenic lysosomal storage disease, is caused by mutations in the GLB1 gene, which encodes the lysosomal enzyme beta -galactosidase (β-gal). Reduced β-gal activity results in the accumulation of toxic levels of GM1 in neurons throughout the brain, causing rapidly progressive neurodegeneration. GM1 manifests 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 60 percent of the global GM1 incidence of 0.5 to 1 in 100,000 live births. “It is gratifying to see...