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Passage Bio Doses First Patient in Global Clinical Trial for Infantile Krabbe Disease, A Rare Fatal Pediatric Condition

March 10, 2022
Company expects to report interim safety and biomarker data for Cohort 1 patients in the global phase 1/ 2 clinical trial, GALax-C, by end of 2022

PHILADELPHIA, March 10, 2022 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (Nasdaq: PASG), a clinical-stage genetic medicines company focused on developing transformative therapies for central nervous system (CNS) disorders, announced that the first patient with early infantile Krabbe disease has received PBKR03, an adeno-associated virus (AAV)-delivery gene therapy, in its global Phase 1/2 clinical trial, GALax-C.

“It is gratifying to dose the first patient in our GALax-C trial,” said Bruce Goldsmith, Ph.D., president and chief executive officer of Passage Bio. “We now have two global clinical trials with patients dosed under way. The advancement of these two clinical trials represents significant progress for our company in our mission to develop transformative medicines for people with devastating CNS disorders. We look forward to reporting initial safety and biomarker data from GALax-C by end of 2022.”

Krabbe disease is a rare pediatric lysosomal storage disorder caused by mutations in the GALC gene, which encodes galactosylceramidase, an enzyme that breaks down galactosylceramide and psychosine. Without adequate levels of galactosylceramidase, psychosine accumulates, causing widespread death of myelin-producing cells and progressive damage to nerves in both the brain and peripheral tissues of affected children. This is characterized in children by loss of acquired milestones, staring episodes, apnea, peripheral neuropathy, severe weakness, unresponsiveness to stimuli, seizures, blindness, and deafness. Life expectancy for early infantile Krabbe disease, the most severe form, is two years.

Eliseo Salinas, M.D., MSc., chief research and development officer at Passage Bio, noted: “For GALax-C, we are using a proprietary AAVhu68 capsid, the same vector that we are using in our GM1 gangliosidosis clinical trial. We were pleased to recently report interim clinical data from the first two patients in the GM1 trial, showing meaningful clinical improvements and a positive safety profile for the therapy. We are hopeful about the benefits PBKR03 may have for patients with Krabbe disease. We also are grateful to the children, families, and clinical trial investigators who have chosen to participate in our studies.”

Roberto Giugliani, M.D., Ph.D., Department of Genetics UFRGS and Medical Genetics Service HCPA, Porto Alegre, Brazil, who is an expert in lysosomal storage disorders and a clinical trial investigator for Passage Bio’s Imagine-1 and GALax-C clinical studies, said: “It is encouraging to see this clinical trial program advancing, especially considering the urgent medical need for this patient population. Currently there are no approved disease-modifying treatment options for children with Krabbe disease, which has devastating impact on patients as well as their families.”

The U.S. Food and Drug Administration (FDA) has granted PBKR03 Fast Track, Orphan Drug, and Rare Pediatric Disease designations. PBKR03 has also received an Orphan designation from the European Commission.

About GALax-C (NCT04771416)

GALax-C is a Phase 1/2 global, open-label, dose-escalation study of PBKR03 administered by single injection into the cisterna magna in pediatric subjects with early infantile Krabbe disease who are >1 to 9 months of age at enrollment.

The GALax-C study will investigate low and high dose PBKR03 in two age groups, beginning with a low dose cohort of children aged 4 to 9 months. Enrollment is currently ongoing for Cohort 1 at sites globally. The primary endpoint of the study is to evaluate the safety and tolerability of PBKR03. Secondary endpoints include disease biomarkers, and clinical outcome measures. GALax-C is a two-year study with a three-year safety extension.

Passage Bio is undertaking a number of initiatives to identify eligible patients, such as screening efforts to support early and accurate identification of children with Krabbe disease, as well as no-charge genetic testing for patients suspected of having Krabbe disease through collaboration with Invitae.

More information about GALax-C can be found at

About PBKR03

PBKR03 utilizes a next-generation proprietary AAV capsid to deliver, through intra-cisterna magna (ICM) administration, a functional GALC gene to patients with mutations in the gene that codes for galactosylceramidase (GAL-C). The gene therapy has the potential to treat both the central nervous system and peripheral nerve manifestations observed in patients with Krabbe disease.

PBKR03 is supported by extensive preclinical studies conducted by Passage Bio’s collaborator, the University of Pennsylvania’s Gene Therapy Program. The studies showed meaningful transduction of both the central and peripheral nervous system in animal models, with restoration of myelination in the brain and peripheral nerves. In a naturally occurring Krabbe animal model, a single ICM injection of an AAVhu68 capsid containing the normal canine GALC gene showed normalization of GALC activity, reduction of cerebral spinal fluid psychosine levels, normalization of peripheral nerve conduction velocity, improvement in brain myelination, reduction in brain inflammation and increased survival.

About Passage Bio

At Passage Bio (Nasdaq: PASG), we are on a mission to provide life-transforming genetic medicines for patients with CNS diseases that replace their suffering with boundless possibility, all while building lasting relationships with the communities we serve. Based in Philadelphia, PA, our company has established a strategic collaboration and licensing agreement with the renowned University of Pennsylvania’s Gene Therapy Program to conduct our discovery and IND-enabling preclinical work. This provides our team with enhanced access to a broad portfolio of gene therapy candidates and future gene therapy innovations that we then pair with our deep clinical, regulatory, manufacturing and commercial expertise to rapidly advance our robust pipeline of optimized gene therapies into clinical testing. As we work with speed and tenacity, we are always mindful of patients who may be able to benefit from our therapies. More information is available at

Forward-Looking Statements

This press release contains “forward-looking statements” within the meaning of, and made pursuant to the safe harbor provisions of, the Private Securities Litigation Reform Act of 1995, including, but not limited to: our expectations about timing and execution of anticipated milestones, including subsequent events in our GALax-C study; initiation of clinical trials and the availability of clinical data from such trials; our expectations about our collaborators’ and partners’ ability to execute key initiatives; our expectations about manufacturing plans and strategies; our expectations about cash runway; and the ability of our lead product candidates to treat their respective target monogenic CNS disorders. These forward-looking statements may be accompanied by such words as “aim,” “anticipate,” “believe,” “could,” “estimate,” “expect,” “forecast,” “goal,” “intend,” “may,” “might,” “plan,” “potential,” “possible,” “will,” “would,” and other words and terms of similar meaning. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including: our ability to develop and obtain regulatory approval for our product candidates; the timing and results of preclinical studies and clinical trials; risks associated with clinical trials, including our ability to adequately manage clinical activities, unexpected concerns that may arise from additional data or analysis obtained during clinical trials, regulatory authorities may require additional information or further studies, or may fail to approve or may delay approval of our drug candidates; the occurrence of adverse safety events; the risk that positive results in a preclinical study or clinical trial may not be replicated in subsequent trials or success in early stage clinical trials may not be predictive of results in later stage clinical trials; failure to protect and enforce our intellectual property, and other proprietary rights; our dependence on collaborators and other third parties for the development and manufacture of product candidates and other aspects of our business, which are outside of our full control; risks associated with current and potential delays, work stoppages, or supply chain disruptions caused by the coronavirus pandemic; and the other risks and uncertainties that are described in the Risk Factors section in documents the company files from time to time with the Securities and Exchange Commission (SEC), and other reports as filed with the SEC. Passage Bio undertakes no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise.

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