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Passage Bio Receives FDA Clearance of IND Application for PBML04 for Treatment of Metachromatic Leukodystrophy

June 8, 2022

• 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.

About Passage Bio
Passage Bio (Nasdaq: PASG) is a clinical-stage genetic medicines company on a mission to provide life-transforming therapies for patients with CNS diseases with limited or no approved treatment options. Our portfolio spans pediatric and adult CNS indications, and we are currently advancing three clinical programs in GM1 gangliosidosis, Krabbe disease and frontotemporal dementia with several additional programs in preclinical development. 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. Through this collaboration, we have 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. 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 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.

For further information, please contact:

Passage Bio Investors:
Stuart Henderson
Passage Bio

Passage Bio Media:
Mike Beyer
Sam Brown Inc. Healthcare Communications

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