Passage Bio Receives FDA Clearance of IND Application for PBML04 for the Treatment of Metachromatic … | Your money


• 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 drug 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 application (IND) for PBML04, an adeno-associated virus (AAV) gene therapy that is being investigated for the treatment of leukodystrophy metachromatic (MLD). MLD is a rare, fatal, pediatric lysosomal storage disease for which available treatment options are currently limited.

“We are delighted to have received IND clearance for our metachromatic leukodystrophy program, marking our fourth IND clearance as a company and our third pediatric lysosomal storage disorder program to reach clinical development,” said Edgar (Chip) Cale, Acting General Manager of Passage Bio. “The clearance of this IND is a testament to the quality of the preclinical data supporting PBML04 through our partnership with the University of Pennsylvania Gene Therapy Program and the strong CMC and analytical capabilities we have developed in-house. MLD, GM1 gangliosidosis and Krabbe disease are similar diseases each caused by a single genetic mutation resulting in a deficiency of a critical enzyme. Our approach and clinical development plans in these three programs also share similarities, including the use of our proprietary next-generation 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 caused by mutations in the arylsulfatase-A (ARSA) gene which reduces enzyme activity, resulting in progressive accumulation of toxic sulfatides in the central and peripheral nervous system. We are targeting childhood-onset MLD, which is characterized by progressive muscle weakness, stiffness, gait disturbance, developmental delays and is usually fatal by age five. The estimated worldwide incidence of MLD is approximately 1 in 100,000 live births.

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

About Passage Bio Passage Bio (Nasdaq: PASG) is a clinical-stage genetic drugs company whose mission is to provide life-transforming therapies for patients with CNS diseases with limited or no 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, Pennsylvania, our company has established a strategic collaboration and licensing agreement with the renowned University of Pennsylvania Gene Therapy Program to conduct our discoveries and preclinical work enabling IND. Through this collaboration, we have enhanced access to a broad portfolio of gene therapy candidates and future gene therapy innovations which we then combine with our deep clinical, regulatory, manufacturing and commercial expertise to rapidly advance our strong portfolio of optimized gene therapies. As we work with speed and tenacity, we are always attentive to patients who could benefit from our therapies. More information is available at

Forward-Looking Statements This press release contains “forward-looking statements” within the meaning of and pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to: our expectations regarding the timing and achievement of planned milestones, including the initiation of clinical trials and the availability of clinical data from such trials; our expectations regarding the ability of our employees and partners to execute key initiatives; our expectations regarding manufacturing plans and strategies; our expectations regarding the cash trail; and the ability of our lead product candidates to treat their respective target single gene CNS disorders. These forward-looking statements may be accompanied by words such as “aim”, “anticipate”, “believe”, “could”, “estimate”, “expect”, “anticipate”, “target”, “intend of”, “could”, “could”, “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 these 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 additional studies, or may not approve or 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 that the success of early-stage clinical trials may not be predictive of later-stage clinical trial results; failure to protect and enforce our intellectual property and other proprietary rights; our reliance on collaborators and other third parties for the development and manufacture of product candidates and other aspects of our business, which are beyond our complete control; risks associated with actual and potential delays, work stoppages or supply chain disruptions caused by the coronavirus pandemic; and other risks and uncertainties described in the Risk Factors section of the company’s filings from time to time with the Securities and Exchange Commission (SEC) and other reports filed with the SEC. Passage Bio assumes no obligation to publicly update any forward-looking statement, written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise.

For more information, please contact:

Passage Bio Investors: Stuart Henderson Passage Bio 267.866.0114 [email protected]

Passage Bio Media: Mike Beyer Sam Brown Inc. Healthcare Communications 312-961-2502 [email protected]

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