PHILADELPHIA, Aug. 04, 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, today reported financial results for the second quarter ended June 30, 2022 and provided recent business highlights.
“We continue to be excited by the data emerging from our Imagine-1 program and the promise of our investigational therapy to potentially offer the first disease-modifying treatment for GM1 gangliosidosis. Our initial data have been met with enthusiasm from the clinical and patient communities, and we are thrilled to have begun recruiting patients for the final cohort in the dose-ascending phase of the trial,” said Edgar (Chip) Cale, interim chief executive officer of Passage Bio. “As we look towards the rest of the year, we remain focused on delivering several important milestones. These include reporting initial safety and biomarker data from Cohorts 2 and 3 in Imagine-1 and sharing initial data from a subset of Cohort 1 in our GALax-C trial for Krabbe disease. We also look forward to dosing the first patient in our upliFT-D trial for frontotemporal dementia. The team at Passage is driven to fulfill our vision of developing ground-breaking therapies that transform the lives of patients with CNS diseases.”
- Advanced Imagine-1 clinical trial to recruitment of patients in final cohort, Cohort 4 (early infantile, high dose), in dose-ascending phase of study: The Independent Data Monitoring Committee reviewed interim safety data from Cohort 3 (early infantile, low dose) and recommended advancement to Cohort 4. Patient recruitment for this cohort is underway.
- Presented encouraging longer-term clinical and biomarker data for patients with GM1 gangliosidosis in Imagine-1 study at the American Society of Gene and Cell Therapy (ASGCT) 25th Annual Meeting: In May 2022, the company presented new data showing continued and meaningful developmental improvement, as assessed by study investigators and caregivers. For the two patients in Cohort 1, safety data at 13 and seven months showed that a low dose of PBGM01 was well tolerated and had a favorable safety profile. Additionally, longer-term biomarker data for beta-galactosidase enzyme activity in cerebrospinal fluid (CSF) and serum showed functional transgene expression.
- Received clearance of Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA) for a Phase 1 clinical program for PBML04: In May 2022, the FDA cleared the company’s IND application for PBML04 in metachromatic leukodystrophy (MLD), a rare, pediatric, lysosomal storage disorder. PBML04 utilizes the same next-generation proprietary capsid as PBGM01 and PBKR03 to deliver, through intra-cisterna magna (ICM) administration, a functional ARSA gene into the CSF. PBML04 represents the company’s fourth program to receive IND clearance.
- Expanded board of directors with appointment of Michael Kamarck, Ph.D.: In July 2022, the company announced the appointment of Michael Kamarck, Ph.D., to its board of directors and his appointment to the Nominating and Corporate Governance Committee. Dr. Kamarck is a seasoned biopharmaceutical executive with over forty years of experience in discovery research, process development and technical operations.
Anticipated Upcoming Milestones
- Present interim safety and biomarker data from Cohorts 2 (late infantile, high dose) and 3 (early infantile, low dose) for Imagine-1 clinical trial for GM1 in the second half of 2022.
- Present interim safety and biomarker data from subset of Cohort 1 for GALax-C clinical trial for Krabbe disease by year-end 2022.
- Dose first patient in Phase 1/2 study with PBFT02 for FTD-GRN in mid-2022.
Second Quarter 2022 Financial Results
- Cash Position: Cash, cash equivalents and marketable securities were $239.3 million as of June 30, 2022, compared to $267.1 million as of March 31, 2022. The Company expects current cash and cash equivalents to fund operations into the second quarter of 2024.
- Research and Development (R&D) Expenses: R&D expenses were $26.8 million for the second quarter ended June 30, 2022, compared to $33.1 million for the same quarter in 2021.
- General and Administrative (G&A) Expenses: G&A expenses were $13.0 million for the second quarter ended June 30, 2022, compared to $15.4 million for the same quarter in 2021.
- Net Loss: Net loss was $39.5 million, or a net loss of $0.73 per basic and diluted share, for the second quarter ended June 30, 2022, compared to $48.4 million, or a net loss of $0.90 per basic and diluted share, for the same quarter in 2021.
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 www.passagebio.com.