Passage BIO(US:PASG)2020-11-10 18:48Financial Data and Key Metrics Changes - The company ended the quarter with cash, cash equivalents, and marketable securities of approximately $336 million, compared to $159 million as of December 31, 2019, indicating strong financial health [36] - R&D expenses were $20.8 million for the quarter ended September 30, compared to $10.4 million for the same quarter in 2019, primarily due to increased clinical manufacturing and development costs [37] - G&A expenses increased to $7.8 million for the quarter ended September 30, compared to $1.2 million for the same quarter in 2019, driven by personnel-related costs and professional fees [39] - The net loss was $28.5 million for the third quarter of 2020, compared to $11.4 million in the same quarter of 2019, with a net loss per share of $0.63 compared to $2.02 in the prior year [40] Business Line Data and Key Metrics Changes - The company is focused on three lead programs: PBGM01 for GM1 gangliosidosis, PBFT02 for frontotemporal dementia, and PBKR03 for Krabbe disease, with plans to initiate clinical trials in early 2021 [10][25] - PBGM01 has received orphan drug and rare pediatric disease designations in the U.S. and orphan disease designation in Europe, highlighting its potential in addressing unmet medical needs [19][31] Market Data and Key Metrics Changes - The incidence of GM1 is predicted to be about 0.5 in 100,000 live births, translating to approximately 50 patients potentially diagnosed per year [58] - The company is actively engaging with patient advocacy groups and initiatives like the New York ScreenPlus pilot program to enhance patient identification and support for GM1 and Krabbe disease [22][54] Company Strategy and Development Direction - The company aims to provide life-transforming gene therapies for patients with rare, monogenic CNS diseases, leveraging partnerships with leading research institutions [8][9] - The completion of a dedicated GMP manufacturing suite is a strategic milestone to secure control of the supply chain and support clinical and commercial success [33] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in addressing FDA concerns regarding biocompatibility and anticipates initiating the Phase 1/2 trial for PBGM01 in the first quarter of 2021 [14][44] - The company is preparing for clinical trial initiations for FTD and Krabbe programs in the first half of 2021, with ongoing efforts to expand clinical manufacturing and operations teams [45] Other Important Information - The company has a robust pipeline with a total of 17 programs focused on rare, monogenic disorders of the CNS, supported by a strong financial position [11] - Recent preclinical data supports the potential of PBGM01 as a treatment for GM1, showing improvements in neurological function and increased survival in animal models [23] Q&A Session Summary Question: Plans for Invitae collaboration regarding Krabbe disease - Management indicated that while Krabbe is not currently included in the New York ScreenPlus, there are ongoing pilot programs in multiple states for Krabbe screening [54] Question: Expected patient identification from Invitae partnership - The company estimates that the incidence of GM1 is around 50 patients per year, but recognizes that underdiagnosis may increase this number through enhanced awareness and testing initiatives [58][59] Question: Impact of FDA discussions on filing plans for other diseases - Management confirmed that IND submissions for FTD and Krabbe are planned for early 2021, following the resolution of the clinical hold for GM1 [63]