Passage BIO(US:PASG)2020-08-13 18:17Financial Data and Key Metrics Changes - The company ended the quarter with cash and cash equivalents of approximately $353 million, compared to $159 million as of December 31, 2019, indicating a strong cash position expected to fund operations into 2023 [38] - R&D expenses increased to $19.9 million for the quarter ended June 30, compared to $6.3 million for the same quarter in 2019, primarily due to increased clinical manufacturing costs and preparation for clinical trials [39] - G&A expenses rose to $7.4 million for the quarter ended June 30, compared to $1 million for the same quarter in 2019, driven by increased personnel-related costs and expanded operations [40] - The net loss for the second quarter of 2020 was $27.2 million, compared to $13.4 million in the same quarter of 2019, with a net loss per share of $0.60 compared to $3.19 in the prior year [42] Business Line Data and Key Metrics Changes - The company is advancing its lead program PBGM01 for the treatment of infantile GM1 gangliosidosis, with an IND submission to the FDA in June 2020, although it is currently on clinical hold due to device biocompatibility concerns [12][14] - The company is preparing for clinical trials for its other programs, including PBFT02 for frontotemporal dementia and PBKR03 for infantile Krabbe disease, with plans to initiate trials in the first half of 2021 [28][35] Market Data and Key Metrics Changes - The company is focused on rare, monogenetic CNS disorders, which represent a significant unmet medical need, and is working to establish a global clinical supply chain for its gene therapy programs [25][27] Company Strategy and Development Direction - The company aims to build a premier gene therapy company focused on rare CNS disorders and is expanding its internal team to enhance clinical and manufacturing capabilities [8][10] - The strategy includes securing a dedicated manufacturing suite to control the supply chain and support clinical and commercial success [26] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in addressing FDA concerns regarding the biocompatibility of the delivery device and anticipates enrolling the first patient in the Phase 1/2 trial of PBGM01 by late 2020 or early 2021 [17][46] - The company remains on track to report initial biomarker and safety data in the first half of 2021 and is excited about the potential of its pipeline candidates [47] Other Important Information - The company has secured clinical stage manufacturing capacity and is working with external experts to evaluate options for additional risk assessment and testing related to the FDA's clinical hold [16][25] Q&A Session Summary Question: Clarification on the proposed ICM device components and FDA discussions - Management clarified that the components of the proposed ICM device are similar to those used in other clinical studies, and the current discussions with the FDA are specific to the GM1 IND filing [51][53] Question: Competitive dynamics of the FTD program - Management highlighted differentiation in their FTD program through the use of AAV1, which can significantly increase progranulin levels compared to other approaches, potentially stabilizing the disease [58][61] Question: Immunosuppression plans for clinical trials - Management indicated that a prophylactic immunosuppression regimen will begin with steroid treatment, with adjustments possible based on emerging data from ongoing studies [66][68] Question: Additional testing required by the FDA - Management noted that the additional testing relates to biocompatibility and that they are preparing for various risk assessments and testing as required by the FDA [70][71] Question: DRG or sensory testing in the Phase 1/2 trial - Management confirmed that they will monitor nerve conduction in the GM1 study to detect any changes, following assessments in non-human primates [73]