Passage BIO(PASG) - 2022 Q2 - Earnings Call Transcript

Financial Data and Key Metrics Changes - The company ended Q2 2022 with approximately $239.3 million in cash, cash equivalents, and marketable securities, down from $267.1 million as of March 31, 2022, indicating a decrease in cash reserves [27] - R&D expenses were $26.8 million for Q2 2022, a decrease from $33.1 million in the same quarter of 2021, primarily due to a reduction in clinical manufacturing expenses [28] - G&A expenses were $13 million for Q2 2022, down from $15.4 million in Q2 2021, mainly due to a reduction in personnel-related expenses [29] - The net loss for Q2 2022 was $39.5 million, compared to a net loss of $48.4 million in Q2 2021, reflecting an improvement in financial performance [30] Business Line Data and Key Metrics Changes - The company is advancing three ongoing clinical programs, with significant progress in the Imagine-1 trial for GM1 gangliosidosis, now recruiting for the final cohort [6][7] - The PBKR03 program for early infantile Krabbe disease is actively recruiting patients, with initial safety and biomarker data expected by the end of 2022 [20][22] - The PBFT02 program for frontotemporal dementia is preparing to dose the first patient shortly, with ongoing patient identification efforts [23][24] Market Data and Key Metrics Changes - The company has established a global network of trial sites across the United States, Brazil, Canada, the UK, Israel, and the Netherlands to enhance patient recruitment [10] - Increased genetic testing among frontotemporal dementia patients has been observed, aiding in patient identification for clinical trials [10][45] Company Strategy and Development Direction - The company aims to develop transformative therapies for CNS disorders with limited or no approved treatment options, focusing on advancing clinical programs and achieving key milestones [6][32] - The company is evaluating its financial situation and prioritizing assets for clinical development, indicating a strategic approach to resource allocation [37] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving several important milestones in the coming months, including reporting initial data from ongoing clinical trials [32] - The company remains focused on managing cash carefully to ensure the delivery of meaningful clinical milestones [27][32] Other Important Information - The FDA cleared the IND application for the PBML04 program for metachromatic leukodystrophy, marking the company's fourth consecutive IND clearance [7][26] - The company has added Michael Kamarck, PhD, to its Board of Directors, bringing extensive biopharmaceutical experience [11] Q&A Session Summary Question: Will the initial Krabbe and GM1 data be released through a press release or a medical meeting? - The company is evaluating the best way to release the data, considering both scientific conferences and press releases [36] Question: Can you expand on the decision for clinical development for MLD? - The decision is based on evaluating the current financial situation and prioritizing assets rather than issues with the drug itself [37] Question: What safety events were observed in the initial patient dosed with PBKR03? - The first patient developed acute hydrocephalus but is currently stable after treatment, and additional safety monitoring has been implemented [41] Question: What is the status of patient recruitment for FTD? - Recruitment challenges are multifactorial, including patient identification and competition with other trials, but efforts are ongoing to improve throughput [49] Question: What is the status of the CEO search? - The Board is actively engaged in a public search for a new CEO and is making good progress [47]