Passage BIO(US:PASG)2022-05-16 18:07Financial Data and Key Metrics Changes - The company ended Q1 2022 with approximately $267.1 million in cash, cash equivalents, and marketable securities, down from $315.8 million as of December 31, 2021 [42] - R&D expenses for Q1 2022 were $26.2 million, compared to $25 million for the same quarter in 2021, primarily due to increases in clinical manufacturing and development expenses [43] - G&A expenses rose to $15.1 million in Q1 2022 from $12.5 million in the same quarter of 2021, driven by increased personnel-related expenses [45] - The net loss for Q1 2022 was $42.8 million, compared to $38.9 million for the same quarter in 2021 [46] Business Line Data and Key Metrics Changes - The company is focusing on three ongoing clinical programs: GM1 gangliosidosis, Krabbe disease, and frontotemporal dementia (FTD) [12][34] - The Imagine-1 trial for GM1 gangliosidosis reported positive interim safety and clinical efficacy data, with dosing of patients in multiple cohorts [9][21] - The GALax-C trial for Krabbe disease has opened additional clinical sites and reported a Grade-4 adverse event in the first patient, but the trial will continue with modifications [27][31] Market Data and Key Metrics Changes - The company has established a global network of trial sites across the U.S., Brazil, Canada, UK, Israel, and The Netherlands for its clinical programs [10] - The strategic decision to focus on specific programs has resulted in returning rights for certain programs to the University of Pennsylvania [13] Company Strategy and Development Direction - The company is committed to developing transformative therapies for CNS disorders with limited treatment options and has streamlined its operations to extend its cash runway into Q2 2024 [15] - The strategic prioritization includes a focus on ongoing clinical programs and a reduction in operating expenses through workforce reduction and slowed investment in manufacturing [11][14] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the progress made in Q1 2022 and anticipates reporting initial data from ongoing clinical trials in the second half of 2022 [48] - The company is optimistic about delivering multiple value-creating clinical milestones over the next two years [15] Other Important Information - The company has retained eight additional license options for future CNS indications through its partnership with Penn's Gene Therapy Program [13] - The company is actively working to improve patient recruitment for its clinical trials, particularly in the FTD program [36][84] Q&A Session Summary Question: Clarification on the Krabbe hydrocephalus event - Management noted that imaging prior to dosing showed changes in the ventricles, suggesting a potential link to disease progression rather than the drug itself [52][53] Question: Key outcomes measures for GM1 presentation - Management highlighted that the upcoming presentation will include updated data on biomarkers and clinical outcomes from the Imagine-1 trial [59] Question: Recruitment dynamics for GM1 program - Management indicated that they are open to collaborating with other centers to accelerate patient recruitment following the discontinuation of a competitor program [61] Question: Update on Krabbe disease trial - Management confirmed that the IDMC has approved proceeding with dosing and that they are revising the clinical trial protocol based on recommendations [68][70] Question: Changes to inclusion criteria for PBFT02 - Management confirmed that inclusion criteria were updated to broaden eligibility and improve enrollment [82] Question: Screening for GRN mutations in FTD - Management acknowledged challenges in identifying patients with GRN mutations and emphasized efforts to improve genetic testing and patient identification [110][113]