REGENXBIO(US:RGNX)2025-03-13 20:30Financial Data and Key Metrics Changes - REGENXBIO ended the quarter on December 31, 2024, with cash, cash equivalents, and marketable securities of $245 million, down from $314 million as of December 31, 2023, primarily due to cash used for operating activities [26] - R&D expenses were $209 million for the year ended December 31, 2024, compared to $232 million in 2023, attributed to decreases in headcount and preclinical activities [26][27] - The company expects the cash runway to fund operations into the second half of 2026, with potential extensions through non-dilutive financing options [27] Business Line Data and Key Metrics Changes - The pivotal study for RGX-202 is advancing rapidly, with nearly half of the enrollment completed, and the company expects to submit a BLA under the accelerated approval pathway by mid-2026 [12][29] - The BLA for RGX-121 has been submitted, with potential FDA approval anticipated in the second half of 2025 [7][24] - The diabetic retinopathy program is moving towards a pivotal program with AbbVie, with successful end-of-Phase II meetings held with the FDA [14][20] Market Data and Key Metrics Changes - The partnership with Nippon Shinyaku for MPS programs is strategically significant, combining REGENXBIO's development expertise with Nippon's commercialization experience [9][10] - The company is preparing for potential approval of RGX-121 for MPS II in Q4 2025, which could enhance its market position [10][24] Company Strategy and Development Direction - REGENXBIO aims to launch multiple first or best-in-class gene therapies, focusing on sustainable profitability and leveraging over 15 years of gene therapy leadership [8][29] - The company is positioned to deliver on multiple late-stage opportunities, with a focus on differentiation against standard care and available treatments [29][31] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the strong momentum across the pipeline, with key milestones approaching [29] - The company believes it is well-positioned to deliver transformative treatments for patients in need of new options, particularly in the Duchenne muscular dystrophy and retinal disease markets [29][31] Other Important Information - The company has a robust commercial capability and global partnerships, which are expected to support the launch of its gene therapies [7][10] - REGENXBIO is actively working on non-dilutive financing options, including milestone payments and potential monetization of a priority review voucher [27] Q&A Session Summary Question: Can you delve deeper into the components of non-dilutive financing? - Management outlined three components: expected milestone payments from the diabetic retinopathy program, potential monetization of a priority review voucher upon regulatory approval, and the reversion of royalty income from Zolgensma once a cap is reached [36][39][40] Question: What are the expectations around potentially going to an advisory committee for RGX-202? - Management indicated that while they do not foresee a significant issue that would necessitate an advisory committee, they are prepared for it if required [46][47] Question: Can you provide an update on the NDA and patient data for the one to three-year-old cohort? - Management plans to update on one patient from the one to four cohort at the upcoming MDA meeting, with expectations of higher microdystrophin levels in younger patients [51][53] Question: What is the current pace of enrollment for the DMD pivotal trial? - Management reported encouraging enrollment and expects acceleration as more sites are activated, with a significant number of patients showing interest [58][59] Question: Are cardiac endpoints being measured in the DMD study? - Management confirmed that they are measuring cardiac endpoints, including ejection fraction and troponin levels, but noted that significant changes may not be observable in younger patients [67][72] Question: Will enrollment for the wet AMD study be cut short to accelerate timelines? - Management stated that they will not cut enrollment short, as they aim to fully characterize safety and efficacy in collaboration with AbbVie [75][76]