REGENXBIO(RGNX) - 2023 Q1 - Earnings Call Transcript

Financial Data and Key Metrics Changes - REGENXBIO ended Q1 2023 with cash, cash equivalents, and marketable securities totaling $474 million, down from$565 million as of December 31, 2022, primarily due to cash used for operating activities [73] - R&D expenses for Q1 2023 were $59 million, an increase from$56 million in Q1 2022, attributed to higher personnel costs and laboratory expenses related to the manufacturing innovation center [74] Business Line Data and Key Metrics Changes - The company is advancing its 5x'25 strategy, aiming to move five AAV therapeutics into pivotal or commercial stages by 2025 [22][25] - The RGX-314 program has dosed over 600 patients across seven trials, demonstrating good tolerability and potential as a one-time treatment for major retinal vascular diseases [29][30] Market Data and Key Metrics Changes - The global market for diabetic retinopathy (DR) is estimated to reach $15 billion in the next decade, with RGX-314 positioned as a potential one-time treatment option [47] - The anti-VEGF market is calculated to be over$14 billion globally, with RGX-314 targeting over 30 million patients with high unmet needs [66] Company Strategy and Development Direction - REGENXBIO is focused on expanding its global reach through collaboration with AbbVie, particularly in the eye care sector, to bring RGX-314 to patients worldwide [44][62] - The company is leveraging its NAVXpress platform for manufacturing, which is operational and designed to meet global regulatory standards [64] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the regulatory pathway for RGX-314, anticipating submissions to the FDA and EMA in late 2025 to early 2026 [62] - The company is optimistic about the potential of RGX-314 to address the high unmet need in retinal diseases, particularly given the limitations of current anti-VEGF treatments [63] Other Important Information - The company announced the transfer of INDs for ongoing clinical trials to AbbVie, marking a significant step in their collaboration [23][26] - REGENXBIO's manufacturing innovation center is set to produce commercial-scale GMP material for the RGX-314 clinical program [45] Q&A Session Summary Question: What is the impact of the increased enrollment in ATMOSPHERE and ASCENT on regulatory submissions? - Management indicated that the increased sample size enhances power for both primary and secondary endpoints, which is crucial for regulatory submissions [80][81] Question: Was there an alteration in the agreement with AbbVie regarding regulatory responsibilities? - Management clarified that the transition of regulatory responsibilities was part of the original collaboration agreement and is now being implemented as planned [84][109] Question: How does the decision to expand enrollment impact the registration pathway for suprachoroidal delivery? - Management believes that the expansion will support important milestones for regulatory approvals in both the U.S. and internationally [91][92] Question: What is the expected safety profile for RGX-202 in the Duchenne program? - Management stated that the trial is excluding patients with preexisting AAV8 antibodies to ensure safety and efficacy [95][97] Question: How will the fellow eye study be conducted in relation to the pivotal trials? - The fellow eye study will treat patients at a later time to avoid confounding results, aligning with FDA discussions [120][135]