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REGENXBIO(RGNX) - 2020 Q1 - Earnings Call Transcript

Financial Data and Key Metrics Changes - As of March 31, 2020, cash, cash equivalents, and marketable securities totaled $356.6 million, down from $400 million as of December 31, 2019, primarily due to net cash used in operating activities of $35.6 million and cash used for property and equipment purchases of $4.6 million [38] - Revenues for Q1 2020 were $17.6 million, a significant increase from $900,000 in Q1 2019, driven by $10 million in royalty revenue from Zolgensma and $7.2 million in license revenue from a new agreement with Ultragenyx [39] - Research and development expenses rose to $37 million in Q1 2020 from $25.2 million in Q1 2019, attributed to increased personnel costs and clinical trial expenses [41] - General and administrative expenses increased to $14.8 million in Q1 2020 from $11.6 million in Q1 2019, mainly due to higher personnel-related costs and professional fees [42] - The net loss for Q1 2020 was $40 million, or $1.08 per share, compared to a net loss of $32.2 million, or $0.89 per share, in Q1 2019 [43] Business Line Data and Key Metrics Changes - The RGX-314 gene therapy program demonstrated stable and consistent results, with 73% of patients remaining anti-VEGF injection-free nine months after a single administration [15][24] - The company is advancing its internal gene therapy pipeline, with ongoing trials for RGX-121 and RGX-111, and expects to provide updates in mid-2020 [16][28] Market Data and Key Metrics Changes - Novartis reported over $530 million in net sales of Zolgensma as of the end of Q1 2020, indicating progress towards the $1 billion milestone that would trigger an $80 million payment to REGENXBIO [40] Company Strategy and Development Direction - The company is focused on improving lives through gene therapy and has made significant progress in its NAV Technology Platform, with over 20 partnered product candidates [30][36] - REGENXBIO is expanding its gene therapy pipeline and has plans to initiate trials for RGX-314 using a suprachoroidal delivery approach in 2020 [25][30] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the potential of gene therapy to provide safer treatment alternatives during the COVID-19 pandemic, highlighting the importance of single administration treatments [50][51] - The company anticipates regulatory updates and continued progress in its clinical programs throughout 2020 [36][52] Other Important Information - The construction of a GMP production facility in Rockville is ongoing and expected to be operational in 2021, which will enhance production capabilities [18] Q&A Session Summary Question: Regarding the suprachoroidal data and initiation in the first half of this year - Management confirmed that they are targeting to start the suprachoroidal delivery development program for wet AMD by the end of the first half of 2020 and will provide updates based on data available at that time [55][56] Question: Concerns about inflammation with the suprachoroidal approach - Management acknowledged the potential for inflammation but noted that preclinical data with their proprietary NAV technology showed no inflammation with suprachoroidal delivery, providing comfort regarding this route [64][66] Question: Financial aspects regarding CapEx build-out and Abeona license agreement - Management clarified that the CapEx for the new headquarters and GMP facility will be in the tens of millions over the life of the program and confirmed that there are outstanding amounts due from Abeona due to a breach of contract [84][86]