REGENXBIO(US:RGNX)2020-11-05 05:01Financial Data and Key Metrics Changes - REGENXBIO reported revenues of $98.9 million for Q3 2020, a significant increase from $14.7 million in Q3 2019, primarily due to an $80 million milestone fee from Novartis and a $9.6 million increase in Zolgensma royalty revenue [36] - The company ended Q3 2020 with cash, cash equivalents, and marketable securities totaling $289.8 million, down from $400 million as of December 31, 2019, mainly due to net cash used in operating activities of $93.5 million [35] - Net income for Q3 2020 was $8.8 million, compared to a net loss of $34.6 million in Q3 2019, reflecting a significant turnaround in financial performance [38] Business Line Data and Key Metrics Changes - The RGX-314 program is advancing with two Phase II studies underway for the treatment of wet AMD and diabetic retinopathy, indicating progress in the clinical pipeline [9][13] - The company has expanded its MPS II program, allowing for additional patients in the ongoing Phase 1/2 study of RGX-121, demonstrating growth in its rare disease portfolio [23] Market Data and Key Metrics Changes - Zolgensma sales increased by 82% in Q3 2020 compared to Q3 2019, contributing significantly to the company's revenue growth [36] - The ALTITUDE trial for RGX-314 in diabetic retinopathy is expected to enroll 40 patients, highlighting the company's focus on addressing significant unmet medical needs in the market [22] Company Strategy and Development Direction - REGENXBIO is focused on advancing its NAV technology platform and expanding its clinical pipeline, with plans to initiate pivotal studies for RGX-314 in early 2021 [15][30] - The company is also enhancing its manufacturing capabilities with a new GMP facility in Rockville, aimed at ensuring product consistency and scalability [42][44] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the long-term durability of RGX-314, citing positive results from ongoing studies and the potential for a one-time treatment option for patients [15][21] - The impact of the COVID-19 pandemic on clinical development timelines is being actively monitored, with a focus on maintaining operational stability [11] Other Important Information - REGENXBIO received an $80 million milestone payment from Novartis based on Zolgensma's cumulative net sales exceeding $1 billion, reflecting the success of its partnered product [33] - The company is engaged in ongoing discussions regarding intellectual property rights related to its NAV technology platform, emphasizing the importance of protecting its innovations [100][102] Q&A Session Summary Question: Update on suprachoroidal microinjector program and safety data - Management confirmed that no inflammation has been observed in patients dosed so far in the AAVIATE trial, with a total of 20 patients planned for cohort 1 [55][56] Question: Update on MPS II program - Management plans to provide an update on the MPS II program by the end of 2020, with additional patients enrolled under the expanded protocol [62] Question: Frequency of rescue VEGF injections for RGX-314 - Management indicated that discussions with physicians suggest a target of at least a 50% reduction in the need for rescue injections, emphasizing the value of gene therapy in improving patient outcomes [68][70] Question: Status of CLN2 programs and IND filing - Management confirmed that preclinical studies for RGX-181 and RGX-381 are nearing completion, with IND filings expected by the end of 2020 [90][92] Question: Update on IP dispute with Passage Bio and Sarepta - Management stated there are no specific updates on the IP dispute but emphasized the importance of protecting their NAV technology platform [100][102]