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Cash Equivalents and Marketable Securities: $245 million as of December 31, 2024, down from $314 million as of December 31, 2023.
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R&D Expenses: $209 million for the year ended December 31, 2024, compared to $232 million in 2023.
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Net Proceeds from Public Offering: $131 million received from an upsized public offering of common stock and pre-funded warrants in March 2024.
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Cash Runway Guidance: Expected to fund operations into the second half of 2026, excluding potential commercial revenue from RGX-121.
Release Date: March 13, 2025
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
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Regenxbio Inc (NASDAQ:RGNX) has submitted its first Biologics License Application (BLA) for RGX-121, targeting Hunter syndrome, with potential FDA approval expected in Q4 2025.
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The company is advancing its diabetic retinopathy program into a pivotal stage, with plans for future BLA filings for RGX-202 for Duchenne Muscular Dystrophy and ABBV-RGX-314 for Wet AMD.
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Regenxbio Inc (NASDAQ:RGNX) has a strong partnership with Nippon Shinyaku, enhancing its commercial capabilities and providing potential milestones and revenue.
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The company is well-positioned with robust commercial capabilities and global partners, aiming for sustainable profitability through its gene therapy leadership.
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Regenxbio Inc (NASDAQ:RGNX) has a solid financial position with $245 million in cash, cash equivalents, and marketable securities, expected to fund operations into the second half of 2026.
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Regenxbio Inc (NASDAQ:RGNX) experienced a decrease in cash and marketable securities from $314 million to $245 million over the year, primarily due to funding operating activities.
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The company faces risks and uncertainties associated with forward-looking statements, which may cause actual results to differ from forecasts.
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There is a potential regulatory approval risk for the priority review voucher (PRV) associated with RGX-121, which could impact non-dilutive financing options.
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The competitive landscape in the Wet AMD space poses challenges, with other long-acting TKI studies anticipated to read out in the same 2026 timeframe.
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Regenxbio Inc (NASDAQ:RGNX) has not yet initiated studies in non-ambulatory patients for its Duchenne Muscular Dystrophy program, focusing resources on current pivotal trials.
Q: Can you delve deeper into the components of non-dilutive financing available and the probabilities around realizing those? Also, any expectations around potentially going to an ADCOM for RGX-202 once you have the full data set? A: Patrick Christmas, Executive Vice President, Chief Legal Officer, explained that non-dilutive options include the DR milestone expected in the second half of the year, the potential monetization of a priority review voucher upon regulatory approval, and the reversion of the Zolgensma royalty stream. Curran Simpson, Chief Operating Officer, added that the pre-BLA meeting with the FDA has de-risked the process, making approval and PRV high-probability events. Stephen Pakola, Chief Medical Officer, noted that while they can’t predict an ADCOM with certainty, they are prepared if it occurs.
