• Cash Equivalents and Marketable Securities: $245 million as of December 31, 2024, down from $314 million as of December 31, 2023.
• R&D Expenses: $209 million for the year ended December 31, 2024, compared to $232 million in 2023.
• Net Proceeds from Public Offering: $131 million received from an upsized public offering of common stock and pre-funded warrants in March 2024.
• Cash Runway Guidance: Expected to fund operations into the second half of 2026, excluding potential commercial revenue from RGX-121.

• Warning! GuruFocus has detected 5 Warning Signs with RGNX.

Release Date: March 13, 2025

For the complete transcript of the earnings call, please refer to the full earnings call transcript.

Positive Points

• 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.
• 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.
• Regenxbio Inc (NASDAQ:RGNX) has a strong partnership with Nippon Shinyaku, enhancing its commercial capabilities and providing potential milestones and revenue.
• The company is well-positioned with robust commercial capabilities and global partners, aiming for sustainable profitability through its gene therapy leadership.
• 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.

Negative Points

• 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.
• The company faces risks and uncertainties associated with forward-looking statements, which may cause actual results to differ from forecasts.
• There is a potential regulatory approval risk for the priority review voucher (PRV) associated with RGX-121, which could impact non-dilutive financing options.
• 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.
• 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 & A Highlights

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.

Q: Regarding the DMD program, how is the pace of enrollment for the pivotal trial, and have you started enrolling younger patients? A: Curran Simpson stated that enrollment is encouraging, with significant interest from patients and families. They expect enrollment to accelerate as more sites are activated. Stephen Pakola added that they are enrolling patients across all age ranges, including younger patients, and these will count towards the pivotal trial.

Q: Are you measuring cardiac endpoints in the AFFINITY study, and could this differentiate your program from others? A: Stephen Pakola confirmed that they are measuring cardiac endpoints like ejection fraction and troponin levels. While cardiac deterioration typically occurs in older patients, they are optimistic about the potential benefits of their therapy based on preclinical data.

Q: On the regulatory discussions for diabetic retinopathy, are there differences between US and OUS regulators? A: Stephen Pakola explained that while the US has a clear path using the diabetic retinopathy severity scale, they are working with EMA and Japan to establish a similar path. They believe the case for using this scale is strong and are optimistic about global regulatory discussions.

Q: What should we expect from the upcoming functional data update for the DMD program, and how will you analyze this data? A: Curran Simpson mentioned that the update will include new patients and longer-term data for previously reported patients. Stephen Pakola added that they will compare the data to matched external natural history controls, a method discussed with the FDA during the end of Phase 2 meeting.

For the complete transcript of the earnings call, please refer to the full earnings call transcript.