• Cash, Cash Equivalents, and Marketable Securities: $279 million as of September 30, 2024, compared to $314 million as of December 31, 2023.
• R&D Expenses: $54 million for Q3 2024, compared to $58 million for Q3 2023.
• Net Proceeds from Public Offering: $131 million received from an upsized public offering in March 2024.
• Cash Runway Guidance: Expected to fund operations into 2026.

• Warning! GuruFocus has detected 6 Warning Signs with RGNX.

Release Date: November 06, 2024

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

Positive Points

• Regenxbio Inc (NASDAQ:RGNX) is making significant progress in advancing its late-stage programs, including RGX 202 for Duchenne and RGX 121 for Hunter syndrome.
• The company has initiated the BLA filing for RGX 121, which could become the first gene therapy for Hunter syndrome, marking a major milestone.
• RGX 202 is positioned as a potential best-in-class gene therapy for Duchenne, with promising clinical data and a favorable safety profile.
• Regenxbio Inc (NASDAQ:RGNX) has a strong cash position, with $279 million in cash and equivalents, expected to fund operations into 2026.
• The company is collaborating with AbbVie on RGX 314, with plans to initiate a global pivotal trial for diabetic retinopathy in the first half of 2025.

Negative Points

• Regenxbio Inc (NASDAQ:RGNX) faces uncertainties and risks associated with forward-looking statements and regulatory approvals.
• The company experienced a decrease in cash and equivalents from $314 million at the end of 2023 to $279 million as of September 30, 2024.
• There is concern about the small sample size for RGX 202, which may impact the interpretation of functional data.
• The potential impact of biosimilars on the market for RGX 314 in wet AMD remains uncertain.
• Regenxbio Inc (NASDAQ:RGNX) is preparing for a potential Adcom for RGX 121, which could delay the approval process.

Q & A Highlights

Q: Are there any concerns about the ability to treat the 1 to 3-year-old cohort in DMD, given their young age and potential for growing additional muscle tissue? A: Stephen Pakola, Executive Vice President, Chief Medical Officer, explained that while the issue of replicating cells is a topic of discussion, they are confident in their AAV8 vector's durability, as evidenced by its use in liver-directed hemophilia treatment, which shows sustained activity beyond 12 years. Curran Simpson, President and CEO, added that there are no requests for additional biomarker data beyond what's included in the protocol.

Q: Could you recap your latest view on how best to gauge the functional data expected later this month for RGX 202? A: Stephen Pakola stated that they will look at a variety of measures, including time function tests like the 10-meter walk/run and time to stand, as well as NSA A and caregiver-reported outcomes. Curran Simpson added that they will also use external controls to compare individual patient data against natural history databases.

Q: Will the initial group of patients' data be included in the pivotal data set for RGX 202? A: Curran Simpson confirmed that they expect to use data from the phase 1/2 study, particularly for patients dosed at dose level two, as the process and product quality will remain consistent through phase 1/2 and pivotal development. Stephen Pakola added that the primary endpoint for accelerated approval is microdystrophin, making it natural to use this data.

Q: What are your plans for the fellow eye study for the superchorioidal delivery program in wet AMD and DR? A: Stephen Pakola mentioned that they plan to evaluate bilateral treatment with superchorioidal delivery, similar to their subretinal program. They are confident in dosing both eyes due to the compartmentalized delivery method, which minimizes immune response.

Q: What was your reaction to Pfizer's phase 3 data, which showed expression but no functional benefit, and could this affect regulatory standards? A: Stephen Pakola emphasized the importance of their second-generation construct, which closely resembles the native dystrophin molecule, including key functional elements like the C-terminal domain. He noted that the FDA has acknowledged the importance of these elements, and they have seen no change in discussions with the FDA regarding their pivotal program.

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