REGENXBIO Inc. Q1 FY2025 earnings call

Key Highlights

• RGX-121 (MPS II/Hunter syndrome): Expected FDA BLA acceptance soon, on track for potential approval in H2 2025; strategic partnership with Nippon Shinyaku for commercialization, goal to deliver to patients in H1 2026.
• RGX-202 (Duchenne muscular dystrophy): Pivotal study >50% enrolled, on track to submit BLA in mid-2026; planning commercial supply manufacturing in Q3 2025; expects to share updated functional data in H1 2025.
• Retinal Programs (ABBV-RGX-314): Advancing in wet AMD and diabetic retinopathy trials; completed end-of-Phase II meeting with FDA for DR, working on Phase III plans; subretinal wet AMD pivotal trials on track to complete enrollment in 2025.
• Financials: Cash, cash equivalents, and marketable securities at $272M as of March 31, 2025; R&D expenses decreased to $53M in Q1 2025 due to clinical trial expenses for RGX-314 and RGX-202.

No specific financial performance data for product segments provided in the transcript.

Guidance

• Cash runway of $272M as of March 31, 2025 expected to fund operations into H2 2026, with potential extension via non-dilutive financing.
• RGX-121 on track for potential FDA approval in H2 2025.
• RGX-202 pivotal study aims to complete enrollment in H2 2025 and submit BLA in mid-2026.
• ABBV-RGX-314 subretinal wet AMD pivotal trials to complete enrollment in 2025 and share top-line data in H1 2026; working on DR pivotal study with AbbVie in 2025.

Risks

• Forward-looking statements subject to risks and uncertainties detailed in REGENXBIO's Form 10-K and 10-Q.
• Regulatory risks: Potential delays or changes in FDA approval pathways for BLA reviews.
• Market risks: Impact of macroeconomic factors on gene therapy pricing and commercialization.

Question and Answer

Q: Timing for Hunter BLA acceptance; potential FDA delay?

A: Expect imminent BLA acceptance, no significant delays anticipated; no hypothetical FDA delays accounted for beyond normal review processes.

Q: Thoughts on FDA's focus on safety and impact on DMD approvability; biomarker vs functional data?

A: Program on track, strong functional data and safety profile; accelerated approval pathway considered, conviction in program's benefit to patients.

Q: Changes in FDA interactions; impact on retinal programs?

A: Business as usual, no significant changes in FDA interactions; safety focus in non-rare diseases is a priority, but no delays seen for retinal programs.

Q: Regulatory changes and impact on gene therapy in non-life-threatening conditions; DR indication delays?

A: Safety focus in non-rare is a priority, but subretinal program safety profile is excellent; no delays seen for DR indication.

Q: RGX-202 commercial supply manufacturing; data updates?

A: Initiating commercial supply manufacturing in Q3 2025; expects functional data press release in H1 2025, focusing on dose level two patients' outcomes.

Q: Wet AMD subretinal bilateral dosing; suprachoroidal opportunity?

A: Prospective data on bilateral dosing included in filings; suprachoroidal delivery offers in-office one-time treatment for diabetic retinopathy, addressing unmet need.

Q: Impact of recent drug pricing announcements on gene therapy pricing?

A: Early to tell, no immediate impact; monitoring macroenvironment, but cell and gene therapy may be less impacted initially.