REGENXBIO Inc. (RGNX)·Q1 2025 Earnings Summary

Executive Summary

• Q1 2025 revenue was $89.012M and GAAP diluted EPS was $0.12; both missed Wall Street consensus ($105.350M revenue, $0.34 EPS). Revenue strength came from $71.8M recognized under the Nippon Shinyaku collaboration, while EPS benefitted from higher investment income and restrained R&D, but fell short of estimates .
• Cash, cash equivalents and marketable securities rose to $272.7M, with management reiterating cash runway into H2 2026; shortly after quarter-end, FDA accepted RGX‑121 (Priority Review; PDUFA Nov 9, 2025), and non‑dilutive financing further extended runway (early 2027) .
• Strategic catalysts: RGX‑121 Priority Review in MPS II, RGX‑202 pivotal enrollment >50% with planned BLA mid‑2026, and AbbVie-partnered RGX‑314 (wet AMD/DR) progressing toward pivotal/2026 data; commercial supply manufacturing for RGX‑202 begins in Q3 2025 .
• Stock-relevant narrative: near-term regulatory de‑risking (RGX‑121 acceptance), robust pipeline momentum, and reiterated cash runway, offset by the revenue/EPS miss vs consensus and continued operating/investment expense headwinds .*

What Went Well and What Went Wrong

What Went Well

• RGX‑121 BLA accepted with Priority Review; management highlighted potential first-in-class gene therapy for MPS II and PRV monetization optionality: “Acceptance of the RGX‑121 BLA marks an exciting milestone...” (PDUFA 11/9/2025) .
• RGX‑202 pivotal study “beyond 50% enrolled”; commercial supply manufacturing starts in Q3 2025; in-house capacity up to 2,500 doses/year with >80% full capsid purity, supporting fast-follower positioning in Duchenne .
• Strong balance sheet and non-dilutive financing levers: CFO reiterated runway into H2 2026 and outlined additional options (milestones, PRV sale, Zolgensma royalty reversion) to extend well beyond 2026 .

What Went Wrong

• Revenue/EPS both missed consensus despite collaboration revenue recognition; revenue $89.012M vs $105.350M est., EPS $0.12 vs $0.34 est.* The delta appears tied to timing/mix of license and service revenue and higher interest expense ($8.57M) .*
• Operating expense/investment headwinds persist: total operating expenses were $76.885M; interest expense rose to $8.57M, tempering bottom-line leverage .
• Regulatory/safety scrutiny in DMD heightened post-Elevidys event; management emphasized immunomodulation and high product purity, but investors remain sensitive to FDA expectations around accelerated approval and functional data requirements .

Financial Results

Consolidated Results vs Prior Periods and Consensus

Values with asterisks retrieved from S&P Global.*

Margins

Values with asterisks retrieved from S&P Global.*

Revenue Breakdown

Management disclosed $71.8M of license and service revenue recognized under Nippon Shinyaku in Q1 2025 .

KPIs

Guidance Changes

Earnings Call Themes & Trends

Management Commentary

• “We have made tremendous progress towards delivering multiple commercial gene therapies, starting this year.” — Curran M. Simpson, President & CEO .
• “Our manufacturing innovation center is a state-of-the-art integrated GMP facility that can produce up to 2,500 doses of RGX‑202 annually… delivering industry-leading purity levels in Duchenne with over 80% full capsids.” — Curran M. Simpson .
• “Our accumulating Phase I/II results are showing impressive evidence of differentiation on safety, biomarker and functional outcomes… with all participants above 10% expression.” — Dr. Steve Pakola, CMO .
• “We expect the balance in cash… $272M… to fund our operations into the second half of 2026… many non-dilutive financing optionalities could extend our cash runway well beyond.” — Mitchell Chan, CFO .

Q&A Highlights

• Timing/acceptance of RGX‑121 BLA: management guided to imminent acceptance (now achieved), citing “business normal” FDA interactions and confidence in AA pathway .
• DMD safety/regulatory bar post-Elevidys: team emphasized proactive immunomodulation, high purity product, consistent safety profile; enrollment on track for N=30 pivotal dataset .
• DR Phase III planning with AbbVie: global regulatory feedback gathered (FDA/EMA/Japan), endpoint strategy under consideration; first patient dose expected in 2025 .
• RGX‑202 commercial build-out: in-house capacity of 2,500 doses/year; plan to stockpile inventory for 2027 launch to address large prevalent market .
• Wet AMD bilateral dosing: fellow-eye data supportive; intent to include in regulatory package .

Estimates Context

• Q1 2025 actual vs consensus: Revenue $89.012M vs $105.350M estimate (miss); EPS $0.12 vs $0.34 estimate (miss).* Estimate counts: revenue (9), EPS (8).* Sequentially, revenue increased from $21.214M in Q4 2024, and improved year-over-year from $15.622M, driven by collaboration revenue recognition under Nippon Shinyaku, but came in below estimates likely due to timing/mix of license/service revenue recognition .*
• FY 2025 consensus: Revenue $206.942M; EPS $(2.97).* Post-Q1, model revisions may modestly reduce near-term revenue/EPS to reflect actuals and interest expense trajectory.*

Values retrieved from S&P Global.*

Key Takeaways for Investors

• Revenue/EPS miss vs consensus despite strong collaboration revenue recognition; monitor revenue timing/mix and interest expense trajectory near term .*
• Regulatory de-risking: RGX‑121 Priority Review accepted with 11/9/2025 PDUFA date, offering a tangible near‑term catalyst and PRV monetization optionality .
• DMD fast-follower: RGX‑202 pivotal enrollment >50%, robust safety/functional profile, and Q3 2025 start of commercial supply manufacturing position the asset well for mid‑2026 BLA filing .
• Manufacturing advantage: In-house GMP capacity (2,500 doses/year) and >80% full capsid purity are strategic differentiators for launch readiness and product quality .
• AbbVie partnership: Wet AMD pivotal trials on track for 2026 topline; DR Phase III initiation targeted in 2025; milestone revenue potential supports non‑dilutive funding .
• Cash runway: $272.7M at quarter-end into H2 2026; subsequent royalty financing extends to early 2027, reducing financing overhang .
• Near-term trading: Expect sensitivity to any incremental RGX‑202 functional data releases in 1H 2025, RGX‑121 review updates, and DR Phase III initiation timing; narrative skew improving on regulatory momentum, but quarterly prints may be volatile given collaboration revenue timing and interest expense .*