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REGENXBIO Inc. (RGNX)·Q4 2024 Earnings Summary

Executive Summary

  • Q4 2024 revenue was $21.2M, down 4% year over year and 12% sequentially; GAAP diluted EPS improved to ($1.01) from ($1.43) in Q4 2023 and from ($1.17) in Q3 2024, with operating loss narrowing on lower R&D and credit loss recoveries .
  • Guidance: cash, cash equivalents and marketable securities of $244.9M at 12/31/24 plus $110M upfront from Nippon Shinyaku fund operations into the second half of 2026 (raised vs prior “into 2026”) .
  • Catalysts: RGX-121 BLA submitted; potential FDA approval 2H 2025; RGX-202 pivotal trial nearly 50% enrolled with BLA targeted mid‑2026; AbbVie retinal programs targeting topline wet AMD data in 2026 and planning DR Phase III in 2025 .
  • Key stock reaction drivers ahead: regulatory milestones (RGX‑121 approval, PRV monetization), nondilutive capital (AbbVie DR milestone), and pivotal RGX‑202 biomarker/functional data flow in 2025–2026 .

What Went Well and What Went Wrong

What Went Well

  • BLA for clemidsogene lanparvovec (RGX‑121) submitted; potential approval 2H 2025; management: “We’ve submitted our first BLA and expect our first FDA approval in the fourth quarter [2025]” .
  • RGX‑202 pivotal advancing rapidly; nearly 50% enrolled; management emphasized manufacturing readiness: “can produce 2,500 doses of RGX‑202 per year… purity levels >80% full capsid” .
  • Retinal franchise progressing: DR pivotal planning with AbbVie in 2025; wet AMD pivotal topline expected in 2026; fellow‑eye data showed 97% reduction in anti‑VEGF burden with durable control and no intraocular inflammation .

What Went Wrong

  • Revenue decline driven by lower Zolgensma royalties (FY 2024 $81.5M vs $85.3M FY 2023), pressuring top line in Q4 and full year .
  • Continued operating losses: Q4 loss from operations ($51.4M), though improved YoY; interest expense rose to ($9.4M) in Q4 2024 .
  • Cash declined to $244.9M at year‑end from $314.1M prior year, reflecting funding of operations despite March 2024 offering proceeds .

Financial Results

Income Statement and EPS vs prior periods and estimates

MetricQ4 2023Q3 2024Q4 2024
Revenues ($USD Millions)$22.2 $24.2 $21.2
License and Royalty Revenue ($USD Millions)$22.2 $24.2 $21.2
Cost of Revenues ($USD Millions)$11.2 $12.4 $6.3
Gross Profit ($USD Millions)$11.0 (computed from cited data) $11.8 (computed from cited data) $14.9 (computed from cited data)
Gross Margin (%)49.4% (computed from cited data) 48.8% (computed from cited data) 70.2% (computed from cited data)
Loss from Operations ($USD Millions)($63.9) ($62.1) ($51.4)
Operating Margin (%)(287.6)% (computed from cited data) (256.6)% (computed from cited data) (241.9)% (computed from cited data)
GAAP Diluted EPS ($)($1.43) ($1.17) ($1.01)
Consensus Revenue Estimate ($USD Millions)N/A (S&P Global unavailable)N/A (S&P Global unavailable)N/A (S&P Global unavailable)
Consensus EPS Estimate ($)N/A (S&P Global unavailable)N/A (S&P Global unavailable)N/A (S&P Global unavailable)

Note: Consensus estimates unavailable via S&P Global; estimate comparisons not provided.

OpEx and Cash KPIs

MetricQ4 2023Q3 2024Q4 2024
R&D Expense ($USD Millions)$55.7 $54.4 $50.4
G&A Expense ($USD Millions)$19.1 $19.4 $20.1
Credit Losses (Recoveries) ($USD Millions)$0.0 N/A($5.0)
Investment Income ($USD Millions)$2.4 $3.3 $9.5
Interest Expense ($USD Millions)($1.4) ($0.8) ($9.4)
Cash, Cash Equivalents & Marketable Securities ($USD Millions)$314.1 (12/31/2023) $278.6 (9/30/2024) $244.9 (12/31/2024)

Annual Royalty KPI

MetricFY 2023FY 2024
Zolgensma Royalty Revenue ($USD Millions)$85.3 $81.5

Guidance Changes

MetricPeriodPrevious GuidanceCurrent GuidanceChange
Cash RunwayCorporateFund operations into 2026 Fund operations into 2H 2026, including $110M Nippon upfront Raised
RGX‑121 Regulatory TimelineMPS IIRolling BLA completion Q1 2025 Potential FDA approval in 2H 2025; PRV retained New/More specific
RGX‑202 BLA TimingDMDBLA submission in 2026 BLA under accelerated approval pathway targeted mid‑2026 Refined
ABBV‑RGX‑314 DR PivotalDRInitiate first global pivotal in H1 2025 Planning Phase III program in 2025 to support global filings Maintained
ABBV‑RGX‑314 Wet AMD Pivotal ToplineWet AMDTopline expected H1 2026 Topline expected 2026; enrollment to complete in 2025 Maintained
Nondilutive FinancingCorporate$200M AbbVie DR milestone potential disclosed (Q2) Expect DR milestone in 2H 2025; PRV monetization upon approval; Zolgensma royalties revert after $300M cap Clarified timing/options

Earnings Call Themes & Trends

TopicQ2 2024 (Aug)Q3 2024 (Nov)Q4 2024 (Mar)Trend
RGX‑202 clinical differentiation & pivotal pathHigh microdystrophin across ages; EOP2 success; pivotal in Q4 2024; manufacturing 2,500 doses/yr First functional data planned; pivotal initiation imminent; expansion to 1–3 yrs Nearly 50% enrolled; biomarker/functional updates in H1 2025; BLA mid‑2026 Accelerating with robust data and enrollment
RGX‑121 regulatory progressPre‑BLA success; rolling BLA to start Q3 2024 Rolling BLA initiated; completion Q1 2025 BLA completed; potential approval 2H 2025 Entering review/approval window
Retinal franchise (wet AMD, DR)DR EOP2 moved up to Q4; DR pivotal H1 2025; wet AMD pivotal ongoing DR EOP2 Q4; wet AMD global submissions H1 2026; fellow‑eye data positive DR Phase III planning; wet AMD topline 2026; enrollment completion expected 2025 On track; derisked with safety/efficacy
Manufacturing readinessNAVXpress suspension; scalable to 2,000L; commercial‑ready Emphasis on consistent product quality bridging P1/2 to pivotal 2,500 doses/yr capacity; >80% full capsid purity Strengthening
Financing optionalityHighlighted AbbVie $200M DR milestone; PRV monetization potential Milestone recognition upon first patient dosed; runway into 2026 DR milestone expected 2H 2025; PRV upon RGX‑121 approval; royalties revert post cap Expanded/clearer timing

Management Commentary

  • “2025 is a transformational year… We’ve submitted our first BLA and expect our first FDA approval in the fourth quarter for RGX‑121… then head into 2026 and 2027 with potential BLA filings for RGX‑202 and ABBV‑RGX‑314” — Curran M. Simpson, CEO .
  • “Our manufacturing innovation center can produce 2,500 doses of RGX‑202 per year… commercial‑ready manufacturing… second to market position in Duchenne remains strong” — CEO .
  • “ALTITUDE® DR… end of Phase II meeting was very positive… planning a Phase III clinical program to support global regulatory filings” — CMO .
  • “CAMPSIITE trial met its primary pivotal endpoint… BLA using the accelerated approval pathway… D2S6 reasonably likely to predict clinical benefit” — CMO .

Q&A Highlights

  • Nondilutive financing: Mgmt expects DR milestone in 2H 2025 upon first patient dosed; PRV monetization post RGX‑121 approval; Zolgensma royalties revert after $300M cap — “timing risk” more than binary risk; view approval probability high given pre‑BLA feedback .
  • DMD pivotal enrollment outlook: Screening robust; enrollment to accelerate as sites add and functional data shared; younger cohorts (1–3 yrs) included in pivotal; interest strong across ages .
  • Cardiac endpoints in DMD: AFFINITY collects EF via echo/MRI and troponin; differentiation may be more apparent over longer follow‑up as patients age .
  • Wet AMD pivotal strategy: No plan to cut enrollment; goal is full characterization vs standard regimens; AbbVie to drive fast filing once topline available .
  • Regulatory continuity (CBER): Company not seeing meaningful changes in review cadence or team availability; remains “business as usual” .

Estimates Context

  • S&P Global consensus estimates for Q4 2024 were unavailable at the time of analysis; therefore, comparisons to Street expectations are not provided. Future revisions may be needed as nondilutive milestones and regulatory timelines firm up (AbbVie DR, RGX‑121 approval/PRV) .

Key Takeaways for Investors

  • Regulatory path is crystallizing: RGX‑121 BLA filed with potential approval 2H 2025, positioning PRV monetization and initial commercial revenue stream with partner Nippon Shinyaku .
  • RGX‑202 is the near‑term growth driver: pivotal nearing half enrollment; mid‑2026 BLA targeted; manufacturing capacity and product purity support a fast, broad launch strategy .
  • Retinal franchise provides large optionality: DR pivotal program planning in 2025 and wet AMD topline in 2026; fellow‑eye subretinal data de‑risk bilateral use, enhancing commercial potential .
  • Financial runway extends into 2H 2026 with the $110M Nippon upfront; additional nondilutive options (AbbVie DR milestone; PRV sale; royalty reversion) could meaningfully extend runway .
  • Operating discipline evident: R&D down YoY/QoQ; credit loss recoveries and higher investment income helped offset expenses; but interest expense rose, keeping focus on cash management .
  • Near‑term trading catalysts: any FDA acceptance milestones for RGX‑121 BLA, DR Phase III initiation, additional RGX‑202 biomarker/functional readouts, and updates on nondilutive financing timing (AbbVie milestone) .
  • Medium‑term thesis: multiple first/best‑in‑class one‑time gene therapies across rare and retinal indications, with in‑house manufacturing as a strategic advantage and partnerships (AbbVie, Nippon) to scale commercialization .

Appendix: Additional Data Cross‑Checks

  • Q4 2024 revenue $21.2M; net loss ($51.2M); GAAP EPS ($1.01); cash & marketable securities $244.9M .
  • Q3 2024 revenue $24.2M; GAAP EPS ($1.17); cash & marketable securities $278.6M .
  • FY Zolgensma royalties: $81.5M (2024) vs $85.3M (2023) .