REGENXBIO Inc. (RGNX)·Q2 2025 Earnings Summary

Executive Summary

• Q2 2025 missed Street: revenue $21.36M vs $40.87M consensus and EPS $(1.38) vs $(0.90) consensus; larger operating loss and higher interest expense drove the EPS delta, while royalties (Zolgensma) declined YoY to $18.4M . S&P Global estimates marked with asterisks; see disclaimer below.*
• Cash runway extended to “into early 2027” following $150M non‑dilutive royalty financing; quarter-end cash, cash equivalents and marketable securities were $363.6M .
• Pipeline catalysts tightened: RGX‑202 pivotal enrollment now expected complete in October 2025 (earlier than prior), topline 1H26, BLA mid‑2026; RGX‑121 BLA accepted with Priority Review (PDUFA Nov 9, 2025); sura‑vec DR advancing to pivotal Phase IIb/III with $100M milestone upon first patient dosed from AbbVie .
• Management emphasized differentiated safety strategies (immune modulation, high full capsid purity), AbbVie’s increased investment (ACHIEVE Phase IIIb), and non‑dilutive funding optionality (potential PRV sale, milestones) as supports into multiple launches .

What Went Well and What Went Wrong

What Went Well

• RGX‑202 DMD program timelines pulled forward; enrollment completion now targeted for October 2025; commercial manufacturing to start in Q3 2025 to enable launch scale capacity (“up to 2,500 doses per year”) .
  • CEO: “We now expect to complete enrollment in the ongoing pivotal study this October… initiating commercial manufacturing this fall… can produce up to 2,500 doses of RGX‑202 per year.”
• RGX‑121 BLA accepted with Priority Review; FDA mid‑cycle, PLI and BIMO inspections completed with no observations; PDUFA Nov 9, 2025 .
• Sura‑vec (ABBV‑RGX‑314) DR program advanced to pivotal Phase IIb/III on positive two‑year ALTITUDE data; AbbVie milestone structure updated ($100M upon first patient in Phase IIb/III; $100M upon first patient in second Phase III); AbbVie to fund ACHIEVE Phase IIIb wet AMD study .
  • CMO: “A single in‑office injection… was well tolerated… dose level 3 achieving at least a 70% reduction in vision‑threatening events vs historical control.”

What Went Wrong

• Financial miss vs consensus: revenue $21.36M vs $40.87M consensus and EPS $(1.38) vs $(0.90); YoY royalty decline (Zolgensma royalties $18.4M from $21.8M) and higher R&D burden pressured results .*
• Operating spend rose: R&D $59.5M (vs $48.9M YoY) driven by manufacturing/clinical supply and pivotal trial costs for sura‑vec and RGX‑202; G&A also higher YoY to $19.9M .
• Interest expense jumped to $11.0M (vs $0.45M YoY), contributing to net loss $(70.9)M; company completed a $150M royalty monetization during the quarter, increasing financing costs .

Financial Results

GAAP results and comps

Q2 2025 vs Street consensus

Revenue mix

Balance sheet and liquidity

• Cash, cash equivalents and marketable securities: $363.6M at 6/30/25; expected to fund operations into early 2027 .
• Executed $150M non‑dilutive royalty monetization in May 2025; two optional $50M tranches remain available .

Guidance Changes

Earnings Call Themes & Trends

Management Commentary

• CEO (RGX‑202 timelines and scale): “We now expect to complete enrollment in the ongoing pivotal study this October… initiating commercial manufacturing this fall… can produce up to 2,500 doses of RGX‑202 per year.”
• CMO (DR efficacy and safety): “At two years in dose level 3… zero cases of intraocular inflammation… dose level three achieving at least a 70% reduction in [vision‑threatening events] compared to historical control.”
• CFO (runway and non‑dilutive options): “We expect the June 30 cash balance… to fund our operation into early 2027… does not include… PRV sale… AbbVie milestones… additional funds from the May 2025 health care royalty agreement.”

Q&A Highlights

• DR pivotal design and dosing: Phase IIb/III under one protocol, double‑masked with sham control, interim data to inform Part 2; evaluating DL3 and DL4 to “not leave efficacy on the table” given strong safety with short‑course steroids .
• AbbVie amendment economics: Splits prior $200M DR milestone into $100M at Phase IIb/III first patient and $100M at first patient in second Phase III; management said the first $100M will “more than cover” the Phase IIb costs .
• DMD regulatory posture: Continued confidence in agreed pivotal path; will keep enrolling confirmatory to expand safety database; leadership changes at FDA not seen altering review interactions to date .
• wAMD subretinal endpoints: Aim for NI on VA and materially reduce injection burden (>50% reduction viewed as clinically meaningful), while maintaining vision .
• Device supply: Confident in ongoing access to suprachoroidal microinjectors despite partner restructuring; inventory and contractual provisions in place .

Estimates Context

• Q2 2025 results vs S&P Global consensus: revenue $21.36M vs $40.87M consensus; EPS $(1.38) vs $(0.90). 9 revenue and 10 EPS estimates contributed to consensus.* Drivers included lower YoY Zolgensma royalties ($18.4M vs $21.8M), higher R&D for pivotal programs, and higher interest expense .
  *Values retrieved from S&P Global (GetEstimates).

Where estimates may adjust:

• Revenue models likely trim near‑term royalties and service revenue cadence; expense models to reflect sustained R&D through multiple pivotal programs and higher interest expense run‑rate post‑financing .

Key Takeaways for Investors

• Near‑term catalysts: RGX‑121 PDUFA Nov 9, 2025; DR Phase IIb/III study initiation and $100M milestone from AbbVie upon first patient; wAMD subretinal pivotal enrollment completion updates ahead of 2026 topline .
• RGX‑202 remains a potential “next‑to‑market” DMD gene therapy; earlier enrollment completion (Oct’25) and commercial mfg initiation support launch readiness in 2027 pending positive data/BLA .
• Sura‑vec DR program de‑risked by durable two‑year efficacy/safety and AbbVie’s added investment/ACHIEVE trial, supporting both regulatory and commercialization narratives .
• Balance sheet stronger post $150M royalty monetization; runway into early 2027 excludes potential PRV sale and milestones, offering non‑dilutive upside optionality .
• Stock‑moving risks: execution on pivotal initiations/completions, safety consistency at higher dosing (DL4), regulatory clarity in DMD, and royalty trajectory from Zolgensma .
• Q2 miss should be weighed against tightened clinical timelines and funding visibility; trading may hinge on DR pivotal start/milestone timing and RGX‑121 FDA review progression .

SOURCES

• Q2 2025 8‑K/Press release (financials, programs, AbbVie amendment, guidance):
• Q2 2025 earnings call transcript (prepared remarks and Q&A):
• Q1 2025 8‑K/Press release (comparatives, prior guidance):
• Q4/FY 2024 8‑K/Press release (baseline, prior commentary):
• Royalty monetization financing press release:
• RGX‑121 BLA Priority Review press release:

Notes: S&P Global consensus estimates used for Street comparisons. Actuals reflect GAAP as reported. Contingent milestones and PRV monetization are excluded from runway guidance per company statements.