RGNX
REGENXBIO Inc.14.11
+0.28+2.02%
Dec 16, 4:00:00 PM EST
Earnings Call Transcripts
This Quarter (Q1 '26)
No earnings call transcript available yet
Last Quarter (Q4 '25)
FY Q4 '25
Q&A affirms DMD approval path
Q&A bolstered confidence in RGX-202's accelerated approval path for Duchenne, as management detailed prospectively FDA-reviewed protocol using external controls, functional improvements in older patients trumping placebo ethics concerns, and zero liver injuries or thrombocytopenia in Phase 1/2 versus peers' 40% rates. Analysts pressed on FDA scrutiny and confirmatory study; team noted rapid enrollment expands safety database to ~50 patients at filing. DR NAVIGATE affirmed dose level 3 for its 50% two-step DRSS gains and zero intraocular inflammation. MPS responses addressed CRL sampling issues and heparan sulfate data, eyeing Denali precedent. Safety differentiation shines. Investors eye Q2 DMD topline and pre-BLA feedback.
Key Stats
Market Cap
714.29MP/E (TTM)
-Basic EPS (TTM)
-3.46Dividend Yield
0%Recent Filings
8-K
Q4 revenues jump; cash to 2027
REGENXBIO reported Q4 and full-year 2025 results on March 5, 2026, with revenues up to $30.3M and $170.4M from $21.2M and $83.3M last year, fueled by Nippon Shinyaku collaboration. Cash stands at $240.9M, funding operations into early 2027. Pipeline advances: RGX-202 Duchenne topline data early Q2 2026; sura-vec wet AMD pivotal results Q4 2026. Cash burn quickens.
10-K
FY2025 results
REGENXBIO posted FY2025 revenues of $170M, up sharply from $83M in 2024, fueled by $73M from the new Nippon Shinyaku deal and steady Zolgensma/Itvisma royalties hitting $83M on $1.23B Novartis sales—Itvisma launched late Q4. Yet R&D climbed to $228M from manufacturing and trials for ABBV-RGX-314, RGX-202, RGX-121, while interest on royalty debt quadrupled to $45M, driving net loss to $194M versus $227M prior year. Cash burned $124M in operations but $241M liquidity lasts into early 2027. Q4 saw Itvisma royalties kick in; AFFINITY DUCHENNE pivotal enrollment wrapped. Pivotal readouts loom 2026. Clinical holds on RGX-121/111 threaten pipeline momentum.
8-K
FDA rejects RGX-121 BLA
REGENXBIO received an FDA Complete Response Letter on February 7, 2026, rejecting its BLA for RGX-121 gene therapy targeting MPS II due to issues with study eligibility, natural history controls, and CSF HS D2S6 surrogate endpoint. The company plans a Type A meeting and BLA resubmission with added data. Paths forward prove challenging in ultra-rare disease. Delays hit hard.
8-K
FDA halts MPS gene trials
REGENXBIO disclosed FDA clinical holds on its Phase I/II RGX-111 trial for MPS I and Phase I/II/III RGX-121 trial for MPS II, triggered by a single neoplasm case in an asymptomatic RGX-111 patient four years post-dosing. Holds cite product similarities despite clean safety in nine other RGX-111 and 32 RGX-121 patients. Causality remains unestablished. Investigation ongoing.
8-K
Q3 results, pipeline advances
REGENXBIO reported Q3 2025 results, with cash at $302M funding operations into early 2027. Revenues rose to $29.7M from $24.2M year-over-year, yet net loss widened slightly to $61.9M. Pipeline advanced: RGX-202 enrollment complete, topline data early Q2 2026; RGX-121 PDUFA February 8, 2026. Momentum builds across Duchenne, MPS II, retinal programs.
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