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uniQure N.V. (QURE)·Q2 2025 Earnings Summary

Executive Summary

  • Q2 2025 revenue of $5.26M declined year over year and improved sequentially; EPS loss narrowed to $0.69, with management highlighting strong regulatory progress toward AMT-130’s accelerated approval pathway .
  • Versus Wall Street consensus, revenue modestly missed ($5.26M actual vs $5.82M consensus) while EPS was a clear beat (-$0.69 actual vs -$0.89 consensus). Bolded for significance: EPS beat; revenue miss. Values retrieved from S&P Global* .
  • Guidance reinforced: topline three-year AMT-130 data in September 2025, pre-BLA meeting in Q4 2025, and BLA submission in Q1 2026; cash runway guided into 2H 2027, supporting U.S. launch preparations .
  • Stock catalysts: September AMT-130 three-year data (cUHDRS vs Enroll-HD external control), Q4 pre-BLA feedback, and continued clarity on CMC and PPQ campaign; early AMT-260 case study (92% seizure reduction) and initial AMT-191 Fabry data add pipeline breadth .

What Went Well and What Went Wrong

What Went Well

  • Regulatory alignment: FDA supported accelerated approval framework for AMT-130, with primary efficacy analysis on three-year cUHDRS and propensity score-matched external control; final SAP submitted and PPQ campaign initiated .
  • Commercial readiness: appointment of Chief Customer and Strategy Officer and disciplined launch planning; ~50–55 U.S. centers capable of the neurosurgical procedure for administration .
  • Pipeline signals: AMT-260 case study showed 92% seizure reduction over five months with no serious safety events; AMT-191 initial cohort data showed supraphysiological α-Gal A expression and ERT withdrawal .

Management quotes:

  • “AMT-130 is well-positioned to potentially become the first disease-modifying therapy for people living with Huntington’s disease.” — CEO Matt Kapusta .
  • “The FDA’s stated preference was for propensity score matching... we decided to submit the SAP aligned with the FDA preference.” — CMO Walid Abi‑Saab .

What Went Wrong

  • Revenue decline YoY driven by lower collaboration and contract manufacturing revenues post-Lexington divestment; contract manufacturing now recorded net within other expenses .
  • Non-operating sensitivity: Q2 had favorable FX boosting non-operating items; management noted changes in interest income and expense dynamics year over year .
  • Trial recruitment challenges for AMT-260 acknowledged; additional sites activated to counter enrollment headwinds .

Financial Results

Quarterly Financials (sequential comparison)

MetricQ1 2025Q2 2025
Total revenues ($USD Millions)$1.57 $5.26
Net loss ($USD Millions)$43.64 $37.72
Diluted EPS ($USD)$(0.82) $(0.69)
R&D expenses ($USD Millions)$36.14 $35.38
SG&A expenses ($USD Millions)$10.91 $13.50
Other non-operating items, net ($USD Millions)$(3.81) $6.57
Cash, cash equivalents & current investment securities ($USD Millions)$409.0 $377.0
Weighted avg. shares (Millions)53.11 54.81

Year-over-Year Snapshot (Q2)

MetricQ2 2024Q2 2025
Total revenues ($USD Millions)$11.13 $5.26
Diluted EPS ($USD)$(1.16) $(0.69)

Estimates vs Actuals (S&P Global consensus*)

MetricQ4 2024Q1 2025Q2 2025
Revenue Consensus Mean ($USD Millions)$19.10*$6.39*$5.82*
Revenue Actual ($USD Millions)$5.22*$1.57 $5.26
Primary EPS Consensus Mean ($USD)$(0.54)*$(1.01)*$(0.89)*
Primary EPS Actual ($USD)$(1.49)*$(0.82) $(0.69)

Values retrieved from S&P Global*.

KPIs and Operating Drivers

KPIQ1 2025Q2 2025
Contract manufacturing revenues recorded net (post-Lexington divestment)Yes Yes
PPQ campaign statusPlanned Q3 initiation Initiated
Final SAP submissionPlanned Q2 Submitted
Cash runwayInto 2H 2027 Into 2H 2027

Guidance Changes

MetricPeriodPrevious GuidanceCurrent GuidanceChange
AMT-130 topline three-year dataSeptember 2025“Q3 2025” planned September 2025 Maintained
Pre-BLA meeting (AMT-130)Q4 2025Q4 2025 planned Q4 2025 Maintained
BLA submission (AMT-130)Q1 2026Q1 2026 planned Q1 2026 Maintained
PPQ campaign2H 2025Initiate PPQ Q3 2025 PPQ initiated; complete by Q4 2025 Clarified/On track
Fourth cohort (lower striatal volume)Q3–Q4 2025N/A previouslyInitiate Q3; full enrollment by Q4 New
Cash runwayThrough 2H 2027Into 2H 2027 Into 2H 2027 Maintained

Earnings Call Themes & Trends

TopicPrevious Mentions (Q4 2024, Q1 2025)Current Period (Q2 2025)Trend
Regulatory path (accelerated approval)RMAT/accelerated pathway alignment; external controls acceptable Final SAP submitted; propensity score-matched primary; PPQ initiated; pre-BLA in Q4 Strengthening alignment, execution underway
External control methodology (Enroll-HD)Enroll-HD accepted; propensity-weighted primary contemplated FDA preference for propensity-matched; sensitivity includes weighted; results expected similar Method refined per FDA preference
Commercial planning & capacityStreamlining ops; planning for potential launch Center-of-excellence strategy; 50–55 capable sites; bed capacity not a constraint Increasing confidence in launch feasibility
AMT-260 (MTLE) executionProtocol broadened to aid enrollment 92% seizure reduction case; 14 sites; recruitment challenging but improving Positive signal; building enrollment
AMT-191 (Fabry)Cohort enrollment completed; data 2H 2025 Initial cohort data: supraphysiological α-Gal A; ERT withdrawal (Sept) Early efficacy signal; safety monitored
NfL biomarkerFDA said NfL can be supportive No specific FDA thresholds; 3-year comparator lacking; expect supportive role Supportive, interpretive constraints at 36 months

Management Commentary

  • “Our momentum is strong across clinical advancement, regulatory alignment, BLA readiness, and commercial launch preparation... we believe the second half of 2025 is shaping up to be an eventful period for uniQure.” — CEO Matt Kapusta .
  • “The FDA agreed that cUHDRS measured against a propensity score-adjusted external control constructed using Enroll-HD could serve as the registrational endpoint for an accelerated approval BLA.” — CMO Walid Abi‑Saab .
  • “We very much look forward to presenting top-line pivotal data anticipated in September, which we expect will support a planned BLA submission in the first quarter of 2026.” — CEO Matt Kapusta .

Q&A Highlights

  • Regulatory threshold: FDA did not request a minimum clinical effect; company expects substantial evidence supporting accelerated approval from three-year data .
  • Methodology: SAP aligns with FDA preference for propensity score matching; sensitivity analyses include propensity weighting; estimates across methods expected to be similar .
  • Launch approach: Center-of-excellence rollout; AMT-130 local administration minimizes systemic exposure; no meaningful bed-capacity constraints expected .
  • Label considerations: Fourth cohort aims to generate safety data for lower striatal volumes; label specifics to be determined during review .
  • Market sizing: ~35,000 diagnosed patients in the U.S.; many thousands likely eligible; 50–55 sites capable of procedure .

Estimates Context

  • Q2 2025: Revenue $5.26M vs $5.82M consensus → miss; EPS $(0.69) vs $(0.89) consensus → beat. Values retrieved from S&P Global* .
  • Sequential improvement vs Q1 2025 on revenues and EPS; operating loss narrowed, aided by favorable FX in non-operating items .
  • Street may adjust models to reflect lower collaboration/contract manufacturing contributions post-Lexington divestment, offset by license revenue variability and non-operating FX sensitivity .

Key Takeaways for Investors

  • September AMT-130 three-year data vs Enroll-HD (propensity-matched) is the pivotal near-term catalyst; strong execution on PPQ and CMC de-risks the BLA path .
  • Regulatory posture is constructive: FDA alignment on endpoints and controls, with supportive stance on non-RCT evidence in severe unmet need indications .
  • Commercial feasibility looks solid: 50–55 capable U.S. centers; center-of-excellence launch can scale with demand and safety oversight .
  • Pipeline breadth underpins medium-term optionality: AMT-260 early efficacy signal in MTLE; AMT-191 Fabry cohort data demonstrating high α-Gal A levels and ERT withdrawal .
  • Financial runway into 2H 2027 provides capacity to execute on regulatory, commercial, and development plans without near-term financing pressure from operations; note September offering further strengthened balance sheet (post-Q2 event) .
  • Model updates: Expect consensus revisions for revenue mix and EPS given lower contract manufacturing visibility and FX-driven non-op volatility; regulatory milestones may drive multiple expansion contingent on September readout .
  • Risk monitor: Enrollment pace (AMT-260), external control comparability, and FDA feedback at pre-BLA; watch for any divergence between matched vs weighted analyses and interpretive challenges for 36-month NfL .