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uniQure N.V. (QURE)·Q1 2025 Earnings Summary

Executive Summary

  • Q1 2025 revenue was $1.57M, down sharply year-over-year due to lower collaboration and the shift of HEMGENIX contract manufacturing to “other expenses” post-Lexington divestiture; revenue materially missed Wall Street consensus of ~$6.39M. EPS of -$0.82 was better than the -$1.01 consensus, aided by lower R&D/SG&A and a $6.0M one-time reagent gain; net loss improved to -$43.6M from -$65.6M YoY . Revenue consensus and EPS consensus values marked with an asterisk are from S&P Global.*
  • The FDA granted Breakthrough Therapy designation to AMT-130 in April, and management held Type B FDA meetings on CMC and the statistical analysis plan; a regulatory update (including BLA timing) is expected after receipt of formal minutes in Q2 2025 .
  • Initial safety data for the third AMT-130 cohort (with prophylactic immunosuppression) showed no treatment‑related SAEs; management favors a short, 2‑week steroid regimen going forward, with pivotal data from the Phase I/II program expected in Q3 2025 (3‑year follow-up on 24 treated patients) .
  • Cash, cash equivalents and investment securities were $409.0M at March 31, 2025; management reiterated runway into 2H 2027. A follow-on equity offering added ~$80.5M net in Q1, supporting BLA readiness and commercialization preparations for AMT‑130 .

What Went Well and What Went Wrong

  • What Went Well

    • FDA Breakthrough Therapy designation for AMT-130, reinforcing the accelerated pathway alignment and clinical promise. “Breakthrough Therapy designation…underscores the FDA’s continued engagement with AMT‑130” (CEO) .
    • Cohort 3 immunosuppression learnings: AMT‑130 generally well-tolerated; no treatment-related SAEs; management proposes optimized perioperative steroids only. “We believe a short 2‑week course of steroids represents an appropriate…strategy” (CMO) .
    • Balance sheet strengthened: $409.0M cash and investments, runway into 2H 2027, and $80.5M net proceeds from Q1 follow-on; reduced expense structure after Lexington sale and restructuring .

  • What Went Wrong

    • Revenue fell to $1.6M vs $8.5M YoY on lower collaboration and the reclassification of contract manufacturing (post-Lexington sale) to “other expenses,” driving a significant top-line miss vs consensus (~$6.39M). Collaboration revenue -$3.3M YoY; contract manufacturing -$4.0M YoY .
    • Dependence on non-operating items: $8.3M other income driven by a one-time $6.0M reagent sale; ongoing amortization of the Genezen favorable purchase right lifted “other expense” to $2.0M .
    • Continued royalty financing and debt load: liability from royalty financing rose to $446.7M; long-term debt ~$51.6M at March 31, 2025, remaining a structural headwind as the company advances BLA and commercialization .

Financial Results

Quarterly trend (oldest → newest)

MetricQ3 2024Q4 2024Q1 2025
Revenue ($USD)$2.287M $5.221M*$1.567M
Net Income ($USD)-$44.378M -$73.261M*-$43.637M
Diluted EPS ($USD)-$0.91 -$1.50*-$0.82

Values with an asterisk are from S&P Global.*

Q1 2025 vs prior year and estimates

MetricQ1 2024Q1 2025 (Actual)Q1 2025 Consensus
Revenue ($USD)$8.485M $1.567M $6.389M*
EPS ($USD)-$1.36 -$0.82 -$1.01*
# of Estimates (Revenue/EPS)——10 / 10*

Values with an asterisk are from S&P Global.*

Operating KPIs (Q1 2025 vs Q1 2024)

MetricQ1 2024Q1 2025
R&D Expenses ($USD)$40.7M $36.1M
SG&A Expenses ($USD)$13.9M $10.9M
Cost of Contract Manufacturing ($USD)$9.076M $0
Other Income ($USD)$1.4M $8.3M
Other Expense ($USD)$0.2M $2.0M
Weighted Avg. Shares48,384,510 53,110,580

Liquidity

MetricSep 30, 2024Dec 31, 2024Mar 31, 2025
Cash, Cash Equivalents & Investment Securities ($USD)$435.2M $367.5M $409.0M

Guidance Changes

MetricPeriodPrevious GuidanceCurrent GuidanceChange
Cash RunwayThrough 2H 2027“Through 2H 2027” (Feb 27, 2025) “Through 2H 2027” (May 9, 2025) Maintained
AMT‑130 Regulatory Update (incl. BLA timing)Q2 2025“Provide regulatory update in Q2 2025 after Type B meetings” “Regulatory update in Q2 2025 after receipt of formal minutes” Maintained/Clarified
AMT‑130 Clinical Data UpdateQ3 2025“Q3 2025 update; 3‑year follow‑up on 24 treated patients” (Feb) “Q3 2025 update; 3‑year follow‑up on 24 treated patients” (May) Maintained
AMT‑260 initial case study (first patient)May 29, 2025Not previously set as a case study date“Case study incl. initial safety/exploratory efficacy at Epilepsy Symposium May 29, 2025” New milestone
AMT‑162 (SOD1‑ALS)2025“Proceed to second cohort” “Completed second cohort; start third in Q3 2025” Raised execution

Earnings Call Themes & Trends

TopicPrevious Mentions (Q3 2024 and FY/Q4 2024)Current Period (Q1 2025)Trend
Regulatory pathway for AMT‑130RMAT, planning Type B meeting, accelerated path discussions Breakthrough Therapy designation; Type B meetings on CMC and SAP; regulatory update pending minutes in Q2 Strengthening/advancing
External control & endpointUse of cUHDRS and natural history datasets (TRACK/PREDICT/Enroll) under RMAT alignment Confirmed focus on 3‑year primary analysis; natural history comparator selection aligned; top-line Q3 to follow SAP Converging on plan
Immunosuppression strategy (Cohort 3)Cohort design initiated No treatment‑related SAEs; favor 2‑week perioperative steroids only Optimization achieved
CMC & manufacturing readinessLeveraging HEMGENIX experience; post-Lexington sale CMC validation preparation ongoing; timeline details to come in Q2 update On track
Commercial planningCapital preservation, restructuring to support milestones Preparing for US launch; 50+ centers can perform MRI‑guided administration; ex‑US path to be engaged Readiness building
Pipeline execution (AMT‑260/191/162)Initiations/dosing and protocol amendments AMT‑260 case study May 29; AMT‑191 treated 4 pts; AMT‑162 2nd cohort complete; 3rd starts Q3 Advancing

Management Commentary

  • “The FDA granted breakthrough therapy designation…we look forward to providing a detailed regulatory update later this quarter after we receive formal meeting minutes.” (CEO) .
  • “AMT‑130 continues to be generally well tolerated at both doses with no treatment‑related serious adverse events…a short 2‑week course of steroids represents an appropriate…strategy.” (CMO) .
  • “Cash…totaled $409 million…This strong balance sheet provides uniQure with the resources…including the planned U.S. launch of AMT‑130. We expect…sufficient to fund operations into the second half of 2027.” (CFO) .

Q&A Highlights

  • Regulatory continuity under new CBER leadership: management emphasized reliance on 3+ years of clinical outcomes (not surrogate biomarkers) and confidence in their dataset .
  • Statistical analysis plan and external control: primary 3‑year analysis with propensity methods; comparator finalized with FDA; top-line Q3 data aligned to SAP .
  • Immunosuppression protocol: triple regimen observed 3 SAEs related to immunosuppression (mania, infection, fever), all resolved; moving to short steroid course .
  • CMC timeline: leveraging HEMGENIX experience and same site/personnel; further details to be provided in Q2 regulatory update .
  • Commercial rollout capacity: >50 US sites could administer MRI‑guided intracranial therapy; focus on early-manifest HD patients initially .

Estimates Context

  • Q1 2025: Revenue $1.567M vs consensus ~$6.389M (miss); EPS -$0.82 vs consensus -$1.01 (beat). Ten estimates for both revenue and EPS.* Actuals per press release .
  • Q4 2024: Revenue ~$5.221M vs consensus ~$19.097M (miss); EPS -$1.49 vs consensus -$0.54 (miss). Eleven revenue and eight EPS estimates.*
    Values marked with an asterisk are from S&P Global.
MetricQ4 2024 ConsensusQ4 2024 ActualQ1 2025 ConsensusQ1 2025 Actual
Revenue ($USD)$19.097M*$5.221M*$6.389M*$1.567M
EPS ($USD)-$0.54*-$1.49*-$1.01*-$0.82
# of Estimates (Rev/EPS)11 / 8*—10 / 10*—

Values with an asterisk are from S&P Global.*

Implications: Street models likely need to lower near-term revenue and adjust opex/other income assumptions; EPS resilience this quarter (vs consensus) tied to one-time gains and lower operating expenses .

Key Takeaways for Investors

  • Near-term catalyst stack: Q2 2025 regulatory update (BLA timing) and Q3 2025 clinical update (3‑year follow-up on 24 treated AMT‑130 patients) should drive narrative and potential re-rating .
  • The quarter’s top-line shortfall was known structural (collaboration and HEMGENIX manufacturing reclassification) rather than a pipeline setback; monitor future collaboration/licensing inflows .
  • EPS beat vs consensus was quality-mixed (lower opex plus $6.0M one-time reagent sale); durability hinges on opex discipline and non-operating headwinds (royalty financing) .
  • Immunosuppression optimization reduces procedural risk; focus shifts to durability and magnitude of cUHDRS benefit at 3 years—key to accelerated approval case .
  • Liquidity is ample ($409.0M; runway into 2H 2027), enabling BLA and commercial build-out; follow-on offering de‑risked capital needs near term .
  • Commercial readiness: >50 centers capable of MRI-guided administration; potential first-in-class, best‑in‑class positioning in a large genetic patient pool may attract partners/ex‑US strategies .
  • Trading lens: stock sensitivity to Q2 regulatory clarity and Q3 clinical data is high; downside protection depends on perceived CMC readiness and consistency of 3‑year cUHDRS effect vs external control .
Notes: 
- Values marked with an asterisk (*) are retrieved from S&P Global.