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uniQure N.V. (QURE)·Q4 2024 Earnings Summary

Executive Summary

  • uniQure reported FY 2024 revenue of $27.1M (vs. $15.8M in 2023) and a net loss of $239.6M (vs. $308.5M), driven by higher license and collaboration revenue and materially lower operating expenses following a restructuring and Lexington facility sale; cash and investments were $367.5M at 12/31/24 (pro forma ~$448M after Q1’25 offering), with runway into 2H 2027 .
  • For Q4 2024, quarterly revenue implied by company-reported figures was approximately $5.22M; net loss implied approximately $73.26M; quarterly EPS was not disclosed (only FY EPS of $(4.92)) .
  • The quarter’s key catalyst: FDA alignment on an Accelerated Approval pathway for AMT-130 (cUHDRS as intermediate clinical endpoint and CSF NfL as supportive evidence), with BLA-readiness and Type B meetings underway in 1H’25; initial third-cohort safety update expected in 2Q’25 and an AMT-130 data update in 3Q’25 supporting a potential BLA submission .
  • Strategic actions reduced annual cash burn by ~$70M and retired $50M of debt; a Q1’25 equity raise further extended runway to support BLA submission and potential U.S. launch of AMT-130 .

What Went Well and What Went Wrong

  • What Went Well

    • FDA alignment on key elements of Accelerated Approval for AMT-130 (natural history external control acceptable; cUHDRS as intermediate endpoint; CSF NfL supportive), enabling BLA-readiness activities and defined FDA touchpoints in 1H’25 .
    • Cost discipline and restructuring: R&D down to $143.8M (from $214.9M) and SG&A down to $52.7M (from $74.6M) for FY 2024; cash runway extended into 2H 2027, bolstered by Q1’25 equity raise .
    • Pipeline execution: AMT-260 first patient dosed (mTLE), protocol broadened to accelerate enrollment; AMT-162 (SOD1-ALS) moved into second cohort with positive IDMC; AMT-191 (Fabry) completed first-cohort enrollment with favorable IDMC and H2’25 data timing .
    • Management quote: “This past year was transformative… securing alignment with the FDA on key elements of the Accelerated Approval pathway… We have initiated BLA-readiness activities” — CEO Matt Kapusta .

  • What Went Wrong

    • Shareholders’ equity turned negative by year-end 2024 (−$6.75M), reflecting accumulated losses and royalty liability accretion despite reduced operating expenses .
    • Other non-operating items, net expense increased sharply to $52.8M for FY 2024 (vs. $23.7M in 2023), primarily from higher non-cash interest expense tied to the 2023 royalty agreement and FX losses .
    • Contract manufacturing revenue for HEMGENIX shifted to be recorded net within other expenses post-Lexington sale, reducing reported revenue line item and complicating comparability; cost of contract manufacturing moved net as well .

Financial Results

  • Quarterly snapshot (USD, millions except per-share)
MetricQ2 2024Q3 2024Q4 2024
Revenue ($M)$11.13 $2.29 ≈$5.22 (derived: FY $27.12 less Q1 $8.49, Q2 $11.13, Q3 $2.29)
Net Loss ($M)$(56.30) $(44.38) ≈$(73.26) (derived: FY $(239.56) less Q1 $(65.62), Q2 $(56.30), Q3 $(44.38))
EPS ($)$(1.16) $(0.91) N/A (Company disclosed only FY EPS $(4.92))
Cash + Investments (End of Period, $M)$524.4 $435.2 $367.5 (pro forma ~$448 incl. Q1’25 raise)
  • Annual revenue mix (USD, millions)
MetricFY 2022FY 2023FY 2024
License Revenues$100.00 $2.76 $10.13
Contract Manufacturing Revenues$1.72 $10.84 $6.11
Collaboration Revenues$4.77 $2.25 $10.87
Total Revenues$106.48 $15.84 $27.12
  • KPIs and balance sheet indicators
KPIQ2 2024Q3 2024Q4 2024
Total Cash, Cash Equivalents & Investment Securities ($M)$524.4 $435.2 $367.5 (pro forma ~$448 incl. $80.7M raise)
Long-term Debt ($M)$102.51 $51.11 $51.32
Royalty Financing Liability ($M)$415.94 $426.69 $434.93
Shareholders’ Equity ($M)$97.11 $55.82 $(6.75)

Notes:

  • Q4 2024 quarterly revenue and net loss are arithmetic derivations from company-reported FY and Q1–Q3 figures; EPS was not reported quarterly for Q4 (only FY) .
  • Company attributes cash reduction partly to non-recurring outflows including $53M debt retirement, $31.5M milestones, $12.0M Lexington-related payments, and $4.7M severance .

Guidance Changes

MetricPeriodPrevious GuidanceCurrent GuidanceChange
Cash runwayMulti-year“Through end of 2027” (as of 9/30/24) “Through 2H 2027” (as of 12/31/24); pro forma ~$448M incl. Q1’25 raise Maintained (terminology refined; runway intact)
AMT-130 regulatory path1H 2025Type B meeting late Nov 2024; additional FDA meetings in 1H’25 FDA alignment on Accelerated Approval criteria; Type B CMC meeting in Q1’25; Type B stats plan meeting in Q2’25; regulatory update incl. BLA timing in Q2’25 Raised clarity; de-risked path
AMT-130 Cohort 34Q 2024–1Q 2025Enrollment expected complete in 4Q’24 Enrollment of 12 patients completed in Feb 2025; initial safety update expected in 2Q’25 Slightly delayed but completed; near-term safety update
AMT-130 data update3Q 2025Mid-2025 interim update 3Q 2025 data (incl. 3-year follow-up on 24 treated patients) to support potential BLA Timing specified; scope expanded
AMT-260 (mTLE)2024–2026Observational phase enrollment; site activations ongoing First patient dosed (Nov 2024); FDA-approved protocol broadening to accelerate enrollment; initial data 1H 2026 Progressed; enrollment acceleration
AMT-162 (SOD1-ALS)2024–2026First patient dosed (Oct 2024) IDMC favorable on Cohort 1; Cohort 2 enrolling; initial data 1H 2026 Advanced; data timing set
AMT-191 (Fabry)2024–2025First patient dosed (Aug 2024) First cohort enrollment completed; IDMC favorable; initial data H2 2025 Advanced; data timing set

Earnings Call Themes & Trends

TopicPrevious Mentions (Q2 2024)Previous Mentions (Q3 2024)Current Period (Q4 2024)Trend
AMT-130 FDA/regulatoryRMAT granted; planning FDA interactions 2H’24 Type B meeting scheduled late Nov; discussing expedited pathway FDA alignment on Accelerated Approval elements; Type B CMC/statistics meetings in 1H’25 Increasing regulatory clarity
Cost actions / restructuring65% headcount reduction; ~$75M cash burn reduction; Lexington sale Reiterated restructuring; further expense decline expected in 1H’25 ~$70M lower recurring burn; cash runway into 2H’27; debt retired; follow-on offering Execution on cost/financing
Pipeline executionScreenings for AMT-260/191/162; timelines set Dosed first patients in AMT-162 & AMT-191; mTLE observational phase begun AMT-260 first patient dosed; protocol broadened; AMT-162 IDMC favorable & Cohort 2; AMT-191 Cohort 1 complete Advancing to PoC milestones
Manufacturing/HEMGENIX accountingFacility sale to Genezen; contract mfg costs elevated in Q2 Costs recognized net post-sale; “other expenses” classification Contract mfg revenue/costs recorded net within other expenses post-sale Cleaner OpEx, less revenue recognition
Balance sheet / runway$524.4M cash & investments; runway to end 2027 $435.2M; runway through end 2027 $367.5M (pro forma ~$448M); runway into 2H 2027 Runway maintained via financing

Management Commentary

  • “This past year was transformative… securing alignment with the FDA on key elements of the Accelerated Approval pathway… We have initiated BLA-readiness activities and look forward to further engagement with the FDA throughout the first half of 2025.” — Matt Kapusta, CEO .
  • “Operationally, we took decisive steps… sale of our Lexington manufacturing facility and a company-wide restructuring that significantly reduced our cash burn… enabling… potential approval and commercial launch of AMT-130.” — Matt Kapusta, CEO .
  • “We are very pleased to reach agreement with the FDA on core components of an Accelerated Approval pathway for AMT-130… an important milestone… putting us on the most rapid and efficient pathway…” — Walid Abi‑Saab, M.D., CMO .

Q&A Highlights

  • No Q4 2024 earnings call transcript was available in the document set; the company did host a Dec 10, 2024 investor call regarding FDA alignment, but a transcript was not available for review here .

Estimates Context

  • Wall Street consensus (S&P Global) for Q4 2024 EPS and revenue was unavailable via the S&P Global tool at this time due to access limits; therefore we cannot assess beat/miss vs. consensus for Q4 2024. We would normally anchor to S&P Global consensus for this comparison (consensus data unavailable).

Key Takeaways for Investors

  • Regulatory de‑risking for AMT‑130: FDA alignment on Accelerated Approval with cUHDRS and CSF NfL as acceptable endpoints materially increases odds of a 2025 BLA submission; watch for 2Q’25 regulatory update and 3Q’25 data package disclosure .
  • Cash runway intact into 2H 2027 post-restructuring, facility sale, debt retirement and Q1’25 equity raise; sufficient capital to reach major regulatory and clinical catalysts (including potential U.S. launch preparations) .
  • Operating efficiency improving: steep YoY reductions in R&D and SG&A support narrowing annual losses; however, non-cash interest from royalty financing elevated non‑operating expense — monitor the royalty liability accretion and FX impacts .
  • Pipeline momentum across three additional programs (mTLE, SOD1‑ALS, Fabry) with initial data readouts slated H2’25–H1’26 — potential multi‑asset value inflections beyond AMT‑130 .
  • Accounting shift post‑Lexington sale reduces reported revenue from contract manufacturing (now recorded net in other expenses), but improves cost structure; focus on license/collaboration revenue for top‑line trajectory .
  • Balance sheet risk: shareholders’ equity turned negative at year‑end; continued reliance on external financing and royalty structure warrants scrutiny as the company approaches commercialization .
  • Near‑term trading catalysts: 2Q’25 FDA CMC/statistics meetings and regulatory update; 2Q’25 Cohort 3 safety readout; 3Q’25 AMT‑130 data supporting BLA — outcomes could drive significant stock volatility given binary regulatory and clinical events .

Appendix: Source Documents Reviewed

  • Q4 2024 8‑K (Press release Exhibit 99.1 embedded): company results and financial statements ; standalone press release version –.
  • Q3 2024 8‑K (Press release Exhibit 99.1): quarterly results and pipeline updates –.
  • Q2 2024 8‑K (Press release Exhibit 99.1): quarterly results and restructuring announcement –.
  • Other relevant Q4 2024 press releases: FDA alignment on Accelerated Approval (Dec 10, 2024) –; first patient dosed in AMT‑260 (Nov 21, 2024) ; first patient dosed in AMT‑162 (Oct 15, 2024) .

Disclosures:

  • Quarterly Q4 revenue and net loss are implied from company-reported FY and Q1–Q3 figures; the company did not disclose Q4 EPS (only FY EPS). Consensus estimates via S&P Global were unavailable at time of analysis.