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uniQure N.V.: Q1 FY2026 earnings call

What management said about Q1 FY2026, from the documents filed with the SEC on 5 May 2026: press release. Guidance figures: 2 new, 3 kept. The quarter’s numbers.

Key takeaways

  • Net loss widened to $53.5 million: The company reported a net loss of $53.5 million, or $0.85 basic and diluted loss per ordinary share, compared with $43.6 million, or $0.82, a year earlier. Source
  • SG&A rose on commercial planning: SG&A was $20.1 million, against $10.9 million. The rise was mainly employee and contractor costs for staff recruited in 2025 to support AMT-130 commercial planning. Source
  • FDA will not accept external-control data: After the January 2026 Type A meeting, the FDA said it cannot agree that Phase I/II data compared with an external control are sufficient as primary evidence of effectiveness. Source
  • Progressing toward UK submission: After a constructive pre-submission meeting with the UK MHRA, the company is progressing AMT-130 toward its expected UK regulatory submission. Source
  • All 11 Fabry patients off ERT: As of February 18, 2026, all 11 dosed AMT-191 patients had been withdrawn from enzyme replacement therapy. Source
  • AMT-162 development discontinued after review: Following review of EPISOD1 data, the company discontinued AMT-162 development. Safety data continue to be collected from the five patients dosed in the trial. Source
  • Cash fell to $586.6 million: Cash, cash equivalents and current investment securities were $586.6 million at March 31, 2026, compared with $622.5 million at December 31, 2025. Source

“We believe our data continue to support the potential for AMT-130 to fundamentally change the treatment landscape for Huntington’s disease”

Matthew Kapusta, chief executive officer at uniQure

Guidance

AreaPeriodFigureAgainst last quarter
AMT-130 clinical data Sourcethird quarter of 2026four-year AMT-130 data analysisNew
AMT-130 FDA interaction Sourcesecond quarter of 2026Type B meeting scheduled for the second quarter of 2026Kept
AMT-260 clinical data SourceJune 2026first-cohort data, up to six months of follow-up, at June 18-19, 2026 conferenceKept
AMT-260 enrollment Sourcemid-2026second cohort enrollment completed in mid-2026New
Cash runway Sourceinto the second half of 2029into the second half of 2029Kept

What changed from last quarter

  • New guidance: The company set a third-quarter 2026 target for an MAA submission to the UK MHRA, following a pre-submission meeting. Source
  • Ended: The company decided to discontinue development of AMT-162 after review of EPISOD1 data. Source
  • New: In April 2026, uniQure, CSL Behring and Genezen entered into agreements on HEMGENIX supply. Source
  • New: All 11 dosed AMT-191 patients had been withdrawn from enzyme replacement therapy by February 18, 2026, up from six in the prior release. Source
  • Guidance kept: Cash runway guidance into the second half of 2029 was restated. Source

Quarterly results and costs

  • Net loss widened to $53.5 million: The company reported a net loss of $53.5 million, or $0.85 basic and diluted loss per ordinary share, compared with $43.6 million, or $0.82, a year earlier. Source
  • Revenue rose on license income: Revenue was $3.6 million, compared with $1.6 million, and the company attributed the increase to higher license revenue. Source
  • R&D spend fell $6.9 million: R&D expenses were $29.2 million, against $36.1 million. Lower contingent consideration fair value, external program spend and employee costs drove the decrease. Source
  • SG&A rose on commercial planning: SG&A was $20.1 million, against $10.9 million. The rise was mainly employee and contractor costs for staff recruited in 2025 to support AMT-130 commercial planning. Source
  • Other income fell on reagent sale: Other income was $1.6 million, against $8.3 million, as the prior period included a one-time gain on the sale of critical reagents. Source
  • Currency losses lifted non-operating expense: Non-operating items, net, were an expense of $7.3 million, against $3.8 million, mainly from higher net foreign currency losses. Source

AMT-130 regulatory path

  • FDA will not accept external-control data: After the January 2026 Type A meeting, the FDA said it cannot agree that Phase I/II data compared with an external control are sufficient as primary evidence of effectiveness. Source
  • Type B meeting granted for Q2: The company has been granted a Type B meeting with the FDA to discuss a potential new trial design and to seek feedback on the statistical analysis plan for four-year data. Source
  • Progressing toward UK submission: After a constructive pre-submission meeting with the UK MHRA, the company is progressing AMT-130 toward its expected UK regulatory submission. Source

“following a constructive interaction with the MHRA, we are preparing to submit an MAA in the third quarter”

Matthew Kapusta, chief executive officer at uniQure

AMT-260 epilepsy program

  • Enrollment on track in epilepsy program: Management said enrollment in the AMT-260 temporal lobe epilepsy program is on track, with a first-cohort update to be presented at a conference. Source
  • First dose cohort completed in 2025: Enrollment in the first dose cohort of six patients in the Phase I/IIa study was completed during 2025. Source
  • Second dose cohort enrolling: Enrollment in a second dose cohort, expected to include an additional six patients, is ongoing. Source

Fabry and ALS programs

  • All 11 Fabry patients off ERT: As of February 18, 2026, all 11 dosed AMT-191 patients had been withdrawn from enzyme replacement therapy. Source
  • Enzyme activity increases remained durable: Dose-dependent increases in α-Gal A activity were durable across follow-up periods of four months to more than one year, while lyso-Gb3 levels stayed stable. Source
  • Fabry dosing paused over liver events: Additional dosing in the mid- and high-dose cohorts remains paused pending review of Grade 3 liver enzyme elevations, confirmed as dose-limiting toxicities. Source
  • AMT-162 development discontinued after review: Following review of EPISOD1 data, the company discontinued AMT-162 development. Safety data continue to be collected from the five patients dosed in the trial. Source

“With these important milestones ahead, we remain committed to advancing our programs with urgency while maintaining disciplined capital allocation to drive long-term shareholder value”

Matthew Kapusta, chief executive officer at uniQure

Cash and balance sheet

  • Cash fell to $586.6 million: Cash, cash equivalents and current investment securities were $586.6 million at March 31, 2026, compared with $622.5 million at December 31, 2025. Source
  • Runway into second half of 2029: Management expects cash, cash equivalents and investment securities to fund operations into the second half of 2029. Source
  • Warrant liability produced a gain: Non-operating items included a $3.8 million gain from changes in the fair value of the pre-funded warrant liability. Source

HEMGENIX supply agreements

  • HEMGENIX supply agreements signed: In April 2026, the company entered agreements with CSL Behring and Genezen that end its remaining HEMGENIX supply after mid-2026 batch delivery. Source
  • CSL now contracts directly with Genezen: CSL Behring entered a direct relationship with Genezen as the HEMGENIX supplier. The company said royalties and milestones are unaffected. Source
  • HEMGENIX supply costs fell: Other expense was $1.5 million, against $2.0 million, with a $0.3 million decrease in costs for supplying Hemgenix to CSL Behring. Source

Coming up, as management said on 5 May 2026

  • Type B meeting with FDA in Q2: The company plans to discuss key elements of a potential new clinical trial design at the FDA Type B meeting. Source
  • AMT-260 first-cohort data in June: First-cohort AMT-260 data are to be provided at the Epilepsy Foundation Pipeline Conference on June 18-19, 2026. Source
  • Four-year AMT-130 data in Q3: Four-year AMT-130 data analysis is expected in the third quarter of 2026. Source
  • UK MAA submission in Q3: The company expects to submit an MAA for AMT-130 to the UK MHRA in the third quarter of 2026. Source
  • Second cohort enrollment completes mid-2026: Enrollment in the second AMT-260 cohort is expected to finish in mid-2026. Source
  • HEMGENIX supply ends mid-2026: Remaining HEMGENIX supply and minimum purchase commitments are expected to end after batch delivery in mid-2026. Source
  • RBC healthcare conference in May: Management plans to attend the 2026 RBC Capital Markets Global Healthcare Conference in New York. Source

Figures management reported

FigureReported
R&D expenses (three months ended March 31, 2026) Source$29.2 million
AMT-191 patients withdrawn from ERT Source11 of 11 dosed patients
AMT-260 first-cohort data timing SourceJune 18-19, 2026 conference

Risks management discussed

  • Regulatory: The FDA said it cannot accept Phase I/II data compared with an external control as primary evidence of effectiveness for AMT-130. Source
  • Operational: Dosing in the Fabry mid- and high-dose cohorts is paused after Grade 3 liver enzyme elevations confirmed as dose-limiting toxicities. Source
  • Operational: AMT-162 development was discontinued after a serious adverse event determined to be a dose-limiting toxicity, following review of EPISOD1 data. Source
  • Financial: Non-operating items included higher net foreign currency losses, which increased non-operating expense by $3.5 million. Source

Earlier plans management reported on

  • On track: Management said the UK MAA is on track for the third quarter of 2026, following a pre-submission meeting with the MHRA. Source
  • On track: Management said enrollment in the AMT-260 epilepsy program is on track. Source
  • Not stated: The prior release said a Type B meeting would be requested in the second quarter of 2026; this release says the meeting has been granted. Source

Other quarters

Summarised with AI from the documents the company filed with the SEC. Every point is checked against the filing before it is shown: management’s own words word for word, figures against the line or table row they come from. A point that fails the check is left out. Source opens the filing at that line.