uniQure N.V.: Q2 FY2026 earnings call
What management said about Q2 FY2026, from the documents filed with the SEC on 29 July 2026: press release. Guidance figures: 1 raised, 2 new, 2 kept. The quarter’s numbers.
Key takeaways
- FDA agrees accelerated approval path is reasonable: Official FDA minutes received July 2026 confirmed alignment that a BLA under the accelerated approval pathway, based on existing clinical data, is reasonable. The company cites a June 2026 Type B meeting. Source
- Confirmatory study design still being aligned: The FDA seeks alignment on the confirmatory study design before BLA submission, including a possible randomized standard-of-care control instead of a sham procedure. The company plans to start the study once design is agreed. Source
- AMT-260 low-dose seizure reductions reported: At the May 29, 2026 cutoff, three of six low-dose patients had 79% to 100% seizure declines in months four through six. The other three ranged from a 33% decrease to a 36% increase. Source
- AMT-191 dosing paused in two cohorts: Dosing in the mid- and high-dose cohorts remains paused after two patients had asymptomatic liver enzyme elevations confirmed as dose-limiting toxicities. The elevations resolved by end of May 2026 after immunosuppression. Source
- Net loss widened to $81.1 million: Net loss was $81.1 million, or $1.22 basic and diluted loss per ordinary share, compared with $37.7 million, or $0.69, in the second quarter of 2025. Source
- Cash rose after June offering: Cash, cash equivalents and current investment securities were $810.3 million at June 30, 2026, up from $622.5 million at December 31, 2025. Source
- Runway extended into 2030: Management expects these resources to fund projected operating expenses into 2030. The prior release gave runway into the second half of 2029. Source
“This has been a defining quarter - not just for uniQure, but for the Huntington's disease community,”
Matthew Kapusta, chief executive officer at uniQure
Guidance
| Area | Period | Figure | Against last quarter |
|---|---|---|---|
| AMT-130 U.S. BLA submission Source | third quarter of 2026 | third quarter of 2026 | New |
| AMT-130 U.K. regulatory submission Source | third quarter of 2026 | third quarter of 2026 | Kept |
| AMT-130 topline four-year data Source | September 2026 | September 2026 | Kept |
| AMT-260 second cohort enrollment Source | third quarter of 2026 | third quarter of 2026 | |
| AMT-260 updated Phase I/IIa results Source | first half of 2027 | first half of 2027 | New |
| Cash runway Source | into 2030 | into 2030 | Raised from second half of 2029 |
What changed from last quarter
- Guidance raised: Cash runway extended from the second half of 2029 to into 2030. Source
- New guidance: First guidance for a U.S. BLA submission in the third quarter of 2026, following FDA alignment on the accelerated approval pathway. Source
- New guidance: Updated Phase I/IIa AMT-260 results are now guided for the first half of 2027. Source
- Guidance kept: U.K. regulatory submission for AMT-130 is still guided to the third quarter of 2026. Source
- New: A Type B meeting with the FDA was held in June 2026, and official minutes were received in July 2026. Source
- New: The company closed a $259 million follow-on offering in June 2026. Source
- New: Preliminary six-month follow-up data for the first low-dose AMT-260 cohort were announced in June 2026. Source
AMT-130 regulatory path
- FDA agrees accelerated approval path is reasonable: Official FDA minutes received July 2026 confirmed alignment that a BLA under the accelerated approval pathway, based on existing clinical data, is reasonable. The company cites a June 2026 Type B meeting. Source
- Confirmatory study design still being aligned: The FDA seeks alignment on the confirmatory study design before BLA submission, including a possible randomized standard-of-care control instead of a sham procedure. The company plans to start the study once design is agreed. Source
- FDA expects confirmatory study well underway: Under FDA draft accelerated approval guidance, the confirmatory study must be feasible within a reasonable timeline and well underway, potentially fully enrolled, at the time of accelerated approval. Source
- U.K. submission progressing as planned: Following a March 2026 pre-submission meeting with the U.K. MHRA, the regulatory submission is progressing as planned for the third quarter of 2026. Source
- Four-year data set for September: The company plans to present follow-up on all patients in the first two AMT-130 cohorts, including four years on 24 patients, 12 at high dose and 12 at low dose, in September 2026. Source
Pipeline clinical progress
- AMT-260 low-dose seizure reductions reported: At the May 29, 2026 cutoff, three of six low-dose patients had 79% to 100% seizure declines in months four through six. The other three ranged from a 33% decrease to a 36% increase. Source
- AMT-260 first cohort adverse events mild: No serious adverse events related to AMT-260 or the surgical procedure were reported as of June 19, 2026. No immunosuppression was required, and the most common event was headache, in two patients. Source
- AMT-191 dosing paused in two cohorts: Dosing in the mid- and high-dose cohorts remains paused after two patients had asymptomatic liver enzyme elevations confirmed as dose-limiting toxicities. The elevations resolved by end of May 2026 after immunosuppression. Source
- AMT-191 enzyme activity rose across doses: Alpha-Gal A activity rose dose-dependently across 11 patients at three dose levels. All 11 dosed patients were withdrawn from enzyme replacement therapy, and lyso-Gb3 stayed stable post-dose. Source
“we remain on track to submit our BLA for AMT-130 in the third quarter - a milestone that reflects years of rigorous science, disciplined execution”
Matthew Kapusta, chief executive officer at uniQure
Second quarter financial results
- Revenue rose on higher license income: Total revenue was $5.8 million, against $5.3 million a year earlier. The company attributed the increase to higher license revenue. Source
- Net loss widened to $81.1 million: Net loss was $81.1 million, or $1.22 basic and diluted loss per ordinary share, compared with $37.7 million, or $0.69, in the second quarter of 2025. Source
- Non-operating expense driven by currency and warrants: Non-operating expense was $27.0 million, against a $6.6 million gain a year earlier. Foreign currency moved $20.4 million, and a $16.0 million loss came from changes in pre-funded warrant liability fair value. Source
Operating costs and expenses
- R&D spend slightly lower overall: R&D expenses were $34.0 million, down $1.4 million. Lower AMT-130 direct spend was partly offset by higher spend on AMT-260, AMT-162 and AMT-191 programs. Source
- SG&A rose on commercial team hiring: SG&A was $17.4 million, up $3.9 million. Employee and contractor costs rose $4.3 million from staff recruited in the second half of 2025 for potential AMT-130 launches. Source
- Other expense rose on HEMGENIX supply costs: Other expense was $8.0 million, against $2.2 million a year earlier, mainly from a $6.0 million increase in costs for supplying HEMGENIX to CSL Behring. Source
“With four-year data expected in September and the U.K. regulatory activities progressing as planned, we enter the second half of 2026 with real momentum”
Matthew Kapusta, chief executive officer at uniQure
Cash, funding and runway
- Cash rose after June offering: Cash, cash equivalents and current investment securities were $810.3 million at June 30, 2026, up from $622.5 million at December 31, 2025. Source
- Runway extended into 2030: Management expects these resources to fund projected operating expenses into 2030. The prior release gave runway into the second half of 2029. Source
- $259 million follow-on offering completed: The company sold 5,686,813 ordinary shares at $45.50 per share, including full exercise of the underwriters' option, for $259 million gross proceeds before discounts and expenses. Source
Strategy and upcoming events
- Broader pipeline work continues: Management said it continues to execute across the broader pipeline, which covers Huntington's disease, refractory temporal lobe epilepsy, Fabry disease and other severe diseases. Source
- Investor conference planned for August: Management plans to attend the 2026 Biotech Summer Summit in Newport, Rhode Island, from August 10 to 12. Source
Coming up, as management said on 29 July 2026
- Four-year AMT-130 data in September: Topline four-year data from the Phase I/II AMT-130 study is expected in September 2026. Source
- AMT-130 BLA submission in third quarter: The company expects to submit a BLA for AMT-130 in the third quarter of 2026, after aligning with the FDA on the confirmatory study design. Source
- Confirmatory study start after FDA alignment: The company plans to start the confirmatory study as quickly as possible once the FDA agrees on the trial design. Source
- AMT-260 second cohort enrollment completion: Enrollment in the second AMT-260 dose cohort is expected to complete in the third quarter of 2026. Source
- Updated AMT-260 results in 2027: Updated Phase I/IIa AMT-260 results are expected in the first half of 2027. Source
- Investor conference in August: Management will attend the 2026 Biotech Summer Summit in Newport, RI, from August 10 to 12. Source
Figures management reported
| Figure | Reported |
|---|---|
| R&D expenses, second quarter 2026 Source | $34.0 million vs $35.4 million |
| Total revenues, second quarter 2026 Source | $5.8 million |
| Trial readout, AMT-260 low-dose cohort Source | 3 of 6 patients with 79% to 100% seizure decline |
| Pipeline milestone, AMT-130 BLA Source | Submission expected third quarter 2026 |
Risks management discussed
- Regulatory: Confirmatory study design is still under FDA discussion, including whether a standard-of-care control replaces a sham arm, before BLA submission. Source
- Regulatory: Under FDA draft accelerated approval guidance, the confirmatory study is expected to be well underway and potentially fully enrolled at approval. Source
- Operational: Mid- and high-dose AMT-191 dosing remains paused pending evaluation of liver enzyme elevations confirmed as dose-limiting toxicities. Source
- Financial: Net non-operating expense rose $33.6 million, driven by a $20.4 million foreign currency change and a $16.0 million warrant liability fair value loss. Source
Earlier plans management reported on
- On track: Management states the U.S. and U.K. AMT-130 submissions remain on track as planned for the third quarter of 2026. Source
- Not stated: The prior release said first-cohort data would be presented at the June 2026 conference; this release reports preliminary six-month data announced in June. Source
- Not stated: The prior release guided second-cohort enrollment completion to mid-2026; this release expects completion in the third quarter of 2026. 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.