uniQure reaffirms Q3 2026 BLA plans for AMT-130 in Huntington's disease

uniQure reported second-quarter 2026 results on July 29, 2026, and said meeting minutes received in July 2026 from a June 2026 Type B meeting with the FDA confirmed that the agency and the company had aligned that a Biologics License Application under the accelerated approval pathway for AMT-130, based on existing clinical data, was reasonable.¹ The FDA also said the confirmatory study design still needs alignment, including possible use of a randomized standard-of-care control instead of a sham procedure¹, and indicated that under its draft accelerated approval guidance, the confirmatory study should be feasible within a reasonable timeline and potentially well underway or fully enrolled at the time of accelerated approval.¹ The company said it still expects to file the BLA in the third quarter of 2026 and will start the confirmatory study once trial design is finalized with the FDA.

Separately, the company held a pre-submission meeting with the U.K.'s MHRA in March 2026 and said its U.K. regulatory submission remains on track for the third quarter of 2026.¹ uniQure said it plans to disclose four-year follow-up data from its Phase I/II AMT-130 studies in September 2026, covering all patients in the first two cohorts, including 24 patients with four years of follow-up (12 at high dose, 12 at low dose).¹

In epilepsy, as of a May 29, 2026 data cutoff, three of six patients in the first, low-dose AMT-260 cohort showed reductions in disabling seizures of 79% to 100% from baseline during months four through six¹, while the other three patients had changes ranging from a 33% decrease to a 36% increase.¹ No related serious adverse events were reported as of the June 19, 2026 presentation, with all adverse events mild or moderate, most commonly headache in two patients.¹ Enrollment in the second dose cohort is expected to complete in the third quarter of 2026.¹

For AMT-191 in Fabry disease, dosing in mid- and high-dose cohorts remains paused pending evaluation of asymptomatic liver enzyme elevations in two mid-dose patients, confirmed as dose-limiting toxicities, which resolved by the end of May 2026 following immunosuppression.¹

On financing, uniQure closed an upsized offering of 5,686,813 ordinary shares at $45.50 per share in June 2026, raising $259 million in gross proceeds.¹ As of June 30, 2026, cash, cash equivalents and current investment securities totaled $810.3 million, which the company said should fund operations into 2030.¹