LEXINGTON, Mass. and AMSTERDAM — May 5, 2026 — Leads & Copy —
uniQure N.V. (NASDAQ: QURE) reported its financial results for the first quarter of 2026 and highlighted recent business progress, including advancements in its gene therapy programs for Huntington’s disease, temporal lobe epilepsy, and Fabry disease.
The company is advancing interactions with the FDA for AMT-130 for Huntington’s disease, with a Type B meeting scheduled for the second quarter of 2026. Progress is also being made toward an expected UK regulatory submission for AMT-130, with a Marketing Authorization Application (MAA) on track for the third quarter of 2026 following a successful pre-submission meeting with the UK MHRA.
Enrollment in the AMT-260 temporal lobe epilepsy program is on track, and a clinical update from the first cohort in the Phase I/IIa study will be presented at the Epilepsy Foundation Pipeline Conference. Updated data from the AMT-191 Phase I/IIa study in Fabry disease showed sustained increases in α-Gal A Enzyme Activity and stable Lyso-Gb3 levels, leading to all 11 dosed patients discontinuing enzyme replacement therapy.
According to the press release, uniQure has a strong balance sheet with $586.6 million in cash, cash equivalents, and current investment securities as of March 31, 2026, providing a financial runway into the second half of 2029. uniQure hosted an earnings call on May 5, 2026, at 8:30 a.m. ET.
uniQure CEO Matthew Kapusta said the company remained focused on advancing AMT-130 to patients globally and executing across its broader pipeline during the first quarter of 2026. Kapusta added that data continues to support the potential for AMT-130 to fundamentally change the treatment landscape for Huntington’s disease, and he looks forward to continued engagement with the FDA.
The company held a Type A meeting with the FDA in January 2026 to discuss the regulatory path forward following an October 2025 pre-Biologics License Application (BLA) meeting. The FDA stated it could not agree that data from the Phase I/II studies, compared to an external control, are sufficient to provide the primary evidence of effectiveness required to support a marketing application for AMT-130. uniQure has been granted a Type B meeting with the FDA to discuss key elements of a new clinical trial design and to solicit feedback on the proposed statistical analysis plan for the four-year data expected in the third quarter of 2026.
Following a constructive pre-submission meeting with the UK’s MHRA, uniQure expects to submit an MAA for AMT-130 based on three-year data in the third quarter of 2026.
In 2025, uniQure completed enrollment in the first dose cohort of six patients in the Phase I/IIa study of AMT-260. Enrollment is ongoing in a second dose cohort, expected to include an additional six patients, with completion expected in mid-2026. Data from the first cohort of six patients in the Phase I/IIa study with up to six months of follow-up is expected to be presented at the Epilepsy Foundation Pipeline Conference, June 18 -19, 2026.
In February 2026, uniQure presented updated safety and exploratory efficacy data from the Phase I/II study of AMT-191 in Fabry disease (data cutoff as of January 8, 2026). Dose-dependent elevations were observed across 11 patients in three dose levels with α-Gal A activity ranging from 0.34- to 82.2-fold above mean normal range at the lowest dose, 1.6- to 312.5-fold at the mid dose, and 27.7- to 223.7-fold at the highest dose. These increases were durable across follow-up periods ranging from four months to more than one year. Plasma lyso-Gb3 levels were stable post-dose across all cohorts, regardless of enzyme replacement therapy (ERT) status. As of February 18, 2026, all 11 dosed patients were withdrawn from ERT.
AMT-191 continued to show a manageable safety profile at all dose levels. Additional dosing in the mid- and high-dose cohorts has been paused pending further evaluation of asymptomatic Grade 3 liver enzyme elevations reported in two patients from the mid-dose cohort, which were confirmed as dose-limiting toxicities.
In April 2026, uniQure, CSL Behring and Genezen entered into agreements under which uniQure’s remaining HEMGENIX® supply and any minimum purchase commitments will terminate after delivery of contractually specified batches to CSL Behring, which uniQure expects to occur in mid-2026. Concurrently, CSL Behring entered into a direct relationship with Genezen as the supplier of HEMGENIX®. These agreements have no impact on future royalties or milestones to uniQure under the license agreement with CSL Behring.
Financial highlights for the first quarter of 2026 include a cash position of $586.6 million, revenues of $3.6 million, R&D expenses of $29.2 million, and SG&A expenses of $20.1 million. The net loss for the quarter was $53.5 million, or $0.85 basic and diluted loss per ordinary share.
Upcoming investor events include the 2026 RBC Capital Markets Global Healthcare Conference on May 19 in New York, NY.
uniQure is advancing a pipeline of proprietary gene therapies for the treatment of patients with Huntington’s disease, refractory temporal lobe epilepsy, Fabry disease, and other severe diseases.
Source: uniQure N.V.
