Recent Updates — QURE
On September 29, 2026, uniQure N.V. released updated clinical data for its gene therapy candidate ifezuntirgene inilparvovec (AMT-130) in the treatment of Huntington’s disease. The update covers Phase I/II results at 36 and 48 months with a data cutoff of June 30, 2026. At 36 months, high-dose patients showed an 80% slowing of disease progression based on cUHDRS (p=0.005) and 67% based on TFC (p=0.011). At 48 months, the primary endpoint cUHDRS showed a non-significant 44% slowing (p=0.144), while TFC demonstrated a significant 61% slowing (p=0.008). Post-hoc analysis using prior controls suggested stronger effects at 48 months. The therapy was generally well-tolerated, with one treatment-related serious adverse event of CNS inflammation reported since September 2025. uniQure N.V. is a gene therapy company developing treatments for rare diseases including hemophilia and Huntington’s disease.
On September 2, 2026, uniQure N.V. announced the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration for accelerated approval of ifezuntirgene inilparvovec (AMT-130), an investigational gene therapy for Huntington’s disease. The company also submitted a Marketing Authorisation Application (MAA) to the UK Medicines Healthcare products Regulatory Agency. Both applications are supported by three-year data from Phase I/II studies demonstrating slowed disease progression, and uniQure has requested priority review for the BLA. The filing does not contain specific financial figures or dividend announcements.
uniQure N.V. reported second quarter 2026 financial results and provided a corporate update on July 29, 2026. The company generated $5.8 million in revenue and recorded a net loss of $81.1 million, or $1.22 per share, compared to a $37.7 million loss in the prior year period. Cash reserves increased to $810.3 million following the June 2026 closing of a $259 million follow-on equity offering at $45.50 per share, extending the cash runway into 2030. Regulatory progress includes alignment with the FDA on a Biologics License Application (BLA) for AMT-130 for Huntington’s disease, with submission expected in Q3 2026 and four-year clinical data to be presented in September. Additionally, preliminary data for AMT-260 showed seizure reductions in epilepsy patients, while development of AMT-191 for Fabry disease remains paused pending evaluation of liver enzyme elevations. uniQure N.V. operates as a gene therapy company developing transformative treatments for severe genetic diseases.
uniQure N.V. entered into an underwriting agreement with Leerink Partners LLC to conduct an underwritten public offering of 4,945,055 ordinary shares at $45.50 per share, with an option for underwriters to purchase an additional 741,758 shares. The underwriters exercised the option in full on June 24, 2026, and the company expects to receive approximately $242.7 million in net proceeds. uniQure N.V. is a biotechnology company focused on gene therapy development.
uniQure N.V. announced preliminary data from the first cohort of its Phase I/IIa clinical trial for AMT-260, targeting refractory mesial temporal lobe epilepsy. In the low-dose cohort (1x10 12 gc/mL), three of six patients achieved 79% to 100% reductions in disabling seizures during months four through six of follow-up. The remaining three patients showed variable changes in disabling seizures, ranging from a 33% decrease to a 36% increase. No serious adverse events related to AMT-260 or the surgical procedure were reported as of June 19, 2026. uniQure N.V. is a biotechnology company focused on gene therapy development.
uniQure N.V. announced its plan to submit a Biologic License Application (BLA) for AMT-130 in Huntington's Disease during the third quarter of 2026. This follows a Type B meeting with the FDA, where the agency communicated that the 3-year analysis from the Phase I/II study would be acceptable as the primary basis for an accelerated approval. uniQure N.V. is a biotechnology company focused on developing gene therapies for rare genetic diseases.