Uniqure NV
NASDAQ:QURE
Uniqure NV
uniQure NV engages in the discovery, development, and commercialization of innovative gene therapies. The company is headquartered in Amsterdam, Noord-Holland and currently employs 463 full-time employees. The company went IPO on 2014-02-05. The company seeks to develop one-time administered treatments with potentially curative results for patients suffering from genetic and other devastating diseases. The company develops, both internally and through partnerships, a pipeline of gene therapies. The company produces adeno-associated virus based, or AAV-based, gene therapies in its own facilities with a proprietary, commercial-scale, current good manufacturing practices, compliant, manufacturing process. AMT-061, the Company’s lead product candidate for patients with hemophilia B, is going through a dosing phase of a pivotal study. AMT-130, the product candidate for patients with Huntington’s disease is in Phase I/II clinical study.
uniQure NV engages in the discovery, development, and commercialization of innovative gene therapies. The company is headquartered in Amsterdam, Noord-Holland and currently employs 463 full-time employees. The company went IPO on 2014-02-05. The company seeks to develop one-time administered treatments with potentially curative results for patients suffering from genetic and other devastating diseases. The company develops, both internally and through partnerships, a pipeline of gene therapies. The company produces adeno-associated virus based, or AAV-based, gene therapies in its own facilities with a proprietary, commercial-scale, current good manufacturing practices, compliant, manufacturing process. AMT-061, the Company’s lead product candidate for patients with hemophilia B, is going through a dosing phase of a pivotal study. AMT-130, the product candidate for patients with Huntington’s disease is in Phase I/II clinical study.
Regulatory update: FDA told uniQure it is unlikely to accept Phase I/II AMT-130 data as the primary evidence for a BLA and strongly recommended a randomized, double‑blind sham surgery–controlled Phase III.
Clinical strength: Management reiterates confidence in AMT-130 — 3‑year data showed a 75% slowing on the composite UHDRS, 60% slowing on TFC and reductions in neurofilament light — and plans a 4‑year analysis in Q3 2026.
Next regulatory steps: uniQure will request a Type B meeting in Q2 2026 to discuss Phase III design and will submit an amended statistical analysis plan to include the 4‑year analysis.
Other programs: AMT‑191 (Fabry) showed durable, dose‑dependent increases in alpha‑Gal A in 11 patients and all have been withdrawn from enzyme replacement therapy; dosing paused in mid/high cohorts after two grade‑3 LFTs confirmed as dose‑limiting. AMT‑260 (TLE) cohort 1 has six patients with a Q2 update planned; AMT‑162 (SOD1 ALS) remains on voluntary hold after DRG toxicity in one patient.
Financials & runway: 2025 revenue was $16.1 million; cash and investments totaled $622.5 million at year‑end, which management expects is sufficient to fund operations into the second half of 2026.
Patient/community focus: uniQure emphasizes engagement with the HD community and plans to pursue ex‑U.S. regulatory/early access options while continuing dialogue with global regulators.
Commercial posture: Company is prepared to commercialize AMT‑130 itself but will evaluate partnering as Phase III design and cost become clearer.
