uniQure Q3 2026: Misses on Both Lines as AMT-130 Launch Costs Rise

uniQure reported a loss of $0.88 per share for the third quarter of 2026, missing the consensus estimate of roughly $0.82 by about 7%. Revenue of $5.2 million also fell short of the $5.4 million estimate by 4.3%, though it was 40.4% higher than the same period a year earlier, driven by increased license revenue.

The headline miss is largely a story about deliberate spending. SG&A climbed to $23.9 million from $19.4 million as the company hired field medical, market access, and strategic account staff and paid professional fees tied to preparing a commercial launch of AMT-130, its Huntington's disease gene therapy. R&D expenses also moved higher on increased employee and contractor costs, partially offset by a $4.1 million reduction in AMT-130 process validation expenses.

The central event investors are watching is the FDA's PDUFA target action date of May 1, 2027, for AMT-130, which is under Priority Review and Accelerated Approval consideration. The company's BLA was accepted by the FDA, and the UK's MHRA has also validated a Marketing Authorization Application. Among the clinical data supporting the filing: a 36-month analysis of 15 high-dose patients showed 80% slowing on the cUHDRS scale and 67% slowing on the TFC scale versus an external control, though the 48-month primary endpoint on cUHDRS in 12 high-dose patients did not reach statistical significance.

With $768.4 million in cash and investment securities on hand, management says the company is funded well into 2030, providing a long runway through the FDA decision and beyond into any post-approval confirmatory study. That confirmatory study, targeting roughly 200 patients randomized against standard of care with TFC at 36 months as the primary endpoint, is expected to begin U.S. patient screening by the end of 2026.

CEO Matthew Kapusta pointed to the Priority Review status and ongoing trial activity as signs of meaningful momentum: "We strongly believe AMT-130 has the potential to deliver meaningful benefit to HD patients. With our BLA under Priority Review and preparations for the confirmatory study underway, we are committed to bringing this therapy to patients and families who continue to live with this devastating disease." The key risk the company itself flags is that the FDA may conclude the Phase I/II data are not sufficient for approval, making the May 2027 decision date the defining moment for the stock.