On November 10, 2025, uniQure reported third‑quarter results that fell short of analyst expectations but reinforced its long‑term cash position. Revenue rose 60.9% to $3.7 million, driven by a $1.5 million lift in license income, but missed the consensus estimate of $5.13–$6.93 million. The company posted a net loss of $80.5 million, a widening from the $44.4 million loss in Q3 2024 and the $37.7 million loss in Q2 2025, largely due to a $10.1 million increase in R&D expenses for the AMT‑130 BLA and higher professional fees. Non‑operating expenses surged to $20.9 million from $4.2 million year‑ago, driven by foreign‑currency losses, reduced interest income, and fair‑value losses on pre‑funded warrants.
The cash, cash equivalents and current investments climbed to $694.2 million, up from $409.0 million at the end of March. This balance now supports operations through 2029, extending the company’s runway beyond the previously cited second half of 2027. The stronger liquidity cushion comes despite the larger quarterly loss, reflecting uniQure’s continued investment in its pipeline and the high cost of bringing a gene‑therapy product to market.
Revenue and earnings misses are largely attributable to the company’s aggressive investment in the AMT‑130 program. The Phase I/II study of AMT‑130 in Huntington’s disease met its primary and key secondary endpoints, but the FDA’s recent feedback on the BLA raised uncertainty about the adequacy of external control data. This regulatory pause has delayed the expected BLA filing, increasing R&D and professional fees and contributing to the widened loss. Management noted that the FDA’s comments “introduce uncertainty in the timing of our BLA submission,” underscoring the headwind that could affect future cash burn.
EPS of $‑1.38 per share fell short of the consensus estimate of $‑0.85, a miss of $0.53. The miss reflects the combined impact of higher R&D and professional fees, non‑operating losses, and the absence of any revenue from product sales. While the company’s revenue grew sharply, the lack of commercial revenue and the heavy regulatory costs have eroded profitability. The miss signals that, although the pipeline is progressing, the company remains in a high‑cost development phase.
Management emphasized that the positive clinical data for AMT‑130 remain a key tailwind, but the regulatory uncertainty and the need for additional data will likely keep the company in a loss‑making state for the foreseeable future. The company’s cash runway to 2029 provides a buffer, but investors will watch for a clear regulatory path and any changes in the cost structure. The market reaction was tempered by the earnings miss and the FDA feedback, with analysts noting that the company’s valuation is now more sensitive to regulatory outcomes than to revenue growth.
The company’s guidance for the next quarter and full year was not updated in the release, leaving investors to interpret the current results as a baseline. The lack of forward guidance, combined with the regulatory pause, suggests a cautious outlook for the near term, while the strong cash position and clinical milestones keep the long‑term prospects intact.
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