uniQure reports US$768.4M liquidity and runway into 2030
Cash and investments fell US$41.9 million over the quarter as the gene therapy developer prepared for a potential AMT-130 launch.
Adrian Kessler
Operations are expected to be funded into 2030 as uniQure N.V. NASDAQQURE reported US$768.4 million in cash and investments on Wednesday. The Sept. 30 balance fell US$41.9 million over the quarter.
The comparison is with US$810.3 million at June 30, disclosed in the company's July 29 financial update. The unchanged runway forecast extends beyond the FDA's May 1, 2027 target for acting on the AMT-130 Huntington's disease application.
uniQure is a specialist gene therapy developer based in Lexington, Massachusetts, and Amsterdam, advancing treatments for severe neurological and inherited diseases.
The available NASDAQQURE quote showed uniQure shares closing at US$24.83 on Nasdaq at 4 p.m. EDT on Oct. 30. That price predates Wednesday's results and provides no measure of the market's response.
Launch Preparation Raises Administrative Spending
Revenue for the quarter ended Sept. 30 rose to US$5.2 million from US$3.7 million a year earlier, driven by higher licence revenue. The business remains loss-making as it prepares for a potential commercial launch.
The third-quarter net loss narrowed to US$61.2 million from US$80.5 million in the corresponding period of 2025. Net non-operating expense fell to US$7.6 million from US$20.9 million, reflecting lower expenses related to pre-funded warrants and improved net interest income.
Research and development spending reached US$35.5 million in the third quarter, compared with US$34.4 million a year earlier. Higher employee and contractor costs outweighed a decline in direct research and development expenses.
Selling, general and administrative expenses for the quarter ended Sept. 30 rose to US$23.9 million from US$19.4 million a year earlier. Recruitment drove higher personnel costs, while professional fees and information technology costs also increased as uniQure prepared for potential commercial launches.
"As we continue to build our commercial capabilities and prepare for a potential launch, we are energized by what lies ahead, and our financial strength provides us the runway to deliver on our plans," uniQure chief executive Matthew Kapusta said in the release.
Read more: Rhythm Pharmaceuticals reports US$92.1M sales as IMCIVREE launch grows
Confirmatory Trial Screening Is Planned For 2026
The regulatory milestone was announced on Nov. 2, making Wednesday's financial figures the new information. Priority review applies to an application seeking accelerated approval; it does not authorize sales.
uniQure says it has aligned with the FDA on key elements of a randomized confirmatory trial enrolling approximately 200 patients. Treatment and standard care will be allocated two to one, with Total Functional Capacity measured at 36 months as the primary endpoint.
The evidence still carries uncertainty. In September's analysis of 12 high-dose patients, the composite disease-rating endpoint showed a non-significant comparison against an external control at 48 months, with p=0.144.
The company described AMT-130 as generally well tolerated across 51 treated patients in Wednesday's update. Its application relies on an earlier 36-month comparison involving 12 high-dose patients and a matched external control.
Read more: uniQure gains FDA priority review for Huntington's gene therapy
Among other RNA medicine developers, Wave Life Sciences, Inc. NASDAQWVE reported US$431 million in cash, equivalents and marketable securities at Sept. 30. Its Oct. 27 update projected a runway into the third quarter of 2028.
For sector context, the iShares Biotechnology ETF NASDAQIBB closed at US$213.79 at 4 p.m. EST on Nov. 2, according to that Google Finance quote.
U.S. patient screening for uniQure's confirmatory trial is expected to begin by the end of 2026. The FDA's May 1, 2027 action target is the next dated regulatory decision on whether AMT-130 can receive accelerated approval.
