QURE
uniQure N.V.21.89
+1.46+7.15%
Dec 16, 4:00:01 PM EST
Earnings Call Transcripts
This Quarter (Q1 '26)
No earnings call transcript available yet
Last Quarter (Q4 '25)
FY Q4 '25
Reaffirms Phase III commitment, ex-U.S. focus
Q&A largely reaffirms prepared remarks on FDA's Phase III sham-controlled mandate for AMT-130, but management commits to advancing it if feasible and ethical, citing patient burden risks like prolonged uncertainty post-sham surgery. They plan a Q2 Type B meeting to pitch designs leveraging Enroll-HD's vast natural history data. Ex-U.S. paths gained traction, with named patient programs modeled on Roche's playbook in priority markets. Analysts grilled on enrollment speed, costs, and partnering; answers stayed vague, premature. Sham burdens HD patients. Watch Type B outcomes and ex-U.S. progress for thesis clarity.
Key Stats
Market Cap
1.36BP/E (TTM)
-Basic EPS (TTM)
-4.40Dividend Yield
0%Recent Filings
10-K
FY2025 results
uniQure's FY2025 delivered positive topline from AMT-130 Phase I/II Huntington's trial, showing high-dose slowing disease by 75% on cUHDRS (p=0.003) vs external control at 36 months—yet FDA rejected BLA path sans Phase III sham trial, stalling momentum. No quarterly financials disclosed; annual net loss narrowed to $199M from $240M prior amid $404M equity raises funding pipeline. Clinical ramps hit Q4: AMT-260/AMT-191 cohorts filled, though ALS paused on SAE. Cash swelled to $623M into H2 2029 runway; $50M Hercules debt at 9.45%. FDA delays risk derailing AMT-130 approval.
8-K
FDA rejects AMT-130 Phase I/II data
uniQure reported 2025 net loss of $199.0 million, down from $239.6 million in 2024, with cash at $622.5 million funding operations into H2 2029. FDA rejected Phase I/II data for AMT-130 Huntington's therapy versus external control, urging Phase III randomized sham-controlled trial; uniQure plans Q2 2026 Type B meeting. Cash burn eased, yet regulatory pivot delays approval.
8-K
AMT-130 slows HD progression 75%
uniQure updated its corporate presentation on January 13, 2026, highlighting AMT-130's 36-month Phase I/II data showing 75% slowing of Huntington's disease progression on cUHDRS (p=0.003) versus external controls. High-dose met primary and key secondary endpoints; CSF NfL dropped below baseline. Cash stands at $694.2M as of September 30, 2025. Data readouts loom.
8-K
FDA Type A meeting scheduled
8-K
FDA rejects AMT-130 BLA data
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