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The call communicated major positive clinical advances (notably strong 3‑year AMT‑130 efficacy, supportive Fabry and TLE data) and a strengthened balance sheet, but these were tempered by significant regulatory resistance from the FDA (recommendation for a sham‑controlled Phase III), safety‑related pauses/holds in two programs (AMT‑191 dosing pause and AMT‑162 hold), and a substantial year‑over‑year revenue decline. Management emphasized active regulatory engagement, continued data generation (4‑year AMT‑130 analysis) and pursuit of ex‑U.S. pathways and early access options, while acknowledging material uncertainty around Phase III design, cost and feasibility.
The key regulatory guidance from the call was that the FDA reaffirmed it is unlikely to accept Phase I/II data versus an external control as primary evidence and strongly recommended a randomized, double‑blind, sham surgery‑controlled Phase III study (discussed at an October 2025 pre‑BLA and a Type A meeting on Jan 30, 2026, with minutes received Feb 27), prompting uniQure to plan a Type B meeting in Q2 2026 to discuss Phase III design and to amend the Phase I/II SAP to include a 4‑year analysis expected in Q3 2026 (including 12 patients at 4 years and all patients reaching 3 years); uniQure noted its 3‑year AMT‑130 data showed a 75% slowing on the composite UHDRS and 60% slowing on Total Functional Capacity with reductions in neurofilament light versus an Enroll HD external comparator (>30,000 participants, 14 years of data), but the 12‑month sham‑controlled U.S. cohort showed no worsening at 12 months, which influenced FDA views; the company will pursue constructive FDA dialogue, ex‑U.S. regulator discussions (e.g., EMA/MHRA), and feasibility work (including patient burden/ethical considerations around multiyear sham surgery), while continuing program updates (AMT‑191: 11 patients, all off ERT, dose‑dependent alpha‑Gal A increases durable >1 year to 4 months with 2 mid‑dose Grade 3 LFTs leading to a dosing pause; AMT‑260: six patients in Cohort 1 with ≥6 months follow‑up and Cohort 2 enrollment expected complete by midyear) and citing a cash balance of $622.5M as of 12/31/25 (vs. $367.5M a year earlier, including ~$404.2M raised) with runway into H2 2026.
Phase I/II 3‑year data demonstrated a statistically significant 75% slowing of disease progression on the composite UHDRS and a 60% slowing on Total Functional Capacity versus an external Enroll HD comparator; reduction in neurofilament light and supportive trends across other clinically meaningful endpoints. Company plans a 4‑year analysis in Q3 2026 and intends to amend the SAP and submit the analysis to regulators.
Preliminary Phase I/II data from 11 patients showed dose‑dependent elevation in alpha‑Gal A enzyme activity durable from 4 months up to >1 year; as of the call all 11 patients have been withdrawn from enzyme replacement therapy.
Case study of first patient with up to 6 months follow‑up showed a promising reduction in seizure frequency and no serious adverse events; enrollment completed for five additional patients in cohort 1 (six total) with cohort 2 enrollment underway and a planned Q2 update on six patients with ≥6 months follow‑up.
Cash, cash equivalents and investment securities totaled $622.5 million at Dec 31, 2025 vs $367.5 million at Dec 31, 2024, an increase of ~$255.0 million (+69.4%), primarily from ~$404.2 million in proceeds from public offerings and prefunded loans; company expects cash to fund operations into the second half of 2026.
Cost of contract manufacturing revenues was nil in 2025 versus $17.1 million in 2024 following the 2024 divestiture of the Lexington manufacturing facility; R&D 'other' costs decreased by $26.0 million driven by lower employee/contractor/severance and facility costs.
Company is actively engaging the FDA (Type B planned for Q2 2026), pursuing dialogue with ex‑U.S. regulators (MHRA, EMA and others), and evaluating named‑patient/early access programs and ex‑U.S. regulatory pathways to accelerate patient access.
Broad advocacy from the Huntington's disease community and positive feedback from neurosurgeons, neurologists and HD centers of excellence reinforce clinical interest and urgency for regulatory flexibility and access.
Thank you for standing by. My name is Kate, and I will be your conference operator today. At this time, I would like to welcome everyone to the uniQure Fourth Quarter and Year-End 2025 Earnings Call. [Operator Instructions] I would now like to turn the call over to Chiara Russo, Senior Director of Investor Relations. Please go ahead.
Good morning. and thank you for joining us for uniQure's year-end 2025 earnings call. Earlier this morning, uniQure released its financial results for the fourth quarter and year-end of 2025, and our press release is available on the Investors and Media section of our website at uniqure.com. Our 10-K was also filed with the SEC earlier this morning. Joining me on the call this morning are Matt Kapusta, Chief Executive Officer; Dr. Walid Abi-Saab, Chief Medical Officer; Kylie O'Keefe, Chief Customer and Strategy Officer; and Christian Klemt, Chief Financial Officer. After our formal remarks, we'll open up the call for Q&A. Before we begin, please note that we will be making forward-looking statements during this investor call.
All statements other than statements of historical fact are forward-looking statements. They are based on management's beliefs and assumptions and on information available to management only as of the date of this conference call. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including without limitation, the factors described in uniQure's most recent SEC filings. Given these risks, you should not place undue reliance on these forward-looking stateme...