uniQure(QURE) - 2025 Q2 - Quarterly Results

Executive Summary & Recent Highlights uniQure made significant clinical and regulatory progress for AMT-130 and AMT-260, strengthened its leadership, and maintained a strong cash position Recent Company Progress Overview uniQure achieved significant progress in Q2 2025, including FDA alignment for AMT-130 BLA submission, positive early clinical signals for AMT-260, and a strengthened leadership team - Achieved FDA alignment on AMT-130 statistical analysis plan and CMC requirements to support a planned Biologics License Application (BLA) submission in the first quarter of 2026[1][3] - Company expects to present AMT-130 topline three-year data in September 2025[1] - Presented case study from first participant treated with AMT-260 for refractory mesial temporal lobe epilepsy showing 92% seizure reduction with no serious adverse events through first five months of follow up[1] - Appointment of Kylie O'Keefe as Chief Customer and Strategy Officer[1] High-Level Financial Overview The company reported a strong cash position of approximately $377.0 million as of June 30, 2025, which is expected to fund operations into the second half of 2027 Key Financial Highlights (Q2 2025) | Metric | Value | | :----- | :---- | | Cash, cash equivalents and current investment securities (as of June 30, 2025) | ~$377.0 million | | Expected Cash Runway | Into second half of 2027 | Recent Company Developments and Updates uniQure advanced its gene therapy pipeline with significant regulatory and clinical progress for AMT-130, promising early data for AMT-260, and strengthened its leadership team AMT-130 Program (Huntington's Disease) uniQure achieved significant regulatory and clinical milestones for AMT-130, including FDA alignment on the BLA pathway, Breakthrough Therapy designation, and plans for a Q1 2026 BLA submission - FDA alignment on statistical analysis plan (SAP) and Chemistry, Manufacturing and Controls (CMC) requirements for BLA submission in Q1 2026. The primary efficacy analysis will evaluate the three-year change in cUHDRS[3][4] - Submitted final SAP to FDA and initiated Process Performance Qualification (PPQ) campaign in July 2025[4] - AMT-130 was generally well-tolerated in Phase I/II study (third cohort); a short-term steroid regimen is expected to be incorporated as prophylactic immunosuppression[4] - Granted Breakthrough Therapy designation by FDA in April 2025 based on clinical evidence showing potential for slowing progression of Huntington's disease[4] AMT-130 Upcoming Milestones | Milestone | Expected Timing | | :-------- | :-------------- | | Present topline, three-year data from Phase I/II trials | September 2025 | | Initiate fourth cohort (high-dose AMT-130 in 6 patients with lower striatal volumes) | Q3 2025 | | Hold pre-BLA meeting with FDA and complete PPQ campaign | Q4 2025 | | Submit BLA with request for priority review designation | Q1 2026 | Advancing Additional Clinical Programs uniQure is progressing other gene therapy candidates, including AMT-260 with promising early efficacy, and AMT-191 and AMT-162 which are actively enrolling patients with initial data expected in late 2025 and H1 2026, respectively - AMT-260 (refractory mesial temporal lobe epilepsy): Initial data from the first treated patient showed a 92% reduction in seizure frequency through five months with no serious safety events. Clinical sites continue to screen patients[4] - AMT-191 (Fabry disease): Continues to enroll patients in the Phase I/IIa study; initial safety and exploratory efficacy data expected at the 15th ICIEM 2025 in September 2025[4] - AMT-162 (SOD1 amyotrophic lateral sclerosis - ALS): Continues to enroll patients in the Phase I/II EPISOD1 study; initial data expected in the first half of 2026[4] Strengthening the Leadership Team uniQure strengthened its leadership team with the appointment of Kylie O'Keefe as Chief Customer and Strategy Officer, bringing decades of commercial experience to lead commercial and medical affairs - Kylie O'Keefe was appointed as Chief Customer and Strategy Officer in June 2025, responsible for leading all commercial and medical affairs. She previously served as Chief Commercial Officer at PTC Therapeutics[4][11] Financial Review uniQure reported a strong cash position extending into H2 2027 and a reduced net loss in Q2 2025, driven by lower operating expenses and increased non-operating income Cash Position and Runway uniQure's cash, cash equivalents, and current investment securities increased to $377.0 million as of June 30, 2025, primarily due to a follow-on offering, extending its operational funding into the second half of 2027 Cash Position (in millions USD) | Metric | June 30, 2025 | December 31, 2024 | Change | | :----- | :------------ | :---------------- | :----- | | Cash, cash equivalents and current investment securities | $377.0 | $367.5 | +$9.5 | - The net increase in cash was primarily related to net proceeds of $80.5 million from a follow-on offering in January and February 2025[5] - Based on the current operating plan, including the planned U.S. launch of AMT-130, the cash position is expected to fund operations into the second half of 2027[5][11] Q2 2025 Operating Results Overview The company reported a reduced net loss of $37.7 million in Q2 2025 compared to $56.3 million in Q2 2024, driven by decreased revenue but also lower operating expenses and a significant increase in non-operating income Key Financial Results (Three months ended June 30, in thousands USD, except per share) | Metric | Q2 2025 | Q2 2024 | Change (YoY) | | :----- | :------ | :------ | :----------- | | Total revenues | $5,262 | $11,126 | -$5,864 | | Operating expenses | ($49,539) | ($56,883) | +$7,344 | | Loss from operations | ($43,865) | ($44,010) | +$145 | | Non-operating items, net | $6,571 | ($11,341) | +$17,912 | | Net loss | ($37,719) | ($56,299) | +$18,580 | | Basic and diluted net loss per ordinary share | ($0.69) | ($1.16) | +$0.47 | | Weighted average shares | 54,807,967 | 48,622,440 | +6,185,527 | Revenues Analysis Total revenue decreased by $5.8 million year-over-year, primarily due to a significant drop in collaboration revenue and contract manufacturing, partially offset by an increase in license revenue Revenue Breakdown (Three months ended June 30, in millions USD) | Revenue Type | Q2 2025 | Q2 2024 | Change (YoY) | | :----------- | :------ | :------ | :----------- | | Total Revenues | $5.3 | $11.1 | -$5.8 | | License revenue | N/A | N/A | +$3.4 | | Collaboration revenue | N/A | N/A | -$7.1 | | Contract manufacturing (HEMGENIX®) | N/A | N/A | -$2.1 | - Following the divestment of the Lexington facility in July 2024, revenue from contract manufacturing is recorded net of cost within other expenses[6] Operating Expenses Analysis Operating expenses decreased by $7.3 million year-over-year, mainly driven by the absence of contract manufacturing costs and reductions in employee-related and professional fees, despite an increase in R&D external program spend Operating Expenses (Three months ended June 30, in millions USD) | Expense Type | Q2 2025 | Q2 2024 | Change (YoY) | | :----------- | :------ | :------ | :----------- | | Cost of contract manufacturing revenues | $0.0 | $7.2 | -$7.2 | | Research and development (R&D) expenses | $35.4 | $33.7 | +$1.7 | | Selling, general and administrative (SG&A) expenses | $13.5 | $15.8 | -$2.3 | - R&D increase of $1.7 million was due to a $6.3 million increase in external program spend and $4.0 million higher expenses related to an increase in the fair value of contingent consideration, offset by decreases in employee-related, facility, and preclinical supply expenses[8] - SG&A decrease of $2.3 million was primarily related to a $1.6 million decrease in employee-related expenses and a $0.6 million decrease in professional fees[9] Other Income and Expense Other expense increased significantly due to non-cash amortization related to HEMGENIX® purchase rights, while non-operating items shifted from a net expense to a net income, primarily driven by foreign currency gains Other Income/Expense (Three months ended June 30, in millions USD) | Item | Q2 2025 | Q2 2024 | Change (YoY) | | :--- | :------ | :------ | :----------- | | Other expense | $2.2 | $0.2 | +$2.0 | | Other non-operating items, net | $6.6 (income) | $11.3 (expense) | +$17.9 (increase in income) | - Increase in other expense primarily related to $1.4 million of non-cash expense recognized to amortize the right to purchase HEMGENIX® from Genezen on favorable terms[10] - The $17.9 million increase in other non-operating items, net, was primarily related to an increase in net foreign currency gains of $19.6 million, partially offset by a decrease in interest income and reduction of interest expense[12] Consolidated Financial Statements The consolidated financial statements provide detailed figures for the balance sheet as of June 30, 2025, and the statement of operations for the three months ended June 30, 2025 and 2024 Unaudited Consolidated Balance Sheets As of June 30, 2025, total assets increased to $584.9 million from $556.5 million at year-end 2024, primarily driven by an increase in cash and cash equivalents, while total liabilities also increased, resulting in a reduced shareholders' deficit Consolidated Balance Sheet Highlights (in thousands USD) | Metric | June 30, 2025 | December 31, 2024 | Change | | :----- | :------------ | :---------------- | :----- | | Total assets | $584,890 | $556,536 | +$28,354 | | Total liabilities | $588,893 | $563,288 | +$25,605 | | Total shareholders' (deficit) / equity | ($4,003) | ($6,752) | +$2,749 | Key Balance Sheet Changes (in thousands USD) | Item | June 30, 2025 | December 31, 2024 | Change | | :--- | :------------ | :---------------- | :----- | | Cash and cash equivalents | $253,778 | $158,930 | +$94,848 | | Current investment securities | $123,196 | $208,591 | -$85,395 | | Prepaid expenses | $18,538 | $9,281 | +$9,257 | | Intangible assets, net | $75,604 | $71,043 | +$4,561 | | Goodwill | $25,340 | $22,414 | +$2,926 | | Liability from royalty financing agreement | $455,259 | $434,930 | +$20,329 | | Contingent consideration, net of current portion | $15,931 | $10,860 | +$5,071 | Unaudited Consolidated Statements of Operations The detailed statement of operations shows a net loss of $37.7 million for Q2 2025, an improvement from the $56.3 million loss in Q2 2024, reflecting changes in revenues, operating expenses, and non-operating items Consolidated Statements of Operations (Three months ended June 30, in thousands USD, except per share) | Item | 2025 | 2024 | | :--- | :--- | :--- | | Total revenues | $5,262 | $11,126 | | Cost of license revenues | ($656) | ($234) | | Cost of contract manufacturing revenues | — | ($7,227) | | Research and development expenses | ($35,383) | ($33,655) | | Selling, general and administrative expenses | ($13,500) | ($15,767) | | Total operating expenses | ($49,539) | ($56,883) | | Other income | $2,597 | $1,983 | | Other expense | ($2,185) | ($236) | | Loss from operations | ($43,865) | ($44,010) | | Non-operating items, net | $6,571 | ($11,341) | | Loss before income tax (expense) / benefit | ($37,294) | ($55,351) | | Income tax (expense) / benefit | ($425) | ($948) | | Net loss | ($37,719) | ($56,299) | | Basic and diluted net loss per ordinary share | ($0.69) | ($1.16) | | Weighted average shares used in computing basic and diluted net loss per ordinary share | 54,807,967 | 48,622,440 | Company Information This section provides an overview of uniQure's mission and pipeline, details investor relations, and outlines important forward-looking statements and associated risks About uniQure uniQure is a leading gene therapy company focused on developing single treatments with potentially curative results for severe medical needs, building on its success with a gene therapy for hemophilia B and advancing a pipeline for Huntington's disease, epilepsy, ALS, and Fabry disease - uniQure is delivering on the promise of gene therapy – single treatments with potentially curative results[15] - Achieved a major milestone with the approval of its gene therapy for hemophilia B (HEMGENIX®)[15] - Advancing a pipeline of proprietary gene therapies for the treatment of patients with Huntington's disease, refractory temporal lobe epilepsy, ALS, Fabry disease, and other severe diseases[15] Investor Relations and Contacts uniQure hosted an investor conference call and webcast on July 29, 2025, and provides contact information for investor and media inquiries - Hosted an investor conference call and webcast on Tuesday, July 29, 2025, at 8:30 a.m. ET[14] - The event webcast replay will be archived for 90 days under the Events & Presentations section of uniQure's website[14] - Contact information for investors (Chiara Russo) and media (Tom Malone) is provided[18][19] Forward-Looking Statements This section outlines forward-looking statements regarding the company's future expectations, including cash runway, regulatory interactions, clinical trial timing, commercialization plans, and potential risks and uncertainties that could cause actual results to differ materially - Forward-looking statements include those concerning the Company's cash runway, timing and outcome of regulatory interactions for AMT-130, plans for clinical updates (AMT-130, AMT-191, AMT-162), and commercialization plans[16] - Actual results could differ materially due to risks and uncertainties such as clinical trial results, regulatory approvals, ability to build infrastructure, intellectual property, and funding[16][17] - These risks and uncertainties are more fully described in the Company's periodic filings with the U.S. Securities & Exchange Commission (SEC), including its Annual Report on Form 10-K and Quarterly Report on Form 10-Q[17]