uniQure(QURE) - 2025 Q1 - Quarterly Results

Executive Summary & Recent Progress First Quarter 2025 Highlights uniQure announced its Q1 2025 financial results and highlighted significant progress, including FDA Breakthrough Therapy designation for AMT-130, positive safety data from its Phase I/II study, and a strong cash position expected to fund operations into the second half of 2027. - AMT-130 granted Breakthrough Therapy designation by FDA[1] - Initial safety data from the third cohort of the Phase I/II study indicate AMT-130 continues to be generally well-tolerated, with no treatment-related serious adverse events reported[1] - Cash, cash equivalents, and current investment securities totaled approximately $409.0 million as of March 31, 2025, projected to fund operations into the second half of 2027[1] CEO's Statement CEO Matt Kapusta highlighted constructive interactions with the FDA for AMT-130, emphasizing the significance of its Breakthrough Therapy designation and upcoming regulatory and clinical data updates. He also noted continued progress across the broader gene therapy pipeline and the company's strong financial position. - CEO anticipates providing a regulatory update in Q2 2025, including guidance on potential timing of BLA submission for AMT-130, and expects to share new clinical data from AMT-130 Phase I/II trials in Q3 2025[3] - Initial data for AMT-260 (refractory mesial temporal lobe epilepsy) from the first treated patient is expected later this quarter, with AMT-191 (Fabry disease) initial data on track for the second half of the year[3] Recent Company Developments and Updates Advancing AMT-130 Toward Accelerated Approval for Huntington's disease uniQure has made significant strides with AMT-130, securing FDA Breakthrough Therapy designation and engaging in Type B meetings to advance Biologics License Application (BLA) preparations. Clinical trials show AMT-130 is generally well-tolerated, with further data expected in Q3 2025. - In April 2025, the FDA granted Breakthrough Therapy designation to AMT-130, based on Phase I/II clinical evidence indicating potential to slow Huntington's disease progression[4] - In March 2025 and Q2 2025, the Company conducted Type B meetings with the FDA to discuss CMC requirements and the pivotal statistical analysis plan for a planned BLA submission[7] - In February 2025, enrollment of all 12 patients in the third cohort of the Phase I/II study was completed, with AMT-130 generally well-tolerated at both dose levels and no treatment-related serious adverse events reported[7] Advancing additional clinical programs to proof-of-concept uniQure is progressing its pipeline with upcoming data presentations for AMT-260 (refractory mTLE) in May 2025 and AMT-191 (Fabry disease) in H2 2025, following favorable safety reviews. Enrollment for AMT-162 (SOD1 ALS) is also advancing, with initial data anticipated in H1 2026. - Initial safety and exploratory efficacy data from the first treated patient for AMT-260 (refractory mTLE) are expected to be presented at the Epilepsy Therapies & Diagnostics Development Symposium on May 29, 2025[7] - For AMT-191 (Fabry disease), a favorable recommendation from the independent data monitoring committee (IDMC) was announced in February 2025, with initial study data planned for the second half of 2025[7] - For AMT-162 (SOD1 ALS), enrollment in a second dose cohort is complete, with the third dose cohort expected in Q3 2025, and initial study data anticipated in the first half of 2026[7][8] Financial Highlights Cash Position uniQure's cash, cash equivalents, and current investment securities increased to $409.0 million as of March 31, 2025, primarily due to $80.5 million net proceeds from a follow-on offering, extending its operational funding into the second half of 2027. Cash, Cash Equivalents and Current Investment Securities | Date | Amount (Millions USD) | | :--- | :--- | | March 31, 2025 | $409.0 | | December 31, 2024 | $367.5 | - The increase in cash was primarily driven by net proceeds of $80.5 million from a follow-on offering in January and February 2025[9][16] - Based on the current operating plan, cash, cash equivalents, and current investment securities are projected to fund operations into the second half of 2027[9] Revenues Total revenues for the first quarter of 2025 significantly decreased to $1.6 million from $8.5 million in the prior year, primarily due to reductions in collaboration revenue and contract manufacturing revenue for HEMGENIX®. Total Revenues (Three months ended March 31) | Year | Amount (Millions USD) | Change (YoY) | | :--- | :--- | :--- | | 2025 | $1.6 | -$6.9 million | | 2024 | $8.5 | - | - The $6.9 million decrease in revenue stemmed from a $0.4 million increase in license revenue, a $3.3 million decrease in collaboration revenue, and a $4.0 million decrease in contract manufacturing revenue for HEMGENIX®[10] Operating Expenses Total operating expenses decreased by $16.6 million to $47.2 million in Q1 2025, driven by the nil cost of contract manufacturing revenues (due to facility divestment), and reductions in R&D and SG&A expenses. Total Operating Expenses (Three months ended March 31) | Year | Amount (Millions USD) | Change (YoY) | | :--- | :--- | :--- | | 2025 | $47.2 | -$16.6 million | | 2024 | $63.9 | - | Cost of Contract Manufacturing Revenues Cost of contract manufacturing revenues was nil for Q1 2025, a $9.1 million decrease from Q1 2024, following the divestment of the Lexington facility in July 2024. Cost of Contract Manufacturing Revenues (Three months ended March 31) | Year | Amount (Millions USD) | Change (YoY) | | :--- | :--- | :--- | | 2025 | $0.0 | -$9.1 million | | 2024 | $9.1 | - | - Following the divestment of the Lexington facility in July 2024, the cost of contract manufacturing is recorded net of revenue within other expenses[11] Research and Development (R&D) Expenses R&D expenses decreased by $4.6 million to $36.1 million in Q1 2025, primarily due to lower employee-related and facility expenses, partially offset by increased external program spend. R&D Expenses (Three months ended March 31) | Year | Amount (Millions USD) | Change (YoY) | | :--- | :--- | :--- | | 2025 | $36.1 | -$4.6 million | | 2024 | $40.7 | - | - The decrease was primarily attributed to a $7.7 million reduction in employee-related expenses, a $2.5 million decrease in facility expenses, and a $1.1 million decrease in preclinical supply costs, partially offset by a $7.1 million net increase in external program spend and $1.1 million higher expenses from an increase in the fair value of contingent consideration[12] Selling, General and Administrative (SG&A) Expenses SG&A expenses decreased by $3.0 million to $10.9 million in Q1 2025, mainly due to reductions in employee-related expenses and intellectual property fees. SG&A Expenses (Three months ended March 31) | Year | Amount (Millions USD) | Change (YoY) | | :--- | :--- | :--- | | 2025 | $10.9 | -$3.0 million | | 2024 | $13.9 | - | - The decrease was primarily due to a $2.2 million reduction in employee-related expenses and a $0.3 million decrease in intellectual property fees[13] Other Income and Expense Other income significantly increased in Q1 2025 due to a one-time gain, while other expense also rose. Non-operating items, net, showed a reduced expense compared to the prior year, driven by lower interest expense and higher foreign currency gains. Other Income Other income increased by $6.9 million to $8.3 million in Q1 2025, primarily driven by a one-time gain of $6.0 million from the sale of critical reagents. Other Income (Three months ended March 31) | Year | Amount (Millions USD) | Change (YoY) | | :--- | :--- | :--- | | 2025 | $8.3 | +$6.9 million | | 2024 | $1.4 | - | - The increase was primarily attributable to a one-time gain of $6.0 million from the sale of critical reagents[14] Other Expense Other expense increased to $2.0 million in Q1 2025 from $0.2 million in Q1 2024, mainly due to a $1.4 million non-cash expense for amortizing the right to purchase HEMGENIX®. Other Expense (Three months ended March 31) | Year | Amount (Millions USD) | Change (YoY) | | :--- | :--- | :--- | | 2025 | $2.0 | +$1.8 million | | 2024 | $0.2 | - | - The increase was primarily due to $1.4 million in non-cash expense recognized for amortizing the right to purchase HEMGENIX® from Genezen on favorable terms[15] Other Non-Operating Items, Net Other non-operating items, net, decreased as an expense by $6.9 million to $3.8 million in Q1 2025, driven by a reduction in interest expense and an increase in net foreign currency gains. Other Non-Operating Items, Net (Three months ended March 31) | Year | Amount (Millions USD) | Change (YoY) | | :--- | :--- | :--- | | 2025 | -$3.8 (expense) | +$6.9 million (less expense) | | 2024 | -$10.7 (expense) | - | - The $6.9 million decrease in other non-operating items, net, was primarily due to a $2.4 million decrease in interest income, a $1.0 million reduction in interest expense from the $50.0 million repayment of Hercules debt in July 2024, and an $8.3 million increase in net foreign currency gains[17] Net Loss and EPS uniQure significantly narrowed its net loss for Q1 2025 to $43.6 million, or $0.82 per ordinary share, compared to a $65.6 million net loss, or $1.36 per ordinary share, in Q1 2024. Net Loss (Three months ended March 31) | Year | Amount (Millions USD) | Basic and Diluted Loss Per Ordinary Share | | :--- | :--- | :--- | | 2025 | $43.6 | $0.82 | | 2024 | $65.6 | $1.36 | Corporate Information Investor Conference Call and Webcast Information uniQure hosted an investor conference call and webcast on May 9, 2025, at 8:30 a.m. ET, with replay available on their website for 90 days. - uniQure management hosted an investor conference call and webcast on Friday, May 9, 2025, at 8:30 a.m. ET[19] - The event was webcast under the Events & Presentations section of uniQure's website, with a replay archived for 90 days[19] About uniQure uniQure is a leading gene therapy company focused on developing single treatments for severe medical needs, having achieved a historic milestone with its approved hemophilia B gene therapy and advancing a pipeline for Huntington's disease, epilepsy, ALS, and Fabry disease. - uniQure is a leading gene therapy company developing transformative therapies for patients with severe medical needs[20] - The approvals of uniQure's gene therapy for hemophilia B represent a major milestone in genomic medicine[20] - uniQure is advancing a pipeline of proprietary gene therapies for Huntington's disease, refractory temporal lobe epilepsy, ALS, Fabry disease, and other severe diseases[20] Forward-Looking Statements This section contains forward-looking statements regarding uniQure's future expectations, including its cash runway, regulatory outcomes for AMT-130, commercialization plans, and clinical updates for various programs. It also highlights inherent risks and uncertainties that could cause actual results to differ materially from these projections. - Forward-looking statements encompass the Company's cash runway into the second half of 2027, regulatory outcomes for AMT-130, commercialization plans, and clinical updates for AMT-130, AMT-260, AMT-191, and AMT-162 programs[21] - Actual results may differ materially due to risks and uncertainties related to clinical results, regulatory interactions, infrastructure development, clinical trial management effectiveness, continued development and acceptance of gene therapies, intellectual property, and funding operations[21][22] Contacts Contact information is provided for investor relations (Chiara Russo) and media inquiries (Tom Malone). - For investors, contact Chiara Russo at c.russo@uniQure.com[24] - For media, contact Tom Malone at t.malone@uniQure.com[24] Unaudited Consolidated Financial Statements Unaudited Consolidated Balance Sheets As of March 31, 2025, uniQure's total assets increased to $605.4 million, driven by a rise in cash and cash equivalents. Total liabilities also increased, but shareholders' equity moved from a deficit to a positive balance, reflecting improved financial positioning. Key Balance Sheet Figures (in thousands USD) | Item | March 31, 2025 | December 31, 2024 | Change | | :--- | :--- | :--- | :--- | | Cash and cash equivalents | $217,229 | $158,930 | +$58,299 | | Current investment securities | $191,782 | $208,591 | -$16,809 | | Total current assets | $435,421 | $390,289 | +$45,132 | | Total assets | $605,403 | $556,536 | +$48,867 | | Total current liabilities | $36,310 | $40,053 | -$3,743 | | Total liabilities | $571,717 | $563,288 | +$8,429 | | Total shareholders' equity / (deficit) | $33,686 | $(6,752) | +$40,438 | Unaudited Consolidated Statements of Operations uniQure reported a significantly reduced net loss of $43.6 million for Q1 2025, down from $65.6 million in Q1 2024. This improvement was primarily due to lower operating expenses and higher other income, despite a decrease in total revenues. Key Income Statement Figures (in thousands USD, three months ended March 31) | Item | 2025 | 2024 | Change (YoY) | | :--- | :--- | :--- | :--- | | Total revenues | $1,567 | $8,485 | -$6,918 | | Total operating expenses | $47,245 | $63,855 | -$16,610 | | Other income | $8,306 | $1,376 | +$6,930 | | Other expense | $1,959 | $234 | +$1,725 | | Loss from operations | $39,331 | $54,228 | -$14,897 | | Non-operating items, net | $3,810 | $10,734 | -$6,924 | | Net loss | $43,637 | $65,618 | -$21,981 | | Basic and diluted net loss per ordinary share | $0.82 | $1.36 | -$0.54 | | Weighted average shares | 53,110,580 | 48,384,510 | +4,726,070 |