Core Insights - uniQure reported a quarterly loss of $1.38 per share, which was worse than the Zacks Consensus Estimate of a loss of $0.85, marking a 62.35% earnings surprise [1] - The company's revenues for the quarter were $3.7 million, missing the Zacks Consensus Estimate by 46.62%, but showing an increase from $2.29 million a year ago [2] - The stock has gained approximately 57.4% year-to-date, outperforming the S&P 500's gain of 14.4% [3] Financial Performance - The company has surpassed consensus EPS estimates two times over the last four quarters [2] - The current consensus EPS estimate for the upcoming quarter is -$0.40 on revenues of $40.73 million, and for the current fiscal year, it is -$3.12 on revenues of $39.82 million [7] Market Outlook - The sustainability of the stock's price movement will depend on management's commentary during the earnings call [3] - The Zacks Rank for uniQure is currently 3 (Hold), indicating expected performance in line with the market in the near future [6] - The Medical - Biomedical and Genetics industry is currently in the top 36% of Zacks industries, suggesting a favorable outlook compared to the bottom 50% [8]

Financial Performance - Total revenues for Q3 2025 were $3.701 million, a 62% increase from $2.287 million in Q3 2024[15] - License revenues increased to $3.582 million in Q3 2025 from $2.111 million in Q3 2024, representing a 70% growth[15] - Net loss for Q3 2025 was $80.529 million, compared to a net loss of $44.378 million in Q3 2024, reflecting an increase in losses of 81%[15] - Total revenue for the nine months ended September 30, 2025, was $10.5 million, down from $21.9 million in the same period in 2024, indicating a 52% decrease[91] - The company reported a net loss of $161.9 million for the nine months ended September 30, 2025, compared to a net loss of $166.3 million for the same period in 2024[167] Assets and Liabilities - Total assets as of September 30, 2025, were $888.382 million, up from $556.536 million as of December 31, 2024, indicating a 59% increase[13] - Total current liabilities increased to $100.632 million as of September 30, 2025, from $40.053 million at the end of 2024, a 151% rise[13] - Shareholders' equity increased to $228.746 million as of September 30, 2025, from a deficit of $6.752 million at the end of 2024[13] - The accumulated deficit as of September 30, 2025, was $1.29 billion, compared to $1.06 billion as of September 30, 2024, indicating an increase in the deficit of 21.8%[20] Cash Flow - Cash and cash equivalents rose significantly to $597.056 million as of September 30, 2025, compared to $158.930 million at the end of 2024, marking a 276% increase[13] - Cash used in operating activities for the nine months ended September 30, 2025, was $100.4 million, an improvement from $129.9 million in the same period of 2024[22] - The total cash, cash equivalents, and restricted cash at the end of the period was $598.5 million, up from $253.1 million at the end of September 2024, reflecting a significant increase of 135.1%[22] - The company generated $119.8 million in net cash from investing activities for the nine months ended September 30, 2025, compared to $189.6 million in 2024, indicating a decrease of 36.8%[22] Research and Development - Research and development expenses for the nine months ended September 30, 2025, were $105.889 million, slightly up from $104.942 million in the same period of 2024[15] - Direct research and development expenses for Huntington's disease (AMT-130) rose to $30.6 million in 2025 from $9.8 million in 2024, an increase of $20.8 million[177] - The pivotal Phase I/II study of AMT-130 for Huntington's disease demonstrated a statistically significant 75% slowing of disease progression as measured by cUHDRS[113] - AMT-130 received Breakthrough Therapy designation from the FDA in April 2025, indicating potential for meaningful improvement over existing therapies[115] Shareholder Information - The number of ordinary shares outstanding increased to 62,165,900 as of September 30, 2025, from 48,988,087 at the end of 2024[13] - The company raised $70.1 million through a follow-on public offering of 4.4 million ordinary shares at a price of $17.00 per share in January 2025[73] - In September 2025, the Company received net proceeds of $323.7 million from a follow-on public offering of 6.7 million ordinary shares at a price of $47.50 per share[76] - The company has authorized an additional 2,400,000 shares for issuance under the 2014 Share Incentive Plan as of June 2025[78] Financing Activities - The Company received an upfront payment of $375.0 million from the Royalty Financing Agreement with HemB SPV, L.P., in exchange for rights to certain royalties[67] - The net proceeds from the Royalty Financing Agreement were recorded as $370.1 million, with an effective interest rate expected to be between 10.5% and 12.0% per annum as of September 30, 2025[71] - The company entered into a $175.0 million senior secured term loan facility with Hercules Capital, with a floating interest rate of 9.70% as of September 30, 2025[118] Expenses - Total operating expenses for Q3 2025 were $54.2 million, an increase of 25.5% from $43.2 million in Q3 2024[142] - Selling, general and administrative expenses for the three months ended September 30, 2025, were $19.4 million, an increase from $11.6 million in the same period in 2024[156] - Personnel-related accruals increased to $26.5 million as of September 30, 2025, compared to $12.3 million on December 31, 2024[57] Taxation - The effective income tax rate for the three months ended September 30, 2025, was 12.0%, significantly lower than the enacted rate of 25.8% in the Netherlands[96] - The company recorded a current tax expense of $7.5 million related to Dutch operations for the three months ended September 30, 2025[99]

Financial Performance - uniQure reported a net loss of $80.5 million for Q3 2025, compared to a net loss of $44.4 million in Q3 2024, resulting in a basic and diluted loss per share of $1.38[18] - Revenue for the three months ended September 30, 2025, was $3.7 million, an increase from $2.3 million in the same period in 2024, driven by a $1.5 million increase in license revenues[10] - Total revenues for Q3 2025 were $3.701 million, a 62% increase from $2.287 million in Q3 2024[29] - Net loss for Q3 2025 was $80.529 million, compared to a net loss of $44.378 million in Q3 2024, indicating increased operational costs[29] - The company reported a basic and diluted net loss per ordinary share of $1.38 for Q3 2025, compared to $0.91 for Q3 2024[29] Cash and Assets - As of September 30, 2025, the company held cash, cash equivalents, and current investment securities of $694.2 million, up from $367.5 million as of December 31, 2024, primarily due to $404.2 million raised through public offerings[9] - Cash and cash equivalents as of September 30, 2025, were $597.056 million, significantly up from $158.930 million at the end of 2024[27] - Total assets increased to $888.382 million as of September 30, 2025, compared to $556.536 million at the end of 2024[27] - Total current liabilities rose to $100.632 million in Q3 2025, up from $40.053 million at the end of 2024, indicating increased financial obligations[27] Research and Development - Research and development expenses for Q3 2025 were $34.4 million, an increase from $30.6 million in Q3 2024, largely due to $6.6 million related to the preparation for the Biologics License Application submission for AMT-130[12] - Research and development expenses increased to $34.366 million in Q3 2025, compared to $30.595 million in Q3 2024, reflecting a focus on advancing clinical programs[29] - The pivotal Phase I/II study of AMT-130 for Huntington's disease showed a statistically significant 75% slowing in disease progression at 36 months, meeting the primary endpoint[4] - The key secondary endpoint of Total Functional Capacity (TFC) demonstrated a statistically significant 60% slowing of disease progression compared to an external control[6] - AMT-260 for mesial temporal lobe epilepsy is advancing, with additional clinical data expected in the first half of 2026[4] - AMT-191 for Fabry disease showed a 27- to 208-fold increase in α-Gal A activity in treated patients, with further data expected in the first half of 2026[4] Administrative Expenses - Selling, general and administrative expenses rose to $19.4 million in Q3 2025 from $11.6 million in Q3 2024, primarily due to increased employee-related expenses and professional fees[13] Future Plans - The company plans to urgently interact with the FDA to define next steps for AMT-130 following unexpected feedback during a pre-BLA meeting[3] - The company plans to progress AMT-130 in the U.S. and interact with the FDA regarding its development[23] - The anticipated completion of dosing in a fourth cohort evaluating high-dose AMT-130 is expected before year-end 2025[23] - The company aims to expand the first cohort in the AMT-260 study into mesial temporal lobe epilepsy and initiate a second cohort[23]

Core Insights - The third quarter of 2025 was significant for uniQure, presenting topline three-year data for AMT-130, a gene therapy for Huntington's disease, showing statistically significant slowing of disease progression [3][4] - The company raised approximately $323.7 million in net proceeds from a public offering, enhancing its financial position with cash and equivalents totaling $694.2 million as of September 30, 2025 [7][9] - uniQure plans to urgently interact with the FDA regarding AMT-130 following unexpected feedback that introduced uncertainty in the timing of the Biologics License Application (BLA) submission [4][8] Recent Developments - AMT-130 for Huntington's disease met primary and key secondary endpoints, showing a 75% slowing in disease progression and a 60% slowing in Total Functional Capacity at 36 months [5][4] - Enrollment for AMT-260 in mesial temporal lobe epilepsy has commenced, with updated data expected in the first half of 2026 [6][8] - Initial data for AMT-191 in Fabry disease showed a 27- to 208-fold increase in α-Gal A enzyme activity, with further results anticipated in 2026 [6][8] Financial Performance - Revenue for Q3 2025 was $3.7 million, up from $2.3 million in Q3 2024, primarily due to increased license revenues [10] - Research and development expenses rose to $34.4 million in Q3 2025 from $30.6 million in Q3 2024, driven by preparations for the BLA submission for AMT-130 [12] - The net loss for Q3 2025 was $80.5 million, compared to a net loss of $44.4 million in the same period in 2024, reflecting increased operating expenses and non-operating items [17][28]

Group 1 - U.S. stock futures are higher, with Nasdaq futures gaining approximately 1.5% [1] - Monday.Com Ltd is expected to report quarterly earnings of 88 cents per share, an increase from 85 cents per share year-over-year [2] - The consensus estimate for monday.com's quarterly revenue is $312.26 million, up from $251 million a year earlier [2] Group 2 - Monday.Com shares fell 2.6% to $184.70 in pre-market trading [2] - Metsera Inc shares dipped 15% to $70.68 in pre-market trading following Pfizer's $10 billion acquisition deal [4] - Ionis Pharmaceuticals Inc declined 11.5% to $65.00 after disclosing results from pivotal Phase 3 studies [4] - Other companies such as UniQure NV, Oscar Health Inc, Centene Corp, and Immunovant Inc also experienced declines in pre-market trading [4]

Group 1 - U.S. stock futures are higher, with Nasdaq futures gaining approximately 1.5% on Monday [1] - Monday.Com Ltd is expected to report quarterly earnings of 88 cents per share, an increase from 85 cents per share in the previous year [2] - The consensus estimate for monday.com's quarterly revenue is $312.26 million, up from $251 million a year earlier [2] Group 2 - Monday.Com shares fell 2.6% to $184.70 in pre-market trading [2] - Metsera Inc shares dipped 15% to $70.68 after Pfizer secured a $10 billion deal to acquire the company [4] - Ionis Pharmaceuticals Inc declined 11.5% to $65.00 following the disclosure of pivotal Phase 3 study results [4] - Other companies such as UniQure NV, Oscar Health Inc, Centene Corp, and Immunovant Inc also experienced declines in pre-market trading [4]

Core Viewpoint - uniQure N.V. is set to report its third quarter 2025 financial results on November 10, 2025, highlighting its ongoing commitment to advancing gene therapies for severe medical conditions [1]. Company Overview - uniQure is recognized for its pioneering work in gene therapy, particularly with its approved treatment for hemophilia B, marking a significant achievement in genomic medicine [3]. - The company is developing a pipeline of proprietary gene therapies targeting various severe diseases, including Huntington's disease, refractory temporal lobe epilepsy, ALS, and Fabry disease [3]. Event Details - The earnings call will take place at 8:30 a.m. ET and will be accessible via webcast on uniQure's website, with a replay available for 90 days post-event [2]. - Analysts can participate in the Q&A session by dialing the provided numbers and entering the specified passcode [2].

Core Viewpoint - Bragar Eagel & Squire, P.C. is investigating potential claims against uniQure N.V. regarding possible violations of federal securities laws and unlawful business practices following a significant drop in share price due to FDA concerns about the adequacy of data for a BLA submission [1][2]. Investigation Details - On November 3, 2025, uniQure announced that the FDA no longer believes that the data from Phase I/II studies of AMT-130 is sufficient for a BLA submission, leading to a share price decline of over 57% in morning trading on the same day [2]. Next Steps - Investors who purchased uniQure shares and experienced losses are encouraged to contact Bragar Eagel & Squire for more information regarding their rights and potential claims [3]. About Bragar Eagel & Squire, P.C. - Bragar Eagel & Squire, P.C. is a law firm recognized nationally, representing individual and institutional investors in various types of litigation across state and federal courts [4].

Core Points - U.S. stock futures are lower, with Dow futures falling over 50 points [1] - Super Micro Computer Inc reported disappointing first-quarter results, with earnings of 35 cents per share, missing the consensus estimate of 40 cents, and revenue of $5.01 billion, below the expected $5.99 billion [2] - Super Micro's revenue decreased from $5.93 billion in the same period last year [2] - The company's shares fell 9.2% to $43.12 in pre-market trading [2] Company-Specific Movements - Biohaven Ltd experienced a significant drop of 46.2% to $7.51 after receiving an FDA Complete Response Letter for VYGLXIA [4] - Trex Company Inc declined 31.9% to $32.02 following worse-than-expected third-quarter results and lowered FY25 sales guidance [4] - Soleno Therapeutics Inc fell 22.2% to $49.65 after reporting third-quarter results [4] - Axon Enterprise Inc's shares dropped 19.6% to $567.50 after mixed third-quarter results and an acquisition announcement [4] - Pinterest Inc dipped 18.8% to $26.69 after posting disappointing third-quarter earnings [4] - SSR Mining Inc fell 17.5% to $17.90 after its third-quarter results [4] - Uniqure NV's shares decreased by 15.8% to $25.61 after a prior gain [4] - Clover Health Investments Corp fell 15.5% to $2.97 after mixed third-quarter results [4] - Upstart Holdings Inc declined 15.3% to $39.15 after mixed results and lowered FY25 guidance [4] - Corsair Gaming Inc fell 12.4% to $6.59 following worse-than-expected third-quarter results [4] - Advanced Micro Devices, Inc. declined 5% to $237.80 despite reporting better-than-expected third-quarter results and projecting fourth-quarter revenue of approximately $9.6 billion [4]

Core Viewpoint - Kirby McInerney LLP is investigating potential claims against uniQure N.V. regarding possible violations of federal securities laws or unlawful business practices [1][3]. Group 1: Company Disclosure - On November 3, 2025, uniQure announced that the FDA no longer agrees that data from Phase I/II studies of AMT-130 may be adequate for a Biologics License Application (BLA) submission [3]. - The timing of the BLA submission for AMT-130 is now unclear following the FDA's stance [3]. Group 2: Market Reaction - Following the announcement, uniQure's share price fell by $33.40, a decline of approximately 49.33%, from $67.69 on October 31, 2025, to $34.29 on November 3, 2025 [3].