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Financial Data and Key Metrics Changes - Revenue for Q3 2025 was $3.7 million, an increase from $2.3 million in Q3 2024, resulting in a $1.5 million increase in license revenues and a slight decrease in collaboration revenues [17] - Research and development expenses rose to $34.4 million in Q3 2025 from $30.6 million in Q3 2024, driven by a $10.1 million increase in direct R&D expenses, primarily for BLA submission preparation [18] - Selling, general, and administrative expenses increased to $19.4 million in Q3 2025 from $11.6 million in Q3 2024, mainly due to higher employee-related expenses and professional fees [18] - Cash, cash equivalents, and investment securities totaled $649.2 million as of September 30, 2025, up from $376.5 million at the end of 2024, primarily due to net proceeds from public offerings [19] Business Line Data and Key Metrics Changes - AMT-130 for Huntington's disease showed a statistically significant 75% slowing of disease progression at three years compared to an external control, meeting the pivotal study's primary endpoint [9] - AMT-260 for mesial temporal lobe epilepsy has activated 17 recruiting sites in the U.S. and completed enrollment of the first three patients in the first cohort, with updated data expected in the first half of 2026 [11] - AMT-191 for Fabry disease demonstrated supra-physiological alpha-galA enzyme activity in treated patients, with all successfully withdrawn from enzyme replacement therapy [12] Market Data and Key Metrics Changes - The company is focusing on building a foundational strategy for the U.S. market for AMT-130 while also exploring potential markets in the European Union and the United Kingdom [16] Company Strategy and Development Direction - The company remains committed to working with the FDA to clarify next steps for AMT-130 and is also planning discussions with other regulatory agencies [20] - The management emphasizes the importance of stakeholder engagement and education to prepare for a potential launch of AMT-130 [15] Management's Comments on Operating Environment and Future Outlook - Management expressed disappointment regarding the FDA's feedback on the BLA submission for AMT-130 but remains confident in the data and its potential to benefit patients [5][10] - The company plans to engage urgently with the FDA to discuss the next steps and remains committed to transparency and collaboration [6][11] Other Important Information - Enrollment in the phase I-II trial of AMT-162 for SOD1 ALS has been paused due to safety concerns, with ongoing data collection from treated patients [13] Q&A Session Summary Question: Treatment effect and external control arm construction - The company conducted rigorous propensity score matching with Enroll-HD and performed various sensitivity analyses to ensure robustness of findings [27] Question: Details on AMT-162 and dose differences - The middle dose of AMT-162 was about three-fold higher than the low dose, and the company is monitoring data to determine next steps [31] Question: Expectations from final meeting minutes with the FDA - The company hopes the minutes will outline FDA concerns and provide guidance on addressing those in future meetings [34] Question: Confirmation of prior meeting minutes regarding accelerated approval - The company confirmed that previous meetings indicated the data could support a BLA submission and that the composite UHDRS was an acceptable endpoint [37] Question: Future investment in Huntington's program - The company is committed to collaborating with the FDA to find an expedited path for BLA submission, emphasizing the urgency of patient needs [41] Question: EMA or MHRA feedback on AMT-130 - The company has not yet engaged with EMA or MHRA but plans to prioritize discussions with the FDA first [47]

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].