Latest news with #uniQure
Business Insider
4 days ago
- Business
- Business Insider
Leerink Partners Keeps Their Buy Rating on uniQure (QURE)
Leerink Partners analyst Joseph Schwartz reiterated a Buy rating on uniQure (QURE – Research Report) on June 3 and set a price target of $48.00. The company's shares closed yesterday at $14.96. Confident Investing Starts Here: Schwartz covers the Healthcare sector, focusing on stocks such as uniQure, KalVista Pharmaceuticals, and Sarepta Therapeutics. According to TipRanks, Schwartz has an average return of 10.5% and a 42.05% success rate on recommended stocks. Currently, the analyst consensus on uniQure is a Strong Buy with an average price target of $38.40, implying a 156.68% upside from current levels. In a report released on June 3, H.C. Wainwright also reiterated a Buy rating on the stock with a $70.00 price target. Based on uniQure's latest earnings release for the quarter ending March 31, the company reported a quarterly revenue of $1.57 million and a GAAP net loss of $43.64 million. In comparison, last year the company earned a revenue of $8.49 million and had a GAAP net loss of $65.62 million Based on the recent corporate insider activity of 37 insiders, corporate insider sentiment is negative on the stock. This means that over the past quarter there has been an increase of insiders selling their shares of QURE in relation to earlier this year. Most recently, in March 2025, Jeannette Potts, the CLO of QURE sold 7,076.00 shares for a total of $72,812.04.
Associated Press
7 days ago
- Business
- Associated Press
uniQure Provides Regulatory Update on AMT-130 for Huntington's Disease
~ Alignment with FDA continues to support Accelerated Approval pathway ~ ~ BLA submission planned for first quarter of 2026 ~ ~ Conference call today at 8:30 a.m. ET ~ LEXINGTON, Mass. and AMSTERDAM, June 02, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today provided a regulatory update on AMT-130, its investigational gene therapy for the treatment of Huntington's disease. Following recent Type B meetings and further guidance from the U.S. Food and Drug Administration (FDA), the Company has reached alignment with the FDA on several key components of the statistical analysis plan and Chemistry, Manufacturing and Controls (CMC) information that will support a Biologics License Application (BLA) submission expected in the first quarter of 2026. 'We are very pleased with our continued, productive engagement with the FDA and the progress we've made toward a planned BLA submission for AMT-130 in the first quarter of 2026,' said Walid Abi-Saab, M.D., chief medical officer of uniQure. 'We are pursuing an accelerated approval pathway supported by multiple years of clinical data – a rigorous and differentiated approach that reflects the urgent need in Huntington's disease and our commitment to delivering the first disease-modifying treatment for people affected by this devastating disease. We are grateful to the FDA for their continued engagement and look forward to sharing three-year top-line data in the third quarter of 2025.' Statistical Analysis Plan In the second quarter of 2025, the Company held a Type B meeting with the FDA to discuss the proposed use of external control data and the prospectively defined statistical analysis plan (SAP) in support of the planned BLA submission for AMT-130. The FDA continued to support its prior agreement that the composite Unified Huntington's Disease Rating Scale (cUHDRS) may serve as an acceptable registrational, intermediate clinical endpoint for accelerated approval. The FDA agreed that the primary efficacy analysis for the BLA will evaluate the 3-year change in cUHDRS in high-dose AMT-130 patients compared to a propensity score-adjusted external control arm. The Company plans to use propensity score-weighted external control derived from the ENROLL-HD dataset for the primary analysis and to submit certain sensitivity analyses, including one using a propensity score-matched external control, as additional support. The FDA also agreed that ENROLL-HD – a large, prospective, longitudinal, natural history study of patients with Huntington's disease – may be acceptable as the external control dataset for the primary analysis of the trial data along with additional sensitivity analyses using the TRACK-HD/TRACK-ON and PREDICT-HD datasets. To date, approximately 33,000 patients have enrolled in ENROLL-HD. Compared to previously used natural history studies like TRACK-HD and PREDICT-HD, ENROLL-HD offers a substantially larger sample size, lower attrition rates, and longer average patient follow-up. The Company expects the larger sample size to reduce variability in the external control data and enhance the robustness of the SAP. The Company plans to submit an updated SAP consistent with discussions from the recently held Type B meeting to the FDA in the second quarter of 2025. Chemistry, Manufacturing and Controls (CMC) In the first quarter of 2025, the Company held a Type B meeting with the FDA to discuss CMC requirements in support of the planned BLA submission for AMT-130. The FDA agreed that validation of the AMT-130 manufacturing process should be possible using experience and prior knowledge from the etranacogene dezaparvovec-drlb (HEMGENIX®) process, complemented with additional full-scale AMT-130 GMP batches and a single Process Performance Qualification (PPQ) batch. The FDA also agreed with the Company's proposed drug product release testing plan, including the proposed potency assay, pending completion of qualification and specification setting activities. Next Steps for the Planned BLA Submission Based on these recent Type B meetings with the FDA, the Company continues to prepare for a BLA submission for AMT-130 in the treatment of Huntington's disease. Certain key next steps and expected timing include: Investor Conference Call and Webcast Information uniQure management will host an investor conference call and webcast today, Monday, June 2, 2025 at 8:30 a.m. ET. The event will be webcast under the Events & Presentations section of uniQure's website at and following the event a replay will be archived for 90 days. Interested parties participating by phone will need to register using this online form. After registering for dial-in details, all phone participants will receive an auto-generated e-mail containing a link to the dial-in number along with a personal PIN number to use to access the event by phone. If you are joining the conference call, please dial in 15 minutes before the start time. About the Phase I/II Clinical Program of AMT-130 uniQure is conducting two multi-center, dose-escalating, Phase I/II clinical studies to explore the safety, tolerability, and exploratory efficacy signals of AMT-130 for the treatment of Huntington's disease. In the U.S. study, a total of 26 patients with early manifest Huntington's disease were randomized to treatment (n=6 low dose; n=10 high dose) or an imitation (sham) surgical procedure (n=10). Treated patients received a single administration of AMT-130 through MRI-guided, convection-enhanced stereotactic neurosurgical delivery directly into the striatum (caudate and putamen). The study consists of a blinded 12-month core study period followed by unblinded long-term follow-up of treated patients for five years. An additional four control patients crossed over to treatment. The European open-label Phase Ib/II study of AMT-130 enrolled 13 patients with early manifest Huntington's disease (n=6 low dose; n=7 high dose). A third cohort enrolled an additional 12 patients across sites in the U.S. and EU. This cohort was randomized to explore both doses of AMT-130 in combination with immunosuppression, using the current, established stereotactic administration procedure. Additional details are available on (NCT0543017, NCT04120493) AMT-130 has been granted the FDA's Regenerative Medicine Advance Therapy (RMAT) designation and Breakthrough Therapy designation, the first therapy for Huntington's disease to receive an RMAT designation. About Huntington's Disease Huntington's disease is a rare, inherited neurodegenerative disorder that leads to motor symptoms including chorea, behavioral abnormalities and cognitive decline resulting in progressive physical and mental deterioration. The disease is an autosomal dominant condition with a disease-causing CAG repeat expansion in the first exon of the huntingtin gene that leads to the production and aggregation of abnormal protein in the brain. According to 2021 study in Neuroepidemiology, approximately 70,000 people have been diagnosed with Huntington's disease in the U.S. and Europe, with hundreds of thousands of others at risk of inheriting the disease. Despite the clear etiology of Huntington's disease, there are currently no approved therapies to delay the onset or to slow the disease's progression. About uniQure uniQure is delivering on the promise of gene therapy – single treatments with potentially curative results. The approvals of uniQure's gene therapy for hemophilia B – an historic achievement based on more than a decade of research and clinical development – represent a major milestone in the field of genomic medicine and ushers in a new treatment approach for patients living with hemophilia. uniQure is now 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. uniQure Forward-Looking Statements This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as 'anticipate,' 'believe,' 'could,' 'establish,' 'estimate,' 'expect,' 'goal,' 'intend,' 'look forward to', 'may,' 'plan,' 'potential,' 'predict,' 'project,' 'seek,' 'should,' 'will,' 'would' and similar expressions and the negatives of those terms. Forward-looking statements are based on management's beliefs and assumptions and on information available to management as of the date of this press release. Examples of these forward-looking statements include, but are not limited to, statements concerning: the availability of accelerated approval pathways and the need for additional pre-approval studies for AMT-130; the Company's anticipated timing of the BLA submission; the Company's plans to submit a revised SAP and CMC information to the FDA; the Company's ability to deliver a potentially life-changing therapy to people living with Huntington's disease and related timeline for doing so; the potential clinical and functional effects of AMT-130; the Company's plans to continue clinical development of AMT-130; the Company's plans to share clinical data of AMT-130 in the third quarter of 2025; and the utility of the ENROLL-HD patient dataset with respect to Phase I/II study. The Company may not actually achieve the plans, intentions or expectations disclosed in these forward-looking statements. Furthermore, the Company's actual results could differ materially from the plans, intentions and expectations anticipated in these forward-looking statements for many reasons. These risks and uncertainties include, among others: risks related to the Company's Phase I/ll clinical trials of AMT-130, including the risk that interim data from the trials may not be predictive of later data readouts that will serve as a basis for further regulatory interactions, may not support BLA submissions or accelerated approvals, may not be satisfactory to the FDA and other regulators, and new analyses of existing data and results may produce different conclusions than established as of the date hereof; risks related to the Company's current and future interactions with regulatory authorities, which may affect the initiation, timing and progress of clinical trials, its BLA submission plans and pathways to regulatory approval; risks related to the Company's ability to pursue business development efforts with respect to AMT-130; risks related to the Company's use of propensity-weighted external controls in connection with its statistical analysis of clinical outcomes to date; uncertainties as to the FDA's and other regulatory authorities' interpretation of the data from the Company's Phase I/ll clinical trials of AMT-130 and acceptance of the Company's clinical programs and the regulatory approval process;later developments with the FDA and other regulators that could be inconsistent with the feedback received to date; and whether regulatory authorities will accept the Company's approach as a basis for accelerated approval; risks related to the Company's use of nominal p values as a basis for its statistical analyses; whether the measurements that the Company is evaluating continue to be viewed as robust and sensitive measurements of disease progression; whether RMAT designation or any accelerated pathway, will lead to regulatory approval; the Company's ability to continue to build and maintain the infrastructure and personnel needed to achieve its goals; the Company's effectiveness in managing current and future clinical trials and regulatory processes; the Company's ability to demonstrate the therapeutic benefits of its gene therapy candidates in clinical trials; the continued development and acceptance of gene therapies; the Company's ability to obtain, maintain and protect its intellectual property; and the Company's ability to fund its operations and to raise additional capital as needed and on acceptable terms. These risks and uncertainties are more fully described under the heading 'Risk Factors' in the Company's periodic filings with the U.S. Securities & Exchange Commission (SEC), including its Annual Report on Form 10-K filed with the SEC on February 27, 2025, its Quarterly Reports on Form 10-Q filed May 9, 2025, and in other filings that the Company makes with the SEC from time to time. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements and, except as required by law, the Company assumes no obligation to update these forward-looking statements, even if new information becomes available in the future.
Business Insider
12-05-2025
- Business
- Business Insider
Analysts Are Bullish on These Healthcare Stocks: Iovance Biotherapeutics (IOVA), uniQure (QURE)
There's a lot to be optimistic about in the Healthcare sector as 2 analysts just weighed in on Iovance Biotherapeutics (IOVA – Research Report) and uniQure (QURE – Research Report) with bullish sentiments. Protect Your Portfolio Against Market Uncertainty Discover companies with rock-solid fundamentals in TipRanks' Smart Value Newsletter. Receive undervalued stocks, resilient to market uncertainty, delivered straight to your inbox. Iovance Biotherapeutics (IOVA) In a report issued on May 9, Peter Lawson from Barclays maintained a Buy rating on Iovance Biotherapeutics, with a price target of $4.00. The company's shares closed last Friday at $1.75. According to Lawson is currently ranked with 0 stars on a 0-5 stars ranking scale, with an average return of -10.1% and a 35.4% success rate. Lawson covers the Healthcare sector, focusing on stocks such as Springworks Therapeutics, Adaptimmune Therapeutics, and Karyopharm Therapeutics. Currently, the analyst consensus on Iovance Biotherapeutics is a Moderate Buy with an average price target of $13.83, a 612.9% upside from current levels. In a report issued on May 8, TD Cowen also maintained a Buy rating on the stock with a $5.00 price target. uniQure (QURE) Wells Fargo analyst Yanan Zhu maintained a Buy rating on uniQure on May 9 and set a price target of $30.00. The company's shares closed last Friday at $12.79. According to Zhu is ranked 0 out of 5 stars with an average return of -5.6% and a 26.4% success rate. Zhu covers the Healthcare sector, focusing on stocks such as Adaptimmune Therapeutics, Iovance Biotherapeutics, and Crispr Therapeutics AG. Currently, the analyst consensus on uniQure is a Strong Buy with an average price target of $36.86, representing a 203.4% upside. In a report issued on April 29, Leerink Partners also maintained a Buy rating on the stock with a $48.00 price target.
Yahoo
10-05-2025
- Business
- Yahoo
uniQure NV (QURE) Q1 2025 Earnings Call Highlights: Navigating Revenue Decline with Strategic ...
Revenue: $1.6 million for Q1 2025, down from $8.5 million in Q1 2024. Research and Development Expenses: $36.1 million, down from $40.7 million in Q1 2024. Selling, General, and Administrative Expenses: $10.9 million, down from $13.9 million in Q1 2024. Cash, Cash Equivalents, and Investment Securities: $409 million as of March 31, 2025, up from $367.5 million as of December 31, 2024. Cash Flow from Follow-On Offering: Net proceeds of $80.5 million in Q1 2025. Warning! GuruFocus has detected 6 Warning Signs with QURE. Release Date: May 09, 2025 For the complete transcript of the earnings call, please refer to the full earnings call transcript. uniQure NV (NASDAQ:QURE) reported significant progress in its Huntington's disease program, with AMT-130 receiving breakthrough therapy designation from the FDA. The company has a strong financial position with over $400 million in cash, providing flexibility to advance its pipeline. uniQure NV (NASDAQ:QURE) has expanded its clinical pipeline with new studies in refractory temporal lobe epilepsy, Fabry disease, and SOD1 ALS. The company successfully completed an $80 million follow-on offering, further strengthening its balance sheet. uniQure NV (NASDAQ:QURE) is on track for a planned BLA submission for AMT-130, with productive interactions with the FDA supporting this timeline. Revenue for the first quarter decreased significantly to $1.6 million from $8.5 million in the same period in 2024, primarily due to reduced collaboration and contract manufacturing revenue. There were three serious adverse events related to immunosuppression in Cohort 3 of the AMT-130 trial, although they were resolved with standard care. The company faces challenges in maintaining momentum with its pipeline, as evidenced by the need for ongoing regulatory interactions and data analysis. uniQure NV (NASDAQ:QURE) is still in the early stages of commercial planning for AMT-130, with details on market strategy and partnerships yet to be finalized. The company has not yet finalized its approach for a confirmatory study for AMT-130, which could impact future regulatory approvals. Q: How confident are you on the three-year follow-up data on the cUHDRS, and what would you consider a good outcome? A: Walid Abi-Saab, Chief Medical Officer, stated that they have been monitoring patients for a long time and have no indication of losing efficacy over time. They believe the dose-dependent reduction in cUHDRS observed at the two-year data will be maintained at three years, and they feel very confident about their results going forward. Q: Will the third quarter update include the propensity match scoring based on the agreed-upon SAP, or will that analysis be withheld for the BLA submission only? A: Walid Abi-Saab explained that they plan to agree with the FDA on the statistical analysis plan before locking the database and analyzing the data. They will share topline results but must be careful about the degree of data shared before FDA review. Q: Can you elaborate on the three immunosuppression-related SAEs reported in Cohort 3 and any changes made to the protocols or perioperative management? A: Walid Abi-Saab noted that AMT-130 is generally safe and well-tolerated. In Cohort 3, they observed three adverse events related to immunosuppression: mania, infection, and fever, all of which resolved with standard interventions. They believe a short two-week course of corticosteroids is optimal for future use. Q: How are you thinking about potentially evaluating AMT-130 in patients with earlier or later-stage disease? A: Walid Abi-Saab mentioned that the initial trial focused on early manifest patients to maximize function preservation. They are considering earlier or later interventions but need to discuss with regulators to determine the best path forward. The decision will depend on generating data to show benefit across different stages. Q: What is the latest data set the FDA has seen, and have they seen any Cohort 3 data with the updated immunosuppression regimen? A: Walid Abi-Saab clarified that the FDA has seen data from 24 patients at two years, based on a June 2024 cut. No additional data, including Cohort 3, has been shared since then. The two-week steroid regimen will be new, as previous cohorts did not include systematic perioperative immunosuppression. For the complete transcript of the earnings call, please refer to the full earnings call transcript. This article first appeared on GuruFocus. Sign in to access your portfolio
Yahoo
21-04-2025
- Health
- Yahoo
FDA grants breakthrough status to uniQure's Huntington's disease therapy
The US Food and Drug Administration (FDA) has awarded breakthrough therapy designation to uniQure's AMT-130 for treating Huntington's disease, a rare neurodegenerative condition. This designation for the therapy adds to a list of the agency's recognitions, including regenerative medicine advanced therapy (RMAT), fast track and orphan drug designations. Clinical data from the ongoing Phase I/II studies of the therapy support this latest designation. Interim data presented by the company last July demonstrated a dose-dependent slowing of progression of the disease in individuals treated with the therapy, against a propensity-weighted natural history. Till now, 45 subjects received the treatment. uniQure chief medical officer Walid Abi-Saab said: 'Receiving breakthrough therapy designation underscores both the urgent need for effective treatments for Huntington's disease and the encouraging interim data demonstrating that AMT-130 has the potential to slow disease progression. 'It's a powerful recognition of the promise of AMT-130 and the important progress we've made. We deeply value the FDA's continued commitment to advancing innovative gene therapies for patients with critical unmet needs, and we look forward to working closely with the agency to bring AMT-130 to the Huntington's disease patient community as quickly as possible.' The breakthrough therapy status is granted to accelerate investigational therapies development for serious conditions when preliminary clinical evidence suggests a substantial improvement over existing treatments. uniQure noted that Huntington's disease, which results in chorea, cognitive decline, and behavioural abnormalities, affects approximately 70,000 people in Europe and the US, with many more at risk. The company's gene therapy for haemophilia B has paved the way for new treatment approaches in genomic medicine. uniQure is currently progressing with its pipeline of gene therapies aimed at treating Huntington's disease, refractory temporal lobe epilepsy, Fabry disease, amyotrophic lateral sclerosis (ALS), and other severe conditions. "FDA grants breakthrough status to uniQure's Huntington's disease therapy" was originally created and published by Pharmaceutical Technology, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site. Sign in to access your portfolio
