Latest news with #AMT-130
Yahoo
5 days ago
- Business
- Yahoo
Huntington's Disease Clinical Trial Pipeline Analysis: 20+ Key Companies Shaping the Future of Dopamine Receptor Antagonists Therapeutics
The Huntington's disease treatment market is poised for significant growth, driven by rising prevalence rates and increased awareness globally. Advancements in gene-targeting and disease-modifying therapies, along with a robust clinical pipeline, are addressing critical unmet needs. Active government support and educational initiatives are further enhancing diagnosis and treatment uptake. Together, these factors create a strong foundation for market expansion in the coming years. New York, USA, June 03, 2025 (GLOBE NEWSWIRE) -- Huntington's Disease Clinical Trial Pipeline Analysis: 20+ Key Companies Shaping the Future of Dopamine Receptor Antagonists Therapeutics | DelveInsight The Huntington's disease treatment market is poised for significant growth, driven by rising prevalence rates and increased awareness globally. Advancements in gene-targeting and disease-modifying therapies, along with a robust clinical pipeline, are addressing critical unmet needs. Active government support and educational initiatives are further enhancing diagnosis and treatment uptake. Together, these factors create a strong foundation for market expansion in the coming years. DelveInsight's 'Huntington's Disease Pipeline Insight 2025' report provides comprehensive global coverage of pipeline Huntington's disease therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the Huntington's disease pipeline domain. Key Takeaways from the Huntington's Disease Pipeline Report DelveInsight's Huntington's disease pipeline report depicts a robust space with 20+ active players working to develop 20+ pipeline Huntington's disease drugs. Key Huntington's disease companies such as Hoffmann-La Roche, Medibiofarma, PTC Therapeutics, Annexon, Alnylam Pharmaceuticals, Neuvivo, Skyhawk Therapeutics, BPG Bio, and others are evaluating new Huntington's disease drugs to improve the treatment landscape. Promising pipeline Huntington's disease therapies, such as RG6042, MBF 015, PTC518, ANX-005, ALN-HTT02, NP001, SKY 0515, Research Programme: HDD, and others are in different phases of Huntington's disease clinical trials. In April 2025, the FDA granted a breakthrough therapy designation to AMT-130 (uniQuire) for the treatment of Huntington's disease. Previously, AMT-130 was also granted a regenerative medicine advanced therapy designation and orphan drug designation for this indication. In April 2025, Prilenia Therapeutics announced that it had entered into a collaboration and license agreement with Ferrer for the development and commercialization of pridopidine in Europe and other select markets. In March 2025, Skyhawk Therapeutics presented their novel SKY-0515 small molecule RNA splicing modulator targeting Huntington's Disease to members of the Huntington's Disease Youth Organization (HDYO), at the biannual HYDO International Congress in Prague, Czech Republic. In December 2024, Novartis announced that it had entered into a global licensing and collaboration agreement with PTC Therapeutics for PTC518, an HTT mRNA splice modulator for the treatment of Huntington's disease. Under the terms of the agreement, Novartis will pay USD 1 billion upfront and up to USD 1.9 billion in development, regulatory, and sales milestones. Novartis will also share profits in the US and pay tiered royalties on sales outside the US. In September 2024, PTC Therapeutics announced that the FDA had granted Fast Track designation to the PTC518 program for the treatment of Huntington's disease. In September 2024, the European Medicines Agency (EMA) accepted the marketing authorization application (MAA) for Prilenia Therapeutics' pridopidine for the treatment of adults with Huntington's disease. Request a sample and discover the recent advances in Huntington's disease drugs @ Huntington's Disease Pipeline Report The Huntington's disease pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage Huntington's disease drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the Huntington's disease clinical trial landscape. Huntington's Disease Overview Huntington's disease is a hereditary, progressive neurodegenerative condition marked by the gradual onset of involuntary muscle movements—affecting the hands, feet, face, and trunk—and a steady decline in cognitive abilities and memory. It impacts the central region of the brain, leading to difficulties in movement, emotional regulation, and cognitive functions. Symptoms often appear between ages 30 and 50 but can emerge as early as age 2 or as late as 80. A hallmark feature of the disease is the presence of rapid, uncontrolled movements such as muscle jerks or tics. As the condition advances, patients may experience loss of coordination, speech difficulties, memory deterioration, and worsening choreiform movements, along with personality and behavioral changes. Huntington's disease follows an autosomal dominant inheritance pattern, meaning only one copy of the faulty gene is needed to cause the disease. It is triggered by a mutation in a single gene on chromosome 4, which encodes the huntingtin protein. While the exact role of huntingtin remains unclear, its mutated form disrupts normal brain function, leading to involuntary movements, severe cognitive decline, and emotional disturbances such as depression and irritability. The mutation involves an expansion of a CAG repeat segment within the gene. Typically, this segment repeats 17 to 20 times in a healthy gene, but in Huntington's disease, the number of repeats rises to 40 or more. Diagnosing Huntington's disease involves a comprehensive clinical assessment, patient history, and several specialized tests. These may include blood work, genetic testing to detect mutations in the HTT gene, and brain imaging techniques such as CT scans, MRI, and EEG. CT and MRI provide detailed cross-sectional images of the brain, while EEG measures the brain's electrical activity to support diagnosis. Currently, there is no cure for Huntington's disease, and treatment is aimed at alleviating symptoms. The only two FDA-approved medications—Austedo and Xenazine—are indicated specifically for managing chorea related to Huntington's. Additional symptom management may include antidepressants, antipsychotics, and mood stabilizers to address psychiatric symptoms. Supportive therapies and multidisciplinary care are also critical in improving the quality of life for individuals affected by this out more about Huntington's disease drugs @ Huntington's Disease Treatment A snapshot of the Pipeline Huntington's Disease Drugs mentioned in the report: Drugs Company Phase MoA RoA MBF 015 Medibiofarma II HDAC1/C2 inhibitors Oral PTC518 PTC Therapeutics II HD protein inhibitors; RNA splicing modulators Oral ANX-005 Annexon II Complement C1 inhibitors Intravenous ALN-HTT02 Alnylam Pharmaceuticals I RNA interference Intrathecal NP001 Neuvivo I Macrophage modulators Unspecified SKY 0515 Skyhawk Therapeutics I RNA splicing modulators Oral Learn more about the emerging Huntington's disease therapies @ Huntington's Disease Clinical Trials Huntington's Disease Therapeutics Assessment The Huntington's disease pipeline report proffers an integral view of the emerging Huntington's disease therapies segmented by stage, product type, molecule type, route of administration, and mechanism of action. Scope of the Huntington's Disease Pipeline Report Coverage: Global Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Therapeutics Assessment By Route of Administration: Intra-articular, Intraocular, Intrathecal, Intravenous, Oral, Parenteral, Subcutaneous, Topical, Transdermal Therapeutics Assessment By Molecule Type: Oligonucleotide, Peptide, Small molecule Therapeutics Assessment By Mechanism of Action: HDAC1/C2 inhibitors, RNA interference, HD protein inhibitors, RNA splicing modulators, Macrophage modulators, Complement C1 inhibitors Key Huntington's Disease Companies: Hoffmann-La Roche, Medibiofarma, PTC Therapeutics, Annexon, Alnylam Pharmaceuticals, Neuvivo, Skyhawk Therapeutics, BPG Bio, and others. Key Huntington's Disease Pipeline Therapies: RG6042, MBF 015, PTC518, ANX-005, ALN-HTT02, NP001, SKY 0515, Research Programme: HDD, and others. Dive deep into rich insights for new Huntington's disease treatments, visit @ Huntington's Disease Drugs Table of Contents 1. Huntington's Disease Pipeline Report Introduction 2. Huntington's Disease Pipeline Report Executive Summary 3. Huntington's Disease Pipeline: Overview 4. Analytical Perspective In-depth Commercial Assessment 5. Huntington's Disease Clinical Trial Therapeutics 6. Huntington's Disease Pipeline: Late-Stage Products (Pre-registration) 7. Huntington's Disease Pipeline: Late-Stage Products (Phase III) 8. Huntington's Disease Pipeline: Mid-Stage Products (Phase II) 9. Huntington's Disease Pipeline: Early-Stage Products (Phase I) 10. Huntington's Disease Pipeline Therapeutics Assessment 11. Inactive Products in the Huntington's Disease Pipeline 12. Company-University Collaborations (Licensing/Partnering) Analysis 13. Key Companies 14. Key Products in the Huntington's Disease Pipeline 15. Unmet Needs 16. Market Drivers and Barriers 17. Future Perspectives and Conclusion 18. Analyst Views 19. Appendix For further information on the Huntington's disease pipeline therapeutics, reach out @ Huntington's Disease Therapeutics Related Reports Huntington's Disease Epidemiology Forecast Huntington's Disease Epidemiology Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted Huntington's disease epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Huntington's Disease Market Huntington's Disease Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Huntington's disease companies, including Prilenia Therapeutics, Neurocrine Biosciences, SOM Biotech, Annexon Biosciences, Vaccinex, Sage Therapeutics, UniQure Biopharma, Wave life sciences, Takeda, Medesis Pharma, among others. Wilson Disease Market Wilson Disease Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Wilson disease companies, including Alexion Pharmaceuticals, Vivet Therapeutics, Orphalan, among others. Wilson Disease Pipeline Wilson Disease Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key Wilson disease companies, including Alexion Pharmaceuticals, Vivet Therapeutics, Orphalan, among others. Bipolar Disorder Pipeline Bipolar Disorder Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key bipolar disorder companies, including Vanda Pharmaceuticals, Pear Therapeutics, Sunovion, Lyndra Therapeutics, among others. DelveInsight's Pharma Competitive Intelligence Service: Through its CI solutions, DelveInsight provides its clients with real-time and actionable intelligence on their competitors and markets of interest to keep them stay ahead of the competition by providing insights into the latest therapeutic area-specific/indication-specific market trends, in emerging drugs, and competitive strategies. These services are tailored to the specific needs of each client and are delivered through a combination of reports, dashboards, and interactive presentations, enabling clients to make informed decisions, mitigate risks, and identify opportunities for growth and expansion. Other Business Consulting Services Healthcare Conference Coverage Pipeline Assessment Healthcare Licensing Services Discover how a mid-pharma client gained a level of confidence in their soon-to-be partner for manufacturing their therapeutics by downloading our Due Diligence Case Study About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. Connect with us at LinkedIn CONTACT: Contact Us Shruti Thakur info@ +14699457679 in to access your portfolio
Yahoo
6 days ago
- Business
- Yahoo
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: Q2 2025: submit updated SAP to the FDA Q3 2025: initiate PPQ run and present topline Phase I/II data per the SAP Q4 2025: hold pre-BLA meeting with the FDA Q1 2026: submit BLA with a request for priority review designation 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. uniQure Contacts: FOR INVESTORS: FOR MEDIA: Chiara Russo Tom Malone Direct: 617-306-9137 Mobile: 617-306-9137 Mobile: 617-306-9137 Mobile:339-223-8541 in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
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
Associated Press
17-04-2025
- Business
- Associated Press
uniQure Announces FDA Breakthrough Therapy Designation Granted to AMT-130 for the Treatment of Huntington's Disease
~ Breakthrough Therapy designation based on clinical evidence from Phase I/II trials showing meaningful slowing of disease progression ~ ~ Additional regulatory update and guidance on the Biologics License Application submission expected in the second quarter of 2025 ~ LEXINGTON, Mass. and AMSTERDAM, April 17, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to AMT-130 for the treatment of Huntington's disease, a rare, inherited neurodegenerative disorder for which there are currently no disease-modifying therapies available. This designation is in addition to Regenerative Medicine Advanced Therapy (RMAT) designation, Orphan Drug designation and Fast Track designation, all previously granted by the FDA to AMT-130. '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,' said Walid Abi-Saab, M.D., chief medical officer of uniQure. '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 designation is supported by clinical data from the ongoing Phase I/II trials of AMT-130 for the treatment of Huntington's disease. In July 2024, uniQure presented interim data at 24 months that showed dose-dependent slowing of disease progression based on the cUHDRS of treated patients compared to a propensity-weighted natural history. To date, a total of 45 patients have received AMT-130. Breakthrough Therapy designation is intended to expedite the development and review of investigational therapeutic candidates that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s). In general, the preliminary clinical evidence should show a clear advantage over available therapy. A drug that receives Breakthrough Therapy designation is eligible for all Fast Track designation features, intensive guidance on an efficient drug development program, and FDA commitment involving senior managers1. 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 Company's plans for further interactions with the FDA to discuss the requirements for its planned BLA submission for AMT-130; the Company's ability to utilize an accelerated pathway to progress AMT-130 through regulatory approval; the Company's plans to announce additional interim data and regulatory updates from its ongoing Phase I/II clinical studies of AMT-130, along with an initial safety update on the third cohort of the AMT-130 study and other program updates; the potential clinical and functional effects of AMT-130; and the Company's plans to continue clinical development of AMT-130. The Company's actual results could differ materially from those anticipated in these forward-looking statements for many reasons. These risks and uncertainties include, among others: risks associated with the clinical results and the development and timing of the Company's programs; the Company's interactions with regulatory authorities, which may affect the initiation, timing and progress of clinical trials and pathways to regulatory approval; the Company's ability to continue to build and maintain the company infrastructure and personnel needed to achieve its goals; the Company's effectiveness in managing current and future clinical trials and regulatory processes; the continued development and acceptance of gene therapies; the Company's ability to demonstrate the therapeutic benefits of its gene therapy candidates in clinical trials; the Company's ability to obtain, maintain and protect intellectual property; and the Company's ability to fund its operations and to raise additional capital as needed. 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, 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. 1 Guidance for Industry Expedited Programs for Serious Conditions - Drugs and Biologics, May 2014
Associated Press
27-02-2025
- Business
- Associated Press
uniQure Announces 2024 Financial Results and Highlights Recent Company Progress
~ Announced alignment with the U.S. Food and Drug Administration (FDA) on key elements of the Accelerated Approval pathway for AMT-130 in Huntington's disease; Initiated preparations for a potential Biologics License Application (BLA) submission ~ ~ Completed patient enrollment in the third cohort of the Phase I/II study of AMT-130 ~ ~ Initiated dosing of the Phase I/II study of AMT-260 in mesial temporal lobe epilepsy (mTLE); Implementing protocol changes to expand study inclusion criteria ~ ~ Completed enrollment of the first cohorts in the Phase I/II studies of AMT-191 in Fabry disease and AMT-162 in SOD1-ALS; Received favorable recommendations from the respective Independent Data Monitoring Committees (IDMC) to proceed with dosing the second cohorts ~ ~ Cash and cash equivalents of approximately $367.5 million as of December 31, 2024, combined with $80.7 million in net proceeds from the recently completed financing, are expected to fund operations into the second half of 2027 ~ LEXINGTON, Mass. and AMSTERDAM, Feb. 27, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today reported its financial results for the fourth quarter and full year of 2024 and highlighted recent progress across its business. 'This past year was transformative for uniQure, marked by significant clinical and operational progress,' stated Matt Kapusta, chief executive officer of uniQure. 'On the clinical front, we made great strides advancing AMT-130 for Huntington's disease, including securing alignment with the FDA on key elements of the Accelerated Approval pathway, a major milestone that brings us closer to delivering the first potentially disease-modifying treatment for this devastating condition. With this regulatory clarity, we have initiated BLA-readiness activities and look forward to further engagement with the FDA throughout the first half of 2025. Our RMAT designation has enabled a productive and expedited dialogue with the FDA, which we will continue leveraging to advance AMT-130 through the regulatory process as rapidly as possible. This is welcome news for patients awaiting further development of our groundbreaking therapies.' Mr. Kapusta continued, 'Beyond AMT-130, we continue to advance our broader pipeline of investigational gene therapies, with patient enrollment progressing in the Phase I/II studies of AMT-191 for Fabry disease and AMT-162 for SOD1-ALS. Additionally, we are implementing FDA-approved protocol changes to the Phase I/II study of AMT-260 in mTLE, including broadening the inclusion criteria for certain patients in the first cohort – a step we anticipate will help accelerate trial enrollment.' 'Operationally, we took decisive steps in 2024 to streamline our organization, including the sale of our Lexington manufacturing facility and a company-wide restructuring that significantly reduced our cash burn and strengthened our financial position,' Mr. Kapusta continued. 'These strategic actions enable us to prioritize investments and achieve multiple value-creating milestones, including the potential approval and commercial launch of AMT-130.' Recent Company Developments and Updates Pursuing Accelerated Approval of AMT-130 for the treatment of Huntington's disease In May 2024, the FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation for AMT-130, stating that preliminary clinical evidence indicates that AMT-130 has the potential to address unmet medical needs for treatment of Huntington's disease. In December 2024, uniQure reached agreement with the FDA on key elements of an Accelerated Approval pathway for AMT-130 in Huntington's disease. As part of the RMAT Type B meeting, the FDA agreed that data from the ongoing Phase I/II studies, compared to a natural history external control, may serve as the primary basis for a BLA submission, eliminating the need for an additional pre-submission study. The FDA also agreed that the composite Unified Huntington's Disease Rating Scale (cUHDRS) may be used as an intermediate clinical endpoint and reductions in neurofilament light chain (NfL) in the cerebrospinal fluid (CSF) may serve as supportive evidence of therapeutic benefit. The Company has scheduled a Type B meeting with the FDA in the first quarter of 2025 to discuss chemistry, manufacturing and control (CMC) requirements to support its planned BLA submission. A separate Type B meeting to discuss the pivotal statistical analysis plan for the BLA is anticipated to take place in the second quarter of 2025. After the completion of these interactions, the Company expects to provide a regulatory update in the second quarter of 2025, including the expected timing of a potential BLA submission. In February 2025, the Company completed enrollment of all 12 patients in the third cohort investigating an optimized immunosuppression regimen. The Company expects to provide an initial safety update on the third cohort in the second quarter of 2025. In the third quarter of 2025, the Company expects to present data from its ongoing Phase I/II studies of AMT-130 in support of a potential BLA submission. The update will include follow-up data on all patients treated with AMT-130 in the first two cohorts, including three years of follow-up on 24 treated patients. Advancing additional clinical programs to proof-of-concept AMT-260 for the treatment of refractory mesial temporal lobe epilepsy (mTLE) – In November 2024, the Company announced the first patient dosed in the Phase I/II clinical trial of AMT-260 for the treatment of mTLE. The FDA recently approved a protocol amendment expanding the inclusion criteria for certain patients in the first cohort to include patients with non-lesional mesial temporal lobe epilepsy in the non-dominant hemisphere. This broader inclusion criteria may assist in accelerated enrollment. The Company expects to present initial data from the study in the first half of 2026. AMT-162 for the treatment of SOD1 amyotrophic lateral sclerosis (ALS) – In January 2025, the Company announced a favorable recommendation from the IDMC based on the review of 28-day safety data from the first study cohort in the Phase I/II EPISODE1 study. The Company initiated enrollment in the second dose cohort in the first quarter of 2025 and expects to present initial data from the study in the first half of 2026. AMT-191 for the treatment of Fabry disease – In February 2025, the Company announced completion of enrollment in the first cohort in the Phase I/IIa clinical trial of AMT-191 and a favorable recommendation from the IDMC having reviewed safety data from the initial two patients. The Company expects to initiate enrollment in the second dose cohort in the second quarter of 2025 and to present initial data from the study in the second half of 2025 . Strong financial position In the first quarter of 2025, the Company completed a public offering of 5.1 million ordinary shares, including the full exercise of the underwriters' overallotment option, at a price of $17.00 per share. Net proceeds from the offering are expected to fund operations into the second half of 2027, including the potential BLA submission and U.S. commercial launch of AMT-130. In the second quarter of 2024, the Company completed the sale of its Lexington, MA manufacturing facility to Genezen and retired $50 million of its outstanding debt with Hercules Capital. In the third quarter of 2024, the Company announced an organization restructuring which, combined with the Lexington facility sale, eliminated approximately 65% of the global workforce and reduced recurring cash burn by approximately $70 million per year. Upcoming Investor Events TD Cowen 45th Annual Healthcare Conference, March 3rd – Boston, MA Leerink's Global Healthcare Conference 2025, March 10th – Miami, FL Kempen Life Sciences Conference, April 3rd – Amsterdam, NL Financial Highlights Cash position: As of December 31, 2024, the Company held cash, cash equivalents and investment securities of $367.5 million, compared to $617.9 million as of December 31, 2023. Including the net proceeds of $80.7 million from the recently completed follow-on offering, the Company's proforma cash, cash equivalents and investment securities was approximately $448 million. The reduction in cash was in part driven by non-recurring payments, including $53 million related to the retirement of debt, $31.5 million related to milestone payments, $12.0 million of one-time payments related to the divestment of the Lexington facility transaction, and $4.7 million of severance payments related to the Company's restructuring. Based on the Company's current operating plan, including the planned U.S. launch of AMT-130, the Company expects cash, cash equivalents and investment securities will be sufficient to fund operations through the second half of 2027. Revenues: Revenue for the year ended December 31, 2024 was $27.1 million, compared to $15.8 million in the same period in 2023. The increase of $11.3 million in revenue resulted from a $7.4 million increase in license revenue, an increase of $8.6 million from collaboration revenue, and a decrease of $4.7 million from contract manufacturing of HEMGENIX® for CSL Behring. Following the divestment of the Lexington facility in July 2024, revenue from contract manufacturing is recorded net of cost within other expenses. Cost of contract manufacturing revenues: Cost of contract manufacturing revenues were $17.1 million for the year ended December 31, 2024, compared to $13.6 million for the same period in 2023. Following the divestment of the Lexington facility in July 2024, cost of contract manufacturing is recorded net of revenue within other expenses. R&D expenses: Research and development expenses were $143.8 million for the year ended December 31, 2024, compared to $214.9 million during the same period in 2023. The $71.1 million decrease was related to a decrease of $30.5 million in employee-related expenses, $17.7 million lower expenses related to changes in the fair value of contingent consideration, a net decrease of $8.3 million in external program spend and an $8.1 million decrease in costs related to preclinical supplies. SG&A expenses: Selling, general and administrative expenses were $52.7 million for the year ended December 31, 2024, compared to $74.6 million during the same period in 2023. The $21.9 million decrease was primarily related to a $7.6 million decrease in employee-related expenses, a decrease of $5.4 million in information technology costs, a $4.4 million decrease in professional fees and a $2.1 million decrease in intellectual property fees and compared to the prior year period. Other income: Other income was $7.9 million for the year ended December 31, 2024, compared to $6.1 million during the same period in 2023. The increase was primarily related to the $1.2 million gain recorded on divesting the Lexington manufacturing facility. Other expense: Other expense was $4.6 million for the year ended December 31, 2024, compared to $1.7 million during the same period in 2023. The increase was primarily related to $2.5 million of non-cash expense recognized to amortize the right to purchase HEMGENIX® from Genezen on favorable terms. Other non-operating items, net: Other non-operating items, net was an expense of $52.8 million for the year ended December 31, 2024, compared to $23.7 million for the same period in 2023. The $29.1 million increase in other non-operating items, net was primarily related to an increase in non-cash interest expense of $23.9 million related to the royalty agreement that the Company entered into in May 2023 and an increase in net foreign currency losses of $8.8 million. Net loss: The net loss for the year ended December 31, 2024, was $239.6 million, or $4.92 basic and diluted loss per ordinary share, compared to $308.5 million net loss for the same period in 2023, or $6.47 basic and diluted loss per ordinary share. 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. 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. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. Examples of these forward-looking statements include, but are not limited to, statements concerning the Company's cash runway and its ability to fund its operations into the second half of 2027 and the planned use of proceeds from its first quarter 2025 public offering; the Company's plans for further interactions with the FDA to discuss the requirements for its planned BLA submission for AMT-130; the Company's ability to utilize an accelerated pathway to progress AMT-130 through regulatory approval; the Company's plans to announce additional interim data and regulatory updates from its ongoing Phase I/II clinical studies of AMT-130, along with an initial safety update on the third cohort of the AMT-130 study and other program updates; the effectiveness of planned protocol changes in accelerating enrollment in the AMT-260 study; and the Company's organizational restructuring and other actions designed to increase shareholder value and fund its pipeline of gene therapy candidates. The Company's actual results could differ materially from those anticipated in these forward-looking statements for many reasons. These risks and uncertainties include, among others: risks associated with the clinical results and the development and timing of the Company's programs; the Company's interactions with regulatory authorities, which may affect the initiation, timing and progress of clinical trials and pathways to regulatory approval; the Company's ability to continue to build and maintain the company infrastructure and personnel needed to achieve its goals; the Company's effectiveness in managing current and future clinical trials and regulatory processes; the continued development and acceptance of gene therapies; the Company's ability to demonstrate the therapeutic benefits of its gene therapy candidates in clinical trials; the Company's ability to obtain, maintain and protect intellectual property; and the Company's ability to fund its operations and to raise additional capital as needed. 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 to be filed February 27, 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 the Company assumes no obligation to update these forward-looking statements, even if new information becomes available in the future. uniQure Contacts: uniQure N.V. UNAUDITED CONSOLIDATED BALANCE SHEETS December 31, December 31, 2024 2023 (in thousands, except share and per share amounts) Current assets Cash and cash equivalents $ 158.930 $ 241.360 Current investment securities 208.591 376.532 Accounts receivable 5.881 4.193 Inventories, net — 12.024 Prepaid expenses 9.281 15.089 Other current assets and receivables 7.606 2.655 Total current assets 390.289 651.853 Non-current assets Property, plant and equipment, net $ 20.424 $ 46.548 Other investments 27.464 $ 2.179 Operating lease right-of-use assets 13.647 28.789 Intangible assets, net 71.043 60.481 Goodwill 22.414 26.379 Deferred tax assets, net 9.856 12.276 Other non-current assets 1.399 3.184 Total non-current assets 166.247 179.836 Total assets $ 556.536 $ 831.689 Current liabilities Accounts payable $ 7.227 $ 6.586 Accrued expenses and other current liabilities 29.225 30.534 Current portion of contingent consideration — 28.211 Current portion of operating lease liabilities 3.601 8.344 Total current liabilities 40.053 73.675 Non-current liabilities Long-term debt 51.324 101.749 Liability from royalty financing agreement 434.930 394.241 Operating lease liabilities, net of current portion 11.136 28.316 Contingent consideration, net of current portion 10.860 14.795 Deferred tax liability, net 7.043 7.543 Other non-current liabilities 7.942 3.700 Total non-current liabilities 523.235 550.344 Total liabilities 563.288 624.019 Shareholders' equity Total shareholders' equity (6.752) 207.670 Total liabilities and shareholders' equity $ 556.536 $ 831.689 uniQure N.V. UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS Year ended December 31, 2024 2023 2022 (in thousands, except share and per share amounts) License revenues 10.133 $ 2.758 $ 100.000 Contract manufacturing revenues 6.114 10.835 1.717 Collaboration revenues 10.872 2.250 4.766 Total revenues 27.119 15.843 106.483 Operating expenses: Cost of license revenues (1.267) (65) (1.254) Cost of contract manufacturing revenues (17.060) (13.563) (2.089) Research and development expenses (143.782) (214.864) (197.591) Selling, general and administrative expenses (52.657) (74.591) (55.059) Total operating expenses (214.766) (303.083) (255.993) Other income 7.926 6.059 7.171 Other expense (4.573) (1.690) (820) Loss from operations (184.294) (282.871) (143.159) Non-operating items, net (52.833) (23.686) 14.900 Loss before income tax (expense) / benefit $ (237.127) $ (306.557) $ (128.259) Income tax (expense) / benefit (2.429) (1.921) 1.470 Net loss $ (239.556) $ (308.478) $ (126.789) Earnings per ordinary share - basic and diluted Basic and diluted net loss per ordinary share $ (4,92) $ (6,47) $ (2,71) Weighted average shares - basic and diluted 48.649.129 47.670.986 46.735.045
