Summary of uniQure's AMT-130 Conference Call Company and Industry - **Company**: uniQure - **Industry**: Gene therapy for Huntington's disease Key Points and Arguments Clinical Trial Results - **Pivotal Data Announcement**: uniQure announced pivotal data from the Phase I/II clinical trials of AMT-130 for Huntington's disease, showing a statistically significant 75% slowing of disease progression at 36 months as measured by the Composite Unified Huntington's Disease Rating Scale (CUHDRS) [4][18] - **Total Functional Capacity (TFC)**: The study also demonstrated a 60% reduction in disease progression on the TFC, a key measure of a patient's ability to live independently [14][19] - **Neurofilament Light Chain (NFL)**: CSF neurofilament light chain levels were below baseline at 36 months, indicating a reduction in neurodegeneration [16][19] Regulatory and Commercial Plans - **Biologics License Application (BLA)**: uniQure plans to submit a BLA for AMT-130 in the first quarter of 2026, with a request for priority review [5][18] - **FDA Engagement**: A pre-BLA meeting with the FDA is scheduled to discuss the data and the content of the BLA [18][34] Patient Population and Market Opportunity - **At-Risk Population**: Approximately 200,000 Americans are at risk for Huntington's disease, with about 100,000 being genetically identifiable [26] - **Diagnosed Patients**: There are around 40,000 symptomatic HD patients in the U.S., with about 20,000 currently diagnosed [26][27] - **Initial Treatable Patients**: An estimated 6,000 patients could be considered initially treatable at launch [27] Treatment Characteristics - **Durable Treatment**: AMT-130 is designed as a durable, once-administered treatment, which is crucial for a slowly progressing disease [6] - **Targeted Delivery**: The treatment allows for precision-based delivery to affected brain regions, maximizing therapeutic concentrations and minimizing systemic exposure [7] - **Mechanism of Action**: AMT-130 targets the first exon of the Huntington gene, suppressing both the full-length mutant protein and toxic splice isoforms [7] Safety Profile - **Well Tolerated**: AMT-130 has shown a manageable safety profile, with no new treatment-related serious adverse events reported since December 2022 [17][19] - **Common Adverse Events**: Most adverse events were related to study procedures, such as headache and procedural pain [17] Expert Perspectives - **Clinical Impact**: Dr. Sarah Tabrizi emphasized the potential of AMT-130 to significantly impact the quality of life for patients and families affected by Huntington's disease [24] - **Long-term Benefits**: The slowing of disease progression could allow patients to maintain independence and work longer, potentially preventing symptoms from occurring [41] Future Considerations - **Payer Engagement**: Early engagement with payers is critical to educate them on the value of AMT-130 and its impact on patients' lives [51] - **Labeling and Eligibility**: Discussions are ongoing regarding the potential labeling of AMT-130, including whether it will be restricted to symptomatic patients [80] Additional Important Content - **Statistical Analysis**: The study's primary analysis showed a 75% reduction in disease progression with a p-value of 0.003, indicating strong statistical significance [18] - **Longitudinal Data**: The data from the Enroll-HD study provided a robust external control for comparison, enhancing the credibility of the results [55] This summary encapsulates the critical findings and strategic plans discussed during the conference call regarding AMT-130 and its implications for Huntington's disease treatment.

Pivotal Phase I/II AMT-130 Huntington's Disease Update September 24, 2025 Disclaimer This presentation 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," "estimate," "expect," "goal," "intend," "look forward to," "may ...

uniQure (NASDAQ:QURE) announced on Wednesday that its one-time gene therapy AMT-130 reached the main goals in a pivotal Phase 2/3 trial for patients with Huntington’s disease, a rare neurodegenerative disorder. Citing topline data from the trial, the Dutch biotech noted that ...

Core Viewpoint - uniQure N.V. has secured a $175 million non-dilutive senior secured term loan facility with Hercules Capital to enhance financial flexibility for the potential commercial launch of its investigational gene therapy AMT-130 for Huntington's disease [1][2]. Financial Details - The loan facility includes three tranches: a first tranche of $50 million to refinance existing debt, a second tranche of $100 million available upon achieving regulatory and financial milestones, and a third tranche of up to $25 million subject to Hercules' approval [3]. - The interest rate for all tranches is floating, set at the greater of 9.45% or the prime rate plus 2.45%, with a current floating rate of 9.70%, down from a previous rate of 11.95% [3]. Company Background - uniQure is a leader in gene therapy, focusing on transformative therapies for severe medical conditions, including Huntington's disease, refractory temporal lobe epilepsy, ALS, and Fabry disease [5]. - The company has achieved significant milestones, including the approval of its gene therapy for hemophilia B, marking a major advancement in genomic medicine [5]. Partner Information - Hercules Capital is a prominent specialty finance company that provides senior secured venture growth loans to high-growth, innovative companies in technology and life sciences, having committed over $22 billion to more than 680 companies since its inception [6].

Core Insights - uniQure N.V. announced positive topline data from the pivotal Phase I/II study of AMT-130 for Huntington's disease, demonstrating statistically significant slowing of disease progression [1][2][3] Study Results - The pivotal study met its primary endpoint, showing a 75% slowing of disease progression at 36 months as measured by the composite Unified Huntington's Disease Rating Scale (cUHDRS) compared to a matched external control [3][4] - High-dose AMT-130 also achieved a statistically significant 60% slowing of disease progression as measured by Total Functional Capacity (TFC), meeting a key secondary endpoint [4][6] - Favorable trends were observed in additional clinical measures, including an 88% slowing of disease progression as measured by the Symbol Digit Modalities Test (SDMT) [4][6] Safety Profile - AMT-130 was generally well-tolerated with a manageable safety profile, and no new drug-related serious adverse events were reported since December 2022 [8][9] - The most common adverse events were related to the administration procedure, all of which resolved [8] Regulatory Plans - uniQure plans to submit a Biologics License Application (BLA) in the first quarter of 2026, with a potential U.S. launch later that year pending approval [1][9] - AMT-130 has received Breakthrough Therapy designation and Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA [9] Clinical Program Overview - The Phase I/II clinical program includes two multi-center, dose-escalating studies to explore the safety, tolerability, and exploratory efficacy signals of AMT-130 for Huntington's disease [11] - Data from the studies will be compared to a propensity score-matched external control derived from the Enroll-HD natural history dataset [11] Background on Huntington's Disease - Huntington's disease is a rare, inherited neurodegenerative disorder affecting approximately 75,000 people in the U.S., EU, and the UK, with no approved therapies currently available to slow its progression [15]

Core Insights - Solid Biosciences has entered into a non-exclusive worldwide license and collaboration agreement with Kinea Bio for the use of its proprietary capsid, AAV-SLB101, to develop KNA-155, a gene therapy targeting dysferlinopathy [1][2] - Solid will receive an upfront payment and is eligible for additional milestone payments and tiered royalties on net sales [2] - AAV-SLB101 is designed for enhanced muscle tropism and reduced liver biodistribution, showing promising results in preclinical studies and early clinical trials [3][7] Company Overview - Solid Biosciences focuses on developing precision genetic medicines for neuromuscular and cardiac diseases, with a diverse pipeline including therapies for Duchenne muscular dystrophy and other rare diseases [8][9] - The company aims to license AAV-SLB101 broadly to various institutions and companies pursuing treatments for rare diseases, having already established over 25 agreements [7] Collaboration Details - The agreement allows Kinea Bio to utilize AAV-SLB101 as the delivery mechanism for KNA-155, which is currently advancing into IND-enabling preclinical activities [2] - Both companies express confidence in the potential of AAV-SLB101 to deliver safe and effective therapies for patients with dysferlinopathies [6]

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Core Insights - Adverum Biotechnologies is advancing its pivotal Phase 3 trial, ARTEMIS, for Ixo-vec in wet age-related macular degeneration (wet AMD), with full enrollment expected by 4Q 2025 and data readout anticipated in 1Q 2027 [1][6]. Company Overview - Adverum Biotechnologies is a clinical-stage company focused on gene therapy to treat prevalent ocular diseases, aiming to establish functional cures to restore vision and prevent blindness [4]. - The company is developing ixoberogene soroparvovec (Ixo-vec), designed for a one-time intravitreal injection, which aims to provide long-term efficacy and reduce the need for frequent anti-VEGF treatments [3][4]. Trial Details - ARTEMIS is the first of two Phase 3 trials evaluating Ixo-vec, comparing a single administration of Ixo-vec (6E10 vg/eye) to aflibercept (2mg) administered every 8 weeks [2]. - The trial includes both treatment-naïve and previously treated patients, with the goal of demonstrating the efficacy of Ixo-vec as a potential one-time treatment for wet AMD [2][3]. Regulatory Designations - Ixo-vec has received Fast Track and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA, as well as PRIME designation from the EMA and Innovation Passport from the UK's Medicines and Healthcare Products Regulatory Agency [3].

Core Insights - Bayer has made significant progress in developing two potential therapies for Parkinson's disease (PD) [1] Group 1: Clinical Trials - The first participant has been randomized in the pivotal Phase III clinical trial, exPDite-2, for bemdaneprocel, which is an investigational cell therapy for PD [1] - The first European participants have been randomized in the Phase II clinical trial, REGENERATE-PD, for AB-1005, an investigational gene therapy [1] - Both therapies are aimed at treating moderate-stage Parkinson's disease [1]

Group 1 - Adverum Biotechnologies, Inc. (NASDAQ:ADVM) is recognized as a fundamentally strong penny stock with significant potential for investment [1][2] - The company reported impressive progress in the ARTEMIS Phase 3 trial, with increased enrollment driven by enthusiasm from retina specialists and patients, indicating a strong pipeline and future plans [2][3] - Strategic partnership discussions are ongoing, supported by the recognition of Ixo-vec's potential as a leading therapy, and a recent vote of confidence from Frazier Life Sciences, a major investor [3] Group 2 - Trexquant Investment LP significantly reduced its stake in Adverum by 80.5%, now holding 18,694 shares valued at approximately $82,000, which reflects a 0.09% ownership [1]