uniQure (NasdaqGS:QURE) FY Conference Transcript

Summary of uniQure FY Conference Call - March 02, 2026 Company Overview - Company: uniQure (NasdaqGS:QURE) - Focus: Gene therapies for neurodegenerative diseases, particularly Huntington's disease and epilepsy Key Points on Huntington's Disease Program (AMT-130) - Phase 3 Data: The pivotal phase 1/2 trial showed a 75% slowing of disease progression at three years, measured by the composite Unified Huntington's Disease Rating Scale (UHDRS) [9] - Total Functional Capacity: A 60% decline in disease progression was observed, which is significant for FDA approval [9] - Neurofilament Light: A decrease from baseline was noted, contrasting with the expected 10%-15% annual increase in neurodegeneration markers [9] - FDA Interactions: The FDA granted RMAT designation in early 2024 based on initial data, but later expressed concerns about the Phase 1/2 study being hypothesis-generating and not suitable for BLA submission [15][20] - Next Steps: A Type B meeting with the FDA is planned to discuss phase 3 study design and the use of external controls [44][46] - Patient Community Support: Strong advocacy from patient groups has been noted, with significant engagement in policy discussions and petitions to the FDA [82][84] Key Points on AMT-260 (Temporal Lobe Epilepsy Program) - Initial Data: A case study showed a 92% reduction in seizure frequency in the first patient treated, with no significant adverse events reported [84] - Current Status: Six patients have been dosed in the first cohort, with data expected in the second quarter of 2026 [85] - Future Goals: Aiming for a 50% or higher reduction in seizures to support progression to pivotal trials [96] Key Points on AMT-191 (Fabry Disease Program) - Study Progress: A Phase I/II study is ongoing with 11 patients treated, all of whom are off enzyme replacement therapy [99] - Outcomes: Dose-dependent increases in the deficient enzyme have been observed, with ongoing monitoring for long-term outcomes [99] Financial and Strategic Considerations - Funding Position: The company has a strong balance sheet with a runway into the second half of 2029, allowing for continued investment in promising programs [104] - Partnership Strategy: The company remains open to partnerships but emphasizes the need for data to support investment decisions [105] Safety Profile - AMT-130 Administration: The procedure is reported to be safe and well-tolerated, with most adverse events being procedure-related rather than drug-related [64] Regulatory Landscape - International Engagement: The company is engaging with regulators outside the U.S. for potential expedited reviews and expanded access pathways [71][74] Conclusion - Commitment to Patients: The company is focused on advancing its therapies while ensuring ethical considerations in study designs, particularly for slow-progressing diseases like Huntington's [46][54]