uniQure N.V.

Thank you for standing by. My name is Kate and I will be your conference operator today. At this time, I would like to welcome everyone to the Unicure fourth quarter and year end 2025 earnings call. All lines have been placed on mute to prevent any background noise. After the speaker's remarks, there will be a question and answer session. If you would like to ask a question during this time, simply press star followed with the number one on your telephone keypad. If you would like to withdraw your question, press star one again. Thank you. I would now like to turn the call over to Cara Rosso, Senior Director of Investor Relations. Please go ahead.

Good morning, and thank you for joining us for Unicure's year-end 2025 earnings call. Earlier this morning, Unicure released its financial results for the fourth quarter and year-end of 2025, and our press release is available on the Investors and Media section of our website, at unicure.com. Our 10K was also filed with the SEC earlier this morning. Joining me on the call this morning are Matt Capista, Chief Executive Officer, Dr. Wally Abisab, Chief Medical Officer, Kylie O'Keefe, Chief Customer and Strategy Officer, and Christian Klempt, Chief Financial Officer. After our formal remarks, we'll open up the call for Q&A. Before we begin, please know that we will be making forward-looking statements during this investor call. All statements, other than statements of historical fact, are forward-looking statements. They are based on management's beliefs and assumptions and on information available to management only as of the date of this conference call. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, the factors described in Unicure's most recent SDC filings. Given these risks, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these statements, even if new information becomes available in the future. Now, let me introduce Matt Capista, Unicure's CEO.

Thanks, Chiara. Good morning, everyone, and thank you for joining us on our year-end 2025 conference call. For more than 25 years, Unicure has been driven by a singular mission to deliver transformative medicines to patients living with rare genetic and other debilitating diseases. Along that journey, we have successfully developed two approved gene therapies. For the past decade, we have been deeply focused on advancing AMT130, a novel one-time administered treatment designed to address the underlying cause for Huntington's disease. In September of last year, we reported groundbreaking three-year data from our Phase 1-2 study of AMT-130, which were widely embraced by the HD community. These data demonstrated a statistically significant 75% slowing of disease progression as measured by the composite unified Huntington's disease rating scale, a statistically significant 60% slowing as measured by total functional capacity, a reduction in neurofilament light, a key indicator of neurodegeneration from baseline, and supportive trends across other key clinically meaningful endpoints. Importantly, these outcomes were assessed against a carefully and methodically constructed patient-matched external comparator derived from EnrollHD. the largest independent Huntington's disease natural history data set in existence, encompassing longitudinal clinical grade data for more than 30,000 participants that has been rigorously and painstakingly collected over the past 14 years. While the months since sharing our landmark data have presented certain challenges, they have only strengthened our conviction that AMT130 has the potential to benefit patients with Huntington's disease and reinforced our unwavering commitment to the HD community. As previously disclosed during the October 2025 pre-BLA meeting, the FDA conveyed that data submitted from the Phase 1-2 studies of ANT130 were unlikely to provide the primary evidence to support a BLA submission. We subsequently held a Type A meeting with the agency on January 30th to discuss next steps. In the meeting minutes we received on Friday, February 27th, the FDA explicitly affirmed their commitment to exercising appropriate regulatory flexibility to facilitate the development of safe and effective therapies for HD. Still, the FDA strongly recommended we conduct a phase three randomized double-blind sham surgery controlled study of AMT130. While we respect the agency's perspective and share its commitment to rigorous science, we believe it's appropriate to fully and carefully consider how regulatory flexibility is applied in the context of a rare, monogenic, slow progressive, and ultimately fatal neurodegenerative disorder for which there are no approved disease-modifying treatments. In our view, the totality of evidence generated to date for AMT130 warrants continued substantive dialogue regarding the most scientifically grounded and feasible regulatory pathways given the severity of the unmet need and the irreversible nature of the disease. While this was not the feedback we were hoping for, we remain highly confident in the strength and durability of our data. Our focus now is on constructive engagement with the FDA to further define a clear and efficient regulatory path forward. We are actively evaluating the agency's recommendations, including potential Phase III study designs, while preserving our commitment to advancing the program responsibly and expeditiously. In addition, we intend to update our Phase I-II statistical analysis plan to incorporate a four-year analysis that we expect to conduct in the third quarter of 2026. We believe extended follow-up will further inform the durability and magnitude of effect observed to date. Let me be clear, we remain unwavering in our commitment to the HD community and greatly appreciate their tireless support over the past months and years. The urgency in the community is real and we strongly believe AMT130 has the potential to deliver meaningful disease-modifying benefits. Our team is fully engaged in determining the clearest and most efficient regulatory pathways to bring this therapy to patients as quickly as possible around the world, and we look forward to providing further updates as these discussions progress. With that, I will turn the call over to Waleed to provide additional color on HD and our other clinical programs. Waleed? Thank you, Matt.