uniQure N.V.

Good day, and thank you for standing by. Welcome to the Unicure first quarter 2025 earnings conference call. At this time, all participants are in a listen-only mode. After the speaker's presentation, there'll be a question and answer session. To ask a question during the session, you'll need to press star 11 on your telephone. You will then hear an automated message advising your hand is raised. To answer your question, press star 11 again. Please be advised that today's conference is being recorded. I would now like to turn the conference over to your speaker for today, Chiara Russo, Senior Director of Investor Relations. Please go ahead.

Thank you. Good morning, and thank you for joining us for Unicare's inaugural quarterly earnings call. Earlier this morning, Unicare released its financial results for the first quarter of 2025. The press release is available on the Investors in Media section of our website at unicure.com, and our 10Q will be filed with the SEC later today. Joining me on the call this morning are Matt Capusa, Chief Executive Officer, Dr. Walid Abisab, Chief Medical Officer, and Christian Klemp, our Chief Financial Officer. After our formal remarks, we'll open up the call for Q&A. Please know that we'll 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 limitations, the factors described in UNICUR's most recent SEC filings. Given these risks, you should not put 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 Cavista, Unicare's CEO.

Thanks, Kiara, and good morning, everyone. Thank you for joining our call today. I'm very pleased to share with you our first quarter 2025 financial results and provide an update on the progress of our four clinical stage gene therapy programs. As we continue to advance our pipeline and drive AMT 130 closer to a planned BLA submission, we are commencing quarterly earnings calls to provide regular updates on our progress. I'm joined today by Dr. Waleed Abisab, our Chief Medical Officer, and Christian Klent, our Chief Financial Officer, who each provide updates on their respective areas. I'd like to begin by reflecting on what has been a truly remarkable last 12 months for Unicure. and why we believe 2025 has the potential to be a transformational year for the company, with multiple important value-driving milestones ahead. Last July, we presented compelling two-year data from our Huntington's program, demonstrating AMT 130's potential to meaningfully slow disease progression. Then in November, we announced alignment with the FDA on key elements of an accelerated approval pathway, which include the use of a natural history external control and CUHDRS as a registrational intermediate clinical endpoint. In 2025, we continue to make strong progress with AMT130. In April, the FDA granted breakthrough therapy designation, underscoring both the urgent need in Huntington's disease and the strength of our clinical data. In recent weeks, we've had multiple productive interactions with the FDA focused on preparing for a planned BLA submission, and we look forward to providing a detailed regulatory update later this quarter after we receive formal meeting minutes. We're also making good progress towards initiating CNC process validation, another important step in support of the planned BLA submission. In addition to the strong progress made with our Huntington's program, We've also expanded our clinical pipeline with the initiation of three additional clinical studies in refractory temporal lobe epilepsy, or TLE, Fabry disease, and SOD1 ALS. In the quarter, we continue to advance enrollment in both our Fabry and SOD1 ALS studies, and we look forward to presenting initial Fabry data in the second half of 2025. We've completed enrollment of the first two dose cohorts in our SOD1 ALS trial, and currently have several patients screening for our TLE clinical study. We also expect to share preliminary results from the first patient dose in the TLE study at an upcoming scientific meeting later this month. Lastly, Unicure continues to be in a strong financial position with more than $400 million of cash on hand as of March 31st. Last year, we significantly reduced our cash burn with the divestiture of our GMP manufacturing facility, and organizational restructuring. We further strengthened our balance sheet with the completion of a targeted $80 million follow-on offering earlier this year. Together, these actions provide us the financial flexibility to advance our pipeline, including the planned BLA submission and launch of AMT 130, and support key data readouts from our other clinical programs. Overall, I'm incredibly proud of the progress our team made in the first quarter. We delivered on our key objectives and remain on track to meet the strategic goals outlined for 2025. Our continued focus and disciplined execution positioned us well for what we expect will be a transformative year for Unicure. I'll now turn the call over to Waleed to provide a more detailed clinical update. Waleed?