uniQure N.V.

All participants are in a listen-only mode. After the speaker's remarks, we will conduct a question and answer session. To ask a question at this time, you'll need to press star followed by the number one on your telephone keypad. As a reminder, this conference call is being recorded. I would now like to turn the call over to Kiara Russo, Senior Director of Investor Relations. Thank you. Please go ahead.

Good morning, and thank you for joining us for Unicure's third quarter of 2025 earnings call. Earlier this morning, Unicure released its financial results for the third quarter of 2025, and our press release is available on the investors and media section of our website at unicure.com. Our 10Q was also filed with the SEC earlier today. Joining me on the call this morning are Matt Capista, Chief Executive Officer, Dr. Walid 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 the call-up 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 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 SEC 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, Kiara, and good morning, everyone. Thank you for joining today's third quarter conference call. As you know, in the third quarter, we announced positive top-line data from our pivotal Phase 1-2 study of AMT130 in Huntington's disease, the first gene therapy to demonstrate statistically significant slowing of disease progression in Huntington's disease. These groundbreaking results represent an important milestone not only for Unicure, but also for patients and families who have long awaited a potential disease-modifying therapy. As previously disclosed, we met with the FDA in late October to review our data and discuss the potential submission of a BLA for AMT130. Based on discussions at the meeting, we believe the FDA currently no longer agrees that the data from the Phase 1-2 studies of AMT130, in comparison to an external control, may be adequate to provide primary evidence in support of a BLA submission. Consequently, the timing of a BLA submission for AMT130 is now uncertain. This feedback represents a notable shift from prior communications with the FDA during multiple Type B meetings over the past year. We plan to urgently engage with the FDA to discuss next steps, and we expect to receive the formal meeting minutes within the next 30 days. While the latest FDA feedback is certainly surprising and disappointing, we continue to strongly believe that AMT130 has the potential to provide significant benefit to patients. We believe the data presented to date, widely recognized as the most compelling ever generated in Huntington's disease, provides substantial evidence of therapeutic effect. Every year, thousands of Americans die because of Huntington's disease and thousands more are newly diagnosed. We believe AMT 130 has the potential to significantly slow disease progression and exemplifies the type of transformative innovation in rare diseases the FDA has pledged to support. We remain fully committed to our partners, investigators, and most importantly, to Huntington's patients and their families, and to working collaboratively with the FDA to bring this therapy to Huntington's patients in the U.S. as rapidly as possible. We will continue to act with urgency, transparency, and discipline as we work to deliver on the promise of gene therapy to transform lives.