uniQure N.V.

Good day and thank you for standing by. Welcome to the AMT 130 Huntington's Disease Program update. At this time, all participants are in a listen-only mode. After the speaker's presentation, there will be a question and answer session. To ask a question during that session, you'll need to press star 1 1 on your phone. Please be advised that today's conference is being recorded And I would now like to hand the conference over to Ms. Maria Cantor, Chief Corporate Affairs Officer. Ms. Cantor, please go ahead.

Good morning, and thank you for joining us. This morning, Unicure announced that it has postponed patient enrollment in the higher dose cohort of the Phase 1-2 clinical trial of AMT-130 in Huntington's disease based on reported SUSAR events at this dose. We will discuss these events and provide an update on the program during this call. Joining me for the investor event and webcast are Matt Capistar, our Chief Executive Officer, and Dr. Ricardo Domelch, our President of Research and Development. We'll be making forward-looking statements during this investor call. All statements, other than those 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 the factors described under the heading Risk Factors in Unicure's quarterly report on Form 10Q, filed today, August 8, 2022, and other securities filings. Given these risks and uncertainties, Investors 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 CEO.

Thank you, Maria, and good morning, everyone. On June 23rd, we announced encouraging 12-month follow-up data from the low-dose cohort of our U.S. Phase 1-2 study that showed AMD-130 was generally well-tolerated with neurofilament light levels near baseline in treated patients and meaningful lowering of mutant Huntington protein supportive of TORX engagement. As announced in our press release this morning, in July we were informed of two suspected unexpected serious adverse reactions, or SUSARs, in the higher dose cohort of our open-label phase 1B2 clinical trial of AMT-130 in Europe. Both of these patients, which were the first two patients dosed at a single clinical site, presented with localized inflammatory responses and other related symptoms within one to two weeks after their procedures. A third patient who had previously been treated with a higher dose of AMT-130 in the US in March of this year experienced severe headache and other related symptoms also shortly after their procedure. That event was initially deemed by the investigator as not related to AMT-130, but related to the procedure. Upon further review and discussion with the Clinical Trials Independent Data Safety Monitoring Board, or DSMB, we have reclassified and reported that patient's reaction as a SUSAR. Importantly, these three patients are no longer hospitalized and have since fully or substantially recovered. The DSMB does not view these events as a dose-limiting toxicity, and thus far in our investigation, we have not yet identified the root cause of these events. Nevertheless, we in the DSMB believe It is prudent to voluntarily and temporarily delay the enrollment and dosing of additional patients with the higher dose until we complete a comprehensive safety review in conjunction with our GSMB. During that time, we will continue our investigation and consider potential risk mitigation plans. We expect to do this over the next 60 to 90 days. This delay does not apply to any future procedures at the low dose where no significant adverse events related to AMT-130 have been reported. Thus far, a total of 36 patients have been enrolled across our U.S. and European clinical trials, including 26 patients treated with AMT-130 and 10 control patients. Of those treated, 14 patients have received a higher dose, with some having now been followed for up to one year. An additional 12 patients have received a lower dose, with some having now been followed for up to two years. Over the course of our studies, the independent DSMB has conducted six separate safety reviews, and aside from these three events, no other SUSARs have been reported. We do not expect this delay will have any impact on the data readouts in 2023. In the U.S. Phase 1-2 study, all 26 patients in the first two dose cohorts have been enrolled, and the company continues to expect to present one- to two-year follow-up data in the second quarter of 2023. In the European Phase 1B study, Phase 1B2 study, the six-patient lower-dose cohort is fully enrolled, and the company continues to expect to present one-year follow-up data in 2023. Four of nine patients in the European study have been enrolled in the higher-dose cohort. Before I hand the call over to Ricardo, I want to stress that patient safety and well-being will always be Unicure's top priority. and we are committed to working with the DSMV to complete our investigation as soon as possible. Now, let me turn the call over to Ricardo.