ProQR Therapeutics N.V.

Good morning, and welcome to the ProQR Investor Call. Today's conference call is being recorded. At this time, I'd like to turn the call over to Sarah Kiley, Vice President of Investor Relations and Corporate Affairs. Please go ahead.

Thank you, and good day, everyone. We appreciate you joining our event today. Today we are pleased to highlight new preclinical proof of concept data for our AX081 and 0810 program targeting NTCP, which was presented yesterday at the ASGCT annual meeting in Baltimore. On slide two, you'll find the agenda for our call and our speakers. From the management team are Daniel DeBoer, our founder and CEO, who will provide a brief introduction, and then Sarah Plattenberg, our Chief Scientific Officer, who will take us through the data presented at ASGCT. Following the presentation, we will have a Q&A session with covering analysts before we conclude the call. Today's event is being recorded and we will have the replay available on our website following the event. You can also find our ASGCT poster under the publications and presentation section of our website. On slide three is our forward-looking statements. During the call today, we will make forward-looking statements There are risks and uncertainties associated with an investment in ProQR, which are described in detail in our SEC filings. I will now turn the call over to Daniel. Daniel?

Thank you, Sarah, and good morning and good afternoon, everyone. Thank you for joining us today. We are pleased to share an update with you on the data that we shared at the ASTTC annual meeting. But first, I'll provide a brief overview of the company's strategy and our proprietary Axiomar RNA editing platform. Axiomer was invented at the Procure Labs in 2014 and uses the well-proven modality of oligonucleotides to recruit a novel mechanism of action. Axiomer uses editing oligonucleotides, or EONs, to recruit endogenous ADAR to edit individual bases in the RNA. ADAR is present in all human cells, and RNA editing is a naturally occurring process. In fact, it's happening in all of us right now. Our proprietary Axiomar platform makes use of the ADAR mechanism that Nature has developed and recruits it to edit specific nucleotides in a targeted way. Preclinical platform data demonstrate that Axiomar is broadly validated across multiple genes. And today we'll focus on the preclinical proof of concept for our AX810 program, targeting NTCP for cholestatic diseases. Our strategy includes both in-house development of pipeline programs, initially including AXA10, as well as AX1412, targeting the B4K1 gene for cardiovascular disease, and using the Axiomar technology for selective partnering and expertise in technology, like in our partnership with Eli Lilly, allowing us to capture the full value of this platform technology. Procure has quite literally led the field of RNA editing since 2014. When Procure scientists invented the RNA editing, technology using endogenous ADAR and performed the first experiments using our editing oligonucleotides to recruit natural and endogenously expressed ADARs. These experiments also led to the first IP filings through this technology back in 2014, which laid the foundation for our leading IP estate today. Procure holds more than 10 platform patents protecting the use of oligonucleotides to recruit endogenous ADAR broadly. Several of these patents have been granted and were subsequently opposed in several jurisdictions, where the patent courts ruled in favor of Procure and upheld the patents. So our foundational IP estate is not only granted, but also tested in opposition and survived that, which reaffirms the conviction in our leading IP estate. Finally, as we reported in our Q1 financials this morning, I also note our strong cash position, N&Q 1 with approximately 103 million euros, providing a runway into proper mid-2026. Now moving on to our pipeline on slide five. As you will see, our pipeline contains a variety of targets for rare and prevalent disease, as well as wholly owned and partner programs. We plan to capture the value of our platform across two strategies. First, through the development of an internal pipeline of high-impact medicines, and second, through selective partnering. We're initially prioritizing AX810 for cholesthetic disease and targeting NTCP, and the AX1412 program for cardiovascular disease, which targets the B4 GALT1 gene. There are a number of earlier stage programs as well, which we will share more about in due course. We've partnered with Eli Lilly on currently 10 targets on the Axiomar platform, where ProQ leads the discovery phase, and Lilly all phases beyond that. This partnership so far brought in $125 million in upfront payments, and Lilly holds an option to expand the partnership from 10 to 15 targets, for which they would pay an additional $50 million in opt-in fee. In addition to this, Procure is eligible to receive $3.75 billion in milestone payments plus royalties. We are very pleased with the partnership with Eli Lilly and continue to execute on that with high priorities. We're also pleased to note that at ASCCC, we actually have a poster together with Lilly on some of the development work we were doing with Lilly. We also earlier this year announced a partnership with the Red Syndrome Research Trust, which is focused on developing editing oligonucleotides, targeting an underlying genetic variant that causes Red Syndrome, a progressive neurodevelopmental disorder caused by genetic mutations in the MECP2 gene. Given the vast opportunity with the platform, we have appetite and capacity to selectively form additional multi-target discovery partnerships. Moving on to slide six, building on our experience from the last 10 plus years, we have designed a translational strategy that we believe gives a high probability of success for our first in human trials. Our objectives are to generate a data set in human studies where we are studying editing and disease-relevant biomarkers with proper sample sizing. To do so, we select the targets that introduce a variant in a wildcard sequence such that this allows us to study target engagement and biomarkers in healthy volunteers. The advantage is that in a healthy volunteer study, we can, in a much more efficient way, have appropriate sample sizing in each cohort get a data set without disease background noise, and get to a high-value data set in a short amount of time. The targets are largely de-risked because we source these from human genetics research, from populations that carry these variants, which are associated with health benefits. So in these trials, we can, in a cost- and time-effective manner, demonstrate RNA editing or target engagement, disease-relevant biomarkers, proper PK and dose finding, in addition to safety. and vulnerability. With that, I'm now pleased to hand over the call to Gerhard Plattenburg, who will take us through our data presented here at ASGTC.