Orchard Therapeutics plc

Welcome to the Orchard Therapeutics Q1 2022 earnings conference call. My name is John Aubrey, operator for today's call. At this time, all participants are in listen-only mode. Later, we will conduct a question and answer session. During the question and answer session, if you do have a question, press 01 on your touchtone phone. I will now turn the call over to Bobby Gaspar.

Hello, everyone, and welcome to Orchard Therapeutics first quarter 2022 earnings Financial Results and Corporate Update webcast. I'm CEO and co-founder, Bobby Gaspar. Before we get started, I want to remind everyone that this presentation contains forward-looking statements. Please refer to this slide and our latest SEC filings for more information. I have three colleagues joining me on today's call. Braden Parker, our Chief Commercial Officer, is joining us to highlight the success we've recently had expanding commercial access for Lubeldi. Frank Thomas, President and Chief Operating Officer will review our financial results for the quarter. Leslie Meltzer, our Chief Medical Officer, will also be joining for Q&A. In March, we spoke about our decision to strategically prioritize the focus of our platform to synergistic indications where we believe HSC gene therapy is scientifically and clinically differentiated and where it can deliver the most value. Through the insights we've gained, for many years of scientific and clinical development in this field, we believe indication selection in our space is paramount, and this is what is driving our strategic approach and decision-making. Firstly, we want to focus where the HSC approach has differentiated itself scientifically and clinically. That is, in neurodegenerative conditions like MLD, where the migration of gene-modified HSCs into the CNS is key to delivering the gene where it is needed. With Lumeldi, we have seen how focusing in this area can lead to clinical benefit, regulatory approval, and increasingly commercial success. Throughout the start of 2022, Orchard has built sustained commercial momentum for Lumeldi as the first HSC gene therapy with meaningful reimbursement coverage in Europe. We're extremely pleased by the recognition of Lumeldi's value commensurate with its clinical impact, which is reflected in the published net price of 2.4 million euros attained in Germany, and which is indicative of our agreement throughout the region. We're encouraged by the continued progress we're making to identify, refer, and treat eligible patients with this therapy. In addition, we believe that a further focus on neurometabolic diseases, such as MPS disorders, will offer a synergistic approach, and enables us to leverage a common infrastructure from manufacturing and CMC to distribution, supply chain, and our commercial network, as well as regulatory capabilities. We've also chosen to focus on our research programs using HSC gene therapy for specific larger disease indications with genetic susceptibilities, such as the progranulin form of frontotemporal dementia or the NOD2 form of Crohn's disease. As we develop these further and as the preclinical data matures, we believe these programs provide opportunities for significant value creation, either on our own or through potential partners with an interest in our platform or indications. At the upcoming ASGCT meeting, Data from our neurodegenerative programs will be presented that continue to demonstrate the unique ability of our approach to enable broad distribution of gene-corrected cells and localized delivery of therapeutic enzymes and proteins in the brain. Featured presentations include updated results on the OTR203 clinical program for MPS1 hurlers, as well as preclinical work to advance the platform's applicability to address other neurodegenerative and CNS-related conditions including the progranulin form of FTD. So we continue to build on the deep body of data driving our mission to develop the curative potential of HSC gene therapy and create even greater value for the communities we serve. I'll now turn the call over to Braden to review some exciting progress on the MELDI commercial launch.

Braden. Thanks, Bobby. Since Lameldi's approval and launch, we've been building out multiple facets of our commercial infrastructure to identify and treat as many eligible MLD patients as possible. By design, this foundation can also support our next wave of neurodegenerative programs in the MPS space. These additional indications represent exciting commercial opportunities that can leverage much of the investment we are making in Lameldi today. Since the launch, we've prioritized the activities and initiatives we believe will generate the most value for MLD patients and providers. On the access and reimbursement front, we've seen our positive engagement with health technology assessment bodies and payers throughout the region yield agreements securing reimbursement for all eligible MLD children. After announcing an agreement with NHS England in February, Italy and Germany became the second and third major European markets to recognize the value of Lameldi commensurate with its clinical impact. The German net price of 2.475 million euros is indicative of the level at which we've been able to secure access to Lameldi. These agreements come after Lameldi received the highest possible therapeutic benefit rating in pre-symptomatic patients by the German Federal Joint Commission, which only four other medicines have ever received. and the Italian Medicines Agency recognized the significant potential clinical benefit Lameldi offers by granting it innovative status. We believe the progress in Europe and the value recognized by payers has differentiated this product and our team and demonstrated a willingness to ensure access for MLD patients across the continent. We intend to continue expanding access by securing agreements in additional European markets, We are in various stages of technology assessment and reimbursement discussions with joint HTA assessment consortia, such as Benalexa and Phenose, as well as Insane. We have plans to enlarge our network of treatment centers in a similar fashion by qualifying centers to administer Lomeldi in Sweden and Spain, in addition to our existing centers in the UK, Netherlands, Germany, Italy, and France. Over time, this same network of centers would be utilized to support future potential launches in similar indications. And in order to help ensure we are identifying as many eligible patients as possible, we are continuing to increase our support for newborn screening to drive early diagnosis. We now have seven studies ongoing in Europe and one in the U.S., which are intended to generate data to support the adoption of regular screening at the state, national, and regional levels. These programs could also identify MLD patients to be treated commercially if reimbursement is in place at the time of diagnosis. We are continuing to assess how to best expand our geographic footprint beyond Europe and the U.S. to regions such as the Middle East, Latin America, and Asia Pacific in a capital-efficient manner. As we've mentioned previously, our partner GenPharm in the Middle East has already identified a patient who received the MLD through reimbursed international treatment abroad at our qualified treatment center in Milan, Italy. I'll hand it back to Bobby to discuss our U.S. regulatory plan and the rest of the neurometabolic portfolio. Bobby?