Prothena Corporation plc

Good afternoon. My name is Audra, and I will be your conference operator today. At this time, I would like to welcome everyone to the Prothena Corporation fourth quarter and full year 2022 financial results conference call. There will be a question and answer session to follow. Please be advised that this call is being recorded at the company's request. I would now like to turn the call over to Mr. Eric Endicott, Prothena's Senior Vice President, Corporate Affairs and Communications. Please go ahead.

Thank you, operator. Good afternoon, everyone, and welcome to Perthena's investor conference call to review our business progress, our fourth quarter and full year 2022 financial results, and our 2023 financial guidance. Please review the press release we issued earlier today, which is available on our website at perthena.com and is also attached to a form 8K filed today with the SEC. On today's call, Dr. Gene Kinney, our president and chief executive officer, will provide introductory remarks followed by an overview of Prathena's portfolio and development strategy as we continue advancing toward becoming a fully integrated commercial biotechnology company. Following Jean's opening remarks, Dr. Hideki Guerin, our Chief Medical Officer, will provide an overview of the significant achievements and progress made in 2022 across our entire portfolio. Tran Nguyen, our Chief Financial Officer and Chief Strategy Officer, We'll then review our financial results for the fourth quarter and full year of 2022, and we'll discuss our 2023 financial guidance. Jean will then provide closing remarks, and then we will open the call up for a Q&A session where Dr. Wagner-Zago, our Chief Scientific Officer, and Susana Mesa, our Senior Vice President, Strategy and Operations, will also be participants. Before we begin, I would like to remind you that during today's presentation, we will be making forward-looking statements that are subject to certain risks, uncertainties, and other factors that could cause actual results to differ materially from those referred to in any forward-looking statements. For discussion of the risks and uncertainties associated with our forward-looking statements, please see our press release issued today, as well as our most recent filings with the SEC. We disclaim any obligation to update our forward-looking statements. With that, I'd like to turn the call over to Gene.

Thank you, Eric, and thank you all for joining us today to review our 2022 financial results and business highlights. We're excited today to share Prathena's major achievements in 2022 and how we are advancing a portfolio of drug candidates targeting both neurodegenerative and rare peripheral amyloid diseases. At Prathena, We are driven by our mission to create impactful treatments for the millions of patients that are affected by diseases caused by protein dysregulation. That mission is enabled by our deep scientific expertise, which serves as a unifying thread between our business strategy, our portfolio development, and the dedication that propels Cothenians every day. Turning now to slide five. Today, Prathena is a late-stage clinical biotechnology company with a robust pipeline, which includes four wholly-owned programs and five partner programs. This intentional mix allows us to build a diverse portfolio by leveraging partner payments while still maintaining full upside potential for our wholly-owned assets. For our Alzheimer's programs in 2022, we advanced both PRX-12 and PRX-5 into the clinic, and expect to report top-line Phase I multiple ascending dose data from both programs this year. For PRX123, we have completed several IND-enabling studies and look forward to submitting an IND by year-end. For our Parkinson's disease program, presenizumab is being evaluated by Roche in both the Phase IIb padova and open-label extension portion of the Phase II Pasadena study. We also have two rare peripheral amyloid disease programs. First for Tamimab, a wholly owned program, which is being evaluated in a confirmatory phase three setting and NMC6019, formerly PRX4, which is being evaluated by Novo Nordisk in a phase two study. Collectively, these achievements position us well for a transformational period over the next 24 months. And finally, I will highlight that we remain well-funded to execute on our strategic objectives. We ended 2022 with a strong cash position of $713 million, which included $40 million received for a clinical milestone from Novo Nordisk. Our partnership with Novo, together with our Bristol-Myers Squibb and Roche collaboration, and our wholly-owned programs, allows us to advance our robust pipeline with blockbuster potential, further supporting our goal to address the unmet medical needs of millions of patients affected by diseases caused by protein dysregulation. We believe that our focus on treating and preventing neurodegenerative and rare peripheral amyloid diseases addresses significant unmet medical needs. Our clinical expertise and differentiated approach enables us to advance best-in-class therapies that have the potential to transform the treatment landscape for protein dysregulation diseases. Our focus on developing treatments for neurodegenerative disorders includes therapeutic approaches for Alzheimer's disease and Parkinson's disease, which sadly are growing exponentially. Combined, these two diseases affect an estimated 60 million people globally, a number that is expected to increase rapidly with an aging population. This tremendous social and economic burden is not only experienced by patients, but also by family members, caregivers, and the overall healthcare system. In rare peripheral amyloid diseases, Bertamamab and NNC6019 are being developed in targeted patient populations at high risk for early mortality, which underscores our commitment to develop therapies for patients with an urgent need for improved survival. Before I turn the call over to Hideki, I'd like to highlight several breakthroughs that occurred in 2022 that have meaningfully advanced the overall treatment landscape for Alzheimer's disease. Over the past several months, multiple milestones have been achieved in the Alzheimer's community. Notably, developments include the presentation last November at the Clinical Trials on Alzheimer's Disease, or CTAD, conference of statistically significant and clinically meaningful results across the primary endpoint and all key secondary endpoints from ESI and Biogen's Confirmatory Phase III Clarity AD trial for licanumab. an antibody that targets immunoterminous of A-beta in patients with Alzheimer's disease to both clear plaques and neutralize soluble protofibers. In early January, licanumab received accelerated approval from the FDA for the treatment of early Alzheimer's disease. Notably, the approval was based on Phase II data that demonstrated that licanumab reduced the accumulation of A-beta plaque in the brain which was noted by the agency as a surrogate endpoint reasonably likely to predict positive clinical outcomes. The results from CLARITY-AD, plus the accelerated approval of leucanumab, continues to support the A-beta treatment pathway in Alzheimer's disease, and we believe paved the path for the next generation of plaque-clearing anti-A-beta antibodies, including PRX12, a potential best-in-class subcutaneous treatment for Alzheimer's disease. Another notable development at the CTAD conference was the many scientific advancements in the area of biomarkers. This included research from Dr. Randy Bateman's team at Washington University in St. Louis, which showed that cerebral spinal fluid levels of MTBR tau-243 closely tracked with tau PET, distinguishing amyloid-positive individuals with tau tangles from amyloid-positive individuals without tau tangles. Hideki will cover this exciting data in more detail shortly. At Prothena, we celebrate these advancements and also strive to create novel treatments that further improve efficacy and quality of life for patients. Finding solutions to treat and prevent this disease is crucial. This is why we are advancing one of the most robust Alzheimer's disease portfolios in the industry, guided by our comprehensive therapeutic strategy to address the unmet needs in Alzheimer's. Our portfolio is well positioned in light of these recent scientific advances in the field and positions Prothena as a leader in the transformation of Alzheimer's therapeutic approaches. We are currently advancing three product candidates, PRX12, PRX5, and PRX123, targeting key pathological pathways of the disease cascade, which have the potential to expand from next-generation disease-modifying treatments to combination and prevention paradigms. With that, I'll now turn the call over to Hideki to highlight the progress made across our robust R&D portfolio in 2022.