Arcturus Therapeutics Holdings Inc.

Greetings and welcome to Arcturus Therapeutics second quarter 2024 earnings call. At this time, all participants are in a listen only mode. A brief question and answer session will follow the formal presentation. If anyone should require operator assistance during the conference, please press star zero on your telephone keypad. As a reminder, this conference is being recorded. It is now my pleasure to introduce your host, Netta Savarsadeh, Vice President, Head of Investor Relations, Public Relations, and Marketing. Thank you, Netta. You may begin.

Thank you, Operator. Good afternoon and welcome to Arcturus Certificates Quarterly Financial Update and Pipeline Progress Call. Today's call will be led by Joe Payne, our President and CEO, and Andy Sassine, our CFO. Dr. Pat Chivukula, our CSO and COO, will join them for the Q&A session. Before we begin, I would like to remind everyone that the statements made during this call regarding matters that are not historical facts are forward-looking statements within the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements are not guarantees of performance. They involve known and unknown risk, uncertainties, and assumptions that may cause actual results, performance, and achievements to differ materially from those expressed or implied by the statement. Please see the forward-looking statement disclaimer on the company's press release issued earlier today, as well as the risk factors section in our most recent Form 10-K and in subsequent filings with the SEC. In addition, any forward-looking statements We present our views only as of the date such statements are made. Arcturus specifically disclaims any obligation to update such statements. And with that, I will now turn the call over to Jim.

Thank you, Netta. It's good to be with you again, everybody. We look forward to providing our updates today on our quarterly investor call. I will begin my remarks with an update on progress with our vaccine franchise led by Costave, our self-amplifying mRNA COVID-19 vaccine. We're happy to report that we remain on track for the Costave Q4 commercial launch in Japan. Our manufacturing team is working diligently to deliver commercial batches of Costave this quarter. On the regulatory front, our partner Meiji has submitted a partial change application to Japan's PMDA to support the use of the updated CoStave JN1 COVID-19 vaccine for the upcoming 2024 and 2025 season. The European Medicine Agency or EMA continues to review the CoStave marketing authorization application or MAA. The review is ongoing as planned. In May, we published pivotal phase three efficacy, immunogenicity and safety data for CoStave in Nature Communications. The results demonstrate that two-dose primary vaccination at the five-microgram dose level of CoStave, which is our self-amplifying mRNA vaccine, was well-tolerated and immunogenic and provided significant protection against COVID-19 disease. The efficacy of CoStave against severe COVID-19 was 100% in healthy persons aged 18 to 59. and more than 90% in persons at risk of severe consequences of the disease due to comorbidities or older age. We continue to build a meaningful data set for our proprietary STAR self-amplifying mRNA platform as a durable and more persistent vaccine technology. The 12-month persistence results from the CoStave Phase 3 study will be disclosed in September at the 12th Options Conference in Brisbane, Australia. Bivalent CoStave, also known as ARCT2301, continues to demonstrate more broad and durable immune response compared to the bivalent version of Comirnaty. The six-month antibody persistence results from the ongoing bivalent CoStave Phase III study will also be presented at the upcoming Options Congress. The enrollment for the Phase III study of ARCT 2303 is now complete with interim data available later this year. ARCT 2303 is the XBB 1.5 variant version of COSTAVE that's being evaluated in multiple ethnicities in the Southern Hemisphere. The data from this Phase III study is intended to support regulatory filings globally for COSTAVE. and future products utilizing the STAR self-amplifying mRNA platform. Moving on to the ARCT2138 program. This is our quadrivalent seasonal influenza program. The participants recruited for this phase one study in both healthy young and older adults received one of four dose levels of the study vaccine or a licensed influenza vaccine. In addition to the flu-specific data, this study will help us learn more about the scope of the self-amplifying mRNA platform, and more specifically, how high of a dose is reasonably tolerated and how low of a dose is suitably immunogenic. This past quarter, there has been elevated concern regarding the highly pathogenic avian influenza, also known as H5N1 bird flu. The World Health Organization has reported For H5N1 bird flu, a case fatality rate, or CFR, of 52% as of July 22, 2024. They referred to 889 cases of H5N1 and 463 deaths. Many of the human cases reported in the U.S. have been confirmed as H5N1. Arcturus is on track to start a Phase I H5N1 pandemic flu study with support from BARDA in Q4. This vaccine, named ARCT2304, utilizes our proprietary STAR self-amplifying mRNA and lunar delivery platform technologies. The study is to be conducted in the United States and is designed to enroll approximately 200 healthy adults. Now shifting attention to our mRNA therapeutics franchise, let's begin with an update on ARCT032. ARCTO32 is an inhaled messenger RNA therapeutic candidate for cystic fibrosis formulated with Arcturus' lunar delivery technology. And we're pleased to report that we recently submitted an IND application for a phase two multiple ascending dose study to evaluate the safety, tolerability, and efficacy of ARCTO32 in CF patients. I'd like to take a moment to commend our team for working diligently to get this IND submission completed in a timely manner. The IND application for the Phase 2 study is supported by safety and tolerability data collected in a Phase 1 study in 32 healthy volunteers and the two-administration Phase 1b study in seven subjects with CF. No serious adverse events have been observed in any clinical trial participants to date. No febrile reactions have been observed within the target dose range of the planned Phase 2 study. The Phase 2 study intends to recruit CF patients who are ineligible for modulator treatment and additional CF subjects who are eligible but are not prescribed modulators. The CF Foundation patient registry estimates approximately 8% of CF patients are ineligible for modulator therapy and an additional 10% of the CF population are eligible but are not prescribed modulators. Further details pertaining to the design of this Phase II CF study will be provided at an appropriate time later this year. I'll now move on to the ARCT810 program. This is our messenger RNA therapeutic candidate for ornithine transcarbamylase or OTC deficiency. In July, the company announced that the double-blind ARCT 810 Phase 2 study in the EU and the United Kingdom completed enrollment of eight participants, including adults and adolescents, at the 0.3 milligram per kilogram dose level. We're pleased to report that the dosing phase of this first Phase 2 European cohort of eight is near completion, with interim data to be available in the fourth quarter. The company is expanding the Phase II clinical program of ARCT 810 by enrolling OTC deficiency patients in the United States with more serious disease and to recruit younger patients. Patient screening has been initiated, and the company expects the remainder of the Phase II clinical program to be completed here in the U.S. The ARCT 810 U.S. Phase II study has an open-label multiple ascending dose design, It's a study to evaluate the pharmacodynamics and safety of ARCT810 in adult and adolescent participants with OTC deficiency and includes the option to recruit younger patients. Each participant will receive five doses administered intravenously every two weeks at doses ranging from 0.3 to 0.7 milligrams per kilogram. The pharmacodynamic or biological effect of ARCT810 will be assessed by measuring multiple biomarkers that indicate a potential improvement in the activity of the urea cycle. Before passing the mic to Andy, I'd like to take a moment to recognize the recent appointment of Dr. Monsef Slaoui to our Board of Directors. He was previously the Chief Scientific Advisor for Operation Warp Speed. He advised the US President's Council of Advisors on Science and Technology was a member of the advisory committee to the director of the NIH. He built a very respectful career in pharma, leading GSK's global R&D in vaccines, therapeutics, and oncology, which makes him an excellent fit to help Arcturus. We are already implementing his strategic expertise in our product innovation, development, and commercialization strategies. We're fortunate to have him join the Arcturus team as a member of our board. With that, I'll now pass the call to Andy.