Arcturus Therapeutics Holdings Inc.

Good afternoon, ladies and gentlemen, and welcome to the Arcturus Therapeutics first quarter 2023 earnings conference call. At this time, all lines are in a listen-only mode. Following the presentation, we will conduct a question and answer session. If at any time during this call you require immediate assistance, please press star zero for the operator. This call is being recorded on Tuesday, May 9, I would now like to turn the conference over to Nada Safarzadeh, Vice President, Head of Investor Relations, Public Relations, and Marketing. Please go ahead.

Thank you, Operator. Good afternoon and welcome to Arcturus Therapeutics' first quarter 2023 financial update and pipeline progress call. Today's call will be led by Joseph Payne, our President and CEO, and Andy Sassine, our CFO. Dr. Pat Chivikula, our CSO and COO, will join in for the Q&A session as well. 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 risks 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 factor section in our most recent Form 10-K and in subsequent filings with the SEC. In addition, any forward-looking statements represent our views only as of the date such statements are made. Arcturus specifically disclaims any obligation to update such statements to reflect future information, events, or circumstances. And with that, I will now turn the call over to Joe.

Thank you, Netta, and good afternoon to all, and good evening to our friends on the East Coast. I will begin my remarks by highlighting our ARCT 154 COVID-19 vaccine phase 3 program. This is our most advanced clinical program. ARCT 154 has the potential to offer effective and longer lasting protection against COVID-19. I'm very pleased to report that last month our collaborator Meiji submitted a new drug application to support potential approval of ARCT 154 as a primary immunization vaccine based on our placebo controlled phase three study. The phase three study was conducted during a period of multiple variants of concern and met its primary endpoint of preventing COVID-19 and demonstrated a favorable safety profile. The study was conducted to assess efficacy against COVID-19 in approximately 16,000 individuals and we're very pleased with the results. The ARCT154 Phase 3 comparative study of ARCT154 as a booster is being conducted by Meiji Seika Pharma and they have completed enrollment of approximately 828 subjects with interim results expected later this quarter. This non-inferiority study is designed to evaluate the safety and immunogenicity of ARCT 154 compared to Carbon Addi of Pfizer and BioNTech and administered as a booster dose. We expect the interim analysis data to be submitted to the PMDA later this quarter and to seek registration of the ARCT 154 as a primary series and booster later this year, potentially representing our company's first product approval. Such a meaningful milestone could be indicative of the broader platform opportunity for our mRNA medicine technologies to result in novel vaccines and therapeutics over the coming years. If approved, Japanese sales of ARCT154 could represent a significant commercial opportunity for Arcturus. I will also remind you that the ARCT 154 phase 3 Japanese booster study as well as product manufacturing related to this collaboration are being funded by Meiji Seika Pharma and the Japanese government. In April, Meiji Seika Pharma entered into an agreement with CSL Securus whereby Meiji will be responsible for the distribution and sales of ARCT 154 in Japan. We are indeed fortunate to be partnered with competent and experienced commercial partners. In April 2023, we received an advance payment of $23.6 million for the manufacturing and supply of ARCT 154 from CSL. The advance payment is for specified manufacturing runs of ARCT 154, which includes the drug substance as well as the reservation fees and related manufacturing requirements. So, ARCT is different than conventional mRNA vaccines in meaningful and important ways. The dose is very much lower. The product is lyophilized. It's not a liquid or a frozen liquid. These features bring potential dose-related safety benefits and provide a much better shipping, storage, and supply chain. ARCT 154 has shown and continues to show broad neutralizing capability against multiple variants of concern. ARCT 154 has the potential to offer not only effective, but also longer lasting protection against COVID-19. And on top of this backdrop, we will soon be able to share the phase three safety and immunogenicity booster data with multiple regulators across the globe. This is indeed an exciting time for our vaccine franchise. I'll now move on to update on ARCT810. This is our mRNA therapeutic candidate for OTC deficiency. This investigational medicine is designed to address the deficient OTC enzyme in the liver and thereby restore urea cycle activity and to prevent metabolic crises that cause neurological damage. ARCT 810 could potentially liberalize the strict dietary protein restrictions that OTC patients face today and improve quality of life for those living with this condition. ARCT 810 utilizes Arcturus's proprietary lunar delivery technology. An important attribute of our technology is that the lipids administered are rapidly degraded, which we expect lead to a favorable safety profile. ARCT 810 is being evaluated in two ongoing clinical studies in patients, a phase 1B study in adults and a multi-dose phase 2 study in adolescents and adults. Enrolment has begun in the ARCT 810 phase 2 study in the UK and Europe. The phase 2 multiple dose study is designed to enrol up to 24 adolescents and adults with OTC deficiency. And Arcturus plans to share interim phase two data on a subset of participants later this year in 2023. Now I'll move on to ARCT032, our inhaled messenger RNA therapeutic candidate for cystic fibrosis. This program is designed to express fully functional CFTR protein in the lungs of individuals with CF. Utilizing our lunar delivery technology, that has been optimized for inhaled lung delivery. Our approach is agnostic to the underlying mutations associated with the disease, and as a result, ARCTO32 could provide clinical benefit across a wide range of those living with CF, including those that are not well served by currently approved CFTR modulators. The clinical development of ARCTO32 is supported by encouraging preclinical data, demonstrating successful CFTR protein expression in the airway epithelium of the lung in different animal species and the restoration of the CFTR channel in a CFTR knockout mouse model and shown functional delivery of mRNA in a CF ferret model. In addition, in vitro administration of ARCT032 to bronchial epithelial cells from CF patient donors has also demonstrated robust expression of CFTR protein as well as functional restoration of chloride current. The ARCTO32 clinical development program continues to advance according to plan. We're pleased to report today that we have successfully completed the enrollment and administration of a phase one study with 32 healthy participants, including eight subjects in each of the four doses being tested. and we anticipate reporting study results later this year. The safety and tolerability data support the study expansion and inclusion of patients with CF. Arcturus is working on a protocol amendment to allow the dosing of CF patients and expects to initiate the enrollment in Q3 of this year.