Good day ladies and gentlemen and welcome to the Arcturus Therapeutics Third Quarter 2022 Earnings Call. Today’s conference is being recorded. At this time, I would like to turn the conference over to Kyle Gutsadt, Senior Analyst, Investor & Public Relations. Please go ahead, sir.

Thank you, Keith. Good afternoon and welcome to Arcturus Therapeutics third quarter 2022 financial update and pipeline progress call. Thank you all for joining us. Today’s call will be led by Joseph Payne, our President and CEO; and Andy Sassine, our CFO. Dr. Pad Chivukula, our CSO and COO, will join in for the Q&A session. Before we begin, I would like to remind everyone that 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 statements. 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 Forms 10-Q and 10-K filed with the SEC. In addition, any forward-looking statements represent our views only as of the date such statements are made, May 9, 2022. 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 and good afternoon to all. Thank you for joining our third quarter quarterly call. The recent period has been characterized by remarkable progress we have made both with our pipeline and also in completing transformational business development agreements with CSL and BARDA. I will begin with our recently announced collaboration with CSL. I want to first express my gratitude to the entire team here at Arcturus that worked tirelessly on this deal, particularly the deal team led by Lance Carta, our Chief Legal Officer and Kevin Skull, who leads our Business Development Efforts. They all did an exceptional job. Indeed, there is an extraordinary story behind every extraordinary deal, so thank you to the team, many of whom are listening to the call. This collaboration with CSL is designed to develop and commercialize self-amplifying mRNA vaccines targeting COVID-19, influenza, three additional pathogens, and pandemic preparedness. This broad strategic collaboration will drive the development, manufacture, and global commercialization of novel self-amplifying mRNA-based vaccines. This was a transformative deal for Arcturus, both from a financial perspective as well as in positioning our company to become a leader in the development and delivery of mRNA vaccines and therapeutics. CSL Seqirus is one of the top two companies in the multi-billion dollar influenza vaccine market, and they bring profound capabilities, especially related to advanced vaccine manufacturing, development, distribution, and commercialization. The collaboration combines CSL Seqirus’ well-established global vaccine, commercial and manufacturing infrastructure with Arcturus’ manufacturing expertise in innovative self-amplifying mRNA vaccine and LUNAR delivery platform technologies. Together, we will focus on the development of self-amplifying mRNA vaccines targeting COVID-19, influenza, three additional defined respiratory infectious disease vaccines, and pandemic preparedness, and we look forward to providing continued updates on our plans and progress in the coming months. The agreement will become effective upon the expiration of the Hart-Scott Rodino waiting period. Andy will provide further details about the collaboration, but I will briefly mention some highlights. Arcturus will receive $200 million upfront, up to $4.3 billion in potential development and commercial milestones, a 40% profit sharing for COVID-19 vaccines, and up to double-digit royalties for influenza and three additional defined respiratory infectious disease vaccines. These are very meaningful figures and we expect this deal to provide meaningful funding to support the development of our pipeline over the coming years. With the CSL partnership in place, we are prioritizing our regulatory and clinical efforts toward larger commercial markets. CSL is responsible for providing guidance and updates pertaining to ARCT-154 and any of its future derivatives. Now onto the BARDA agreement. This is our second recent external agreement with the Biomedical Advanced Research and Development Authority or BARDA. This transaction provided Arcturus with an award valued at up to $63.2 million over three years. The award will support preclinical, manufacturing, and nonclinical safety studies, along with development and regulatory support for Arcturus’s self-amplifying mRNA vaccine platform technology for rapid pandemic influenza response through Phase 1 clinical studies. Our low-dose lyophilized vaccines are preferable when stockpiling footprint and pandemic preparedness are taken into consideration. Self-amplifying mRNA vaccines have the potential to provide safe and effective protection against disease with the specific advantages of rapid scale-up, lower doses, and easier transport and storage. These are qualities essential to a rapid response against pandemic influenza and are consistent with the strategic objectives of the U.S. government’s national strategy for pandemic influenza. We believe that this highly sought-after BARDA award provides further validation for our technology and its promise to deliver important new vaccines and medicines. This agreement with BARDA establishes a meaningful contractual relationship with the U.S. government. So when the next pandemic occurs, Arcturus may have a more streamlined process to access pandemic-related government funding. I also want to acknowledge that the CSL Collaboration Agreement allows for Arcturus to perform its obligations under the BARDA contract. Now moving to ARCT-810, this is our therapeutic candidate for Ornithine Transcarbamylase deficiency or OTC deficiency. Our therapeutic candidate aims to address the deficient OTC enzyme in the liver of individuals living with this disease. ARCT-810 has the potential to restore urea cycle activity, prevent or slow the progression of neurological damage, and potentially expand dietary options and improve the quality of life for people living with this condition. All subjects in our Phase 1B single ascending dose study have completed dosing, including the cohort dosed at 0.4 milligrams per kilogram without requiring steroid co-treatment. We continue to advance ARCT-810 in a Phase 2 randomized double-blind, placebo-controlled, nested single and multiple ascending dose clinical trial whose design will enroll 24 adolescents and adults living with this disease. The study is being executed in multiple European countries. The participating sites are working with several dozen identified patients through the pre-screening process with dosing beginning this quarter. Next year, the company will strategically share interim ARCT-810 clinical data simultaneously with the announcement of new additional liver therapeutic programs. So we look forward to that. Now moving on to our cystic fibrosis program, we continue to progress the necessary preclinical and nonclinical studies to enable ARCT-032 to move to the clinic. ARCT-032 is our inhaled messenger RNA therapeutic candidate for cystic fibrosis. New preclinical data was presented and well-received at the 2022 North American Cystic Fibrosis Conference last week, and we included some of that data in our press release today. The new data slides presented at the NACFC are readily available on our website if you click on the publications tab. These data provide further support for the therapeutic potential of ARCT-002. Three critical steps for an inhaled messenger RNA are required to be successful: delivery, protein expression, and functional restoration. The first critical step is delivery, getting that messenger RNA to where it needs to be. And these new preclinical data demonstrated effective delivery of messenger RNA to bronchial and tracheal epithelial cells even in the presence of CF sputum or mucus. These successful delivery data are attributed to Arcturus' proprietary LUNAR technology. We have previously shared successful inhaled delivery data in multiple animal models including healthy mice, rats, ferrets, and primates. These new data were collected utilizing a well-established CF ferret model, wherein these animals present mucus that coats the airway epithelial cells in their lungs. The observed effective delivery of messenger RNA in this CF ferret model is noteworthy. On to the second critical step of protein expression, treated human bronchial epithelial cells from CF donors were treated with ARCT-032 in-vitro and demonstrated robust expression of mature CFTR protein at levels comparable to control non-CF or wild-type donors. As for the third critical step of functional restoration, additional in-vitro data demonstrated robust restoration of CFTR transporter activity. Bronchial epithelial cells obtained from human CF donors were treated with ARCT-032. After treatment, we observed a significant increase in chloride-ion current, up to a 70% restoration compared to control BECs or bronchial epithelial cells obtained from non-CF donors. In summary, we have observed successful delivery of mRNA to tracheal and bronchial epithelial cells in the presence of mucus in a CF ferret model. We also observed robust in-vitro expression of CFTR protein alongside functional restoration of chloride-ion current, all comparable to controls. All of these are important milestones for the ARCT-032 program. We believe that this therapeutic candidate, ARCT-032, may bring significant benefits to individuals living with cystic fibrosis, including those unaided by currently available treatments. We continue to anticipate submission of a clinical trial application or CTA for ARCT-032 by year-end. I will now pass the call on to Andy Sassine, our CFO, to provide financial updates.

Thank you, Joe, and good afternoon, everyone. The press release issued earlier today includes financial statements for the third quarter of 2022 and provides a summary and analysis of year-over-year and sequential financial performance. Please reference our 10-Q for more details on the financial performance. I will begin with a summary of the CSL agreement we signed last week. Under the terms of the agreement, Arcturus will receive $200 million upfront and is eligible to receive over $1.3 billion in development milestones and over $3 billion in commercial milestones. In addition, the company is eligible to receive a 40% net profit share for COVID-19 vaccine products and up to double-digit royalties for vaccines against flu, pandemic preparedness, and three other respiratory pathogens. This is a 60/40 profit-sharing agreement on COVID-19 vaccine products, and with respect to the program cost, we expect the milestones will cover all of our expenses going forward. We provided CSL some R&D credits, which are spread out over five years and will likely be applied to the flu and other programs. The CSL transaction is subject to filing under the Hart-Scott Rodino Antitrust Act. The HSR filings have been made by both parties and we are in the waiting period. Assuming the transaction closes and we receive the $200 million upfront payment, we expect that Arcturus will be funded for three years based on our current pipeline, assuming no revenues from any product sales. Let’s assume the milestones will cover all of our COVID program costs. In evaluating new collaborations, we take a comprehensive view of new potential agreements and how they align with our existing agreements to ensure that our plans support our longer-term strategy to advance our pipeline and create shareholder value. Last week, Vinbiocare and Arcturus mutually terminated our mRNA license and COVID-19 supply agreement and entered into a study support agreement. Vinbiocare did not strategically fit in with the combined CSL-Arcturus global manufacturing plan. However, we continue to work closely with Vinbiocare in Vietnam to prepare the clinical data collected for regulatory approval in certain countries. I will now provide a quick summary of our financial results. We reported revenues of $13.4 million for the third quarter of 2022 compared to revenues of $2.4 million in the three months ended September 30, 2021. The increase in revenues was predominantly driven by the Vinbiocare agreement. We reported total operating expenses of $50.2 million during the third quarter of 2022 compared to operating expenses of $56.3 million in the three months ended September 30, 2021. The decline in operating expenses was primarily due to lower manufacturing costs. Finally, we reported a net loss of approximately $35.3 million or $1.33 per basic and diluted share for the third quarter of 2022, compared to a net loss of $54.1 million or $2.05 per basic and diluted shares in the three months ended September 30, 2021. As Joe mentioned earlier, we also signed an agreement with BARDA that will provide up to $63 million in funding to the company over the next three years. Supported by these two agreements, Arcturus is expected to be in a very strong financial position over the next few years. As discussed, the company is expecting to receive $200 million from the CSL upon expiration of the waiting period under the Hart-Scott Rodino Antitrust Improvements Act. We believe that our company has the resources needed to achieve multiple value-creating milestones for vaccine and therapeutic programs. I will now pass the call back to Joe.

Thanks, Andy. We are highly encouraged about the potential of our mRNA vaccine and therapeutic pipeline. We are very pleased with our recent progress. This recent period has been highlighted by the closing of meaningful agreements and continued advancement of our pipeline of mRNA vaccines and therapeutics. We believe our agreement with CSL Seqirus opens enormous opportunities for our company, and we are excited to continue to execute on our mission to bring meaningful new treatments to patients while also rewarding our shareholders. We look forward to providing you with future updates on our progress, and I will now turn the call back to the operator for questions.

Thank you. We will take our first question from Yasmeen Rahimi with Piper Sandler. Please go ahead.

Hi team, this is Lauren on for Yaz. Just two quick questions for you. The first, can you provide some color on the timing for the interim analysis and what exactly will be included in that interim? And then second, how far are you away from filing the CF program in the U.S. and what is left in regard to the CTA? Thanks.

Sure. So the first question, are you referring to ARCT-154 or ARCT-810?

The 810.

Okay, the 810. We indicated that we are going to strategically share that data simultaneously or concurrently with the announcement of our new liver therapeutic programs. And so that is going to be sometime in 2023. Of course, we are disappointed with the progress of our recruitment this year. However, we fully intend not to repeat some of those challenges that we have incurred this year. And we look forward to getting the data as soon as we can next year. But what is important to understand is there is going to be a strategic communication of the interim data that will be concurrent with the disclosure or announcement of additional liver therapeutic programs. And the second question is around the CTA. Now there is very little left remaining to do. We indicated that we remain on track for a filing of that CTA this year, this quarter, so there are not many weeks left. So anything to add, Pad, with respect to the final remaining elements? It is just the application process, the drafting, yes. So all the work has been done and we are just in the process of drafting the filing. That is it.

Perfect. Thank you so much.

We will take our next question from Seamus Fernandez with Guggenheim. Please go ahead.

Thanks for the questions, guys. So can you just maybe help us to understand how it sounds like most of the communications for the COVID program are going to be rolling from CSL. Just hoping to get a better understanding of how you guys are working towards in that process. How the reimbursement for R&D expenses given the sort of 40% profit-sharing agreement, how that is going to work. I just want to get a better sense of what the timelines for communication around the 154 program might be and what kind of is written into the contract for you guys to at least influence that on the margin. And then the second question at the R&D event that CSL posted recently, they did talk about their own self-amplifying mRNA program. Just wondering if you guys have rights to that program, should that program advance or if your own influenza program and theirs may in fact be competing? Thanks.

Sure, sure. There are a few questions there. I will address them one at a time. First of all, it is correct that CSL is responsible for providing guidance and updates pertaining to ARCT-154 and any of its future derivatives. But we are actively supporting them and working with them on the clinical efforts and regulatory efforts. With respect to the question on any sort of CSL flu development programs, if CSL uses Arcturus’ technology or its platform or manufacturing know-how in any way, that will trigger the financials for that program, meaning, the associated milestones and royalties. Now there was a question about the 40/60 or the 60/40 ratio. I don’t know if you wanted to elaborate on that, and I can turn that to Andy. But did I address the two major questions?

Yes, you did. Thank you.

We will take our next question from Yigal Nochomovitz with Citi. Please go ahead.

Hi, John, Andy. How are you? Just a few questions here. This is sort of forward-looking and you may not have all the answers obviously. But with the CF program, what is the envisioned target profile there? I mean, are you thinking this is going to be a daily, a weekly, a monthly inhaled? I know it is very early, but if you have a hypothesis or a vision for what kind of profile you are looking to achieve?

Yes. So what we understand is that we will be able to express the protein CFTR shortly after administration. But what we also recognize is that protein does not have a very long half-life. However, the effect, the functional effect of that protein may extend the durability of it. And that is still being determined by the field. So how often these treatments will be is speculative at this point. We are anticipating weekly, but we do not know that detail until we collect the data.

Okay, got it. And then also a clinical question on OTC. In terms of what you want to see on ammonia reduction to achieve proof of concept, what is your threshold there that you are looking to achieve?

Yes, thankfully the field is maturing in its understanding of what sort of restoration of the enzyme is required for normal function. We have seen that in analyses and publications that have been shared, that it ranges from 4 to 16%. So if there is 4% protein restoration that is viewed as preventing death, which would be very meaningful in this disease, especially in young males. And then all the way up to 16% protein restoration would be required for or expected to establish normalcy in general.

Okay, got it. And then, I don’t know if you can answer this one. It might be more of a CSL question, but I think in the CSL press release they mentioned that they are starting a program with their own flu vaccine next year. So, is your flu vaccine that they might work on going to be a parallel program or a backup program, or how does that all work?

Yes. I think it is best to say that we are combining our efforts on this program. Whatever comes out of it, a combined or one of the either programs is successful, as long as it uses a portion of our technology or manufacturing know-how, it will trigger the full financials of milestones and subsequent royalty. But with respect to which of these programs are ultimately going to be selected to move forward in advanced development, we refer you to CSL.

We will take our next question from Pete Stavropoulos with Cantor Fitzgerald. Please go ahead.

I have one quick question on the OTC program. It is more of a so, when you think about the patient population and the heterogeneity in terms of presentation, the neonatal and the late-onset OTC, as well as other programs in development, such as gene therapies. Assuming 810 achieves the target product profile you hope for, how and where do you see this fitting into the evolving treatment landscape?

There are significant advantages to the Arcturus Therapeutic candidate for OTC being a messenger RNA therapeutic. It is a transient effect, which means that any side effects that are observed would also be transient in nature. There is flexibility and adjustability of the dosing. There is a wide variety of presentation in this disease, so that in some cases it may require lower or higher dosing and adjustable dosing there. This is something that this provides. We also lack steroid co-treatment at least so far with respect to this therapeutic candidate. This is potentially a considerable advantage especially in the OTC deficiency community. So we bring forward all of those elements. And then, Pad, anything else to add?

One other point is that this is a neonatal disease, so going after the younger kids, we think a protein replacement with an mRNA is the ideal solution.

Correct. Versus the other options out there.

Yes. So, I guess part of that neonatal OTC patients usually need a liver transplant from my understanding. So do you view this more as a replacing that need for a liver transplant or as a bridging agent?

Both, depending on the age of the participant and the data that we collect as throughout the next year will help guide those decisions. But absolutely the full intent is to replace any need for protein or, I mean, for liver transplant and establish normalcy and really be a disruptive medicine. But in some cases it may be used as a bridge just to extend life to get you to the next stage of therapy.

Okay. And can you provide any color on near-term milestones from the CSL agreement?

I will let Andy address that. We did mention that there is $1.3 billion in development milestones across five targets, but Andy.

Yes. We are not going to give specifics to near-term, because what we have articulated is that the COVID-19 programs are going to be covered by the milestones. So that certainly will extend our runway significantly. I think I have given kind of guidance that it will be at least three years. So from that perspective, that should alleviate any near-term terms about funding.

Alright. Thank you very much for taking my questions.

We will take our next question from Gena Wang with Barclays. Please go ahead.

Hi, this is Sheldon now for Gena. We have two, one on the CF program, another one on the BARDA agreements. For the CF program, you show the data from F508 del patients’ BC cell line. So in your Phase 1 trial on from the CTA, what patient population are you going to evaluate first, because I suppose the mRNA therapy is most potent in the class one patient, which are not addressable by the current drugs. So could you share your thoughts on the target patient population in Phase 1 and beyond? And my second question is on the BARDA award. So you mentioned that the $63.2 million is going to be spread out over three years. So at what pace or what cadence should we expect those milestones and because it also covers the pandemic flu, is there an overlap with the CSL collaboration or we receive benefit from both?

Great. There are a few questions there. I will begin to address the first question that the Phase 1 trial for CF is intended to involve healthy volunteers. And the strategic thinking there is it allows us to escalate the dose and identify the dose parameters quickly and therefore lock in the dose quickly. And then when we transition to patients, we will be more optimistic about the specific dose. With respect to - I can address the BARDA related questions as well. But Pad anything to add or did I capture - did I address the question on the CTL Phase I question?

Yes, exactly as Joe mentioned. So we are going into healthy volunteers. And that is the regulatory feedback that we received. And based on that regulatory feedback, we are going into healthy volunteers to pick the dose. And then, of course, in our Phase I, we will be looking into patients and that will be a multiple-dose study.

Yes. And with respect to the next question on, yes, it is correct that, that $63 million value award is spread over three years. But this is a cost reimbursement structure that is very typical that you see in BARDA agreements to agreements with the United States government. So there is nothing out of the ordinary with respect to how it is reimbursed. And was there a third question? I think that is it.

No. I mean, is this flu pandemic program also one of the indications that are covered by the CSL collaboration, so are you going to also receive reimbursement from CSL?

No, the BARDA agreement is considered separate. I mentioned that the CSL will - I’m referring to my notes in the script, but just give me a moment. I mentioned that because I want to be careful in my messaging and see us just entering a new partnership. But it is that the CSL collaboration agreement allows for Arcturus to perform its obligations under the BARDA contract just to provide clear guidance there. So it is a separate agreement. We have a relationship with the government, we can operate accordingly. And an ailment on the CSL R&D call recently said that they would approach working with Arcturus as using the best of both companies’ technologies to what is advanced in each of the vaccine fields.

Got it, very helpful. Thank you so much.

Thank you. We will take our next question from Yale Jen with Laidlaw and Company. Please go ahead.

Good afternoon and thanks for taking the question. I just want to be clear that 154 will be the same or similar COVID vaccine for the CSL or CSL will start over with a new version of the COVID vaccine?

Well, you can imagine all of the above. But the strategic thinking and direction and messaging pertaining to ARCT-154 and any sort of future derivatives is going to be communicated by CSL. So would be inappropriate for me to provide guidance with respect to their strategic thinking on any future derivatives.

Okay. Maybe just one more follow-up here, which is the ARCT-032, how should we think about based on the current preclinical data comparing to some other mRNA-based CF treatment maybe earlier one or any comparisons you can make from that.

Yes, I will start and allow Pad to fill in any gaps or provide any additional comments. But we have some unique aspects to our CF therapeutic. It is the only one that I know that utilizes the LUNAR delivery technology. We have applied our proprietary and trade secret know-how on the manufacturing of this messenger RNA, so it is suitably pure for inhalation applications. With respect to the data we have showed data in healthy animals in mice, rats, ferrets, and primates. This new data set is a fifth animal model, and this time it is a CF ferret model, and I think we are the only ones to have this collective set of data. But Pad, anything else to add with how this differentiates compared to others?

Yes, from the data that we have seen at least it generated in the in-vitro model, we look very promising. Obviously, we have restored the CFTR expression to almost wild type levels. So, that data is pretty encouraging. We haven’t seen similar data sets with others yet.

Okay, great, that is very helpful. And congrats on the development.

Okay. Thank you, Yale.

We will take our next question from Yanan Zhu with Wells Fargo Securities. Please go ahead.

Hi, thanks for taking my questions. So first on the COVID-19 or rather the CSL milestones, is it reasonable to assume that the weight across the five different programs might be different and the COVID-19 may be weighted more heavily in a milestone? And if so, could you share a proportion for that? And then also on the new program that you are talking about, you plan on announcing sometime next year. I’m wondering what features are you looking at in making the decision, in terms of the biology, whether de-risked targets or novel targets and any other considerations that goes into that decision? Thanks.

Sure, yes, two good questions. Thanks. So with respect to the first one, I will reiterate that there is $1.3 billion in development milestones across five programs and while we haven’t disclosed and will not disclose the relative amounts of each of those milestones and timelines, it is true that the COVID-19 vaccine is the most advanced and has the shortest development timeline remaining to potential approval, of course, as being the most advanced of those five programs. But other than that, we are not disclosing any additional information. With respect to the new targets that we are seriously considering adding to deliver therapeutic targets, we hold those cards very close to our chest. We understand the value and the specific value of our therapeutic technologies. We have come to realize that the LUNAR technology is exceptional at delivering large RNA molecules and that the technology is biodegradable and non-accumulating and the applicability of this technology through systemic administrations and inhaled administrations. So we are going to definitely leverage our technology differentiation with the LUNAR delivery technology. But with respect to specific guidance, it is too early to do that. There are other competitors, as you can appreciate, in this space. So we will be strategic when we provide more hints there.

Got it. Thank you for the color. If I may add a question for the CF program, with all the animal models you have studied, how in terms of multiple dosing what is the experience there in terms of any potential immune reactions after multiple doses or anti-drug antibodies and things of that sort? Thank you.

Yes. I will begin by discussing the most advanced animal model and human subjects, and then I will hand it over to Pad to cover some additional preclinical evaluations related to the LUNAR franchise. I want to emphasize that in our Phase 1 study, our therapeutic has now been administered to 29 individuals in both Phase 1 and Phase 1B. During the Phase 1 studies, we thoroughly examined lipid decomposition and found that there were no lipids left in the plasma after 48 hours, which was received positively in humans. As for other safety parameters or immunogenic concerns, I will now turn it over to Pad.

Yes. We have talked about this in the past. You might recall that we shared data that of our nine-month safety data - the nine-month tox data for the OTC program. Essentially, we gave weekly dosing for nine months in non-human primates, and we didn’t see any loss in activity of the delivered protein. So that was pretty encouraging for us. Showing the LUNAR was at least pre-clinically it showed quite a promise.

Got it. Thank you for the color.

Thanks.

At this time, there are no further questions in the queue. I would like to turn the conference back to your presenters for additional or closing remarks.

No closing remarks. Just thanks for participating in the call, and if there are any remaining questions, please reach out to the team and we will get back to you right away. Thanks to everyone. Goodnight.

Ladies and gentlemen, this concludes today’s conference. We appreciate your participation. You may now disconnect.