Arcturus Therapeutics Holdings Inc. (ARCT) Earnings Call Transcript & Summary

Greetings, and welcome to Arcturus Therapeutics Second Quarter 2024 Earnings Call. [Operator Instructions] As a reminded this conference is being recorded. It is now my pleasure to introduce your host, Neda Safarzadeh, Vice President, Head of Investor Relations, Public Relations and Marketing. Thank you, Neda. You may begin.

Thank you, operator. Good afternoon, and welcome to Arcturus Therapeutics' 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. Pad Chivukula, our CFO and COO, who will join in 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 and 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 Factors 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. And with that, I will now turn the call over to Joe.

Thank you, Neda. 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 Kostaive, our self-amplifying mRNA COVID-19 vaccine. We're happy to report that we remain on track for the Kostaive Q4 commercial launch in Japan. Our manufacturing team is working diligently to deliver commercial batches of Kostaive 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 Kostaive JN.1 COVID-19 vaccine for the upcoming 2024 and 2025 season. The European Medicine Agency, or EMA, continues to review the Kostaive Marketing Authorization Application or MAA. The review is ongoing as planned. In May, we published pivotal Phase III efficacy, immunogenicity and safety data for Kostaive in Nature Communications. The results demonstrate that 2-dose primary vaccination the 5-microgram dose level of Kostaive, which is our self-amplifying mRNA vaccine was well tolerated and immunogenic and provided significant protection against COVID-19 disease. The efficacy of Kostaive 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 STARR, self-amplifying mRNA platform, as a durable and more persistent vaccine technology. The 12-month persistence results from the Kostaive Phase III study will be disclosed in September at the XII OPTIONS Conference in Brisbane, Australia. Bivalent Kostaive also known as ARCT-2301, continues to demonstrate more broad and durable immune response compared to the bivalent version of Comimaty. The 6-month antibody persistence results from the ongoing bivalent Kostaive 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 Kostaive 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 Kostaive and future products utilizing the STARR, self-amplifying mRNA platform. Moving on to the ARCT-2138 program. This is our quadrivalent seasonal influenza program. The participants recruited for this Phase I study in both healthy young and older adults received 1 of 4 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 the 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 refer 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 ARCT-2304 utilizes our proprietary STARR 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 ARCT-032. ARCT-032 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 II multiple ascending dose study to evaluate the safety, tolerability and efficacy of ARCT-032 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 II study is supported by safety and tolerability data collected in a Phase I study in 32 healthy volunteers and the 2 administration Phase Ib study in 7 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 II study. The Phase II 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 ARCT-810 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 II study in the EU and the United Kingdom completed enrollment of 8 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 II European cohort of 8 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 ARCT-810 in adult and adolescent participants with OTC deficiency and includes the option to recruit younger patients. Each participant will receive 5 doses administered intravenously every 2 weeks at doses ranging from 0.3 to 0.7 milligrams per kilogram. The pharmacodynamic or biological effect of ARCT-810 will be assessed by measuring multiple biomarkers that indicate a potential improvement in the activity of the urea cycle. Now before passing the mic to Andy, I'd like to take a moment to recognize the recent appointment of Dr. Moncef Slaoui to our Board of Directors. He was previously the Chief Scientific Adviser for Operation Warp Speed, advised the U.S. 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 them 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.

Thank you, Joe, and good afternoon, everyone. The press release issued earlier today includes financial statements for the second quarter of 2024, and provides a summary and analysis of year-over-year and sequential financial performance. Please also reference our most recent Form 10-Q for more details on the financial performance. We are very pleased with the progress Kostaive, our first COVID-19 vaccine has made in Japan. We believe that this product could represent an improved vaccine option for the Japanese population as well as an important source of revenue for our company. I will now provide a summary of our financial results for the second quarter of 2024. For the 3 months ended June 30, 2024, we reported revenue of $49.9 million, a significant increase of $39.4 million from the $10.5 million reported in the same period in 2023. The increase was primarily due to the CSL agreement during the second quarter of 2024. This increase in CSL revenue recognized was driven by the recognition of COVID vaccine manufacturing activity and higher milestone achievements compared to the previous year's quarter. Additionally, revenue related to the BARDA agreement increased due to progress in the pandemic flu program. Total operating expenses for the 3 months ended June 30, 2024, were $71 million compared to $65.9 million for the 3 months ended June 30, 2023. Total operating expenses for the 6 months ended June 30, 2024, was $139.4 million compared with $131.4 million for the 6 months ended June 30, 2023. Research and development expenses were $58.7 million for the 3 months ended June 30, 2024 compared to $52.7 million for the 3 months ended June 30, 2023. The increases in research and development expenses were primarily driven by higher clinical-related expenses and manufacturing expenses across all programs in our pipeline. Additionally, investments increased in early stage and discovery technology, including the initiation of preclinical research related to Lyme disease and Gonorrhea vaccine programs. For the 3 months ended June 30, 2024, Arcturus reported a net loss of approximately $17.2 million or $0.64 per diluted share. Compared with a net loss of $52.6 million or $1.98 per diluted share in the 3 months ended June 30, 2023. Cash, cash equivalents and restricted cash were $317.2 million as of June 30, 2024, and $348.9 million on December 31, 2023. Arcturus achieved a total of approximately $437.1 million in upfront payments and milestones from CSL as of June 30, 2024. We continue to expect to receive future milestone payments from CSL supporting the ongoing development of the COVID and flu programs and 3 additional vaccine programs by CSL. I'm happy to report the expected cash runway extends through the first quarter of fiscal year 2027 based on the current pipeline and programs. In summary, we believe that the company remains on a strong financial position and has the resources needed to achieve multiple near-term value-creating milestones for the vaccine and therapeutic programs. Furthermore, with the anticipated launch of Kostaive later this year in Japan, we look forward to beginning to report potential commercial milestones and revenues. I'll now pass the call back to Joe.

Thanks, Andy. We've continued to make excellent progress on our mRNA vaccines and therapeutics pipeline. We are excited about our upcoming launch for Kostaive in Japan. And we are pleased that the aggregate safety data for our mRNA therapeutics platform supports the continued clinical development of both our flagship rare disease therapeutic programs. I will now turn the call to the operator for Q&A.

We will now be conducting a question-and-answer session. [Operator Instructions] Our first question comes from the line of Whitney Ijem with Canaccord Genuity.

This is Joohwan on for Whitney. Congrats on the positive progress this quarter. Maybe just a few questions here. I know you said you would provide the full details on the trial later this year. But is there anything else you can tell us about the CF Phase II study, like how long the study would be? Or what endpoints do you plan to assess other than FEV1. And then maybe just a quick one on the Phase Ib study. Have you dosed the 7 patients yet? And are there still plans to provide an update on the last 3 patients later this year?

Thanks, Joohwan. Yes, both questions are pertaining to the CF trial. First is on the Phase II trial, I think we mentioned that further details pertaining to the design of the Phase II CF study will be provided at an appropriate time later this year. We did share in today's call that we intend to recruit CF patients who are ineligible for modulator treatment and that includes Class I patients and also the additional CF subjects who are potentially eligible, but they're not prescribed modulators for a variety of reasons. So that helps people understand where the focus, what type of patients we're recruiting, which are the most unmet medical need. With respect to specific dose levels, we've just said that there's no febrile reactions that have been observed within the target dose range of our planned Phase II study based on what we saw in Phase I and Phase Ib. So we're not disclosing that at this time. But there will be an appropriate time for us to share more details pertaining to the design with respect to dosing and how long each cohort will be receiving doses, et cetera. With respect to the Phase Ib, the dosing has successfully completed. No SAEs were observed in any of the clinical participants to date. No febrile reactions again were observed within the target dose range of our planned Phase II study. The LCI, the Lung Clearance Index, was collected. And while it may be encouraging, it's not a validated assay in adults. So our focus now going forward is just simply on the Phase II study, which is going to come here fairly quickly now that we filed an IND. So that's where we'll likely be able to collect some very meaningful and statistically significant data.

Our next question comes from the line of Yasmeen Rahimi with Piper Sandler.

Thank you so much for all the great updates. A few questions. Joe, could you -- you alluded to that the design of the CF study will be coming shortly. Are we just awaiting IND clearance and then you're going to announce the study design? Simple question one. Question two, could you maybe talk about what's the duration of the study, as I apologize if I missed it. And then third question is, could you maybe help us understand the timing for the COVID vaccine approval here in Europe as we were expecting that in the third quarter? And how should we be thinking about sort of -- if there's any eligibility for any orders for 2024 and thoughts around 2025 in Europe?

Yes. Well, with respect to orders, I'll defer to Andy, but I can address your question on the COVID vaccine timing in Europe. CSL is going to be providing all commercial guidance in Europe and U.S. So we defer to them with respect to guidance in Europe. Having said that, the review is active and ongoing as planned. And we're excited to get the first self-amplifying mRNA product approved in Europe as part of that process. But we defer guidance to CSL on that note. With respect to the design and duration of the Phase II trial, there -- we first get approval to proceed as the plan, and then we get the sites up and running and we initiated recruitment of those folks that are ineligible for modulator therapy. And then sometime later this year will be an appropriate time to provide more details to the Phase II design and maybe after we get approval to proceed, assuming success. But we're not going to be sharing any more details on this call today.

Okay. Thank you again. I'll jump back in the queue.

Or but Andy, you had a question with respect to sales, correct? Just wanted to make sure I addressed your question.

No. Unfortunately, we don't provide guidance with respect to vaccine sales that will be provided by Meiji and CSL. So please respect the fact that they have the rights to those 2 vaccines in the various jurisdictions. And they will provide guidance when it's appropriate.

Thanks, Yasmeen.

Our next question comes from the line of Evan Wang with Guggenheim Securities.

Just first on OTC -- just wondering what you consider good data from the EU portion of the trial, just given that there's -- I think that's focused on the 0.3 mg per kg dose while going up to 0.7 mg per kg in the U.S. And then if you can share anything in terms of how recruitment in the U.S. study is going or how patient identification certification is going? And then second, just if you can provide any sort of updates in terms of the Japanese COVID vaccine market in terms of, maybe, I guess, expectations for shots in arm versus the potential upside of having the additional batches called for it?

Okay. So first, with respect to the OTC question, we're definitely interested in collecting the safety and tolerability of the up to 6 doses that these from this initial cohort in Europe. It would be great to see that it's safe and well tolerated up to 6 doses because this is a key precursor to allowing us to get access to younger sicker patients. But we also want to get smarter on biomarkers. This is becoming more and more crucial for the OTC program. It's key that we design an efficient and pivotal trial and that efficient pivotal trial design is going to be closely associated with the biomarker strategy. So what we hope to at least start to initially get some understanding of -- on biomarkers with -- associated with this first batch of data. Moving on to your second question about the recruitment in the U.S. While we've just initiated that the Phase II expansion or the additional Phase II trial in the U.S. So that's all we've said at this point. At a later time this year at the quarterly calls, we will provide subsequent updates to give people an understanding of how we're doing with recruitment there. But one of the primary reasons we're focusing on OTC in the U.S. is to gain access to these younger and more severe patients, right? So we look forward to recruiting those people, but that update will come on the next call. With respect to -- I think your third question was dealing with the Japanese market. Could you restate that for Andy?

Yes. Can you just remind us how you and your partners are thinking about the Japanese COVID vaccination opportunity? And anything you can share on, I guess, maybe timing of the vaccination campaign, pricing, that would be helpful.

With respect to timing and pricing, that's Andy. Go ahead.

Sure. Thank you, Evan. We're pretty excited about the opportunity in Japan and working very closely with our partner with Meijan and CSL. On June 11, just a few months ago, Mr. Kobayashi, the CEO of the Meiji Seika Pharma operations, provided some guidance on how many vaccines they ordered from CSL which was approximately 4 million doses. And they stated that the Japan market for COVID vaccine was about 20 million doses for the last year. So that hopefully gives you kind of a perspective of where it is. And then they hope to increase orders to about 10 million doses next fiscal year if everything is successful in the current fiscal year with respect to sales. They provided Kostaive pricing guidance of about JPY 10,000 per dose. Obviously, with the volatility in the Japanese yen, it's going to be a little bit challenging to try to figure out the dollar equivalent. But is a very reasonable and attractive price for all 3 parties involved. So we're excited. We're going to be shipping the vaccine, as we stated on previous calls, to Meiji probably starting at the beginning of Q4. And hopefully, that gives you the response you were looking for.

Our next question comes from the line of Myles Minter with William Blair.

The first one is just on OTC and it's back on enrollment. I know you're doing this at a single center in the U.S. I'm just very curious as to why you picked that single center to work with. And I'm wondering whether you do have a head start of at least some of the 9 patients already being identified or whether you're going into this relatively blind and recruiting from scratch. That's the first one. And the second one, maybe for you, Andy, is just any update to the sale of the ARCALIS equity position in Japan? Because I know you've been working on that? Anything to update there?

Sure. The site that we're working with is based in Washington, D.C. for OTC, but it's extremely well connected with the OTC community as a whole. So even though it's functionally 1 site, it's actually very well connected with the community and other sites in the United States. And so we we're going to be depending on them to help facilitate recruitment. In terms of -- we'll be able to provide an update of where we are. We're optimistic that we can continue to recruit here in the U.S. and get access to these younger sicker patients. We look forward to providing an update on the next call. With respect to the ARCALIS update, Andy, do you have something there?

Sure, Myles. Thanks for the question. We don't really have an update on the strategic review engagement with JPMorgan Investment Bank at this point in time. The process is ongoing and multiple parties are continuing to conduct due diligence in our data room. We will provide updates when appropriate. And hopefully, that will answer your question.

Our next question comes from the line of Yanan Zhu with Wells Fargo.

So on the cystic fibrosis study enrollment criteria for this part of patients eligible for modulators, but not prescribed modulators. Could you talk about the characteristics of those patients? And also, do you expect this to be a substantial proportion of the enrollment for the study? The COVID vaccine front, I was just wondering, could you give us an update on the progress in manufacturing of the 4 million doses ordered or to be ordered by Meiji. And lastly, on a [ positive event ] flu vaccine Phase I study front, could you remind us of the time line for readout? And also, what is considered a successful outcome for this study. What is the bar for success set by the conventional flu vaccines?

Thanks, Yanan. Well, first of all, that we are focusing on modulator ineligibles, which is typically the null patients, people that do not have any CFTR in their lungs who are -- they're also termed as Class 1 subjects. But there's -- and that's approximately 8% of the CF community, but there is an additional 10% of the CF community that may be eligible for modulators, but they're not getting prescribed modulators, and there's many, many reasons for this. It would be difficult to outline them on a call but there's significant unmet medical need in this group. And so they will be accessing this trial potentially. But the numbers that you're looking for is 8% are modulator ineligibles and 10% of the CF community are not taking modulators for a variety of other reasons. So it's about half and half. Whether that will translate into recruitment for this Phase II trial is yet to be proven out. So we can't provide any guidance there. But it's all about addressing unmet medical need and working with the CF Foundation to do this, and that's where it is. It's in those 2 groups at least in this initial Phase II trial. With respect to the manufacturing update, we're on track. But I can defer to Andy for that in a moment. You also asked a question about the flu study. Now there's a growing number of successful mRNA flu vaccines, which is good to see. Their efficacy numbers are decent, both of those products to support those commercial strategies. And with respect to guidance, we're under fairly strict restrictions to provide guidance on what their commercial strategy is. But that's where the ongoing conversation is, and that's the opportunity for us to step in to help contribute to the flu shot arena. But, Andy, back to you on the -- I think you were just -- I just wanted to clarify, Yanan, you're just asking if manufacturing is update for the 4 million doses or something like that in Japan. Is that what you wanted to clarity on?

Yes. Any progress on the manufacturing of those 4 million doses.

We do have some good news. ARCALIS has successfully completed 2 GMP batches and is currently in the process of completing the final batch. Upon completion, of course, ARCALIS and Meiji will petition the PMDA for approval of their manufacturing plant for commercial production. So as we alluded to in the previous call, the first 4 million doses that was ordered by Meiji through CSL will be provided from our U.S. and European CDMO. And of course, once ARCALIS is qualified to deliver and produce commercial batches, that will give me the -- hopefully the opportunity to provide additional orders directly made in Japan. So we're cautiously optimistic at this point in time, but we still await the completion of the third GMP batch, and we'll certainly update the market upon that progress. Hopefully, that answers your question.

Our next question comes from the line of Yigal Nochomovitz with Citi Group. Please proceed with your question.

Could you give a bit more color on how you plan to recognize the revenue for Kostaive? Is it going to be when it's ordered by CSL or when it's manufactured at ARCALIS or at the CDMO as you referenced, Andy? Or is it only when it's actually shipped out to the Japanese government to the clinics?

Sure. Thanks for the question. We typically don't provide guidance with respect to sales and how we're going to record them. But I did on the previous call, alluded to the fact that we would qualify for a commercial milestone with the delivery of the vaccine to Japan in the first sale. So we'll certainly provide more color around that milestone when it is achieved and earned and reported in our financial results. And with respect to the opportunity to record revenue, that's really going to depend on Meiji successfully selling those doses in Japan. And so once they do sell them and they'll report the sales to CSL and they'll determine the appropriate allocation for CSL, Arcturus and Meiji with respect to the profit split. And then at that point in time, we'll be able to record the revenue when appropriate. So we don't really have any color at this point in time. It's going to depend on Meiji sell-through. But obviously, they're highly motivated and eager to get the process on the road. They're very successful and #1 rated in flu vaccines in Japan. So we have the right commercial partner for this strategy and certainly Meiji have been talking about the opportunity to recognize revenues and sales on the recent conference call. And so we're encouraged. And if appropriate, I think there's a few people from our team that would probably be delighted to fly over to Japan and get our own vaccine as well if we can qualify. Hopefully, that answers your question.

Okay. And of the 4 million doses that have been ordered, is there any way to have a sense as to what percent of those are actually going to be converted to sales in the fourth quarter of this year by CSL?

Yes, I wish I could tell you more specifically. The problem, though, is that Meiji will be responsible for the commercialization of that. And so if you assume that we deliver the vaccines in the fourth quarter, Meiji has a successful opportunity to sell them in the fourth quarter and assume the first quarter of the following year. Then by the time they determine the price and net of whatever fee they have to pay and commission. Then they'll report that result to CSL who will then determine the profit breakout among all 3 parties. So it is not -- it is a straightforward profit but does take time. And we're certainly encouraged by the opportunity and the partnership that we have. So please stay tuned. And hopefully, we'll be able to provide more color around the fourth quarter.

Can you provide any sense as to order of magnitude of the milestone for delivering the vaccine to CSL in the fourth quarter?

It's -- we hesitate to provide specific guidance, but we did say that we will actually earn a milestone. So we are excited to provide that forward-looking statement. And I think in the fourth quarter, we'll be able to share more specifically the amount. The opportunity you'll have with respect to our guidance. So please stay tuned. We're not that far away.

Our next question comes from the line of Pete Stavropoulos with Cantor Fitzgerald.

This is [ Samantha Schaeffer ] on the line for Pete. So the 032 data shows that the 2 doses were well tolerated with no major safety signals. Does this safety profile give you confidence to advance 032 into other indications with an inhaled formulation. And how are you thinking about next potential programs such as other rare diseases or an inhaled vaccine. And final question, does your partner CSL have access to inhaled formulations for vaccine candidates? Or do they solely focus on injection-based formulations?

It's a good question, Samantha. Yes, the Phase I and Phase Ib data, definitely gives us a lot of positive confidence to continue to grow and expand the platform in lung because traditionally, one of the biggest issues, if not the biggest issue in the lung is toxicology, right? So if -- by navigating our way through that with not just a single administration, but with 2 administrations, it does give us more confidence that we can continue to grow the lung franchise. Now before we allocate real hard money to it, I think it will be prudent for us to see some efficacy readout that's relatively short coming here with this Phase II trial. But yes, as a whole, it does provide us some additional confidence in the platform. With respect to the application of inhaled mRNA therapeutics or vaccines, it's unlikely that we'll apply that to the CSL collaboration. It's limited in scope to just 5 targets. And we've already validated the intramuscular administration for use. And so there's only so many targets and so only so many things you can do with that. So it's very unlikely that we'll do an inhaled vaccine with CSL. But we have that flexibility to do that on our own at some point in the future, for sure.

Our next question comes from the line of Ed Arce with H.C. Wainright.

Great. And congrats on the progress. I think some of my questions around Kostaive have been answered, but I just want to ask turning to the therapeutics portfolio. First, on 032 a lot of discussion there, and perhaps this is a bit premature, but just wondering if there is any potential time line to at least preliminary or interim data from that Phase II. And then with 810, I recognize that this fourth quarter readout is an interim. And just so just looking for specifics around the data that you expect and more specifically, what would you view as the bar for success on that readout as you continue the study towards full results.

Sure, sure. With respect to the 032 time line, it's as soon as possible. That is such a strategically important therapeutic for Arcturus, has an extraordinary value upside. If we establish proof of concept, not only for the asset but for the platform. So we -- ASAP, we haven't given specific guidance as to when the data will be forthcoming. We need to get approval to proceed and get the sites on board and then determine the pace of recruitment thereafter. So at each of these quarterly calls, we'll be able to provide some more detailed or more granularity on time line. But rest assured, it is a very important program for a Arcturus strategically. So we want to get it done as soon as possible. With respect to the 810 interim data, it is the first complete cohort out of Europe. It is a 8 folks. We discussed this a little bit with the previous question, but just to emphasize that the safety and tolerability of up to 6 administrations for injectable mRNA is a substantial hurdle. So just by getting that completed, it allows us to open up regulatory conversations into the younger children. And a lot of these rare diseases are pediatric in nature. And so I think that, that's very meaningful data set. Now whether Wall Street appreciates it or not is another question. But internally, that's very important for us to gain access to a sicker and younger patients in general. And the biomarker strategy, just to reemphasize that, in OTC an efficient pivotal trial is going to be leaning significantly on the biomarker strategy. It can be shorter and leaner, and faster if we have [ first smart ] biomarkers. so getting smarter is what we want to do with -- we're measuring a lot of biomarkers in this group of 8 out of Europe, and we just want to understand which ones to focus on, which ones can really drive a pivotal trial design. So that's what we're trying to get out of that. Thanks, Ed.

Thanks, Joe. Actually, following up on that, I meant to ask about those biomarkers. I'm not sure if some or all of those are on clinical trials or not. But any take on what are the ones that you're particularly interested in that would be key to sort of moving forward from an efficacy perspective?

Yes. The challenge here, ammonia is the easiest biomarker measure, unfortunately, in the advanced disease area, those that are in serious or severe disease. They're all on ammonia scavengers which complicates the measurement of ammonia as a biomarker. So this is where the strategy is key. We need to identify a biomarker in folks that are on ammonia scavengers. So we are looking at other amino acids that are implicated in the urea cycle that include glutamate, for example. But we also are looking at OTC itself, and we're looking at ureagenesis. This is a urea cycle disorder. So the genesis or generation of urea can be tracked and measured. So we're looking at ways to do that. So we're -- but the overarching statement here is we want to get smarter at looking at these biomarkers. And in the urine, you can look at orotic acid as well. So there's lots that we're going to be collecting, but we just want to get smarter and figure out what we can apply more in a targeted way, especially with this U.S. expansion into sicker and younger OTC subjects.

Our next question comes from the line of I-Eh Jen with Laidlaw & Company.

We got two here. The first one is that for the Omicron XBB 2303 vaccine you are completing that. So when we should anticipate the top line readout? And another follow-up on that particular one is that what might be the next move? I know you want for the U.S. and other global applications, but what -- any specific move that could -- you might be able to share at this point?

Yes. So the 2303 study has completed enrollment, and the meaningful aspect of that study is the fact that our platform is getting experience in multiple ethnicities and this will support regulatory applications globally, not just the U.S. So it's -- that's the primary strategic role of that. So whether we share that data or not is uncertain. Again, this is licensed -- this is a license program to CSL and they'll ultimately be deciding what gets shared publicly, but that's the purpose of it, and it's completed and that data will be coming in throughout the remainder of this year, but it is intended again to support regulatory conversations and regulatory filings, marketing authorization applications, et cetera, for not only Kostaive but subsequent products that utilize this platform. And then you said you had another question. Yes, go ahead.

Yes, I have one more question here, which is Meiji had filed changing the adjustment for the vaccine to the Japanese regulatory authority. Do we know any time line or projected time line in terms of the agency will approve that change to the JN.1 vaccine.

Yes. The time line for approval is suitable for us to launch and market this year. And it's in line with the other approved vaccines. So now that we have an approved platform, it's nice that we can piggyback those that are already approved and they usually funnel all of these together through the same process of approval for the updated JN.1 variant, for example. So very similar time lines are even identical in some cases to the competitors in the space. But it's suitable for us to market it and launch it successfully this year.

Thanks [indiscernible] and congrats on progresses.

Thank you. There are no further questions at this time. I would like to turn the floor back over to Joe for closing comments.

Thanks for participating in the call. And if there's any remaining questions, please don't hesitate to reach out to the team, and we'll get back to you right away. Thanks, everyone.

Thank you. This concludes today's teleconference. You may disconnect your lines at this time. Thank you for your participation.