Arcturus Therapeutics Holdings Inc.

Jim, welcome to the Arcturus Therapeutics first quarter 2022 earnings call. Today's conference is being recorded. At this time, I'd like to turn the conference over to Dipankar Roy, Senior Director of Investor Relations. Please go ahead, sir.

Thank you, James. Good afternoon and welcome to Arcturus Therapeutics first quarter 2022 financial results and corporate update call. Thank you all for joining us. Today's call will be led by Joseph Pate, our President and CEO, and Andy Sassine, our CFO. Dr. Pat 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 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 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 9th, 2022. AFTURA specifically disclaims any obligation to update such statements to reflect future information, events, or circumstances. With that, I'll now turn the call over to Jill.

Hey thank you Dipankar. Good afternoon to all. Thank you for joining Arcturus' Q1 quarterly call. My comments today initially capture a list of summary points for the for the quarter and I'll follow that up with additional detail per program before I turn the time over to Andy for financial updates. This quarter has been highlighted by several key achievements. Indeed we've made significant progress advancing our pipeline of mRNA based vaccines and therapeutics. So let's begin with ARCT 154. This is our most advanced COVID vaccine candidate that has shown impressive activity against SARS-CoV-2, both as a primary vaccine and as a booster. We're pleased with the promising results coming from a Phase 1-2 study evaluating ARCT 154 and its potential as a booster in individuals primed with Comirnaty. The boost with ARCT 154 generated high booster numbers, these are the geometric mean fold rises or GMFRs in neutralizing antibody titers and we'll share more of those results in a moment. Importantly, this quarter we learned from VinBiocare that the phase one slash two slash three study has met its primary endpoint objective of vaccine efficacy. And that ARCT 154 demonstrated very strong protection against severe and fatal COVID disease, even when up against the most challenging variants that have escaped other vaccines like Delta and Omicron. We believe this is a significant milestone for the company and for the field of RNA vaccines. This represents the first time ever that phase three vaccine efficacy endpoint has been achieved. with a low-dose self-amplifying mRNA vaccine, showing that we can prevent COVID-19 symptomatic disease, severe disease, and death. With over 19,000 participants involved in this study and a comparable safety profile in the ARCT154 vaccinated arm and placebo arms, respectively, we view this as important validation of our self-amplifying mRNA technology platform and its applicability towards developing vaccines. The vaccine efficacy data, along with the available safety and immunogenicity data, has been shared with the Vietnam Ministry of Health, and we anticipate a decision around the EUA in the near future. The application for full market authorization is expected to be filed later this year. We're also pleased to share that Arcturus has provided an expression of interest letter to the World Health Organization to potentially include ARCT154 in its emergency use listing process. We're sincerely thankful to the WHO and to the Vietnam Ministry of Health for their support and encouragement in this process. Coming back to our booster trial, last week we released additional data from our Phase 1-2 booster clinical trial here in the United States and Singapore, showing the durability of neutralizing antibody response extending to at least three months after a five-microgram ARCT154 booster dosing following primary vaccination with Comirnaty. We announced new data today that our low-dose self-amplifying mRNA vaccine shows a 46-fold rise in neutralizing antibody levels for Omicron BA.2, and that's a month or 29 days post-boost. All of these data, including those from the current Omicron variants, provide important clinical validations and highlight ARCT154's potential as an effective booster candidate against a broad range of variants of SARS-CoV-2. As the world transitions now into an endemic period of COVID, the booster market represents the most significant commercial opportunity. These data are a step towards ARCT154 realizing that potential. We have begun our startup activities toward a global registrational booster trial and look forward to starting dosing soon. Now let's get into the details now about the ARCT154 clinical results. I'll begin with a recap of the ARCT154 phase three efficacy data that we reported a few weeks ago. The ongoing phase 1, 2, 3 registrational study sponsored by Arcturus' collaborator in BioCare enrolled over 19,000 adult participants in Vietnam, including a large percentage of individuals at higher risk of severe complications of COVID disease. The phase 3 placebo-controlled vaccine efficacy portion of the study enrolled over 16,000 participants. Evaluation of vaccine efficacy demonstrated that the study met its primary endpoint of prevention of virologically confirmed COVID-19 disease. Data show that in an analysis of COVID-19 cases accrued between seven days and 56 days after completion of a two-dose vaccination series, two five-microgram doses of ARCT154 demonstrated 55% vaccine efficacy for preventing symptomatic COVID-19. Key secondary endpoint evaluating severe COVID-19 disease including COVID-19 related deaths was analyzed and demonstrated over 95% efficacy for prevention of severe COVID including deaths. Notably, this study was conducted during a period characterized by the dominance of the Delta and Omicron variants. We are currently conducting additional analyses of the swabs taken from the COVID cases in this study to characterize which SARS-CoV strains led to infections in this study, and particularly to confirm if there's further evidence of protection against these two very challenging variants of concern that have escaped protection from many other vaccines. The data from the study are also reassuring from the safety and tolerability perspective. The incidence of unsolicited adverse events in the ARCT154 and placebo group are comparable in the safety data collected from over 17,000 participants enrolled in the Phase 1, 2, and 3 through one month post-fold vaccination. In addition, the frequency and severity of those events was generally consistent upon the second administration. No cases of myocarditis or pericarditis have been reported. Although the study size was not large enough to detect large numbers of these rare events, the absence of such events in a study of this size is still comforting. We and our partners at VinBioCare will continue to carefully monitor the safety of participants in this ongoing study. The study met its primary objectives in both the demonstration of acceptable immunogenicity and safety in the Phase 1-2-3a readout and in meeting the protocol-specified vaccine efficacy endpoint in Phase 3b. These results have been submitted by VinBiocare to the Vietnam Ministry of Health for the express purpose of seeking emergency use authorization. We also continue to work with VinBiocare to support filing for full marketing authorization to be completed later this year. We are very encouraged by the data from this Phase 1-2-3 study. With two low-dose administrations of the ARCT154 vaccine, effective prevention of COVID disease and particularly severe and fatal disease has been shown. The safety profile was well-tolerated, and we view these findings as an important illustration of the potential of our self-amplifying mRNA platform, which includes the STAR technology, and STAR is the trademark for self-transcribing and replicating RNA technology, and the Lunar Delivery Platform, our trade secret know-how and proprietary manufacturing processes, including purification formulation and lyophilization methods toward the mRNA drug substance and the vaccine drug product. Given the continued circulation of SARS-CoV-2 and the discussion of its movement into an endemic phase, we are moving forward with development of ARCT154 as a booster vaccine. In an ongoing Phase 1-2 study evaluating ARCT154 given as a 5-microgram booster dose approximately five months after primary vaccination with Comirnaty, the ARCT154 vaccine responses are promising. The acceptable reactogenicity accompanied by robust immune responses continue to build our enthusiasm toward next steps. We've observed noteworthy neutralizing antibody responses soon after vaccination with ARCT154 and now have shown the high GMFRs to be sustained through three months after vaccination. In addition, the neutralizing antibody responses to the parent strain of the vaccine, also known as D614G, and antibody responses against a panel of SARS-CoV-2 variant strains have demonstrated antibody responses that have remained 13 to 30 fold elevated over three months, after three months, 91 days to be exact, over day one baselines for all SARS-CoV-2 strains tested on the panel. We're even further encouraged by additional data from this study demonstrating strong neutralizing antibody activity against the Omicron BA.2 variant upon boosting with ARCT154 with a 46-fold increase seen in NAB activity at day 29 over day 1 baseline. This data, paired with the 54-fold increase in neutralizing antibodies against BA1 over the 29-day window that we reported earlier this year, generates an appealing profile for ARCT154 as a broadly immunogenic and next-generation low-dose booster. Given these collective data, we have been in discussions with major regulatory agencies, including the FDA, the MHRA and EMA, regarding the path to pivotal trial and future approval as a booster. These conversations have informed the design elements of our pivotal booster trial intended to support global registration. In parallel with our development activities, our global manufacturing footprint continues to advance. with ongoing technology transfer activities to VinBiocare's manufacturing facility, and we expect it to become operational this year with a capacity to manufacture 200 million doses annually. The development of the Hanoi manufacturing facility is fully sponsored and funded by VinBiocare, and we are looking forward to the next steps in our collaboration to potentially add Hanoi as an international hub for manufacturing self-amplifying mRNA medicines. Our self-amplifying mRNA influenza vaccine program has continued its preclinical work, and we expect to make a final decision of our lunar flu development candidate this year. Given the comparative speed of mRNA vaccine production and precise antigenic matching against circulating influenza strains, this is expected to offer an important improvement over currently marketed influenza vaccines. With its comparatively lower dose, than other mRNA vaccines in development. We hope to understand if there are other meaningful ways to differentiate in the influenza vaccine space. Well, now I'd like to shift from our vaccine franchise over to our therapeutic mRNA pipeline programs. Indeed, we also continue to make progress in our mRNA therapeutic programs. I'll begin with an update on ARCT 810. our therapeutic candidate for Ornithine Transcarbamylase Deficiency or OTC deficiency. OTC is the number one urea cycle disorder. It's a rare and serious disease with no approved treatments that address the root cause of the disease. Our approach aims to augment the deficient or absent OTC enzyme in the liver of patients living with this disease. ARCT 810 has the potential to restore urea cycle activity preventing or slowing the progression of neurological damage and potentially expanding dietary options and improving on quality of life for people living with this condition. Well, we've obtained approval from the United Kingdom's HRA or Health Research Authority and other European authorities to initiate a phase two multiple dose clinical trial for ARCT810 designed to enroll 24 adolescents and adults. Patient screening and enrollment is underway at multiple sites in the United Kingdom, Spain, and Belgium, with other countries soon to follow. We anticipate that phase two enrollment will commence before the end of the second quarter, and we expect to obtain interim proof of concept data in the second half of 2022 from a subset of participants. We've also completed dosing of the first cohort of our ascending dose phase 1B study here in the U.S., wherein the participants received a dose of 0.2 milligrams per kilogram of ARCT A10. We're pleased also to report that the Data Safety Review Committee has recommended continuation of this study without modification. Therefore, the dosing of the screened second cohort will begin imminently. Moving briefly now to our Cystic Fibrosis Program. We've continued to progress the necessary preclinical studies to enable ARCT 032 This is our inhaled messenger RNA therapeutic candidate for cystic fibrosis to move into clinical studies, and we anticipate the submission of a clinical trial application, or CTA, for ARCT 032 in the third quarter of 2022. In addition to our internally developed pipeline programs, Arcturus continues to support our partnered or licensed therapeutic programs. The most advanced of these is a very promising therapeutic candidate for glycogen storage disease, which continues to be evaluated by Ultragenyx in a Phase 1-2 study. Well, with that, I will now pass the call on to Andy Sassine, our CFO, to provide the financial updates.

Thank you, Joe, and good afternoon, everyone. Press release issued earlier today includes financial statements for the first quarter of 2022 and provides a summary and analysis of sequential financial performance. Please reference our 10Q for more details on the financial performance. I will go over our financial and present some operating metrics as we continue to transition to a late stage clinical company with several assets in our pipeline. I will also provide some details regarding our manufacturing strategy as we prepare for the potential of emergency use authorization in Vietnam. Finally, I will provide some insights regarding our cash position and expected run rate. As you heard Joe mention, we had a very productive first quarter, including highly encouraging efficacy trial and boosted durability results for ARCT 154. As you know, we've partnered our ARCT 154 Next Generation Lunar COVID-19 Vaccine Candidate in Vietnam with VIN BioCare. VIN BioCare is part of the VIN Group, which is one of Vietnam's largest corporations, and is sponsoring and funding our Phase 1, 2, 3 study in Vietnam targeting COVID-19 and the variants of concern. This partnership includes the trial and collaboration around building a vaccine manufacturing facility in Hanoi resulting in significant cost savings for our tourists. Our technology transfer activities remain on track for the facility to become operational later this year, with a capacity of over 200 million doses annually. We are also continuing to work with other manufacturing partners to mature our global footprint, including Europe, Japan, and the United States of America. Revenues from strategic alliances and collaboration for the first quarter of 2022 was $5.2 million, which was relatively consistent with the December quarter. Our operating expenses for the first quarter were $55.6 million, which declined quarter over quarter, an increase from the quarter ended December 2021. This increase was primarily due to one-time manufacturing and CMC quality runs for our 154 vaccine program, and an increase in costs associated with the startup activities towards initiation of the booster trial. In January 2022, the company entered into an agreement with a pharmaceutical company, whereby the pharmaceutical company agreed to fund up to $25 million for a clinical trial for lunar COVID-19 vaccine candidate as a booster. Net loss for the first quarter of 2022 was approximately $51.2 million or $1.94 per share, basic and diluted share. We reported a net loss of approximately $56.3 million or $2.15 per basic and diluted share in the first quarter of 2021 and a loss of $38.7 million or $1.47 per basic and diluted share in the first quarter of 2021. Our cash balance at the end of the first quarter was $319.7 million, and based on our current pipeline, our cash position is expected to be sufficient to support operations into late 2023. I will now pass the call back to Joe.

Hey, thanks, Andy. We're definitely pleased to have another productive quarter, advancing our pipeline of mRNA vaccines and therapeutics, in particular the recent clinical data, ARCT154, that helps validate our self-amplifying mRNA technology platform, our lunar lipid nanoparticle delivery platform, and our proprietary trade secret manufacturing processes, including lyophilization. So I'll now turn the time over back to you, Doug, our operator, to field any questions.

Thank you. If you'd like to ask a question, please signal by pressing star 1 on your telephone keypad. If you're using a speakerphone, please make sure your mute function is turned off to allow us to not reach our equipment. Again, press star 1 to ask a question. And we'll take our first question today from Yasmine Rahimi with Piper Sandler.

Good afternoon, team, and thank you for taking my questions. A couple for you. Maybe the first one is I know you made in your prepared remarks, you noted that you're going to be analyzing in the primary efficacy data. the distribution of how many of the cases were due to Omicron and Delta. Just to kind of give us an idea out of the top line efficacy event rate, those two data points, how soon can we expect that? Like, do you think we could, how long does the process take and in which format would you share it with us? That's one. And then the second thing is, have you gotten any communication back from Vietnam in the last two and a half weeks after the primary advocacy shared with them. And then I have a last follow-up.

Sure. Thanks, Yas, for joining the call. With respect to the swab data and Delta and Omicron, this data will be collected over a matter of weeks. It's not several months. So we aim to get that part of the puzzle completed soon. but we did provide references for those that read our press release and the previous one as well that provides some guidance as to the prevalence of Delta and Omicron during the period of the study, and we encourage people to refer to that in the meantime while we collect the hard data for that. The second question, I'll turn the time over to Pat to address that.

I think your question was about the EUA and what's sort of the status in Vietnam. So I think the review process is still ongoing, but we can share that over the last few weeks, the clinical section, which is one of the key parts of the EUA process, including the safety, the immunogenicity, and the efficacy data, was reviewed by the National Committee of Ethics. and during the meeting it was concluded and it was given positive feedback. So we're encouraged by that. The larger application is still under review and we hope to hear back from them very shortly.

Thank you. And then my last question is in regards to the booster study that you'll be kicking off in 2400 at all. I just want to confirm just, you know, In terms to receive approvability here in the U.S., is this it? Is it just the execution of the booster study in conjunction to the large study that was done in Vietnam to support full approvability here in the U.S. and in Europe? So if you could just kind of reconfirm if there are any other rate-limiting steps left, that could be helpful. And thank you again for taking my questions.

No, I appreciate the question. So we have met with the FDA and consulted with them and received their feedback pertaining to the design elements of this pivotal booster trial with respect to the appropriate size of the trial and to what degree our data collected in Vietnam will be accepted or supportive. And after that feedback, not just from the FDA, but the MHRA and the EMEA, that we have you know, felt comfortable to proceed with the pivotal trial. So that's all we've given specific guidance to. But, you know, we've previously said approximately 2,400 people will be participating in the trial. So this is not an extraordinarily large trial. And clearly that's not going to be an efficacy-based trial. Like you'd see, there's no more, you know, placebo-controlled randomized vaccine efficacy trials and stuff like that. So I can confirm that we've received feedback from these major market regulatory agencies and we're proceeding accordingly.

Okay, thank you team. Thank you, yes. Our next question will come from Seamus Fernandez with Guggenheim.

Hi, this is Evan Wang on for Seamus. I have two questions. First on the booster, Any more details you can share in terms of trial design? I know you guys have shared that's 2,400 patients, but in terms of, you know, if it's a head-to-head, whether it's non-inferiority or superiority, any additional color would be appreciated. And it's still on timelines for the trial. The second question is, you know, I know you shared some durability data in terms of the booster for COVID. non-Omicron variants, but any timelines in terms of when we can see derivative data for the BA.1 and BA.2 variants? Thank you.

Sure. Okay. So, additional color on the trial. Of course, we have a tight understanding of the design of our trial, but we have to remain not just coy, but strategically silent on key elements of this. We realize that the endemic booster market is going to be a significant commercial opportunity with competitors, large pharmaceutical competitors. So we want to make sure we hold those cards close to our chest. And because we're first movers in the self-amplifying mRNA space with anticipated competitors in the coming years, we want to keep those cards close to our chest as well. What we feel is important to disclose to our investors is that this is not going to be an astronomically large trial or something that's difficult or time-consuming. With respect to the time, we remind people this is a single administration-type booster vaccine trial. It's not a two-shot trial. And recruitment times are anticipated to be much faster because the population of people that have already been administered vaccines is plentiful. So we're not looking for naive folks or people that have not been exposed to the virus. We're simply looking for people that have already been vaccinated. So we believe recruitment times will be reasonable or much faster than our first experience. And with respect to the other design elements, we're just holding those cards close to our chest, if that makes sense, strategically. With respect to the BA1 and BA2 durability, We've shown 29-day data, so that kind of gives you an idea that we'll be able to subsequently show durability data for those Omicron variants as well. That, again, won't be several months, but it may be some weeks here ahead of time. But I refer everyone on the call to the relatively substantial amount of variant data that we've already provided against Alpha, Beta, Delta, Gamma and other variants of interest And very consistently, this vaccine is responsive and durable. And there's something special about self-amplifying mRNA. We're going to continue to learn about it. We just wanted to share three-month data at this point. But we're cautiously optimistic that this durability will continue. And that's where we are. But wait some additional.

It won't be several months. Thank you. Thank you. We'll now hear from Nick Abbott with Wells Fargo.

Oh, good afternoon. Thanks for taking my question. You know, first one, Joe, when I look at the GMT chart here in the press release, you know, you have one patient roughly a two-fold increase. Perhaps it's the same patient, a three-fold increase over BA1 and BA2, and obviously that's over the mean for day one so you know what was the that those individuals increase over their own day one values and if it's sort of substantially less than this you know 40 to 50 fold range you know have you looked at you know what what is it about this particular individual that means they have a poor you know apparently possibly a poor response

Right. Now, we don't want to disclose details for specific individuals, but I think this is an appropriate question to remind that a significant, a large percentage of our participants in our trials were high-risk individuals, whether that's due to elderly or comorbidities, you know, immune-suppressed individuals, right? So considering that a significant percentage was... at-risk individuals. It's likely one of those that gave a lower response than others, but we've provided all the data. If you look at the mean, clearly it's highly encouraging.

Agreed. And then you mentioned this expression of interest letter to WHO. Could you elaborate on that, Joe? What What does it mean? What is the timeline? You know, what happens if they say yes?

Oh, yeah. Well, the WHO, of course, is a charitable organization at first. So, you know, one of our intent here is to help in any way we can with primary vaccination. I know in the U.S. and Europe, the majority of people have been vaccinated. But in the developing nations, this is still a core issue for the WHO. And we want to see if there's potential here for us to help there. I think it's also helpful to remind people that I don't know the exact number of countries involved in the WHO emergency use listing, but it is a large number of developing nations. So if we do successfully capture a spot on this EUL or this emergency use list, then it'll open up opportunities for not just helping in the near term, but commercial opportunities down the road. So that's about it. You know, with respect to the timeline of going through the process, we just wanted to make people aware that, you know, Vietnam Ministry of Health and the WHO have been engaged and have been, you know, sent a letter and have been supportive of this process. And we'll give updates as they arise. But we haven't given any tight guidance on a timeline to an official spot on the emergency use list.

Great. Thanks, Ed. Igal Nochemovitz with Citigroup has our next question.

Hi, great. Thanks, John and Andy, for taking the questions. Just curious on what regulatory work you've done with regulatory consultants or other outside experts to clarify whether FDA would be amenable to approving a fourth vaccine only as a booster, given obviously that's not the paradigm that we've seen for Moderna, Pfizer, and J&J. Thank you.

Right. Yeah, the only thing that we've learned is with respect to vaccines, another approved under emergency use, I think we'd have to see another wave. Like some biostatisticians are predicting a wave of COVID coming this fall. If that indeed happens, then that likelihood of garnering emergency use authorization would be higher. But I think the expectation is to go through a traditional BLA process in the US to capture a traditional booster. And the reason, the justification for that is simply this is a lower dose, longer lasting potentially option. So this is a different mechanism of action of expressing the antigen and it could be beneficial to subpopulations here in the US. So we believe that regulatory agencies will be respectful of that, of these key differences of allowing their citizens to have access to a lower dose next generation mRNA vaccine.

Got it. And have you had discussions with the FDA regarding the acceptability of the Vietnam 20,000 patient trial for your application? What does it stand with that?

Well, we haven't given a tight, detailed response to that question. It's a good question, but we have asked this question to these regulatory agencies, and they have provided their feedback and have provided a path forward for the Vietnamese data to be supportive. The details as to what's specifically required for this data to be supportive has not been shared. However, we felt comfortable enough for us to proceed with this global registrational study based upon the feedback we've received.

And for the global registrational study for the booster, you vetted that design with FDA, correct?

Correct.

In detail, that's correct. Okay. Thank you very much. Next, we'll hear from Kumar Raja.

Next, we'll hear from Kumar Raja with Brookline Capital Markets.

Thanks for taking my questions. One more on the booster trial. So some patients, as of now in the U.S., some have received two, some have received three doses, as well as some have received four doses. You have data from patients who have completed two doses. So how are you thinking about including patients who have received three or four doses in the trial? And also with regard to the data, what are you thinking in terms of timeline to follow that?

Sure. So I can refer, you know, those on the call to, you know, there was a white paper for some guidance provided. by regulatory agencies with respect to booster trials? Can they include people that have received two shots, three shots? And the answer is generally yes. But I cannot speak on behalf of the FDA or other regulatory agencies specifically to ARCT 154. But there has been general guidance provided that booster trials can recruit not just people that have received two shots, but people that have received three shots. So that allows us flexibility and and benefits from a recruitment perspective, definitely.

Okay, and in terms of the timeline, how long do you think you will need to track these patients? And also, your thoughts on including older, high-risk patients in the trials.

Correct, correct. We received guidance on all of these details from the regulatory agencies. They were fairly consistent. I can speak to that. with respect to the percentage of the elderly and how long we need to track these folks for. Those design elements are implemented into our booster trial design and it has definitely taken into consideration the feedback from the regulatory agencies that we asked that specific question to.

I just needed a clarification on something Andy mentioned. So you have received $25 million from a pharmaceutical company for the booster trial. Did I hear that right?

Yeah, we haven't gotten the $25 million. We've gotten an agreement to reimburse us up to $25 million of our Phase III booster trial. So hopefully that clarifies it for you.

Okay, and what do you guys need to do in return for that?

We need to obviously spend money on the trial and bill our pharmaceutical company partner and hopefully get reimbursed in a reasonable timeframe.

Okay. Thanks so much. Our next question will come from Steven Seedhouse with Wayman James.

Great. Thanks for taking the question. I wanted to ask about ARCT810, the Phase 2 study that's getting underway. What, in your view, would constitute proof of concept emerging from the data in that study? And then on 154 booster study, I wanted to ask if you settled on a dose for that study, if it's the same dose you've been contemplating, or if you think about increasing the dose to squeeze out more efficacy potentially. Thanks.

Yeah, at this point, five micrograms is our dose. We'll continue to monitor that dose level in the booster environment. But right now, we believe that we'll be keeping the dose at five micrograms. With respect to biological proof of concept for the A10 program, there's multiple biomarkers associated with ornithine transcarb amylase deficiency, as you're well aware. including ammonia in the blood or the plasma and erotic acid in the urine, and the urea cycle itself and ureogenesis, and also levels of OTC itself. So a lot of biomarkers associated with this disease. So we're defining proof of concept as biological proof of concept. So if any or all of these biomarkers are impacted by OTC, ARCT 810 treatment, then we would determine that as biological proof of concept.

Thank you.

Thanks, Steve. Our final question will come from Ed Arcee with HC William Wright.

Hello, everyone. This is Thomas. You're asking a couple questions for Ed. Hi, Joe. This is Thomas. Hi. You mentioned earlier that the Vietnam Ministry of Health is reviewing the data that's been submitted, which includes Phase 1, 2, and 3A. Can you remind us what is their review process regarding a timeframe? Do they adhere to a specific guideline that they hope to render a response regarding the EUA?

No specific timeline was given, but all those data that we've referred to in this review today have been provided, and they're working hard and diligently at reviewing it. I forget the exact number of papers, but this was an extraordinary amount of data. Usually, regulatory agencies review subsections of data over, you know, a decade of time, and this is 38 weeks of data involving 19,000 participants. So we do recognize the amount of data that needs to be reviewed in this process, but they're working as fast as they can. You know, we've guided, you know, the decision as soon. But, Pad, anything to add?

No, and I think, you know, we've been clear about the timing for that. We'll have some... clear guidance on that by end of the quarter.

Got it. And then regarding the data publication that is to be in the future from how we understand it is driven by VinBioCare. Can you talk about when should we expect the data package to be published, and what about Phase 3b data? When can we expect that?

Oh, the publication of the data? Well, the data is intimately associated with VinBioCare, our partner as well. But yes, they understand, we understand. the expectation is that at some point here we're going to be publishing this data for sure. So at least that's our expectation. But we haven't given any guidance on timing of that data. But, Pat, anything else to add?

Again, we and WinBio can understand the importance of sharing the data that we've generated and the positive nature of the data. So I think we will provide more details around some of the efficacy aspects analysis as well as further analysis of some of the subgroups as well.

And I know our teams are actively drafting these publications right now, but you're asking the specific timing of the publication of those and we just haven't provided that specific guidance yet.

Got it. Thank you so much for the kind of questions and we're looking forward to the publication.

Yeah. Thank you.

That will conclude today's question and answer session, and I'll turn the conference over to Mr. Payne for any additional closing remarks.

Thanks to everyone that joined the call. If we didn't get a chance to address your question, feel free to reach out to us directly. We'll be in touch, guys. Thanks, everyone. Bye-bye.

That will conclude today's conference call. Thank you for your participation. You may now disconnect.