Arcturus Therapeutics Holdings Inc (ARCT) 2025 Q3 法說會逐字稿

Good afternoon, everyone. Welcome to the Arcturus Therapeutics third-quarter 2025 earnings call. (Operator Instructions) Also, today's call is being recorded, (Operator Instructions)

Now, at this time, I'd like to turn things over to Neda Safarzadeh, Vice President, Head of Investor Relations, Public Relations, and Marketing. Please go ahead, ma'am.

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, CSO and COO, will join them 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 in 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. I will begin today with an update on our ARCT-032 program. This is our messenger RNA therapeutic candidate for cystic fibrosis, or CF. ARCT-032 utilizes Arcturus's LUNAR lipid-mediated aerosolized platform to deliver CFTR messenger RNA to the lungs. Expression of a functional copy of the CFTR mRNA in the lungs of people with CF has the potential to restore CFTR activity and mitigate the downstream effects that cause progressive lung disease.

In October, the company announced interim data from its ongoing Phase 2 clinical trial of ARCT-032. Treatment with inhaled 10-milligram doses daily over 28 days in six Class I CF adults was generally safe and well tolerated. A protocol prespecified analysis of high-resolution computed tomography lung scans, or HRCT lung scans, using FDA 501(k)-cleared AI technology revealed reductions in mucus burden in 4 of the 6 Class I CF participants in our second cohort.

The ongoing third cohort is enrolling up to six subjects to assess the safety and tolerability of the 15-milligram dose daily over 28 days and the impact on the efficacy endpoints. The company intends to evaluate daily dosing of ARCT-032 over a 12-week duration in up to 20 CF participants. Safety and preliminary efficacy data will be collected in this study, which is planned to begin in the first half of 2026 after the third cohort top-line data is understood.

Two weeks ago, I, along with our team, had the privilege of attending the North American Cystic Fibrosis Conference in Seattle. It was great to meet with the CF Foundation leadership team and share our enthusiasm for the Class I population based on our encouraging data. I met with the physicians and principal investigators involved in our ongoing clinical trials and was very pleased to hear their anecdotes, positivity, and encouragement. I enjoy meeting with multiple CT scan experts and and felt their passion as they described the present and future importance of HRCT imaging data in lung disease trials.

I affirmed my appreciation of the significant unmet medical need represented by Class I CF and other CFTR modulator nonresponders here in the United States. There's an even higher prevalence of people with Class I CF in countries outside the US especially in Europe, India, the Middle East, and Israel. All in all, the conversations with the CF Foundation, people with Class I CF, their physicians, investigators, CT scan experts, and global CF representatives, all reinforced Arcturus' commitment to advance ARCT-032 further into development.

The safety and tolerability profile data along with the before and after treatment HRCT scan images showing mucus plug reduction were well received by the CF community. We look forward to collecting additional and potentially meaningful clinical data in 2026 for our CF program.

Moving on to the ARCT-810 program. This is our messenger RNA therapeutic candidate for ornithine transcarbamylase deficiency or OTC deficiency. With positive interim Phase 2 data in hand, the company is diligently preparing for meetings with regulatory agencies in the first half of 2026 to discuss pivotal trial strategy for both pediatric and adult populations. Understanding what the FDA requires for ARCT-810's path to approval is the next key milestone for this program. We aim to provide more details pertaining to these regulatory alignment meetings in the first half of 2026.

I will now provide regulatory updates for our partnered COVID-19 vaccine program, also known as KOSTAIVE. Our Japanese partner, Meiji Seika Pharma, had launched the two-dose vial of KOSTAIVE updated for the JN.1 variant XEC in Japan. This is the first time the two-dose vial presentation is being distributed in Japan. Meiji received approval from the Pharmaceuticals and Medical Devices Agency or PMDA in August.

Also in August, the company published the Phase 3 manuscript on the immunogenicity and safety of our self-amplifying mRNA COVID-19 vaccine, ARCT-2303. The study shows that ARCT-2303 induces a robust immune response against SARS-CoV-2 and can be co-administered with licensed influenza vaccines in adults with no impact on safety or immunogenicity of either vaccine. The results were published in eClinical medicine.

Moving on to ARCT-2304. This is our next-gen star vaccine candidate for pandemic A/H5N1 influenza virus. This is the program contracted with and funded in part by BARDA. We conducted a Phase 1 study in 132 young adults and 80 older adults. ARCT-2304 induced a humoral immune response after a single dose in all tested dose levels. The administration of a second dose of ARCT-2304 further increased immune responses.

ARCT-2304 at dose levels of 1.5, 5, and 12 micrograms induced a hemoglutinin-specific immune response similar to or higher than the MF59-adjuvanted pandemic vaccine in both young and older adults. No safety or tolerability concerns were raised from available data. These data further validate our STARR sa-mRNA platform. The study results support the further development of the self-amplifying mRNA pandemic influenza vaccine candidate.

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 third quarter of 2025 and provides a summary and analysis of year-over-year performance. Please also reference our most recent Form 10-Q for more details on the financial performance.

The KOSTAIVE BLA filing had been delayed indefinitely. Due to the sudden regulatory changes by the FDA, combined with uncertain commercial visibility of KOSTAIVE in the United States, we have decided to reduce additional expenses to extend the runway for the Cystic Fibrosis and OTC programs. The company expects continued support from CSL to commercialize KOSTAIVE in Asia and Europe and will provide additional detail on our year-end call in March.

Revenues for the three and nine months ended September 30, 2025, was $17.2 million and $74.8 million, respectively, representing a decrease of $24.5 million and $54.7 million compared to the same period in 2024. These declines were primarily driven by reduced revenues from the CSL collaboration, reflecting lower supply agreement activity and lower amortization of the upfront payment as KOSTAIVE became a commercial product. Total operating expenses for the three months ended September 30, 2025 were $33.7 million compared with $52.4 million for the three months ended September 30, 2024. Total operating expenses for the nine months ended September 30, 2025 were $119.8 million compared with $191.8 million in the prior year.

R&D expenses were $23.3 million for the three months ended September 30, 2025 compared with $39.1 million in the prior year. The decrease was primarily driven by lower manufacturing cost for the COVID, flu, and CF program as well as reduced clinical trial expenses for COVID and Cystic Fibrosis. Lower payroll and employee benefits further contributed to the decrease. R&D expenses were $87.7 million for the nine months ended September 30, 2025, compared with $151.4 million in the prior year. The decrease was primarily driven by lower manufacturing and clinical costs related to the COVID program, reflecting the program's transition from a development program to the commercial phase.

Additional decreases were attributable to lower manufacturing costs for the Cystic Fibrosis and flu program. These reductions were partially offset by higher clinical costs for Phase 2 of the Cystic Fibrosis program. Payroll and benefits expenses also decreased primarily due to lower stock-based compensation expense. G&A expenses were $10.4 million and $32.1 million for the three and nine months ended September 30, 2025, compared with $13.3 million and $40.4 million in the comparable period last year.

The decrease in both periods were primarily due to reduced share-based compensation expense as well as reduced payroll and benefits. We expect general and administrative expenses to continue to decrease slightly in fiscal year '26.

For the three months ended September 30, 2025, Arcturus reported a net loss of approximately $13.5 million or $0.49 per diluted share, compared with a net loss of $6.9 million or $0.26 per diluted share in the three months ended September 30, 2024. Cash, cash equivalents, and restricted cash were $237.3 million as of September 30, 2025 and $293.9 million on December 31, 2024. Based on the additional planned cost reductions in Q4 and the delay in the Phase 3 Cystic Fibrosis clinical trial commencement, the cash runway remained extended into 2028. More details regarding our cost reduction and runway will be provided on our year-end call in March.

In summary, the company remains in a strong financial position and has the cash runway needed to achieve multiple near-term value-creating milestones for both therapeutic programs.

I will now pass the call back to Joe.

Thanks, Andy. Arcturus continues to make progress across our mRNA therapeutics and vaccines pipeline. We look forward to initiating the planned 12-week CF study for ARCT-032 in the first half of 2026. And engaging regulatory agencies regarding the pivotal trial designs for ARCT-810.

With that, let's turn the time over to the operator for questions.

(Operator Instructions) Yasmeen Rahimi, Piper Sandler.

I guess the first question is, given this data, have you been able to do some PK/PD modeling to help us understand the expectations as you are initiating the third dose cohort and what you hope to gain and how we should be thinking about that? That's question one.

And then question two is, as you are preparing for the meeting with the agency to discuss your OTC pivotal programs, what are some of the optionalities of base case, best case, both in development for the pediatric population as well as development in the adult population. And thank you so much, and I'll jump back in the queue.

With respect to PK/PD modeling, well, the third cohort, which is being evaluated at a dose level of 15 milligrams is being conducted in a very similar manner to the first two cohorts at 5 and 10 milligrams, respectively. So all of the activities with respect to data collection are going to be in line with the first two cohorts. With respect to the fourth cohort or I guess you would say this planned 12-week safety and preliminary efficacy study. This is an expanded study.

So in addition from extending the duration of the study from 4 weeks to 12 weeks, we're also increasing the population up to 20, but a large amount of the data that's being collected is very similar to what we're doing in the first three cohorts. There is some noted differences that we're intending to conduct this trial under. First of all, we're going to be adding an extra screening visit which to establish a more stable FEV1 baseline.

We're also going to be, of course, looking at the high-res CT scan before the study, but at 12 weeks this time instead of at 4 weeks. So we'll allow those two additional months to occur before we take an imaging scan. We're also going to be looking at adding questions to the questionnaire that there's what's called an EQ-5D-5L general health questionnaire, where we're going to be looking at mobility and self-care and usual activities, pain and discomfort, anxiety depression. We're going to be adding this general health questionnaire to the standard validated CFQR questionnaire that people are familiar with CF, but with respect to PK and PD markers, it would be very similar to the first three cohorts.

With respect to your second question, you were asking about OTC, we're looking at two separate populations, the adult population and then the more severe disease in children. And these will likely are requiring two separate conversations with regulatory agencies to gain alignment on a pivotal study. The adults will likely be involving glutamine as a biomarker because that's where we captured success already, and we've learned -- we've collected some positive data already in our trials to date with respect to glutamine in adults. With respect to children that are suffering of more severe disease, the focus will be more on pneumonia itself and getting alignment with the FDA on that, and can we do a single-arm study, for example, in children to capture approval? But these sorts of conversations are separate and distinct enough to have separate meetings to address them as our expectation.

The best case scenario, of course, is that we gain alignment with both adults and pediatrics, and that would be very exciting for this program to have line of sight in a broader population to get this approved as soon as possible. And any different scenarios would be if one of these were approved to proceed, for example. But anyway, thanks for your question.

Myles Minter, William Blair.

This is Jake on for Myles. Just reflecting back on the imaging data you showed for CF. Do you expect that the improvements you see in mucus over time, especially in that 20-week study, will be bronchial- or alveolar-specific as you sort of showed in that initial dataset? And is that what you saw in the mucus burden from the ferret model preclinically?

And then just wanted to also check in on cost saves and see if you've updated your guidance as to when you're going to start realizing revenue from that program?

Okay. So the first question is with respect to mucus plug reduction, one of the key observations that we've observed and is familiar with the field is mucus plugs form in the smaller airways, right? So as you resolve that, you measure smaller changes in airway improvement. FEV on the other hand is mainly a measure of larger airways. So they're complementary, but they do not measure the same thing. And given enough time, we believe that both of these will improve. And so that's one of the purposes of the 12-week study.

Before I move on to the KOSTAIVE commercial question, did I address your question, Jake?

I guess I wanted to know whether you expect those mucus reductions to occur across the entirety of the lung or whether a specific bronchial or specific alveoli are going to be resolved given that you sort of boxed specific bronchials in your presentation, denoting that because potentially LNPs are directed there primarily that that's where you're going to see an effect. I just wanted to know that. And whether you also saw sort of bronchial specific reductions in the lungs of the ferrets when you dosed those.

No. Great question. So first of all, with respect to the data that we collected in the images, you do see in the lower register, the lower lobes that they are first to resolve and show a reduction of mucus plugs and that's simply because this is an inhaled therapeutic. We've talked to now several pulmonologists that view this as confirmational that that we see, first, the lower register, the lower lobes being addressed simply because this is an inhaled therapeutic. We expect over time that the effect will continue to improve. And that's one of the primary purposes of the 12-week study is through an extended duration that will continue to address not only the lower lobes, but the upper lobes as well.

With respect to your question about ferrets, this is a different lung type entirely. They're not a vertical animal. So gravity is not -- and this was an injected process. This wasn't a traditional inhaled therapeutic like humans experience, so we didn't expect to see a similar dataset, and we didn't do CT scans in these ferrets as well. We analyze the data separately through mucociliary clearance.

Now with respect to the second question on KOSTAIVE guidance. Andy, do you want to provide maybe an answer there?

Yeah. Thank you for the question. Typically, we do not provide guidance with respect to KOSTAIVE commercial revenue. And the last comment that came in press release that came from Meiji, they did order about 1 million doses for the fourth quarter, and that was delivered to them in October, November. So they're in the process of selling those doses in Japan.

We don't really have an update subsequent to that, but probably look for an update sometime at the end of the year call in next March. Hope that helps.

Seamus Fernandez, Guggenheim.

This is Evan Lang on for Seamus. Just two questions on Cystic Fibrosis with the upcoming 15 mg dose. Can you just talk about the metrics that will drive to know your decision reflect 10 or 15 mg in the subsequent Phase 2, whether it's FEV1, CT scan, or both and what specifically you may be looking for? And then with the subsequent 12-week study, curious what you define as success then with longer treatment in terms of either FEV1 or high-res CT as you think about data relative to the interim value shown so far?

Yeah. With respect to the 15-milligram cohort, we want to gain additional confidence in the dose response. We did not see any mucus plug reduction at 5 milligrams, yet we saw four out of fix in the second cohort at 10 milligrams exhibit mucus plug reduction. So one of the things we're looking for at 15 milligrams is, is there a continued or elevated response? And is that a dose response? But the most important dataset that we're collecting from this third cohort is really safety and tolerability. If it's well tolerated, then I think we have our dose that we will select for this 12-week study coming up in the first half of next year.

With respect to what we would define as success is if we see continued or further reduction of mucus plugs, and that translates into additional benefits that can be either imaged or or experienced in terms of lung function improvements, and that would be fantastic, given that this is a first-in-line therapy for a considerable unmet medical need in Class I subjects and modulator nonresponders. So anything positive for FEV would be viewed positively if we see an improvement with the extended duration or elevated dosing in this 12-week study with respect to the mucus plug reduction and mucus burden being decreased, that would be also very promising to encourage the Board and our company to advance this into a Phase 3 trial.

And if I could ask one follow-up. Just curious in terms of the CT scan and mucus plugs, what commissions you as clinically meaningful? And what in terms of some of the regulatory discussions you've had, especially as an approach to include CT scan as an exploratory end point, what they might view as a potentially approvable endpoint or whether focus would be on FEV1?

Well, we'll be the first company in CF to establish that. That's one of the key tasks at hand here as a group. As we share this data with the FDA, we need to determine what's clinically meaningful. What we know now is that the more optimized mature modulators out there after a year of treatment, you can see near-complete resolution of mucus plugs. And at an interim time point, you'll see not a complete resolution of mucus plugs.

Unfortunately, we're the first -- well, unfortunately, we're the first company in therapeutic to evaluate CT scan measurements after only 28 days of treatment. So the fact that we saw some of these subjects responding 30%, 40% mucus reduction after just 28 days is encouraging. But the question you asked is what is meaningful?

I think we're already in that phase of a meaningful reduction. We just now need to extend treatment to see if that translates into other benefits and lung function improvements over an extended term. But the specific number, we're not prepared to share right now, no one is. That is something that we can discuss at a later time with the agency.

Whitney Ijem, Canaccord.

This is Angela on for Whitney. So you're planning to start this 12-week study starting in the first half of next year. Any idea when we should expect to see data from the 15-milligram cohort? And any thoughts on what endpoints you would show for the 15 milligrams?

Well, for the 15-milligram cohort, if you're referring to the third cohort, that's the 28-day study. It's the same data being collected under the same protocol for the first two cohorts. And that data is expected likely in the first quarter of next year. And as soon as that top-line data is understood, we will be able to quickly then transition to the 12-week study. And focus on that.

There -- but the parameters and the efficacy endpoints and safety and tolerability investigations are all identical to what we did for the first two cohorts for the 15-milligram third cohort. Did I address --

Yeah. Maybe just one quick follow-up. Any chance you would show the analysis of the CT scans for patients who might not have responded and seen the decrease in mucus plugs? Or do you expect it to be similar?

Well, there's going to be a time for us to share the complete data package for the first three cohorts, right? We do have a 5-, 10- and 15-milligram cohort, I'm sure that will be a nice presentation or publication at some point. We haven't determined exactly when, but that would be an appropriate time to to just share all the data we've collected to determine if there's a dose response and provide those details.

I've already shared on this call already that at 5 milligrams, we did not see any mucus plugs reduction. There was none observed. And at 10 milligrams, we saw four out of six. So the 15-milligram cohort, we'll see if that recapitulates or gets better, and we'll have the opportunity to see if there's a dose response at that time.

Yanan Zhu, Wells Fargo.

This is Kuan on for Yanan. So our question is also around CF. We are wondering, have you seen any data from 15 mg? And if so, any update on the safety? And will you be planning to evaluate any dose higher than that? And I have a quick follow-up.

Yeah. 15 milligram will be the highest dose that we're evaluating. We intend to choose either 10 or 15 milligrams or something in between, perhaps, for the 12-week study in next year. When the 15-milligram cohort data is completed in the first quarter of next year, that's when we'll be able to make that decision. In terms of when we share data around that third cohort, that hasn't been guided. We'll first collect it and then make a determination how and when to share it. Did I address your question?

Yes. And I'm wondering, do you need to show a clear correlation between mucus plug reduction and FEV1? I'm wondering hypothetically, for example, only mucus or plug reduction data is positive and FEV1 is not, would that affect how you view the program and how you make (inaudible)

I had great conversations with a lot of the experts here at the NACFC in Seattle last month. And yes, CT imaging has been a primary endpoint previously. However, that's not our present expectation. The FDA may not yet consider CT imaging as a surrogate endpoint at this time. But I think it's safe to assume that it will be a supportive endpoint for like a Phase 3 or a pivotal trial.

We first have to collect the data from the 12-week study and then share it with the FDA to have that conversation. If the data is convincing, yes, we'll look at primary or a co-primary endpoint that involves CT scan. But the present expectation is that CT imaging will be a supportive endpoint, very similar to what or can be had to go through where they have their primary being FEV, but the support of data played a key role in getting that approved as the first modulator or one of the early modulators.

Yigal Nochomovitz, Citi.

This is Joohwan Kim on for Yigal. Regarding the extra screening visit that you had mentioned for the 12-week study, can you just provide additional clarity on whether you're planning on averaging the screening business together to get a more reliable baseline? And also, are you planning on just doing the one extra measurement? And I guess, why not multiple?

Yeah, and why not do multiple? It's a great question. We haven't had that conversation with the FDA yet. But our present thinking around designing the trial is not only increasing the duration from 4 to 12 weeks and increasing the number of participants from 6 to 20, but it's also strengthening the baseline. And whether that's an additional FEV previsit or a second one? And do we average screening and to FEV pretreatments or not, that conversation will be one of the key questions that we'll have with the FDA.

Once we have aligned on that, I can provide more clarity. But the present expectation is just an additional pretreatment value that can be averaged and strengthening the baseline twofold.

Got it. And if I could just follow up with one more. I believe you had mentioned previously that the quality of life assessment for the Phase 2 for the CFQ RSS was also variable, and so you have decided not to share that. But I guess, is there a strategy in mind to reduce the variability there, so you could better interpret its meaning implied to the future, or is the EQ-5D-5L questionnaire just less subject to variability?

Yeah. So just to catch everybody up, the validated questionnaire that's used in the modulator space is well validated and quite comprehensive and it addresses multiple organs in the body. Our CFQ-R is truncated and focused on just the lungs because it's an inhaled therapeutic, so there's questions in the pulmonary section. And because of that and for other reasons and because of the smallness of the nature of our of 6 cohorts. The variability is just -- these questionnaires are more powerful and larger Phase III studies, but it's still variable to address that.

In our upcoming 12-week study, we do intend to add additional general health questions, what's called an EQ-5D. And I touched on this earlier, but when we look at mobility and self-care and pain, discomfort, anxiety depression, and this general health questionnaire will be coupled with this truncated CFQ-R that's more validated just to add weight to the questionnaire, and we'll see what we can glean from that. And to what extent we modify it or keep it for the Phase 3 trial will also be helpful in this upcoming 12-week study.

Tom Shrader, BTIG.

I'm wondering in the next CF cohort if there's any interest or thoughts about adding slightly less impacted patients where the lungs might be a little cleaner and delivery might be easier. And then I appreciate you at arm's length on Kostaive. But can you comment, are there ongoing discussions? Or has the FDA kind of made a statement and there's no room to discuss anything?

There's definitely room to discuss that they, of course, are interested in this first-in-line therapy for such a huge unmet medical need in the CF space. So there is still flexibility to discuss. With respect to your first question about less impacted individuals, we did -- you'd have to go to our website, but we enabled the cohort patient 5 -- cohort 2, patient 5. This is someone who had more advanced disease and much more numerous plugs and even larger plugs.

And we just got considerable positive feedback from the subject even though their mucus plug reduction was only 9%. And because the size of the plugs that were reduced were meaningful, were larger. So we found -- we had just a success, I would call it, with one particular more advanced subject. So the short answer to your question is, no. We're not refining the list of who's going to be getting the drug for this upcoming 12-week study because we found success in less advanced and more advanced disease.

Yale Jen, Laidlaw & Company.

I apologize, I missed the earlier part of the conversation. Just a quick question about CSL in terms of the -- I understand the initial contract or deal with them, there's sort of four target infectious disease treatments via treatment. So I wonder any progress on those two other than the influenza and COVID. Any comment on that?

Yeah, it's a great question. CSL and Seqirus are considering a demerging process, and they've publicly disclosed that. The timing of that is -- it remains uncertain. But if CSL and Seqirus demerge, then Seqirus will be focused on the vaccine enterprise going forward, especially the flu enterprise. So with respect to the future of the program, the collaboration will come through that arm of the company.

As of right now, COVID is, of course, still very active. Any guidance on the flu program will come from them going forward and likely the Seqirus branch if they demerge. With respect to any new programs, we have communicated in the past that those are being considered or active in certain degrees. But we haven't disclosed any of those details that they will be the right time and place to do it and it will likely be coinciding with any updates we hear from a CSL-Seqirus demerger.

Lili Nsongo, Leerink.

Two questions. First, on the Cystic Fibrosis program. So the program -- the study was initiated in January, about nine patients were dosed by September. You had mentioned initially that the three additional patients for Cohort 3 would be dosed by year-end. How should we think about the enrollment pace? Is 3 patients a quarter what we should expect moving forward? And does that also apply going into the 12-week study?

Yeah. We've expanded the third cohort from 3. At our last quarterly call, we indicated that we -- or estimated that we would have three subjects in our third cohort. We've now communicated that that could be expanded up to six, and that would take us into the first quarter of next year. With respect to -- did I address that question first?

I mean, this wasn't the question, but based on that pattern of having about three patients enrolled per quarter, should we also think about the pace of enrollment for the 20 patients for the 12-week study to be similar?

Yeah. No, we're looking to add a considerable amount of sites in different countries as well to facilitate enrollment for this 12-week study. I alluded to this previously. But when we were at the Seattle conference or at the NACFC, we got to meet with a variety of investigators globally, not just limited to the US. So the percentage and prevalence of Class I and modulator nonresponders in other countries is extraordinary and untapped.

So in addition to the dozen or so sites that we have opened here in the US, I believe that we'll be adding additional sites outside of the US. And that will -- is intended to accelerate the enrollment pace to support the end of 20 rather than the end of 6 for this upcoming 12-week study. So the short answer to your question is that we anticipate enrollment rate to increase with these additional sites and additional access to Class I and modulator nonresponders. To what extent that increases, we'll be the first to find out.

And is the global expansion and the additional sites? Is that captured in the current cash runway guidance?

Yes. But Andy, you can confirm that that's captured in the runway guidance, correct?

Yeah, that is all captured in the runway guidance. And the good news is that we had produced additional material for the CF clinical trials and consequently, the additional cost of expanding the trial is pretty much de minimis. So we're in very good shape there financially. Thank you.

Second question regarding the OTC program. So can you give us an age range in terms of what your pediatric population target would be? Because the data we've seen so far is in 12 and older. And I was wondering what would it take to go to the younger patient? Or would you solely focus on pediatric patients that are 12 to 18?

That's one of the agenda-related questions that we expect for these Type C type meetings with the regulatory agency. Pertaining to pediatrics is what the cutoff age is. Whether it's 5 years old or 8 years old is going to be determined and finalized as part of that meeting. And then adults as well, is it going to be 12 and above or 16 and above are -- that will be -- that's one of the purposes of these meetings to get aligned with the pivotal trial protocol. And that clarity will be provided in the first half of next year is our anticipation.

Do you expect to be able to go into pivotal in pediatric patients without an additional study needed to reach between the dynamic team?

Well, if we can accomplish that, that would be fantastic. But yes, that's the intent. To what extent we can accomplish that, we'll find out. And all I know is there's a much more considerable unmet medical need in these young, specially X-linked males or boys. And the younger they are, the more severe the disease. And the less the requisite or requirement that we have to track glutamine as a primary driver, like it is in the adults. Adults is likely going to be more closely associated with glutamine as a biomarker.

So there are two separate discussions.

And Mr. Payne, it appears we have no further questions this afternoon. Sir, I'd like to turn the conference back to you for any closing comments.

Thanks, everyone, for participating on the call. And if there are remaining questions by those that weren't able to pose them, don't hesitate to reach out to our team, and we'll get back to you as soon as we can. Thanks again.

Thank you very much, Mr. Payne. Again, ladies and gentlemen, that will conclude the Arcturus Therapeutics third-quarter earnings conference call. Again, thanks so much for joining us, everyone, and we wish you all a great afternoon. Goodbye.