Arcturus Therapeutics Q1 2025 Earnings Call Transcript

Quartr
Provided by Quartr
May 12, 2025

There are 14 speakers on the call.

Operator

Today's call is being recorded. I would now like to turn the call over to Nadeus Saffarzadeh, Vice President, Head of Investor Relations, Public Relations and Marketing. Please go ahead.

Speaker 1

Thank you, operator. Good afternoon, and welcome to Arctera Therapeutics Quarterly Financial Update and Pipeline Progress Call. Today's call will be led by Joe Payne, our President and CEO, and Andy Sousin, our CFO. Doctor. Patri Bakula, our CSO and COO will join them for the Q and A session.

Speaker 1

Before we begin, I would like to remind everyone that the statements made during this call regarding matters that are not historical facts are forward looking statements within the Safe Harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward looking statements are not guarantees of performance. They involve known and unknown risks, uncertainties and assumptions that may cause actual results, performance and achievements to differ materially from those expressed or implied by the statement. Please see the forward looking statement disclaimer on the company's press release issued earlier today, as well as the Risk Factors section in our most recent Form 10 ks 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.

Speaker 1

Arcturus specifically disclaims any obligation to update such statements. And with that, I will now turn the call over to Joe.

Speaker 2

Thank you, Neda. It's good to be with you again, everybody. Over the last few months, there has been elevated interest in our mRNA therapeutics pipeline, given that we have meaningful clinical data sets forthcoming. So I will begin today's call with updates pertaining to our mRNA therapeutics pipeline. I will begin with an update on ARCT032.

Speaker 2

This is our messenger RNA therapeutic candidate for cystic fibrosis. Arcturus is advancing enrollment in the open label Phase II multiple ascending dose CF study in adults with CF, who are not eligible for CFTR modulator therapy, or are not taking CFTR modulators due to drug intolerance, poor response, or lack of access to modulators. Each adult participant in the phase two CF study is expected to receive daily inhaled treatments of ARCT032, or over a period of twenty eight days. The study began last December, and to date, the study continues in multiple subjects without safety or tolerability issues. The company expects to complete Phase two enrollment by the end of the year and provide Phase two interim data for the first two cohorts in mid twenty twenty five.

Speaker 2

I'll now move on to our ARCT-eight ten program. This is our messenger RNA therapeutic candidate for ornithine transcarbamylase or OTC deficiency. Arcturus continues to enroll participants in the open label Phase II OTC deficiency study with five intravenous infusions of ARCT-eight ten over a period of two months. The company previously completed the dosing phase involving a cohort of eight people and the dosing was at 0.3 of a placebo controlled European study enrolling OTC deficient individuals. The company expects to provide Phase II interim data this quarter or Q2 twenty twenty five.

Speaker 2

The U. S. Phase II study evaluates several biomarkers, including glutamine and ammonia. Elevated glutamine levels can occur when ammonia scavengers are used, particularly in individuals with urea cycle disorders such as OTC deficiency. While ammonia scavengers reduce circulating ammonia, glutamine may still be elevated due to its role as a temporary ammonia repository.

Speaker 2

And therefore, may be used as a useful biomarker to ascertain proper urea cycle function in OTC deficient individuals. We are also utilizing a newly developed and improved 15N uriogenesis assay. This is supported by a newly published paper out of Haverley's lab at the University of Zurich. This 15N Ureogenesis Assay is expected to provide important data for monitoring the effect of ARCT-eight ten in the company's clinical development program. We are encouraged by the comprehensive data we have collected to date with our CF and OTC programs.

Speaker 2

And given the current market conditions, we have made a strategic decision to focus our resources toward our mRNA therapeutics pipeline. I now shift your attention to our partnered COVID-nineteen vaccine program. We are pleased about the recent EU approval of Costave. This is our self amplifying mRNA COVID-nineteen vaccine. Arcturus received an initial milestone payment from CSL, our global vaccine partner in relation to the EU approval of Costave.

Speaker 2

We continue to make progress expanding the global Costay franchise. Company anticipates a Marketing Authorization Application or MAA filing in The United Kingdom in Q2 twenty twenty five, followed by a U. S. BLA filing in Q3 twenty twenty five. The WHO is expected to announce the updated COVID strain later this week.

Speaker 2

And we, along with our partner, CSL Securis, will update Costave accordingly to support Meiji's distribution efforts in Japan this upcoming season. Our STAR self amplifying mRNA platform continues to benefit from meaningful publications. The company recently published a comprehensive analysis of safety data for Costave with a twelve month follow-up from the pivotal clinical study in Vietnam, which had over 17,000 participants who received at least one dose of the study vaccine. The study confirmed the favorable reactogenicity profile. Acceptable tolerability of ARCT-one hundred fifty four was also observed in older participants and individuals at high risk of severe COVID-nineteen due to underlying medical conditions.

Speaker 2

Long term data from this large trial suggests that the SA mRNA COVID vaccine is safe and well tolerated and did not include any reports of myocarditis or pericarditis. In April, Arcturus' Japanese partner, Meiji Seikopharma published an analysis characterizing the distribution and clearance of ARCT-one hundred fifty four encoded spike protein and non structural proteins in the lymph nodes and injection site muscle in mice, following a single intramuscular vaccination. The study showed the encoded spike protein reached its highest level, approximately three days after vaccination and quickly disappeared from the injection site muscle. The spike protein persisted up to twenty eight days in lymph nodes after vaccination. And the data suggests that this prolonged spike protein expression may be credited for the observed higher immunogenicity.

Speaker 2

And as expected, the study also confirmed that the replication is limited. Now moving to ARCT2304. This is our SA mRNA vaccine candidate for pandemic influenza A, which is also known as H5N1 or the bird flu virus. In April, Arcturus received US FDA Fast Track designation for ARCT2304. As a reminder, this project has been supported in whole with federal funds from the HHS, ASPR and BARDA.

Speaker 2

Company recently completed the recruitment of two twelve adults, which included 80 participants over the age of 60 years old. In a randomized placebo controlled phase one trial being conducted here in The US. The company expects interim phase one data in the second half of twenty twenty five. And with that, I will now pass the call to Andy.

Speaker 3

Thank you, Joe, and good afternoon, everyone. The press release issued earlier today includes financial statements for the first quarter of twenty twenty five 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 detail on the financial performance. I am pleased to announce we were able to extend our cash runway till the first quarter of twenty twenty eight. However, it was not an easy decision.

Speaker 3

Due to the current market environment and uncertainty regarding our regulatory process, we have made the decision to focus our research and development expenditures on our CF and OTC programs exclusively. I am happy to announce that we have received the initial milestone payment from CSL in relation to the UUA EU cost save approval. We anticipate an additional milestone payment and we'll update provide an update on our second quarter call. I will now provide a summary of our financial results for the first quarter of twenty twenty five. For the three months ended 03/31/2025, revenues were $29,400,000 compared to $38,000,000 in the second period of twenty twenty in the same period of 2024.

Speaker 3

The decrease primarily reflects lower development milestone revenues recognized under the CSL collaboration agreement as Coastave transitions from the development stage to commercialization. Research and development expenses were $34,900,000 for the three months ended 03/31/2025, compared to $53,600,000 in the comparable period last year. The decrease in research and development expenses was primarily driven by lower manufacturing costs associated with the Coast Day, Lunar Flu, and BARDA programs, partially offset by increased manufacturing and clinical costs for the Lunar CF and Lunar OTC programs. R and D expenses also declined sequentially for fourth quarter from fourth quarter 'twenty four by almost $9,000,000 We anticipate additional quarterly declines in the second half of fiscal year 'twenty five. General and administrative expenses were $11,300,000 for the three months ended 2025, compared to $14,900,000 in the comparable period last year.

Speaker 3

The decrease in general and administrative expenses was primarily attributable to reduced share based compensation costs. We expect general and administrative expenses to decrease during the next twelve months, driven by lower share based compensation costs and a reduction in expenses related to the commercial transition of the COVID program to CSL. For the three months ended 03/31/2025, Arcturus reported a net loss of approximately $14,100,000 or $0.52 per diluted share compared with a net loss of $3,000,000 or $1 per diluted share in the three months ended 03/31/2024. Cash and cash equivalents and restricted cash were $273,800,000 as of 03/31/2025 compared to $293,900,000 in 12/31/2024. This $20,000,000 decline in cash is reflective of our annualized cash burn this fiscal year.

Speaker 3

As stated earlier, I am pleased to report that the cash runway now extends into 2028 with the reallocation of resources to the CF and OTC programs, which include significant cost reductions. In summary, company remains in a strong financial position and has the cash runway needed to achieve multiple near term value creating milestones for the therapeutic programs. We look forward to another exciting year with multiple upcoming clinical data from our CF and OTC programs. I will now pass the call back to Joe.

Speaker 2

Hey, thanks, Andy. Arcturus continues to make excellent progress across our therapeutics pipeline. And we look forward to multiple key clinical data sets across our mRNA therapeutics and our partnered vaccine programs this year. So with that, let's turn the time over to the operator for some questions.

Operator

Thank you. We'll take our first question from Lily Sanago with Leerink Partners. Please go ahead.

Speaker 4

Hi, good afternoon. This is Lily Sanago from Leerink. Thank you for taking the question and thank you for the update this quarter. I just had two questions. So the first one, could you provide maybe a little more color in terms of the changes that have taken place in order to be able to extend the cash runway as well as you know the potential cash flow incoming cash flows that you anticipate within this new guidance.

Speaker 4

And then the second question relating to the development of COVID vaccine, how should we think about potential milestone related to UK approval and U. S. As well as the timing for those? Thank you.

Speaker 3

Sure, Lily. Thank you for that question. It was not an easy decision Lily, but as you know in this environment I think it was very prudent for us to focus on our two most critical programs. And as a consequence of that, we had to make some tough decisions with respect to cost reductions including some elimination of early development in R and D programs as well as consolidation of our facility. So a combination of all these factors contributed to the extension of the runway, but more importantly as well, you can probably surmise that guidance we originally provided remained relatively conservative based on our cash burn historically.

Speaker 3

So hopefully you'll appreciate that we tend to be hopefully under promising and over delivering with respect to our cash runway. Hope that answers your question.

Operator

It does. Thank you.

Speaker 5

Go ahead.

Speaker 2

Yeah, you also asked a second question with respect to milestones, potential milestones associated with The UK filing or US filing. And there's no milestones associated with those. Andy, you wanted to comment further?

Speaker 3

We have a milestone associated with the first US revenues from Co State but we're not anticipating that Lilly till 2028 frankly assuming approval in '26 or so with The US BLA filing this year. So we're not including that in our forecast. Hopefully you'll appreciate that we'd like to remain conservative with respect to that milestone.

Speaker 2

Thank you.

Operator

And we'll next go to Yasmeen Rimi with Piper Sandler. Please go ahead.

Speaker 5

Good afternoon, team. Thank you so much for all the updates. Wanted to spend some time as many investors are very much looking forward to ARCT032 interim readout here mid-twenty twenty five. I just want to get a good understanding as we get into this mid readout, now that we have a little bit more granular color, and the press release has noted two doses are going to be provided. I guess, how large could the initial interim cohort be across these two doses?

Speaker 5

What sort of the bar for success you wanna see? And then to the extent you can talk about the patient population you're recruiting for this study, that would be really helpful. Sorry, these are like sort of three pronged questions along the interim readout. Just wanna have a good understanding of what we're gonna be getting at that interim look.

Speaker 2

No, good questions, Yaz. We're also excited about this mid year update for our CF program. There's considerable interest in it. It's a great commercial opportunity and it's an exciting program. But you asked about how large of a data set to expect.

Speaker 2

The entire phase two study has been listed at clinicaltrials.gov as 12. So because this is an interim data cut of drawing from two cohorts, you would expect in that six to nine region of subjects. So with respect to the bar of success, clearly this is 28 daily administrations. So it's a significant hurdle to have reasonable safety and tolerability achieved. In addition to that, we'd be looking for FEV or lung function improvements.

Speaker 2

And the bar that has been established recently is 3%. We believe is if we achieve that, that that would justify advancing the program. Clearly the higher percentage that we would get would impact the size and nature and cost of our phase three trial. In terms of the types of patients, I think you might be inferring to maybe class one and non class it would approximately half of these subjects would be class one subjects and the other half would be non class one. But every single one would be similar in that they're all non modulator responders.

Speaker 2

And so we don't tend to bifurcate the data. We view this group as having similar unmet medical need and presentation. So that's it.

Speaker 5

Very helpful. Thank you, Joe. I'll jump back in the Thanks, Yas.

Operator

We'll next go to Seamus Fernandez with Guggenheim Securities. Please go ahead.

Speaker 6

Hey, guys. This is Evan Wang on for Seamus. Thanks for the questions. Just following up on cystic fibrosis. We've seen some tolerability issues from a competitor program.

Speaker 6

Can you remind us again the difference of thirty two versus some of the other competitor mRNA programs? And just to hone in on that a little bit more, in terms of the dose range you're exploring and what we may see from these first or these three dose cohorts, especially relative to what you studied in healthy volunteers and the initial CF patient studies? And then, you know, in terms of the first two dose cohorts, how should we be thinking about that? I guess, and whether you expect each dose cohort to be efficacious or whether you expect a dose response here? Thank you.

Speaker 2

Yeah, a lot there, all good questions. First of all, our therapeutic is different from our competitors. I think the key points of differentiation would be number one, our delivery technology. Our LUNAR lipid nanoparticle delivery technology is exclusive to us. We discovered this and are applying it to the CF program.

Speaker 2

And it's chemically different from what everyone else is utilizing. And we've showcased these differences in preclinical animal models. So clearly, there's something there with respect to our delivery technology. When you're talking about safety and tolerability, you also have to recognize the level of purity of an mRNA construct. And Arcturus has significant intellectual property pertaining to the purification of our mRNA.

Speaker 2

And how this could potentially impact safety and tolerability is on removing impurities effectively. If we do that, then that would logically enhance or expand the therapeutic index and improve a safety and tolerability profile. And those would be two key areas of differentiation. We mentioned that there's two cohorts and each of those is attributed to a different dose level. We have of course flexibility built into this phase two study to have a third cohort or third dose level or expand the second level.

Speaker 2

We haven't provided any guidance on the nature of that third dose level, but the data that we intend to share mid year will involve two, we'll be drawing from two different dose levels. None of these levels of dosing is wasted, we believe. You asked if all of these could be efficacious and somewhat that may indeed be the case. But we'll save the details for that disclosure for mid year.

Speaker 6

Got it. Thank you.

Operator

Thank you. And next we'll go to Myles Minter with William Blair. Please go ahead.

Speaker 7

Hi, this is Jake on for Myles. Thanks so much for taking our question. Couple for you. First on CF, we're just wondering if you guys have talked about any sort of accelerated approval pathway for ARCT032 given the unmet need there in null mutant patients? And then a second question on OTC, you guys mentioned measuring glutamine as a biomarker there.

Speaker 7

Just wondering if you have any sort of initial bar for success where we should expect a percentage of normal on glutamine? Thank you.

Speaker 2

Great questions. First of all, with respect to some sort of regulatory acceleration. Whenever you see excellent Phase two data, that would always warrant a discussion with regulators to accelerate the approval pathway, Especially like you mentioned for serious diseases where there's significant unmet medical need. We have not shared any data. So we don't know.

Speaker 2

It would be too early for me to guide whether this is good or great or excellent data. But to simply address your question, if it's considered excellent data, then yes, we would be looking to accelerate the clinical path for sure. With respect to glutamine, I think it I'd like to remind everyone listening that if you have a dysfunctional urea cycle, then your body cannot convert that ammonia into urea. So what does it do? It converts ammonia into glutamine.

Speaker 2

So glutamine is just a great biomarker that can help us understand this disease. In terms of what would we be looking for in glutamine levels, it would be simply to restore normal levels. So very often you would, it's well known that folks suffering from urea cycle disorders, including OTC deficiency, have elevated glutamine levels. And if we could restore those into the normal range, that could be some convincing evidence that our therapeutic

Operator

And next we'll go to Whitney Ijem with Canaccord. Please go ahead.

Speaker 8

Hey, guys. Thanks for taking the questions. Just sticking with CF, can you talk about the time points that you're measuring FEV in the study and what the duration of follow-up I guess we should expect from both cohorts? I'm I guess curious if there's interim time points you're looking at between the zero and twenty eight days and if there's subsequent time points and if we should expect all those for both cohorts in the upcoming data.

Speaker 2

Yeah, details pertaining to this trial design are available on clinicaltrials.gov. And also the CF Foundation Tracker that tracks not only our phase two trial and the details there, but also our competitors. And that's a good reference for people to look at. We're gonna be measuring FEV or lung function improvement this twenty eight day treatment cycle. So what do I mean by before?

Speaker 2

Well, there's a screening process where FEV is measured. Then of course at day zero, prior to first administration. And then there's a few measurements throughout the cycle. And then of course afterwards, it's approximately seven FEV measurements taken. We haven't shared the specific details of that just for competitive purposes.

Speaker 2

But there's plenty of data points in the study to give a decent level of conviction to advance this program further into development. Did I address your question, Whitney?

Speaker 8

I think so. Just to clarify though, so we should expect the full twenty eight days and then maybe some additional follow-up from both cohorts or is it Yeah. Okay. Okay.

Speaker 2

Maybe not the entire follow-up because these can be a few months in nature, the follow-up. So we may not have the entire follow-up for Right. We intend to have safety, tolerability and the lung function values for the complete treatment course, correct.

Speaker 8

Okay, perfect, excellent. Okay, and then just a follow-up to clarify on the US Coastalefe milestone, is it that there is not a milestone on approval or that you are not including a milestone on approval in your guidance to be conservative?

Speaker 3

Okay. Yes, we there is a milestone associated with first commercial sales in The U. S. And that milestone is not included in our guidance because we're not anticipating first commercial sales until 2028. And so hopefully that gives you some perspective as to the cash runway does not include a U.

Speaker 3

S. Milestone related to first commercial sale.

Speaker 8

Okay. And also not one related to EU I'm sorry, US approval.

Speaker 3

Right. There is no milestone with US approval. It's related

Speaker 2

to

Speaker 3

sales. Correct.

Operator

Okay, perfect. Perfect.

Speaker 8

Thank you very much.

Speaker 2

Thank you, Winnie.

Operator

Thank you. And next we'll go to Pete Stavropoulos with Cantor Fitzgerald. Please go ahead.

Speaker 9

Hi, this is Samantha on the line for Pete. Thanks for taking our questions. So on the CF program, given that the impaired CFTR function impedes mucociliary clearance and mucous plugging. Do you see potential for 32 to normalize or remodel the lungs? And if it can, can you speculate on the length of time this may take?

Speaker 9

I'm curious to hear your thoughts.

Speaker 2

No, it's a great question. We're hoping to be the first to answer that question with data. But just some basic comments is that these patients that are participating in our trial do not have 100% lung function. And some of that dysfunction is attributed to irreversible damage. So there's gonna be some portion of the lung that just isn't recoverable.

Speaker 2

But we're also optimistic that there is a portion that is. And so the lung is very well known to regenerate and heal effectively if you can reverse the dysfunctional biology. So we hope to see that our upcoming trial results. In terms of the time, because this is a topically administered agent, the time course to restoration, it may be different from systemic treatments like you see in the modulator business. We're different.

Speaker 2

We're topically administered. So accessibility to these cells is going to be a key marker of success. And as the lung heals, then more cells would be accessible. So at least theoretically we anticipate a steady improvement over an extended period of time. But what is just unknown at this point is how long is that time period and how much material is required.

Speaker 2

But we were anxiously awaiting the data.

Speaker 9

That's very helpful. And one short follow-up question. Besides FEV, are there any other measurements that you could or will look at that might enhance the investigator's conviction in the program, something like quality of life measurements or others?

Speaker 2

Yeah, there's a survey that were provided. There's questionnaire over the last couple of decades in the modulator field. Have improved these questionnaires significantly and that will complement our FEV data. In terms of other mechanisms like mucociliary clearance or others, the FDA came back with some simple guidance there that all they need to see is FEV to justify advancing this into a phase three trial. But we are supporting that data set with some questionnaires to see if we can support what we observe with respect to lung function improvements.

Speaker 9

That's great. Thank you guys so much.

Speaker 2

Thank you, Samantha.

Operator

Next we'll go to Tom Schrader with BTIG. Please go ahead.

Speaker 10

Good afternoon. Thanks for taking the question. You've commented that the next CF trial is going to depend a lot on the size of the efficacy signal. But do you have a sense of the minimum safety database for indication of this? Is 100 about right?

Speaker 10

Or are you applying sort of completely blind at this point?

Speaker 2

We're definitely not completely blind. I think 100 is reasonable. Unfortunately, depends on how successful the Phase II results are. If the more successful they are, the more leverage we have to decrease this number and get this into real subjects commercially as fast as possible. But 100 is reasonable.

Speaker 2

So that would imply that we had 39 subjects in phase one and phase 1B, we add another dozen, that would get us over 50 after phase two, that we'd probably need a minimum of 50 people to strengthen the database to achieve that 100. But we have not just to be clear, we haven't received that. But that's a reasonable estimate.

Speaker 10

And then in OTC, it seems like there's two deliverables. One, you're helping OTC, but in a grander plan that you're getting stuff into the liver. Is that the same readout? Or what I'm really asking is, do you have any way to monitor uptake into the liver that isn't correction of OTC or is it really the same readout for both questions?

Speaker 2

Yeah, there's not going to be a bio sampling or some sort of tissue removal as part of this process. The FDA doesn't require that for these significant rare diseases. But we do have a biomarker strategy. We've alluded to glutamine, for example, as one of those biomarkers. But then there's also a functional readout.

Speaker 2

And the 15N Ureogenesis Assay is also of interest there. A great paper just came out last week out of the University of Zurich that I encourage people listening to refer to. But that's another, a new exciting way to track disease progression or healing.

Speaker 10

Okay, great. Thank you.

Speaker 2

Thank you, Tom.

Operator

Next we'll go to Yigal Nochomovitz with Citi. Please go ahead.

Speaker 11

Yeah, hi, thanks. I had one question on CF and one on OTC. On CF, think in the past you've talked about some thresholds for FEV1 improvement that you would deal with the sufficiently strong to move forward. I'm wondering if you could comment on that with respect to the upcoming data. And then on OTC you mentioned some biomarkers, you mentioned the N15 assay.

Speaker 11

I'm wondering are you also going to measure orotic acid and uridine because those are also urinary biomarkers that would be interesting. And you mentioned glutamine but I think alanine is another one that could be interesting as biomarker in lieu of ammonia which you can't measure. Thanks.

Speaker 2

Good, Yigal. I'll first start with the thresholds in CF. These numbers that have been communicated over this last quarter, especially are based on historical precedents, but in the modulator space. But if you can have a three percent and if you can show a three percent improvement in FEV over a treatment course, in order to reprove that in a Phase III study would require potentially hundreds of people. And so that's a significant endeavor.

Speaker 2

Nonetheless, one that we would pursue in order to prove that this is real. But if the FEV value goes up to 5%, then your phase three trial could be as low as 50 people. Definitely or at least confidently below under 100 people. So there, if you go north of 5%, then in higher FEV responses, It wouldn't change the size of the phase three trial because earlier on this call there was a question about how much, how many people would you need to establish a significant enough safety database and 100 is approximated at this point. But, so whether you see higher levels of FED, you'd still need at least fifty percent, we estimate, or 50 participants in the phase three trial.

Speaker 2

You know, shifting to the OTC program, erotic acid is a biomarker we are tracking as well, and that's in the urine. And that is associated with, you know, the urea cycle and hyperammonemia. And then also there is several amino acids and biomarkers that we're exploring. Glutamine is just more well understood and frequently used by clinicians and doctors to track people on ammonia scavengers to look at glutamine to see if that's elevated as well. Then they still have some challenges with hyperammonemia that's outside of their ammonia scavenger medicine and their diet.

Speaker 2

So glutamine is a more common biomarker that's well more well understood in the community. But you're absolutely right, there's other biomarkers we're measuring as well.

Speaker 11

Okay, thanks. And then just going back to the three percent versus five percent, what are you assuming there in terms of the dispersion of the data given it's obviously a relatively small sample size, six or 12 people. So what does that mean in terms of how much dispersion you're willing to accept and be comfortable with whether it's the 3% or the 5% because obviously that will impact the way you power things.

Speaker 2

Yeah, yeah, yeah. Of course, we'd like to see elevated consistency in small data sets such as this. We'll have the opportunity to share that data and discuss it. But at this time, it's just too early to comment further on that. But yes, to address the core of your question, a more consistent data set would be preferred.

Speaker 11

Got it. All right. Thanks so much.

Operator

And next we'll go to Yanan Zhu with Wells Fargo. Please go ahead.

Speaker 12

Great. Thanks for taking our questions. So on cystic fibrosis, I thought previously the guidance was the data second quarter. Given that the guidance is midyear, is it okay to assume that more likely the data will be in the third quarter? And what might be the venue for the update?

Speaker 12

And on efficacy, I was wondering, you know, in terms of, you know, you talk about the bar for FEV1. I was wondering what is the bar for proportion of patients meeting that 3% minimum threshold of improvement? Would like if the response rate, if we can call them that, is expected to be fairly broad, or could this be even like a 20% response rate still considered a worthwhile product to move forward with? Thanks.

Speaker 2

Yeah, thanks, Yunnan. You bring up a great question. With respect to the bar of a response rate, that hasn't really been established by the field. We're going to have to make a call based upon this data, the FEV, and the consistency of it. There isn't some sort of feedback we've received from the FDA, for example, on this question.

Speaker 2

We have received feedback from the FDA that 12 subjects may very likely be sufficient to advance this. So as long as there's some level of consistency within these 12 subjects, and remember multiple dose levels will be evaluated within this group, then that is likely considered success and we'll be able to receive approval to advance this into a phase three trial. Then you asked another question though. It was about

Speaker 12

The timing and venue.

Speaker 2

Oh, the timing and venue. Yeah, well, because this data set is involving data from two cohorts now, We wanted to allow that extra few weeks just to schedule and get additional numbers to strengthen the data set. So it's just that's the reason for focusing on a midyear interim data update. In terms of a venue, we haven't decided on that. But that is something that we'll communicate relatively soon here.

Speaker 12

Okay. If I may, also wondering since you are moving ahead with filing the BLA, so you're still on track for filing the BLA for Costave in The U. S. I was wondering with the new leadership at CBIR, have you had any interactions with FDA very recently? And what is the expectation for this filing?

Speaker 13

Thanks.

Speaker 2

Our interactions with the FDA, I'll first comment on that, have been normal. The feedback we've received has been within the expected timeline or even earlier in the example of the fast track designation on the vaccine side. So we haven't seen any changes to regulatory environments for at least our mRNA based vaccines and therapeutics. We do recognize that there's a passionate new administration that, but all of them and all of the employees we've talked to at each of these agencies are passionate about gaining access to safer and better medicines and vaccines. And that is almost a credo at Arcturus.

Speaker 2

We want to provide that. And we're believing we're providing data to support that notion. So I think the science will stand in its own legs throughout this process.

Speaker 12

Great. Thanks for taking the questions.

Speaker 2

Yeah, thank you, Yunon.

Operator

We'll go next to Yale Jen with Laidlaw and Company. Please go ahead.

Speaker 13

Good afternoon and thanks for taking the questions. Just two from us. The first one is for the urogenesis asset you mentioned during the call. Could you elaborate a little bit more in terms of any specific readout that can, you know, be relevant to the data release? And maybe the second question really here is that on the big picture perspective, that given that you are focusing much more on your in house programs, and so far you have two very active ones.

Speaker 13

Would you consider a little bit more maybe even early stage, so maybe enrich the pipeline, maybe sometime come out later this year or late next year to enrich that? And thanks.

Speaker 2

Yeah, the early, early stuff does not consume a lot of resources. So clearly we want to be in a position of being able to announce new programs when it's appropriate upon establishing any sort of proof of concept for each of these therapeutic programs. We've already done a considerable effort there already to date. So we're in a position to move if needed. Our focus on our budget and extending the runway hasn't impacted our subsequent programs.

Speaker 2

That work's already been done.

Speaker 13

Okay, in terms of the urogenesis assay, any specific regard that we should pay attention to?

Speaker 2

Yeah, I encourage people to go to the 15N urogenesis paper that I've highlighted a couple times on this call out of the University of Zurich. It's new. It's called the it comes out of the Journal of Metabolic Health and Disease. And the title of it is Characterization and Treatment Monitoring of Ureogenesis Disorders Using stable isotopes. But we just believe it's a significant upgrade on the uriogenesis assays of decades ago.

Speaker 2

It's a considerably better process. And we'll be collecting data within the context of what's been utilized in this paper. That's very helpful. We'll provide more details on this at the right time. Just planted that seed.

Speaker 2

Okay.

Speaker 13

Well, great. Thanks a lot and congrats on all the progress.

Operator

And this concludes our question and answer session. I would now like to turn the call back over to Joe Payne for final and closing remarks.

Speaker 2

Hey, thanks everyone for participating on the call. I know there's some conferences up and we can meet face to face. But if there's any remaining questions, don't hesitate to reach out to our team and we'll get back to you. Thanks everybody.

Operator

Thank you. And this does conclude today's conference. We thank you for your participation. You may disconnect at any time.

Powered by Quartr

Key Takeaways

  • ARCT032 (cystic fibrosis): Phase II inhaled mRNA therapy enrolling adults ineligible for CFTR modulators, with daily dosing over 28 days, no safety issues to date, full enrollment by year-end and interim data for the first two cohorts (6–9 patients) expected mid-2025.
  • ARCT810 (OTC deficiency): Open-label Phase II IV study continues, evaluating biomarkers including ammonia, glutamine and a new 15N ureogenesis assay, with interim results due in Q2 2025.
  • Strategic focus and cash runway: R&D resources have been narrowed to the CF and OTC programs amid market challenges, delivering cost cuts that extend the company’s cash runway into Q1 2028.
  • Partnered COVID-19 vaccine (Costava): EU approval secured and initial milestone from CSL received; UK MAA filing in Q2 2025 and U.S. BLA in Q3 2025, supported by 12-month safety data in 17,000+ participants showing no myocarditis or pericarditis.
  • Q1 2025 financials: Revenues of $29.4 M (down from $38 M), R&D expenses of $34.9 M, net loss of $14.1 M, and cash of $273.8 M as of March 31, supporting near-term pipeline milestones.
AI Generated. May Contain Errors.
Earnings Conference Call
Arcturus Therapeutics Q1 2025
00:00 / 00:00