Arcturus Therapeutics Holdings Inc. Stock Price: Quote, Forecast, Splits & News (ARCT)

ARCT

Arcturus Therapeutics Holdings Inc.

ARCT

Overview

Financials

Historical Data

$12.35

+$0.07

0.57%

At close: Jun 18, 4:00:01 PM EDT

$12.40

+$0.05

0.4%

After hours: 7:59:09 PM EDT

Financial information provided by Financial Modeling Prep. Options data provided by Unusual Whales. Live transcripts provided by Quartr. Reported revenue and EPS data from Earnings powered by FinChat.io. All data is provided for informational purposes only, and is not intended for trading purposes or financial, investment, tax, legal, accounting or other advice.

Market Cap: $335M
IPO Date: 5/22/13
CEO: Mr. Joseph E. Payne M.Sc.
Fulltime Employees: 174
Sector: Healthcare
Industry: Biotechnology
Country: US
Exchange: NASDAQ Global Market

Peers

$0.00

Watchlist

$0.00

Perplexity Finance

ARCT

Arcturus Therapeutics Holdings Inc.

ARCT

Overview

Financials

Historical Data

$12.35

+$0.07

0.57%

At close: Jun 18, 4:00:01 PM EDT

$12.40

+$0.05

0.4%

After hours: 7:59:09 PM EDT

Market Cap: $335M
IPO Date: 5/22/13
CEO: Mr. Joseph E. Payne M.Sc.
Fulltime Employees: 174
Sector: Healthcare
Industry: Biotechnology
Country: US
Exchange: NASDAQ Global Market

Peers

$0.00

Watchlist

$0.00

Arcturus Therapeutics Holdings Inc. Stock Price: Quote, Forecast, Splits & News (ARCT)

Change

+$0.07

0.57%

Financial Profile

Prev Close

$12.28

Market Cap

$334.94M

Day Range

$12.10 - $12.96

Open

$12.18

P/E Ratio

-4.9

52W Range

$8.04 - $28.05

24H Volume

$4.60M

Dividend Yield

0.00%

EPS

-2.52

Key Issues

Latest Price Movement

Jun 20

Earnings

Report Date

May 12, 2025

Reported Revenue

29.4M

Beat +25.84%

Avg 1d Change

4.04%

Report Period

Q1 2025

Reported EPS

-0.52

Beat +67.09%

Implied Move 1d

13.49%

Transcript

11m 25s

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 in subsequent filings with the SEC. In addition, any forward looking statements represents 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.

12m 16s

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 therapeutic pipeline. I will begin with an update on ARCT032. This is our messenger RNA therapeutic candidate for cystic fibrosis. Arcturus is advancing enrollment in the open label Phase two 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 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. I'll now move on to our ARCT eight ten program. This is our messenger RNA therapeutic candidate ornithine transcarbamylase or OTC deficiency.

13m 51s

Arcturus continues to enroll participants in the open label Phase two 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 zero point three mgs per kilogram 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. 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 ammonia repository. 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 uriogenesis assay is expected to provide important data for monitoring the effect of ARCT-eight ten the company's clinical development program.

15m 32s

We are encouraged by the comprehensive data we have collected to date with our CF and OTC programs. 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 vaccine partner in relation to the EU approval of Costave. 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 And we, along with our partner, CSL Securus, will update Costave accordingly to support Meiji's distribution efforts in Japan this upcoming season. Our STARR 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.

17m 15s

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. 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 54 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. 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. [indiscernible] In April, Arcturus received US FDA fast Track designation for ARCT2304.

19m 6s

As a reminder, this project has been supported in whole with federal funds from the HHS, ASPR, and BARDA. 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 U. S. 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. Thank you, Joe, and good afternoon everyone. 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. Due to the current market environment and uncertainty regarding our 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 UAEU costave approval.

20m 41s

We anticipate an additional milestone payment and will 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 29,400,000.0 compared to $38,000,000 in the second period in the same period of 2024. The decrease primarily reflects lower development milestone revenues recognized under the CSL collaboration agreement. As Coast Abe 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 as associated with the Coast Day lunar flu and BARDA program. Partially offset by increased manufacturing and clinical costs for the LUNAR CF and LUNAR OTC programs. R and D expenses also declined sequentially, 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 14,900,000.0 in the comparable period last year. The decrease in general and administrative expenses primarily attributable to reduced share based compensation costs.

22m 39s

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 14,100,000,000.0 or $0.52 per diluted share compared with a net of $3,000,000 or $1 per diluted share the three months ended 03/31/2024. Cash and cash equivalents restricted cash were $273,800,000 as of 03/31/2025, compared to 293,900,000.0 in 12/31/2024. This $20,000,000 decline in cash is reflected of our annualized cash burn this fiscal year. 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.

24m 21s

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.

24m 42s

Thank you. Take our first question from Lily Sonago with Leerink Partners. Please go ahead. Hi, good afternoon. This is Lily from Leerink. You for taking the question and thank you for the update this quarter. I just have two questions. So the first one 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 in cash flow, incoming cash flows that you anticipate within the this new guidance? And then the second question relating to the development of co state vaccine, how should we think about potential milestone related to UK approval and US as well as the timing for those? Thank you.

25m 45s

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. 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.

26m 55s

It does. Thank you. Go ahead. Yeah. With 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 wanted to comment further We have a milestone associated with the first U. S. Revenues from Coastal but we're not anticipating that literally till 2028 frankly. Assuming approval in 2026 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.

27m 43s

Thank you.

27m 46s

And we'll next go to Yasmeen Rimi with Piper Sandler. Please go ahead. Good afternoon, team. Thank you so much for all the updates. I wanted to spend some time as many investors are very much looking forward to ARCT 30 two's interim readout here mid-twenty twenty five. I just want to get a good understanding as we get into this mid readout now that have a little bit more granular color, In the press release it's noted two doses are gonna be provided. I guess how large could the initial interim cohort be across these two doses? What sort of the bar that for success you wanna see. And then the extent you can talk about the patient population, that you're recruiting for the study, would be really helpful. Sorry. These are, like, sort of three pronged questions along the interim readout. Wanna have a good understanding of what we're gonna be getting at that interim look. [indiscernible] 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. The entire Phase two study is has been listed at clinicaltrials.gov as 12.

29m 13s

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. And the bar that has been established recently is 3%. We believe is is if we achieve 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 [indiscernible] In terms of the types of patients, I think you might be inferring to maybe Class I and non Class I. Approximately half of these subjects would be Class I 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. 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.

30m 33s

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

30m 40s

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

30m 45s

Hey, guys. This is Evan Wang on for Seamus. Thanks for the questions. Just following up on cystic fibrosis. 've seen some tolerability issues from a competitor program. Can you remind us again the difference of 32 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 whether and whether you expect each dose cohort to be efficacious or whether you expect a dose response here. Thank you.

31m 32s

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

32m 58s

We haven't provided any guidance on the nature of that third dose level. But the data that we intend to share in mid year will involve two will be drawing from two different dose levels. None of these levels have 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 and for that disclosure for midyear. Got it. Thank you.

33m 38s

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

33m 44s

Hi, this is Jake on for Myles.

33m 46s

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 that unmet need there in null mutant patients? And then a second question on OTC, you guys mentioned measuring glutamine as a biomarker there. Wondering if you have any sort of initial bar for success where we should expect a percentage of normal on glutamine. Thank you.

34m 17s

Okay, 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 unmet medical need. We have not shared any data, so we don't know 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 then yes, we would be looking to accelerate the clinical path for sure. With respect to glutamine, 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. 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? Would it be 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 a glutamine levels. And if we could restore those into the normal range, that could be some convincing evidence that our therapeutic And next we'll go to Whitney Ijem with Canaccord.

36m 9s

Please go ahead. Hey, guys. Thanks for taking the questions. Just sticking with CF, can you talk about the time points that you're measuring FEV in this 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.

36m 39s

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? 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 a FEV measurements taken. We shared the specific details of that. Just for competitive purposes. 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?

37m 47s

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? Is it that [indiscernible] 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 We intend to have safety, tolerability and the lung function values. For the complete treatment course, correct?

38m 15s

Okay, perfect. Excellent. Okay. And then just a follow-up to clarify on The U. S. Coastal milestone. Is it that you there is not a milestone on approval or that you are not including a milestone on approval in your guidance to be conservative?

38m 32s

Okay. Yes, 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 to the cash runway does not include a U. S. Milestone related to first commercial sale.

39m

Okay. And also not one related to EU I'm sorry, U. S. Approval.

39m 5s

Right. There is no milestone with US approval. It's related to sales, correct.

39m 12s

Okay. Perfect. Perfect. Thank you very much. Thank you, Winnie.

39m 17s

Thank you. And next we'll go to Pete Stavropoulos with Cantor Fitzgerald. Please go ahead. Hi, this is Samantha on the line for Pete. Thanks for taking our questions. So, on the CF program, given that the impaired CF 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 I'm curious to hear your thoughts. No, it's a great question. We're We're hoping to be the first to answer that question with data. But just some basic comments is that these 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. 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 in our upcoming trial results. In terms of the time, because this is a topically administered agent, the time course to to restoration it may be different from systemic treatments like you see in the modulator business.

40m 44s

We're different. 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 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. But we were anxiously awaiting the data.

41m 17s

That's very helpful. And one short 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?

41m 34s

Yeah, there's a survey that we're provided. There's a validated CF questionnaire over the last couple decades 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 III trial. But we are supporting that data set with some questionnaires to see if we can support what we observe in respect to lung function improvements.

42m 15s

That's great. Thank you guys so much.

42m 17s

Thank you, Samantha.

42m 20s

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

42m 25s

Good afternoon. Thanks for taking the question.

42m 27s

You've commented that the next CF trial is going to depend a lot on the 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? Or are you applying sort of completely blind at this point?

42m 46s

We're definitely not completely blind. Think 100 is reasonable. Unfortunately, depends on how successful the phase two results are. If 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. So that would that would imply that we had 39 subjects in phase one and phase 1b, we had 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.

43m 32s

And then in OTC, it seems like there's two deliverables. One, your helping OTC 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? 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 and the 15N Ureogenesis Assay. [indiscernible] 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 [indiscernible] exciting way to track disease progression or healing. Okay, great. Thank you.

44m 43s

Thank you, Tom.

44m 45s

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

44m 50s

Yeah, hi thanks. I had one question on CS and one on OTC On CF, I 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 Then on OTC you mentioned some biomarkers, you mentioned the N15 assay. I'm wondering, you also going to measure urtic acid and uridine because those are also urinary biomarkers would be interesting 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.

45m 37s

Good, thanks 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 3% if you can show a 3% improvement in FEV, over a treatment course, in order to reprove that in a Phase three study would require potentially hundreds of people And so that's a significant endeavor. 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 and if you go north of 5%, then in higher FEV responses. It wouldn't change the size of the Phase III trial because earlier on this call, was a question about how much many people would you need to establish significant enough safety database. And 100 is approximated at this point. But so whether we see higher levels of FEV, you'd still need at least 50%. We we estimate, or 50 participants in the Phase III trial. Shifting to the OTC program, Ironic acid is is a biomarker we are tracking as well and that's in the urine.

47m 11s

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

47m 57s

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 relatively small sample size, you know, six or 12 people So what does that mean in terms of how much dispersion you're willing to accept be comfortable with? Know, whether it's the three percent or the 5% because obviously that will impact the the way you power things.

48m 23s

Yeah, yeah, yeah. Of course, we'd like to see elevated consistency in small data sets such as this. 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 your the core of your question, a more consistent data set would would be preferred.

48m 51s

Got it. All right. Thanks so much.

48m 56s

Next we'll go to Yanan Xu with Wells Fargo. Please go ahead.

49m

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 mid year, it okay to assume that more likely the data will be in the third quarter? And what might be the venue for the update? And on efficacy, I was wondering you know, in terms of, you know, you talk about the bar for SEV1. I was wondering what is the bar for proportion of patients meeting that three percent minimum threshold of improvement would Would it be like if the response rate, if we can call them that, is like expected to be fairly broad, or could this be even like a twenty percent response rate still considered a worthwhile product to move forward with? Thanks. Yes. Thanks, Shinon. 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. We have received feedback from the FDA that 12 subjects may very likely be sufficient to advance this.

50m 35s

So as long as there's some level of consistency within these 12 subjects. And remember multiple dose levels will be evaluated in within this group, then that is likely considered success and we'll be able to receive approval to advance this into a Phase III trial. Then you asked another question, though, It was about timing and venue. Oh, the timing and venue. Yeah. Well, because this dataset 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.

51m 44s

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 cost save 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 for this filing? Thanks.

52m 15s

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. 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 the these agencies are passionate about gaining access to safer and better medicines and vaccines. And that is almost a credo at Arcturus. We wanna provide that. And we're believing we're providing data support that notion. So I think the science will stand in its own legs throughout this process.

53m 8s

Great, thanks for taking the questions. Yeah, thank you, Yuno.

53m 14s

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

53m 19s

Good afternoon and thanks for taking the question. Just two from us.

53m 25s

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, very relevant to the data release? [indiscernible] 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. Would you consider a little bit more, maybe even in the early stage so maybe enrich the pipeline, maybe sometime come out later this year or late next year to to enrich that. And thanks.

54m 13s

Yeah, the early, early stuff does not consume a lot of resources. So you know, 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.

54m 32s

And we've already done considerable effort there already to date. So we're in a position to move if needed. [indiscernible] focus on our budget and extending the runway hasn't impacted our programs. That work has already been done.

54m 51s

Okay, in terms of the asset any specific readout that we should pay attention to?

54m 59s

Yeah, I encourage people to go to the 15N Ureogenesis Paper that I've 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 Ureogenesis Disorders Using Stable Isotopes. But we just believe it's a significant upgrade on on the Ureogenesis assays of decades ago. It's a considerably better process and we'll be collecting data within the context of what's been utilized in this paper.

55m 46s

Okay, that's very helpful.

55m 48s

And we'll provide more details. On this at the right time. We just planted that seed. If that makes sense. Okay.

55m 57s

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

56m 3s

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.

56m 10s

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, hesitate to reach out to our team and we'll get back to you. Thanks everybody.

56m 23s

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

Key Insights

Arcturus is focusing its resources on its mRNA therapeutics pipeline, specifically the ARCT032 program for cystic fibrosis (CF) and ARCT810 for ornithine transcarbamylase (OTC) deficiency, with Phase 2 enrollment for CF expected to complete by year-end and interim data for both programs anticipated in mid-2025.
The company received EU approval for its partnered COVID-19 vaccine, Costave, resulting in an initial milestone payment from CSL; further regulatory filings are planned for the UK (Q2 2025) and US (Q3 2025), but no additional milestone payments are expected until first US commercial sales, likely in 2028.
Financially, Arcturus reported Q1 2025 revenues of $29.4M (down from $38M YoY), a net loss of $14.1M ($0.52 per diluted share), and ended the quarter with $273.8M in cash; cost reductions and program prioritization have extended the cash runway into Q1 2028.
R&D and G&A expenses declined year-over-year and sequentially, with further reductions expected as the company narrows its focus to CF and OTC programs; no new early-stage programs will be prioritized until further notice.
For the CF program, the interim Phase 2 readout in mid-2025 will include data from two dose cohorts (6–9 subjects), with a 3% FEV improvement considered a meaningful efficacy bar; safety, tolerability, and lung function are primary endpoints, and regulatory acceleration will be considered if results are strong.

Recent Transcripts

Arctera extends cash runway to 2028, focusing on CF and OTC mRNA therapeutics.

CoStave mRNA COVID-19 vaccine received EU approval; key Phase II data for CF and OTC due in 2025.

Earnings

Report Date

May 12, 2025

Reported Revenue

29.4M

Beat +25.84%

Avg 1d Change

4.04%

Report Period

Q1 2025

Reported EPS

-0.52

Beat +67.09%

Implied Move 1d

13.49%

Transcript

11m 25s

12m 16s

13m 51s

15m 32s

17m 15s

19m 6s

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24m 21s

24m 42s

25m 45s

26m 55s

27m 43s

Thank you.

27m 46s

29m 13s

30m 33s

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

30m 40s

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

30m 45s

31m 32s

32m 58s

33m 38s

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

33m 44s

Hi, this is Jake on for Myles.

33m 46s

34m 17s

36m 9s

36m 39s

37m 47s

38m 15s

38m 32s

39m

Okay. And also not one related to EU I'm sorry, U. S. Approval.

39m 5s

Right. There is no milestone with US approval. It's related to sales, correct.

39m 12s

Okay. Perfect. Perfect. Thank you very much. Thank you, Winnie.

39m 17s

40m 44s

41m 17s

41m 34s

42m 15s

That's great. Thank you guys so much.

42m 17s

Thank you, Samantha.

42m 20s

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

42m 25s

Good afternoon. Thanks for taking the question.

42m 27s

42m 46s

43m 32s

44m 43s

Thank you, Tom.

44m 45s

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

44m 50s

45m 37s

47m 11s

47m 57s

48m 23s

48m 51s

Got it. All right. Thanks so much.

48m 56s

Next we'll go to Yanan Xu with Wells Fargo. Please go ahead.

49m

50m 35s

51m 44s

52m 15s

53m 8s

Great, thanks for taking the questions. Yeah, thank you, Yuno.

53m 14s

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

53m 19s

Good afternoon and thanks for taking the question. Just two from us.

53m 25s

54m 13s

54m 32s

54m 51s

Okay, in terms of the asset any specific readout that we should pay attention to?

54m 59s

55m 46s

Okay, that's very helpful.

55m 48s

And we'll provide more details. On this at the right time. We just planted that seed. If that makes sense. Okay.

55m 57s

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

56m 3s

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.

56m 10s

56m 23s

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

Key Insights

Arcturus is focusing its resources on its mRNA therapeutics pipeline, specifically the ARCT032 program for cystic fibrosis (CF) and ARCT810 for ornithine transcarbamylase (OTC) deficiency, with Phase 2 enrollment for CF expected to complete by year-end and interim data for both programs anticipated in mid-2025.
The company received EU approval for its partnered COVID-19 vaccine, Costave, resulting in an initial milestone payment from CSL; further regulatory filings are planned for the UK (Q2 2025) and US (Q3 2025), but no additional milestone payments are expected until first US commercial sales, likely in 2028.
Financially, Arcturus reported Q1 2025 revenues of $29.4M (down from $38M YoY), a net loss of $14.1M ($0.52 per diluted share), and ended the quarter with $273.8M in cash; cost reductions and program prioritization have extended the cash runway into Q1 2028.
R&D and G&A expenses declined year-over-year and sequentially, with further reductions expected as the company narrows its focus to CF and OTC programs; no new early-stage programs will be prioritized until further notice.
For the CF program, the interim Phase 2 readout in mid-2025 will include data from two dose cohorts (6–9 subjects), with a 3% FEV improvement considered a meaningful efficacy bar; safety, tolerability, and lung function are primary endpoints, and regulatory acceleration will be considered if results are strong.