Arcturus (ARCT) Q3 2026 Earnings Call Transcript

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DATE

Thursday, Nov. 5, 2026

CALL PARTICIPANTS

• Vice President, Head of Investor Relations, Public Relations and Marketing - Neda Safarzadeh
• President and Chief Executive Officer - Joseph Payne
• Chief Financial Officer - Dennis Mulroy
• Chief Scientific Officer and Chief Operating Officer - Padmanabh Chivukula
• Chief Medical Officer - Alan Cohen

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TAKEAWAYS

• Revenue -- $18.3 million for the quarter, an increase from $17.2 million primarily driven by a $12 million termination payment and the recognition of deferred revenue from the CSL Seqirus collaboration.
• Net Loss -- $7.4 million, or $0.26 per share, narrowing from a net loss of $13.4 million, or $0.49 per share, in the comparable period last year.
• Cash Balance -- $184.8 million as of Sept. 30, 2026, which management expects will provide a cash runway through year-end 2028.
• ARCT-032 Enrollment -- Enrollment in Cohort 4 of the Phase II cystic fibrosis study is expected to be completed by year-end 2026, supported by accelerated clinical site activations in Israel and Turkey.
• ARCT-032 Dosing -- Participants in the study receive 10 mg of the therapeutic candidate daily via inhalation for a period of 12 weeks.
• Cystic Fibrosis Data Timeline -- Management plans to present full Phase II data for ARCT-032 in the second quarter of 2027, with top-line data available before that presentation to inform a Phase III decision.
• LUNAR 2.0 Potency -- The next-generation delivery technology demonstrated a 38-fold improvement in potency compared to the original LUNAR formulation in nonhuman primate models for ornithine transcarbamylase expression.
• ARCT-2601 Dosing -- Phase II dosing for this messenger RNA therapeutic for OTC deficiency is planned to initiate in the first quarter of 2027.
• OTC Deficiency Data Timeline -- Top-line data for the ARCT-2601 Phase II study is expected in the second half of 2027.
• Research and Development Expenses -- $17 million for the quarter, a decrease from $23.3 million reflecting reduced manufacturing and clinical activities across the LUNAR-COVID and BARDA programs.
• General and Administrative Expenses -- $10.5 million for the quarter, remaining consistent with $10.4 million in the prior-year period.
• PKU Program -- Management plans to submit an Investigational New Drug application for a LUNAR 2.0 formulated therapy for phenylketonuria in the second half of 2027.
• ARCT-810 Clinical Results -- The candidate demonstrated biomarker activity at 0.3 mg/kg and 0.5 mg/kg doses, reducing fasting ammonia and maintaining mean glutamine concentrations within the normal range during treatment.
• CSL Seqirus Settlement -- The company received a $12 million payment during the first nine months of 2026 as part of the termination and settlement agreement with CSL Seqirus.
• Japan Vaccine Commercialization -- KOSTAIVE, a self-amplifying mRNA COVID vaccine, is being distributed in Japan for the 2026 to 2027 season through partner Meiji.
• European Marketing Rights -- The company completed the transfer of Marketing Authorization Holder rights for KOSTAIVE in the European Union and the United Kingdom in Sept. and Oct. 2026.
• Thermo Fisher Collaboration -- A decision to advance ARCT-032 into Phase III would trigger financial contributions from Thermo Fisher Scientific under the existing agreement.
• Acquisition Completion -- The company finished the acquisition of myNEO, integrating AI-driven antigen design and target identification capabilities into its mRNA platform.
• ARCT-2601 Age Range -- The amended Phase II protocol for the OTC deficiency candidate will evaluate patients aged 12 years and older.
• Cash Usage -- Cash and cash equivalents decreased by $46.1 million during the first nine months of 2026, primarily due to ongoing clinical development activities.

SUMMARY

Arcturus Therapeutics Holdings Inc. (NASDAQ:ARCT) reported third-quarter results including revenue of $18.3 million and a cash balance of $184.8 million. Management stated that the company regained strategic control of its vaccine portfolio, which includes the COVID vaccine KOSTAIVE, while maintaining a cash runway extending through year-end 2028. The company reported operational activity in its cystic fibrosis program, with enrollment in Cohort 4 expected to finish by year-end 2026. Management noted the completion of the myNEO acquisition to incorporate AI-driven antigen design into its development pipeline.

• Chief Medical Officer Cohen noted that the company is monitoring cystic fibrosis data for safety and has observed patients successfully complete the full 12-week dosing regimen at 10 mg.
• CEO Payne stated, "In this nonhuman primate model, we demonstrated a 38-fold improvement in potency using LUNAR 2.0 compared to the original LUNAR formulation that we used in ARCT-810."
• Management indicated that the decision to advance ARCT-032 to Phase III remains under the company's control, with a potential timeline for regulatory engagement in the second half of 2027.
• Payne stated, "Beyond OTC deficiency, LUNAR 2.0 is enabling us to expand our liver therapeutics pipeline into additional genetic diseases, including phenylketonuria or PKU and gout."
• The company confirmed that top-line data for the cystic fibrosis program will be available to inform the Phase III decision prior to the full data presentation in Q2 2027.
• Chief Financial Officer Mulroy stated, "We continue to maintain a strong balance sheet and a cash runway of over 2 years through year-end 2028."

INDUSTRY GLOSSARY

• LUNAR: A proprietary lipid-mediated delivery technology used to transport messenger RNA into target cells.
• sa-mRNA: Self-amplifying messenger RNA, a technology designed to enable the body to produce therapeutic proteins over an extended period from a lower dose.
• OTC Deficiency: Ornithine transcarbamylase deficiency, a rare genetic disorder of the urea cycle that causes toxic ammonia to accumulate in the blood.
• PKU: Phenylketonuria, an inherited disorder that causes the amino acid phenylalanine to build up in the body due to a deficiency in the PAH enzyme.
• IND: An Investigational New Drug application submitted to the Food and Drug Administration to initiate clinical trials in humans.
• MAH: Marketing Authorization Holder, the entity that holds the legal rights to market a pharmaceutical product in a specific jurisdiction.
• BARDA: The Biomedical Advanced Research and Development Authority, a U.S. government office responsible for the development of medical countermeasures.

Full Conference Call Transcript

Operator: Hello, and welcome, everyone joining today's Arcturus Therapeutics Third Quarter 2026 Earnings Call. [Operator Instructions] Please note this call is being recorded. [Operator Instructions] It is now my pleasure to turn the meeting over to Neda Safarzadeh, Vice President, Head of Investor Relations, Public Relations and Marketing. Please go ahead.

Neda Safarzadeh: 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 Dennis Mulroy, our Chief Financial Officer. Dr. Pad Chivukula, our CSO and COO; and Dr. Alan Cohen, our Chief Medical Officer, will join them for the Q&A session. Before we begin, I would like to remind everyone that the statements made during this call regarding matters that are not historical facts are forward-looking statements within the safe harbor provisions of the Private Securities Litigation 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 this statement. Please see the forward-looking statement disclaimer on the company's press release issued earlier today, as well as the Risk Factors section in our most recent Form 10-K and in subsequent filings with the SEC. In addition, any forward-looking statements represent our views only as of the date such statements are made. Arcturus specifically disclaims any obligation to update such statements. And with that, I will now turn the call over to Joe.

Joseph Payne: Thank you, Neda. Good afternoon, everyone, and thank you for joining us today. The third quarter was an important period for Arcturus as we continued to advance our key clinical programs, introduced our next-generation LUNAR 2.0 delivery platform and expanded our pipeline of mRNA therapeutics. I'd like to begin with our liver therapeutics franchise, where we believe we are entering a new, exciting phase of development. In September, we presented encouraging Phase II data from ARCT-810 in patients with ornithine transcarbamylase or OTC deficiency.

At the 0.3 and 0.5 milligram per kilogram dose levels, ARCT-810 demonstrated meaningful biomarker activity reducing and/or maintaining first morning fasting ammonia within the normal range, including during increased protein intake and reducing glutamine levels in all participants with the 0.5 milligram per kilogram dose achieving mean glutamine concentrations within the normal range during treatment and was generally safe and well tolerated. These results provide important clinical validation of our mRNA approach to liver therapeutics and contributed to the development of our next-generation OTC program called ARCT-2601. ARCT-2601 is particularly significant for Arcturus because it is the first mRNA therapeutic candidate utilizing our new LUNAR 2.0 delivery technology.

Nonhuman primate models have consistently demonstrated strong translational predictivity for lipid nanoparticle formulated RNA therapeutics. This is well documented. And in nonhuman primates, we can measure human ornithine transcarbamylase expression. And in this nonhuman primate model, we demonstrated a 38-fold improvement in potency using LUNAR 2.0 compared to the original LUNAR formulation that we used in ARCT-810. We believe this substantial improvement in delivery efficiency has the potential to enable lower and less frequent dosing and potentially broaden the range of diseases we can address with our mRNA therapeutics. We shared the LUNAR 2.0 data with the FDA.

Following favorable FDA feedback and clarity on the regulatory pathway from our Type C meeting in June, we now plan to initiate Phase II dosing of ARCT-2601 next quarter. The amended protocol will evaluate patients with OTC deficiency ages 12 and older, and we expect top line data in the second half of 2027. Central to our thesis is safe and effective delivery of mRNA. We worked in the field of RNA therapeutics where delivery is everything as -- so you can understand why we are looking forward to seeing this first potential clinical top line data for ARCT-2601 as this is the first opportunity to confirm how the 38-fold improvement we observed in nonhuman primates translates into patients.

Beyond OTC deficiency, LUNAR 2.0 is enabling us to expand our liver therapeutics pipeline into additional genetic diseases, including phenylketonuria or PKU and gout. For PKU, we are developing an investigational LUNAR 2.0 formulated mRNA therapy designed to enable the liver to produce functional PAH. This is the enzyme that is deficient in patients with PKU. Our objective is to restore PAH activity, improve phenylalanine metabolism and ultimately address the underlying cause of the disease. We currently plan to submit an IND for this program in the second half of 2027. Turning now to our respiratory franchise. We continue to make progress with ARCT-032 in CF.

Enrollment in Cohort 4 of the Phase II study has accelerated as we have activated additional clinical sites in Israel and Turkey. We expect enrollment to be completed by year-end with full Cohort 4 data planned for presentation in the second quarter of 2027. Cohort 4 is evaluating 10 milligrams of ARCT-032 administered by inhalation for 12 weeks in up to 20 adults with cystic fibrosis, primarily Class I CF subjects. We are also pleased with the continued support of Thermo Fisher Scientific for the ARCT-032 program.

Under our collaboration, a decision to advance ARCT-032 into Phase III would trigger significant contributions from Thermo Fisher, with that decision expected when top line data is available before our planned Phase II full data presentation. With the ongoing bolus of patients enrolling in Turkey and Israel, the data generated from these patients may be included to inform that decision. Thermo Fisher receives regular updates in accordance with our agreement. And per the agreement, the decision to advance ARCT-032 into Phase III remains under Arcturus's control. During the quarter, we also continued to strengthen our broader technology capabilities and AI infrastructure. We completed the acquisition of myNEO, adding AI-driven capabilities in antigen design, mRNA optimization and target identification.

We believe integrating these capabilities with our existing mRNA design and delivery expertise can accelerate pipeline development and create additional strategic opportunities, including oncology, given myNEO's 7 years of experience expertly designing neoantigens. On the vaccine side of our business, KOSTAIVE, our self-amplifying mRNA COVID vaccine, continues to be commercialized in Japan through our partner, Meiji, for the 2026-2027 season. We also successfully completed the transfer of the marketing authorization holder rights for KOSTAIVE in both the European Union and the United Kingdom from CSL Seqirus to Arcturus. These transfers are an important step as we move toward executing commercial objectives for KOSTAIVE in 2027.

We also recently published the results of our Phase I study for our self-amplifying mRNA pandemic influenza vaccine in Nature Communications. The study demonstrated immunological benefits compared with standard of care, a favorable safety profile and the potential for a single-dose pandemic response. Taken together, we believe these developments in our vaccine enterprise demonstrate the breadth of our mRNA platform and the evolution of Arcturus. We now have important programs progressing across respiratory and liver therapeutics, a next-generation delivery technology with LUNAR 2.0, an expanding pipeline in genetic diseases, additional AI-enabled discovery capabilities through myNEO and a commercial vaccine platform led with KOSTAIVE. Looking ahead, we have several important milestones approaching.

We expect to complete enrollment in the ARCT-032 Phase II study by year-end, present the full Phase II CF data in the second quarter of 2027, initiate Phase II dosing of ARCT-2601 in the first quarter of 2027 and report top line ARCT-2601 data in the second half of 2027. Our focus remains on disciplined execution and advancing the programs where we believe our technology can deliver meaningful differentiation in patients and long-term value for shareholders. With that, I'll turn the call over to Dennis to review our third quarter financial results.

Dennis Mulroy: Thanks, Joe, and good afternoon, everyone. Our press release issued earlier today includes financial statements for the 3 and 9 months ended September 30, 2026, and provides a summary and analysis of year-over-year performance. Please also reference our Form 10-Q for more details on our financial performance. Cash and cash equivalents were $184.8 million as of September 30, 2026. and $230.9 million on December 31, 2025, for a decrease of $46.1 million during the first 9 months of 2026.

The decrease was primarily driven by cash used for ongoing clinical development activities, including the continued advancement of the company's cystic fibrosis and OTC deficiency programs, partially offset by $12 million payment received from CSL Seqirus under the termination and settlement agreement. Revenue was $18.3 million and $23.3 million for the 3 and 9 months ended September 30, 2026, compared to $17.2 million and $74.8 million in the comparable periods last year.

The increase in revenue for the 3-month period was primarily driven by the recognition of the $12 million termination payment and the remaining deferred revenue upon termination of the CSL Seqirus collaboration, under which the company regained strategic control of KOSTAIVE and its broader vaccine portfolios, partially offset by lower grant revenue. The decrease in -- of the 9-month period was primarily attributable to revenue recognized under the CSL Seqirus collaboration prior to the termination and lower BARDA grant revenue. Research and development expenses were $17 million and $56 million for the 3 and 9 months ended September 30, 2026, compared to $23.3 million and $87.7 million for the corresponding periods in 2025.

The decreases were primarily driven by reduced clinical and manufacturing activities, particularly across the LUNAR-COVID and BARDA programs, together with lower personnel and facility costs as the company continues to advance its highest priority programs, while maintaining a disciplined approach to capital allocation. General and administrative expenses were $10.5 million and $30.9 million for the 3 and 9 months ended September 30, 2026, compared with $10.4 million and $32.1 million in the comparable period last year. Overall, general and administrative expenses remained relatively consistent for the quarter and the year-to-date decrease primarily driven by lower payroll and benefit costs and lower share-based compensation expense, all resulting with lower headcount and particularly offset by increased legal and professional fees.

Net loss was $7.4 million and $58.2 million for the 3 and 9 months ended September 30, 2026, or net loss per share of $0.26 and $2.05 compared to a net loss of $13.4 million and $36.7 million for the 3 and 9 months ended September 30, 2025, or a net loss per share of $0.49 and $1.35 per share. We continue to prioritize disciplined investment in the programs and technologies that we believe will create the greatest long-term value. We're excited with the recent acquisition of myNEO and working with their talented team as we incorporate their strong expertise in our future strategic relationships.

We continue to maintain a strong balance sheet and a cash runway of over 2 years through year-end 2028, allowing the company to reach important clinical and regulatory milestones in our mRNA therapeutic pipeline. With that, I'll pass the call back to Joe.

Joseph Payne: Thank you, Dennis. As you can hear, Arcturus is entering an important period with multiple potential, value-creating data milestones across our pipeline with a strong balance sheet and a disciplined approach to capital allocation. So with that, operator, we're ready to take questions.

Operator: [Operator Instructions] We will take our first question from Lili Nsongo with Leerink Partners.

Lili Nsongo: So maybe a couple of questions related to the cystic fibrosis program. Can you remind us how many patients have been dosed to date? I know you mentioned that you expect completion of dosing by year-end 2026. And I think the target enrollment was 20 patients. But in terms of the go/no-go decision along with top line data, should we expect that to be at a minimum of 12 weeks after the completion of the cohort? Or do you plan on presenting maybe a smaller data set first?

Joseph Payne: Thanks, Lili, for the questions. Yes, first of all, the cohort size remains consistent. It's approximately 20 people and up to 20 subjects, primarily Class I CF individuals. The bolus of these have been coming out of Israel and Turkey, and we highlighted that. We haven't disclosed exactly the number that we've recruited to date, but we have shared our confidence in completing that enrollment by year-end. With respect to the data presentation, this is intended to be a full data set of all -- approximately 20 of these subjects when we complete enrollment, the entire fourth cohort. So it will be a fulsome data set, that presentation we've guided now in Q2.

Lili Nsongo: I'm sorry. And just to confirm, the -- it will also be a full data set for the top line data that will guide a go/no-go decision?

Joseph Payne: Yes, exactly. Well, the go/no-go decision, that is expected when top line data is available, and that will be available before likely our planned Phase II full data presentation in Q2.

Operator: We will move next with Seamus Fernandez with Guggenheim Securities.

Boran Wang: This is Evan Wang on for Seamus Fernandez. Just I guess, 2 for me. One is a clarification on cystic fibrosis. So with the planned presentation in 2Q, is that referring to a medical meeting if you're talking about a separate top line readout? And then secondly, on the OTC program in 2601, curious if you've kind of -- any more color you can provide on the design there, evaluation of 1 or 2 doses, dose levels? And now that you've kind of guided to top line data in the second half, is that like a full data set of like 3 to 6 patients? Curious, anything you can share there?

Joseph Payne: Sure. So we haven't disclosed the exact method of the presentation in Q2, but it will likely be at conference. So stay tuned for that. With respect to the 2601 data set, yes, we want to make sure that we have sufficient data to give us some initial confidence into the proof-of-concept of 2601 so that we can share that data, coupled with the ARCT-810 data at the EOP2 meeting after we collect that initial data set. Now Alan, are you available to comment further?

Alan Cohen: Yes, Joe, I'm on the call. So just to align and reemphasize what Joe was just saying, as it relates to the 2601 data set, the plan is to enroll upwards to 6 subjects. And the plan would be to have the full data set in hand, in combination with the 810 data, so that we could present the totality of that data at an end of Phase II meeting with the FDA and then based on that data and what we believe we've observed and achieved, use that to construct and design a Phase III program moving forward.

Boran Wang: If I could ask one follow-up on cystic fibrosis, just kind of clarify when top line press release data may be then?

Joseph Payne: Top line data, all we've indicated it will be before -- for CF, yes, the top line data will be available prior to the full data presentation. Or you're talking about the OTC data?

Boran Wang: I was talking about cystic fibrosis but...

Joseph Payne: Yes, the CF data, yes. The CF data, top line data will be before the full data presentation.

Operator: Our next question comes from Yanan Zhu with Wells Fargo.

Kuan-Hung Lin: This is Kuan on for Yanan. So on the CF program, given that this is an open-label study, I assume the company is monitoring the data in real time. So can you share with us whether you remain confident that 12 weeks would be sufficient to capture consistent benefits across patients?

Joseph Payne: Yes. We're definitely confident that the 12 weeks is sufficient, but -- And with respect to limiting real-time access to the data, Alan, maybe you can comment on both of those questions.

Alan Cohen: Sure. So just to reemphasize Joe's response, we're confident that if there's going to be an ascertainable clinical signal, that 12 weeks should be sufficient using clinically relevant measures such as changes in percent predicted FEV1 based on spirometry and changes in MVW via lung clearance index, as well as the 2 quality of life measures and the changes in HRCT over that 12-week period. In terms of the fact that it's open label, we are spending a lot of time making sure that the only people that have access to that open-label data right now are the medical people, including myself. And the access to that is really primarily for safety monitoring, not efficacy monitoring at this time.

We don't want to have that data inadvertently influence how we proceed with the study and how we collect the balance of the remaining data set. So right now -- and I can say that we have had patients make it through all 12 weeks at the current regimen of 10 milligrams, once daily by inhalation. So we're confident that based on the data that we've collected thus far that we'll be able to continue to enroll and collect that data in its totality ideally by the end of this calendar year.

Kuan-Hung Lin: Got it. That's super helpful. And one quick question on OTCD. So based on your interactions with FDA-810 so far, do you think there is a clarity on a potential pivotal endpoint for OTCD program in general? And does 2601's preclinical data have any implication on the likelihood of success on that endpoint?

Joseph Payne: Yes. With respect to the likelihood of success, obviously, we are very optimistic in light of the 38-fold improvement we saw in primates and that this will translate positivity -- positively into human data. But with respect to the endpoint clarity on 2601, maybe you can elaborate, Alan?

Alan Cohen: Sure. If history serves as an appropriate reference point, and we believe that it does, given that the drugs that are currently approved for OTC deficiency, a focus on stabilizing and bringing down serum ammonia levels, we certainly know that the biomarkers that we have, observed changes in that we believe are clinically relevant and important. Both reductions in glutamine levels and reductions in ammonia levels will undoubtedly be the focused area that the FDA will want to be able to see continued improvements and changes in over time. As you'll recall also, we had patients that liberalized their dietary intake of protein.

So we were also able to observe that in the context of patients taking in more protein, they were still able to have reductions and stabilization in their glutamine and ammonia levels through the balance of the data set that we collected thus far. So those are going to be the focused areas of attention that we believe the agency will want to continue to see demarcations for clinical efficacy and will undoubtedly serve as relevant primary and secondary endpoints for a Phase III study of this kind.

Operator: Our next question comes from Myles Minter with William Blair.

Myles Minter: First one is just on 2601. I think you've previously said that the patients you'd enroll in that cohort would have elevated ammonia levels at baseline. And I know the patients you treated with 810 prior to that have had relatively controlled ammonia levels at baseline. So just wondering how that different patient population might change your expectations for data there? And then the second one, Joe, you mentioned oncology. I'd be amiss not to ask, are you going to become a personalized neoadjuvant vaccine company?

Joseph Payne: Sure, sure. How about if I address the cancer question first, and then Alan, you can talk about the elevated ammonia question for 2601. But yes, Arcturus is focused on executing on our mRNA therapeutics pipeline. I want to make that clear. However, we do have a technology and capabilities that can enable key players in the oncology space. With the recent acquisition of myNEO, we now have capabilities of neoantigen design, and we have clearly a differentiated sa-mRNA platform that is better at engaging T cells, for example, and proven mRNA delivery and manufacturing. So for these reasons, of course, we're always open to enabling key players in oncology.

But I also want my message to be equivalently clear that we're focused on executing on our mRNA therapeutics pipeline this year. And Alan on to you.

Alan Cohen: Yes. And let me just try to elaborate on your question relevant to ammonia levels and glutamine levels in the study patient populations. Despite the best efforts by patients under their current standards of care, where they're significantly restricting their protein intake and most, if not all of them are on ammonia binding agents like RAVICTI. Despite their best efforts, more often than not, there are patients out there that are walking around with elevated ammonia levels on the high end of what would be considered the normal range. But as a result of that, don't feel well. They have mental cloudiness and fog. They have GI upset, they have headaches, et cetera.

What we're going to try to do in the 2601 study is within the audience of patients that we have the pleasure of accessing for this trial, we're going to try to prioritize those patients whose ammonia levels and glutamine levels are running on the higher end so that the deltas can be as pronounced as possible. But we recognize that right now, if we can just show continued reductions and stabilization of those levels, particularly as we go down in the age groups over time, the problem with the stable adults is just quality of life and functionality and their ability to just take in normal protein amounts during the day.

For the kids, it's these unexpected spikes that they sometimes experience with very high ammonia levels, which cross the blood-brain barrier and cause irreversible neurological damage. So as we go down in the age ranges from stable adults into adolescents into the younger kids, certainly, the kinds of things we're going to be looking for and the stability of those changes over time will become increasingly important. But you're correct. We're going to be continuing to go into patients for whom, despite their best efforts, their baseline levels are running on the higher end of things so that we can ascertain stability and changes and reductions for both glutamine and ammonia over the study period.

Operator: Our next question comes from Whitney Ijem with Canaccord.

Whitney Ijem: Congrats on the progress. I -- Sorry to go back to this, but I just want to try to triple clarify the CF data timing. So just to make sure I understood, is it that -- you've now said the go/no-go decision will be based on the top line data that will be available before 2Q. Previously, the go/no decision was expected before year-end. So does that imply that the top line data is expected in the fourth quarter of this year? Just want to be really clear on that.

Joseph Payne: Yes. No, we've provided very clear goalposts here with the completion of enrollment at year-end and a full data presentation guidance for Q2. With respect to when the decision is expected, we have to recognize a couple of things. One is top line data available and what does that include. We've already indicated that top line data by definition is before our planned full data presentation. But the ongoing bolus of patients that we're enrolling in Turkey and Israel, that data generated from these patients may be included to inform that decision. It doesn't have to be, but it may be. And we just want to keep that above ground and open for everybody.

And so the timing of that data, we haven't guided the specifics, but when we have it, it will provide us the opportunity to share and to provide that decision.

Whitney Ijem: Okay. Got it. So not -- the go/no-go decision might not happen before year-end? Might...

Joseph Payne: We have not indicated -- the only new guidance that we've provided today is the data presentation. When the team was out at the bank conference season in September, we learned. We heard feedback they would like guidance on data presentation timing. And so that's what we've added.

Whitney Ijem: Okay. That makes sense. I just wanted to be clear on that. Okay. And then just my second question was just on the plans for KOSTAIVE in U.S. and Europe. I think there was language about kind of moving towards commercialization. Next year is that something you plan to do on your own? Or will you be seeking a partner?

Joseph Payne: Yes. Well, now that the market authorization holders have been shifted from CSL Seqirus to Arcturus, that provides us an opportunity to engage in commercial activities -- additional commercial activities outside of Japan in 2027. With respect to U.S., we've been consistent that we would pursue a partner for the U.S. region. But we've indicated that since the separation agreement of CSL, it provides us the opportunity to explore commercial activities next year in Europe and the United Kingdom. And now we have the opportunity to do so. That is correct.

Operator: We will move next with Adam Walsh with ROTH Capital Partners.

Adam Walsh: The first one is on the CF. If there is a go decision there, what would be the main hurdles to starting a Phase III? And could that be done in 2027?

Joseph Payne: Yes. Alan can comment on that. But -- yes, go ahead, Alan.

Alan Cohen: Yes. The goal -- as Joe has just been laying out the timeline. So let's just go through it real quick. We're going to enroll upwards to 20 patients in the U.S., Israel and Turkey through the balance of this calendar year, so by the end of December. It's a 12-week study. So -- And then we have to do data lock on last patient out and then analysis. And some of the endpoints that we're looking at like high-resolution CT scans take a little bit longer than things like spirometry.

So we would want to have all that data in hand before we sit down with the agency to have a conversation about based on the data that we've seen and the access to the data that we intend to have available, if needed, from the CF Foundation from the REACH study, which is the normative data set that you've heard us talk about and that they're continuing to complete their data set and analysis on right now, the likelihood would be that we would have a conversation with the agency in the second half of next year.

And then the likelihood of starting a study, we could probably have a protocol written and ready to go, but the ability to start that by, let's say, late December, I think it would be speculative at this point to put a flag in the ground and say absolutely, but we would certainly want to move the program forward based on positive data in a population for whom standards of care are just simply not available. So there's a sense of urgency for this patient population. I think the agency is consistent in that feeling. And the CF Foundation, of course, is advocating strongly for rapid, stealth, thoughtful studies that can be done efficiently and quickly.

So we would certainly do everything we can to try to get a study started as soon as possible. I think it would be a push to say end of 2027, but I certainly think that if it's feasible, we would go for it.

Adam Walsh: And my question -- second question is just also on CF. You've talked a little bit about the safety and kind of having the open-label trial, you're able to see the safety data. You said previously that if there were any very serious adverse events, you would publicly disclose them. And you've also said that patients have -- some patients have finished the full 12 weeks. That sounds pretty encouraging given the fact that competitor programs all failed on safety and tolerability. If you could just maybe give some thoughts and color around where we stand on safety, that would be helpful.

Alan Cohen: Sure, of course. I'm unable, of course, to speak to anything. I think what you're referring to is the most recent mentioned, by Vertex in a recent quarterly earnings report that they had stopped advancing their own messenger RNA LNP program. And I believe they clarified that it was for largely safety concerns. What we are aware of in that study was that in order to continue to conduct that trial, they were actually requiring things like systemic steroids on a daily basis in a population that has pancreatic insufficiency already and has problems with blood glucose levels. They were also using moderate dose inhaled long-acting bronchodilators and inhaled corticosteroids on top of that.

Fortunately, we've not needed to do that in our protocol. We're just using a bronchodilator, which most of these patients use daily as part of their standard of care for airway clearance. And we only have them doing that for the first 2 weeks. And then if they don't need the additional pre-dosing before self-administration at home, they can stop it. Also, the other programs were unable to proceed with home administration.

And our understanding was that they were requiring towards the end of their program before they discontinued it that patients were needing to come in daily for administration in the clinic setting, which obviously would be incompatible with a drug that's inhaled and that needs to be administered on a daily basis. Once again, we're not having those problems. So currently, what I can say and what I will reiterate is that of the patient population that we've currently enrolled, we have patients that have successfully completed all 12 weeks.

We also have not had any severe significant safety events which the Drug Safety Monitoring Committee would either put stipulations into our ability to advance it or would ask us to stop the program or we would need to discontinue it. We have not had any of those triggers or any of those events thus far. So what I can say is that we are continuing to successfully enroll this trial as planned. And we have interested patients across the globe, which we are actively continuing to screen and enroll with the hope of getting the full data set and patient data set in by end of calendar year. Hopefully, that answers your question.

Operator: Our next question comes from Mayank Mamtani with B. Riley Securities.

Mayank Mamtani: I appreciate the level of detail. One more, if I may, on 032, sorry, if you could clarify what proportion of already dosed patients or those you plan to enroll at the year-end -- you're planning -- you've already been at the 15 mg escalated? Or are the -- is the entire 20 patient cohort now going to be at the 10 mg dose and you don't need the escalation? And then maybe just answer that and then I have a follow-up.

Joseph Payne: No, the -- yes, we anticipate the entire 20 subjects will be primarily Class I CF subjects and all of them will have received 10 mgs daily for the 12-week period, just for clarity there.

Mayank Mamtani: Okay. Great. And then, Alan, on the REACH study learnings, if you're able to speak to any magnitude and consistency of what the annual FEV1 or LCI data you're learning from there? And if there's a opportunity to use that as an external control versus doing a placebo-controlled trial in this population as the Phase III, I don't know how challenging that is?

Joseph Payne: Yes. With respect to the REACH study, I'll start there and pass it over to -- the mic over to Alan. But we got a detailed update on the REACH study from the CF Foundation in October at the NACFC. And it was good to hear from them that there's hundreds of participants in this REACH study over a wide age span and includes multiple lung function parameters, FEV1 and LCI, which we're also collecting in the present study. So this is all very encouraging. But Alan, I'll turn the time over to you to elaborate further.

Alan Cohen: Yes. I -- And in addition, so obviously, the REACH study is being run and is completely under the control of the -- both at the CF Foundation and the academic physicians that are working with them, who were kind enough to meet with us during the time of the NACFC meeting in Atlanta in October and at least give us an update as to how they're progressing. Right now, what I can say is that they're -- they've done an excellent job, and there's been very high interest across the CF community to want to participate and contribute to this natural history study.

And this was intended, as you know, to make a natural history database available, which doesn't exist in the literature. So that sponsors like ourselves could have access to it and use it when sitting down with the FDA to allow us to ideally do a smaller number of patient studies and use it as a potential natural history control so that the relationship of placebo versus active drug arm could be minimized. Also, the number of patients could be minimized.

The CF Foundation is also working on novel approaches for statistical directions for how these studies might be able to be conducted, recognizing that there is really a relatively small patient population that can be accessed and that would be interested in participating in the Phase III study. So the good news. The good news is that they're progressing. The data set looks to be useful. It will be supportive. And we hope to be able to have access to that in the second half of next year around the time that we would be planning engagement with the FDA with our end of Phase II data.

But obviously, a lot of things are going to need to happen between now and then. But right now, we're continuing to coordinate and work in a collaborative way with the CF Foundation and the academics running that study so that if and when we need to have that information in hand to help us with our negotiations and discussions with the agency on steps moving forward, that we will have it in our pocket so that we'll be able to reference it and use it to this end.

So hopefully, that gives you a little bit more color and flavor for the timing and the expectations and the hopes of what we plan to do with it as well as other sponsors.

Mayank Mamtani: Super helpful. And lastly, on the 2601, has the GLP-tox study read out internally? And maybe what starting dose levels you're submitting as part of the protocol as you get ready to file IND, that would be helpful.

Joseph Payne: Yes. With respect to 2601, we're preparing to file the IND here very shortly. We just have to include our the final GLP-tox studies into that report and send it off. We've already indicated with clear guidance that we plan to initiate dosing in Q1 or next quarter for 2601. There's no other activities that we need to highlight. Pad, anything you want to add or?

Padmanabh Chivukula: No. And again, the starting dose, we have not disclosed that, but it will be a dose that we've previously tested with 810.

Operator: Our next question comes from Thomas Shrader with BTIG.

Thomas Shrader: Really to return to 2601 dosing. I mean 6 patients is not a lot. Do you feel like you've have good agreement with the FDA? I assume you're going to try to crosswalk to the big 810 data set. Do you have good agreement? You've had very positive interactions, it sounds like -- do you have good agreement on what you need to show, what you need to match to say you understand dose -- Any color you could provide?

Joseph Payne: No, Yes, we had a pair of Type C meetings to discuss this program and a significant discussion on LUNAR 2.0 specifically, and we incorporated that into this IND filing. So yes, we have a high degree of confidence, but that's what the IND process for, to formalize that. Once that's formally approved to proceed, then we can provide more details pertaining to the protocol. With respect to our confidence on the small level of patients, this is a luxury that you have when you're utilizing a program that's potentially 38x better. And that can be determined -- it's not like 2x better, 3x better. We have to power the study significantly.

And it also takes into consideration that this is simply a reformulation of ARCT-810. And so that played a key role in making this a relatively smooth process. But did I address your question, Tom? I wanted to make sure.

Thomas Shrader: Yes. I mean, that makes sense. It is the falloff and stuff should all be the same. It's the same protein. It's only the sort of the starting level. One quick vaccine question. I mean a single-dose pandemic vaccine is pretty exciting, kind of what the world needs. Do you think, is there still public money for something that has RNA in the title? Or just any thoughts on how promising you could get a BARDA grant or something to move what seems on paper a fantastic vaccine?

Joseph Payne: Yes. Although our focus is on mRNA therapeutics, we do acknowledge the nondilutive monies associated with business development type deals in the vaccine enterprise. And one of those type of deals that is more relevant is pandemic preparedness contracts and deals. And yes, we're sitting on a technology that's ideal for the next pandemic and helping countries prepare for that is something that we'll continue to provide that opportunity to different countries going forward. And you're spot on, self-amplifying mRNA, yet another example of a single administration to protect against pandemic bird flu, like that's significant data that was quietly shared today. And hopefully, it doesn't become relevant in the future.

But it does showcase the breadth of our platform and the capability of self-amplifying mRNA, not only in infectious disease vaccines, but also into oncology, which is a hotter topic now in the mRNA field.

Operator: We will move next with Yale Jen with Laidlaw & Company.

Yale Jen: I just want to go back to the earlier question regarding the natural history study by CF Foundation, I understand which is not completed yet. But at this time, with the data you have already seen as well as to your knowledge about the current study you are conducting, does that -- put those 2 together, does that increase your confidence for the potential success of the 032 can make -- study at this moment? And then I have a follow-up.

Joseph Payne: That's the entire purpose of the REACH study is to enable sponsors, like the CF Foundation is trying to help companies like Arcturus get drugs approved for Class I CF. This is a completely untreated population, and we're first movers in this area, and they're trying to help us. And the CF Foundation came up with this REACH study to help us for this purpose to provide some sort of baseline control, a normative study on what happens to Class I subjects over time, and natural history like study.

And so the short answer to your question is yes, this is going to very likely help us, especially if we're fortunate to advance this into a Phase III study, then it can be leveraged at that time even more so. So the short answer is yes.

Yale Jen: Okay. Maybe one follow-up here, which is the PKU program that you have not talked too much about. Should we be able to see some preclinical data maybe over the next period of time and start to let investors understand a little bit more about that program as well?

Joseph Payne: Well, as part of the -- we indicated today that we're guiding an IND filing for the PKU program in the second half. As part of that process, there's a copious amount of preclinical data that's included in the filing. And they will provide opportunities for us to share that. But maybe do you want to provide some color, Pad?

Padmanabh Chivukula: Yes. Again, a few weeks ago, during the Science Day, I think we did present a little bit of data about our optimization process in terms of RNA design. Once we select, obviously, the candidate, we will be doing a more thorough study, looking at duration, looking at activity. And at some point, yes, absolutely, we will be sharing that publicly as well.

Joseph Payne: And the one thing I'd like to add, I'm glad you asked that question in closing because I just want to commend Pad and his team. It's not just innovations around the delivery technology, but extending the half-life of -- and the durability of expression of the mRNA molecule, too. So there's a lot of innovation at Arcturus that we're very proud of, and I wish we had more time to highlight that on this call. But -- So thank you for that question.

Operator: And at this time, there are no further questions in queue. I will now turn the meeting back to Joe for closing remarks.

Joseph Payne: Thanks, everyone, for participating on the call. Don't hesitate to reach out to our team for any remaining questions. We'll get back to you as soon as we can. Thanks again.

Operator: Thank you. This brings us to the end of today's meeting. We appreciate your time and participation. You may now disconnect.

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