Earnings call: Arcturus Therapeutics reported a net loss of $6.9 million

Arcturus Therapeutics (NASDAQ: NASDAQ:ARCT) shared updates on its financial status and progress in its product pipeline during the Q3 2024 earnings call on November 14, 2024. CEO Joe Payne and CFO Andy Sassine discussed the company's first commercial product launch, KOSTAIVE, in Japan and its financial results.

Despite a reported net loss of $6.9 million for the quarter, an improvement from the previous year, the company remains optimistic about its future, with a cash runway extending into Q1 2027 and promising developments in its mRNA therapeutic programs.

Key Takeaways

• Arcturus launched KOSTAIVE, its first commercial product, in Japan.
• A $25 million milestone payment was received following KOSTAIVE's first sale.
• The European Medicines Agency (EMA) is close to giving a decision on KOSTAIVE, with a CHMP opinion expected in December 2024.
• Arcturus plans to file a Biologics License Application (BLA) for KOSTAIVE in the U.S. in H1 2025.
• Interim Phase 2 data for ARCT-032 and ARCT-810 are anticipated in early 2025.
• Q3 2024 revenues were $41.7 million, down from $45.1 million in Q3 2023.
• The company's cash reserves stood at $294.1 million as of September 30, 2024.
• Arcturus secured around $462.1 million in upfront payments and milestones from CSL (OTC:CSLLY) and expects further payments.

Company Outlook

• Arcturus is focusing on the commercial revenue potential of KOSTAIVE in 2025.
• The company is working with Meiji Seika Pharma to facilitate domestic manufacturing of KOSTAIVE in Japan.
• Arcturus is optimistic about the strategic importance of its manufacturing partner ARCALIS for mRNA production, especially for the CF program.

Bearish Highlights

• The company reported a net loss for the quarter, although it was an improvement from the previous year.
• Revenues declined compared to the same quarter in the previous year.

Bullish Highlights

• The company's mRNA therapeutic programs, ARCT-O32 and ARCT-810, are progressing well, with interim Phase 2 data expected in H1 2025.
• Arcturus has a strong cash position with a runway extending into Q1 2027, excluding potential revenue from KOSTAIVE.

Misses

• There was a decrease in revenue from Q3 2023 to Q3 2024.

Q&A Highlights

• The management is confident in the timeline of the CF program and the strategic importance of manufacturing capabilities.
• Specifics about manufacturing orders from partners will be disclosed by the partners themselves.
• The company did not disclose specific numerical goals for improvements in FEV1 for CF patients.

Arcturus Therapeutics' management team remains confident in their strategy and the potential of their mRNA technology, particularly in the CF and vaccine markets. With several key milestones on the horizon, the company is poised to make significant advancements in the coming year.

InvestingPro Insights

Arcturus Therapeutics' (NASDAQ: ARCT) recent earnings call paints a picture of a company in transition, with its first commercial product launch and ongoing development of mRNA therapeutics. To complement this information, InvestingPro data and tips provide additional context for investors.

According to InvestingPro data, Arcturus has a market capitalization of $519.48 million, reflecting its position as a smaller player in the biotech space. The company's revenue for the last twelve months as of Q2 2024 stood at $163.87 million, with a significant quarterly revenue growth of 373.99% in Q2 2024. This aligns with the company's narrative of transitioning towards commercialization, particularly with the launch of KOSTAIVE in Japan.

However, InvestingPro Tips highlight some challenges. The company currently suffers from weak gross profit margins, which is evident in the reported gross profit margin of -18.43% for the last twelve months. This negative margin underscores the financial hurdles Arcturus faces as it scales up its operations and brings products to market.

On a positive note, an InvestingPro Tip indicates that Arcturus holds more cash than debt on its balance sheet. This corroborates the company's statement about having a cash runway extending into Q1 2027, providing some financial stability as it pursues its development pipeline.

The stock's performance has been volatile, with a one-month price total return of -16.6% as of the data's timestamp. This volatility is consistent with the company's current phase, where investor sentiment can be heavily influenced by clinical trial results and regulatory decisions.

For investors seeking a more comprehensive analysis, InvestingPro offers additional tips and metrics. In fact, there are 10 more InvestingPro Tips available for Arcturus Therapeutics, which could provide deeper insights into the company's financial health and market position.

Full transcript - Arcturus Therapeutics Holdings Inc (ARCT) Q3 2024:

Operator: Good day, everyone, and welcome to today's Arcturus Therapeutics Third Quarter 2024 Earnings Conference Call. At this time, all participants are in a listen-only mode. Later, you will have the opportunity to ask questions during the question-and-answer session. [Operator Instructions]. Please note this call may be recorded, and I will be standing by if you should need any assistance. It is now my pleasure to turn the conference over to Ms. 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 Andy Sassine, our CFO. Dr. Pad Chivukula, our CSO and COO, 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 Legation 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 present 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. It's good to be with you again, everybody. We look forward to providing our updates today on our quarterly investor call. I will begin my remarks with an update on progress with our vaccine franchise led by KOSTAIVE, our self-amplifying mRNA COVID-19 vaccine. We're thrilled about our recent commercial launch of KOSTAIVE in Japan. Last month, members of our senior management team, including myself, had the wonderful opportunity to travel to Japan to be vaccinated with KOSTAIVE in Tokyo. In addition to our team from Arcturus, we shared this experience with senior management from Meiji, CSL, and Arcalis. As you can imagine, it was an experience our team will never forget. In connection with the first sale of our COVID-19 vaccine, Arcturus received a $25 million commercial milestone. On the regulatory front, CSL Secures' partner in Japan, Meiji Seika Pharma, announced earlier this year that they submitted a partial change application for an amendment to the manufacturing and marketing approval of KOSTAIVE to include domestic manufacturing sites in Japan, including Arcalis. An Arcalis is Arcturus' manufacturing joint venture in Japan. When approved, this will allow for Meiji Seika Pharma to begin selling domestically produced KOSTAIVE this season. The European Medicine Agency continues to review the KOSTAIVE marketing authorization application. I've been impressed with how our team has worked diligently with the agency as they review the first potential self-amplifying mRNA product in Europe. The process is near completion with the CHMP opinion expected next month. As we look forward to achieving marketing approval in the U.S., we plan to file a BLA for KOSTAIVE in the first half of next year, which will be supported by positive results from multiple Phase 3 studies. We continue to collect meaningful clinical data for our proprietary next generation STARR mRNA platform. The company announced today another set of positive Phase 3 results wherein ARCT-2303, this is the monovalent XBB variant derivative of KOSTAIVE, met all four primary study objectives and key secondary objectives. The study supports co-administration of KOSTAIVE with licensed influenza vaccines. ARCT-2303 demonstrated superior immune response versus ARCT-154 as measured by neutralizing antibodies against Omicron XBB.1.5.6 in terms of geometric mean titer or GMT ratio and a seroconversion rate or SCR difference. Co-administration of ARCT-2303 and cell-based quadrivalent influenza vaccine showed non-inferior immune response versus standalone QIV administration. Co-administration of ARCT-2303 and QIV also showed non-inferior immune response versus standalone ARCT-2303 administration. And lastly, co-administration of ARCT-2303 and adjuvanted QIV in older adults showed similar responses versus standalone administration of ARCT-2303 and adjuvanted QIV. In September, the company along with our partners, CSL, Seqirus, and Meiji, announced new 12-month post-vaccination data for KOSTAIVE at Options 12 for the Control of Influenza Conference. The results of a head-to-head Phase 3 study demonstrated that KOSTAIVE maintained superior immunogenicity compared to the conventional mRNA vaccine COMIRNATY for up to one year against Wuhan-Hu-1 and Omicron, BA.4 and 5 and certain other variants, and at one-sixth of the dose of the comparator. These results were published in the Lancet Infectious Disease. Additional Phase 3 data presented at the Options Conference showed that bivalent KOSTAIVE, also known as ARCT-2301, induced superior immunogenicity over conventional bivalent mRNA vaccine COMIRNATY that persisted against key variants up to six months post-vaccination. Now shifting our attention to our mRNA therapeutics franchise, let's begin with an update on ARCT-032. ARCT-032 is an inhaled messenger RNA therapeutic for cystic fibrosis, and it's formulated with Arcturus' lunar delivery technology that differentiates us from our competitors. In September, we received clearance of an investigational new drug application to the U.S. Food and Drug Administration. The FDA clearance of the IND application enables Arcturus to initiate a Phase 2 multiple ascending dose study to evaluate the safety, tolerability, and efficacy of ARCT-032 in people with cystic fibrosis. Our team is actively engaged in onboarding a substantial number of clinical sites to help us in this effort. We are fortunate to be able to be working closely with the CF Foundation in this process. The Phase 2 study is presently screening individuals with CF who do not qualify for or benefit from CFTR modulator medicines due to dysfunctional or absent CFTR protein and/or drug intolerance. This study will allow us to evaluate FEB lung function improvement in individuals with CF. And I'm very pleased to report that the company is on track to share in-term Phase 2 proof of concept data for our CF program in the first half of 2025. I'll now move on to our ARCT-810 program. This is our messenger RNA therapeutic candidate for Ornithine Transcarbamylase or OTC deficiency. Earlier this year, Arcturus announced the expansion of the Phase 2 clinical program of ARCT-810 into the United States. This open label multiple dose study evaluating pharmacodynamics and safety is currently enrolling adults and adolescents requiring clinical management for OTC deficiency. Our placebo-controlled Phase 2 European study has completed the dosing phase. So these concurrent Phase 2 studies in Europe and the U.S. will allow us to evaluate meaningful biomarker changes in individuals with OTC deficiency. And I'm happy to report that the company is on track to share in-term Phase 2 proof of concept data in the first half of 2025. With that, I'll now pass the call to Andy.

Andrew Sassine: Thank you, Joe, and good afternoon, everyone. The press release issued earlier today includes financial statements for the third quarter of 2024 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 details on the financial performance. We are very pleased with the launch of KOSTAIVE, our COVID-19 vaccine candidate in Japan. This represents an important milestone for Arcturus as it is the first commercial product in the company's history. We believe that this product highlights the differentiating aspect of samRNA technology and how it can potentially represent an improved vaccine option for patients. I am also happy to announce that we have received a $25 million commercial milestone with the first KOSTAIVE sale in Japan. I also went to Tokyo last month to get the KOSTAIVE vaccine with 32 executives from Arcturus, Arcalis, CSL, and Meiji. Due to the early clinical success of our cystic fibrosis program, Arcalis has become a strategic manufacturing asset for Arcturus and therefore we have decided not to sell our stake in Arcalis at this point in time. The strategic review process conducted by JP Morgan generated interest from financial and strategic participants which will benefit Arcurus and Arcalis in the future. We decided to expand our manufacturing product line with Arcalis to include respiratory mRNA therapeutics and therefore we are planning to transfer our cystic fibrosis manufacturing process technology to Arcalis. I will now provide a summary of our financial results for the third quarter of 2024. For the three months ended September 30th, 2024, we reported revenues of $41.7 million, a slight decrease from the $45.1 million reported in the same period in 2023. This small decrease is attributable to a decrease in CSL revenue as we achieved a milestone of $35 million during Q3 of 2023 compared to a milestone of $25 million during Q3 of 2024. This was offset by an increased revenue from the BARDA agreement. Total (EPA:TTEF) operating expenses for Q3 2024 were $52.4 million compared with $64.5 million for Q3 2023. Total operating expenses for the nine months ended September 30th, 2024 were $191.8 million compared with $195.9 million for the nine months ended September 30th, 2023. Research and development expenses with $39.1 million for Q3 2024 compared with $51.1 million for Q3 2023. The decrease was primarily due to $15.9 million in manufacturing expenses incurred in the Q3 2023 related to the Meiji supply agreement and other clinical trials manufacturing batches as well as decreased facilities and equipment expenses. The decreases were primarily offset by a $3.6 million increase in clinical trial related expenses for the COVID and flu programs. For Q3 2024, our tourists reported a net loss of approximately $6.9 million or $0.26 per diluted share compared with a net loss of $16.2 million or $0.61 per diluted share for Q3 2023. Cash, cash equivalents and restricted cash were $294.1 million as of September 30th, 2024 and $348.9 million as of December 31st, 2023. Arcturus achieved a total of approximately $462.1 million in upfront payments and milestones from CSL as of September 30th, 2024 and expects to continue to receive future milestone payments from CSL supporting the ongoing development of the COVID and flu programs and three additional vaccine programs by CSL. Based on the current pipeline and programs, the cash runway is expected to extend into the first quarter of fiscal year 2027 and does not include any contribution from the sale of KOSTAIVE vaccines in Japan. In summary, the company remains in a strong financial position and has the cash runway needed to achieve multiple near-term value creating milestones for the vaccine and therapeutic program. Furthermore, with the recent launch of KOSTAIVE in Japan, we look forward to reporting potential commercial revenues in 2025. I will now pass the call back to Joe.

Joseph Payne: Thanks, Andy. We've continued to make exceptional progress on our mRNA vaccines and therapeutics pipeline. We are particularly excited about the launch of KOSTAIVE, the first commercial product in the company's history. And we're also pleased that both of our flagship mRNA therapeutic programs, ARCT-O32 and ARCT-810 are on track for interim Phase 2 POC clinical data in the first half of 2025. I will now turn the call to the operator for Q&A.

Operator: Thank you. [Operator Instructions]. We'll go first to Lili Nsongo with Leerink.

Lili Nsongo: Hi. Good afternoon, and thank you for taking the question. I guess two questions from my side. So, first question regarding the commercial launch of KOSTAIVE in Japan. Anything you could give us maybe a little more granularity. So you had mentioned that 4 million doses had been delivered to Japan through partner Meiji, that they are also upping production. I was wondering if you could give maybe a little more color on the launch trajectory in Japan and expectations for the fall -- for the winter season going into 2025. Secondly, so for the OTC deficiency study, so the European study has completed for a while now, and so the U.S. study is ongoing. Would you mind giving maybe a little more color on the number of patients and type of data we should expect in the upcoming readout in the first half of 2025?.

Joseph Payne: Thanks, Lili, for the question. Andy, with respect to the additional nuance on the commercialization process in Japan, do you want to handle that question?

Andrew Sassine: Sure. Thanks, Lily. We're pretty excited about working with Meiji in Japan because, as you know, they're the number one flu vaccine company, and so they're in a very strong position to be able to launch the product very effectively. And if you may have paid attention into a recent press release, they've actually articulated that they were planning to sell roughly 4.5 million vaccines during the season with their guidance. And of course, we only shipped 4 million. And of course, the remaining vaccine must have come from the opportunity to produce them in ARCALIS in Japan. So we're all awaiting the announcement of that approval by the PMDA shortly, which should enable Meiji to have a full launch by probably December or so, with a vaccine that's actually made in Japan, so pretty exciting, I think, for not only Meiji and CSL, but the Japanese people overall. So we're not really pretty to give specific guidelines other than what they've been able to articulate publicly, but I would closely pay attention to any communications coming from Meiji and CSL regarding the launch and progress of the vaccine sales in Japan.

Joseph Payne: And pertaining to the OTC question, Lili, the U.S. expansion is going to be similar in size relative to what we did in Europe. We are enrolling younger and more advanced disease subjects. We did see some early signals that were encouraging in the European trial, but we're going to be combining this data in what we share in the first half of next year.

Lili Nsongo: Thank you.

Joseph Payne: Thanks Lili.

Operator: We'll go next to Yasmeen Rahimi with Piper Sandler.

Unidentified Analyst: Hey, good afternoon, team. This is [Indiscernible] for Yas. Thanks for taking our questions. First, to the extent that you can, for the Phase 2 cystic fibrosis study, could you provide any color on the size and cohorts that you're thinking about? What doses are you planning to move forward with? And then for the second question, what is needed to demonstrate proof of concept in your view?

Joseph Payne: Pad, do you want to address that question, the CF study?

Pad Chivukula: Yes, I mean, obviously, if you look at -- we are going to be looking at what a lot of our competitors are doing in terms of looking at the various biomarkers. And we can't discuss a lot about the actual study design. And our plan is to, again, recruit. We finished our Phase 2 study. We plan to start our study in the U.S. and we'll provide more data around our CF study later on this year, I mean, early next year.

Joseph Payne: Yes, multiple doses are going to be evaluated in the CF study. It's an open-label study. There's no bronchoscopy included in this study. And FEV is going to be measured throughout the study. But with respect to specific time points and additional details, there'll be an appropriate time for us to share that. And that'll be at a later time.

Unidentified Analyst: All right. Thank you.

JosephPayne: Thanks.

Operator: We'll move next to Whitney Ijem with Canaccord Genuity.

Whitney Ijem: Hey, guys, thanks for taking the question. First, I just wanted to follow up on the commentary around vaccines in Japan and the switch to ARCALIS once it's approved. Is the idea that when ARCALIS is approved and it's manufactured domestically in Japan, that there will be kind of a step up or an acceleration, just as we think about modeling quarter-over-quarter next year? And then the second question, to follow up on OTC, I think looking back, the interim Phase 2 data had originally been expected in the fourth quarter. So maybe I missed it. But what drove the shift to the first half of next year? Thanks.

Joseph Payne: Andy, do you want to address the first question?

Andrew Sassine: Sure, Whitney. No, I think you were spot on there with that assessment. And as you can tell, Meiji and ARCALIS are very proud to obviously be able to manufacture the first samRNA vaccine in Japan. And it was pretty evident by the response and the press that were all in attendance. They had over 30 press officials there. So a pretty well attended press conference. And certainly, I think, you know, they would probably prefer to launch and articulate that this vaccine is made in Japan. And certainly having shipments from ARCALIS in December will enable them to articulate that more clearly. So with respect to probably a more aggressive commercial launch, you could probably anticipate, it would happen in December, January timeframe, in my opinion. So if you're looking at the timing of the revenue, it probably would happen in the, you know, probably the first two quarters of next year.

Joseph Payne: And with respect to the OTC data, we did complete dosing in Europe, and there was some early evaluation of some of that data. The data is not locked, and we initiated the enrolling process in the U.S. prior to that process. So we thought it was wise to just couple these together and provide the interim data update in the first half of next year.

Whitney Ijem: Understood. Thanks.

Joseph Payne: Thanks.

Operator: We'll move next to Evan Wang with Guggenheim Securities.

Evan Wang: Hi, guys. Thanks for the question. Just a few from me. Firstly, on KOSTAIVE, anything you can share on broader vaccination trends in Japan? So not just KOSTAIVE-specific. I know the Japanese season starts later, but has this been in line with, you know, Meiji's estimate to support the dose totals for the season? And also on KOSTAIVE, you highlighted kind of recognizing revenue in 2025. Can you remind us some of the reporting here and how Arcturus recognizes some of this revenue? And then third, on OTC and specific viruses, I'm just wondering, it's great to see some timelines for proof of concept in the first half of 2025. I'm just wondering what gives confidence in some of these timelines. Is dosing and recruitment thus far better than expected? Any additional color there would be helpful. Thanks.

Joseph Payne: Sure. Let's see, you had a question about revenues. Do you want to address that one first, Andy?

Andrew Sassine: Sure. As you can see, when Meiji will sell the vaccines in Japan, they will be reporting those sales to CSL on a quarterly basis. CSL then will in term determine the allocation of the profit share between CSL, Meiji and Arcturus. At that point in time, we'll be able to recognize those revenues once that allocation is contributed to Arcturus. And keep in mind that we do have to offset the initial revenue by the 40% of the production cost that we are responsible for in the development of the program. And so that amount has not been communicated officially, but you can assume that that would probably incur at least a few million doses before you're able to offset those initial 40% of the development and production costs for the KOSTAIVE vaccine. I hope that was helpful.

Joseph Payne: And addressing your second, or questions about the KOSTAIVE trend and the CF timeline. I can comment that the team was in Japan and we got a really good feel for Meiji's presence in Japan in the vaccine industry. They have a large sales force. They have approximately 40% of the flu shot business in Japan. So it was really good to see what kind of materials they're providing to just a large number of physicians in Japan, as you can imagine. So, clearly there's a educational phase of the launch teaching people about this next generation technology, but any additional details than that, it would be more appropriate to just for them to provide with their regular updates with respect to commercial guidance. But all I can say is that I was very impressed with the management team and the commercial staff there that they really knew what they were doing. With respect to the CF timeline, the reason we're very comfortable on this is, first of all, if you notice the design of our trial is, I would say, open label. It's not placebo controlled. There's no bronchoscopy involved or lung brushing, which can deter participants from participating. And we also have some early data that we've already shared in Phase 1B, including a Class 1 subject that had some early and promising data. So I think that data collection is helping us. We're also working with the CF Foundation. And as we're onboarding a substantial number of sites, I think it's given us encouragement with these preliminary conversations that we should be well on track to deliver some data in the first half of next year.

Operator: We'll move next to Myles Minter with William Blair.

Myles Minter: Hey, thanks for taking the questions. Three quick ones if I may. The first one is just on the guidance that you'd get the EMA approval milestone from CSL for potential approval of KOSTAIVE in the first quarter of '25. Does that imply that you'd expect to see a chimp opinion issued at either the November or December meeting by the end of this quarter? First one. The second one is on the decision to keep the ARCALIS equity stake. Does that mean if Meiji does give additional manufacturing orders of cost save to ARCALIS, that's something you could actually report on rather than just pushing back to Meiji for commercial guidance? And the third question is moving the CF program manufacturing to ARCALIS and having a U.S. focus Phase 2 clinical trial, does that mean you have to get FDA inspection of that facility? Thanks very much.

Joseph Payne: Andy, do you want to provide a first half?

Andrew Sassine: Sure. No, it's -- we were obviously very encouraged by the opportunity with the early progress we've had in cystic fibrosis. So obviously manufacturing and trying to plan strategically a global production base is quite daunting, because just to address, just to give you an idea, just to address the Class 1 population, you're looking at probably 17, roughly, kilograms per year. And so you need to put together a very well orchestrated manufacturing base to be able to address all that. And of course, ARCALIS has now become very strategic because of that opportunity. And consequently, they are a very low cost and very efficient operation, because as you know, the drug substance, drug product and DNA is all made there. And so it's by authorization and the sale finish. So having all that in one location reduces a substantial amount of transportation logistical risk, especially with transferring drug substance to Europe for the drug product completion stage. And so, there are many implications and opportunities. And obviously, as you have just heard, the amount of mRNA we're going to need to make for the CF program is quite substantial. And based on just the capacity ARCALIS has now, that's about 100 million a year just for ARCALIS and sales. And that's only considering two kilograms out of the 17. So just to give you the significant impact of this opportunity is pretty substantial. So we've had to reevaluate our global supply base and working very closely with Aldevron and Danaher (NYSE:DHR), of course, and Catalent (NYSE:CTLT) and Recipharm and Polymun. And so this has taken on a very global concerted effort. It's exciting, but it's going to take a little bit of work. And certainly it makes ARCALIS a much more valuable partner right now, and we need them. And we're kind of excited about working more closely with them on the CF program.

Joseph Payne: And pertaining to EMA approval, we've clearly guided today that we're anticipating a CHMP decision in December. And that obviously precedes a formal approval process shortly thereafter that takes us into Q1. So I think your assumption is fair. Did we address all your questions, Myles?

Myles Minter: Just a quick one on the second one. Just with the manufacturing orders that could come to Meiji when the PMDA issues approval in December, if they do that, is that something you, as Arcturus, would be able to report on if they do indeed receive a bulk manufacturing order for KOSTAIVE? Thanks.

Joseph Payne: Yes. The short answer is no. But Andy, yes, go ahead.

Andrew Sassine: We really can't comment on those, because that'll be up to Meiji and CSL to articulate that. Of course, we'll give you as much color as we can post quarter, and hopefully that'll enable you to have a better insight as to the ramp-up and the success of the Arcalis production on a quarterly basis.

Myles Minter: Awesome. Appreciate you taking all the questions. Thanks.

Joseph Payne: Thanks, Myles.

Operator: We'll move next to Yanan Zhu with Wells Fargo (NYSE:WFC).

Unidentified Analyst: Hi, thanks for taking our question. This is Quan [ph] for Yanan. So I have a question on your CF program. Can you share with us your barcode for success on FEV1? And also, Vertex (NASDAQ:VRTX) Moderna (NASDAQ:MRNA) will report their VX-522 phase 1 with data also in first half '25. So can you remind us the differentiation of 032 versus VX-522? Thank you.

Joseph Payne: Yes, it's definitely a wonderfully competitive area. And I think this is great for patients in general in the CF community. But we do have key differentiation elements to our program and our technology compared to our competitive peers. So thank you for the question. The first and foremost, we have a different delivery technology that we call Lunar. But this not only has a different registered trademark, but it's a chemically different lipid nanoparticle that is biodegradable, non-accumulating. And we believe these chemical differences have proven out to provide differentiated data pre-clinically. So we have pre-clinical data in the ferret model that has shown that we have a very significant response that supersedes positive control after a single administration. I think that would be representative of the differentiation I'm speaking to. We're also sharing data more visibly. We've already provided some phase 1B data and a class 1 subject that has provided some promising early response after just two administrations. And then finally, a purification IP, I think, is a differentiator. And this is a big deal in the therapeutic space. When you're dosing chronically and larger amounts of mRNA, it's very important, especially in compromised lungs, like the CF patient population, that these mRNA molecules are substantially pure. And we have a potentially leadership position in this space. And with intellectual property behind it, it's different. How we purify our mRNAs is likely different than our competitors. But I'll stop there. I could talk for a while. But I think those are the key differentiators.

Unidentified Analyst: Right. Thanks for the colors. And would you mind sharing your barcode success on FEV1?

Joseph Payne: Did you say our expectations around FEV1? What was your question?

Unidentified Analyst: Right. Yes.

Joseph Payne: Because we're addressing the patient population where there's substantial unmet medical need. Again, these are the non-modulator responders. That's about 15% to 18% of the CF population. So we're going after these folks that do not really have an excellent treatment option. So the barrier for entry, the bar that we need to establish for lung function improvement, I believe, is very small. We haven't provided any details on that. And with respect to our conversations with regulatory agencies, for example, we hold those cards close to our chest. But the short answer is anything measurable, I believe, would be very significant for this patient population. A small percentage improvement would be very meaningful, in our view. But we haven't given that specific number yet. There will be an appropriate time later down the road.

Unidentified Analyst: Yes, thank you for that. And one quick question on 2303. Congrats on the data. Can you share with us what's the next step for the program? Are you ready to file, or what's your strategy? Thank you.

Joseph Payne: 2303. The strategic purpose for these other Phase III trials was just to showcase the breadth of the platform, that the technology can be multi-antigenic, for example, in the bivalent trials we're doing, and also in where we're conducting these trials, is meaningful because we're collecting an expanded safety database in multiple ethnicities around the world with these additional Phase III trials. Taking that all together, it helps beef up and support a really strong BLA application in the first half of next year. So they're strategically important to support the BLA application in the U.S. I don't foresee us marketing these products. The data is used to support the platform of KOSTAIVE in the United States.

Unidentified Analyst: Got it. Thank you for all the colors.

Joseph Payne: Thank you, Quan.

Operator: We will move next to Pete Stavropoulos with Cantor Fitzgerald.

Samantha Schaeffer: Hi, this is Samantha Schaeffer on the line for Pete. Thanks for taking our question. Can you touch on the H5N1 pandemic flu program? If you could remind us on key details for this non-CSL partner program and what to expect. Thanks.

Joseph Payne: Thank you. The short answer to that question is we remain on track to get into the clinic this year. So thank you for the question. H5N1 is definitely important to BARDA. We do have some BARDA references in our filing documents and our press release and probably the script as well. You can see that we're elevating our relationship with them. But that stage, that stage of getting into the clinic is coming up here shortly.

Samantha Schaeffer: Thank you.

Operator: We'll move next to Ed Arce with H.C. Wainwright.

Unidentified Analyst: Hi, everyone. This is Thomas [Indiscernible], of course. Congratulations on your progress today. So first question, wondering what the Phase 2 data readout for [Indiscernible] and OTC expected in first half next year. Which efficacy measurement do you believe has the potential to support approval endpoints, or which efficacy endpoints should investors focus on?

Joseph Payne: That's actually a really good question. The data we're collecting is not only important to establish proof of concept for intravenously dosed mRNA in our platform, but a key part of the OTC strategy is to identify the appropriate biomarker if we choose to advance this into a pivotal trial or a phase three trial. So it's not just the data that's important in this first half of 2025, but understanding which of these many biomarkers that we're collecting data on, many of them. And we believe we have a strategy that is going to be appropriate for a phase three or pivotal trial. But we won't be communicating specifically what that biomarker strategy is today. That's something that we can do concurrently with the interim data sharing in the first half of next year.

Unidentified Analyst: Got it. And then for the other program, I do have this partner with CSL. Are there any updates with the Lunar flu program? I believe last we heard this Phase 1 [Indiscernible]?

Joseph Payne: Yes, good question. Our CSL collaboration in the flu is a very active one. I'll say that. Its multiple programs are involved. The funding for these programs has been increasing. We're meeting regularly in JSC meetings with CSL. But with respect to the cadence of data sharing and any sort of commercial strategy on these, we respectively agree with CSL that will allow them to provide that information. So all we can say with respect to the flu program is that it's very active. There's multiple programs and that funding is increasing for these programs. And it's definitely a priority for our collaboration. But again, how the data is shared and the cadence of that data and any sort of commercial strategy and what it's combined with and the bundling commercial strategies, these kind of things we can't refer to at all. But thanks for the question.

Unidentified Analyst: Got it. One more question from us, this one first, Andy. Just wonder, what's the entire 25 million milestone from UG? Was that entire amounts recognized in third quarter or it's going to be spread over several quarters?

Joseph Payne: Yes, the 25 million when it's recognized, go ahead, Andy, yes.

Andrew Sassine: Yes, thank you, Joe. Yes, the 25 million is going to be reported just like all of our other development milestones at this point in time. And the SC-606 requires that we probably amortize around 90% to 93% of the milestone in the quarter that it was earned. And then the remaining amount is amortized over a production to complete method. And so that's why you see the accruals on some of the CSL revenues that occur on a recurring basis in our quarters. So hopefully that gives you a perspective of what we've recorded here in the quarter.

Unidentified Analyst: Got it. Thank you for taking all the questions. Looking forward to upcoming progress with KOSTAIVE in Japan.

Joseph Payne: Thank you.

Andrew Sassine: Thank you.

Operator: We'll move next to Yale Jen with Laidlaw & Company.

Yale Jen: Great, thanks for taking the question. My first question is that giving the three parties will be involved in terms of determining the allocations. So should we anticipate any potential royalty from revenue from Japan will be something of next year instead of the last quarter of this year?

Joseph Payne: Yes, timing of revenue recognition. Yes, go ahead, Andy.

Andrew Sassine: Yes, Yale. I think if you listen closely to what we were articulating, by the time Meiji reports the sale to CSL and then they will in turn determine the allocation, you're probably better off assuming that the sales will be recognized in the first half of next year rather than this year. So hopefully that gives you some perspective because remember we also have the 40% of the production costs that we have to absorb before we're able to recognize any revenues. So I think I would prefer to caution on the conservative side and probably anticipate the first half of next year, the better predicament of when we can see some of those revenues. Hopefully that helps you.

Yale Jen: Yes, it does. And maybe just to tag on one more question, at least on the P&L side. I noticed that this quarter's R&D expenses was lower than the sort of prior two quarters. I understand you have changes from the last years, but should we anticipate this a little bit lower R&D expenses will continue or how should we think about that?

Andrew Sassine: That's a good question. And one of the reasons why it's so difficult to give quarterly guidance because of the functionality of when trials are completed and when inventory is shipped. So that's why I prefer to provide a runway guidance, and so if you can be reassured that we had restated that our guidance is in the first quarter of '27, so that's remained consistent now for a few quarters. So you can assume that our burn is going to be somewhere around 100 million a year. And if you divide that by the cash we have, that should give you some comfort that we should be easily achieving that first quarter of '27 goal. And keep in mind that it doesn't include any revenue contributions from Japan. So hopefully we'll be able to update the market next year on that progress.

Yale Jen: Great, and maybe the last question here in terms of 2303 combined with the QIV, how should we think about that going forward in terms of these combined vaccines? Would that be something that the CSL will make a decision on the specific?

Joseph Payne: Exactly, yes, yes. We now have the phase three data to show that non-inferiority or equivalence, right? So CSL will be determining anything to doing with commercial strategy and especially with the flu, they provide that, whether it's combos or co-promotions or bundling, anything like that, that'll all come from them.

Yale Jen: Okay, great, thanks a lot and congrats on the progress.

Joseph Payne: Yes, thank you.

Andrew Sassine: Thank you.

Operator: We'll move next to Yigal Nochomovitz with Citi.

Yigal Nochomovitz: Hi, guys, I hope you can hear me okay. Just first of all, could you just clarify 25 million milestone from Japan? Is that counts receivable and or is it actually your cash report at the end of the quarter? Thanks.

Joseph Payne: Yes. Go ahead.

Andrew Sassine: Yes, no, good question. We give CSL about 60 days to pay the bill. So, and they've been a pretty good customer. So I'm not too worried about getting that $25 million. Hopefully that alleviates your concern about them paying us.

Yigal Nochomovitz: Okay. And then on the manufacturing, I'm just wondering, so you're saying that you're going to transfer the manufacturing on the CF over there. The capacity is left at our college after including CF on top of COVID. And do you have, do they have the capability there to do the fill and finish for the specialization of the CF product given that it's going to be the nebulized product? Is there anything else that they need to incorporate into the manufacturing chain to do that piece of things?

Joseph Payne: No, it's a short answer, no. Go ahead.

Andrew Sassine: Yes, we can go ahead, let Pad answer that if he has any more color on the production.

Pad Chivukula: Yes, again, the process for making our drug substance for both our CF product as well as our vaccine products are very similar. So that's the great beauty about messenger RNA. I think we can use the very similar process for all the APIs and all of our programs that we're currently having internally. There's obviously nuances related to a lyophilized product or a frozen drug product. And I think that there is some specific and the components, exact components that are in our CF product are different than what we're currently using for our vaccines. So because of that, there is a know-how or a text transfer process that we have to undergo. But we're confident that that text transfer process is going to be just fine and they're capable of making our drug substance. And ultimately, you asked a question a little bit about fill finish, they are building out not just drug substance capabilities, but also drug product and final fill finish capabilities as well. And because of that, we're going to be leveraging our partners.

Yigal Nochomovitz: So the product produced for CF in Japan will be the final commercial product, correct?

Pad Chivukula: Yes, that would be the vision of the, yes.

Yigal Nochomovitz: Okay. And then as far as capacity, I guess the question is, what about OTC? Is that something you would consider transferring over there or is there a reason you are deciding not to do that? Is it a capacity question?

Joseph Payne: It's rare to do a small amount, so.

Andrew Sassine: We can handle that with our current partners. Just the demand for the amount of cystic fibrosis, mRNA that's going to be required is pretty substantial as I articulated early, on a year-over-year basis of 17 kilogram, that's a lot of mRNA. And as you know, Arcalis has capacity up to five kilograms right now. So, and so certainly there's an opportunity to potentially expand that as we are able to achieve some clinical success with the CF program.

Yigal Nochomovitz: And then the last one is, so you have the four million doses that you're shipping over there, and then you said another half a million that's going to be made locally in Japan. So are they basically, and then at some point, they're all going to go into the channel and there may be some overlap potentially. Are they all going to kind of look indistinguishable from a labeling, branding perspective?

Andrew Sassine: Well, they all have the same label. Yes, the label is uniform. And so the excitement here is that, ARCALIS is now in the process of producing the KOSTAIVE vaccine. And that's important because as you know, the Japanese government has given Arcalis $165 million to help construct that facility. So it's a very strategic plant, not only for the Japanese people, but the government to protect the people in any future pandemics that should arise. And so that I think is why the people over there are very excited about this opportunity.

Joseph Payne: And one thing to correct Yigal, the four million has already been shipped. I just wanted to make sure that was clear.

Yigal Nochomovitz: Okay. Understood, thank you.

Operator: And with no further questions holding at this time, I'll turn the conference back to Joe Payne for any additional or closing remarks.

Joseph Payne: Hey, thanks everyone for participating on the call. If there's any remaining questions, please don't hesitate to reach out to our team and we'll get back to you as soon as we can. Thanks and good night.

Operator: Thank you, ladies and gentlemen, that will conclude today's program. We thank you for your participation. You may disconnect at any time.

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