Q3 2023 Arcturus Therapeutics Holdings Inc Earnings Call
Okay.
[music].
Speaker 1: Greetings and welcome to the Arcturus Therapeutics 3rd Quarter 2023 Earnings Conference Call. At this time, all participants are in a listen-only mode. A brief question and answer session will follow the formal presentation. If anyone should require operator assistance during the conference, please press star zero on your telephone keypad. As a reminder, this conference is being recorded.
Greetings and welcome to the Arcturus Therapeutics third quarter 2023 earnings conference call at.
At this time all participants are in listen only mode. A brief question and answer session will follow the formal presentation.
Anyone should require operator assistance please.
Zero on your telephone keypad.
Reminder, this call is being recorded it is not my pleasure to introduce your host that as the holiday.
Speaker 1: It is now my pleasure to introduce your host, Nerissa Robinson.
Speaker 1: President, Head of Investor Relations, Public Relations, and Marketing. Thank you. You may close.
Or is it head of Investor Relations public relations and marketing. Thank you you May proceed.
Thank you operator, good afternoon, and welcome to Arcturus Therapeutics third quarter 2023 financial update on pipeline progress.
Speaker 2: Today's call will be led by Joe Payne, our President and CEO , and Andy Satine, our CFO .
Today's call will be led by Joe Payne, our president and CEO and Andy <unk> our CFO.
Speaker 2: Dr. Patrick Vakula, our CSO and CEO , will join them for the Q&A.
First Patrick Nicola our CSO MTO will join them for the Q&A session.
Speaker 2: 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 1992.
Before we begin I would like to remind everyone that the statements made during this call regarding matters that are not just starting cold facts are forward looking statements within the safe Harbor provisions of the private Securities Litigation Reform Act of 1995.
Speaker 2: Forward-looking distinctions 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.
Forward looking statements are not guarantees of performance.
Both known and unknown risks uncertainties and assumptions that may cause actual results performance and achievements could differ materially from those expressed or implied by the statements.
Speaker 2: 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.
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 this date.
Speaker 2: In addition, any forward-looking statements represent our views only as of the date such statements are made.
They are made.
Speaker 2: Arcturus specifically disclaims any obligation to update such a statement. And with that, I will now turn the call over to Joe.
Arcturus has specifically disclaims any obligation to update such statements.
And with that I will now turn the call over to Joe.
Thank you it's good to be with you again everybody.
Speaker 3: I'm going to begin my remarks with an update on progress regarding our monovalent COVID-19 vaccine, ARCT 154.
I'm going to begin my remarks, with an update on progress regarding our model D M and COVID-19 vaccine E. R. C T 154.
Speaker 3: Following favorable clinical results from the phase three pivotal studies, the new drug application is currently under review by Japan's Pharmaceuticals and Medical Devices Agency.
Following favorable clinical results from the phase III pivotal studies the new drug application is currently under review by Japan's Pharmaceuticals, and medical devices agency or the P. M D. A.
Speaker 3: The ARCP 154 Japan NDA submission is supported by an active controlled phase 3 booster vaccine study, which was conducted in Japan, and a placebo-controlled phase 3 primary vaccination series efficacy and safety study, which was.
C. A R. C. T 154, Japan NDA submission is supported by an active controlled phase III booster vaccine study, which was conducted in Japan, and a placebo controlled phase III primary vaccination series efficacy and safety study, which was conducted in Vietnam.
The E. R. C T $1 54 phase III booster vaccine study.
<unk> its pre specified primary endpoint demonstrating the non inferiority of an immune response against the Sars C. O V two ancestral strained as compared to commonality.
Speaker 3: In addition, the superiority of ARCT-154 and neutralizing antibody response against the SARS-CoV-2 Omicron, the A4 slash 5 variant, was also demonstrated as a key secondary.
In addition, the superiority a b or C. P 154, and neutralizing antibody response against the Sars C. O P. Two AMA crime E. Four slash five variant was also demonstrated as a key secondary endpoint.
Speaker 3: Updated preliminary phase three booster data was recently presented at the 11th International mRNA Health Conference in Berlin.
David preliminary phase III booster data was recently presented at the International Hey, Marni Health Conference in Berlin.
Speaker 3: in a heads-up comparison to an FDA-approved monovalent mRNA vaccine.
In our heads up comparison to an FDA approved monovalent mrna vaccine.
Speaker 3: Monovalent ARCT 154 showed multi-fold improvement in durability and multi-fold superior titers of neutralizing antibodies against Omicron BA4 and 5. And this was at the six month.
Monovalent E. R. C. G 154 showed multi fold improvement in durability and multifold superior titers of neutralizing antibodies against Oklahoma crime P. A four and five and this was at the six months post boost mark.
Speaker 3: These phase 3 booster results were consistent with the phase 1 slash 2 booster clinical trial durability data that were collected previously and presented at the 9th ESWI Influenza Conference.
A phase III and boost your results were consistent with the phase one slash two booster clinical trial durability data that we're collecting previously and presented at the ninth E. S. Wi influenza conference in Valencia.
Speaker 3: All of these observed clinical benefits were achieved with the STAR Next Generation mRNA technology, which is administered at five micrograms.
All of these observed clinical benefits were achieved with the star next generation mrna technology, which is administered at five micrograms.
Speaker 3: This is an 83 to 92% lower dose level compared to approved mRNA vaccine.
This is an 83% to 92% lower dose level compared to approved mrna vaccines.
Speaker 3: This lower dose level highlights the potential safety and tolerability benefits of this next generation mRNA vaccine platform.
This lower dose level highlights the potential safety and Tolerability benefits of this next generation mrna vaccine platform technology.
Based on all the clinical data collected to date, we believe that our next generation star mrna platform isn't effective and differentiated vaccine technology.
Speaker 3: Based on all the clinical data collected to date, we believe that the next generation star mRNA platform is an effective and differentiated vaccine technology that may offer a longer lasting immune response relative to the older conventional mRNA platform.
It may offer a longer lasting immune response relative to the older conventional mrna platform technologies.
Speaker 3: Supported by the ARCT 154 clinical data, Meiji Seika Pharma, the partner.
Important by the E. R C to 154 clinical data matrix shakeup Barbara.
The partner of CSL Securities submitted a Japan M D. A to support a R. C to 154 as a primary series and booster vaccine for COVID-19.
Speaker 3: The review of this application remains underway and is on track for approval in December .
The review of this application remains underway and is on track for approval in December.
We filed a marketing authorization application in Europe.
Speaker 3: And we are seeking approvals for ARCG 154 and other measures.
And we are seeking approvals for <unk> to $1 54, and other major markets.
We continue to mature the value and scope of the star next generation mrna vaccine platform by collecting meaningful bivalent vaccine clinical data as well.
Speaker 3: We continue to mature the value and scope of the STAR Next Generation mRNA vaccine platform by collecting meaningful bivalent vaccine clinical data as well.
Speaker 3: We're pleased to report today that the planned enrollment target of 850 participants has been reached in the ongoing phase 3 bivalent COVID vaccine trial, comparing immunogenicity to bivalent commerce.
We're pleased to report today that the planned enrollment target of 850 participants has been reached in the ongoing phase III by the Covid vaccine trial, comparing immunogenicity to bivalent carbon copy.
Speaker 3: The initial top line results of this study are expected in Q1 of 2024, followed by an anticipated PMDA approval in Q3 2021.
The initial top line results of this study are expected in Q1 of 'twenty 'twenty four followed by an unanticipated P. M D. A approval in Q3 2024.
Speaker 3: In summary, we are delighted with the rapid progress we have achieved this year with our Star Next Generation mRNA vaccine platform. We believe ARCT 154 provides clear validation of the broader opportunity for Arcturus's mRNA vaccine and therapeutic programs.
In summary, we are delighted with the rapid progress we've achieved this year with our star next generation mrna vaccine platform. We believe <unk> 154 provides clear validation of the broader opportunity for our tourists as mrna vaccine and therapeutic programs.
Our strategic collaboration with CSL.
Speaker 3: which is Arcturus' exclusive global licensee, is focused on developing and commercializing next-generation mRNA vaccines and continues to make substantial progress.
Which is our tourists as exclusive global licensee is focused on developing and commercializing next generation mrna vaccines and continues to make substantial progress.
Our partner lunar flu program.
Speaker 3: which is also now known as ARCT 2138, continues to progress with funding and operational support from CSF.
She is also now known as a C. T 2138 continues to progress with funding and operational support from CSL lunar.
Speaker 3: Lunar Flu utilizes Arcturus's next generation mRNA platform.
Lunar flu utilizes arcturus is next generation mrna platform.
Speaker 3: And we are intending to initiate a phase one clinical trial, which is expected to begin soon.
And we are intending to initiate a phase one clinical trial, which is expected to begin soon.
Well now move on to a R. C T 810.
Speaker 3: This is our messenger RNA therapeutic candidate for ornithine transcarbamylase, or OTC, deficiency.
This is our messenger RNA therapeutic candidate for ornithine transcribed families or OTC deficiency.
Speaker 3: This investigational medicine is designed to functionally replace the deficient OTC enzyme in the liver, restoring urea cycle activity and preventing metabolic crises that cause neurological damage.
They're saying the investigational medicine is designed to functionally replace that deficient OTC enzyme in the liver restoring urea cycle activity in preventing metabolic crises that cause neurological damage.
Speaker 3: ARCT 810 could reduce the need for ammonia scavengers and ease the rigid dietary protein restrictions that OTC patients face today, thus improving the quality
They are C. P 810, it could reduce the need for ammonia scavengers and ease the Richard dietary protein restrictions that O T C patients face today, thus improving the quality of life for those with the disease.
Speaker 3: ARCT 810 has received orphan drug designation and rare pediatric disease designation from
S E T H tenants received orphan drug designation.
And rare pediatric disease designation from the FDA.
Speaker 3: ARCT 810 is currently being evaluated in two ongoing clinical studies in patient.
A R. C. P. H N is currently being evaluated in two ongoing clinical studies in patients a phase one b study in adults and a multi dose phase two study in adolescents and adults with OTC deficiency.
Speaker 3: Phase 1B study in adults and a multi-dose Phase 2 study in adolescents and adults with OTC deficiency.
The phase one single ascending dose study is being conducted in the United States and has completed dosing of all planned four cohorts and a total of 16 subjects.
Speaker 3: Phase 1B Single Ascending Dose Study is being conducted in the United States and has completed dosing of all planned four cohorts in a total of 16 subjects.
We expect the final database lock to occur later in this fourth quarter of 2023.
Speaker 3: We expect the final database lock to occur later in this fourth quarter of 2020.
Speaker 3: The ARCT 810 Phase 2 study is being conducted in the United Kingdom and Europe , and plans to enroll up to 24 adolescents and adults with OTC.
B R. C. A T. H 10 phase two study is being conducted in the United Kingdom, and Europe and plans to enroll up to 24 adolescence and adults with OTC deficiency.
Speaker 3: The ongoing study evaluates two dose levels and includes up to six biweekly administrations for each.
The ongoing study evaluates two dose levels and includes up to six biweekly administrations for each participant.
We remain committed to the development of a R. C. G HN and we're taking various actions to address the continued challenging enrollment rate in Europe by adding study sites and patient services to improve screening participation.
Speaker 3: We remain committed to the development of ARCT 810 and we are taking various actions to address the continued challenging enrollment rate in Europe by adding study sites and patient services to improve screening participation.
Speaker 3: Updated guidance of interim phase two data is expected in H1 or the first half of 2024.
<unk> guidance of interim phase two data is expected in each one or the first half of 'twenty 'twenty four.
Moving now to our a C T O 32 program.
Speaker 3: ARCT032 is an inhaled messenger RNA therapeutic candidate for cystic fibrosis.
A R. C. T O 32, it gives them an inhaled messenger RNA therapeutic candidate for cystic fibrosis formulated with Arcturus has lunar delivery technology, which has been optimized for bronchial epithelial cell delivery.
Speaker 3: formulated with Arcturus's lunar delivery technology, which has been optimized for bronchial epithelial cells.
We completed enrollment and dosing in a phase one study in New Zealand of 32 healthy subjects across four ascending single dose cohorts.
Speaker 3: We completed enrollment and dosing in a phase one study in New Zealand of 32 healthy subjects across four ascending single dose cohorts.
We look forward to presenting safety and Tolerability study results of this phase one study at an appropriate conference in the first half of 2024.
We're pleased to report that we have initiated enrollment in schedule dosing of the first patient in a phase one clinical study in New Zealand.
It was designed to enroll up to eight adults with cystic fibrosis with each participant receiving two administrations of a R. C. T. R 32.
We are presently guiding internal data in H one 2024.
Our tourists are sincerely grateful for the continued support.
The CF Foundation.
In September the organization agreed to increase its financial commitment to $25 million to advance a R. C. T O 32.
In October 2023, a M. A C. T O 32 received rare pediatric disease designation from the F D. A.
As such they are C. T O 32 achieved FDA approval for a pediatric indication.
This is eligible to receive a priority review voucher or a subsequent marketing application for a different product.
New data was presented at the North American cystic fibrosis conference or they earn a CFC in November this new proof of activity in vivo data was collected with a C. F. A ferret model also known as G 551, D, but ferrets and the study require continuous treatment with the CFT.
Our modulator kalydeco to prevent disease progression.
A single administration of a R. C. T. O 32 showed successful transfection of airway epithelium cells and restoration of Mucociliary clearance about the level maintained with Kalydeco.
And with that I'll now pass the call to Andy.
Thank you Joe and good afternoon, everyone.
Our press release issued earlier today includes financial statements for the third quarter ended September 2023, and.
And then provide a summary and analysis of year over year financial results.
Please also reference our most recent 10-Q for more details on our financial performance.
All currency recently achieved a $35 million milestone for yourself.
The milestone payment will be used to fund development activities for the lunar COVID-19 vaccine program.
C S L.
We are very pleased with the yards to 154, new drug application to the P. M D in Japan, and we believe that this product could represent a highly differentiated vaccine option for cruise ships.
Furthermore, the development and manufacturing plans supporting the arts you to want to bore was carried out in a financially disciplined and efficient manner that leverages multiple external collaboration.
The two E. R. C T 154, three Japanese boosters.
On the product manufacturing related to this collaboration.
Are being funded by made you say cup armor and the Japanese government.
Maybe just like a pharma has an agreement with C. S. L secure whereby <unk> will be responsible for the regulatory approval marketing distribution and sale of the.
Our CET 154 in Japan, as well as coordinating the manufacturing Covid vaccine products with our palace for the Japanese market.
Our call is located in a strategic biomedical research and development hub in Japan.
Poised to become a key player in the global mrna drug manufacturing landscape.
This C. D. M. One is designed to support the production of mrna vaccines as well as our mrna based therapeutics and has already completed the construction of a state of the yard M RNA drug substance manufacturing facility.
To date $165 million has been awarded to our Dallas right.
The Japanese government.
These funds are being used to build M RNA drug substance.
Related drug product capability and to construct a DNA template manufacturing facilities.
We expect this facility to become a leading manufacturer of mrna based vaccines and therapeutics.
Ability to manufacture vaccines within 100 days.
Emerging by Wilkes Graham.
We expect this entity to provide meaningful financial dividend to our company over the coming years due to our substantial equity positions.
We have a great leap.
Appreciative of the Japanese government for their financial support.
I will now summarize our financial results for the third quarter of 2023.
Our primary source of revenue from license and consulting and related technology transfer.
Residential and collaborative payments received from research and development arrangements with pharmaceutical and biotechnology partner.
For the three months ended September 30 of 2023 we reported revenue of $45 $1 million compared with $13 $4 million for the three months ended September 30 of 2022.
Revenues increased by 31.7 million during the remarks that at September 30 of 2023.
As compared to the prior year period.
The increase was primarily attributable to revenue recognized under the collaboration agreement with CSL secure and grant revenue recognized from the agreement with BARDA.
Revenue increased by $93 million during the nine months ended September 30, or 23 as.
That was compared to the nine months ended September 30 of 2022.
The increase was attributable to an increase in revenues of about $133 million primarily related to the collaboration agreement.
Oh sure.
This increase was primarily offset by less revenue.
2022 from other Covid program customers.
Total operating expenses for the three months ended September 30 of 2023 was $64 5 million.
Compared with $50 2 million for the three months ended September 32022.
Our research and development expense consist primarily of external manufacturing cost indeed.
In vivo research studies and clinical trial performed by contract Research organization clinic.
Clinical and regulatory consultants.
And they're all related expenses.
Family related expenses and laboratory supplies related to conducting R&D activity.
R&D expenses were $51 1 million for the three months ended September 30th two.
<unk> 23, compared with 37.7 million than the comparable period last year.
Primarily reflecting increased clinical research and manufacturing costs and personnel related expenses.
General and administrative expenses, primarily consist of salaries and related benefits of our executive administrative legal and accounting function and <unk>.
Professional fees for legal and accounting services.
Well in other general and administrative expenses.
G&A expenses were $13 4 million for the three months ended September 30 of 2023, compared with $12 5 million and the.
Comparable period last year.
The increase resulted primarily from personnel expenses due to increased head count and salary increased travel and consulting expenses is lower.
Rather than increased rent expense associated with the new headquarters facility.
For the three months ended September 30 of 2023, Arcturus reported a net loss of approximately $16 2 million was 61 cents per diluted share compared with a net loss of $35 3 million or $1, 33% per diluted share in the three.
Months ended September 30 of 2022.
Cash cash equivalents and restricted cash were $369 1 million.
September 30 of 2023.
394 million at December 31, 2022.
We have achieved approximately $365 million in upfront payments.
The milestone C S L secure.
Timber 30 of 2023.
Do you expect to continue to receive future milestone payments from CFL that will support the ongoing development of the Covid and flu program and three additional vaccine program like yourself.
Finally, I'm happy to report.
Back to cash runway now extends through the end of 'twenty 'twenty six based on our current pipeline and program.
In summary, we believe the company remains in a strong financial position and has the resources to achieve multiple near term value, creating milestone for the vaccine and therapeutic program.
Furthermore, with the anticipated one five for product approval in December in Japan.
We look forward to beginning to report the country a commercial scale in the next few years.
I will now pass the call. Thanks, Andy we've continued to make excellent progress and advance our proprietary messenger RNA and lunar delivery platform technologies toward later stages in clinical development.
And we're excited about the progress toward our first potential product approval in December with a R. C. T 154.
The achievement would definitely marker.
Critical milestones for the platform for our tourists and so with that we'd like to turn the time over to the operator for questions.
Thank you at this time, we will conduct a question and answer session. If you would like to ask a question. Please press star one on your telephone keypad.
Information tone will indicate your line is in the question queue.
You May press star two if someone like did we.
We move to a question from the queue.
All participants using speaker equipment, it may be necessary to pick up your handset before pressing the star keys.
Again, that's star one at this time, one moment, while we poll for our first question.
The first question comes from gasoline Rahimi with Piper Sandler. Please proceed.
Good afternoon team. Thank you so much for all the updates.
First question is directed to the upcoming Japan approval. Many clients are asking us if we should be expecting any update in terms of the commitment of Japan.
Order for vaccines for 'twenty 'twenty four at the time of the approval. That's question. One question two would love to hear your thoughts on.
Given that the bivalent vaccine enrollment has completed one where you should be expecting data and in terms of the next steps with the program and then third is congrats on the CF program.
Patient dosing are you planning to get to eight patients or is there an opportunity to maybe report data on you know a small cohort in 'twenty 'twenty, four and I'll jump back into the queue and thank you for letting me ask my question.
Hey, yes. Thanks.
With respect to.
The government orders that may be associated with the approval of <unk> to $1 54 major is primarily responsible for collecting or soliciting those orders, whether that's from the government or from the private sector in Japan.
We're unfortunately, we do not have insight into that so I'm unable to address that but clearly an order cannot happen until.
The platform or a particular asset is approved.
So I won't be able to speak to any detail on that with respect to the patients being a.
Recruited to support the phase one b trial for cystic fibrosis, we indicated that the first patient is getting dosed here shortly but we're more than willing to share interim data.
Required the guidance. We've provided is in the first half of next year.
With respect to the safety and Tolerability data those are the phase one b trial for cystic fibrosis now you asked a question about when data for another program.
<unk>.
And this is for the bivalent and so the yeah, the bivalent booster data.
We've guided that.
The bivalent I'm just looking at my notes to confirm yes enrollment is completing a very soon and in fact later this month.
We are gonna be providing data next year. So top line data is expected in Q1, and an anticipated approval or P. M. D. A approval in Q3 of 'twenty four.
Yeah.
Thank you so much I'll jump back in the queue.
Thanks, Bob The next question comes from Myles Minter with William Blair. Please proceed.
Hi, Thanks for the questions I'm just.
Relevant to what <unk> been saying previously which was potential approval, if the monovalent vaccine or not sorry, Bob and you've been more conservative same fourth quarter I think that's panned out nicely, but is there anything else going on there from like a regulatory conversations point of view I know you've shine off the six month durability.
Died and now did the Pam D. I request that because you have it I'm just wondering why it is an optical to life from October to December and maybe that's just the fault of my G. In order for you Ryan, but any clarity there would be great and I've got a follow up.
No just the day 29 data was a prerequisite for the primary endpoint.
Six month data was not however, this data is aware to the regulatory agency and taken into consideration as they.
We look at regulatory approval going forward.
We've guided again that are this.
That approval is in December.
Okay.
And then maybe just.
Sorry was there.
No.
Yeah.
Sorry, maybe just one on the cystic fibrosis program.
Do you have to dose as I say if patients in a stepwise fashion sorry, a single patient would receive two administrations to be monitored for safety. The full clearance to start the next patient or can you get those patients in eight of them at a time and touch them altogether. Thank you.
Yeah, Yeah, we anticipate that as Pat and we anticipate dosing on all the cohort altogether that's correct. Okay. Thanks.
Thanks, Pat Thanks for the question.
The next question comes from seamless Fernandez with Guggenheim. Please proceed.
Thanks for the question so just.
Just a couple of quick ones here in terms of the.
Rationale potential milestones going forward.
Just hoping that you guys can help us understand.
You know the sort of key steps forward from a milestone perspective, whether it be from the Covid program.
You know the flu program or potentially other programs.
Perhaps you know just in percentage terms, maybe not without absolute numbers.
Where some of the key.
Sort of catalyst milestones.
Would really lay out or as a percentage of the terms of C. S. L. Whats possible and in 2024, obviously, we know what the runway is now through 2026 with the existing cash, but it seems like that could be drawn out quite significantly.
In 2024 as more of these programs advance and as we see more of the Covid.
154 applications move through in to other jurisdictions.
More broadly so just trying to get a better sense of the breakdown.
Of those potential milestones and then the second question, just and then I'll jump back in the queue.
One O T. C is there a consideration just given how.
Excruciating frankly that the recruitment has been of this patient population is there just a view that the demand among patients.
It's just really not there or that they're just too hard to find the best indication might be smaller than.
What we thought previously, but just sort of begs the question is it worth it to keep pursuing this indication give.
Given the debt.
The very challenging nature of our recruiting trial. Thanks.
Sure. Thanks, Seamus for the questions first I'll walk through some of the near term milestones this year as you've asked.
So with our internal programs, starting with our OTC deficiency program. We've indicated that the database is being locked relative to the phase one and phase one data for the OTC program.
That's that database locks can occur later this quarter with respect to phase two interim data we were guiding the first half of 2024.
Now shifting to the CF program, where hey, Joe Let me answer that question I think he was referring to the financial milestones and and I think what it is that correct. Seamus you were referring to the financial.
Trying to understand the various programs and and obviously with the cash runway going too okay. That's what I thought.
From the back.
No.
Yeah No no. That's that's that's what I thought the question was pertaining to so we typically don't guide specifically on you know the.
The individual milestone because they're they're frankly, you know very lumpy right and we don't know when they're going to start and initiate a certain you know program and when was the catalyst for that program be achieved right. So and in terms of not disappointing people I'd, rather you know announce those.
Milestones when we achieve them in some more freely you're able to articulate how we were able to accomplish that Pete.
What's going to be critical here as we go forward is the guidance that we gave you with the amount of cash that we have to give you a perspective of kind of what we're burning you know outside of the CSL and the BARDA relationships as well as the contribution from the cystic fibrosis Foundation. So.
If you take basically the number of years and divide it by our past you're gonna come close to about $120 million in Bern.
Role will be to bring that down even more so.
Consequently, you know it if it's the guidance that's going to be critical to understanding the timing of when these milestones come in and they're pretty significant obviously those you know over $1 billion in development milestones spread out over three to five program. So.
Yeah, they're pretty meaningful other than I have significant impact on our operations and you know as we achieved them, we will certainly be able to explain.
Explain how we earn them and why we did in <unk>.
Hopefully that will provide you.
Enough comfort that we are well funded into you know at least for the next three years without any revenue milestone and also none no commercial milestone or revenues are included in our forecast that would be certainly considered supplemental and we will update them.
Market you know assuming you do okay.
Revenues in 2024.
Yeah.
And that gave me so I couldn't give you a little bit more color on the OTC program, you know as you know with rare disease programs.
Typically slower to recruit compared to some of the work that we've done with the vaccines and it's a well known phenomenon and.
Specifically, our OTC trial, well you know he has been conducted in our research centers, which can be slower of course, you know so we've taken.
Quite a bit of action to potentially speed that up to there. There are two key things that we've done it in the near term that's going to help in recruitment and trying to speed. This up is we've upped them and get them in.
The patient experience and we've added a concierge service.
So that Oh, we can pay for all of the all of the patients need them and then we've also implemented a ah Ah patient stipend to recognize for so a lot of their efforts to to be part of these trials. So I think both.
Both of these things and in terms of and also opening up more sites and it is going to help in recruitment in the near term.
Great.
A follow up question to that though is there any concern around the size of the market opportunity.
Or a RCT for through two but just trying to get a better sense of you know.
Usually there is also demand for rare conditions, where there can be benefits. Obviously there are other treatment options out there I'm just wondering if there is an assessment that would make sense as it relates to this program.
Just because with the CF program advancing as it as it would seem like resources might be.
Better spent to bring for other rare disease opportunities. It's just been many many years pursuing that so just.
Just trying to get a sense of how.
How are you guys are thinking.
That's a great question, you know and I think you know Seamus if we did not see the success that.
Horizon pharma was having with the rebel.
I think we would've had a different perspective, but the fact that they were able to generate over $250 million in revenue that will be gone only 500 patients is a very encouraging you know opportunity for us and assuming that Victor you only sequesters ammonia.
It's our mrna therapeutics succeed we can prevent people from generating ammonia, hopefully right and so that or at least keeping the ammonia at a baseline level and hopefully having a normal you know protein diet. So obviously you know the opportunity to you know have.
It was small.
In select market share is very lucrative financially for a small company like ours.
And certainly you know we're discouraged by the slow uptake in you know the.
Patient recruitment, but you know I believe the steps that we've taken here in the near term should you know encourage us to accelerate that process here in the first half of next year.
Yeah, we remain committed to the program and it's not just as a value valuable asset, but it also represents the flagship asset for the platform for intravenously, dosed or systemically administered mrna and theres value taken into consideration for that.
Yeah.
I appreciate it thank you guys.
Thanks Seamus.
Our next question comes from Jack I'll know.
So some of it with Citi. Please proceed.
Hi, This is carly on for Yigal. Thanks for taking our questions. We have a couple on the head to head Bivalent study first just wanted to clarify was that study requested by regulators in additional geographies outside of Japan.
And then second.
Second more generally I guess, just wondering how you're thinking about the market opportunity in Japan for bi valent versus 154, and how that affects Macy's commercial launch strategy in Japan. Thank you.
Yes. Thank.
Thank you for the question all the regulatory agencies have United.
Their message for mono Valens Covid vaccines.
The reason that we're proceeding as a as a.
Asian between <unk> selling arcturus here, the reason where we.
We're collecting bivalent data is that we don't have to do it in the future. So if there's ever a reason why.
The regulatory agency changes their view or opinion and by being let's see becomes more important.
Then we will not need to do a trial at that time were just taking care of that now it does strengthen the platform, though to have monovalent phase III comparison data and then.
You add to that the bivalent comparative data.
With with bi valent.
But that's the reason where we're proceeding with.
With with collecting the bivalent data just to strengthen the platform and to prevent us from going back and doing a trial if it's ever requested in the future.
Okay got it that makes sense and then just one quick follow up I think in the past.
And <unk> has had maybe talked about in ex DB specific vaccine candidate just curious if there was an update on the status or the strategy for that okay.
Sure sure they the the the X P. B a vaccine update is a monovalent to update some of our partners have started to communicate about this.
Version of the vaccine, it's called a RCT 2303, and again its a monovalent updated.
Uh huh.
Asset and so so all the monovalent a R. C. T 154 data that we're collecting is very meaningful and relevant to that asset with respect to updates on activities around that asset we havent disclosed those publicly but there'll be an opportunity for us to to to provide an update on our next.
Carl.
Okay. That's very helpful. Thank you.
The next question comes from Yang Ming Xu with Wells Fargo. Please proceed.
Hello, This is quite well your honour and thinks about taking all questions. So two questions on COVID-19.
First of all on your Thunder there.
Hey, How're, you recently present that mrna health care.
Okay Comfrey conference can you tell us how that would translate to differentiation and potentially commercialize this that and the second question is after the potential approval in December.
Sure.
I see yeah, sorry, it would see S O.
Would see S. L launch based on these like mono wafer Fab, then greet you know vaccine or would they.
Would they way for for example, SBB update thank you.
Sure so with respect to the.
The leading commercial or marketing advantages of this platform as we've touched on them already in this call, but I think clearly the buyers here would be very interested in our durability data they want a more durable vaccine technology.
The increased antibodies of course has not.
Not only an improved immune response, but an implied efficacy benefit that would be interesting and then.
You know as we go deeper into the endemic Covid market and as that gets established there's going to be an increased emphasis on safety. So I think the meaningfully.
But the fact that this technology has a much lower dose is going to be.
Emphasize because of the potential safety benefits associated with dose related toxicology, so durability increased antibodies and at a much lower dose with potential safety benefits is going to be the initial strategy.
Now with respect to the you had another question could you repeat that for us.
Sure. So after the potential approval in December with CSL launch based on these.
Our regional vaccine or will they potentially wasteful ex VP update and launch the vaccine can stake. Thank you.
Well with respect to what we want to do strategically is position ourselves with the right approvals and the manufacturing slots and timelines to address what the market desires of rods. So if we have an approval or approvals in place then we can proceed.
To provide a.
And updated monovalent vaccine if the monovalent.
154 gets approved then we'll be in a stronger position to provide an updated monovalent vaccine if that's what the.
The.
If that's where the orders come.
And Hey, this is Pat and I just one other thing to add is so we you know we we Joe mentioned on his call earlier that we we've filed for in the EMA approval and are within our partner CSL. One once we get the approval I think CSL will be looking at what the commercial launch looks like right. So I think there.
They're in charge of that but but as Joe mentioned you know we're kind of we're currently focused on getting the approvals in the various jurisdictions now.
Got it thanks for the colors.
Thank you. The next question comes from Pete Stavropoulos with Cantor Fitzgerald. Please proceed.
Hi, Joe Andy and team nice to hear all the updates for the quarter first question I have for a 154 what are your expectations of when you may hear back from the EMA and sort of rough timelines.
Oh with respect to hearing back from an E. M. A this is gonna be a considerable process. We haven't provided any type guidance on when our approval was expected I can refer to our partner CSL has guided that in 2024, we anticipate.
Approval.
With EMA.
Alright, and then in terms of Carlos you know I'm just curious if you could just discuss a little bit if you can leverage that venture for manufacturing other pipeline candidates and perhaps you know distribution of a drug outside of Japan.
If not already.
When do you expect that.
To be operational and what will be the manufacturing capacity.
Yeah, I can answer that question for you Pete.
We've kind of guided that the drug substance facility as it had been completed.
We've kind of given a timeline for drug product and and fill finish is probably going to be next year and in that timeframe. So in the meantime, we're gonna be supplying Japan than any other country through our current CMO you know a group that we've been working with which includes catalog.
I'll do a bond rescue farm, you know and.
And Europe, so until that plant is up and running.
And running and able to you know support there or.
Not only Japan, but any other MLR and a product that we may be working on or therapeutics.
We certainly would would be delighted to have that diversification of manufacturing you know you know opportunities and in the far east like that so it's a it's going to be a very strategic asset for the major for us and for CSL and certainly want to be able to utilize.
But to the best of.
You know the opportunity to address whatever the demand may be you know on a global basis.
Hopefully that will help answer your question.
It does thank you very much and just one last question for me for 032.
For cystic fibrosis.
You know for the phase one b patients I know, we're going to receive two doses will you be looking at it any pharmacodynamic markers of clinical outcomes again, its only two doses sort of a gauge of 032 activity.
Ah yes, the primary the primary objective of the phase one B study.
Just to understand safety and Tolerability in a in an actual CF patients with two administrations and to understand further what type of administration regimen will be ideal for a nice proof of concept study.
That's to follow them.
Assuming success of course, but that's the now or are we looking at other potential markers of success, yes, but our expectation at this point and the purpose of the study is just to show safety and Tolerability and in CF patients and we'd be very excited to see if multiple administrations are well tolerated because.
As of the recent data that we shared and see a fair et cetera.
Very well.
Indicative or.
Our important data that we bolt onto the the human experience that increases the likelihood of success for this program.
Alright actually throw in one last question. If you don't mind. So this isn't an inhaled product you know is there any potential to actually develop some type of inhaled vaccine you know two respiratory viruses either alone or with partners CSL.
Well, it's a great segue to the the opportunity the overall platform opportunity for our technologies. If if we're showing proof of concept in large vaccine trials and in therapeutics.
It does present the opportunity to potentially combine these we haven't provided any guidance on this but it does give people some sense of excitement at the platform in general that there's a lot of opportunity in the future.
Alright, Thank you for taking my questions.
Thanks. Our next question comes from Ed Arce with H C. Wainwright. Please proceed.
Yeah.
Hi, everyone. This is Thomas Yip asking a couple of questions for Ed. Thank you for taking my questions.
Perhaps first first question Oh a.
And then O T C U E.
You mentioned that patient data.
The study in Europe, I expected in the first half of 'twenty 'twenty four.
Okay can you discuss what about the status of lumpy single ascending dose study in the U S slipped 60 patients. So when should we expect to see some data from that study.
Practically the database lock is later this year.
Once we've gone through that process with them strategically think at the right time to communicate the data.
Because we need to understand the timing of.
The interim phase two data. So we may present. These at the same time, we may present them separately, but we haven't made that strategic decision yet.
But we have informed the market now that the database is getting locked this year for phase one and phase one b.
Understood and then perhaps switching gears just a little too soon.
We successful phase one studies are ongoing.
When should we expect data from these studies.
For the CF study.
Yeah.
One P study, where we are we've guided some interim phase one data in the first half of next year.
With respect to the phase one study.
We've now informed the market that that we intend to provide a presentation at an appropriate conference.
In the first half of next year as well.
And that's the fastest one one last question from US this one's probably for Andy the 35 million milestone every system. Yeah. So okay. Curt can you discuss what was the triggering events for the milestone.
Yeah, the milestone was related to our Covid and the buyback program. So hopefully that will help answer that question.
And we've.
Being able to articulate the progress we're having with the Baidu him up study in Japan and were very encourage by the speed and success of that.
Trial, moving quite quite rapidly. Thank you.
Understood. Thank you again for taking my questions.
Thank you.
The next question comes from Yale Jen with Laidlaw and company. Please proceed.
But do you mean that thanks for taking the question.
Let's switch gears to the flu vaccine that.
To be started soon my question is that giving us the S. L. Oh.
Major player in this space and our.
Recently some of the other messenger RNA back flew back has probably got some issues maybe on the safety or.
Efficacy so.
Both you guys and the.
Thing that Hollywood.
Your messenger RNA vaccine protected overcome some some of those hurdles and Oh.
Product.
Yeah.
Yeah conventional messenger RNA flu vaccines have.
You know potential challenges relating to better dose related challenges and also durability challenges and that's where self amplifying mrna. This next generation technology can prove to be different and.
In good faith that we could because the dose is much lower there may be some additional flexibilities, there with respect to multi valency and including different antigens.
And also what we've shown in the recent data.
In our infectious disease vaccine trials is that this next generation self amplifying mrna technology is more durable.
And that is especially important in the flu shot space, where the flu vaccine space. So those would be areas of differentiation.
Okay, Great. That's very helpful. And then maybe one more question here, which is called the bivalent that Oh good Betsy.
One thing protecting their approved in Japan, what is the commercial strategy outside of Japan.
Being contemplated and was that something also you asked me as well.
Well right.
Right now all the regulatory.
Sid in monovalent.
Vaccines and not necessarily.
Change so if if if that does we're in a position.
Provide whatever the customer wants whether that's an updated five England technology or model.
Okay, great. Thanks, a lot appreciate it yeah.
Thank you again.
Thank you at this time I would like to turn the call back over to Mr. Payne for closing remarks.
Yeah, Thanks, everyone for participating on the call if theres any remaining questions don't hesitate to reach out to the team and we'll get back to you as soon as we can thanks to everyone and good night.
Yeah.
Thank you. This does concludes today's teleconference. You may disconnect. Your lines at this time. Thank you for your participation and have a great day.