Q2 2023 Arcturus Therapeutics Holdings Inc Earnings Call
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Right.
Welcome to Arcturus Therapeutics second quarter 2023 earnings call at this time all participants are in.
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.
A reminder, this conference is being recorded it is now my pleasure to introduce your host NEDA Shockers Sapphires, Judah what vice President head of Investor Relations Public relations and marketing. Please go ahead.
Thank you operator, good afternoon, and welcome to Arcturus Therapeutics second quarter 2023 financial update on pipeline progress call.
Today's call will be led by Joseph Payne, our president and CEO and Andy Sassine CFO .
Dr. Typical our CSO S C O will join them for the Q&A session.
Before we begin I would like to remind everyone that the statements made during this call regarding matters that are not historical facts are forward looking statements within the safe Harbor provisions of the private Securities Litigation Reform Act of 1995.
Forward looking statements are not guarantees of performance they involve known and unknown risks uncertainties and assumptions that may cause actual results performance and achievements to differ materially from those expressed or implied by the statements.
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 view only as of today such as statements were made.
Terrific, specifically disclaims any obligation to update such statements to reflect Florida information events or circumstances.
And with that I will now turn the call over to Jeff.
Thank you Heather 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 by highlighting our progress with our E. R. R. E. R. C T $1 50 for COVID-19 vaccine phase III program.
We're pleased with the rapid development and regulatory process.
Progress recently made with our alliance partner Meiji pharma towards the approval of <unk> 154 by the Japanese regulatory authorities.
Our CET 154 submission is supported by an active controlled phase III booster vaccine study.
That was executed during a period when multiple COVID-19 variance were circulated.
Initial E. R. C T $1 54 phase III booster study results were released as pre prints last month and met archive.
The study achieved its primary endpoint demonstrating non inferiority of neutralizing antibody response against the SAR C. O V. Two ancestral stream when compared to commonality.
The study also demonstrated superiority of <unk> 54, and neutralizing antibody response against the Omicron P. A four slashed five period as a key secondary end point.
The results of this trial indicate increased immunogenicity associated with E. R. C. T 154 at 28 days following booster administration.
For the ancestral strain the geometric mean ratio of neutralizing antibodies between E. R. C. P $1 54 in carbon Eddie was 1.43 fold, indicating a elevated neutralizing antibodies against the vaccine strain versus the comparator.
Based on available phase III clinical data at the time of the interim analysis.
<unk> hundred 54 continues to demonstrate a favorable safety and tolerability profile with no additional safety concerns identified.
In today's earnings release, we also included New E. R C to $1 54, a our phase.
Phase one slash two clinical study data, which suggest a durable immune response up to one year. Following booster administration, notably neutralizing antibody levels remained greater than 10 fold above baseline across a panel of variants, including all the crime.
During the one year observation. This is during the entire ups or one year observation period.
In summary, <unk> $1 54 has shown broad neutralizing capability against multiple variance of concern and has the potential to offer not only effective but also a longer lasting immune response that may suggest durable protection against COVID-19.
We're very pleased with the E. R. C T $1 54 clinical data and now believe that this next generation self amplifying mrna platform is meaningfully different than conventional currently approved mrna vaccines.
I will note that the <unk> hundred 54 doses very much lower and.
And our head to head study a RCT $1 54 was dosed at only five micrograms.
This is an 83, 3% lower dose than commonality and at least 90% lower than other approved mrna vaccines.
As previously as previously reported in April maybe some major <unk> submitted a new drug application in Japan to support potential initial approval of <unk> 54, as a primary immunization vaccine.
And in June we were pleased to hear that made you filed the NDA for the booster and included the relevant data to support that booster application.
The review process with the P. M D. A is ongoing with Japanese NDA approval being our next targeted milestone.
A positive P. M D. A decision would represent Arcturus has first product approval a tremendous achievement for our company.
Okay.
And approval.
Are unapproved all along with the promising data that we've collected is indicative of the broader opportunity for arcturus mrna technology platform.
I'll now move on to provide an update on a R. C. T. A 10. This is our messenger RNA therapeutic candidate for ornithine transcribed families or OTC deficiency.
This investigational medicine is designed to functionally replace that deficient OTC enzyme in the liver and thereby restore urea cycle activity and prevent metabolic crises that cause neurological damage.
Our Cta plan could potentially reduce the need for ammonia scavengers.
And he's the rigid dietary protein restrictions that OTC patients face today, that's improving quality of life for those living with this disease.
In June we announced that the FDA had granted our Cta 10 with fast track designation. The company has also recently received rare pediatric disease designation from the FDA for <unk>.
Rare pediatric disease designation is designed to recognize those rare diseases in children, and which serious or life threatening manifestations, primarily affecting patients from birth to 18 years of age.
Due to this designation F. A R. C. T 810 achieves approval for a pediatric indication are tourists will receive a voucher for priority review of a subsequent marketing application for a different product.
<unk> 10 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.
The phase one single ascending dose study is being conducted in the United States and has completed dosing of all planned cohorts and a total of 16 subjects.
The E. R. C. T 810 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.
The ongoing study is evaluating two dose levels and includes up to six by weekly administrations for each participant.
We expect to share interim data on biological activity from a subset of patients in the coming months.
Now I'll move on to a R. C T O 30 to this.
This is our inhaled messenger RNA therapeutic for cystic fibrosis.
This program is designed to express fully functional C. F T. Our protein in the lungs of individuals with CF utilizing our lunar delivery technology that has been highly optimized for inhaled delivery to the lungs.
Our approach is agnostic to the underlying mutations associated with the disease and as a result of this program could provide clinical benefit across a wide range of those living with CF, including those that are not well served by currently approved C. F. T. R. C FTR modulators.
In June at the European CF Society Conference in Vienna in collaboration with the University of Alabama, Birmingham Lab, we reported promising data demonstrating that a R. C. T O 30 to fully restore C. S. T R expression and function in vitro and bronchial epithelial cells from C F.
Donors.
In the past improvement and chloride transport in these human bronchial epithelial cells treated with see FTR modulators has been associated with improved clinical outcomes.
Yeah.
In the E. R. C. T O 32 clinical development program continues to advance according to plan.
We're pleased to report today the successful completion of dosing in our phase. One study. The study includes 32 healthy participants, including eight subjects in each of four dose cohorts being tested.
We look forward to reporting in terms safety and Tolerability study results later this year.
We received regulatory approval.
I have a protocol amendment to allow the transition to a phase one clinical study of <unk>, our CTO of <unk> 32, and up to eight cystic fibrosis patients.
<unk> will be conducted in New Zealand.
Continuing to advance this important investigational medicine.
Our strategic collaboration with CSL secure us. This is our tourists as exclusive global licensee is focused on the development and commercialization of next generation mrna vaccines and continues to make strong progress.
Our lunar flu program for example continues to progress with funding and operational support from CSL secures.
Lunar flu utilizes arcturus has validated next generation star mrna platform.
And with that I'll now pass the call to Andy.
Okay.
Thank you Joe and good afternoon, everyone.
The press release issued earlier today includes financial statements for the second quarter and six months ended June 2023.
And provides a summary and analysis of year over year financial results.
Please also reference our most recent Form 10-Q for more details on the financial performance.
We are very pleased with the E. R. C. T 154, new drug application to the P. M D a in Japan and.
And we believe that this product could represent an improved vaccine option for patients as well as an important source of potential future revenues for our organization.
Furthermore, the development and manufacturing plans supporting a R. C. T to 154 was carried out in a financially disciplined and efficient manner.
I will remind you that the phase III Japanese booster studies as well as product manufacturing related to this collaboration are being funded by a major U sake of pharma and the Japanese government.
Also in April May June Saker pharma entered into an agreement with CSL secures whereby made you will be responsible for the regulatory approval marketing distribution and sales of <unk> 54 in Japan.
As well as coordinating manufacturing of Covid vaccine products with our catalyst for the Japanese market.
We are thrilled to announce the completion of the state of the art mrna drug substance manufacturing facility in Japan with our partner our palace.
A team of delegates from Arcturus led by our senior executives and scientific team attended the Grand opening ceremony of the cutting edge cgmp facility in Tokyo, Japan. This past week.
The ceremony with the celebration of collaboration and partnership as our tourists joined forces with Japanese health care leaders and senior government officials to inaugurate the first mrna manufacturing facilities in Japan.
Located in a strategic hub for biomedical research and development. Our palace is poised to become a key player in the global mrna drug manufacturing landscape.
Our Calais that new plant meets current good manufacturing practice and is equipped with the world's most advanced manufacturing control and quality control system to efficiently manufacture high quality substance for mrna pharmaceuticals.
In April 2023, we received an advanced payment of $23 6 million for the manufacturing and supply of a R. A T. T 154 booster vaccines from C. S L.
The advanced payment as for specified manufacturing runs.
Our CET 154, which includes the drug substance as well as the reservation fees and related manufacturing requirements.
As Joe mentioned, the lunar flu program continues to progress with funding and operational support from C. S. L and additional updates will be provided soon.
I will now provide a summary of our financial results for the second quarter of 2023.
Our primary source of revenues were from license fees consulting and related technology transfer feed reservation fees and collective collaborative payments received from research and development arrangements with pharmaceutical and biologic biotechnology partners.
For the three months ended June 30 of 2020, we reported revenues of $10 5 million compared with $27 1 million for the three months ended June 32022.
The decrease was primarily attributable to a decrease in revenues of $12 7 million related to the termination of the agreement within bio care and a decrease in revenues of $12 5 million related to the agreement with the Israeli Ministry of Health.
The decrease was primarily offset by an increase in revenue of <unk>.
$8 6 million related to the collaboration agreement with CSL and the grant agreement with BARDA, which were both executed in the second half of 'twenty 'twenty.
Yes.
Revenue increased by $58 5 million during the six months ended June 30 of 2023 compared to the prior year period.
The increase was primarily attributable to an increase in revenue of $87 6 million related to the collaboration agreement with CSL secures and the grant agreement with BARDA.
Total operating expenses for the three months ended June 30 of 2023 was $65 9 million compared with $49 2 million for the three months ended June 32022.
Total operating expenses for the six months ended June 30 of 2023 were $131 4 million compared with one $104 8 million for the six months ended June 20th 30th 2022.
Our research and development expenses consist primarily of external manufacturing cost in vivo research studies and clinical trials performed by contract research organization clinical and regulatory consultants personnel related expenses.
So really related expenses and laboratory supplies related to conducting R&D activities.
R&D expenses were $52 7 million for the three months ended June 32023, compared with $38 2 million in the comparable period last year.
Primarily reflecting increased clinical research and manufacturing cost of $11 4 million and an increase of $2 9 million in personnel related expenses.
R&D expenses were $104 4 million for the six months ended June 30th 2023, compared with $83 1 million in the comparable period last year.
Primarily reflecting increased manufacturing and personnel costs.
General and administrative expenses, primarily consist of salaries and related benefit of our executive administrative legal and accounting functions and professional service fees for legal and accounting services as.
As well as other general and administrative expenses.
G&A expenses were $13 2 million and 27 million for the three and six months.
And at June 30 of 2023, respectively, compared with $11 million in 'twenty $1.7 million in the comparable period last year.
The increases resulted primarily from personnel expenses due to increased headcount and salaries increased travel and consulting expenses.
Well as an increase in rent expense associated with the new headquarters facility.
For the three months ended June 30 of 'twenty twenty-three Arcturus reported a net loss of approximately $52 6 million or $1.98 per diluted share compared with a net loss of $21 6 million or <unk> 82 cents per diluted share in the three months ended June 32022.
<unk>.
For the six months ended June 30 of 2023, Arcturus reported a net loss of approximately $1 8 million or seven cents per diluted share compared with a net loss of $72 7 million or $2.75 per diluted share in the six months ended June 30 of 2022.
Okay.
Cash and cash equivalents and restricted cash was $386 million as of June 30 of 2023 and.
394 million on December 31, 2022.
We have collected approximately 300 million in upfront payment and milestone some CSL as of June 32023.
Additionally, in the second quarter, we received $23 6 million under the manufacturing and supply of <unk> 54 from CSL.
We expect to continue receiving future milestone payments from C. S. L that will support the ongoing development of the Covid and flu programs.
Finally, I'm happy to report the cash run rate remains extended through the beginning of 'twenty 'twenty six based on the current pipeline and programs.
In summary, we believe the company remains in a strong financial position and have the resources needed to achieve multiple near term value, creating milestones for the vaccine and therapeutic programs.
Furthermore, with the anticipated they are C. T $1 50 for product approval later this year in Japan.
We look forward to beginning to report potential commercial sales in the next few years.
I will now pass the call back to Joe.
Hey, Thanks, Andy.
We've continued to make excellent progress and advance our proprietary messenger RNA and lunar delivery platform technologies toward later stages of clinical development.
It was great to hear the progress of our catalysts or partner there on the manufacturing facility in Japan.
And we are especially excited about the progress toward our first product approval potentially later this year with <unk> 54 are.
The achievement would definitely Mark a critical milestone for the platform and for Arcturus, So with that I'd like to turn the time over to the operator for questions.
Thank you.
A question and answer.
If you would like to ask the question.
Please press star one on your telephones.
Yeah.
John .
Your line is in the question queue.
Hey, guys.
Thank you.
All participants.
Gentlemen, you may be necessary.
Chad.
Yes.
Please go ahead.
Sure.
Our first question comes from.
Piper Sandler. Please go ahead.
Good afternoon team and congrats on all that data, especially on the manufacturing facility.
Could you maybe provide some color in terms of the timing of the approval in Japan.
<unk> for the fourth.
Paul second.
You could also provide us some thoughts around manufacturing capacity would you be able to by the time you receive approval in Japan to facilitate and and and and produced over 70 million doses if needed.
Then the third question is on the CF program are you planning to share the single ascending data could you comment on how high you wind it down thing.
And then lastly, you want with a dose of humility pour it into the math question and I'll jump back into the queue and my apologies for so many questions.
Sure well with respect to the first question on the approval timeline I can address that.
You can appreciate that the review process is ongoing with the P. M D. A.
Whether it's CMC and non clan and clinical package insert and risk management plan questions. Right. These are all standard and we're going through this ongoing process with P. M. D. A so it means that we remain on track for later this year.
With respect to approval with respect to capacity I'm going to turn the time to Andy to address that thanks. He has made with respect to capacity in Japan, We've clearly indicated that.
The fact, we will not be operational and producing until next year and Furthermore, we will defer those kind of questions to our partner at palace and <unk> in Japan, and CSL as well. So hopefully you can appreciate there and since the fall of the commercial program here and.
And if license out or a RCT 154, and future Covid products. So we're gonna have to rely on depend on them for guidance with respect to capacity.
But I'm sure they'll provided when the time is appropriate.
And too.
To address your final question about C. F. We have not and by intention not disclosed any of the dose levels that we've evaluated given that.
The environment is.
Competitive.
But I can share with you that that each patient will receive two doses in the phase one b a amendment.
So up to eight to CF patients and each will receive two doses, but the specific dose level, we're not disclosing at this time.
Okay, great. Thank you, so much and I'll jump back into the queue.
Thanks, Yes.
Next question comes from Myles.
Please go ahead.
Hi, everyone. Congrats on the burst of diner as well not to say that.
154 receiver.
We're safe approval by.
By year end in Japan, do you think you've got the rights a strain in there to potentially deliver some doses.
In these current vaccination season, I guess not.
One of your peers out there Daiichi and I got approval I think clearly cited that'd be switching to an expat base trying to supply the current period. So.
So that's the first question.
Yeah.
Myles Thanks for joining the call. It's good to hear you.
Youre, absolutely right all the regulatory agencies, including P. M. D. E are evaluating all the variance of interest in a particular region in this case Japan.
And Arcturus is prepared to address whatever they request.
So right now you've seen the profile of <unk> $1 54, and it's it's broadly acting against a variety of variance, but if if it's deemed by the P. M. D. A for whatever reason to proceed with another variant of interest whether that's X P b or something else, we're prepared to immediately respond and we're positioning the company.
Strategically to do so okay with with CSL and <unk> are involved in this process.
Okay and then the second question is just on duration Ah thanks to supply and that that phase voluntary data, we're gonna say duration of.
He may not initially out of the phase III trial, and you know what are your sort of predictions on on the call Menotti neutralizing antibodies sort of as we get into the six to 12 month period out of that trial.
No I'd say I appreciate the question clearly this data we shared today is very exciting it's very promising.
We're happy to see just a really strong durability signal from <unk> 54, its a good representation of this next generation self amplifying mrna platform, it's clearly different.
With respect to the phase III a dataset.
We intend to share that data for the six month time point later this year.
And we'll be more than happy to share that at that time.
Okay cool thanks for the questions.
Yeah. Thanks Myles.
Next question comes from Seamus Fernandez with Guggenheim Securities. Please go ahead.
Hey, guys.
A few questions first oh.
Just wondering if you share some color on the Japanese market.
What I'm trying to figure out some of the opportunity for 'twenty three 'twenty four particularly with the.
First 25 million vaccine purchase from Japan.
And expectations for additional orders.
On a P. C. Wondering if you can provide an enrollment update there and what we can expect.
From update later this year.
Well that'd be specific to <unk>.
Or should we expect a broader update as well thank you.
Alright. So your first question is with respect to you know.
The opportunity for vaccine purchases in Japan, right, I guess I'll turn that over to Andy do you want to address that.
You know we we obviously are very excited that the Japanese government continues to.
You know purchase the vaccine the COVID-19 related vaccines for population.
And historically, they approached us about 80 million vaccine.
The last 12 months that are we have record from the Japanese government. So certainly the the future awarded that we're currently seeing and anticipating or certainly a testament to their concern about COVID-19 drawing an endemic failure and are certainly of the approval of <unk>.
<unk> been in Japan for a conventional mrna a vaccine made in Japan is certainly are viewed as a very favorable here for our not only our tourists, but our partners <unk> and CSL because the Japanese government is very committed to ensuring that.
In the future, Japan will be vertically integrated and completely independent of any future manufacturers for any related pandemic type of viruses and I think that's pretty exciting that the government has taken on this initiative to be self sufficient and hopefully.
We can be a part of it and and we'll certainly learn more soon but you know with respect to specific orders, we really cannot.
Provide that at this time, it'll it'll certainly be developed in an articulated by major and CSL soon.
And then with respect to your second question on on <unk> enrollment.
I think we haven't provided specific guidance there, but we encourage people to look to other rare liver disease companies on the pace of their enrollment.
As a basic guide as to what to anticipate.
But we have finished our enrolment of the phase one b portion of the trial and as Joe mentioned in his remarks that we intend to share some data on the a 10 later on in the coming months yeah.
Yeah.
Thanks, Kevin.
Next question comes from.
Cancer.
Please go ahead.
Hi, Joe Hi, Andy and team. Thank you for taking my questions. Congratulations on all the progress and the booster data presented in a met Rx. It was nice to see the non inferiority and superiority in non neutralizing capabilities compared to an approved vaccine. So our first question regarding the manufacturing capabilities in Japan.
Can you potentially leverage them for you know outside of Japan, with 150 work and possibly other future candidates or when the facility be strictly focused on COVID-19 vaccine.
Absolutely Peter that's a really good question and thanks for being on the call. We anticipate you know of that are tell us will become a full fledge EMR in a facility with not only the capability of drug substance and drug product, but certainly going to be a world class facility for <unk>.
Any type of mrna capability going forward so it.
Up to our colleagues to announce all of this you know specifics with respect to their capability and I'll leave it up to them, but they do have an exciting future and we are a part of it and.
Certainly we will have more details with our partner as soon as it becomes available.
Alright, Thanks, and 4032 I know that you have fully enrolled the phase one in terms of a healthy volunteers you know of narrow the focus of safety and tolerability, but now that you're expanding into patient population. You know what are some of the ways that you may evaluate.
The Pharmacodynamic response.
Yeah.
What we're gonna be looking primarily at safety and Tolerability in patients.
I know, we're very pleased with the results so far that we've seen in phase one in healthy volunteers. This was the first time anyone has inhaled the lunar technology and so now that we transitioned to patients in phase one b that is the primary objective here.
Because we have a stellar preclinical dataset of Av.
So really positive to hate us. So we just wanted to first see safety and Tolerability in this phase one B S.
Set of patients, having said that we are positioning ourselves to evaluate LCI or lung clearance index.
And this is a sensitive measure of lung function. So we will look to see if after two doses. If we can see something but the primary objective here is just to establish safety and tolerability in patients alright.
Alright.
Thank you for that and the last question is I know you gave a brief sort of update on enrollment and not to look to other programs, but have you activated all the sites and if not you know how many more do you can you potentially activate to to help enrollment move alone.
Are we we are essentially at capacity with respect to activating sites. Our team has been traveling through Europe and following up with all of the principal investigators in this manner. So hum.
Working hard to you.
Enrolled patients and that's the guidance, we've provided and that we remain consistent on our in providing some some data later this year or in the coming months.
Alright, Thank you very much for taking my questions and congrats on the progress once again.
Yeah. Thanks Pete.
Next question comes from.
Wells Fargo. Please go ahead.
Hi, Thanks for taking my question and congrats on the progress.
Wanted to get maybe a little.
More clarity on what might happen after a potential Japan approval.
So I think Daiichi decision at least looking at the news release it is.
By itself and it doesn't look like the P. M D a.
You know instructed not to supply the market into original strain vaccine.
So I was wondering what is your annual partner thinking of last few received the approval.
What's the thinking process in terms of whether to supply the market and if you do supply the market I was wondering who is the manufacturer.
Is that a E. You your fifth Ney York facility or the our Polish facility.
Well with respect to commercial Guy.
As respect to the second obviously thankfully the question yet, but certainly are our current manufacturing partners.
Which represent Scotland and El Dorado in the U S and rest department.
Didn't in Europe would be a you know.
The suppliers of any current supply as you know we've articulated the Japan facility won't come online until next year. So any kind of orders for Japan from that facility, probably will not be until you know next year late or the year. After so hopefully you can appreciate that you know we have a dual.
Process in place to be able to you know provide the Japanese market with our vaccine as soon as possible and certainly once the factory is up and running in Japan that there'll be able to supply going forward hopefully that helps clarify you know that the situation and Joe can address the fruit.
Out of the question, Yeah with respect to the other.
Vaccine or vaccines getting approved in Japan.
We're very happy to see that Japan is following their domestic strategic plan. We've touched on this earlier in the call a little bit we view this very positive because we've if we feel like we're part of this domestic plan with <unk>.
And CSL, but are what what sets this vaccine technology apart from all other conventional mrna vaccines that have been approved or soon to be approved is our dose level. So our dose level is significantly lower.
Our our cereal response rates that we've observed seemed to be differentiating clearly people have to be taking this dose level into consideration when it comes to safety and react to Genesis is potentially differentiating and then finally the data we showed today.
The durability data is very promising.
So that in and can set this technology in part so as we worked with Japan and their strategy, we definitely see a way to fit them in line with their their objectives.
To address some of the key the key issues in protecting their people with a vaccine strategy.
Got it thanks for the color and you know you you touched on durability. Congrats on the data I was wondering can you talk about.
You know the underlying biological basis for this durability and your confidence that.
We could see this observation is that predicated in the longer follow up on the Japan study.
Yeah, It's a great question self amplifying mrna is providing.
Providing very encouraging durability data in your question is why and it's because the mechanism of how this the self amplifying mrna molecules make the antigen.
Conventional mrna makes the antigen for a period of time, and then degrades right, but self amplifying mrna. This next generation mrna technology.
Extends the duration of making that antigen. So the body sees the antigen for a longer period of time.
That means it is allowed to increase the antibody titer levels and that's what we've seen in the phase III a comparison study and.
We've all because the body sees the antigen for a longer period of time. It allows a broader spectrum of antibodies to be made and when you combine those two features of increased antibody titers with a broader spectrum of antibodies being.
Created by the body this translates into a longer lasting vaccine and that would provide an explanation as to you know.
Explaining why why we're seeing what we're seeing.
Got it so we might we should be able to see something similar with the Japan study.
Yeah, correct correct you if that's what you would expect and remember previously we've seen them in our phase one and phase two studies that the antibody titer levels tend to increase beyond one month or beyond four weeks. So will we see that as well in phase III is yet to be.
Understood, but yeah, our expectations are high and very optimistic.
Got it thanks for that color and lastly, the OTC deficiency program I was wondering you know you're in the news.
Maybe you just released your you mentioned that you may announce data from a subset of the patients I was wondering.
We expect our went up both cohorts of the.
Patients data being you called it out.
And what type of changes clinically or in terms of biomarkers can be expected with a roughly a three month duration.
And lastly would you be able to comment on them.
Next steps that we can expect once you have a reported the data. Thanks.
Well I appreciate the question and wanting to understand additional detail on a different dose levels in the OTC clinical trial, but the only guidance. We're providing today is some biological proof of concept or some biological data that will provide in a subset of patients.
So I can't provide any more detail than that.
With respect to any biomarkers that we're tracking we will be tracking them for an extended period of time and that includes ammonia and urea Genesis and other amino acids in can be such as glutamate for example can be measured them.
Several amino acids are impacted by the urea cycle and OTC itself can be measured the ornithine transcribe families enzyme. That's built are expressed in these liver cells are are these liver cells. Just so you're aware can be sloughed off into the circulation and measured.
In the plasma.
And so we can track OTC OTC levels, as well and over an extended period of time.
Yeah.
Got it thanks for all the color.
Thank you.
Good to hear it.
Next question comes from.
C C.
Please go ahead.
Hi, This is carly on for Yigal.
Our questions.
First just curious if there's anything more you can say about <unk> progress.
Seasonal flu vaccine and is that where I can maybe accelerated after the COVID-19 data do you have a sense for timelines for potentially breaking up a little.
For the clinic.
Well I can provide one comment you're absolutely right that the Meiji CSL and Arcturus teams are all United in and our excitement over this recent dataset and that excitement bleeds into other programs of course, it gives us more energy and more ambition vigor in other programs and so yeah.
<unk> about the flu is appropriate we have to be very careful that it's I would consider even though we're working closely with CSL. It is their program and Theres progress there program to guide on so it's very difficult for us to just give a candid response to your question on guidance.
Having said that we look forward to continuously update the market. Because this is an active program for US we've reported milestones on this previously this year.
And clearly this is a program of high interest for CSL, Andy anything to add yeah. In my comments I. Obviously, you said that we will have update provided soon so stay tuned. Thank you yeah.
Yeah.
Okay got it that's helpful. And then just on OTC and Mcs I know that.
Two in OTC is is active in <unk>.
In Europe , and then you're also recruiting in New Zealand for the CF program just curious any.
Any comments on potential timelines and plans for opening sites in the U S. France studies. Thank you.
Well, we already have active Ah trial ongoing and in for Covid in the vaccine enterprise in the U S. Already so we already have an active effort in the United States and for OTC, we have an active effort.
And in the U S with our phase one b trial in patients.
With respect to our C F.
You can see that we have a very strong relationship with with the CF Foundation and they have considerable oversight and influence on the U S.
Area, not just globally, but.
We haven't indicated any guidance as to when we will be conducting any clinical trial efforts in CF and U S. There'll be an appropriate time to do so but today's not that day.
Okay got it great. Thank you very much for taking our questions.
Thanks Karli.
Next question comes from.
H C Wainwright.
Go ahead.
Hi, Good afternoon, everyone. This is Thomas Yip asking a couple of questions for at our Kentucky Graduations.
Progress with our new G H.
So our first perhaps are looking a little bit into the future do you have any plans right now to expand our one platform user populations.
Uh Huh adults is Japan.
So you know what what what can investors think about the estimated timeline.
Yeah. The short answer is yes, there's a lot of activities, we'd like to expand them because our phase III trial was in adults right. So there's there's more than just adults and in Japan, and CSL has a definite and major is a clear interest in expanding the commercial opportunity beyond adults.
But I can't give specific guidance on that but its theres two to provide.
But outside of Japan, CSL is our is our exclusive partner for them for distribution and commercialization in U S and Europe for example.
So I'm looking to them to provide guidance on on those efforts.
Got it perhaps just one more question from us are regarding odor issue with.
Uh huh.
Interest on.
Hum.
Clearly.
Perhaps for the for the Phase one study what what can we expect the first CF patients.
Going into the study.
Richard credit risks.
And then also what.
What can we expect the initial dataset.
Patients.
Ah well, we've conservatively guided the initiation of enrollment in the second half of this year.
And we'll allow that to unfold and then with respect to data.
It'll be at a later time point I think we're focused on the near term objective of enrolling initiating enrollment.
For these up to eight CF patients in New Zealand in the second half of this year.
Okay.
With respect to enter a phase one safety data you know interim phase one safety data is also expected later this year.
Okay got it Oh.
Fourth you those data along with.
Our regulatory status in Japan, as well, but if you look at what's going on.
Yeah. Thanks Thomas.
Next question comes from.
Please go ahead.
Oh, good afternoon, and thank you for taking the question and congrats on all the progress in Japan.
I appreciate the data you provided today in terms of the bulk of duration as well.
The breadth of.
You have seen how long do you keep walking covers.
My question to you is that does this have any read through in terms of the.
Patients in Japan in terms of they'll cover broader.
That's all ups straightening.
Then I have another follow up question.
Yeah. It's a good question the data we shared today was only using a validated assay. The new strains that are coming forward are only exploratory in nature and not validated yet so we've.
We've remained consistent on sharing data that's only validated assays right. So there'll be a time and a place to provide that data once the assets are validated as the answer.
Okay.
What is that referring you mentioned earlier that are you.
<unk> reported a six month data in the future time, so maybe later this year.
Are these two connected in anyway.
Alright, well, if youre, referring to the phase III Japanese comparator study, yes, we were guiding that we'll be able to share a six month durability data later this year.
Correct and you know this is Pat.
You just want to make sure that you understand you know we are an mrna platform company and because of that we get update our vaccine readily so if theres a new strain that emerges we can of course update our vaccine and because the platform will be approved we can quickly commercialize that as well and and all.
Any fashion processes very similar to lot of the other mrna players keep in mind that you know with the trial being completed in February you wont really have the six month completion of the data until August so by the time, they collected and analyze it you have to give us a few months for that so hopefully that gives you some kind of.
You know runway to understand you know when conceivably the six month data could even.
Be presented hopefully it'll be probably a fourth quarter event. If you can appreciate that does that make sense.
Absolutely and replacing the colors and the one follow up question here in terms of the deal.
There are 32 wins you have in your press release, you mentioned that there was some amendment the beam E beam test.
In Florida or theme being suggested or the phase <unk> study.
Any colors in terms of what specific amendment has been placed.
Yeah Yeah.
The amendment is our.
Phase one was in healthy volunteers.
And Oh, well, what we talked about was then in Joe's comments that Theres been an amendment that was filed and it was recently approved to include patients in the study.
So therefore, it just at adding the patient versus E health volunteers, okay, great. Thanks, a lot okay.
Okay.
Right.
Yep Yep. Thank.
Thank you yeah.
Okay.
That was all the time, we had for today.
Oh, sorry.
A closing comment.
Yeah. Thanks for participating on the call today, everyone. If there's remaining questions. Please reach out to the team and we'll get back to you right away Goodnight.
Good night.
This concludes today's teleconference. You may disconnect your lines at this time. Thank you for your partnership.
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