Q2 2022 Arcturus Therapeutics Holdings Inc Earnings Call
Operator: Please stand by.
Unknown Executive: Good day and welcome to the Arcturus Therapeutic second quarter 2022 financial update and pipeline progress call.
Please standby.
Good day and welcome to that.
Our church or therapeutics second quarter, 2022 financial D. N type brain power grids call. Today's conference is being recorded at this time I'd like to turn the conference over to Karl Keirstead. Please go ahead Sir.
Operator: Today's conference is being recorded.
Operator: At this time, I would like to turn the conference over to Kyle Gustat.
Operator: Please go ahead, sir.
Thank you Sarah good afternoon, and welcome to Arcturus Therapeutics second quarter, 2022 financial update and pipeline progress call. Thank you all for joining us.
Kyle Gustat: Thank you, Sarah.
Today's call will be led by Joseph Payne, our president and CEO and Andy <unk>, Our CFO , Dr. Pat <unk>, our CSO and CLO will join us for the Q&A session.
Kyle Gustat: Good afternoon and welcome to Arcturus Therapeutic's second quarter 2022, financial update and pipeline progress call.
Before we begin I would like to remind everyone that 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.
Kyle Gustat: Thank you all for joining us.
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 forms 10-Q, and 10-K filed with the SEC.
In addition, any forward looking statements represent our views only as of the date such statements are made August 19 2022.
Our tourist specifically disclaims any obligation to update such statements to reflect future information events or circumstances with that I'll now turn the call over to Joe.
Kyle Gustat: Today's call will be led by Joseph Payne, our President and CEO, and Andy Sassine, our CFO.
Kyle Gustat: Dr. Pad Chivukula, our CSO and COO will join in for the Q&A session.
Thank you and good afternoon to all thank you for joining Arcturus is Q2 quarterly call.
Kyle Gustat: Before we begin, I would like to remind everyone that 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
My comments today will begin with a brief summary of the business case for our vaccine platform followed by highlighting our recent progress on our pipeline, including additional details per program before I turn the time over to Andy for financial updates.
Kyle Gustat: Litigation Reform Act of 1995.
Kyle Gustat: Forward-looking statements are not guarantees of performance. They involve known and unknown risks, uncertainties, and assumptions that may cause, actual results, performance, and achievements to differ materially from those expressed or implied by the statement.
The business case for our vaccine platform continues to mature and gain momentum we begin by re emphasizing our low dose level, a lower dose level of only five micrograms potentially means improved safety benefits and higher manufacturing related profit margins.
Kyle Gustat: 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 Forms 10-Q and 10-K filed with the SEC.
Kyle Gustat: In addition, any forward-looking statements represent our views only as of the date such statements are made, August 9, 2022.
Our vaccine platform is also devoid of an adjuvant and therefore captures all of the potential safety benefits in manufacturing simplicity associated with that.
In addition to the low dose of five micrograms and the simplicity of zero adjuvant, our vaccine platform utilizes our tourist as Lai authorization technology. We've now collected impressive cold chain data showing favorable stability shipping and storage advantages that our life lives vaccines have relative to frozen liquid.
Vaccines.
Some of the critical challenges with presently approved Covid vaccines are related to durability and broad spectrum nerve neutralizing antibody activity, especially with respect to the omicron variance.
We believe that.
Broadly acting in the potentially longer lasting self amplifying mrna booster vaccine nations would further strengthen our business case for this platform and.
Kyle Gustat: Arcturus specifically disclaims any obligation to update such statements to reflect future information, events, or circumstances.
Indeed, the data we shared today in our press release continues to suggest that self amplifying mrna vaccines may be a superior next generation mrna vaccine technology platform.
I will get into more details pertaining to that data shortly.
This quarter has been highlighted by several key achievements, including significant progress advancing our pipeline of mrna based vaccines and therapeutics.
Kyle Gustat: With that, I'll now turn the call over to Joe.
Let's begin with a CET $1 54.
This is our self amplifying mrna COVID-19 vaccine candidate that has shown impressive activity against Sars Cov, two both as a primary vaccine and as a booster.
Given the continued widespread circulation of Sars cov, two and the anticipation of a long term endemic phase.
We continue to aggressively move forward with development of <unk> $1 54, as a booster vaccine. We're pleased with the favorable results that continue to come from the ongoing phase one two study evaluating $1 54 in the U S and Singapore, giving us a five microgram booster dose approximately.
Five months after primary vaccination with commonality.
The boost with ARCC $1 54 generated robust neutralizing antibody responses that are sustained through three months after booster vaccination. The neutralizing antibody responses to the parents strain of the vaccine also known as <unk> six one for GE and the antibody responses against a panel of SAR CRB to vary.
<unk> strains have also consistently demonstrated anti body responses that have remained elevated over three months.
Now moving our attention to the present and more relevant omicron variance.
We are happy to share new data today from the ongoing analysis of this study demonstrating durable immune responses to both omicron VA, one NBA to variance.
Against be a too we saw a 39 fold increase in neutralizing antibody titer at day 91 over baseline.
This was complemented by a 44 fold increase in neutralizing antibodies against a one at day 91.
With these encouraging data we have growing confidence in <unk> $1 50 force profile as a booster vaccine.
These data suggest that the broadly immunogenetics.
Low dose and well tolerated features of our self amplifying mrna vaccine platform.
It may compete effectively in a booster market currently dominated by vaccines with narrow SAR C. O V two protection and waning immunogenicity and at considerably higher doses.
Okay.
Arcturus is presently collecting six month booster immunogenicity data for all relevant omicron variance, including VA five.
We look forward to seeing that data soon and sharing it with our potential strategic commercial partners.
Earlier this year, we learned from our collaborator Vin bio care that the RCT $1 54 phase one slash two slash three study had met its primary endpoint objective of vaccine efficacy the.
Study demonstrated very strong protection against severe and fatal COVID-19 disease, even went up against the more challenging variance that have escaped other vaccines.
Supported by these collective data and the favorable results of the Vietnam vaccine efficacy trial, we have been in discussions with major regulatory agencies, including the FDA. The MH, our aim and EMA regarding the path to pivotal trial and future approval as a booster.
Now based on additional health authority guidance. The booster trial that was slated to start in Q3 has now been split into two smaller and more time and cost efficient trials. The first of which is expected to start in Q4.
The updated study designs are intended to support global registration of <unk> $1 54, as a booster.
The Vietnam Ministry of Health is undergoing a significant reorganization, resulting in a delay of the <unk> 154, EUA or emergency use authorization for the primary series.
During this reorganization the moh the Ministry of Health communicated that the clinical section review is complete and adequate.
The Moh provided comments to the CMC section and this is the chemistry manufacturing and controls section requesting three manufacturing runs to support full market authorization.
<unk> 154 is now expected to receive EUA and in Q4 2022 with full marketing authorization anticipated in early 2023.
Booster studies meet non inferior immunogenicity and safety objectives.
Now the new stability data that was highlighted in our press release today supporting the supported the advantages of using a lie awful lives vaccine powder to mitigate the cold chain challenges associated with frozen liquid mrna vaccines.
Our lie authorized COVID-19 vaccine powder demonstrated room temperature stability for four days, that's 25 degrees centigrade in the presence of 60% relative humidity and refrigerator stability for six months, that's two to eight centigrade.
And our predicted long term stability at minus 20.
Centigrade for 18 months.
Transportation in the summer and winter months can provide temperature cycling challenges in the supply chain. This is where liquid products can be frozen or thawed unintentionally as you can appreciate and importantly, the lifeline vaccine powder was not impacted by multiple temperature cycling stresses going from minus 22.
Refrigerated temperatures like two through eight a room temperature and vice versa.
This enables global shipping logistics and the supply of a stable usable solid COVID-19 vaccine product at two to eight centigrade.
Yeah.
Taking these significant cold chain advantages into context, with our low dose level potential durability and broader spectrum of activity compared to conventional mrna.
Our next Gen self amplifying mrna platform continues to differentiate itself.
Our self amplifying mrna influenza vaccine has continued its preclinical work.
We expect to make a final decision of our lunar fluid development candidate. This year, given the comparative speed of mrna vaccine production and precise antigenic matching against circulating influenza strains. This is expected to offer an important improvement over currently marketed flu vaccines.
Comparatively lower dose, we believe that our approach may be differentiated and other meaningful ways as well.
Joseph Payne: Thank you, and good afternoon to all.
Now I'd like to shift from our Vac shift from our vaccine franchise over to our therapeutic mrna pipeline programs, which continue to advance.
Joseph Payne: Thank you for joining Arcturus's Q2 quarterly call.
I'll begin with an update on <unk> 10. This is our therapeutic candidate for ornithine, <unk> deficiency or OTC deficiency.
Joseph Payne: My comments today will begin with a brief summary of the business case for our vaccine platform, followed by highlighting our recent progress on our pipeline, including additional details per program, before I turn the time over to Andy for financial updates.
Joseph Payne: The business case for our vaccine platform continues to mature and gain momentum.
OTC deficiency is a result of a urea cycle disorder, it's rare and serious has no approved treatments that address the root cause of the disease and our approach aims to augment the deficient OTC enzyme in the liver of these patients.
Joseph Payne: We begin by reemphasizing our low dose level. A lower dose level of only 5 micrograms potentially means improved safety benefits and higher manufacturing-related profit margins.
Joseph Payne: Our vaccine platform is also devoid of an adjuvant and therefore captures all of the potential safety benefits and manufacturing simplicity associated with that. In addition to the low dose of 5 micrograms and the simplicity of zero adjuvant, our vaccine platform utilizes Arcturus's lyophilization technology. We've now collected impressive cold chain data showing favorable stability, shipping, and storage advantages that our lyophilized vaccines have relative to frozen liquid vaccines.
<unk> 10 has the potential to restore urea cycle activity prevent or slow the progression of neurological damage and potentially expand dietary options and improve on the quality of life for people living with this condition.
Joseph Payne: Some of the critical challenges with presently approved COVID vaccines are related to durability, and broad-spectrum neutralizing antibody activity, especially with respect to the Omicron variants.
Joseph Payne: We believe that broadly acting and the potentially longer-lasting self-amplifying, mRNA booster vaccinations would further strengthen our business case for this platform.
Joseph Payne: Indeed, the data we shared today in our press release continues to suggest that self-amplifying, mRNA vaccines may be a superior next-generation mRNA vaccine technology platform.
We've obtained approval from the United Kingdom's HR, a or health research authority and other European authorities to initiate a phase two multiple dose clinical trial for <unk> 810, designed to enroll 20 for adolescents and adults.
Joseph Payne: I will get to more details pertaining to that data shortly.
As many can appreciate it is often challenging to find and identify rare disease patients. We are pleased to report that our arcturus has identified several dozen patients at multiple sites in the United Kingdom, Spain and Belgium.
Joseph Payne: This quarter has been highlighted by several key achievements, including significant progress advancing our pipeline of mRNA-based vaccines and therapeutics.
Joseph Payne: Let's begin with ARCT154. This is our self-amplifying mRNA COVID vaccine candidate that has shown impressive activity against SARS-CoV-2, both as a primary vaccine and as a booster. Given the continued widespread circulation of SARS-CoV-2 and the anticipation of a long-term endemic phase, we continue to aggressively move forward with development of ARCT154 as a booster vaccine. We're pleased with the favorable results that continue to come from the ongoing Phase 1-2 study, evaluating 154 in the U.S. and Singapore, given as a 5-microgram booster dose approximately five months after primary vaccination with Comirnaty.
With France and other countries soon to follow we plan to screen and dose patients throughout the remainder of this year with the goal of obtaining in term proof of concept data by year end.
We're pleased to report that <unk> was recently granted orphan drug designation by the European Commission for the treatment of ornithine transcribe families deficiency.
There are cute and incentives associated with this designation that include reduced regulatory fees for protocol assistance and marketing applications. The use of the centralized marketing authorization procedure and 10 years of market exclusivity. This.
This European designation complements the orphan drug designation by the U S. FDA that this program has already received previously.
We have also completed dosing of the second cohort of our ascending dose phase <unk> study in the U S where in the participants received a dose of <unk> three milligrams per kilogram of <unk> 10.
The study's data safety review committee or D. Src has recommended continuation of the study and escalation to a higher dose cohort, which will begin dosing imminently.
Moving now to our cystic fibrosis program, we've continued to progress the necessary preclinical and non clinical studies to enable <unk> 32 to move to the clinic.
T O 32 is our inhaled messenger RNA therapeutic for cystic fibrosis we.
We recently presented at the European CF Conference in Rotterdam.
And had a productive update meeting with the CF Foundation there are supportive collaboration partner for this program.
And we anticipate the submission of a clinical trial application or Cta for <unk> 32 in Q4 2022.
Well with that I will now pass the call on to Andy says seen our CFO to provide financial updates.
Thank you Joe and good afternoon, everyone. The press release issued earlier today includes financial statements for the second quarter of 2022 and provides a summary and analysis of year over year and sequential financial performance.
Please reference our 10-Q for more detail on our financial performance.
I will summarize our financial and then some operating metrics.
We continue to transition to a late stage clinical company with several assets in our pipeline.
I will also provide some detail regarding our manufacturing strategy.
As we prepare for the potential of emergency use authorization in Vietnam by.
Finally, I will provide some insights regarding our cash position and expected run rate.
As you heard Joe mention we remain productive in the second quarter.
Banding on the highly encouraging efficacy trials and booster durability results for arc 154.
Joseph Payne: The boost with ARCT154 generated robust neutralizing antibody responses that are, sustained through three months after booster vaccination. The neutralizing antibody responses to the parent strain of the vaccine, also known as D614G, and the antibody responses against a panel of SARS-CoV-2 variant strains have also consistently demonstrated antibody responses that have remained elevated over three months.
Joseph Payne: Now, moving our attention to the present and more relevant Omicron variants. We are happy to share new data today from the ongoing analysis of this study, demonstrating durable immune responses to both Omicron BA1 and BA2 variants. Against BA2, we saw a 39-fold increase in neutralizing antibody titer at day 91 over baseline. This was complemented by a 44-fold increase in neutralizing antibodies against BA1 at day 91.
We have previously discussed we partner to our $1 54 next generation lunar a COVID-19 vaccine candidate in Vietnam, who had been bio care.
Joseph Payne: With these encouraging data, we have growing confidence in ARCT154's profile as a booster, vaccine.
Joseph Payne: These data suggest that the broadly immunogenetic low-dose and well-tolerated features of our self-amplifying mRNA vaccine platform may compete effectively in a booster market currently dominated by vaccines with narrow SARS-CoV-2 protection and waning immunogenicity and at considerably higher doses.
Joseph Payne: ARCTurus is presently collecting six-month booster immunogenicity data for all relevant Omicron variants, including BA5.
Joseph Payne: We look forward to seeing that data soon and sharing it with our potential strategic commercial partners.
Bio care as part of the Venn group, which is one of Vietnam largest corporation in the sponsoring in funding or food 123 study in Vietnam targeting COVID-19.
Joseph Payne: Earlier this year, we learned from our collaborator, VinBioCare, that the ARCT154, Phase 1-2-3 study had met its primary endpoint objective of vaccine efficacy. The study demonstrated very strong protection against severe and fatal COVID disease, even, when up against the more challenging variants that have escaped other vaccines.
Joseph Payne: Supported by these collected data and the favorable results of the Vietnam vaccine efficacy, trial, we have been in discussions with major regulatory agencies, including the FDA, the, MHRA, and EMA, regarding the path to pivotal trial and future approval as a booster.
This partnership includes the trial in collaboration around building a vaccine manufacturing facility in Hanoi.
<unk> and significant cost savings for tourists.
Our technology transfer activities remain on track for the facility to become operational later this year with a potential capacity of over 200 million doses annually.
We are also continuing to work with other manufacturing partners to mature, our global footprint, including Europe , Japan, and the United States of America.
Revenues from strategic alliances and collaborations for the second quarter of 2022 with $27 1 million.
<unk> increased from the previous two quarters.
The increase in revenue during the three months ended June 30 of 2022 as compared to the three months ended March 31, 2022 was primarily due to revenues of $12 5 million related to the onetime recognition of reservation fees from there.
The Israel Ministry of Health and an increase in revenue from been bio care related to shipments of drug substance and the validation of their manufacturing facility.
<unk> bio manufacturing facility in Hanoi is anticipated to achieve commercial scale production capability in Q4 of 2022.
Our operating expenses for the second quarter were $49 million with.
With declining quarter over quarter lower than the same quarter of the previous year.
The decline in operating expenses was primarily due to a decrease in clinical trial expenses related to our CET 154.
Joseph Payne: Now, based on additional health authority guidance, the booster trial that was slated, to start in Q3 has now been split into two smaller and more time and cost-efficient trials, the first of which is expected to start in Q4. The updated study designs are intended to support global registration of ARCT154 as, a booster.
In January 2020, the company entered into an agreement with a pharmaceutical company whereby the pharmaceutical company agreed to fund up to $25 million for a clinical trial for <unk> $1 54 vaccine candidate the booster.
Joseph Payne: The Vietnam Ministry of Health is undergoing a significant reorganization, resulting in, a delay of the ARCT154 EUA, or Emergency Use Authorization, for the primary series. During this reorganization, the MOH, or Ministry of Health, communicated that the clinical, section review is complete and adequate. The MOH provided comments to the CMC section, and this is the Chemistry, Manufacturing, and Control section, requesting three manufacturing runs to support full market authorization.
Joseph Payne: ARCT154 is now expected to receive EUA in Q4 2022, with full marketing authorization, anticipated in early 2023, if booster studies meet non-inferior immunogenicity and safety objectives.
As of June 32022, the company has submitted to the pharmaceutical company or Bill of $4 9 billion from a third party related to the clinical trial.
Bill falls under the expected funding.
$25 million for the booster program.
We reported a net loss of approximately 21.6 million or <unk> 82.
Basic and diluted share for the second quarter of 2022.
Compared to a net loss of $54 6 million.
$2.07 per basic and diluted share in the three months ended June 32021.
Net loss of $51 2 million or $1.94 per basic and diluted share for the three months ended March 31 2022.
Our cash balance at the end of the second quarter with $283 5 million.
Based on our current pipeline our cash position is expected to be sufficient to support operations into late 2023.
I will now pass the call back to Joe.
Hey, Thanks, Andy we're pleased to have another productive quarter advancing our pipeline of mrna vaccines and therapeutics and all the while judiciously controlling our operating expenses as was just nicely summarized by Andy.
Joseph Payne: Now the new stability data that was highlighted in our press release today supported the advantages, of using a lyophilized vaccine powder to mitigate the cold chain challenges associated with frozen liquid mRNA vaccines. Our lyophilized COVID vaccine powder demonstrated room temperature stability for four days. That's 25 degrees centigrade in the presence of 60 percent relative humidity. And refrigerator stability for six months. That's two to eight centigrade. And a predicted long-term stability at minus 20 centigrade for 18 months.
Joseph Payne: Transportation in the summer and winter months can provide temperature cycling challenges, in the supply chain. This is where liquid products can be frozen or thawed unintentionally, as you can appreciate.
Joseph Payne: And importantly, the lyophilized vaccine powder was not impacted by multiple temperature, cycling stresses, going from minus 20 to refrigerated temperatures like two through eight or room temperature and vice versa. This enables global shipping logistics and the supply of a stable usable solid COVID, vaccine product at two to eight centigrade.
Joseph Payne: Taking these significant cold chain advantages into context with our low dose level, potential, durability and broader spectrum of activity compared to conventional mRNA, our next-gen self-amplifying mRNA platform continues to differentiate itself.
We remain highly encouraged about the potential of our clinical pipeline and from our mrna vaccine and therapeutic platforms. We look forward to providing you with updates on our progress everyone. I'll now turn the call back to the operator to field any questions.
Joseph Payne: Our self-amplifying mRNA influenza vaccine has continued its preclinical work.
Joseph Payne: We expect to make a final decision of our Lunar Flu Development Candidate this year. Given the comparative speed of mRNA vaccine production and precise antigenic matching, against circulating influenza strains, this is expected to offer an important improvement over currently marketed flu vaccines.
Joseph Payne: Comparatively lower dose, we believe that our approach may be differentiated in other meaningful ways as well.
Joseph Payne: Now I'd like to shift from our vaccine franchise over to our therapeutic mRNA pipeline programs, which continue to advance.
Joseph Payne: I'll begin with an update on ARCT810. This is our therapeutic candidate for Ornithine Transcarbamylase Deficiency or OTC deficiency. OTC deficiency is a result of a urea cycle disorder.
Joseph Payne: It's rare and serious, has no approved treatments that address the root cause of the disease. And our approach aims to augment the deficient OTC enzyme in the liver of these patients. ARCT810 has the potential to restore urea cycle activity, prevent or slow the progression of neurological damage, and potentially expand dietary options and improve on the quality of life for people living with this condition.
Joseph Payne: We've obtained approval from the United Kingdom's HRA or Health Research Authority, and other European authorities to initiate a phase two multiple dose clinical trial for ARCT810 designed to enroll 24 adolescents and adults.
If you would like to ask a question. Please take note by pressing star one on your telephone keypad, if you're using a speaker phone. Please make sure your mute function. It turned out to them I think not to reach our equipment.
Joseph Payne: As many can appreciate, it is often challenging to find and identify rare disease patients.
Joseph Payne: We are pleased to report that Arcturus has identified several dozen patients at multiple sites, in the United Kingdom, Spain, and Belgium, with France and other countries soon to follow. We plan to screen and dose patients throughout the remainder of this year, with the goal of obtaining interim proof of concept data by year end.
Joseph Payne: We're pleased to report that ARCT810 was recently granted orphan drug designation, by the European Commission for the Treatment of Ornithine Transcarbamylase Deficiency. There are key incentives associated with this designation, that include reduced regulatory fees for protocol assistance and marketing applications, the use of the centralized marketing authorization procedure, and 10 years of market exclusivity. This European designation complements the orphan drug designation by the U.S. FDA, that this program has already received previously.
Joseph Payne: We have also completed dosing of the second cohort of our ascending dose phase 1B study in the U.S., wherein the participants received a dose of 0.3 milligrams per kilogram of ARCT810. The study's Data Safety Review Committee, or DSRC, has recommended continuation of the study, and escalation to a higher dose cohort, which will begin dosing imminently.
Joseph Payne: Moving now to our Cystic Fibrosis Program, we've continued to progress the necessary preclinical, and non-clinical studies to enable ARCT032 to move to the clinic. ARCT032 is our inhaled messenger RNA therapeutic for cystic fibrosis. We recently presented at the European CF Conference in Rotterdam and had a, productive update meeting with the CF Foundation, our supportive collaboration partner for this program, and we anticipate the submission of a clinical trial application or CTA for ARCT 032 in Q4 2022.
Press Star one to ask a question it will pause for just a moment to allow everyone an opportunity to take no further question.
Okay.
And we'll take our first caller from.
Yeah.
Right.
Paper.
I'm Larry.
Joseph Payne: Well with that I will now pass the call on to Andy Sassine, our CFO, to provide financial updates.
Hey, guys.
Andrew Sassine: Thank you Joe and good afternoon everyone.
Okay.
Andrew Sassine: The press release issued earlier today includes financial statements for the second quarter of 2022 and provides a summary and analysis of year-over-year and sequential financial performance.
Oh, one moment. Please we will actually take our first question from Pete Scott.
Ralph.
Sorry, with Cantor Fitzgerald.
Andrew Sassine: Please reference our 10-Q for more details on the financial performance.
Hi, Pizza propagate. Thank you thanks for taking my question.
Andrew Sassine: I will summarize our financials and present some operating metrics as we continue to transition to a late-stage clinical company with several assets in our pipeline.
Oh, one question I have is is.
Is there any more details that you can share in terms of the trial design for the booster I know that you've previously shared that it was going to be about 2400 patients for one study but.
Andrew Sassine: I will also provide some details regarding our manufacturing strategy as we prepare for the potential of emergency use authorization in Vietnam.
Andrew Sassine: Finally, I will provide some insights regarding our cash position and, expected run rate.
And you're going to run through smaller studies.
Andrew Sassine: As you heard Joe mention, we remain productive in the second quarter, expanding on the highly encouraging efficacy trials and booster durability results for ARCT 154.
Can you provide any color on the size of the studies.
Possibly keep differences.
Oh, well, one or both studies to be conducted perhaps with an on the ground specific strain bivalent candidates.
Any color would be appreciated.
Sure. It's a great question. Thanks, Pete for joining the call. Yes, we did emphasize that we're splitting bifurcated. The single trial of 2400 people into two smaller trials, we haven't communicated the exact number of those trials and the specific countries.
Andrew Sassine: As we have previously discussed, we partnered ARCT 154 Next Generation Lunar COVID-19 Vaccine Candidate in Vietnam with Venn, Biocare. Venn Biocare is part of the Venn Group, which is one of Vietnam's largest corporations and is sponsoring and funding our Phase 1, 2, 3 study in Vietnam targeting COVID-19.
Andrew Sassine: This partnership includes the trial and, collaboration around building a vaccine manufacturing facility in Hanoi, resulting in significant cost savings for our tourists.
Andrew Sassine: Our technology transfer activities remain on track for the facility to become operational later this year with a potential capacity of over 200 million doses annually.
Andrew Sassine: We are also continuing to work with other manufacturing partners to mature our global footprint, including Europe, Japan, and the United States of America.
Andrew Sassine: Revenues from strategic alliances and collaboration for the second quarter of, 2022 were $27.1 million, a significant increase from the previous two quarters. The increase in revenue during the three months ended June 30th, 2022, as compared to the three months ended March 31st, 2022, was primarily, due to revenues of $12.5 million related to the one-time recognition of reservation fees from the Israel Ministry of Health and an increase in revenues from Venn BioCare related to shipments of drug substance and the validation of their manufacturing facility.
Andrew Sassine: The VinBio manufacturing facility in Hanoi is anticipated to achieve commercial-scale, production capability in Q4 2022.
Andrew Sassine: Our operating expenses for the second quarter were $49 million, which declined quarter over, quarter and was lower than the same quarter in the previous year. The decline in operating expenses was primarily due to a decrease in clinical trial expenses, related to ARCT 154. In January 2020, the company entered into an agreement with a pharmaceutical company, whereby the pharmaceutical company agreed to fund up to $25 million for a clinical trial for ARCT 154 vaccine candidate as a booster.
Andrew Sassine: As of June 30, 2022, the company has submitted to the pharmaceutical company a bill of $4.9, million from a third party related to the clinical trial. This bill falls under the expected funding of $25 million for the booster program.
Andrew Sassine: We reported a net loss of approximately $21.6 million or $0.82 per basic and diluted share, for the second quarter of 2022, compared to a net loss of $54.6 million or $2.07 per basic and diluted share in the three months ended June 30, 2021, and a net loss of $51.2 million or $1.94 cents per basic and diluted share in the three months ended March 31, 2022. Our cash balance at the end of the second quarter was $283.5 million, and based on our, current pipeline, our cash position is expected to be sufficient to support operations into late 2023.
But one will be emphasized on comparative immunogenicity and the other will be safety centric.
Uh huh.
Uh huh.
Okay.
In terms of.
When you talk to the different agencies.
Do they want sort of like a variation for a comparator arm, if they're asking broken parador them like no.
Our need based versus not we have a protein based vaccines that are approved would die or at least have EUA in the U S and Europe .
So Pat sure, Yes, I mean, we've had those discussions with various health authorities.
And we will be using an active comparator reader, but we'll provide more details on which which vaccine we will be using as a competitor at a future time.
Okay.
If you don't mind, just one last question on the OTC program.
Can you talk a little bit about that trial design and what type of patients.
Sure.
Enrolling well.
Will you be able to will they be unstable background meds and if so will you be enrolling.
Control patients uncontrolled patients boats.
Yeah, we know that there at all it's approximately 24 adults and adolescence as we've communicated.
These are patients there, yes, and they're relatively stable.
But other details in that I don't have in front of me at this time pads is there. Other details you want to provide with respect to the patients sure yeah until phase III trial, Yeah, and again the phase II trial is a multi dose trial and we will be looking at you know all biomarkers associated with the reduction of our board.
Yeah, we will be looking at the enzymatic activity as well as.
Julia Genesis in these patients and we would again are some.
Some of the things that we've done in our phase one that we've talked about the we will be doing that also in our phase two trial and we are hoping to.
To see some changes in the Biomarkers that we're monitoring.
Okay. Thank you very much and congratulations on all the progress.
Joseph Payne: I will now pass the call back to Joe.
Thanks Pete.
Okay.
Thank you and next we will move on to Yasmin Rahimi with Piper Sandler.
Joseph Payne: Hey, thanks, Andy.
Joseph Payne: We're pleased to have another productive quarter, advancing our pipeline of mRNA vaccines and, therapeutics, and all the while judiciously controlling our operating expenses, as was just nicely summarized by Andy.
Joseph Payne: We remain highly encouraged about the potential of our clinical pipeline, and from our mRNA, vaccine therapeutic platforms.
Joseph Payne: We look forward to providing you with updates on our progress, everyone.
Joseph Payne: I'll now turn the call back to the operator to field any questions.
Yes.
Hi, this is lauren on for years.
Just a couple of questions for the approval on Vietnam.
It's looking like the approval contingent on the boost your data. So could you provide a bit of color will you need to boost your studies and if so how large would it be and what is the comparator and then the second question is for the approval.
You have the data.
And for Q2, two how confident are you in that and you can can you comment on the extent of the data you will generate.
Sure. So well the first clarification is we will not the prerequisite of booster data is rich.
Acquired only for full market authorization not for emergency use approval, so just to make that clarification.
What was the follow on question.
The size of the trial, we commented on with respect to the size of the trials of its smaller than 'twenty 400, So it's more cost efficient and faster to recruit and faster to complete by bifurcated. The trial. So we are at.
We haven't disclosed the exact numbers for each of these trials and the purpose again, one is going to be more immunogenicity focused with a comparator vaccine and the other is going to be a safety centric.
Okay and the question of guidance that you know that.
Active comparator again, what we mentioned in the previous question that we will be doing an active comparator trial, but we haven't disclosed which one yeah.
Operator: Thank you.
Okay, great. Thank you.
Thank you.
Next we'll take a question from Nick Abbott with Wells Fargo.
Operator: If you would like to ask a question, please signal by pressing star 1 on your telephone, keypad.
Operator: If you're using a speakerphone, please make sure your mute function is turned off to allow, your signal to reach our equipment.
Operator: You can press star 1 to ask a question, and we'll pause for just a moment to allow everyone, an opportunity to signal for questions.
Good afternoon.
<unk>.
Operator: And we'll take our first caller from Yasmin Rahimi with Piper Sandler.
So the first one Joe in your prepared comments, you say that you will expect.
Operator: My apologies.
Operator: One moment, please.
Operator: We'll actually take our first question from Pete Stavropoulos with, Canter Fitzgerald.
Six months I think antibody data against P. A.
If I heard that correctly.
Have you have you seen them.
One month three month data against <unk> five can you comment on that and there should we be expecting to see this $40 50 volt boost NAD levels three months.
Yeah, well, we sure hope so that'd be fantastic.
Pete Stavropoulos: Hi, it's Pete Stavropoulos.
We have not seen we have not seen any five data we have seen to be a one and two data that we shared today in our press release and what's notable about it is that it remains to be high. It doesn't have this potency drift that you see from other earlier vaccine.
Pete Stavropoulos: Thank you.
Pete Stavropoulos: Thanks for taking my question.
Pete Stavropoulos: So one question I have is, is there any more details that you can share in terms of the, trial design for the booster?
Pete Stavropoulos: I know that you previously shared that it was going to be about 2,400 patients for one study, but you mentioned you're going to run two smaller studies.
Joseph Payne: Can you, provide any color on the size of the studies and possibly key differences?
So so we are we remain hopeful that we will see a high a geometric mean full drives number this GMA foreign number with VA five, but we haven't seen any data and it is correct that the data. We're presently collecting is six month data for all variance, including VA five but also be a one to four and five.
Sure, Okay, and that's that's been coming Australia, sorry.
That's it.
Go ahead, it sounds like you're promoting a movie joke [laughter].
Right.
Joseph Payne: And will one or both studies be conducted perhaps with an Omicron-specific strain by valent candidates?
[laughter].
Joseph Payne: Any color would be appreciated.
Joseph Payne: Sure, that's a great question.
That's right.
Joseph Payne: Thanks, Pete, for joining the call.
These days.
Get this right. So one study is gonna be inactive.
Joseph Payne: Yes, we did emphasize that we're splitting, bifurcating the single trial of 2,400 people, into two smaller trials.
Have an active comparator.
The aim is divided by two maybe its two to one I don't know the other one is gonna be safety centric. So that's gonna be uncontrollably, that's just gonna be.
Joseph Payne: We haven't communicated the exact number of those trials in the specific countries, but one will be emphasized on comparative immunogenicity and the other will be safety-centric.
Joseph Payne: Okay.
Pete Stavropoulos: And in terms of, you know, when you talk to the different agencies, do they want sort of like a variation for a comparative arm, if they're asking for a comparative arm, meaning like, no, RNA-based, you know, versus now we have protein-based vaccines that are approved or at least have EUA in the U.S. and Europe?
Joseph Payne: Sure.
Whatever it is 1000 patients get a booster.
Joseph Payne: Yes.
Likely to think about it.
Yeah, Yeah that's.
Yes that is the right way to think about it.
And then I think.
Well first of all whether you expect to get the data pool cues.
You had a contract with us.
Zero.
Is it just a matter of sort of modifying that contract now you don't have to sell from scratch.
You could stop these studies pretty quickly.
Go ahead, yeah, Yeah, no that's correct yes.
Joseph Payne: I mean, you know, we've had those discussions with various health authorities, and we will be using an active comparative.
This euros that were using are well familiar with and we work with it before so we shouldn't anticipate any delays with the changes okay.
Pete Stavropoulos: But we'll provide more details on which vaccine we will be using as comparative at a future time.
And so then if you expect full approval early 'twenty three than it should have no data considerably be cold and considering you know you'd have to.
Joseph Payne: Okay.
Submit that to the regulators in Vietnam.
Pete Stavropoulos: And if you don't mind, just one last question on the OTC program.
We'd have to review those data.
That's correct and again just like the other vaccine manufacturers. You know this is a process that we went under law, which is a rolling submission. So we will have you know, whereas the various pieces of the regulatory filings and we'll be doing it a couple of things.
Pete Stavropoulos: You know, can you talk a little bit about that trial design?
Pete Stavropoulos: Like, you know, what type of patients you're enrolling?
Okay, and then just last one for me as you speak to the Kols.
Pete Stavropoulos: Will they be on stable background meds?
When you show them the data how do they.
Beauty's data.
First Gen vaccines and <unk>.
One might expect.
Daily specific vaccines.
Right so the.
It's a it's always a fun and interesting academic challenge to compare data with other vaccines, but when we shared this with kols and experts in the field. They do refer to the third shot vaccine data that are.
That other approved vaccines have been that have that.
A shared where they've shown titers in the three hundreds and the <unk> thousand 17. After one month and we've shown a GM fr of 44 after three months.
And.
And.
But there are there you know there's always nuances to this that people prosecute but the short answer to your question is the Kols are generally very encouraged by this data and no doubt.
Okay, great perfect. Thanks, Joe.
Thank you.
Yeah.
Thank you and next we'll move on to Siemens Fernandez.
Security.
Hi, guys. This is evan on for Janice.
Two on Covid and then a follow up on that you see.
I wanted to follow up on the trial design.
Pete Stavropoulos: And, you know, if so, will you be enrolling controlled patients, uncontrolled patients, both?
One word the conversations with regulators.
Before the trial starts for both trials.
Now the first trial starting <unk>.
What kind of population of the trial enrolled.
Adults and elderly and are there any steps you're taking to accelerate development.
And then given that the trials are centered around one five more.
Do you expect the trials and approval to support government sales from U S and European governments or would you need to do though I'm a constitute a variant and I guess if there are any.
Update on a potential development there.
Joseph Payne: Yeah, we know that it's approximately 24 adults and adolescents, as we've, communicated.
Yes, both great questions first of all we're working closely with the CRO.
Joseph Payne: These are patients.
And that interaction is going to be key with respect to the exact start date.
Joseph Payne: They're, yes, and they're relatively stable.
Joseph Payne: But other details than that, I don't have in front of me at this time.
Joseph Payne: Pat, is there other details you want to provide with respect to the patients?
Joseph Payne: Sure.
Joseph Payne: Yeah.
The initial start date for the booster trial.
And we have indicated that there's two separate countries, but it's at.
At least presently the same CRO so were confident there with respect to whether we're going to be focused on just <unk> 54 or building a bi valent strategy is going to be a databased those sorts of decisions.
We've touched on it already on the call today, if <unk> 50 force stands alone and is very strong it generating high GFR as against <unk>, five and we have a.
We're in a fortunate position, where we can continue to progress that as a stand alone monovalent vaccine against.
All the variance of concern if we do see any wavering data against B five or at future variance.
10 for example, there may be an appropriate time to to bridge in a bivariate strategy. We've been building that consistently throughout this process, we're prepared to move quickly there.
And we're working with potential pharmaceutical partners, our commercial strategic partners on that.
Thanks, One question on how do you see.
You highlighted in the remarks that <unk> identified.
Dozens of patients.
Can you clarify how many what claire or qualify for the trial.
And what's next before.
Yeah.
Yeah, it's a well at several dozen I just wanted to clarify that so.
Many of many and so what we're.
We've identified a large number of of OTC patients in Europe .
Specific through after pre screening type efforts. So we're in a good.
We're in a great position there so where we are now is in the the the official screening process and dosing of <unk> of these patients throughout the second half of this year.
And Pat and I can just clarify that that you know, we only need a fraction of the patients that we've identified for the trial. So that's all the color we can give you.
Yeah.
Great. Thank you guys.
Thank you.
You can't really make a move on cucumbers, Roger with Brookline capital markets.
Thanks for taking my questions. So MTN recommended using a biomarker length.
Five a spike protein it's a combination.
So how soon do you think that is going to come on board will you be comparing you by violent are.
In your planned trial.
Any bivalent strategy, we haven't disclosed because we've been working behind the scenes with our potential pharmaceutical partners, but we are prepared to proceed immediately if needed.
The desire I want to reiterate the plan a is to proceed with a C. T $1 54, as a standalone monovalent that has exceptional.
Broad spectrum activity because of the self amplifying mrna technology, there's something special about it at least that's what we're seeing with be a one and two and we hope that continues to be a four and five.
Joseph Payne: And the Phase 2 trial?
Okay.
And with regard to the plan. The second trial protocol 19 vaccine what is your expectation in terms of timing part of that.
Joseph Payne: Yeah.
Oh timing.
We said, we said we're collecting the data presently so we're in the midst of data collection.
Joseph Payne: And again, the Phase 2 trial is a multi-dose trial.
Joseph Payne: And, you know, we will be looking at, you know, all biomarkers associated with, reduction of ammonia.
So once the data set is completed we'll be able to see that data.
And operate accordingly after we.
The U S.
And with regard to the approval in Vietnam. This manufacturing done Remanufacturing.
That is being done by wind biochem.
Yeah, yes. It it it's not my understanding is that's not a requisite, but but that sure can.
<unk> be the case, we've already completed a portion of those runs already at the facility and in Hanoi I believe it's two of them already so but they've requested three just to establish reproducibility for full approval.
Okay, great. Thanks.
Joseph Payne: We'll be looking at enzymatic activity as well as ureogenesis in these patients.
Yeah.
Thank you.
Yeah.
Next we'll move on to Yale Jen with Laidlaw <unk> company.
Joseph Payne: And we will, again, some of the things that we've done in our Phase 1 that we've talked about, we will be doing also in our Phase 2 trial.
Good afternoon, and thanks for taking the question.
Joseph Payne: And we are hoping to see some changes in the biomarkers that we're monitoring.
Pete Stavropoulos: Okay.
The first question is that.
Given the AR. The booster study was started in the fourth quarter of this year and the full approval and then none but actually it will be the first.
Joseph Payne: Thank you very much, and congratulations on all the progress.
In 2023.
Just the bidding on full approval need to looked at the data from the upcoming booster study or they will using some of the studies you already have done.
As a reference the whole basis for potential approval.
Yeah, the combination of both where we've done some booster studies already.
And and there is additional booster studies that we're planning so both of these will be taken into consideration for full market authorization in Vietnam in early next year.
Okay, Great. That's helpful and one more question here is that the as you mentioned about the pharmaceutical company that was all in so Paulo the booster studies.
The question is have you guys reveal any details are what.
I think that the company would have.
Should the drug get approved.
In terms of their pet or ownership.
Well, we haven't provided any details on the pharmaceutical company that's it.
Provided that support.
Other than that we are of course highly respect them.
Yeah, Okay, great. Thanks, Bob I appreciate and congrats on the progress.
Yes. Thank you.
Joseph Payne: Thanks, Pete.
Thank you and next we'll move on to your home.
It can move it with Citigroup.
Joseph Payne: Thank you.
Operator: Next, we will move on to Yasmeen Rahimi with Piper Sandler.
Operator: Hey, Yasmeen.
Operator: Hi, this is Lauren on for YAS.
Hi, guys. Thanks, a lot for taking the questions I just wanted to dig in a little more on the path to booster approval in the U S and Europe . So for these two smaller registrational trials that you've discussed today can you just clarify what will be the primary vaccine series could it be any of Pfizer in return or no buybacks or are you going to restrict.
Some of those are one of those.
Then also regarding splitting into the two smaller trials versus the single trial with a 2400.
Was this based on some specific regulatory feedback or was this basically an internal decision that you guys took to increase the efficiency. Thank you.
Well. The this was based on regulatory feedback and senior management prioritization of efficiency, both time and cost. So it was everything all encompassing but yeah. It did involve specific regulatory feedback for sure with.
With respect to the the comparator vaccine that there is a list of approved vaccines that we can utilize we've we've identified which comparator vaccine, we're using but we're not disclosing that for competitive purposes.
Okay, but with the way the trial design is working you're going to give the competitor vaccine to both arms and then the one on one of the arms will have your booster.
Other arm I assume we will have.
Nothing or some other booster is that the right way to.
<unk> no no where we're just trying to do on a larger Scott scale, what we've already done on phase one to scale. So there's going to be a group. That's received unapproved vaccine as a primary series either two or three shots and then we're going to be providing.
Slot third slashed fourth shot.
Which we described as a booster and then we will be comparing that to a third or fourth shot up to the similar vaccine.
Gotcha Gotcha, Okay, and then Andy is there any reason why you are not able to disclose this pharma company at this point that that's helping you with the booster work and what will we learn the identity of the company at a future point or not.
Well, we hope so, but we have a non disclosure agreement with them. So unfortunately, we won't be able to do that at this point in time.
Okay and then one question about your your early R&D, there's obviously been a lot of talk about another another pathogen the monkey. The monkey pox are you guys doing any early discovery work on that front.
Yeah, I think that's a good question for Pat why don't you address our activities with respect to Monkey pox sure I mean, just like any emerging for a potential path as you know I think our preclinical team is always considering but that's not an actual program currently.
But that might change in the near future.
Got it okay. Thank you very much.
Thank you. Thank you.
Okay.
Thank you we'll take our last question from me.
Uh huh.
Raymond James.
Hey, Steve.
Hey, Thanks for taking the question actually I have a few the first one respectfully I think I'll have to ask this just we noticed the comment in the 10-Q.
Operator: Just a couple questions for the, approval in Vietnam.
Operator: So it's looking like the approval is contingent on the booster data, so could you provide a bit of color?
The completion of the clinical trials in Vietnam in their review.
Operator: Will you need two booster studies?
Operator: And if that's so, how large will they be and what is the comparator?
Operator: And then the second question is for the approval.
Operator: Will you need to have the data from the booster studies in 4222?
Be delayed substantially because of the originally exposed to scandal involved in COVID-19 test kits. So you referenced the reorganization of Moh, but obviously the 10-Q language is a bit more dubious. So I was hoping you could clarify.
Operator: How confident are you in that?
Operator: And you can can you comment on the extent of the data you will generate?
This is just about price gouging, and what we can sort of find online or or.
Operator: Thank you.
There's no impact of data integrity for instance for our 154 or your ability to leverage those different walks out of Vietnam, and if you have any exposure whatsoever to what's happening within the agency.
Joseph Payne: Sure.
Yeah, Thankfully, we haven't had any of that exposure.
This is a this reorganization is an affiliated with with our Arcturus and our vaccine. This is this is a something that's available in the public domain and I.
Feel free to to look into that but it.
It doesn't have any overlap or exposure relation to arcturus whatsoever.
Joseph Payne: So, well, the first clarification is we will not, the prerequisite of booster, data is required only for full market authorization, not for emergency use approval. So just to make that clarification.
Joseph Payne: What was the the follow-on question?
Joseph Payne: The size of the trial.
Okay, and then can you just clarify that thanks.
Joseph Payne: We commented on with respect to the size of the trials, it's smaller than 2400, so it's more cost-efficient and faster to recruit and faster to complete by bifurcating the trial.
That comment could you just clarify that the remaining data.
Joseph Payne: So we haven't disclosed the exact numbers for each of these trials.
They're already frankly for an EUA in Vietnam. It sounded like the CMC issues worked for full authorization.
Joseph Payne: And the purpose, again, one is going to be more immunogenicity focused with a comparator vaccine and the other is going to be a safety centric.
Just wanted to see if there's any data pending or if this is just a timing thing given the moh personnel issues.
Joseph Payne: Okay.
Joseph Payne: And the question again, you know, the active comparator, again, what we, mentioned in the previous question, that we will be doing an active comparator trial, but we haven't disclosed which one.
I think it's an oversimplification to say, it's just a timing thing, but that's how I'm going to address it. It is just a timing thing.
Joseph Payne: Yeah.
Operator: Okay, great.
Operator: Thank you.
Operator: Thank you.
Operator: So the first one, Joe, is the prepared comments you say that we'll expect six months, I think, antibody data against BA5, if I heard that correctly.
Operator: Thank you.
Operator: So, you know, have you seen one-month, three-month data against BA5?
Operator: Next, we'll take our question from Nick, Abbott with Wells Fargo.
Operator: Good afternoon.
Operator: Thanks for taking our questions.
Once their reorganization is complete and and we can go through the normal processes they've already provided feedback on the clinical section in the data. So we feel we feel comfort there.
Operator: Can you comment on that?
Operator: And we should be expecting to see this 40, 50-fold boost in NAB levels at three months for BA5.
Joseph Payne: Yeah, we sure hope so.
Joseph Payne: That'd be fantastic.
Joseph Payne: We have not seen any BA5 data.
And their feedback on the CMC was was more relative to the full market authorization that's correct.
Joseph Payne: We have seen BA1 and BA2 data that we've shared today in our press release.
Joseph Payne: And what's notable about it is that it remains to be high.
Joseph Payne: It doesn't have this potency drift that you see from other earlier, you know, vaccines.
Joseph Payne: So we remain hopeful that we'll see a high geometric mean full rise number, this GMFR number with BA5, but we haven't seen any data.
Joseph Payne: And it is correct that the data we're presently collecting is six month data for all variants, including BA5, but also BA1, 2, 4, and 5.
Joseph Payne: Okay?
Joseph Payne: Sure.
Joseph Payne: Okay.
Operator: Great.
Joseph Payne: And that's coming soon.
Okay, and then lastly, just given 021 is being suspended and the global energy.
Operator: Thank you guys.
Joseph Payne: Sorry.
Joseph Payne: That's it.
Joseph Payne: Go ahead.
Joseph Payne: That sounds like you're promoting a movie, Joe.
Joseph Payne: Right.
Joseph Payne: Just going back to the booster, if I get this right, the one study is going to have an active, comparator, so obviously the N is divided by 2, or maybe it's 2 to 1, I don't know.
Joseph Payne: The other one is going to be safety centric, so that's going to be uncontrolled, that's, just going to be whatever it is, 1,000 patients get the booster, is that the right way to think about it?
Previously mentioned decided not to proceed can you just comment on that.
Joseph Payne: Yeah, yes, that is the right way to think about it.
Joseph Payne: And then I think, you know, the last person asked about whether you expect to get the, data for Q, you know, you had a contract with a CRO, is it just a matter of sort of modifying that contract now, you don't have to start from scratch, you think you can start these studies pretty quickly?
Joseph Payne: Go ahead.
Joseph Payne: Yeah, no, that's correct.
Joseph Payne: Yeah, you know, the CROs that we're using are well familiar with, and we worked with, them before, so we shouldn't anticipate any delays with the changes.
Your level of confidence that you'll get forgiveness of the $47 million, Singapore alone or what needs to happen there.
Joseph Payne: Okay.
Joseph Payne: And so, then if you expect full approval early 23, then we should have, you know, data considering, before then, considering, you know, you'd have to submit that to the regulators in Vietnam, and they would have to review those data.
Joseph Payne: That's correct.
Joseph Payne: And again, just like the other vaccine manufacturers, you know, this is a process that we want to, undergo, which is a rolling submission, so we will have, you know, various pieces of the regulatory filings, and we'll be doing it simultaneously.
Joseph Payne: Okay.
Joseph Payne: And then just last one for me is, you know, you speak to the KOLs, do they, you know, when you show them these data, how do they view these data versus, you know, the current first-gen vaccines, and what one might expect from variant-specific vaccines?
Joseph Payne: Right.
Operator: Yeah.
Okay. Yeah, no. That's a good question we're in the process of negotiating that as we speak and we will certainly have more detailed fool you. Shortly but you are correct. It is a non recourse loan that is clearly and uniquely tied to one to one.
Joseph Payne: So, it's always a fun and interesting academic challenge to compare data with other vaccines, but when we shared this with KOLs and experts in the field, they do refer to the third-shot vaccine data that other approved vaccines have been, that have shared, where they've shown titers in the 300s and a GMFR of 17 after one month, and we've shown a GMFR of, 44 after three months.
Joseph Payne: And, but there, you know, there's always nuances to this that people prosecute, but the short, answer to your question is the KOLs are generally very encouraged by this data, no doubt.
Joseph Payne: Okay.
Joseph Payne: Great.
Joseph Payne: Terrific.
Operator: Thank you.
Joseph Payne: Thanks, Joe.
Joseph Payne: Thank you.
Operator: And next, we'll move on to Seamus Fernandez, Guggenheim Security.
Operator: Hi, guys.
Operator: This is Evan on for Seamus.
Operator: Thank you.
Operator: I have two on COVID and then a follow-up on OTC, so I wanted to follow up on the booster, trial design.
Operator: When were the conversations with regulators, and what's left before a trial starts for, both trials?
Operator: I believe you mentioned the first trial was starting in 4Q.
Operator: What kind of population will the trial enroll?
Operator: Will this only be adults and elderly, and are there any steps you're taking to accelerate, development?
Operator: And then, given that the trials are centered around 154, do you expect the trials and approval, to support government sales from U.S. and European governments, or would you need to develop a Omicron specific variant?
Operator: And, you know, I guess, is there any updates on potential development there?
So we will update the market with that information as soon as possible.
Operator: And next we'll move on to Kumar Raja with Brookline Capital Markets.
Joseph Payne: Yeah, both great questions.
Operator: Thanks for taking my questions.
Joseph Payne: First of all, we're working closely with the CRO and that, interaction is going to be key with respect to the exact start date, the initial start date for the booster trial. And we have indicated that there's two separate countries, but it's at least presently the same CRO.
Operator: So FDA has recommended using a bivalent 4-5 spike protein, as a combination.
Joseph Payne: So we're confident there.
Operator: So how soon do you think that is going to come on board?
Joseph Payne: With respect to whether we're going to be focused on just ARCT154, or building a bivalent strategy is going to be data-based, those sorts of decisions.
Joseph Payne: We've touched on it already on the call today.
Okay. Thank you.
Joseph Payne: If ARCT154 stands alone and is very strong at generating high GMFRs against BA5, then we're in a fortunate position where we can continue to progress that as a standalone monovalent vaccine against all the variants of concern.
Joseph Payne: If we do see any wavering data against BA5 or at future variants, BA10, for example, there may be an appropriate time to bridge in a bivariant strategy.
Joseph Payne: We've been building that consistently throughout this process.
Great. Thank you Steve.
Joseph Payne: We're prepared to move quickly there.
Joseph Payne: And we're working with potential pharmaceutical partners, commercial and strategic partners on that.
Operator: Thanks.
Operator: And one question on OTC.
Operator: Will you be comparing a bivalent in your planned trials?
Thank you and there are no further question I'd like to turn the conference back over to your presenters for any additional or closing remarks.
Operator: You know, you highlighted in the remarks that you've identified dozens of patients.
Joseph Payne: Any bivalent strategy, we haven't disclosed because we've been working behind the scenes with our potential pharmaceutical partners, but we are prepared to proceed immediately if needed.
Joseph Payne: Can you clarify how many would clarify or qualify for the trial?
Joseph Payne: The desire, I want to reiterate the plan A is to proceed with ARCT 154 as a standalone monovalent that has exceptional, you know, broad spectrum activity because of the self-amplifying mRNA technology.
Joseph Payne: And what's next before dosing begins?
Joseph Payne: There's something special about it.
Joseph Payne: Yeah, it's well, several dozen.
Joseph Payne: At least that's what we're seeing with BA1 and BA2.
Joseph Payne: I just want to clarify that.
Joseph Payne: And we hope that continues with BA4.
Operator: Could it be any of Pfizer, Moderna, or Novavax, or are you going to restrict it to some of, those or one of those?
Joseph Payne: So it's many and many.
Joseph Payne: Okay, and with regard to the planned second trial for COVID-19 vaccine, what is the expectation, in terms of timing for that?
Joseph Payne: And then also, regarding splitting into the two smaller trials versus the single trial, with the 2400, was this based on some specific regulatory feedback, or was this basically an internal decision that you guys took to increase the efficiency?
Joseph Payne: So we've identified a large number of OTC patients in Europe with specific through after pre-screening type efforts.
Joseph Payne: Timing, well, we've said we're collecting the data presently. So we're in the midst of data collection.
Joseph Payne: Well, this is based on regulatory feedback and senior management prioritization of efficiency, both time and cost, so it was everything all-encompassing, but yeah, it did involve specific regulatory feedback, for sure.
Joseph Payne: So we're in a great position there.
Joseph Payne: So once the data set is completed, we'll be able to see that data and operate accordingly, after we view it.
Joseph Payne: With respect to the comparator vaccine, there's a list of approved vaccines that we can utilize.
Joseph Payne: So where we are now is in the official screening process and dosing of these patients throughout the second half of this year.
Joseph Payne: And with regard to the approval in Vietnam, these manufacturing runs, three manufacturing, runs, that is being done by WinBioCare?
Joseph Payne: We've identified which comparator vaccine we're using, but we're not disclosing that for competitive purposes.
Joseph Payne: And I can just clarify that, you know, we only need a fraction of the patients that we've identified for the trial.
Joseph Payne: Yes, it's not.
Joseph Payne: Okay, but the way the trial design is working, you're going to give the competitor vaccine to both arms, and then one of the arms will have your booster, the other arm, I assume, will have nothing or some other booster?
Joseph Payne: So we, that's all the color we can give.
Joseph Payne: Is that the right way to conceptualize this?
Joseph Payne: My understanding is that's not a requisite, but that sure can be the case.
Well that's it everybody. Thank you we'll stay in touch for sure to reach out if you have any further questions and bye for now.
Joseph Payne: Yeah.
Joseph Payne: No, we're just trying to do on a larger scale what we've already done on phase 1-2 scale. So there's going to be a group that's received an approved vaccine as a primary series, either two or three shots, and then we're going to be providing a third-slash-fourth shot, which we describe as a booster, and then we'll be comparing that to a third- or fourth-shot of the similar vaccine.
Joseph Payne: We've already completed a portion of those runs already at the facility in Hanoi.
Joseph Payne: Gotcha, gotcha, okay.
Joseph Payne: I believe it's two of them already, but they've requested three just to establish reproducibility, for full approval.
Joseph Payne: And then, Andy, is there any reason why you are not able to disclose this pharma company, at this point that's helping you with the booster work?
Joseph Payne: Okay, great.
Operator: And that does conclude today's teleconference.
Joseph Payne: Will we learn the identity of that company at a future point or not?
Operator: Thanks.
Joseph Payne: Well, we hope so, but we have a non-disclosure agreement with them, so unfortunately, we won't be able to do that at this point in time.
Operator: Yeah.
Joseph Payne: Okay, and then one question about your early R&D.
Operator: Thank you.
Joseph Payne: There's obviously been a lot of talk about, another pathogen, the monkeypox.
Operator: Next, we'll move on to Yale Jin with Laidlaw and Company.
Joseph Payne: Are you guys doing any early discovery work in that front?
Operator: Good afternoon, and thanks for taking the questions.
And that does conclude today's teleconference, and we do appreciate that participation you may now disconnect.
Joseph Payne: Yeah, I think that's a good question for Pat.
Operator: The first question is that given the booster study was started in the fourth quarter of, this year and the potential full approval in Vietnam, potentially will be in 2023, does the Vietnam full approval need to look at the data from the upcoming booster study, or they were using some of the studies you already have done as a reference or basis for potential approval?
Joseph Payne: I want you to address, our activities with respect to monkeypox.
Joseph Payne: Yeah, the combination of both.
Operator: We do appreciate that participation.
Joseph Payne: Sure, I mean, just like any emerging potential pathogen, you know, I think our preclinical team is always considering, but that's not an active program currently, but that might change in the near future.
Joseph Payne: We've done some booster studies already, and there's additional booster studies that we're, planning.
You may now disconnect.
Joseph Payne: Got it, okay.
Joseph Payne: So both of these will be taken into consideration for full market authorization in Vietnam early, next year.
Operator: Thank you very much.
Operator: Okay, great.
Operator: Thank you.
Operator: That's helpful.
Operator: Thank you.
Operator: And one more question here is that, as you mentioned about the pharmaceutical company, that was supporting some part of the booster studies, the question is, have you guys reviewed any details what this particular company would have should the drug get approved in terms of their benefits or ownership?
Operator: Thank you.
Joseph Payne: Well, we haven't provided any details on the pharmaceutical company that has provided that, support, other than that we, of course, highly respect them.
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Operator: We'll take our last question from Steve Seathouse with, Raymond James.
Joseph Payne: Yep.
Operator: Hey, Steve.
Operator: Okay, great.
Operator: Hey, thanks for taking the question.
Operator: Thanks, Bob.
Operator: Actually, I have a few.
Operator: I appreciate it, and congrats on the progress.
Operator: The first one, respectfully, I think I have to ask this.
Joseph Payne: Yes, thank you.
Operator: We noticed a comment in the 10-Q, that the completion of the clinical trials in Vietnam and their review may be delayed substantially because of a recently exposed scandal involving COVID-19 test kits.
Operator: Thank you.
Joseph Payne: So, you referenced the reorganization of MOH, but obviously, the 10-Q language is a bit more dubious.
Operator: And next, we'll move on to Ugo, Dr. Movitz with Citigroup.
Joseph Payne: So, I was hoping you could clarify that this is just about price gouging and what we can sort of find online, and that there's no impact to data integrity, for instance, for ARCT154, or your ability to leverage those data outside of Vietnam, and if you have any exposure whatsoever to what's happening within the agency.
Operator: Hi, guys.
Joseph Payne: Yeah, thankfully we haven't had any of that exposure.
Operator: Thanks a lot for taking the questions.
Joseph Payne: Yeah, this is a, this reorganization, is not affiliated with, with Arcturus and our vaccine.
Operator: I just want to dig in a little more onto the path to booster approval in the U.S. and Europe.
Joseph Payne: This is, this is something that's available in the public domain.
Operator: So for these two smaller registrational trials that you've discussed today, can you just, clarify what will be the primary vaccine series?
Joseph Payne: And I feel free to, to look into that.
Joseph Payne: But it doesn't have any overlap or exposure relation to Arcturus whatsoever.
Okay.
Joseph Payne: Okay.
Joseph Payne: And then can you just clarify that, thanks for that comment.
Joseph Payne: Could you just clarify, that the remaining data, whether there are any, frankly, for an EUA in Vietnam, it sounds like the CMC issues were for full authorization.
Joseph Payne: Just wanted to see if there's any data pending or if this is just a timing thing, given the MOH personnel issues.
Joseph Payne: I think it's an oversimplification to say it's just a timing thing, but that's how I'm, going to address it.
Joseph Payne: It is just a timing thing.
Joseph Payne: Once their reorganization is complete and, and we can go through the, the normal processes, they've already provided feedback on the clinical section and the data.
Joseph Payne: So we feel, we feel comfort there.
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Joseph Payne: And their feedback on the CMC was, was more relative to the full market authorization. That's correct.
Joseph Payne: Okay.
Joseph Payne: And then lastly, just given O2-1 is being suspended and the global entity, previously mentioned decided not to proceed.
Joseph Payne: Can you just comment on the level of confidence that you'll get forgiveness of the $47 million Singapore loan or what needs to happen there?
Joseph Payne: Okay.
Joseph Payne: Yeah.
Joseph Payne: No, that's a good question.
Joseph Payne: We're in the process of negotiating that as we speak, and we'll certainly have more details for you shortly, but you are correct.
Joseph Payne: It is a non-recourse loan that is clearly and uniquely tied to O2-1.
Joseph Payne: And so we will update the market with that information as soon as possible.
Joseph Payne: Okay.
Okay.
Joseph Payne: Thank you.
Joseph Payne: Great.
Yeah.
Joseph Payne: Thank you, Steve.
Operator: Thank you.
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Operator: And there are no further questions.
Joseph Payne: I'd like to turn the conference back over to, your presenters for any additional or closing remarks.
Joseph Payne: Well, that's it, everybody.
Joseph Payne: Thank you.
Joseph Payne: We'll stay in touch.
Joseph Payne: Be sure to reach out if you, have any further questions and bye for now.