Q1 2022 Arcturus Therapeutics Holdings Inc Earnings Call
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Good day and welcome to the Arcturus Therapeutics first quarter 2022 earnings call Today's conference is being recorded.
Operator: Good day, and welcome to the Arcturus Therapeutics First Quarter 2022 Earnings Call. Today's conference is being recorded. At this time, I'd like to turn the conference over to Deepankar Roy, Senior Director of Investor Relations. Please go ahead, sir.
At this time like to turn the comments over to depend car ROI Senior director of Investor Relations. Please go ahead Sir.
Thank you James Good afternoon, and welcome to Arcturus Therapeutics first quarter 2022 financial results and corporate update call. Thank you all for joining US today's call will be led by Joseph <unk>.
Deepankar Roy: Thank you, James. Good afternoon, and welcome to Arcturus Therapeutics' first quarter 2022 financial results and corporate update call. Thank you all for joining us. Today's call will be led by Joseph Payne, our President and CEO, and Andy Sassine, our CFO.
Deepankar Roy: Dr. Pat Chivukula, our CSO and COO, will join in for the Q&A session. 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. 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.
President and CEO and Andy <unk>, our CFO .
<unk> will join us for the Q&A session 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 forward looking statements are not guarantees of performance.
They involve known and unknown risks uncertainties and assumptions.
You may cause actual results performance and achievements to differ materially from those expressed or implied by the statement. Please see the forward looking statement disclaimer on the company's press release issued earlier today as well as the risk factors section in our forms 10-Q, and 10-K filed with SEC.
Deepankar Roy: Please see the forward-looking statement disclaimer in 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 it. In addition, any forward-looking statements represent our views only as of the date such statements are made, May 9th, 2021. Arcturus specifically disclaims any obligation to update such statements to reflect future information, events, or circumstances.
In addition, any forward looking statements represent our views only as of the date such statements Amit mainline 2022.
<unk>, 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.
Joseph Payne: With that, I'll now turn the call over to Joe. Hey, thank you, Dipankar. Good afternoon to all.
Joseph Payne: Thank you for joining Arcturus's Q1 quarterly call. My comments today initially capture a list of summary points for the quarter, and I'll follow that up with additional detail per program before I turn the time over to Andy for financial updates. This quarter has been highlighted by several key achievements.
Hey, Thank you to bank or good afternoon to all thank you for joining our tourist as Q1 quarterly call. My comments today initially capture a list a summary of points for the for the quarter and I'll follow that up with additional detail per program before I turn the time over to Andy for financial updates.
This quarter has been highlighted by several key achievements. Indeed, we've made significant progress advancing our pipeline of mrna based vaccines and therapeutics. So let's begin with <unk> $1 54. This is our most advanced Covid vaccine candidate that has shown impressive activity against Sars Cov, two both as a prime.
Joseph Payne: Indeed, we've made significant progress advancing our pipeline of mRNA-based vaccines and therapeutics. So let's begin with ARCT154. This is our most advanced COVID vaccine candidate that has shown impressive activity against SARS-CoV-2, both as a primary vaccine and as a boost. We're pleased with the promising results coming from a Phase 1-2 study evaluating ARCT-154 and its potential as a booster in individuals primed with comorbidities. The boost with ARCT154 generated high booster numbers. These are the geometric mean fold rises or GMFRs in neutralizing antibody titers.
Mary vaccine and as a booster we're pleased with the promising results coming from our phase one slash two study evaluating <unk> $1 54, and its potential as a booster.
Individuals' prime to a commonality.
The boost with <unk> $1 54 generated high booster numbers. These are the geometric mean fold rises or GMA bars in neutralizing antibody titers and we'll share more of those results in a moment.
Joseph Payne: And we'll share more of those results in a moment. Importantly, this quarter, we learned from VinBioCare that the Phase 1-2-3 study has met its primary endpoint objective of vaccine efficacy. And that ARCT 154 demonstrated very strong protection against severe and fatal COVID, even when up against the most challenging variants that have escaped other vaccines like Delta and Omicron. We believe this is a significant milestone for the company and for the field of RNA vaccines.
Importantly, this quarter, we learned from Vin bio care that the phase one slash two slash three study has met its primary endpoint of objective of vaccine efficacy.
And that <unk> $1 54 demonstrated very strong protection against severe and fatal COVID-19 disease, even went up against the most challenging variance that have escaped other vaccines like delta and omicron.
We believe this is a significant milestone for the company and for the field of RNA vaccines. This represents the first time ever that phase III vaccine efficacy endpoint has been achieved with a low dose self amplifying mrna vaccine showing that we can prevent COVID-19 symptomatic disease.
Joseph Payne: This represents the first time ever that a Phase 3 vaccine efficacy endpoint has been achieved with a low-dose, self-amplifying mRNA vaccine, showing that we can prevent COVID-19 symptomatic disease, severe disease, and death. With over 19,000 participants involved in this study and a comparable safety profile in the ARCT-154 vaccinated arm and placebo arms, respectively. We view this as important validation of our self-amplifying mRNA technology platform and its applicability towards developing vaccines. The vaccine efficacy data, along with the availability of safety or along with the available safety and immunogenicity data, has been shared with the Vietnam Ministry of Health, and we anticipate a decision on the EUA in the near future. The application for full market authorization is expected to be filed later this year.
Severe disease and death.
With over 19000 participants involved in this study and a comparable safety profile in the RCT $1 54, vaccinated arm and placebo arms respectively.
We view this as important validation of our self amplifying mrna technology platform and its applicability towards developing vaccines.
The vaccine efficacy data along with the availability safety along.
Along with the available safety and Immunogenicity data has been shared with the Vietnam Ministry of Health and we anticipate a decision around the EUA in the near future. The application for full market authorization is expected to be filed later this year.
We're also pleased to share that Arcturus has provided an expression of interest letter to the world Health organization to potentially include <unk> $1 54, and its emergency use listing process.
Joseph Payne: We're also pleased to share that Arcturus has provided an expression of interest letter to the World Health Organization to potentially include ARCT 154 in its emergency use listing process. We're sincerely thankful to the WHO and to the Vietnam Ministry of Health for their support and encouragement in this process. Now, coming back to our booster trial.
Sincerely thankful to the W. H O and to the Vietnam Ministry of health for their support and encouragement in this process.
Coming back to our booster trial last week, we released additional data from our phase one two booster clinical trial here in the United States and Singapore, showing the durability of neutralizing antibody response extending to at least three months. After a five microgram <unk> hundred 54 booster dosing fault.
Joseph Payne: Last week, we released additional data from our Phase 1-2 booster clinical trial here in the United States and Singapore showing the durability of the neutralizing antibody response extending to at least three months after a 5-microgram ARCT154 booster dose following primary vaccination with Comirnaty. We announced new data today that our low-dose, self-amplifying mRNA vaccine shows a 46-fold rise in neutralizing antibody levels for Omicron B.A.2, All of these data, including those from the current Omicron variants, provide important clinical validations and highlight ARCT154's potential as an effective booster candidate against a broad range of variants of SARS-CoV-2.
Boeing primary vaccination with commonality.
We announced.
<unk> new data today that are too low dose self amplifying mrna vaccine shows a 46 fold rise in neutralizing antibody levels for <unk> and that's a month or 2009 days post boost all of these data, including those from the current omicron variance provide important clinical.
Nations and highlight <unk> $1 54 has potential as an effect of booster candidate against a broad range of variance of Sars Cov two.
Joseph Payne: As the world transitions now into an endemic period of COVID, the booster market represents the most significant commercial opportunity, and these data are a step towards ARCT 154 realizing that. We have begun our startup activities toward a global registrational booster trial and look forward to starting dosing soon. Now, let's get into the details now about the ARCT 154 clinical results. I'll begin with a recap of the ARCT 154 Phase 3 efficacy data that we reported a few weeks ago. The ongoing Phase 1, 2, and 3 registrational study sponsored by Arcturus' collaborative in BioCare enrolled over 19,000 adult participants in Vietnam, including a large percentage of individuals at higher risk of severe complications of COVID.
As the world transitions now into an endemic period of Covid. The booster market represents the most significant commercial opportunity.
These data are a step towards <unk> $1 54, realizing that potential we have begun our startup activities toward a global Registrational booster trial and look forward to starting dosing soon.
Now, let's get into the details now of about the <unk> $1 54 clinical results I'll begin with a recap of the <unk> $1 54 phase III efficacy data that we reported a few weeks ago.
The ongoing phase 123, Registrational study sponsored by Arcturus is collaborative and bio care enrolled over 19000 adult participants in Vietnam, including a large percentage of individuals at higher risk of severe complications of COVID-19 disease.
Joseph Payne: The Phase 3 Placebo-Controlled Vaccine Efficacy portion of the study enrolled over 16,000 participants. Evaluation of vaccine efficacy demonstrated that the study met its primary endpoint of prevention of virologically confirmed COVID-19 disease. Data show that in an analysis of COVID-19 cases accrued between seven days and 56 days after completion of a two-dose vaccination.
The phase III placebo controlled vaccine efficacy portion of the study enrolled over 16000 participants.
Evaluation of vaccine efficacy demonstrated that the study met its primary endpoint of prevention of Virological confirmed COVID-19 disease.
Data show that in an analysis of COVID-19 cases accrued between seven days and 56 days after completion of a two dose vaccination series.
Joseph Payne: Two 5-microgram doses of ARCT154 demonstrated 55% vaccine efficacy for preventing symptomatic COVID-19. Key secondary endpoints evaluating severe COVID-19 disease, including COVID-19 related deaths, were analyzed and demonstrated over 95% efficacy for Prevention of Severe COVID, including death. Notably, this study was conducted during a period characterized by the dominance of the Delta and Omicron variants
Two five microgram doses of <unk> $1, 54 demonstrated 55% vaccine efficacy for preventing symptomatic COVID-19.
Key secondary endpoints evaluating severe COVID-19 disease, including COVID-19 related deaths was analyzed and demonstrated over 95% efficacy for prevention of severe Covid <unk>.
Including deaths.
Notably this study was conducted during a period characterized by the dominance of the Delta and Omicron variance. We are currently conducting additional analyses of the swaps taken from the Covid cases in this study to characterize which SAR <unk> strains led to infections in this study and particular.
Joseph Payne: We are currently conducting additional analyses of the swabs taken from the COVID cases in this study to characterize which SARS-CoV strains led to infections in them, and particularly to confirm if there's further evidence of protection against these two very challenging variants of concern that have escaped protection from many other vaccines. The data from the study are also reassuring from the safety and tolerability perspective. The incidence of unsolicited adverse events in the ARCT-154 and placebo groups were comparable in the safety data collected from over 17,000 participants enrolled in the Phase I, II, and III through one month post-full vaccination. In addition, the frequency and severity of those events were generally consistent upon the second administration. No cases of myocarditis or pericarditis have been reported.
Really to confirm if there is further evidence of protection against these two very challenging variance of concern that have escaped protection for many other vaccines.
The data from the study are also reassuring from the safety and Tolerability perspective.
The incidence of unnecessary listed adverse events in the <unk> $1 54 in placebo group are comparable in the safety data collected from over 17000 participants enrolled in the phase one two and three through one month post fall vaccination.
In addition, the frequency and severity of those events was generally consistent upon the second administration.
No cases of myocarditis or pericarditis had been reported.
Joseph Payne: Although the study size was not large enough to detect large numbers of these rare events, the absence of such events in a study of this size is still common. We and our partners at VinBioCare will continue to carefully monitor the safety of participants in this ongoing study. The study met its primary objectives in both the demonstration of acceptable immunogenicity and safety in the Phase 1, 2, and 3a readouts and in meeting the protocol specified vaccine efficacy endpoint in Phase 3b. These results have been submitted by VinBioCare to the Vietnam Ministry of Health for the express purpose of seeking emergency use authorization.
Although the study size was not large enough to detect large numbers of these rare events.
<unk> sense of such events in the study of this size is still comforting.
We and our partners have been bio care will continue to carefully monitor the safety of participants in this ongoing study.
The study met its primary objectives in both the demonstration of acceptable Immunogenicity and safety in the phase 123, a readout.
And in meeting the protocol specified vaccine efficacy endpoint in phase III.
These results have been submitted by then bio care to the Vietnam Ministry of Health for the express purpose of seeking emergency use authorization.
Joseph Payne: We also continue to work with VinBioCare to support filing for full marketing authorization to be completed later this year. We are very encouraged by the data from this Phase 1, 2, and 3 study. With two low-dose administrations of the ARCT154 vaccine, effective prevention of COVID disease, and particularly severe and fatal disease, has been shown.
We also continue to work with <unk> to support filing for full marketing authorization to be completed later this year.
We are very encouraged by.
The data from this phase 123 study.
With two low dose administrations of the air <unk> $1 54 vaccine effective prevention of Covid disease, and particular, and particularly severe and fatal disease has been shown.
Joseph Payne: The safety profile was well-tolerated, and we view these findings as an important illustration of the potential of our self-amplifying mRNA platform, which includes the STAR technology, STAR is the trademark for self-transcribing and replicating RNA technology, and the Lunar Delivery Platform, our trade secret know-how and proprietary manufacturing processes, including purification formulation and lyophilization methods toward the mRNA drug substance and the vaccine drug product Given the continued circulation of SARS-CoV-2 and the discussion of its movement into an endemic phase, we are moving forward with the development of ARCT154 as a booster vaccine.
The safety profile was well tolerated and we view these findings as an important illustration of the potential of our self amplifying mrna platform, which includes the star technology and stars the trademark for self transcribing and replicating RNA technology and the lunar delivery platform our tray.
Secret Knowhow and proprietary manufacturing processes, including purification formulation and Lyophilize Asian methods towards the mrna drug substance and the vaccine drug product.
Given the continued circulation of SAR C O V to in the discussion of its movement into an endemic phase we are moving forward with development of <unk> $1 54, as a booster vaccine and.
Joseph Payne: In an ongoing Phase 1-2 study evaluating ARCT-154, given as a 5-microgram booster dose approximately 5 months after primary vaccination with Comirnat, the ARCT-154 vaccine responses are promising. The Acceptable Reactogenicity Accompanied by a Robust Immune Response Continues to Build Our Enthusiasm toward the Next Step. We've observed noteworthy neutralizing antibody responses soon after vaccination with ARCT154 and now have shown the high GMFRs to be sustained through three months after vaccination. In addition, the neutralizing antibody responses to the parent strain of the vaccine, also known as D614G, and antibody responses against a panel of SARS-CoV-2 variant strains demonstrated antibody responses that were 13 to 30-fold elevated after three months, 91 days to be exact, over day one baselines for all SARS-CoV-2 strains tested on the panel.
In an ongoing phase one two study evaluating <unk> 154, given as a five microgram booster dose approximately five months after primary vaccination with commonality.
The E. R. C T $1 54 vaccine responses are promising the.
Acceptable react to Genesis.
Company by robust immune responses continue to build our enthusiasm toward next steps we've observed noteworthy neutralizing antibody responses soon after vaccination with <unk> $1 54, and now have shown the high G. M F ours, there to be sustained through three.
Months after vaccination. In addition, the neutralizing antibody responses to the parents strain of the vaccine also known as <unk> 6004, G and antibody responses against a panel of SAR COPD. Two variant strains have demonstrated anti bot antibody responses that have remained 13 to 30.
Fold elevated over three months after three months 91 days to be exact over day, one baselines for all Saar C. O V. Two strains tested on the panel.
We've Ah.
Joseph Payne: We're even further encouraged by additional data from this study demonstrating strong neutralizing antibody activity against the Omicron B.A.2 variant upon boosting with ARCT154, with a 46-fold increase seen in NAB activity at day 29 over day one baseline. And this data, paired with the 54-fold increase in neutralizing antibodies against BA1 over the 29-day window that we reported earlier this year, generates an appealing profile for ARCT154 as a broadly immunogenic and next-generation low-dose booster.
Or even or even further encouraged by additional data from this study demonstrate demonstrating strong neutralizing antibody activity against the omicron two variant upon boosting with <unk> 154, with a 46 fold increase seen in Nab activity at day 29 over day one baseline.
This data paired with the 54 fold increase in neutralizing antibodies against <unk> one over the 2009 day window that we reported earlier this year generates an appealing profile for <unk> $1 54, as a broadly immunogenic and next generation low dose booster.
<unk>.
Joseph Payne: Given these collective data, we have been in discussions with major regulatory agencies, including the FDA, the MHRA, and the EMA. Regarding the path to pivotal trial and future approval as a booster, these conversations have informed the design elements of our pivotal booster trial intended to support global registration. In parallel with our development activities, our global manufacturing footprint continues to advance with ongoing technology transfer activities to VinBioCare's manufacturing facilities.
Given these collective data we have been in discussions with major regulatory agencies, including the FDA, the MH or a an MAA.
Regarding the path to pivotal trial in future approval as a booster. These conversations have informed the design elements of our pivotal booster trial intended to support global registration.
In parallel with our development activities, our global manufacturing footprint continues to advance with ongoing technology transfer activities to Vince <unk> manufacturing facility and we expect it to become operational this year with a capacity to manufacture 200 million doses annually.
Joseph Payne: And we expect it to become operational this year with a capacity to manufacture 200 million doses a year. The development of the Hanoi Manufacturing Facility is fully sponsored and funded by VIM Biocare, and we are looking forward to the next steps in our collaboration to potentially add Hanoi as an international hub for manufacturing self-amplifying mRNA methods. Meanwhile, our self-amplifying mRNA influenza vaccine program has continued its preclinical work.
The development of the Hawaii manufacturing facility is fully sponsored and funded by the <unk> bio care and we are looking forward to the next steps in our collaboration to potentially add Hanoi as an international hub for manufacturing self amplifying mrna medicines.
Our self amplifying mrna influenza vaccine program has continued its preclinical work and.
Joseph Payne: And we expect to make a final decision on 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 influenza vaccines, with its comparatively lower dose than other mRNA vaccines in development. We hope to understand if there are other meaningful ways to differentiate in the influence of vaccines.
And 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 current currently marketed influenza vaccines.
With its comparatively lower dose than other mrna vaccines in development, we hope to understand if there are other meaningful ways to differentiate in the influenza vaccine space.
Well now I'd like to shift from our vaccine franchise over to our therapeutic mrna pipeline programs.
Joseph Payne: Well, now I'd like to shift from our vaccine franchise over to our therapeutic mRNA pipeline program. Indeed, we also continue to make progress in our mRNA therapeutic programs. I'll begin with an update on ARCT 810, our therapeutic candidate for ornithine transcarbamylase deficiency, or OTC deficiency. OTC is the number one urea cycle disorder. It's a rare and serious disease with no approved treatments that address the root cause of the disease. Our approach aims to augment the deficient or absent OTC enzyme in the liver of patients living with this disease.
Ah Indeed, we also continued to make progress in our mrna therapeutic programs I'll begin with an update on <unk>, our therapeutic candidate for ornithine, transcribe amylase deficiency or OTC deficiency.
Joseph Payne: ARCT 810 has the potential to restore urea cycle activity, preventing or slowing the progression of neurological damage, and potentially expanding dietary options and improving quality of life for people living with it. We've obtained approval from the United Kingdom's HRA, or Health Research Authority, and other European authorities to initiate a Phase II multiple-dose clinical trial for ARCT 810, designed to enroll 24 adolescents and adults. Patient screening and enrollment is underway at multiple sites in the United Kingdom, Spain, and Belgium, with other countries soon to follow.
OTC is the number one urea cycle disorder, it's a rare and serious disease with no approved treatments that address the root cause of the disease.
Our approach aims to augment the deficient or absent OTC enzyme in the liver of patients living with this disease.
<unk> 10 has the potential to restore urea cycle activity, preventing or slowing the progression of neurological damage and potentially expanding dietary options and improving on quality of life for people living with this condition.
Well, we've obtained approval from the United Kingdom from the United Kingdom's HRA 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.
Patient screening and enrollment is underway at multiple sites in the United Kingdom, Spain, and Belgium with other countries soon to follow.
Joseph Payne: We anticipate that phase two enrollment will commence before the end of the second quarter, and we expect to obtain interim proof of concept data in the second half of 2022 from a subset of participants. We have also completed dosing of the first cohort of our Ascending Dose Phase 1B study here in the U.S., wherein the participants received a dose of 0.2 mg per kg of ARCTA-10.
We anticipate that phase two enrollment will commence before the end of the second quarter and we expect to obtain in term proof of concept data in the second half of 2022 from a subset of participants.
We've also completed dosing of the first cohort of our ascending dose phase one B study here in the U S. Wherein the participants received a dose of 0.2 milligrams per kilogram of <unk> 10.
We're pleased also to report that the data Safety Review Committee has recommended continuation of the study without modification.
Joseph Payne: We're pleased also to report that the Data Safety Review Committee has recommended continuation of this study without modification. Therefore, the dosing of the screened second cohort will begin in... Moving briefly now to our Cystic Fibrosis Program. We've continued to progress the necessary preclinical studies to enable ARCT-032, our inhaled messenger RNA therapeutic candidate for cystic fibrosis, to move into clinical studies, and we anticipate the submission of a clinical trial application, or CTA, for ARCT-032 in the third quarter of 2022.
Therefore, the dosing of the screen second cohort will begin imminently.
Moving briefly now to our cystic fibrosis program, we've continued to progress the necessary preclinical studies to enable a RCT or <unk> 32. This is our inhaled messenger RNA therapeutic candidate for cystic fibrosis to move into clinical studies, and we anticipate the submission of a clinical trial application or Cta.
For a R. C T O three or <unk> 32 in the third quarter of 2022.
Joseph Payne: In addition to our internally developed pipeline programs, Arcturus continues to support our partnered or licensed therapeutic programs. The most advanced of these is a very promising therapeutic candidate for glycogen storage disease, which continues to be evaluated by Ultragenyx in a Phase 1-2 study.
In addition to our internally developed pipeline programs are tourists continues to support our partner or license therapeutic programs. The most advanced of these is a very promising therapeutic candidate for glycogen storage disease, which continues to be evaluated by ultra <unk> in a phase one slash II study.
Well with that I will now pass the call onto Andy sustain our CFO to provide the financial updates.
Andrew Sassine: Well, with that, I will now pass the call on to Andy Sassine, our CFO, to provide the financial update. Thank you, Joe, and good afternoon, everyone. The press release issued earlier today includes financial statements for the first quarter of 2022 and provides a summary and analysis of sequential financial performance. Please refer to our 10-Q for more details on financial performance. I will go over our financials and present some operating metrics as we continue to transition to a late-stage clinical company with several assets in our pipeline. I will also provide some details regarding our manufacturing strategy as we prepare for the potential of emergency use authorization in Vietnam. Finally, I will provide some insights regarding our cash position and expected run rate.
Thank you Joe and good afternoon, everyone.
Press release issued earlier today includes financial statements for the first quarter of 2022 and provides a summary and analysis of sequential financial performance.
Please reference our 10-Q for more details on the financial performance.
I will go over our financial and presents some operating metrics as we continue to transition to a late stage clinical company with several assets in our pipeline.
I will also provide some details regarding our manufacturing strategy as we prepare for the potential of emergency use authorization and Hum.
Finally, I will provide some insights regarding our cash position and expected run rate.
You heard Joe mentioned, we had a very productive first quarter, including highly encouraging efficacy trial and booster durability results for <unk> 154.
Andrew Sassine: As you heard Joe mention, we had a very productive first quarter, including highly encouraging efficacy trials and boosted durability results for ARCT 154. As you know, we've partnered our ARCT 154 Next Generation Lunar COVID-19 Vaccine Candidate in Vietnam with Den BioCare. VennBioCare is part of the Venn Group, which is one of Vietnam's largest corporations and is sponsoring and funding our Phase 1, 2, and 3 study in Vietnam targeting COVID-19 and the variants of concern.
As you know we've partnered or they are C. T 154 next generation lunar a COVID-19 vaccine candidate.
In Vietnam within Bioterror and.
Then bio care is part of the <unk> group, which was one of Vietnam largest corporations in the sponsoring and funding our phase 123 study in Vietnam targeting COVID-19, and the variance of concern.
This partnership includes the trial in collaboration around building a vaccine manufacturing facility in Illinois.
Andrew Sassine: This partnership includes a trial and collaboration around building a vaccine manufacturing facility in Hanoi, resulting in significant cost savings for Arcturus. Our technology transfer activities remain on track for the facility to become operational later this year with a 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 collaboration for the first quarter of 2022 were $5.2 million, which was relatively consistent with the December quarter. Our operating expenses for the first quarter were $55.6 million, which declined quarter over quarter, an increase from the quarter ended December 2021.
Noting and significant cost savings for our tourists.
Our technology transfer activities remain on track for the facility to become operational later this year with the 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 first quarter of 2022 was $5 2 million, which was relatively consistent with the December quarter.
Operating expenses for the first quarter were $55 6 million.
It declined quarter over quarter, an increase from the quarter ended December 2021.
This increase was primarily due to onetime manufacturing and CMC quality run for 154 vaccine program.
Andrew Sassine: This increase was primarily due to one-time manufacturing and CMC quality runs for our 154 vaccine program and an increase in costs associated with the startup activities towards initiation of the Booster Trial. In January 2022, 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 a Lunar COVID-19 vaccine candidate as a booster. The net loss for the first quarter of 2022 was approximately $51.2 million, or $1.94 per share.
And an increase in costs associated with the startup activities towards initiation of the booster trial.
In January 2022, the company entered into a farm in agreement with a pharmaceutical company whereby the pharmaceutical company agreed to fund up to $25 million for a clinical trial for Luna and COVID-19 vaccine candidate as a booster.
Net loss for the first quarter of 2022, with approximately $51 2 million or $1 94 per share.
Andrew Sassine: Basic and Diluted Shares. We reported a net loss of approximately $56.3 million, or $2.15 per basic and diluted share, in the first quarter of 2021, and a loss of $38.7 million, or $1.47 per basic and diluted share, in the first quarter of 2021. The cash balance at the end of the first quarter was $319.7 million.
And diluted share.
We reported a net loss of approximately $56 3 million or $2.15 per basic and diluted share in the first quarter of 2021.
And a loss of $38 7 million or $1 47 per basic and diluted share in the first quarter of 2021.
Our cash balance at the end of the first quarter with $319 7 million.
Andrew Sassine: And 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.
And 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.
Joseph Payne: We're definitely pleased to have another productive quarter, advancing our pipeline of mRNA vaccines and therapeutics, in particular the recent clinical data, ARCT-154, that helps validate our self-amplifying mRNA technology platform, our lunar lipid nanoparticle delivery platform, and our proprietary trade secret manufacturing processes, including lyophilization. So I'll now turn the time over back to you, Doug, our operator, to field any questions. Thank you. If you'd like to ask a question, please signal by pressing star one on your telephone keypad. If you're using a speakerphone, please make sure your mute function is turned off to allow us to treat your equipment.
Definitely pleased to have another productive quarter advancing our pipeline of mrna vaccines and therapeutics in particular, the recent clinical data <unk> hundred 54 that helps validate our self amplifying mrna technology platform, our lunar lipid nanoparticle delivery platform and our proprietary trade secret.
<unk> processes, including life realization.
So I'll now turn the time over back to you, Doug our operator to field any questions.
Thank you if you'd like to ask a question. Please signal by pressing star one on your telephone keypad, if you're using a speaker phone. Please make sure. Your mute function is turned off to let us know treat chart equipment.
Again press Star one to ask a question.
And we'll take our first question, Dave from Yasmin Rahimi with Piper Sandler.
Operator: Again, press star one to ask a question. And we'll take our first question today from Yasmeen Rahimi with Piper Zim. Good afternoon, team, and thank you for taking my questions. A couple for you, maybe the first one is...
Good afternoon team and thank you for taking my question a.
Coupled with maybe the first.
First one is.
Yasmeen Rahimi: I know you mentioned in your prepared remarks that you're going to be analyzing the primary efficacy data to see how many of the cases were due to Omicron and Delta just to kind of give us an idea of the top line efficacy event rate, those two data points. How soon can we expect that? Like, do you think we can? How long does the process take, and in which format will you share it with us?
I know you made in your prepared remarks, you noted that you're going to be.
And the primary efficacy data the distribution how many of the cases were due to <unk> Delta.
Just to kind of give us an idea out of the top line efficacy and trade them.
The two data.
Data points.
Can we expect that like do you think.
We could how long does the process take and in what format. What you shared with US. That's one and then the second thing is have you got any communication back from Vietnam in the last two and a half.
Thanks, Gary.
I think as he shared with them and then I have a last follow up.
Sure. It makes you asked for joining the call.
Joseph Payne: That's one. And then the second thing is, have you gotten any communication back from Vietnam in the last two and a half weeks since the primary advocacy was shared with them? And then I have a last follow-up. Sure. Hey, thanks, Yas, for joining the call. With respect to the swab data and Delta and Omicron, this data will be collected over a matter of weeks. It's not several months.
With respect to the swab data in Delta and Autocar.
This data will be collected over a matter of weeks its not its not several months.
Padmanabh Chivukula: So we aim to get that part of the puzzle completed soon. But we did provide references for those that read our press release and the previous one as well, which provide some guidance as to the prevalence of Delta and Omicron during the period of the study. And we encourage people to refer to that in the meantime while we collect the hard data for that. The second question: I'll turn the time over to Pad to address that. Hi Yasmeen, I think your question was about the EUA and what its status is in Vietnam.
So we aim to get that part of the puzzle completed soon.
But we did provide references for those that read our press release and the previous one as well that provide some guidance as to the prevalence of Delta and omicron during the period of the study and we encourage people to refer to that in the mean in the meantime, while we while we collect the hard data for.
Of that.
The second question.
I'll turn the time over to pads or address that yeah.
Yes, I mean I think your question was about the U N. What what's the sort of the status and Vietnam. So I think that the review process is still ongoing but we can we can share a little bit over the last few weeks the clinical section, which is the you know one of the key parts of the <unk> process.
Padmanabh Chivukula: So I think the review process is still ongoing, but we can share that in the last few weeks, the clinical section, which is one of the key parts of the EUA process, including safety, immunogenicity, and efficacy data, was reviewed by the National Committee of Ethics, and during the meeting, it was concluded, and it was given positive feedback. So we're encouraged by that. The larger application is still under review, and we hope to hear back from them very shortly. Thank you.
Including the safety Immunogenicity and efficacy data was reviewed by the National Committee of ethics and during the during the meeting it was concluded and it was given positive feedback. So we're encouraged by that.
The larger application is still under review.
And.
Hope to hear back from them very shortly.
Joseph Payne: And then the last question is in regards to the booster study that you'll be kicking off in 2400 adults. I just want to confirm, you know, in terms of receiving approval here in the US. Is this, Ed, just the execution of the booster study in conjunction with the large study that was done in Vietnam to support full approvability here in the U.S. and in Europe? If you could just kind of reconfirm if there are any other rate limiting steps left, that could be helpful. And thank you again for taking my call.
Thank you and then my last question is in regards to the booster study that you will be kicking off in 'twenty 400 at all I just wanted to confirm.
Got.
Turning to our.
I pulled the ability here in the U S.
This add is it just the execution of the booster study in conjunction to the large study that was done in Vietnam to support full approval ability here in the U S and in Europe . So just if you could just kind of reconfirm that there any other.
Rate limiting steps lap that could be helpful and thank you again for taking my questions.
No I appreciate.
Joseph Payne: No, I appreciate the question. So we have met with the FDA and consulted with them, and received their feedback pertaining to the design elements of this pivotal booster trial with respect to the appropriate size of the trial and to what degree our data collected in Vietnam will be accepted or supportive. And after that feedback, not just from the FDA but the MHRA and the EMEA, we felt comfortable proceeding with the pivotal trial.
The question. So we have met with the FDA and and and consulted with them and received their feedback pertaining to the design elements of this pivotal booster trial.
With respect to the appropriate size of the trial and to what degree are data collected in Vietnam will be.
Accepted are supportive and after that feedback not just from the FDA, but the MH or a in EMEA that we.
Felt comfortable to proceed.
With with the pivotal trial. So that's all we've given specific guidance too but.
Joseph Payne: So that's all we've given specific guidance to, but we've previously said approximately 2,400 people will be participating in the trial, so this is not an extraordinarily large trial, and clearly that's not going to be an efficacy-based trial. Like you would see, there are no more, you know, placebo-controlled, randomized vaccine efficacy trials and stuff like that. So I can confirm that we've received feedback from major market regulatory agencies, and we're proceeding accordingly. Okay, thank you, team. Thank you, Yasmeen. Our next question will come from Seamus Fernandez with Guggenheim. Hi, this is Evan Wang.
We've previously set approximately 2400 people who will be participating in the trial. So this is not an extraordinarily large trial.
And clearly that's not going to be an efficacy based trial.
Like you would see there is no more a.
Placebo controlled randomized vaccine efficacy trials and stuff like that.
So I can confirm that we've received feedback from these major market regulatory agencies and we're proceeding accordingly.
Okay. Thank you team.
Thank you yes.
Our next question will come from Seamus Fernandez with Guggenheim.
Hi, This is Evan Wang on for shipments.
Evan Wang: I have two questions. First, on the booster, any more details you can share in terms of trial design? Unknown Speaker, Unknown Speaker, Unknown Speaker, Superiority.
First on the booster anyway.
Sure in terms of trial design.
You guys I'm sure that's 2400 patients but.
In terms of you know it was a head to head whether its non inferiority or superiority.
Any additional color would be appreciated.
Unknown Speaker: Any additional caller would be appreciated. And it's still on the timelines for the trial. Second question is, you know, I know you shared some durability data in terms of the booster. Non-Omicron Variant. Yigal Nochomovitz, We can see derivate data for the BA1 and BA2 variants.
Yeah, so on timelines for the trial.
Second question is I know you shared.
In terms of the booster.
For a non omicron variance, but any timeline in terms of when we can see durability data for it to be a wanted.
I wanted to be.
Thank you.
Sure Okay.
Joseph Payne: Sure. Okay, so additional color on the trial. Of course, we have a tight understanding of the design of our trial, but we have to remain, not just coy, but strategically silent on key elements of this. We realize that the endemic booster market is going to be a significant commercial opportunity with competitors from large pharmaceutical companies, so we want to make sure we hold those cards close to our chest. And because we're first movers in the self-amplifying mRNA space with anticipated competitors in the coming years, we want to keep those cards close to our chests as well.
So additional color on the trial.
Of course, we have a tight understanding of our or the design of our trial, but we have to remain.
Not just coy, but strategically silent on key elements of this we realized that the endemic booster market is going to be a significant commercial opportunity with competitors.
Large pharmaceutical competitors. So we wanted to make sure we hold those cards close to our chest and because we're first movers in the the self amplifying mrna space with anticipated competitors in the coming years, we want to keep those cards close to our chest as well.
Joseph Payne: What we feel is important to disclose to our investors is that this is not going to be an astronomically large trial or something that's difficult or time-consuming. With respect to time, we remind people this is a single administration-type booster vaccine trial. It's not a two-shot trial. And recruitment times are anticipated to be much faster because the population of people that have already been administered vaccines is much larger. Plentiful.
What we feel is important to disclose to our investors is that this is not going to be an astronomically large trial or something that's difficult or time consuming with respect to the time, we remind people. This is a single administration type booster vaccine trial.
It's not a two shot trial and recruitment times are anticipated to be much faster because the population of people that have already been administered vaccines is plentiful. So we're not looking for naive folks are people that have not been exposed to the virus. We're simply looking for people that have.
Joseph Payne: So we're not looking for naive folks or people that have not been exposed to the virus. We're simply looking for people that have already been vaccinated. So we believe recruitment times will be reasonable or much faster than our first experience. And with respect to the other design elements, we're just holding those cards close to our chest, if that makes sense strategically.
I already have been vaccinated. So we believe recruitment times will be reasonable or much faster than our first experience in.
And with respect to the other design elements, where we're just holding those cards close to our chest that makes sense strategically.
With respect to the VA, one NBA to durability. We've shown 29 day data. So that kind of gives you an idea that we'll be able to subsequently showed durability data for those omicron variance as well that again won't be several months, but it will.
Joseph Payne: With respect to the BA1 and BA2 durability, we've shown 29-day data. So that kind of gives you an idea that we'll be able to subsequently show durability data for those Omicron variants as well. That, again, won't be several months, but it'll...
It may be some weeks here ahead of time.
Joseph Payne: It may be some weeks ahead of time, but I refer everyone on the call to the relatively substantial amount of variant data that we've already provided against alpha, beta, delta, gamma, and other variants of interest. Very consistently, this vaccine is responsive and durable. And there's something special about self-amplifying mRNA.
But I I refer everyone on the call to the <unk>.
Relatively substantial amount of variant data that we've already provided against Alpha beta Delta Gamma and other variants of interest and very consistently. This this vaccine is responsive and durable and there's something special about self amplifying mrna, we're going to continue to learn about it we've just wanted to share.
Joseph Payne: We're going to continue to learn about it. We just wanted to share three-month data at this point. But we're cautiously optimistic that this durability will continue. And that's where we are.
Three months data at this point, but we're cautiously optimistic that this durability will continue.
And that's where we are but wait wait some additional won't be several months.
Joseph Payne: But wait for some additional time; it won't be several months. Thank you. Thank you. We're now here from Nick Abbott with Wells Fargo. Oh, good afternoon.
Thank you.
Thank you.
Well now hear from Nick Abbott with Wells Fargo.
Hello, Good afternoon, Thanks for taking my question.
Nick Abbott: Thanks for taking my question. The first one, Joe, when I look at the GMT chart here, in the press release, you know, you have one patient, roughly a twofold increase, It's the same patient, three-fold increase over BA. Two, and obviously that's over the means for Day One.
First one Joe when I look at the GMT chart here in the press release, you have one patients.
Roughly a twofold increase absolutely the same patients.
Threefold increase.
Joseph Payne: So, what was that those individuals increased over their own Day One value? If it's for the substantially less than, you know, 40 to 50 fold range, you know, have you looked at what it is about this particular individual that means they have a poor, Apparently, possibly a poor... Right now, we don't want to disclose details about specific individuals, but we do, I think this is an appropriate question to remind you that a significant, a large percentage of our participants in our trials were high-risk individuals, whether that's due to the elderly or comorbidities, you know, immune suppressed individuals, right.
Obviously that's me.
Means a day one so what was done.
So that those individuals increase over their own values and if it is.
So there is substantially less than that.
40 to 50 range.
Have you looked at what is it about this yeah individuals.
Paul.
Apparently possibly a poor response.
Right now we don't want to disclose details for specific individuals, but we do I think this is an appropriate question to remind that a significant a large percentage of our participants in our trials were high risk individuals whether that's due to elderly or Comorbidities you know.
Joseph Payne: So considering that a significant percentage was, you know, at-risk individuals, it's likely one of those that gave a lower response than others. But, but we've provided all the data; if you look at the mean, and you know, clearly, it's highly encouraging. And then you mentioned this.
<unk> suppressed individuals right, so considering that a significant percentage.
Does.
At risk individuals, it's likely one of those that gave a lower response than others, but but we've provided all the data. If you look at the mean and you know clearly it's highly encouraging.
Great.
And then you mentioned this.
Joseph Payne: Expression of Interest Letter to WHO. Could you elaborate on that, Joe? What does it mean? What is the timeline? What happens if they say yes?
Expressions of interest in that and so somebody like Jim could you elaborate on that Joe you know what what does it mean what is the timeline you know what happens if they say yes.
Oh, yeah, well the W. H O of course is a charitable organization at first so you know you know one of our intent here is to help them in any way, we can with primary vaccination I know in the U S and Europe . The majority of people have been vaccinated, but in the developing nations. This is still a core issue for the <unk>.
Joseph Payne: Oh, yeah, well, the WHO, of course, is a charitable organization at first. So, you know, one of our intents here is to help in any way we can with primary vaccination. I know, in the US and Europe, the majority of people have been vaccinated, but in developing nations, this is still a core issue for the WHO. And we want to see if there's, I think it's also helpful to remind people that I don't know the exact number of countries involved in the W.H.O.
<unk> show and we want to see if there's a potential here for us to help there.
I think it's also helpful to remind people that Ah I don't know the exact number of countries involved in the W. H O emergency use listing but it is a large number of developing nations. So if if we do successfully capture.
Joseph Payne: Emergency Use Listing, but it is a large number of developing nations. So if we do successfully capture a spot on this EUL, or this emergency use list, then it'll open up opportunities for not just helping in the near term but commercial opportunities down the road. So that's about it, you know, with respect to the timeline of going through the process, we just wanted to make people aware that, you know, Vietnam, the Ministry of Health, and the WHO have been engaged and have... You know, sent a letter and have been supportive of this process, and we'll give updates as they arise. But we haven't given any tight guidance on a timeline, an official spot on the emergency. All right. Thanks, Seth.
Spot on this well.
Or this emergency useless, then it'll open up opportunities for them not just helping in the near term, but commercial opportunities down the road.
So that's about it Oh, you know with respect to the timeline of going through the process. We just wanted to make people aware that Vietnam Ministry of health in the WH show have been engaged and have been.
You know sent a letter and have been supportive of this process and we'll give updates as they as they arise, but we haven't given any tight guidance on a timeline to it.
Officials spot on the emergency use list.
Thanks, Joe.
You go not some of it's with Citigroup has our next question.
Yigal Nochomovitz: Yigal Nochomovitz with Citigroup has our next question. All right, great. Thanks, Jeremy, for taking the questions. I'm just curious about what regulatory work you've done with regulatory consultants or other outside experts. Clarify whether FDA would be amenable to approving a fourth vaccine only as a booster given that obviously that's not the paradigm that we've seen for Moderna, Pfizer, and J&J. Right, yeah, the only thing that we've learned is with respect to another vaccine approved under emergency use. I think we'd have to see another wave, like some biostatisticians are predicting a wave of COVID coming If that indeed happens, then the likelihood of garnering emergency use authorization would be higher.
Right.
To your question.
What regulatory work, you've done with our regulatory consultants or other outside expert.
Clarify, whether FDA would be amenable to approving a fourth vaccine only as a booster.
The paradigm that we've seen or the donor Pfizer and J&J. Thank you.
Yeah. So the only thing that we've learned is like with respect to.
Another approved under emergency use I I.
Yeah, we'd have to see another wave like some some bio statisticians are predicting a wave of COVID-19 coming this fall if that indeed happens then then that likelihood of garnering emergency use authorization would be higher but I think the expectation is to go through a traditional BLA process.
Joseph Payne: But I think the expectation is to go through a traditional BLA process in the US to capture a traditional booster, and the reason, the justification for that, is simply that this is a lower dose, longer-lasting option. And that's what we're trying to do. So this is a different mechanism of action of expressing the antigen, and it could be beneficial to subpopulations here in the U.S.
In the U S to capture a traditional booster and and the reason the justification for that is simply this is a lower dose longer lasting potentially option. So this is a different mechanism of action of <unk> expressing the antigen and it could be beneficial to subpopulations here in the U S. So.
Joseph Payne: So we believe that regulatory agencies will be respectful of that, of these key differences, of allowing their citizens to have access to a lower dose next generation mRNA vaccine. Have you had discussions with the FDA regarding the acceptability of the Vietnam 20,000 patient trial for your application? What is the status on that?
So we believe that regulatory agencies will be respectful of that of those these key differences of allowing their citizens to have access to a lower dose next generation mrna vaccine.
Got it.
Have you had discussions.
Regarding the acceptability of the Vietnam, 20000 patient trial or their application.
Where does it stand with that.
Well, we haven't give a tight detailed response to that question. It's a good question, but we are we have.
Joseph Payne: Well, we haven't given a tight, detailed response to that question. It's a good question, but we have... shared this question with these regulatory agencies, and they have provided their feedback and have provided a path forward for the Vietnamese data to be supportive. The details as to what's specifically required for this data to be supportive have not been shared. However, we felt comfortable enough to proceed with this global registrational study based upon the feedback we've received. And for the Global Registration Study for the booster, you vetted that design with FDA, correct? In detail, that's correct.
Sure you asked this question to these regulatory agencies and they have provided their feedback and have provided a path forward.
For the Vietnamese data to be supportive the details as to which.
What specifically required for for this data to be supportive has not been shared however, we felt comfortable enough for us to proceed with this global Registrational study based upon the feedback we've.
Perceived.
For the global Registrational study for the Lucerne.
You bet is that design.
Yeah correct.
Correct in detail that's correct.
Okay. Thank you very much.
Next to <unk> Kumar Rajat.
Joseph Payne: Okay, thank you very much. Next, we'll hear from Kumar Rajat with Brookline Capital Markets. Thanks for taking my questions.
Next we'll hear from Kumar <unk> with Brookline capital markets.
Kumar Raja: One more on the booster trial. So some patients, as of now, in the US, some have received two, some have received three doses, and some have received four doses. You have data from patients who have completed two doses. So how are you thinking about including patients who have received three or four doses in the trial? And also with regard to the data, what are you thinking in terms of a timeline to follow that? Sure, so I can refer those on the call to a white paper with some guidance provided by regulatory agencies with respect to booster trials.
Thanks for taking my questions one more on the booster try it.
So some patients.
I'll stop now and you are somehow received to somehow received three doses.
How would you do for both of Us.
You have data from patients who have completed two doses.
So how are you thinking about including patients who have received three or port both within the trial.
And also with regard to the data what are you thinking in terms of timeline to follow them.
Sure So yeah I can refer.
You know those on the call to you know there was a white paper or some guidance provided by regulatory agencies with respect to booster trials can they include people that have received two shots three shots or and the answer is generally yes, I do but I cannot speak on behalf of the FDA or other regulatory agencies.
Kumar Raja: Can they include people that have received two shots, three shots, or, and the answer is generally yes, I do, but I cannot speak on behalf of the FDA or other regulatory agencies specifically to ARCT 154, but there has been general guidance provided that booster trials can recruit not just people that have received two shots but people that have received three shots. So that allows us flexibility. Benefits from Recruitment. Perspective.
Specifically the <unk> hundred 54, but there has been general guidance provided that booster trials can recruit not just people that have received two shots, but people that are pushing three shots. So that allows us flexibility in and benefits from a recruitment perspective definitely.
Okay on inbound timeline.
Joseph Payne: Yes. Okay, and in terms of the timeline, how long do you think you will need to track these patients? And also, your thoughts on including older, high-risk patients in the trial? Correct, correct. We received guidance on all of these details from the regulatory agencies, and they were fairly consistent.
How long do you think you would need to track these patients.
And also your thoughts on including all that high risk patients and their clients.
Correct correct, we received guidance on all of these details from the regulatory agencies. They were fairly consistent I can speak to that with.
Joseph Payne: I can speak to that with respect to the percentage of the elderly and how long we need to track these folks for. And so our, our, those design elements are implemented into our. Booster Trial Design. It has definitely taken into consideration the feedback from the regulatory agencies that we ask that specific question. Okay, I just needed clarification on something Andy mentioned. So you have received 25 million from a pharmaceutical company for the booster trial. Did I hear that right? Yeah, we haven't gotten the $25 million yet.
With respect to the percentage of the elderly and how long we need to track. These folks for and so are those design elements are implemented into our booster trial design.
And you know it has it.
It has definitely taken into consideration the feedback from the regulatory agencies that we asked that specific question too.
Okay, I just needed a clarification on something I'm dimension. So you have received $25 million from a pharmaceutical company for the booster time did I hear that right.
Yeah, we haven't gotten the $25 million, we've gotten an agreement to reimburse us up to $25 million of our you know phase III Booth two trials, so hopefully that clarifies it for you.
Joseph Payne: We've gotten an agreement to reimburse us up to $25 million for our, you know, phase three booster trial. So hopefully that clarifies it for you. Okay, and what do you guys need to do in return for that? We need to obviously spend money on the trial and bill our pharmaceutical company partner, and hopefully, get reimbursed in a reasonable timeframe. Okay, thanks so much. Our next question will come from Stephen Seedhouse with Wayman James.
Okay, and what do you guys need to do in return for that.
Oh, we need to obviously you know spend money on the trial and then bill our pharmaceutical company partner in and hopefully get reimbursed in a reasonable timeframe.
Okay. Thanks, so much.
Our next question will come from Stephen seat House with Raymond James.
Great. Thanks for taking the question I wanted to ask you about a strategic 10.
Joseph Payne: Great, thanks for taking the question. I wanted to ask about ARCT 810, the Phase 2 study that's getting underway. What, in your view, would constitute proof of concept emerging from the data in that study? And then, on the 154 Booster Study, I wanted to ask whether you settle on a dose for that study, if it's the same dose you've been contemplating, or if you think about increasing the dose. Squeeze out more efficacy, potentially.
And the phase two study.
Underway what are in your view.
You would constitute.
Emerging from the data in that study.
And.
And then on the 154 booster study wanted to ask.
You settle on a dose for that study would be the same dose you've been contemplating or do you think about increasing the dose.
Just squeeze out more efficacy potentially.
Yeah.
Joseph Payne: Yeah, at this point, 5 micrograms is our dose. We'll continue to monitor that dose level in the booster environment. But right now, we believe that we'll be keeping the dose at 5 micrograms. With respect to biological proof of concept for the A10 program... There are multiple biomarkers associated with ornithine transcarbamylase deficiency, as you're well aware, including ammonia in the blood or the plasma, and erotic acid in the urine, and the urea cycle itself, and ureagenesis, and also levels of OTC itself.
Yeah.
Yeah at this 0.5 micrograms is our dose will.
We will continue to monitor that dose level in the in the booster environment, but right now we believe that we'll be keeping the dose at five micrograms with respect to biological proof of concept for the for the a 10 program.
Joseph Payne: So, there are a lot of biomarkers associated with this disease, so we're defining proof of concept as biological proof of concept. So, if any, or all, of these biomarkers are impacted by ARCT 810 treatment, then we would determine that as biological proof of concept. Thank you. Thanks, Steve. Our final question will come from Ed Arce with 8C. Hello everyone, this is Thomas Yip asking a couple questions for Ed. Hi Joe, this is Thomas.
Multiple biomarkers associated with with only theme transcribe families deficiency as youre well aware.
Putting ammonia in the blood or the plasma and erotic acid in the urine and the urea cycle itself is in urea Genesis.
But and also levels of OTC itself. So a lot of biomarkers associated with this disease. So we're defining proof of concept as biological proof of concept. So if any or all of these biomarkers are impacted by it.
<unk>.
<unk> treatment, then we would determine that is biological proof of concept.
Thank you.
Thanks, Steve.
Our final question will come from Ed Arce with H C Wainwright.
Hello, everybody. This is Thomas Yip, asking a couple of questions for Ed.
Hi, Joe This is probably a good problem.
Thomas Yip: Hi, you mentioned earlier that the Vietnam Ministry of Health is reviewing the data that's been submitted, which includes phase one, two. Can you remind us what the review process regarding a time frame, do they adhere to a specific guideline that they hope to render a response to regarding the EU rate? No specific timeline was given, but, you know, all those data that we've referred to in this review today have been provided, and they're working hard and diligently to review it.
Oh hi.
Earlier that the Ministry of health is a revealing that.
Data that doesn't matter, which include Bruce went through.
Right.
Can you remind us what is there a review process regarding timeframe.
Here to present, the guidelines that they.
To wrap their response.
Good luck with your rig.
No specific timeline was was given but you know all of those data that we've referred to in this review today.
Been provided.
And they're working hard and diligently reviewing it.
I forget the exact number of papers, but this was an extraordinary amount of data usually no regulatory agencies review subsections of data over a decade of time and this is 38 weeks of data involving 19000 participants. So we do recognize the amount of.
Thomas Yip: I forget the exact number of papers, but this was an extraordinary amount of data. Usually, you know, regulatory agencies review subsections of data over, you know, a decade of time, and this is 38 weeks of data involving 19,000 participants. So we do recognize the amount of data that needs to be reviewed in this process, but they're working as fast as they can. You know, we've guided the decision as soon as possible, but any, Pat, anything to add? No, and I think, you know, we've been clear about the timing for that. We'll have some clear guidance on that by the end of the course. I got it.
Data that needs to be reviewed in this process, but they are working as fast as they can.
We've guided.
You know what that.
That decision is soon but any pad anything to add.
And I think you know we've been clear about the timing for that we will have will have some clear guidance on the bags and end of the quarter.
Got it.
Joseph Payne: And then regarding data publication that is to be in the future, from how we understand it, it is driven by VinBioCare. Can you talk about when we should expect the data package to be published? And what about phase 3B data? When can we expect that?
And then regarding the data publication.
That is oh sure from a how we understand that it's not driven by the viral care.
Can you talk about when should we expect that.
That data package to be published and what about <unk>.
We'd be a data or what.
Right.
Oh, the publication of the data well the data is it it is intimately and associated within bio care, our partner as well, but yes. They understand we understand the expectation is that you know at some point here, we're going to be publishing this data for sure. So it.
Joseph Payne: Oh, the publication of the data? Well, the data is intimately associated with VinBioCare, our partner as well. But yes, they understand, we understand. And the expectation is that, you know, at some point here, we're going to be publishing this data for sure. So at least that's our expectation. But we haven't given any guidance on the timing of that data. But, Pad, anything else to add?
That's our expectation.
But we haven't given any guidance on timing of that data.
But.
But had anything else that again, then we and then win win by it because I understand the importance of sharing the data that we've generated and the positive nature of the data. So I think we will provide more details around some.
Padmanabh Chivukula: No, again, we and WinBio can understand the importance of sharing the data that you've generated and the positive nature of the data. So I think we will provide more details around some of the efficacy analysis, as well as, you know, further analysis of some of the subgroups as well. Yeah, and I know our teams are actively drafting these publications right now, but you're asking the specific timing of the publication of these, and we just haven't provided that specific guidance.
Some of the efficacy analysis as well as Oh, you know further analysis of all the US you know some of the subgroups as well.
All of that in there and I know our teams are actively drafting these publications right now, but you're asking the specific timing of the publication of those and we just haven't provided that specific guidance yet.
Joseph Payne: Thank you so much for the kind questions, and we're looking forward to the publication. Yeah, thank you. I will conclude today's question and answer session, and I'll turn the conference over to Mr. Payne for any additional closing remarks. Yeah, thanks to everyone that joined the call. If we didn't get a chance to address your question, feel free to reach out to us directly. We'll be in touch, guys. Thanks, everyone. Bye bye.
And that's been thank you so much worth kind of questions and we're looking forward to the publication.
Yeah. Thank you.
Okay.
That will conclude today's question and answer session I will now turn the conference over to Mr. Payne for any additional or closing remarks.
Okay. Thanks to everyone that joined the call. If we didn't get a chance to address your question feel free to reach out to us directly and we'll be in touch guys. Thanks, everyone Bye bye.
That will conclude today's conference call. Thank you for your participation you may now disconnect.
Joseph Payne: That will conclude today's conference call. Thank you for your participation. You may now disconnect, and Yusuf Kandahar. This is a production of the U.S. Department of State. This is a production of the U.S. Department of State, www.facebook.com. Copyright 2020 Mooji Media Ltd. All Rights Reserved. No part of this recording may be reproduced without Mooji Media Ltd.'s express consent.
Yeah.
Yeah.
Yeah.
Yeah.
Okay.
Okay.