Q4 2022 Arcturus Therapeutics Holdings Inc Earnings Call
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Speaker 2: A question and answer session will follow the formal presentation.
Speaker 2: If anyone should require operator assistance during a conference, please press star zero on your telephone keypad. As a reminder, this conference is being recorded.
Speaker 2: It is now my pleasure to introduce your host, Neda Safarzadeh, Vice President, Head of Investor Relations, Public Relations, and Marketing. Thank you. You may begin.
Speaker 3: Thank you operator. Good afternoon and welcome to our third third PODEX fourth quarter 2022 financial update and pipeline progress call.
Speaker 3: Today's call will be led by Joseph Payne, our President and CEO , and Andy Sessin, our CFO . Dr. Pat Cibucula, our CSO and COO, will join in for the Q's and annotation as well.
Speaker 3: Before we begin, I would like to remind everyone that the statements made during this call regarding matters that are not historical facts are forward-looking statements within the Safe Harbor provisions of the Private Securities Litigation Reform Act of 1995.
Speaker 3: Forward-looking statements are not guaranteed 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.
Speaker 3: 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 Form 10-K file with the SEC.
Speaker 3: In addition, any forward-looking statements represent our views only as of the date such as statements are made.
Speaker 3: Our service specifically disclaims any obligation to update such a statement to reflect future information, events, or circumstances.
Speaker 3: And with that, I will now turn the call over to Jo.
Speaker 2: Thank you Netta and good afternoon to all. Thank you for joining us for today's call.
Speaker 4: The recent period has been characterized by substantial operational and pipeline progress here at Arcturus.
Speaker 4: We will be highlighting four areas on today's update call.
Speaker 4: First, we closed our strategic vaccine collaboration agreement with CSL-Securus at the end of last year. We have received the $200 million upfront payment and invoiced $90 million in additional milestones.
Speaker 4: demonstrating the positive progress that the companies have made on our partnered COVID and seasonal flu vaccine programs.
Speaker 4: Second, we've entered into an agreement with Meiji Pharma to evaluate ARCT154 as a booster vaccine for SARS-CoV-2, also known as COVID-19.
Speaker 4: And we're very happy to report significant operational progress in the ongoing Phase III study in Japan and we'll be providing an update there.
Speaker 4: Third, we have continued to advance our RNA platform technology and our earlier stage clinical programs and we will provide an update on recent progress there.
Speaker 4: Fourth, we have strengthened our management team by the addition of Dr. Jurgen Frohlich as our Chief Medical Officer overseeing mRNA Therapeutics and Dr. Igor Smolanov as our Chief Development Officer overseeing clinical development of our vaccine franchise.
Speaker 4: I'll begin with our recently announced strategic collaboration with CSL.
Speaker 4: We are in the initial phase of our now four-month-old collaboration and very pleased with how the teams are working together. Arcturus has achieved substantial milestones this month associated with nominating next-generation candidates for COVID and seasonal flu programs.
Speaker 4: Mutual respect is evident between the working groups of both companies. There is a clear passion and solid work ethic behind the competent execution that has led to these important and early milestones being achieved.
Speaker 4: We remain eligible for additional development and commercial milestones as Covered Pipeline programs advance.
Speaker 4: Our collaboration with CSL is focused on the development and commercialization of next generation mRNA vaccines, targeting COVID influenza, three additional pathogens, as well as pandemic preparedness.
Speaker 4: Our collaboration combines CSL's well-established global vaccine, commercial, and manufacturing infrastructure with Arcturus's MRNA manufacturing expertise.
Speaker 4: and innovative STAR mRNA vaccine and lunar delivery platform technologies.
Speaker 4: We expect this collaboration to drive the development, manufacture and global commercialization of next generation self amplifying mRNA vaccines over the coming years.
Speaker 4: The impact of this collaboration to our balance sheet and runway continues to be meaningful and Andy will be speaking to that shortly.
Speaker 4: Now onto our agreement with Meiji Pharma to advance ARCT154 development in Japan.
Speaker 4: Our contracted relationship with Maggie is focused on developing a RCT 1 5 4. This is our next generation self amplifying MRNA booster vaccine.
Speaker 4: for COVID-19.
Speaker 4: You may already appreciate that Japan has one of the world's highest rates of COVID booster vaccination.
Speaker 4: Given the Japanese government's focus on public health and infectious disease prevention, we fully expect high levels of COVID booster utilization for many years to come.
Speaker 4: In addition, the Japanese government has been clear about their interest in establishing independence in mRNA vaccines.
Speaker 4: we're very pleased to be part of this effort with Meeji.
Speaker 4: Meiji Group received a significant subsidy from the Japanese government in the fourth quarter of 2022 to support this effort. Meiji is responsible for all development costs related to ARCT154 in Japan.
Speaker 4: In December 2022, we announced that Meiji had initiated a Phase III booster study in Japan designed to compare ARCT154 to Comirnatty.
Speaker 4: in targeting 780 adult participants based on non-inferiority immunogenicity.
Speaker 4: Meiji moved quickly with the enrollment process.
Speaker 4: very impressed and appreciative with the productivity and progress we've seen there.
Speaker 4: I'm very pleased to report today that the study is now fully enrolled, with over 800 participants, exceeding our target enrollment and ahead of schedule.
Speaker 4: In addition, the one month follow-up visits and the one month blood draws have also been completed.
Speaker 4: This timely execution allows us to immediately collect the prerequisite immunogenicity data and be in a position to potentially file our first NDA in Japan.
Speaker 4: Now moving to our earlier stage programs.
Speaker 4: I'll begin with ARCT 810. This is our therapeutic candidate for ornithine transcarbamylase deficiency or OTC deficiency.
Speaker 4: This investigational therapeutic aims to address the deficient OTC enzyme in the livers of individuals with this disease. ARCT 810 has the potential to restore urea cycle activity and prevent metabolic crises that cause neurological damage.
Speaker 4: potentially liberalize strict dietary protein restrictions and improve the quality of life for people living with this condition.
Speaker 4: The program utilizes our proprietary lunar delivery technology and one important attribute of this technology is that the lipids administered are rapidly degraded, which we expect will contribute to a favorable safety profile.
Speaker 4: We are evaluating ARCT 810 and two ongoing clinical studies, a Phase 1B study in adults and a multi-dose Phase 2 study in adolescents and adults with OTC deficiency.
Speaker 4: These studies build upon a completed phase one study that demonstrated a favorable safety profile when dosed up to 0.4 mg per kg, the highest dose evaluated in that study.
Speaker 4: We continue to advance ARCT 810 in the phase 2 study.
Speaker 4: The study will enroll up to 24 adolescents and adults living with OTC deficiency distributed across two dose cohorts.
Speaker 4: The study has enrolled multiple subjects. We remain on track to report ARCT 810 phase 2 interim clinical data later this year. Now moving on to ARCT 032. This is our inhaled messenger RNA therapeutic candidate for cystic fibrosis.
Speaker 4: With this drug we are aiming to express fully functional CFTR protein in the lungs of individuals with cystic fibrosis.
Speaker 4: Our approach is agnostic to the underlying mutations associated with the disease.
Speaker 4: And we believe that ARCT-032 could provide benefit across a very wide range of those living with CF, especially Type 1 CF and for individuals who are not well served by CFTR modulator therapies.
Speaker 4: We're grateful to have obtained support from the CF Foundation for the advancement of this promising investigational medicine. We also benefit from invaluable scientific collaboration with the experts at the CF Foundation.
Speaker 4: Previously, we reported encouraging preclinical data demonstrating successful delivery to the lung in four different animal species, mice, rats, ferrets, and primates.
Speaker 4: Notably, our data have shown the ability to deliver mRNA to airway epithelium in the CF ferret. This is a disease model that produces significant mucus in the airways, similar to patients with CF.
Speaker 4: Finally, in vitro treatment of bronchial epithelial cells from CF donors with ARCTO32 has demonstrated robust expression of CFTR protein and restoration of chloride current.
Speaker 4: Supported by these encouraging data, we are now evaluating ARCTO32 at four different dose levels in a phase one study being conducted in New Zealand.
Speaker 4: We have successfully completed the enrollment of the first two cohorts with expectation to complete enrollment of the entire 32 subject study in the second quarter of this year and plan to report study results later this year. Arcturus is excited to share our lunar HBV data...
Speaker 4: hepatitis summit conference in Paris, France.
Speaker 4: This will be the first opportunity for the scientific community to evaluate the benefits of Arcturus' lunar delivery of systemically administered mRNA.
Speaker 4: for gene editing applications.
Speaker 4: In this past quarter, we have strengthened our management team.
Speaker 4: We have brought on Dr. Jurgen Frohlich to be our CMO, our Chief Medical Officer, to provide seasoned leadership over our mRNA Therapeutics Pipeline, and also have brought on board Dr. Igor Smolinov.
Speaker 4: to be our Chief Development Officer, who will lead Arcturus' clinical development efforts for our promising COVID and seasonal flu self-amplifying mRNA vaccine.
Speaker 4: Now Dr. Froelich has broad and successful experience in the field of rare diseases including OTC deficiency and cystic fibrosis. He will assuredly increase our likelihood of success as Arcturus initiates and navigates through late stage clinical trials.
Speaker 4: for our rare disease therapeutic programs. He has three decades of broad and late stage therapeutic clinical development experience at Genentech, Quintiles, BMS, Ipson, Vertex and Genivant. Jurgen completed medical school at the University of Wurzburg in Germany. He is a diplomat at the American Board of Clinical Pharmacology.
Speaker 4: and holds a dual executive MBA from Zurich, Switzerland, and the State University of New York at Albany. Jurgen has been directly involved in successful global marketing authorizations of drugs in the US, Canada, the European Union, Switzerland, and Australia.
Speaker 4: Since 2011, he's been involved in early and late stage development of CF therapeutics, including the approval of Kaleidoc.
Speaker 4: and clinical development planning for other CFTR modulators.
Speaker 4: He has seasoned experience in phase 1, 2 and 3 trials with inhaled therapeutics in patients with CF to treat chronic lung infection.
Speaker 4: We are fortunate to have Dr. Jurgen Frohlich join our management team as our Chief Medical Officer.
Speaker 4: Now moving on to introduce our Chief Development Officer of Vaccines.
Speaker 4: Dr. Igor Smolanov has a strong record of successfully developing vaccines all the way through approval.
Speaker 4: He will help our vaccine team and our partnership with CSL get our COVID and flu programs to this next level. And we're excited about that. Thanks so much for joining us this past year as a new
Speaker 4: Dr. Smolanoff is a recognized leader in clinical development with a proven record of accomplishment in biotech and large pharmaceutical companies. He contributed to the successful development and licensure of several innovative vaccines.
Speaker 4: Before joining Arcturus, Dr. Smolinov was the Executive Vice President at Clover Pharmaceuticals.
Speaker 4: That's where he built a strong team that was able to rapidly generate pivotal clinical data, leading to a COVID-19 vaccine authorization and product launch there.
Speaker 4: Before that, Dr. Smolanov served as a therapeutic area head, leading the development of several seasonal flu vaccines.
Speaker 4: at CSL-Sicuris. Igor was the head of clinical development at Moderna, managing the initiation of the first clinical trials of messenger RNA vaccines in humans.
Speaker 4: At Novartis vaccines, Dr. Smolinov contributed to the development and global licensure of multiple vaccines there as well.
Speaker 4: Igor graduated from Volgograd State Medical University in Russia where he holds an MD, a PhD and a Doctor of Science degrees from this university. He is the author of more than 50 publications and peer-reviewed journals in clinical pharmacology, infectious disease and vaccine development. We are indeed fortunate to have Dr. Igor Smolinov.
Speaker 4: join our team here at Arcturus as our CDO overseeing our vaccine franchise. I will now pass the call on to Andy Saseen, our CFO , to provide financial updates.
Speaker 5: Thank you, Joe, and good afternoon, everyone.
Speaker 5: The press release issued earlier today includes financial statements for the fourth quarter and fiscal year 2022.
Speaker 5: and provides a summary and analysis of year-over-year and sequential financial performance.
Speaker 5: Please also reference our Form 10-K for more details on the financial performance.
Speaker 5: I'll begin with the CSL agreement.
Speaker 5: Arcturus received a $200 million upfront payment that was received in the fourth quarter of 2022.
Speaker 5: Additionally, in March 2023, Arcturus achieved development milestones primarily associated with nominating next-generation candidates for COVID-19 and seasonal flu programs.
Speaker 5: resulting in $90 million dollars invoiced.
Speaker 5: in 90 million dollars invoiced to CSL.
Speaker 5: We are excited to continue working on these programs under the guidance and leadership of our partner, CSL.
Speaker 5: Our CSL collaboration is a 40-60 profit sharing agreement related to COVID-19 vaccine products.
Speaker 5: With respect to program costs related to the bivalent COVID-19 vaccine, we expect that future anticipated milestones will cover all related expenses going forward.
Speaker 5: Additionally, the program cost for the seasonal flu candidate will be reimbursed in full on an ongoing basis.
Speaker 5: DSL can apply a 37.5 million R&D credit to be used within the next five years against cost incurred on the flu and three other respiratory disease vaccines. As you heard earlier, we are excited that Meiji completed enrollment.
Speaker 5: during the first quarter of 2023 for the Phase III COVID-19 booster trial of ARCT-154 in Japan.
Speaker 5: MAGE is responsible for all related clinical, regulatory, development and manufacturing expenses for the ARCT154 booster vaccine. Our manufacturing loan with the Singapore government, which had a principal and interest balance of $50 million in 2018 and later Gr, Bob in 2017 with the Long-Term
Speaker 6: given.
Speaker 5: As a result, Arcturus has no further loan obligations payable to Singapore.
Speaker 5: On the Treasury side, in March 2023, we paid off the remaining loan with Western Alliance Bank, which had a balance of $10 million as of December 31, 2022. And we entered into a new banking relationship with Weld Fargo.
Speaker 5: Based on the substantial funding provided by the CSL collaboration, we expect our TURF to be in a very strong financial position in the next few years.
Speaker 5: Our cash runway now extends to the beginning of 2026 based on our current pipeline and assuming no milestones or revenues from any commercial product sale.
Speaker 5: I will now provide a quick summary of our financial results for the fourth quarter of 2022.
Speaker 5: We reported revenues of $160.3 million for the fourth quarter compared to revenues of $5.8 million in the fourth quarter of 2021. The increase in revenue is predominantly driven by the licensed portion of the upfront payment from the CSL transaction.
Speaker 5: We reported total operating expenses of $38.8 million during the fourth quarter of 2022 compared to operating expenses of $43.4 million in the fourth quarter of 2021.
Speaker 5: The decline in operating expenses was primarily due to lower COVID-19 related manufacturing and clinical related expenses.
Speaker 5: Finally, we reported a net income of approximately $117.3 million.
Speaker 5: or $4.43 per diluted share during the fourth quarter of 2022, compared to a net loss of $38.7 million, or $1.47 per diluted share during the fourth quarter of 2021.
Speaker 5: I am happy to report for the first time in the history of the company, we reported net income of $9.3 million for the fiscal year ended 2022.
Speaker 5: In summary, we believe that the company is in a strong financial position.
Speaker 5: and has the resources needed to achieve multiple near-term value-creating milestones.
Speaker 5: for the vaccine and therapeutic programs over the next 12 months. I will now pass the call back to Joe.
Speaker 4: Thanks Andy. It's been a productive quarter. We hit the ground running with CSL as indicated by meaningful early milestones being achieved in the partnership. We made measurable progress in each of our clinical programs which has put us in a position to potentially file our first NDA in Japan and collect meaningful clinical data in 2023 for each one of our pipeline programs.
Speaker 4: This will showcase the intramuscular, intravenous and inhaled applications of our proprietary mRNA and delivery technologies.
Speaker 4: And we've also strengthened our management team and look forward to many of you meeting them.
Speaker 4: over the next, the remainder of the year. So with that, we would like to turn the time over to the operator for questions.
Speaker 2: Thank you. Ladies and gentlemen, at this time, we'll be conducting a question-and-answer session.
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Speaker 2: The first question comes from the line of famous Fernandez with Guggenheim. Please proceed with your question. Thanks for the question. My first question is actually on the MAGE trial. It sounds like the team there has made a lot of progress.
Speaker 2: enrolling patients and we're almost on the cusp of actually getting the clinical data. I was just hoping that the team could comment on what your hope for expectations are for those data relative to Comirnaty. And if there will be follow-up data, because I believe there has been in the past some suggestion.
Speaker 2: of greater durability with the self-amplifying RNA as a potential advantage over just the standard modified mRNA. And then the second question, just really wanted to get a better sense if you are willing to share the dosing regimen in a little bit more detail.
Speaker 2: and the number of patients that have been dosed so far in the OTC study. Thanks so much.
Speaker 2: that have been dosed so far in the OTC study. Thanks so much. Okay thanks Seamus.
Speaker 4: Well, with respect to your first question, the primary objective of that trial is to establish non-inferiority with respect to the immunogenicity data. One of the key sets of immunogenicity data is that one month blood data that's been drawn already.
Speaker 4: So you're correct in your assessment that we're collecting data as we speak that's very relevant to the upcoming potential new drug application in Japan.
Speaker 4: So we want to make sure that that immunogenicity data is included in that application. With respect to your durability inference, yes, no doubt we will be tracking that in parallel, but that durability data is not a prerequisite to establishing approval with the PMDA approval in Japan. It'll be in half.
Speaker 4: we've informed, we've communicated externally that we have now enrolled multiple patients. We cannot give any more specifics than that and then with respect to guidance, we want to be clear that we're still on track for sharing Phase 2 data later this year.
Speaker 2: Joe, if you don't mind, can I just clarify enrolled patients or have you started dosing patients officially? Okay, okay, okay, great.
Speaker 4: Yes, ruled means I define as dosing, correct. So whenever I mention the word at home in this context, it's past screening they have been dosed, correct.
Speaker 4: Dose. Dose means I define as dosing, correct. So whenever I mention the word at home in this context, it's past screening, they have been dosed, correct. Okay, great. Thank you. Thank you.
Speaker 7: So, one of the points is that even in the early, the first dosing, we are expecting some pharmacological activity because it is a dose level where we've seen pharmacological effect in preclinical models.
Speaker 8: Our next question comes from the line of Yanan Zhu with Wells Fargo. Please proceed with your question.
Speaker 7: Great. Thanks for taking the questions and congrats on the progress. A few questions. On the Japan COVID study, could you talk about what might be the non-inferiority margin?
Speaker 7: for the primary analysis and have you talked about the potential economics from a Japanese regulatory approval? Yes, my questions are questions of the general public.
Speaker 9: analysis and have you talked about the potential economics from a Japanese regulatory approval? Thanks.
Speaker 4: Well, I want to make sure I understand your question. With respect to the phase 3 trial in Japan, I know that it's been properly powered.
Speaker 4: to achieve statistical relevance with respect to a non-inferior endpoint.
Speaker 4: So there's no concerns there with respect to the margins required. Numerical superiority will obviously be observed, but statistical superiority will have to collect the data to understand that.
Speaker 5: with respect to your second question which was on, I can pass it along to Andy. Yeah, no, thank you for the question. Unfortunately we don't, you know, provide guidance with respect to economics and when we do have that, you know, available we will share with the market.
Speaker 5: what the economics are for CSL, MEGI, and ourselves going forward. So thanks for the question.
Speaker 9: Got it. And then a couple of questions on OTC deficiency. In terms of data later this year, you mentioned a subset of patients. Could we see one or both cohorts of data?
Speaker 4: and also what would define success for that readout? Sure, so the success is biological proof of concept and that's being defined by biomarker changes being observed in this patient population.
Speaker 4: So the biomarkers include ammonia and erotic acid and urea. Ammonia in the blood, erotic acid in the urine. Urea genesis will be measured. Other amino acids will be also measured for and the OTC enzyme itself will be measured.
Speaker 4: measured in the blood through an unvalidated assay. So several biomarkers will be measured and so when we indicate biological proof of concept we mean being able to measure or determine changes in those biomarkers because of the...
Speaker 4: in the blood through an unvalidated assay. So several biomarkers will be measured. And so when we indicate biological proof of concept, we mean being able to measure or determine changes in those biomarkers because of the therapeutic.
Speaker 4: With respect, what was the other question? Would we see one or both cohorts of patients? That depends on the rate of enrollment. We're recruiting up to 24 subjects in this trial and 12 of them are at one particular dose and 12 are at another dose.
Speaker 9: the cystic fibrosis program, which data later this year. Just wondering, for this healthy volunteer study, what would be the most meaningful readout that we should watch out for, and how do you determine the dose to be used in cystic fibrosis patients afterwards? Thank you. Right, so...
Speaker 4: This is the most meaningful exercise here with these four doses being evaluated in these early subjects in phase one It's just ascertaining safety and tolerability of the dosing regimen itself. This is an inhaled therapeutic So we're going to be able to quickly evaluate the maximum tolerated dosing for example. How long can a person inhale this therapeutic for?
Speaker 7: And so that will be the most interesting data that could come out of this. Yeah, this is Pat. And you know, based on our phase one data, obviously we'll be looking at the lowest dose and the top dose. And when we decide to, when we go into phase two, we can probably eliminate some of the lower doses and we'll pick a dose where we feel comfortable with that has a good safety margin to start with.
Speaker 7: And so that will be the most interesting data that could come out of this. Yeah, this is Pat. And you know, based on our phase one data, obviously we'll be looking at the lowest dose and the top dose. And when we decide to, when we go into phase two, we can probably eliminate some of the lower doses and we'll pick a dose where we feel comfortable with that has a good safety margin to start with. Great. Very helpful. Thank you. Thank you.
Speaker 8: Thanks, you know. Our next question comes from the line of Ival Nakomovitz with Citi. Please proceed with your question. Hi, Gene. This is Ashim Mubarak on for you all. Thanks for taking my questions. The OTC program, you had some earlier comments on enrollment and how that
Speaker 8: of response meaning as in how long do you think you need to dose and follow these patients before accumulating enough response data to be meaningful enough to share? Thanks.
Speaker 4: Sure, so I can update the market that we have now onboarded nine active sites for OTC deficiency. The bulk of that effort was last year. This is the year where we've initiated enrollment officially.
Speaker 4: So with respect to the pace of enrollment, it's nothing that is out of the ordinary for a rare liver disease in Europe . So that's the only comment I can provide there. All we've disclosed is multiple patients being enrolled so far. With respect to the kinetics, it's helpful to understand that this is a six administration trial.
Speaker 4: So these doses are separated by two weeks. So there's six administrations. And what we've seen pre-clinically is that OTC is additive in our pre-clinical animal studies. So we may see this in humans as well. So that, with respect to kinetics, we are collecting.
Speaker 7: blood draws after each administration over these half a dozen doses. Yes, this is Pat. Just to point out that we are the first protein replacement therapeutic that is going after this syndication especially for protein replacement using mRNA. So there is a lot of un-paved.
Speaker 8: Got it. That's very helpful. And if I could ask one more on the Cystic Fibrosis Program. Do you think it's possible we could get some initial healthy volunteer data this calendar year or is that you think that's more likely to be a 2024 event? And maybe once you have that data in hand, how quickly do you think you can pivot into treating actual cystic fibrosis?
Speaker 4: had any serious or severe adverse events. All right, we have to report on that.
Speaker 4: So, after the first survey, after the first couple cohorts, it does present the potential opportunity to amend Phase 1 to add CF patients. But whether we add CF patients to the Phase 1 trial itself or quickly pivot to a more traditional Phase 2 regulatory process is yet to be determined and communicated. Very helpful. Thanks very much.
Speaker 8: the OTC deficiency program. You know, what type of patients are you enrolling? Can you speak to their baseline characteristics? You know, are you trying to enroll patients that are stable, unstable background meds? And are they controlled, uncontrolled? And can you speculate, you know, in which type of patients you may be able to see the most pronounced?
Speaker 4: with respect to the other, and what was the other aspect of your question? You know, can you speculate on which type of patients you may actually see the most pronounced effect?
Speaker 4: Oh, okay. Well, I think that this therapeutic has the opportunity to have a biological impact on every patient injected. However, if they are already on ammonia scavengers, for example, the other biomarkers will be more meaningful, like urea, genesis, and OTC itself. But if they are not on ammonia scavengers, then of course ammonia will be looked at.
Speaker 4: and other amino acids are going to be investigated on. So the collective body of data should be sufficient to negotiate the regulatory path efficiently with regulatory agencies.
Speaker 8: Okay. Sorry. Go ahead. Phil, that's it. Did you have another question? Yeah, I have a couple questions. So, if you can give a sense of how many subjects have gone through the full dosing cycle in the phase two MADD study, and are you, is there like a
Speaker 4: safety built in by let's say the SMB? Yeah, there's always safety checkpoints, but you know, we've already got approval to proceed in a multiple ascending dose for six administrations, right? All we've communicated is multiple subjects, but if every two weeks there's another administration so you can make your assumptions based on that.
Speaker 4: There are enrolled subjects that if they continued on in the study, of course, they would have multiple administrations so far.
Speaker 8: Moving on to 032, so congratulations on that. The enrollment seems to be going well. I know you briefly mentioned preclinical data in your prepared remarks, but if you can go into a little bit more detail, perhaps Pat, what from the preclinical data sort of enhanced your conviction to move into the human studies.
Speaker 4: relatively new. It's an exciting model that the CF Foundation and many others are recommending companies to utilize because it's very likely more representative of the human condition because of the mucus that's generated in the lungs in the CF ferret model. So it's difficult to speculate or confirm that this is a validated predictive.
Speaker 4: that we properly modify and also purify our messenger RNA molecule utilizing the Arcturus proprietary technology. This is the first time an inhaled messenger RNA therapeutic for CF has entered the clinic utilizing the lunar technology.
Speaker 4: This technology has been highly optimized for bronchial epithelial cell delivery and been optimized to survive the mucous environment and optimized for inhalation and aerosolization processes.
Speaker 4: And then finally, we also note that this has also been uniquely optimized. The CFTR construct itself to increase functional activity. So there's a lot of differences in this therapeutic than what's been tried before. So we look at this CF ferret model as indicative.
Speaker 4: very meaningful because we just don't see a lot of folks or companies or therapeutics being showcased in this specific model. So I think it could potentially be very meaningful.
Speaker 10: All right, thank you for taking my questions. Yes, thank you, Pete. That is all the time we have for questions at this time. I'd like to hand the call back to Joseph Payne for closing remarks.
Speaker 4: Thanks to everybody. I think that's it. Let me...
Speaker 4: Thanks for participating on the call. If there's any remaining questions, please reach out to the team and we'll get back to you right away. Bye for now.
Speaker 10: Ladies and gentlemen, this does conclude today's teleconference. Thank you for your participation. You may disconnect your lines at this time, and have a wonderful day.