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spk02: Good afternoon, ladies and gentlemen, and welcome to the Atire Pharma fourth quarter and full year 2023 conference call. At this time, all participants are in a listen-only mode. Later, we will conduct a question and answer session, and instructions will be given at that time. To ask a question during the session, please press star 1-1 on your telephone and wait for your name to be announced. To withdraw your question, please press star 1-1 again. As a reminder, this conference is being recorded for replay purposes. It is now my pleasure to hand the conference call over to Ashley Dunstan, ATAR's Director of Investor Relations and Public Affairs. Ms. Dunstan, you may begin.
spk12: Thank you, and good afternoon, everyone. Thank you for joining us today to discuss ATAR's fourth quarter and full year 2023 operating results and corporate update. We are joined today by Dr. Sanjay Shukla, our President and CEO, and Ms. Jill Broadfoot, our CFO. On the call, Sanjay will provide an update on our corporate strategy, including our clinical program for ExoFitimod and research and discovery programs. Jill will review our financial results and our current financial position before handing it back to Sanjay to open the call up for any questions. Before we begin, I would like to remind everyone that except for statements of historical facts, the statements made by management and responses to questions on this conference call are forward-looking statements under the safe harbor provision of the Private Securities Litigation Reform Act of 1995. These statements involve risks and uncertainties that can cause actual results to differ materially from those in such forward-looking statements. Please see the forward-looking statement disclaimer in the company's press release issued this afternoon, as well as the risk factors in the company's SEC filings and included in our most recent annual report on Form 10-K, subsequently filed quarterly reports on Form 10-Q, and in our other SEC filings. Undue reliance should not be placed on forward-looking statements which speak only as of the day they are made as facts and circumstances underlying these forward-looking statements may change. Except as required by law, Atire Pharma disclaims any obligation to update these forward-looking statements to reflect future information, events, or circumstances. I will now turn the call over to Sanjay.
spk19: Thank you, Ashley. Good afternoon, everyone, and thank you for joining us for our fourth quarter and full year 2023 results conference call. At ATAR, we are leveraging evolutionary intelligence to translate tRNA synthetase biology into new therapies for fibrosis and inflammation. Our lead therapeutic candidate, efsofitimod, is a first-in-class biologic immunomodulator based on a naturally occurring lung-enriched splice variant of the tRNA synthetase, HARs. Epsophitamide selectively modulates activated myeloid cells via Neuropilin 2, or NRP2, to resolve inflammation without immune suppression and potentially prevent the progression of fibrosis. We're developing Epsophitamide as a treatment for patients with interstitial lung disease, or ILD, a group of rare immune-mediated disorders that can cause chronic inflammation and fibrosis of the lungs. 2023 was an important year for ATAR as we progressed and expanded our efsofitamide clinical development program, which now includes two ongoing clinical studies. The phase three efsofit study in patients with pulmonary sarcoidosis, a major form of ILD, and the phase two efsoconnect study in patients with ILD related to systemic sclerosis, which is known as SSC or more commonly scleroderma. Throughout the past year, we have also greatly enhanced our mechanistic understanding of the way in which Epsilfitamide is conferring its anti-inflammatory effects. NRP2 is highly expressed on activated immune cells during an inflammatory response, notably myeloid cells, including monocytes and macrophages. By binding NRP2, Epsilfitamide guides the differentiation of monocytes at the site of inflammation into a macrophage subtype that is less pro-inflammatory to resolve aberrant inflammation. Dysregulated inflammation is a hallmark of myeloid-driven diseases such as ILD, where persistent uncontrolled inflammation can lead to the progression of fibrosis. With this new understanding, we now have even greater clarity and confidence as to why F-sulfidamide may represent a breakthrough in treatment for ILD. Our lead indication for esophonamide is pulmonary sarcoidosis, the most prevalent form of ILD, where approximately 70% of patients will have symptomatic disease and nearly 20% will develop lung fibrosis. Current standard of care is primarily oral corticosteroids. A highly toxic treatment that has limited clinical evidence is broadly immunosuppressive and comes with side effects resulting in a high disease burden for patients. Epsilfit is a global pivotal phase three study evaluating Epsilfinamide compared to placebo in the context of a forced steroid taper in patients with pulmonary sarcoidosis. This study is currently enrolling at more than 90 centers in nine countries. We're pleased with the progress we've made thus far with this study, which is expected to be the largest interventional study ever conducted in sarcoidosis. Completing enrollment in Epsilfit is our primary focus. and we anticipate doing so in the second quarter of this year. In the past few months, as patients have completed the 52-week exoFIT study, we've received multiple inquiries from study principals, study principal investigators, or PIs, whose patients are requested to continue treatment once they completed the trial. While ATHER PIs and patients are all blinded to what treatment patients receive as part of the study, either esophitamide or placebo, the feedback we've received has suggested that some patients have performed well and want to continue on study drug rather than returning to the treatment regimen they had prior to the study. For some patients, that may entail resuming or increasing steroid dose, which many patients are reluctant to do. Based on this feedback, we decided to implement an individual patient expanded access program, or EAP, for patients who complete FSOFIT. This individual patient EAP is designed to allow access to FSOFITimod for patients who have or are in the process of completing FSOFIT beyond the duration of the clinical trial. The company, PIs, and patients will remain blinded to the treatment that occurred as part of FSOFIT. Safety is a key component of any EAP. We were able to implement this program based on the existing safety database from prior efsofitamide clinical studies and additional safety and tolerability data from a data safety and monitoring board, or DSMB, review of data from efsofit, which included the evaluation of patients that completed 52 weeks of treatment. The DSMB review recommended the study proceed without modification, suggesting no major safety concerns. And while many types of EAPs are typically implemented after data from a study has been unblinded, we decided to implement this individual patient EAP early, not only based on feedback and demand, but in part to continue to support those patients who have dedicated their time and entrusted us with their health by participating in this important study. This program reflects our ongoing commitment to the sarcoidosis community as we work to develop a safe and effective treatment for those in need. Our second indication for afsofitamide is SSC-ILD. SSC is a form of connective tissue disease where ILD commonly occurs and is the leading cause of mortality. Current treatment options are limited and like sarcoidosis, do not treat the underlying disease or improve quality of life. AfsoConnect is a phase two proof of concept study evaluating afsofitamide compared to placebo in patients with SSC-ILD. This study, which does the first patient last quarter, is currently open for enrollment at multiple centers in the U.S. We're focused on generating data from this study in 2024, and we expect to provide an update on the study later this year. We estimate that the two indications that comprise our current clinical program for afsofitamide Olmosarcoidosis and SSC-ILD collectively represent a potential $2 to $3 billion global market opportunity. This does not include any upside potential in other forms of the more than 200 ILDs, where esophitamide's unique mechanism of action to address complex immune pathology and desirable safety profile may be able to disrupt standard of care. While our primary focus is our clinical program for Epsifidamone, we continue to leverage our intellectual property, or IP at State, covering domains from all 20 human tRNA synthetases and utilize our platform as an engine to generate new pipeline candidates. tRNA synthetases are ancient essential proteins that have evolved novel domains to regulate diverse pathways extracellularly in humans. By identifying extracellular receptors and signaling pathways for these domains, we can elucidate the role these proteins play in cellular response and explore disease areas where they may have therapeutic benefits. Our two most advanced tRNA synthetase candidates in preclinical development are ATYR0101 and ATYR0750, both of which have specific interactions with targets that have implications in fibrosis. These targets include latent transforming growth factor beta-binding protein 1, or LTBP1, and fibroblast growth factor receptor 4, or FGFR4, respectively. AQIRO101, which is derived from a domain of the tRNA synthetase DARS, exerts its anti-fibrotic effects by selectively inducing apoptosis of myofibroblasts, targeting a key hallmark of fibrosis pathology, which is the persistence of activated myofibroblasts. This mechanism may support broad therapeutic application in indications like lung, liver, and kidney fibrosis. The hidden biology that we have been able to unlock from our platform continues to inspire us, including the way in which some of these appended domains, like F-sulfidamide, interact excessively with previously under-the-radar targets, like NRP2, and in particular, its role as an immune regulator, or by more well-known targets in unique ways, like 0101 and 0750. This platform, which is based on signaling pathways that have been have evolved over billions of years to maintain homeostasis is an excellent example of bioinnovation and has the potential to disrupt traditional drug discovery, a process that is increasingly reliant on exploiting existing signaling pathways to generate Me Too therapies. Our conviction as to the potential of tRNA synthetase biology to lead to transformative medicines continues to grow stronger as our research advances. I'll now turn it over to our Chief Financial Officer, Jill Broadfoot, to review our financial results.
spk01: Thank you, Sanjay. We ended 2023 with $101.7 million in cash, restricted cash, cash equivalents, and investments. Collaboration and license revenue related to the current agreement was $0.4 million for the year ended 2023. which consisted of drug product materials sold to Kirin for the Japan portion of Epsofit. As a reminder, Kirin is our partner for the development and commercialization of Epsofitimod for ILB in Japan, where they are responsible for costs related to Epsofit in Japan and purchase drug product material from us with a small markup. Under this agreement, we have received $20 million in upfront and milestone payments from this partnership to date, and we are eligible to receive up to an additional $155 million, which is primarily related to certain development and regulatory milestones. Research and development expenses were $42.3 million for the year-ended 2023, which consisted primarily of clinical trial costs for the EFSA-FIT and EFSA-Connect studies, manufacturing costs for the EFSA-FITimod program, and research and development costs for the ExoPenMod and Discovery programs. General and administrative expenses were $13 million for the year ended 2023. Based on our current operational plans and existing cash, we maintain our prior financial guidance and believe our cash runway is expected to be sufficient to fund the company through the filing of a biologics license application for efsofitimod and pulmonary sarcoidosis. This takes into account the continued allocation of the majority of our resources to our efsofitimod clinical development program, which is our main value driver for the company, while also committing judicious resources to our tRNA synthetase pipeline candidates to maintain an active discovery program and advance our IP estate. Furthermore, our forecast for our cash guidance does not include any potential future milestone payments from Kieran or any proceeds that may result from additional potential partnerships or sources of non-diluted funding, though it does consider proceeds from the prudent use of our at-the-market facility. We implemented this operational plan more than a year ago, driven by our emphasis on maximizing efficiency and adapting to prevailing macroeconomic conditions. And this plan continues to be an effective way to meet our primary corporate objectives relative to optimal capital utilization.
spk00: Now, I'd like to turn the call back over to Sanjay before we open it up to Q&A.
spk16: Thank you, Jill.
spk19: As we look back on the past year, we're filled with optimism for the future. Our scientific expertise in understanding tRNA biology is unparalleled across the industry. We're leveraging this unique biology and harnessing ancient genetic codes developed throughout the course of billions of years in a trillion species. While other companies are just beginning to explore the untapped potential of tRNA biology, we're leading the way with our needs of understanding the science. which we have leveraged to progress a phase three therapeutic candidate. It's not just our understanding of the endless frontier of tRNA synthetase biology that sets us apart. F-sulfidamide represents the reality, not just the concept of a potential near-term meaningful tRNA-based therapy that can change the lives of patients. We're on the cusp of a once-in-a-generation therapy for sarcoidosis. engineered from our natural physiology and are marching towards the chance to transform the lives of ILD patients. Pulmonary sarcoidosis is only the starting point for how efsofitimod may be able to help millions of ILD patients. As the only biotech company in phase three development for this indication, we note the accelerating pipeline of candidates, not just for sarcoidosis, but also for ILD more broadly. Other biopharmaceutical companies who are following our lead also see the multi-billion dollar market opportunity in this space. While these companies have yet to show proof of concept, Epsofitimod is well beyond that, with patients requesting to remain on treatment as they complete our current trial in sarcoidosis due to the benefits they are experiencing.
spk16: So we believe it's just the beginning for us here at Atire.
spk19: New validated targets are emerging from our evolutionary intelligence or EI driven drug discovery platform. Alternatively, artificial intelligence or AI driven drug discovery is thrilling in its infancy. And these unproven hyped approaches do not have the advantage of our EI drug discovery engine. ATAR is uncovering hidden functions embedded in our genetic code that were developed throughout evolution, encompassing infinite real-life biological experiments in over a trillion species over billions of years.
spk06: Just think about that.
spk19: The synthetase domains that we have mapped have developed over billions of years since the beginning of the Tree of Life. Since the evolution of complex systems, all species, including humans, share these same genetic domains, that were created over billions of years of biological stress and strain, allowing all species to thrive and overcome disease and dysfunction. As companies embark on computational models and approaches to quote, unquote, find new and novel targets, Atire, meanwhile, has an IP library of hundreds of potentially efficacious proteins just waiting to be unleashed from our own genetic programming leveraging novel domains that have been tested and validated since the beginnings of life, that we now look to target towards current-day disease and dysfunction. I hope you can now better understand why we're so optimistic here at Atire, not only in the short term with F-sulfuramide, but also in the long-term potential for Atire to become the next transformative biotech company of our time.
spk16: We appreciate your interest at this time,
spk19: Jill and I will be happy to take your questions.
spk02: Thank you. As a reminder, to ask a question, please press star 1-1 on your telephone and wait for your name to be announced. To withdraw your question, please press star 1-1 again. One moment for questions.
spk06: Our first question comes from Gregory Renzo with RBC Capital Markets.
spk02: You may proceed.
spk11: Great. Good afternoon, Sanjay and Jill. Congrats on the progress. Thanks for the updates and thanks for taking my questions. Sanjay, maybe just going to the expanded access program, it's great to hear the call you've provided. Just wanted to ask if you could maybe put into context the significance of this to you and to SFIDIMON, especially as these blinded patients are undergoing the considerable taper for the trial. And then secondly, just any other details that you are seeing, whether it's the patterns from the centers, you know, where that or what that demand is in order to have these patients and these trial participants to continue with respect to the program. And then I have a follow-up as well. Thanks.
spk19: Sure, Greg. Thanks for the question. So really over the last, I would say, six months, if you will, We have been hearing, I've been hearing directly from, oh, I'd say, you know, at this point, dozens of PIs around the world about how happy they are with the performance of the study drug, of the study drug. I'm going to highlight, say that over and over again, because we don't know if it's F-sulfatamide or placebo, and how their patients are performing quite well in the trial. It had reached the point, however, that patients now rolling off the trial frankly, don't want to go back on steroids if they have been able to taper off or increase their steroids. Many of these patients have been on steroids for 5, 10, sometimes 20 years. So this has offered a real unique opportunity in this trial to live their lives with a lot less or no steroids. So It had gotten to the point where I thought it's best for us to even think about an EAP really early on. Most of these programs are put in place after you unblind and you finish the trial. But with data, obviously, we're looking at data next year. We wanted to be able to really fulfill our mission to really be patient oriented, patient first, and look to implement this program. Since we've put out the note, I think it's been received even with more interest. This is certainly something that is a relief to a lot of these patients, and I do think that it's atypical for a company to do this sort of thing ahead of data release. I would much rather have patients wanting to remain in the trial than happy to get out of the trial, put it that way. So I think for me, clinically, when I think about this clinically, I think we're doing the right thing here ethically as well. And I think it's probably the best biomarker you could ask for, in my opinion.
spk09: Yeah, that's helpful. Appreciate the color.
spk11: And then just maybe shifting to SO Connect, just thinking about – That trial opened for enrollment across these multiple centers, and I think I heard you mention an update should come later this year. Just with respect to that update, what is your objective there? Of course, we're all interested in the 12-week skin assessment as well, but just curious how we should be thinking about that. Is it more procedural in nature, or is it something where we'd like to at least look and see some details of that program? Thanks again, and congrats.
spk19: Sure, so we're just in that phase now where I think you'll see six, eight, ten centers up and ready to enroll at this point. Getting that first patient in last quarter was important, but now we're getting into the meat of these sites being able to enroll. And like I said, we'll have a good idea later this year around what kind of data we'll put out here. There are opportunities with that data set. to look at things, even a three-month endpoint, which is some of the skin scoring that you pointed out there. But like I said, I want to see how we do here in the first half of the year with regards to enrolling in that trial now that we are approaching more or less full activation or nearing full activation of the centers that we want to get up and enrolling and screening for that trial.
spk06: Got it. Thanks again. Thanks.
spk02: Thank you. One moment for questions. Our next question goes from Joe Pangen. It's with HC Wainwright. You may proceed.
spk20: Hey, everybody. Good afternoon. Thanks for taking the questions. Sanjay, I want to start first also with the EAP. So it's pretty intriguing, like you said, to be able to have an EAP ahead of data. And if I heard you correctly, both physicians and patients remain blinded even after exiting ESO FIT as to what they had been previously on. So first, was curious if you could provide any more color or anecdotes that the physicians are giving you with regard to wanting to be into the EAP. You alluded to, you know, maybe quote that they were, you know, patients are feeling better, but are there any more details you can share around that? Second, what are the general logistics of setting up an EAP? And related to that, what are the cost implications? Thanks.
spk19: Yeah, great questions, Joe. So I think just at a high level, without sort of getting into specific endpoints, what it really comes down to is these physicians have to put these patients back on something which was presumably steroids as they entered the trial. And now the patients are refusing to want to do that. So it speaks to the lack of options that we have here for sarcoidosis. These patients, of course, are not so fibrotic that they need the anti-fibrotics. So now there's a quandary. Patients are doing well and they've tasted kind of the best experience recently. in their recent care, whether it's removing steroids partially or completely. So I think we have to sort of step up here because many of the experts were sort of in a quandary now. So I think it speaks to the fact that, as I said, this is a really strong biomarker in my mind to have this kind of demand from centers and from patients. With regards to how would this logistically be administered, here in the U.S., it typically is a direct submission from the PI. I would think of this as more like classical compassionate use as opposed to some of the EAPs we know of when drugs are approved and we're waiting for them to get to the market. So this would be an individual PI at their center cross-referencing our IMD and submitting into the FDA. Because we have some of those positive risk evaluations from a DSMB perspective, we feel as though we're also in good shape to support this vertical. And we may provide small grants to kind of help patients with some of their travel or maybe even some infusion costs that's going to be on a case-by-case level but this will be something that the centers themselves will bear a little bit more of the financial burden if you will we'll provide drug for free and we're in good shape from a drug supply perspective but i do not expect nor should any of the investors expect that this will materially impact uh really our our cash position we have the drug we have the drug supply we'll help out with some administrative templates and things like that, and maybe perhaps some small stipends to help patients. When you look at Europe, you look at other places around the world, that's going to be really a little bit more specific. Each country, for example, in Europe, some of them have more formal pathways that are more streamlined, similar to here in the U.S. Other regions, we're working more closely with individual hospitals in determining how best to provide drug to those patients that want it. So it's an atypical program, but I also think it speaks to our commitment to really respond to this demand from patients and PIs.
spk20: Very, very helpful. And then I guess, you know, when you consider, you know, being in a pivotal study right now that we'll go into, call it, you know, roughly mid-next year and the phase two SSC study, How would you sort of characterize at this moment your manufacturing needs to get beyond that and to potential commercialization for FSO?
spk19: Yeah, so we had transitioned a few years ago to a more commercially oriented and scalable type of partnership. So we're in really good shape here to essentially have drug manufactured, clear all the hurdles also with the FDA to support a launch with some of those initial batches and runs, you know, more or less mapped out here. You know, of course, there's, you know, if you go out five, seven years from now, I wouldn't say, you know, we have that type of capacity. But in the short term here to get through our clinical program and the early commercialization work, we're in good shape here. And we're also fortunate that the manufacturer is here in the U.S., so we have good control, good line of sight. I know things are happening worldwide with manufacturers right now where there's a lot of controversy if it's not U.S.-based, but we're de-risked there as well by having a good manufacturing plan that we also spend a lot of time talking to regulators with to make sure they want to make sure that we have that drug product ready for patients because there's going to be a high amount of demand assuming this drug gets approved. These patients have been, as I said, waiting for a therapy their whole lives.
spk20: Well, that five to seven-year comment would be a great problem to have, but thanks for all the comments, Sanjay.
spk02: Sure. Thank you. One moment for questions. Our next question comes from Yasmeen Rahimi with Piper Sandler. You may proceed.
spk07: Hey, team. This is John Guion for Yaz. Congrats on the continued progress, and thanks for taking our questions. For EFSO-FIT, do you still need additional sites to bring enrollment to the finish line, and is there actually an opportunity for additional enrollment beyond the 264 patients? And second, could you provide more color on the percentage or number of patients continuing from EFSO-FIT to the EAP program?
spk19: Yeah, so 264 is kind of our goal. That's A lot of that is based on our power calculations. It assumes that you can have a nominal dropout rate. So we're well powered, actually overpowered in many ways compared to other phase three trials. 92% powered here. 264 is our goal. We still view that as a solid number there. It allows for also a buffer that if you had 10% of the patients discontinue for any reason, you'd still be able to hit your power calculation. So we're still set with that number. Sometimes you can see trials that might be a few patients over, a few patients under. A lot of that has to do with patient screening right there at the end. In our last trial, which was the 36-patient trial, we had eligible patients. The day we stopped screening, we ended up having a few more patients one more patient enrolled in that trial because they were screen eligible and we don't want to sort of shut down enrollment if someone is already screened and is eligible. So I would say that we're still going to be right in that ballpark there of 264. With regards to how many are going to move into the EAP, we don't know yet. We are continuing to generate requests. As you can imagine, as more and more patients finish the trial, we may get more patients interested. My early read on it is we're well built here to have a substantial number of patients. If they want the drug, we're ready to provide it. But I don't have an exact number. But we are prepared to potentially give the AP program, have it there for, you know, a very, very large number of patients. We'll have to wait and see what kind of demand it continues. Early on right now, I'll say, since we put the announcement, we probably had, you know, even more demand since we put the announcement out.
spk07: Thank you so much for the call.
spk02: Thank you. One moment for questions. Our next question comes from Yale Jen with Laidlaw and Co., you may proceed.
spk05: Good afternoon, and thanks for taking the questions. I came in late, so maybe if you can reiterate a little bit in terms of FSO fit enrollment. I know the press release indicated it's on track. Was there any other colors of that? Then I have another question.
spk19: No, Yale, very much on track. I'm very much feeling good. you know, about Q2 this year. So very much on track.
spk05: Okay, great. That's good. And you mentioned a few, two preclinical programs, and they look very interesting at this juncture. Was those the programs you intend to partner out at some point, or what will be the path you are thinking about moving forward with those preclinical programs?
spk19: Well, I think it's, I mean, for everything as a biotech, like, you know, kind of where we are, you always have to look at partnering and those sorts of opportunities. There's no shortage of interests. Obviously, we have, you know, a rather novel platform here, and the platform now is generating more and more validated opportunities. So that will be something that we'll look at. We always look at these sort of potential arrangements. At the same time, we also are potentially sitting on assets here that really are producing signals really never seen before. When you talk about being able to induce apoptosis of myofibroblasts, I don't think you'll hear too many companies really seeing those kinds of early signals. So that's where this biology, I think it's important for everyone to understand. There's hidden potential here. There's hidden potential efficacy here. And I think what ATAR is doing is really looking at these genetic codes and saying, where can they best be applied where there is disease and dysfunction? So unlike, you know, generations of Me Too therapies that have been out there that are not disease modifying, I think all of our approaches, Epsom, Fitamod, and even some of the pipeline approaches we have here we have a very, very high bar here. They have to be disease modifying. They have to really be showing activity here that is two standard deviations better than most opportunities out there. And I think we want to be careful about giving away these gems too early, but we're open to talk to big players at all times.
spk05: Okay, great. Maybe just Just making one more question here, which is that you said the trial is on track to complete enrollment second quarter. And just curious, in terms of the current in Japan, given they have smaller size, have they already completed any colors on that? And thanks.
spk19: Yeah, I can't really get into who's completed it. I mean, it more or less is competitive enrollment. There aren't hard caps per se, country by country. We have our goals on what we want, where we want to get to. So I wouldn't think of it as a separate trial. We've said in the past that a region like Japan, you might expect 25 to 30 patients there. So Japan, like the rest of the world that we're working in right now, very much on track, you know, to meet our projections to get this trial wrapped up here in Q2, at least enrollment.
spk05: Okay, great. Thanks a lot, and congrats on all the progress.
spk16: Thank you, Gail.
spk02: Thank you. And as a reminder, to ask a question, please press star 1-1 on your telephone and wait for your name to be announced. One moment for questions. Our next question comes from Robert LaBoyer with Noble Capital Markets. You may proceed.
spk04: Good afternoon and congratulations on all the progress. I also have a question on the Expanded Access Program and was wondering whether you're going to be compiling endpoints and other data that can be used in the BLA or whether you can present some of the extension study data at medical meetings either before the data is announced or afterwards?
spk19: Yeah, it's a great question, Robert. So, just want to highlight one thing here is this is being conducted outside of our protocol, and that's being done for a few reasons. Number one, we want to hit our BLA timelines and submit, finish the primary trial, if you will, the primary portion of the trial and submit that. This secondary piece of the trial is sitting outside of our protocol. Why are we doing it that way? Well, first of all, health authorities didn't mandate that we do any kind of open label extension. There was no known safety risks observed early in the program, in our last program, and we had long-term safety data already from from this trial. So there was really no need to have a more formal open label extension or anything like that. So this trial will be done outside of the boundaries of our protocol. Now, already I would say many academics are really interested in looking at patients 18, 24 months out, perhaps with or without efsofitamide long-term. So I can't say that there would not be an academic consortium of some of our experts that would want to put out this data. I would support that. I think they could look at different things that within the confines of our own protocol we haven't been able to look at, to look at long-term steroid reduction effects. Some of the early promising things we see with regards to things like weight loss, It was always theoretical. You get off steroids, you could help with weight. We're seeing some rather remarkable signals there. It's early. It's anecdotal. This might be something that we could look at, those investigators could look at in some sort of academic survey. They may want to look at some sort of imaging data two or three years later. But right now, we're focused on getting this drug approved, getting the BLA out. trickle of that kind of interesting data could almost be a head start to phase four. That's the way I look at it. We also don't want to spend money on that sort of downstream side of the fence right now. But I think there's going to be some intriguing potential there to collect data, not only for the patients that continue in the trial, but also those that might have to revert back to steroids and see what happens with those patients. But I think it's a great question, Robert. Really nice how you're thinking about that.
spk16: And it's already things that experts are noodling over potentially to do something outside of the trial.
spk03: Okay, great.
spk02: Thank you very much. Thank you. I would now like to turn the call back over to Sanjay for any closing remarks.
spk19: Great. Well, we thank everybody's interest. Great questions today. I know there's a lot of expectation of Everyone's really looking forward to getting this trial wrapped up here. We are too. Appreciate everyone's interest here. Thank you for following us and look forward to talking to you in the future. Thanks again.
spk02: Thank you for your participation.
spk06: You may now disconnect. Amen. Thank you. Thank you. Thank you. music music
spk02: Good afternoon, ladies and gentlemen, and welcome to the Atire Pharma fourth quarter and full year 2023 conference call. At this time, all participants are in a listen-only mode. Later, we will conduct a question and answer session and instructions will be given at that time. To ask a question during the session, please press star 1-1 on your telephone and wait for your name to be announced. To withdraw your question, please press star 1-1 again. As a reminder, this conference is being recorded for replay purposes. It is now my pleasure to hand the conference call over to Ashley Dunstan, ATAR's Director of Investor Relations and Public Affairs. Ms. Dunstan, you may begin.
spk12: Thank you, and good afternoon, everyone. Thank you for joining us today to discuss Atire's fourth quarter and full year 2023 operating results and corporate update. We are joined today by Dr. Sanjay Shukla, our president and CEO, and Ms. Jill Broadfoot, our CFO. On the call, Sanjay will provide an update on our corporate strategy, including our clinical program for exofitamide and research and discovery programs. Jill will review our financial results, and our current financial position before handing it back to Sanjay to open the call up for any questions. Before we begin, I would like to remind everyone that except for statements of historical facts, the statements made by management and responses to questions on this conference call are forward-looking statements under the safe harbor provision of the Private Securities Litigation Reform Act of 1995. These statements involve risks and uncertainties that can cause actual results to differ materially from those in such forward-looking statements. Please see the forward-looking statement disclaimer in the company's press release issued this afternoon, as well as the risk factors in the company's SEC filings and included in our most recent annual report on Form 10-K, subsequently filed quarterly reports on Form 10-Q, and in our other SEC filings. Undue reliance should not be placed on forward-looking statements which speak only as of the day they are made as facts and circumstances underlying these forward-looking statements may change. Except as required by law, Atire Pharma disclaims any obligation to update these forward-looking statements to reflect future information, events, or circumstances. I will now turn the call over to Sanjay.
spk19: Thank you, Ashley. Good afternoon, everyone, and thank you for joining us for our fourth quarter and full year 2023 results conference call. At Atire, we are leveraging evolutionary intelligence. to translate tRNA synthetase biology into new therapies for fibrosis and inflammation. Our lead therapeutic candidate, efsofitimod, is a first-in-class biologic immunomodulator based on a naturally occurring lung-enriched splice variant of the tRNA synthetase, HARs. Efsofitimod selectively modulates activated myeloid cells via Neuropilin 2, or NRP2, to resolve inflammation without immune suppression and potentially prevent the progression of fibrosis. We're developing efsofitamide as a treatment for patients with interstitial lung disease, or ILD, a group of rare immune-mediated disorders that can cause chronic inflammation and fibrosis of the lungs. 2023 was an important year for ATAR as we progressed and expanded our efsofitamide clinical development program. which now includes two ongoing clinical studies, the Phase III EPSO-Fit study in patients with pulmonary sarcoidosis, a major form of ILD, and the Phase II EPSO-Connect study in patients with ILD related to systemic sclerosis, which is known as SSC or more commonly scleroderma. Throughout the past year, we have also greatly enhanced our mechanistic understanding of the way in which Epsilfitamide is conferring its anti-inflammatory effects. NRP2 is highly expressed on activated immune cells during an inflammatory response, notably myeloid cells, including monocytes and macrophages. By binding NRP2, Epsilfitamot guides the differentiation of monocytes at the site of inflammation into a macrophage subtype that is less pro-inflammatory to resolve aberrant inflammation. is a hallmark of myeloid-driven diseases such as ILD, where persistent, uncontrolled inflammation can lead to the progression of fibrosis. With this new understanding, we now have even greater clarity and confidence as to why F-sulfidamide may represent a breakthrough in treatment for ILD. Our lead indication for F-sulfidamide is pulmonary sarcoidosis, the most prevalent form of ILD. where approximately 70% of patients will have symptomatic disease and nearly 20% will develop lung fibrosis. Current standard of care is primarily oral corticosteroids. A highly toxic treatment that has limited clinical evidence is broadly immunosuppressive and comes with side effects resulting in a high disease burden for patients. Epsilfit is a global pivotal phase three study evaluating Epsilfinamide compared to placebo in the context of a forced steroid taper in patients with pulmonary sarcoidosis. This study is currently enrolling at more than 90 centers in nine countries. We're pleased with the progress we've made thus far with this study, which is expected to be the largest interventional study ever conducted in sarcoidosis. Completing enrollment in Epsil Fit is our primary focus, and we anticipate doing so in the second quarter of this year. In the past few months, as patients have completed the 52-week exofit study, we've received multiple inquiries from study principal investigators, or PIs, whose patients are requested to continue treatment once they completed the trial. While ATHER, PIs, and patients are all blinded to what treatment patients receive as part of the study, either esophitamide or placebo, The feedback we've received has suggested that some patients have performed well and want to continue on study drug rather than returning to the treatment regimen they had prior to the study. For some patients, that may entail resuming or increasing steroid dose, which many patients are reluctant to do. Based on this feedback, we decided to implement an individual patient expanded access program, or EAP, for patients who complete FSOFET. This individual patient EAP is designed to allow access to efsofitamide for patients who have or are in the process of completing efsofit beyond the duration of the clinical trial. The company, PIs, and patients will remain blinded to the treatment that occurred as part of efsofit. Safety is a key component of any EAP. We were able to implement this program based on the existing safety database from prior efsofitimod clinical studies and additional safety and tolerability data from a data safety and monitoring board, or DSMB, review of data from efsofit, which included the evaluation of patients that completed 52 weeks of treatment. The DSMB review recommended the study proceed without modification, suggesting no major safety concerns. And while many types of EAPs are typically implemented after data from a study has been unblinded, we decided to implement this individual patient EAP early, not only based on feedback and demand, but in part to continue to support those patients who have dedicated their time and entrusted us with their health by participating in this important study. This program reflects our ongoing commitment to the sarcoidosis community as we work to develop a safe and effective treatment for those in need. Our second indication for efsofitamide is SSC-ILD. SSC is a form of connective tissue disease where ILD commonly occurs and is the leading cause of mortality. Current treatment options are limited and like sarcoidosis, do not treat the underlying disease or improve quality of life. EfsoConnect is a phase two proof of concept study evaluating efsofitamide compared to placebo in patients with SSC-ILD. This study, which does the first patient last quarter, is currently open for enrollment at multiple centers in the U.S. We're focused on generating data from this study in 2024, and we expect to provide an update on the study later this year. We estimate that the two indications that comprise our current clinical program for absofitamide Olmec sarcoidosis and SSC-ILD collectively represent a potential $2 to $3 billion global market opportunity. This does not include any upside potential in other forms of the more than 200 ILDs, where esophitamide's unique mechanism of action to address complex immune pathology and desirable safety profile may be able to disrupt standard of care. While our primary focus is our clinical program for Epsifidamone, we continue to leverage our intellectual property, or IP at State, covering domains from all 20 human tRNA synthetases and utilize our platform as an engine to generate new pipeline candidates. tRNA synthetases are ancient essential proteins that have evolved novel domains to regulate diverse pathways extracellularly in humans. By identifying extracellular receptors and signaling pathways for these domains, we can elucidate the role these proteins play in cellular response and explore disease areas where they may have therapeutic benefit. Our two most advanced tRNA synthetase candidates in preclinical development are ATYR0101 and ATYR0750, both of which have specific interactions with targets that have implications in fibrosis. These targets include latent transforming growth factor beta binding protein 1, or LTBP1, and fibroblast growth factor receptor 4, or FGFR4, respectively. AQIR 0101, which is derived from a domain of the tRNA synthetase DARS, exerts its anti-fibrotic effects by selectively inducing apoptosis of myofibroblasts, targeting a key hallmark of fibrosis pathology, which is the persistence of activated myofibroblasts. This mechanism may support broad therapeutic application in indications like lung, liver, and kidney fibrosis. The hidden biology that we have been able to unlock from our platform continues to inspire us, including the way in which some of these appended domains, like F-sulfidamide, interact excessively with previously under-the-radar targets, like NRP2, and in particular, its role as an immune regulator, or by more well-known targets in unique ways, like 0101 and 0750. This platform, which is based on signaling pathways that have have evolved over billions of years to maintain homeostasis is an excellent example of bioinnovation and has the potential to disrupt traditional drug discovery, a process that is increasingly reliant on exploiting existing signaling pathways to generate Me Too therapies. Our conviction as to the potential of tRNA synthetase biology to lead to transformative medicines continues to grow stronger as our research advances. I'll now turn it over to our Chief Financial Officer, Jill Broadfoot, to review our financial results.
spk01: Thank you, Sanjay. We ended 2023 with $101.7 million in cash, restricted cash, cash equivalents, and investments. Collaboration and license revenue related to the current agreement was $0.4 million for the year ended 2023. which consisted of drug product materials sold to Kirin for the Japan portion of FSOFIT. As a reminder, Kirin is our partner for the development and commercialization of FSOFIT mod for ILB in Japan, where they are responsible for costs related to FSOFIT in Japan and purchase drug product material from us with a small markup. Under this agreement, we have received $20 million in upfront and milestone payments from this partnership to date, and we are eligible to receive up to an additional $155 million, which is primarily related to certain development and regulatory milestones. Research and development expenses were $42.3 million for the year-ended 2023, which consisted primarily of clinical trial costs for the EFSA-FIT and EFSA-Connect studies, manufacturing costs for the EFSA-FITimod program, and research and development costs for the ExoFinMod and Discovery programs. General and administrative expenses were $13 million for the year end of 2023. Based on our current operational plans and existing cash, we maintain our prior financial guidance and believe our cash runway is expected to be sufficient to fund the company through the filing of a biologics license application for efsofitimod and pulmonary sarcoidosis. This takes into account the continued allocation of the majority of our resources to our efsofitimod clinical development program, which is our main value driver for the company, while also committing judicious resources to our tRNA synthetase pipeline candidates to maintain an active discovery program and advance our IP estate. Furthermore, our forecast for our cash guidance does not include any potential future milestone payments from Kieran or any proceeds that may result from additional potential partnerships or sources of non-diluted funding, though it does consider proceeds from the prudent use of our at-the-market facility. We implemented this operational plan more than a year ago, driven by our emphasis on maximizing efficiency and adapting to prevailing macroeconomic conditions. And this plan continues to be an effective way to meet our primary corporate objectives relative to optimal capital utilization.
spk00: Now, I'd like to turn the call back over to Sanjay before we open it up to Q&A.
spk06: Thank you, Jill.
spk19: As we look back on the past year, we're filled with optimism for the future. Our scientific expertise in understanding tRNA biology is unparalleled across the industry. We're leveraging this unique biology and harnessing ancient genetic codes developed throughout the course of billions of years in a trillion species. While other companies are just beginning to explore the untapped potential of tRNA biology, we're leading the way with our needs of understanding the science. which we have leveraged to progress a phase three therapeutic candidate. It's not just our understanding of the endless frontier of tRNA synthetase biology that sets us apart. F-sulfidamide represents the reality, not just the concept of a potential near-term meaningful tRNA-based therapy that can change the lives of patients. We're on the cusp of a once-in-a-generation therapy for sarcoidosis. engineered from our natural physiology, and are marching towards the chance to transform the lives of ILD patients. Pulmonary sarcoidosis is only the starting point for how efsofitamide may be able to help millions of ILD patients. As the only biotech company in Phase III development for this indication, we note the accelerating pipeline of candidates, not just for sarcoidosis, but also for ILD more broadly. Other biopharmaceutical companies who are following our lead also see the multi-billion dollar market opportunity in this space. While these companies have yet to show proof of concept, Epsofitimod is well beyond that, with patients requesting to remain on treatment as they complete our current trial in sarcoidosis due to the benefits they are experiencing.
spk06: So we believe it's just the beginning for us here at Atire.
spk19: New validated targets are emerging from our evolutionary intelligence or EI driven drug discovery platform. Alternatively, artificial intelligence or AI driven drug discovery is thrilling in its infancy. And these unproven hyped approaches do not have the advantage of our EI drug discovery engine. ATAR is uncovering hidden functions embedded in our genetic code. that were developed throughout evolution, encompassing infinite real-life biological experiments in over a trillion species over billions of years.
spk06: Just think about that.
spk19: The synthetase domains that we have mapped have developed over billions of years since the beginning of the tree of life. Since the evolution of complex systems, all species, including humans, share these same genetic domains. that were created over billions of years of biological stress and strain, allowing all species to thrive and overcome disease and dysfunction. As companies embark on computational models and approaches to quote-unquote find new and novel targets, Atire meanwhile has an IP library of hundreds of potentially efficacious proteins just waiting to be unleashed from our own genetic programming leveraging novel domains that have been tested and validated since the beginnings of life, that we now look to target towards current day disease and dysfunction. I hope you can now better understand why we're so optimistic here at Atire, not only in the short term with F-sulfuramide, but also in the long-term potential for Atire to become the next transformative biotech company of our time.
spk16: We appreciate your interest at this time.
spk19: Joe and I will be happy to take your questions.
spk02: Thank you. As a reminder, to ask a question, please press star 1-1 on your telephone and wait for your name to be announced. To withdraw your question, please press star 1-1 again. One moment for questions.
spk06: Our first question comes from Gregory Renzo with RBC Capital Markets.
spk02: You may proceed.
spk11: Great. Good afternoon, Sanjay and Jill. Congrats on the progress. Thanks for the updates and thanks for taking my questions. Sanjay, maybe just going to the expanded access program, it's great to hear the color you've provided. Just wanted to ask if you could maybe put into context the significance of this to you and to SFIDIMON, especially as these blinded patients are undergoing the considerable taper for the trial. And then secondly, just any other details that you are seeing, whether it's the patterns from the centers, you know, where that or what that demand is in order to have these patients and these trial participants to continue with respect to the program. And then I have a follow-up as well. Thanks.
spk19: Sure, Greg. Thanks for the question. So really over the last, I would say, six months, if you will, We have been hearing, I've been hearing directly from, oh, I'd say, you know, at this point, dozens of PIs around the world about how happy they are with the performance of the study drug, of the study drug. I'm going to highlight, say that over and over again, because we don't know if it's F-sulfatamide or placebo, and how their patients are performing quite well in the trial. It had reached the point, however, that patients now rolling off the trial frankly, don't want to go back on steroids if they have been able to taper off or increase their steroids. Many of these patients have been on steroids for 5, 10, sometimes 20 years. So this has offered a real unique opportunity in this trial to live their lives with a lot less or no steroids. So It had gotten to the point where I thought it's best for us to even think about an EAP really early on. Most of these programs are put in place after you unblind and you finish the trial. But with data, obviously, we're looking at data next year. We wanted to be able to really fulfill our mission to really be patient oriented, patient first, and look to implement this program. Since we've put out the note, I think it's been received even with more interest. This is certainly something that is a relief to a lot of these patients, and I do think that it's atypical for a company to do this sort of thing ahead of data release. I would much rather have patients wanting to remain in the trial than happy to get out of the trial, put it that way. So I think for me, clinically, when I think about this clinically, I think we're doing the right thing here ethically as well. And I think it's probably the best biomarker you could ask for, in my opinion.
spk09: Yeah, that's helpful. Appreciate the color.
spk11: And then just maybe shifting to SO Connect, just thinking about... That trial opened for enrollment across these multiple centers, and I think I heard you mention an update should come later this year. Just with respect to that update, what is your objective there? Of course, we're all interested in the 12-week skin assessment as well, but just curious how we should be thinking about that. Is it more procedural in nature, or is it something where we'd like to at least look and see some details of that program? Thanks again, and congrats.
spk19: Sure, so we're just in that phase now where I think you'll see six, eight, 10 centers up and ready to enroll at this point. Getting that first patient in last quarter was important, but now we're getting into the meat of these sites being able to enroll. And like I said, we'll have a good idea later this year around what kind of data we'll put out here. There are opportunities with that data set to look at things even a three-month endpoint, which is some of the skin scoring that you pointed out there. But like I said, I want to see how we do here in the first half of the year with regards to enrolling in that trial now that we are approaching more or less full activation or nearing full activation of the centers that we want to get up and enrolling and screening for that trial.
spk06: Got it. Thanks again. Thanks. Thank you.
spk02: One moment for questions. Our next question goes from Joe Pangen. It's with HC Wainwright. You may proceed.
spk20: Hey, everybody. Good afternoon. Thanks for taking the questions. Sanjay, I want to start first also with the EAP. So it's pretty intriguing, like you said, to be able to have an EAP ahead of data. And if I heard you correctly, both physicians and patients remain blinded even after exiting ESOFIT as to what they had been previously on. So first, was curious if you could provide any more color or anecdotes that the physicians are giving you with regard to wanting to be into the EAP. You alluded to, you know, maybe quote that they were, you know, patients are feeling better, but are there any more details you can share around that? Second, you know, what are the general logistics of setting up an EAP And related to that, what are the cost implications? Thanks.
spk19: Yeah, great question, Joe. So I think just at a high level, without sort of getting into specific endpoints, what it really comes down to is these physicians have to put these patients back on something which was presumably steroids, you know, as they entered the trial. And now the patients are refusing to want to do that. So it speaks to the lack of options that we have here for sarcoidosis. These patients, of course, are not so fibrotic that they need the antifibrotics. So now there's a quandary. Patients are doing well and they've tasted kind of the best experience recently in their recent care, whether it's removing steroids, partially or completely. So I think we have to sort of step up here because many of the experts were sort of in a quandary now. So I think it speaks to the fact that, as I said, this is a really strong biomarker in my mind to have this kind of demand from centers and from patients. With regards to how would this logistically be administered, Here in the U.S., it typically is a direct submission from the PI. I would think of this as more like classical compassionate use as opposed to some of the EAPs we know of when drugs are approved and we're waiting for them to get to the market. So this would be an individual PI at their center cross-referencing our IMD and submitting into the FDA. Because we have some of those positive risk evaluations from a DSMB perspective. We feel as though we're also in good shape to support this vertical. And we may provide small grants to kind of help patients with some of their travel or maybe even some infusion costs. That's going to be on a case-by-case level. But this will be something that the centers themselves will bear a little bit more of the financial burden, if you will. We'll provide drug for free, and we're in good shape from a drug supply perspective. But I do not expect, nor should any of the investors expect, that this will materially impact really our cash position. We have the drug. We have the drug supply. We'll help out with some administrative templates and things like that. and maybe perhaps some small stipends to help patients. When you look at Europe, you look at other places around the world, that's going to be really a little bit more specific. Each country, for example, in Europe, some of them have more formal pathways that are more streamlined, similar to here in the U.S. Other regions, we're working more closely with individual hospitals in determining how best to provide drug to those patients that want it. So it's an atypical program, but I also think it speaks to our commitment to really respond to this demand from patients and PIs.
spk20: Very, very helpful. And then I guess, you know, when you consider, you know, being in a pivotal study right now that we'll go into, call it, you know, roughly mid-next year and the Phase II SSC study, how would you sort of characterize at this moment your manufacturing needs to get beyond that and to potential commercialization for FSO?
spk19: Yeah, so we had transitioned a few years ago to a more commercially oriented and scalable type of partnership. So we're in really good shape here to essentially have drug manufactured, clear all the hurdles also, with the FDA to support a launch with some of those initial batches and runs more or less mapped out here. Of course, if you go out five, seven years from now, I wouldn't say we have that type of capacity. But in the short term here, to get through our clinical program and the early commercialization work, we're in good shape here. And we're also fortunate that the manufacturer is here in the U.S., so we have good control, good line of sight. I know things are happening worldwide with manufacturers right now where there's a lot of controversy if it's not U.S.-based, but we're de-risked there as well by having a good manufacturing plan that we also spend a lot of time talking to regulators with to make sure they want to make sure that we have that drug product ready for patients because there's going to be a high amount of demand assuming this drug gets approved. These patients have been, as I said, waiting for a therapy their whole lives.
spk20: Well, that five to seven year comment would be a great problem to have, but thanks for all the comments, Sanjay.
spk02: Sure. Thank you. One moment for questions. Our next question comes from Yasmeen Rahimi with Piper Sandler, you may proceed.
spk07: Hey, team. This is Junggu on for YAS. Congrats on the continued progress, and thanks for taking our questions. For EFSO-FIT, do you still need additional sites to bring enrollment to the finish line, and is there actually an opportunity for additional enrollment beyond the 264 patients? And second, could you provide more color on the percentage or number of patients continuing from EFSO-FIT to the EAP programs?
spk19: Yeah, so 264 is kind of our goal. A lot of that is based on our power calculations. It assumes that you can have a nominal dropout rate. So we're well powered, actually overpowered in many ways compared to other phase three trials. 92% powered here. 264 is our goal. We still view that as a solid number there. It allows for also a buffer that if you had 10% of the patients discontinued for any reason, you'd still be able to hit your power calculation. So we're still set with that number. Sometimes you can see trials that, you know, might be a few patients over, a few patients under. A lot of that has to do with patient screening right there at the end. In our last trial, which was the 36-patient trial, we had eligible patients the day we stopped screening we ended up having a few more patients, one more patient enrolled in that trial because they were screen eligible and we don't want to sort of shut down enrollment if someone is already screened and is eligible. So I would say that we're still going to be right in that ballpark there of 264. With regards to how many are going to move into the EIP, We don't know yet. We are continuing to generate requests. As you can imagine, as more and more patients finish the trial, we may get more patients interested. My early read on it is we're well built here to have a substantial number of patients. If they want the drug, we're ready to provide it. But I don't have an exact number. But we are prepared, you know, to potentially give the AP program, have it there for, you know, a very, very large number of patients. We'll have to wait and see what kind of demand it continues. Early on right now, I'll say, since we put the announcement, we probably had, you know, even more demand since we put the announcement out.
spk07: Thank you so much for the call.
spk02: Thank you. One moment for questions. Our next question comes from Yeo Jin with Laidlaw & Co. You may proceed.
spk05: Good afternoon, and thanks for taking the questions. I came in late, so maybe if you can reiterate a little bit in terms of FSO fit enrollment. I know the press release indicated it's on track. Was there any other colors of that? Then I have another question.
spk19: No, Yale, very much on track. Very much feeling good, you know, about Q2 this year. So very much on track.
spk05: Okay, great. That's good. And you mentioned a few, two preclinical programs, and they look very interesting at this juncture. Was those the programs you intend to partner out at some point, or what will be the path you are thinking about moving forward with those preclinical programs?
spk19: Well, I think it's, I mean, for everything as a biotech, like, you know, kind of where we are, you always have to look at partnering and those sorts of opportunities. There's no shortage of interest. Obviously, we have, you know, a rather novel platform here, and the platform now is generating more and more validated opportunities. So that will be something that we'll look at. We always look at these sort of potential arrangements. At the same time, we also are potentially sitting on assets here that really are producing signals really never seen before. When you talk about being able to induce apoptosis of myofibroblasts, I don't think you'll hear too many companies really seeing those kinds of early signals. So that's where this biology, I think, it's important for everyone to understand. There's hidden potential here. There's hidden potential efficacy here. And I think what ATAR is doing is really looking at these genetic codes and saying, where can they best be applied where there is disease and dysfunction? Unlike, you know, generations of Me Too therapies that have been out there that are not disease-modifying, I think all of our approaches, Epsifidamide and even some of the pipeline approaches we have here are, you know, we have a very, very high bar here. They have to be disease-modifying. They have to really be showing activity here that is two standard deviations better than most, you know, opportunities out there. And I think we want to be careful about giving away these gems too early, but we're open to talk to big players at all times.
spk05: Okay, great. Maybe just making one more question here, which is that you said the trial is on track to complete enrollment second quarter, and just curious, in terms of the current in Japan, given they have smaller size have they already completed? Any colors on that? And thanks.
spk19: Yeah, I can't really get into who's completed or not. I mean, it more or less is competitive enrollment. There aren't hard caps per se, country by country. We have our goals on where we want to get to. So I wouldn't think of it as a separate trial. We've said in the past that A region like Japan, you might expect 25 to 30 patients there. So Japan, like the rest of the world that we're working in right now, very much on track, you know, to meet our projections to get this trial wrapped up here in Q2, at least enrollment.
spk05: Okay, great. Thanks a lot, and congrats on all the properties.
spk16: Thank you, Yael.
spk02: Thank you. And as a reminder, to ask a question, please press star 1 1 on your telephone and wait for your name to be announced. One moment for questions. Our next question comes from Robert LaBoyer with Noble Capital Markets. You may proceed.
spk04: Good afternoon and congratulations on all the progress. I also have a question on the Expanded Access Program and was wondering whether you're going to be compiling endpoints and other data that can be used in the BLA or whether you can present some of the extension study data at medical meetings either before the data is announced or afterwards.
spk19: Yeah, it's a great question, Robert. So, just want to highlight one thing here is this is being conducted outside of our protocol. And that's being done for a few reasons. Number one, we want to hit our BLA timelines and submit, finish the primary trial, if you will, the primary portion of the trial and submit that. This secondary piece of the trial is sitting outside of our protocol. Why are we doing it that way? Well, first of all, health authorities didn't mandate that we do any kind of open label extension. There was no known safety risks observed early in the program, in our last program. and we had long-term safety data already from this trial. So there was really no need to have a more formal open label extension or anything like that. So this trial will be done outside of the boundaries of our protocol. Now, already I would say many academics are really interested in looking at patients 18, 24 months out, perhaps with or without long-term. So I can't say that there would not be an academic consortium of some of our experts that would want to put out this data. I would support that. I think they could look at different things that within the confines of our own protocol we haven't been able to look at, to look at long-term, you know, steroid reduction effects. You know, some of the early promising things we see with regards to Things like weight loss. You know, it was always theoretical. You get off steroids, you can help with weight. You know, we're seeing some rather remarkable signals there. It's early. It's anecdotal. This might be something that we could look at, those investigators could look at in some sort of academic survey. They may want to look at some sort of imaging data two or three years later. But right now, we're focused on getting this drug approved, getting the BLA out. Those trickle of that kind of interesting data could almost be a head start to phase four. That's the way I look at it. We also don't want to spend money on that sort of downstream side of the fence right now. But I think there's going to be some intriguing potential there to collect data, not only for the patients that continue in the trial, but also those that might have to revert back to steroids and see what happens with those patients. But I think it's a great question, Robert. Really nice how you're thinking about that.
spk16: And it's already things that, you know, experts are noodling over potentially to do something outside of the trial.
spk03: Okay, great. Thank you very much.
spk02: Thank you. I would now like to turn the call back over to Sanjay for any closing remarks.
spk19: Great. Well, we thank everybody's interest. Great questions today. I know there's a lot of expectation and everyone's really looking forward to getting this trial wrapped up here. We are too. Appreciate everyone's interest here. Thank you for following us and look forward to talking to you in the future. Thanks again.
spk02: Thank you for your participation. You may now disconnect.
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