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spk04: Greetings and welcome to the Tayshia Gene Therapy's fourth quarter and full year 2022 earnings call. At this time, all participants are in a listen-only mode. A brief question and answer session will follow the formal presentation. If anyone should require operator assistance during the conference, please press star zero on your telephone keypad. As a reminder, this conference is being recorded. It is now my pleasure to introduce your host, Haley Collins, Director and Head of Corporate Communications. Thank you, Ms. Collins. You may begin.
spk10: Thank you. Good afternoon and welcome to Tayshia's fourth quarter and full year 2022 Financial Results and Corporate Update Conference Call. I'm Haley Collins, Tayshia's recently appointed Director and Head of Corporate Communications. I will also be overseeing investor relations activities. I bring corporate communications experience in rare diseases, having previously served in a similar role at Jaguar Gene Therapy. I'm excited to join Tayshia at this pivotal time and look forward to working with the team to help bring potentially life-changing therapies to patients with rare diseases with high unmet medical needs. Earlier today, Tayshia issued a press release announcing financial results for the fourth quarter full year 2022. A copy of this press release website and through our SEC filings. Joining me on today's call are Sean Nolan, Taysha's CEO, Sukumar Nagendran, President and Head of R&D, and Cameron Alam, Chief Financial Officer. We will hold a question and answer session following our prepared remarks. Please note that on today's call, we will be making forward-looking statements, including statements relating to the existing clinical data for Taysha 120 and and the therapeutic and commercial potential of Tayshia 120 and Tayshia 102. These statements may include expected timing and results of clinical trials of our product candidates and other clinical and regulatory plans and the market opportunity for those programs. This call may also contain forward-looking statements relating to Tayshia's growth, forecasted cash runway and future operating results, discovery and development of product candidates, strategic alliances and intellectual property, as well as matters that are not historical facts or information. Various risks may cause TASHA's actual results to differ materially from those stated or implied in such forward-looking statements. These risks include uncertainties related to the timing and results of clinical trials of and regulatory interactions for our product candidates, our dependence upon strategic alliances and other third-party relationships, our ability to obtain patent protection for our discoveries, limitations imposed by patents owned or controlled by third parties, and the requirements of substantial funding to conduct our research and development activities. For a list and description of the risks and uncertainties that we face, please see the reports that we have filed with the Security and Exchange Commission, including our annual report on Form 10-K for the year ended December 31, 2022. This conference call contains time-sensitive information that is accurate only as of the date of this live broadcast, March 28, 2023. Patients undertake no obligation to revise or update any forward-looking statements to reflect events or circumstances after the date of this conference call, except as may be required by applicable security laws. With that, I would now like to turn the call over to our CEO, Sean Nolan.
spk06: Thank you, Haley, and welcome, everyone, to our 2022 fourth quarter and full year financial results and corporate update conference call. Today, I will begin providing a brief corporate outlook for 2023. Then, Sukhu Nagendra, President and Head of R&D of Kaysha, will provide an update on our clinical development programs, followed by a financial update from Cameron Alam, our Chief Financial Officer. I will then provide closing remarks before opening the call-up for questions. The actions taken earlier this year to improve execution and expedite progress with our two lead clinical programs in Rett Syndrome and GAN are having a positive effect. For TASIA-102 and Rett Syndrome, we remain on track to execute across our timelines for both initial available safety data and regulatory submissions this year in our ongoing Phase 1-2 Reveal Adult Study. Suku will provide further details here shortly. Fertatia 120 in GAN, an ultra-rare disease with no currently approved treatments. We recently received constructive feedback from the FDA regarding our follow-up questions to the Type B end of Phase II meeting. We are completing a comprehensive review of the data from the ongoing natural history and interventional trial, including functional, biological, and electrophysiological assessments. The preliminary analysis appears encouraging, and we believe there are some compelling new findings that we intend to share with the FDA to further discuss a potential regulatory path forward. Again, Sukhu will discuss this in further detail shortly. In the year ahead, we remain focused on achieving the anticipated near-term milestones in our Rett Syndrome and GAN programs, and continue to work towards our mission of bringing transformational new treatments to patients with these devastating neurodegenerative diseases. I will now turn the call over to Sukhu to provide a more in-depth discussion of our Rett syndrome and GAN programs. Sukhu?
spk08: Thank you, Sean, and good afternoon, everyone. First, I will start with an update on TASHA-102, our gene therapy program for the treatment of Rett syndrome. As a reminder, TASHA-102 utilizes an innovative miRNA-responsive autoregulatory element, or miRARE platform, designed to regulate the cellular expression of MECP2 for the treatment of Rett syndrome. TASHA-102 has received orphan drug and rare pediatric disease designation from the FDA and has been granted orphan drug designation from the European Commission. In our review phase 1-2 trial in adult patients with Rett syndrome, we recently initiated screening for the first potential patient, and we anticipate dosing the first patient in the first half of the year. We remain on track to report initial available clinical data, primarily on safety for TASHA-102 in the first half of 2023, and plan to provide quarterly updates on available clinical data thereafter. Importantly, we recently submitted a protocol amendment to allow patients as young as 15 years old to be included in the study, which we believe will further expedite enrollment. In the second half of the year, we intend to continue dosing patients with Rett syndrome in our reveal trial. For our study in pediatric patients with Rett syndrome, we plan to submit a CTA to the UK MHRA for TASHA 102 in mid-2023. We also have an IND application submission to the US FDA planned in the second half of the year. Now let's turn to TASHA 120, for the treatment of GAN, which, to reiterate, is an ultra-rare neurodegenerative indication with no approved treatments or established regulatory pathway. TASHA 120 has received orphan drug and rare pediatric disease designation from the FDA and has been granted orphan drug designation from the European Commission. In regards to manufacturing, we recently submitted a CMC Module 3 amendment submission to the detailing our commercial process, product manufacturing, and drug comparability analysis. As Shawn mentioned, we also received feedback from the FDA in response to our follow-up questions to formal Type B end-of-Phase II meeting minutes. The FDA clarified MFM32, the primary efficacy scale discussed at the FDA Type B end-of-Phase II meeting, as a relevant primary endpoint only in the setting of a randomized double-blind control trial. while also acknowledging CHR's challenge in executing and enrolling such a study design due to the ulterior nature of GAN. As such, the FDA is open to regulatory flexibility in a controlled trial setting and is willing to consider alternative study designs utilizing objective measurements to demonstrate a relatively large treatment effect that is self-evident and clinically meaningful. We are completing a comprehensive review of data from the ongoing natural history and interventional trial, including functional, biological, and electrophysiological assessments, which will inform our plans for future interactions with the FDA. The ongoing analysis includes functional assessments of MFM32 and ataxia, as progressive gait and lymph ataxia is a common clinical manifestation observed in patients with GANF, that often leads to locus ambulation by the second decade. Additionally, we continue to analyze functional and structural aspects of the retina and optic nerve, given that GAN patients experience deterioration of visual acuity and optic nerve degeneration over time. We are also conducting several objective biological and electrophysiological assessments, including sensory nerve action potential, nerve and skin biopsies, ganglion cell and retinal nerve fiber layer thickness, brain and spine MRI images and muscle responses to nerve activation to determine whether there is a relatively large treatment effect that is self-evident and clinically meaningful. We intend to continue a collaborative dialogue with the FDA regarding the potential registrational path to bring TASIA-120 to patients with GAN who, to reiterate, have no approved treatment or established regulatory pathways. We plan to submit a formal meeting request to the agency in the second quarter of 2023 to further discuss the potential regulatory pathway forward for this ultra rare disease. I will now turn the call over to Cameron to discuss financial.
spk01: Cameron. Thank you, Sukhu. Research and development expenses were $13.9 million for the three months ended December 31st, 2022. compared to $37.9 million for the three months ended December 31, 2021. Research and development expenses were $91.2 million for the full year ended December 31, 2022, compared to $131.9 million for the full year ended December 31, 2021. The $40.7 million decrease was primarily attributable to a decrease of $20.3 million in research and development manufacturing and other raw material purchases, and a $9 million decrease in license fees. The decrease in research and development expenses for the year ended December 31, 2022 was also attributable to a $12 million decrease in third-party research and development fees mainly related to non-clinical and toxicology studies and a $4.7 million decrease in compensation expense as a result of lower headcount. Overall, lower research and development expenses for the year ended December 31, 2022 were partially offset by higher clinical trial expenses of $2.4 million and higher severance expense of $2.9 million in 2022. General and administrative expenses were $7.3 million for the three months ended December 31, 2022, compared to $11.8 million for the three months ended December 31, 2021. General and administrative expenses were $37.4 million for the year ended December 31, 2022, compared to $41.3 million for the year ended December 31st, 2021. The decrease of approximately $3.9 million was primarily attributable to $5 million of lower consultant professional fees and reduced compensation expenses driven by lower headcount in 2022. Lower general and administrative expenses were partially offset by $1.1 million of severance expense. Net loss for the three months ended December 31st, 2022 was $55.7 million, or $0.99 per share, as compared to a net loss of $50.4 million, or $1.32 per share, for the three months ended December 31, 2021. In November 2022, we recorded a $36.4 million non-cash, non-recurring impairment charge related to the North Carolina manufacturing facility. Currently, we are in the process of actively looking for buyers for the North Carolina manufacturing facility. The net loss for the three months ended December 31st, 2022 was partially offset by revenue of $2.5 million recognized related to the Estella's transactions. Net loss for the full year ended December 31st, 2022 was $166 million or $3.78 per share as compared to a net loss of $174.5 million or $4.64 per share for the full year ended December 31st, 2021. As of December 31st, 2022, Tayshia had $87.9 million in cash and cash equivalents. The company continues to expect that its current cash resources will support plant operating expenses and capital requirements into the first quarter of 2024. I will now turn the call back over to Sean for his closing remarks. Sean? Thank you, Cameron.
spk06: I am pleased with the progress we have made with our two lead programs during the first few months of 2023. including initiating screening of the first potential patient in the adult RET study and submitting a protocol amendment that should further expedite patient enrollment. For GAN, we are encouraged by the constructive feedback received from the FDA and the preliminary assessment of the comprehensive data analysis to support a formal meeting request in the second quarter to further discuss the potential regulatory path forward. Our focus throughout this year will be on the execution and delivering across our planned milestones for our Rett Syndrome and GAN programs. We look forward to providing further updates throughout 2023. With that, I will now ask the operator to begin our Q&A session. Operator?
spk04: Thank you. We will now be conducting a question and answer session. If you would like to ask a question, please press star 1 on your telephone keypad. A confirmation tone will indicate that your line is in the question queue. You may press star two if you would like to remove your question from the queue. For participants using speaker equipment, it may be necessary to pick up your handset before pressing the star keys. So that we may address questions from as many participants as possible, we ask that you limit yourself to one question.
spk05: One moment please while we poll for questions.
spk04: Thank you. And our first question is from Yanan Zhu with Wells Fargo Securities. Please proceed with your question.
spk12: Hi, thanks for taking our question. This is Quan for Yanan. So my question is on the RAS syndrome program. So given that a KDS state bill is now approved for RAS syndrome patients aged 5 to 20, how would that affect your clinical study conduct? and enrollment, particularly for the UK study? And do you need to include debut into your trial design? Thank you.
spk06: Thank you very much for the question. We've certainly given you a great deal of thought in our planning, and I'd like to ask Sukhu to provide some perspective on that. Sukhu?
spk08: Yeah, thanks, Sean. So this is an important question that you asked because I think getting a product approved for a terrible disease like RET I think is very important for the whole disease state and the patient population. What I would also identify is that by having atrophylentide approved, it also increases the awareness of the disease state amongst the physician and patient communities, which could potentially increase the number of accessible prevalent patients and also result in, I think, some serious discussion at the state and federal level when it comes to newborn screening, which could further increase the number of patients available from an incident standpoint. So what I'm saying is, collectively, this could be a real plus for the whole disease community, but also for sponsors like us who are doing interventional trials. Now, to address your question about whether, once you have an approved product like roflentide, where the mechanism of action obviously is very different from our product, PASHA 102, which addresses the root cause of the disease, we would ideally like to treat patients that are treatment-naive, such that we can optimize and show the FDA and other regulatory authorities and the patient communities and the healthcare providers who treat this disease, that our product will have significant impact on clinical progression of disease. Now, that is the hope. The caveat is, as you asked, if there is an approved product, there's always possibility that we may have to have an arm which has a combination product evaluation as well. And that is something we would be happy to discuss further with the regulators and take it on a case-by-case basis. but my team is all considering this in the protocol designs for the future. Sean, anything else you'd like to add?
spk06: The only other thing I would add is that given the half-life of the product and some of the adverse event profile, there's opportunity for patients that take the drug may not be able to stay on it, so you've got a pool of of call it non-treated patients that you could potentially access. And, you know, we experienced with Zolgensma studies that even with the addition of Spinraza, you know, patients would be willing to either wait or wash out, you know, from taking those other medicines. So, you know, we'll certainly keep an active eye on the impact that trofenetide is having. We've given it a great deal of consideration into our scenario planning for clinical trial designs. and we remain confident that we'll be able to enroll patients in the U.S. and in other geographies, including Canada and the U.K. So thank you.
spk08: And, Sean, if I can add one more point. Also, if you look at the troponotide label, the discontinuation rates in the trials are very high due to GI symptoms. And I've forgotten the exact numbers, but you may have looked it up. I think it was anywhere from, I don't know, 70% to even 90% in some of the trials. So that may have significant impacts. on more patients being available, even though this product is available for patients with Rett syndrome.
spk03: Got it. Very clear. Thank you.
spk04: Thank you. Our next question is from Whitney Egem with Panaccord. Please proceed with your question.
spk09: Thanks for taking the question. Excuse me. So for GAN, you kind of listed out a lot of additional endpoints functionally, biomarker, et cetera, that you're looking at. And I'm just curious, are those things that were collected all the way along haven't been analyzed or focused on, or are those things that you are calling patients back in now to look at and will be comparing to natural history? And then part two of my one question is, is the goal there to kind of continue the dialogue with the FDA and use that data to potentially support filing or use that data to design another study?
spk06: So I'll take a first and feel free to opine. To answer the first part of your question, this is data that's existing in the database. So these are patients, remember there's been 12 patients that have been treated. We've got the natural history before they were treated and then all the study visits over the course of time, in some cases many, many years. And there are a multitude of assessments that were done. And what Suku's team has been doing is comprehensively and systematically going through all of that data to fully assess it and to see if there are additional data points that can or metrics that can augment and further demonstrate the clinically meaningful effect and the objective measurement effect of the treatment on these patients suffering from GAN. And to answer your second question, and I'll turn it over to Sukhu, the idea would be that we would submit a formal meeting request this quarter, or the second quarter, to dialogue with the FDA about this data. And we will put forward, after we fully have analyzed this, our view on what we think is the appropriate pathway forward. And that could very well be trying to seek approval with the existing data. So we haven't made a final determination yet, but we're encouraged by the data that we've gathered thus far and how it's starting to line up. Sukhu, what would you add or clarify to what I said?
spk08: Yes, John, thank you. So as you highlighted, it's an ultra-rare disease with no available treatment, very small patient population. And the patients were treated, in general, they were more than six years of age. So What we've observed is a slowing of disease as we've all previously talked about when it comes to MFN32. And as Sean pointed out, there were many efficacy endpoints in the protocol that the NIH designed. And it's that data set that we're looking at when it comes to functional biological and electrophysiological endpoints. But we're also doing some modeling work. And we think that comprehensive data analysis should give us enough information to go back to the FDA and request a meeting in the second quarter of this year for further discussion. on what is the best path forward in a data set that we think could really make a difference in these patients' lives, especially given it's an ulcerative disease with no other treatments available. So cautiously optimistic as we collect all the data, work with the NIH, and then go to the FDA, hopefully, and hopefully move this program forward. Thanks for the question.
spk15: Thank you.
spk04: Thank you. Our next question is from Jack Allen with Baird. Please proceed with your question.
spk13: Great. Thank you so much for taking the question. I'm going to stick with Gann here, and I was wondering if you could provide an update as to where things sit as it relates to the ASTELLAS option for Gann. How are you thinking about the XUS opportunity, and then what's the turnaround time for the meeting request, and who have you been meeting with specifically at the FDA? I'd love to get any insights there. It does sound like Peter Marks is... quite optimistic about accelerated approvals based on a recent medical meeting that he's speaking at.
spk06: I'll take a stab at this, and Sukhu, please jump in. I think to answer your last question first, in terms of who from the FDA is going to be there, it's premature for us to say. We could not definitively make a statement relative to that. You know, we'll do everything we can to make sure that the package that we put together is as compelling as possible based on both the natural history and the interventional data. And your point about Peter Marks is a good one. I mean, obviously, we're headquartered in Dallas. The MDA conference was in Dallas. There was a lot of buzz about, you know, some of the comments, you know, that he's made about how to deal with ultra-orphan diseases. And obviously, we feel like this one fits the bill perfectly. and that flexibility may lend itself well to the program, but we also understand we have to make that case. There's new leadership there now with Celia Witten taking over, and we're all watching to see what will happen, and hopefully we can get some additional flexibility, as Peter's kind of outlined in many of his public remarks over the course of time. Suku, do you mind taking on the other two questions from Jack? And Jack, you might want to just, if you're on the line, just... Jack, can you repeat the question?
spk08: Because that's a lot of questions that you asked.
spk13: Thanks so much, and I apologize. I know we're still limited to one, but the other questions I had were surrounding the ASTELLIS option. Where does that fit as it relates to GAN? And then maybe if you could just touch on the XUS path forward for GAN and how you're thinking about potentially filing in Europe, I would think. Thanks so much.
spk08: John, do you want me to take that, or would you like to take that?
spk15: I'll take it.
spk06: On the ex-U.S. piece, Jack, we're focused right now on the U.S., so we have not had additional interactions with anyone overseas since the initial discussions, and we want to focus all of our efforts, energy, and resources right now on the U.S., As it relates to the ASTELLAS option, we really haven't provided too much detail about that, but what I would say is that we're still in a window where ASTELLAS has the option and the time to evaluate whether or not they want to opt in. I think it stands to reason that they would likely make a decision after we have this upcoming FDA meeting that we're planning to have. So we're planning to submit the meeting request in the second quarter. You know, hopefully, you know, mid-ish year we have that actual meeting. And, again, I can't speak for Estella's, but I would say around that time is when they would have the information to make a more fulsome decision on whether or not they want to opt in on the program. So it's still a very active opt-in.
spk08: Great. Thank you so much. And Jack, you had one more question on the regulatory turnaround times. As you know, it depends on the type of meeting we request, right, whether it's a type A, B, C, or D. And I think that's the decision we'll have to make, and that will impact the turnaround time. Some, as you know, could be 30 days or anywhere from 30 to 75 days, depending on the type of meeting requested from the FDA.
spk15: Great. Thank you so much. I appreciate you taking the question. Yeah, thanks.
spk05: Thank you. Our next question is from Gil Bloom with Needham & Company.
spk04: Please proceed with your question.
spk03: Hi, this is Rohit on for Gil. Thanks for taking our questions. Can you just talk about the ex-US market for Tayshia 102, and would you be open to partnering the program? Thank you.
spk06: Well, I would say that the opportunity is significant, right? I think when we talk about the fact there's approximately, you know, 20, 25,000 patients in the U.S. and EU combined. So the opportunity over there is significant. There's good infrastructure over there in terms of clinical study sites, treatment centers. The patients are well identified. So we're encouraged by the opportunity over there. It's one of the reasons You know, we focused our clinical efforts in submitting that CTA mid-year to the UK. And just trying to think of the second part of the question. Well, the partnering aspect. You know, I would say that that's something that we would potentially consider. You know, I think you always have to look at all options, your current capital situation, and make a determination on resource-wise, you know, and otherwise, is it better to partner that or better to keep it to yourself? Right now, you know, the plans are for us, and we're, you know, we have the funding this year to execute the trial as we've outlined it, the planned trial, I should say. So, you know, it's an option that we have, you know, should we decide to pursue that.
spk15: Thank you.
spk05: Thank you.
spk04: Our next question is from Sylvan Turkin with JMP Securities. Please proceed with your question.
spk11: Yeah, thank you. Thanks for taking my question. Just to come back to Gann, I think the last time the FDA tested you, a modest, or I don't remember exactly the wording, effect on the primary endpoint, and that's why they wanted maybe a controlled trial. But, I mean, obviously, those are just words, and so how do we know now that they're open to a strong efficacy measure that you have met the bar with these additional endpoints? Could you stack them together to to show a strong outcome here with the data at hand, or how do you imagine this will play out? Thank you.
spk06: Well, I would say, first of all, that what the FDA said, you know, at the meeting minutes, the initial meeting minutes, was it was very focused on MFM32 because that's where the majority of the discussion happened. That was the primary endpoint. That was the focus of the company. And so all of their comments were generally around that particular endpoint where they basically said, because they believe it's subjective, you need to have a double-blind randomized control trial. So when we went back to them and we asked the subsequent questions, you know, I would say the new news is that they restated their view on MFM32, but then they said that they're willing to – evaluate alternative trial designs that are controlled, and a control can be natural history, as you know, that have endpoints that are clinically meaningful and essentially objective. And so, you know, that is a, I would call it a door that was, a new door open there, you know, opportunity-wise that makes a lot of sense to us. So that it's incumbent upon us to determine What do we think is the data that would justify and address those two aspects? What is clinically meaningful and what is objective? And so with that, I'll turn it over to Sukhu to just give you a flavor of the comprehensiveness and the totality of data that we're looking at and why we're encouraged.
spk08: Sukhu? Thanks, Sean. Thanks, Sean. So your question is an important one. So let me start by saying again, I think somebody earlier asked about Peter Marks. and the FDA's position when it comes to SIBA and ultra-rare disease where there is no treatment available. So I think those remarks at the MD were very important, and hopefully will have some strategic impact on any sponsor that's dealing with an ultra-rare disease, GAN or otherwise. Now, as Sean pointed out, as we continue our in-depth, detailed analysis, the big question here is it's not just MFM32. There are multiple other efficacy endpoints, and the question is will our intrathecal gene therapy actually show broad clinical impact some or most of which could be collectively considered clinically significant, whether it's really in a very large way or even in a moderate fashion in an ulcerative disease, that allows us to make a case on behalf of patients and as a sponsor for potential accelerated approval or an approval that is acceptable such that we can make this medicine available to the patient community. And as Sean pointed out, it's the collective breadth of data, I think, that could eventually make the case for this therapy further review at the FDA and for us to make the case for the submission of a meeting with the FDA in the second quarter. So I hope you understand what I'm trying to explain. It's not just MFM32. There are multiple other endpoints that I think that we've observed with the help of the NIH and other experts that are presenting this ultra rare disease called GAN that our product may have clinical impact. And I think that collective case is what may give strength to the potential review and approval of the product. Stay tuned, and I'm keeping my fingers crossed. That's what we can do for this patient community. Thank you.
spk11: Great. Thank you very much.
spk04: Thank you. Our next question is from Yun Zong with BTIG. Please proceed with your question.
spk00: Hi. Thank you very much for taking the question. So my question is on the Rett syndrome program. And so the change in the patient age to treat patients as young as 15 years old. In addition to helping patient enrollment, maybe, do you have any other goals that you would like to achieve in terms of treatment outcome analysis and the initial data, would that be safety only, or will you be able to provide any additional information?
spk15: Suku, would you like to take this question, please?
spk08: Yes, Sean, I will. I will, absolutely. So this is another very important question. So there was a very important clinical reason that my team and obviously as a sponsor, we decided to submit this updated version for amendment to Health Canada because by dropping the age to 15, and we do have preclinical data and other data that supports us doing this, it enables us to treat a younger group of patients, which we think in this neurodevelopmental disorder may allow us to have greater impact over time. So the question on the table is, A, by dropping the age and having certain endpoints in our efficacy measures and also evaluating safety, will we not only have access, hopefully, to more patients, given that it's now a larger patient pool, but could we also show greater clinical impact that goes beyond safety in a much shorter timeframe? So the question is, when you treat adult patients who have more mature disease or more progressive disease, Over the age of 18, will it take much longer for any product to have impact versus once you drop the age, could you have much greater clinical efficacy impact? So our hope is even though the initial data set that we'll accumulate over time will be safety data, the big question on the table is will we also show greater clinical efficacy impact? And my hope as a clinician is that is what we will also see which will allow us to show the strength of our gene therapy intervention for Rett syndrome, which as you know is a terrible disease, by treating the root cause of the disease. So that is our hope, and I hope that answers your question.
spk15: Okay, great. Thank you.
spk04: As a reminder, if you would like to ask a question, it is star 1 on your telephone keypad. Our next question is from David Huang with SMBC. Please proceed with your question.
spk02: Thanks for the update and taking my question. I had one question just with Dan. Are you formally considering an accelerated approval pathway there? Is that something, given the endpoints and the data that you are now reviewing, do you think that path would be available to pursue? And if so, why? Or if not, why not?
spk15: Suku, would you please take that question?
spk08: Yes, Sean. So I can only give you my opinion based on the data that I've seen and our overall regulatory discussions, right? So obviously what we can do is look at our data set, see if the broad impact of our product, the gene therapy, is truly clinically meaningful, and then ask for the meeting with the FDA. And absolutely, if we believe our product is truly making a difference in this ultra-rare disease, while there's no treatment option available, I would absolutely love to ask for the FDA to consider our product for accelerated approval such that we can make the medicine available to patients. But the caveat is this, right? So this I have to be very clear on, is we have to have a collective data set that truly enables us to make a convincing case with the FDA that this gene therapy qualifies for accelerated approval. So that is what we're hoping for, and that is the plan when we submit a request for an FDA review once we do complete our full analysis, okay? So I hope that helps, and I don't know if you wish to add anything more to what I said.
spk06: Yeah, look, the only thing I would add to that is when you think about some of the remarks from what Dr. Peter Marks has said multiple times publicly, if you think about the disease state of GAN and a product that could merit an accelerated report, It certainly fits the bill. We don't want to get ahead of ourselves at all, and we don't want to prejudge final data. I would just say that based on the preliminary assessment, we're encouraged about the package that we can put together, and we will certainly make the most aggressive presentation we can based on data for patients and to get down this product as soon as possible, given that there's nothing available at this particular point in time. So if we're convinced the data is compelling and meets the bar, we would push hard for something like that.
spk15: Thanks for the question.
spk05: Thank you.
spk04: Our next question is from June Lee with Tourist Securities. Please proceed with your question.
spk14: Hi. Thanks for the update. You know, when you say in your press release that the FDA is open to regulatory flexibility in a, quote, unquote, controlled trial setting, you know, does that necessarily refer to placebo-controlled trial setting, or can that include some other controlled setting? And if it's the latter, what could it be?
spk06: Well, I'll give you my answer to that, and Sukhu, please opine. But when you look at FDA guidance and you look at accelerated approval, there's generally a consideration of a controlled trial. And that controlled trial is one of the designations would be, as an example, natural history. So I think there's a distinction between a controlled trial and a double-blind placebo controlled trial. And the language written in the responses that we got from the FDA are clear on those. When they were talking about MFM32, they were talking about a double-blind placebo-controlled trial. When they were talking about alternate trial designs that met the bar of clinically meaningful and objective endpoints, they talked about a controlled trial. So I think they're speaking to the letter of the guidance, but Suku, I would ask you for further clarification on that.
spk08: Yeah, thanks, Sean. So even though it's an ultra-rare disease, doing a placebo-controlled trial I don't think will be practical and could take forever. So if there are alternative study designs necessary or have to be discussed, the simplest would be to dose a few more patients where you already have pretreatment natural history for comparison. So with that caveat, our hope, is that the collective data set that we submit to the FDA will qualify for a serious review and potential appropriate approval if the FDA thinks that's the best path forward for the patient community. So I would leave it at that. And, Sean, is there any other question you wish me to address? So hopefully we addressed the question that was asked.
spk15: No, I think you did. Okay, thank you. Thanks.
spk04: There are no further questions at this time. I would like to turn the floor back over to Sean Nolan for closing comments.
spk06: Thank everyone for their time and interest in what we're trying to accomplish here at Taysha. I'm very pleased at the progress we've made in the short time period of 2023. We've got a long way to go. We're working hard and we look forward to sharing future updates with you in the coming months. Thank you all and have a good night.
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