Hello, and welcome to the enGene Therapeutics webinar to discuss new data from its LEGEND trial and provide a regulatory update. At this time, all attendees are in a listen-only mode, and a question-and-answer session will follow the formal presentation. As a reminder, this call is being recorded, and a replay will be made available on the enGene website following the conclusion of the event. I'd now like to turn the call over to Lauren Hopfer, Executive Director of Investor Relations at enGene Therapeutics. Please go ahead, Lauren.
Thank you. Good afternoon, everyone, and thank you for joining. With me from enGene on today's call are Ron Cooper, Chief Executive Officer, Amy Pott, Chief Global Commercialization Officer, and we are pleased to welcome the LEGEND trial's primary investigator, Dr. Ashish Kamat, Endowed Professor of Urologic Oncology and Cancer Research at MD Anderson Cancer Center. Before we begin, I would like to remind you that during our prepared remarks and Q and A session to follow, we may make forward-looking statements for purposes of U.S. and Canadian securities laws, and we draw your attention to the disclaimer on the slides that accompany this presentation. At this time, I will turn the call over to Ron Cooper, enGene's President and CEO. Ron?
All right, great. Well, thanks, Lauren, and thank you all for joining us this afternoon on short notice. Here on slide three is a quick overview of this afternoon's agenda. After the market closed today, we're pleased to report updated data from LEGEND's pivotal cohort one, studying our lead- Its tolerability profile continues to trend as potentially best in class, with the lowest rates of discontinuations or interruptions due to treatment-related adverse events, and our primary endpoint, complete response at any time, is 56%, is squarely within the range of approved therapies. Durability, which was very immature during our last update, is also trending towards the range of approved products.
Importantly, during our pre-BLA meeting with the FDA this quarter, we reached alignment on key components of our statistical analysis plan, including the efficacy-ev aluable patient population for cohort one, as well as our plan to initiate a BLA submission for detalimogene before year-end, positioning detalimogene for potential approval in 2027. In tandem with our data and regulatory update, we also issued news that we've appointed a new Chief Medical Officer, Dr. Erik Goluboff, and Chief Legal and Administrative Officer, Jason Duncan, both of whom will play key roles as we enter our next phase of company growth and prepare for potential commercialization. Welcome, Jason, and welcome, Erik. This is an exciting time for our company and for the NMIBC space. With the advent of new treatment options, the sequencing of bladder-sparing therapies will drive market growth by expanding the treatable population.
We're already seeing this play out with earlier approved products, and seeing continued growth, even with the advent of new product launches. When sequencing therapies, urologists are looking for different approaches, and detalimogene's unique non-viral MOA could provide them a different treatment option. As we highlighted in our KOL webinar last month, treatment selection is nuanced through the patient journey with high-risk NMIBC, and it is never a one-size-fits-all model. As the prevalent population continues to grow, this will not be a winner-take-all market. We continue to believe that as a next-gen therapy, detalimogene, with its unique non-viral attributes and MOA, low-cost scalable manufacturing, favorable tolerability, and in-class efficacy, would be uniquely positioned to unlock sequencing more broadly across community urology segments.
75% of NMIBC patients are being treated in community practice settings, where urologists must weigh a variety of factors when selecting treatment, from logistical and infrastructure challenges to financial considerations. If approved, detalimogene would be the first and only non-viral plasmid gene therapy approved for NMIBC and truly suitable for all practice settings. The potential to stock and store detalimogene in a standard freezer or fridge, the lack of BSL-2 handling or post-treatment precautions, and its streamlined preparation and administration help remove logistical and infrastructure challenges across the board. With that, I'm pleased to turn the call over to Dr. Ashish Kamat to review the latest data from LEGEND Cohort 1.
Dr. Kamat is Endowed Professor of Urologic Oncology and Cancer Research at the University of Texas MD Anderson Cancer Center, President of the International Bladder Cancer Group, Co-President of the International Bladder Cancer Network, and primary investigator for our LEGEND trial. Dr. Kamat?
Thank you for that introduction, Ron, and thanks everyone for joining us this afternoon. I will be reviewing the latest interim results from the pivotal cohort of LEGEND, evaluating detalimogene voraplasmid in patients with high-risk BCG-unresponsive non-muscle invasive bladder cancer with carcinoma in situ, both with and without concomitant papillary disease. The treatment landscape for BCG-unresponsive non-muscle invasive bladder cancer has substantially changed over the past several years. We now have several bladder-preserving options, which is welcome progress, of course. But as someone who treats patients with bladder cancer every day, I can tell you we still have a considerable unmet need. Radical cystectomy remains an important curative option, yet many patients are unwilling or unable to undergo it. For those patients, our challenge is to find treatments that provide meaningful and durable disease control while minimizing toxicity and disruption to their lives.
And this is where practical considerations become very important. How often does the patient need treatment? How well can they tolerate it? Can their urologist administer it in their own practice, or will they need to refer the patient to a specialized center? These are conversations that we have with our patients every day, and they provide an important clinical context for evaluating the LEGEND data. As a reminder, the LEGEND trial is an ongoing phase II study. The pivotal cohort enrolled 125 patients with BCG-unresponsive NMIBC with CIS, with or without high-grade Ta or T1 disease, who are ineligible for or elected not to undergo cystectomy. Detalimogene is administered intravesically by a catheter with a 60-minute dwell time. The regimen operates on 12-week treatment cycles, with dosing on week one, two, five, six of each cycle.
A reinduction cycle is permitted at week 12 for patients with persistent or recurrent disease, as specified in the protocol. Patients in a CR at the 48-week disease assessment, or 12-month landmark assessment, then move into a maintenance phase, during which they receive detalimogene on week one and two of each 12-week cycle for up to 36 months. The primary endpoint is complete response, or CR, at any time. Key secondary endpoints include duration of response, the proportion of responders maintaining response for at least 12 months, landmark complete response rates at three, six, nine, and 12 months, safety, and progression-free outcomes. The broader program also includes cohorts that may inform use across additional high-risk NMIBC settings. Something I believe is important to this dataset is that the pivotal cohort population is highly reflective of what we are now seeing in the BCG-unresponsive setting.
40% had concomitant papillary disease, and notably, 26% of patients had received treatment beyond BCG, including intravesical chemotherapy, other viral gene therapies, and systemic immunotherapy. Overall, this patient population is more resistant than some of the earlier NMIBC pivotal studies. Importantly, more than 30% of patients declined cystectomy rather than being medically ineligible, and this highlights the strong patient preference for bladder preservation and the need for additional bladder-sparing alternatives. These are the kinds of patients we increasingly see in our practice. One important development since our previous update is the clarification of the efficacy-evaluable population following discussions with the FDA. Of the 125 enrolled patients, 120 are included in the current efficacy analyses, with five excluded for pre-specified reasons under the agreed analysis approach. The analysis population and statistical approach have been discussed with the FDA.
The exclusions are few and based on one patient withdrawing from study before assessment, two patients who did not receive adequate BCG dosing, and two patients who do not have CIS within one year of receiving adequate BCG. All the data presented today will reflect the FDA-aligned efficacy-evaluable population. Now, turning to efficacy, the primary endpoint is anytime CR rate, which is 56%. Relative to the prior update, which I presented at the AUA in May of this year, we see a 2% improvement, which puts detalimogene squarely in the range observed across approved therapies in this setting. Cross-trial comparisons have limitations, so the relevant point here is not that one bar is higher than the other, but that detalimogene has demonstrated meaningful activity in a challenging, previously treated population. On the right, you can see, of patients who achieved a CR, 91% occurred at first disease assessment.
100% of responders were free from bladder cancer that progressed to T2 or greater, and 96% of responders remain cystectomy-free. Across the entire efficacy-evaluable population, 97% of patients were free from progression to T2 or greater disease, and 86% remain free from cystectomy. This data clearly shows detalimogene may offer a reasonable chance of complete response, which can be determined quickly and with low risk of progression to our patients. These are outcomes that matter when discussing bladder preservation with patients. Perhaps the most important question after achieving a CR is how long that response lasts. An initial response is, of course, encouraging, but sustained disease control is what allows us to preserve the bladder safely over time. Here, we can see the updated duration of response data.
As more responders have reached later disease assessments, the proportion maintaining response for at least 12 months has increased from a Kaplan-Meier estimate of 25% to 43%. The broader durability picture also includes median duration of response, which is currently at 10.1 months. This too has improved since the April 2026 analyses and will continue to evolve. Now, we do recognize that Kaplan-Meier calculations can sometimes overestimate the final effect. There are nine additional patients eligible for their durability assessments, and if they all were in complete response after the 12 months, the duration of response would be 40%. To date, 100% of patients who have entered the maintenance phase of the study and have been assessed for durability have recorded durable responses, which is highly encouraging.
Now, here on slide 14, I also want to highlight the improvement in landmark complete response rates at 12 months, which has moved from 13% to 24%, with just one patient pending their week 48 assessment. This secondary endpoint is now also in the range of approved products, and this is the number that patients always ask about, even though it is not included in product labels. Moving on to safety, detalimogene's tolerability profile has remained favorable even with an increased number of exposures. Treatment-related adverse events were generally mild, localized, and consistent with catheterization or intravesical treatment. Among patients experiencing a TRAE, the great majority, 93%, were grade one or two, and the median time to resolution was approximately six days. The most commonly reported adverse events were fatigue, dysuria, pollakiuria, bladder spasm, micturition urgency, and urinary tract infection. Grade three TRAEs were uncommon, just 4%.
Serious TRAEs were also infrequent, just 2%. There were no grade four or higher events reported. What is especially relevant for real-world use are the low rates of interruptions and discontinuations due to TRAEs, just 4% and 1%, respectively. This data indicates that patients are able to tolerate treatment well and complete the cycles as scheduled. On slide 16, this provides some context for interpreting these results alongside the expanding range of therapies available for BCG-unresponsive NMIBC. Direct comparisons across these studies are difficult because of differences in trial populations, response assessments, follow-up, and adverse event reporting. But what we can clearly see here is that detalimogene has demonstrated clinically meaningful activity and an encouraging tolerability profile. The practical implications of these findings will become clearer, of course, as the data mature. To summarize, detalimogene has shown encouraging clinical activity in the BCG-unresponsive NMIBC population.
The updated durability findings provide important additional information about the potential for sustained disease control with an anytime complete response rate of 56% in a heavily pretreated BCG-unresponsive NMIBC population. This is clinically relevant, and the durability is maturing favorably and will continue to evolve. Furthermore, all responders remain free of muscle-invasive or metastatic disease, and the vast majority of responders remain free of cystectomy. The safety data continues to show favorable tolerability, with relatively few treatment-related interruptions or discontinuations. Events have generally been mild, brief, and localized. The non-viral platform and intravesical administration may also offer practical advantages, particularly for patients treated outside major academic centers. Taken together, I believe these findings support detalimogene as a potentially valuable addition to the bladder-preserving treatment options, of course, subject to regulatory approval. With that, I will turn over the presentation to Amy Pott, enGene's Chief Global Commercialization Officer. Amy?
I would like to discuss how we are thinking about and planning for a potential approval for detalimogene in 2027. Guiding principles for shaping our early go-to-market strategy are grounded in where care happens and how treatment decisions are made. Research continues to confirm that 75% of high-risk BCG-unresponsive NMIBC patients are being seen in community practices. The BCG-unresponsive NMIBC is highly recurrent, and approximately 90% of patients would try anything to avoid radical cystectomy. While it is a serious cancer, it is managed more like a chronic disease with different treatment considerations. This goal, as well as additional treatment goals and lifestyle considerations, are critical decision points between patients and their providers, and this discussion ultimately serves as a guide to treatment selection. However, fewer options are made available to patients being treated in community settings, and we plan to change this.
With bladder preservation a primary goal for the majority of this patient population, they will sequence through multiple lines of bladder-sparing therapies, creating a market in which multiple products can win. With these principles in mind, contextualized by our latest interim data, we are excited by the potential of detalimogene to redefine high-risk NMIBC care in community practice settings and beyond. We believe detalimogene may deliver meaningful value across four key aspects of care. If approved as the first and only non-viral plasmid gene therapy for high-risk BCG-unresponsive NMIBC, detalimogene would need to provide clinical value. With in-class efficacy and differentiated, potentially best-in-class tolerability profile, detalimogene could be suitable for use for the vast majority of this patient population. If a therapy is going to redefine care in high-volume, community-based settings, it not only needs clinical value, but must offer value to patients and practices.
Detalimogene could offer differentiated practice value. It is designed to meet real-world community challenges, no new infrastructure needed, an office-ready workflow, and lower post-treatment burden. Beyond its clinical value, we believe detalimogene could offer patients meaningful value beyond clinical relevancy. With its potential to fit seamlessly into community workflows, it may reduce patient burden following treatment and allow for continuity of care that is close to their home and their long-term providers of care. Of course, there is economic value for the practice. Being the first and only non-viral gene therapy in development for NMIBC, detalimogene would be unique, with no special storage requirements, no special equipment needed, and no need for procedure rooms, all of which ease the financial and capacity pressures on practices, allowing them to have a favorable net cost recovery. On slide 21, here we outline what our market research has uncovered around care settings.
We know that 75% of BCG-unresponsive NMIBC patients are being treated in private community practices. Drilling down deeper, about 30% of these practices struggle to adopt viral and/or novel therapies due to their logistical or financial burden placed on the practice, sometimes a combination of the two, while an additional 10% or so of practices are eager to adopt new therapies that are both effective and offer a positive net cost recovery. If approved, we believe that detalimogene is poised for broad adoption in these segments, given its potential to offer clinical, practice, patient, and economic value. That said, we believe this value will translate beyond these segments and that detalimogene will see use across all care settings, as the desire to avoid radical cystectomy and meet patients' treatment goals will drive adoption more broadly.
The concept of shared decision-making is important, and we know that tolerability, risk of progression, and lifestyle factors are key considerations in treatment selection in all settings, particularly post-BCG failure. Slide 23 illustrates the often long and burdensome treatment journey. Over this time, patients develop a trusted relationship with their urologist, one where they can regularly discuss treatment goals and create long-term care plans to avoid radical cystectomy. We consistently hear that treatment selection is not solely dependent on provider recommendations, but that patient priorities and practice capabilities guide decision-making. The discussion starts with each option's treatment burden, visit frequency, number of doses, and treatment duration, and whether it's manageable for a patient's schedule. Once a baseline for treatment expectation is established, the conversation can shift to efficacy, as well as safety and tolerability.
With many patients experiencing lower urinary tract symptoms, they must weigh efficacy relative to their current bladder health, as well as the level of post-treatment burden their site of care and family care providers can support. Since practice capabilities also play a role in guiding treatment selection, practices facing logistical and financial adoption barriers to certain treatments must discuss available options against the referral to an academic center, weighing continuity of care, convenience, and access. As expected, the patient's insurance coverage is checked. When mapping detalimogene's profile to date with these key treatment selection considerations, we believe it may offer value for all patients and providers. Detalimogene's shorter dwell time and less intensive dosing schedule may ease the burden on patients as compared to their experience with BCG.
When considering sequencing, urologists weigh patient phenotype, prior therapy, tolerability, treatment burden, and a differentiated mechanism of action to decide where a therapy fits. Detalimogene has the potential to be the first and only efficacious non-viral option in this category, with a complete response rate at any time of 56%, which is in line with approved products. As Dr. Kamat mentioned, 91% of patients in cohort one achieved complete response within three months, while the rate of progression was just 3% in responders with an attractive tolerability profile. Thus, detalimogene may be viewed as a low-risk, low-burden option to try. If you combine this with ease of access and utility for a urologist, detalimogene would be a viable and attractive sequencing option.
Slide 26 further highlights not only upfront dosing schedules across approved products and several in development, including detalimogene, but various post-procedural urine bleaching or urine contact precautions required by patients for some. For products requiring bleaching for more than a few hours, this could require patients to bring bleach with them to work, to visit relatives, to dine at a restaurant. While not listed here, certain products outline that a patient's clothing should be washed separately from their family's. Some caution close contact with immunocompromised individuals for specified periods post-treatment, or if a patient is experiencing a febrile illness, respiratory symptoms, or diarrhea, they are advised to avoid contact with others for up to two weeks. These are not insignificant considerations and can present major disruption to daily life. This is an area that detalimogene's unique non-viral mechanism of action may deliver meaningful patient value.
With the continued innovation and new therapies becoming available, we are excited by the prospect of a future where patients can safely sequence through multiple therapies, growing the market while simultaneously enhancing long-term care. We currently estimate there are approximately 20,000 addressable patients in the high-risk BCG-unresponsive setting. Projecting forward the patient journey outlined on slide 23, we see a future where sequencing and shared decision-making carve a new community clinical care pathway. With 75% of BCG-unresponsive NMIBC patients treated in the community setting, we believe that detalimogene's tolerability, in-class efficacy, and low burden to patients and providers could make it an early line choice. Key to continued market growth and patient access is, of course, payer coverage. We have conducted robust payer research, and what we have found is that interviewed payers will cover therapies at parity, assuming NCCN Category 2A guideline inclusion.
This has held even with the launch of new products at increased price points. Increased sequencing, payer support, and pricing dynamics look to support favorable future market growth. Capturing just 10% of the addressable population, or about 2,000 patients, can achieve $1 billion in net sales. Again, we believe detalimogene is poised to provide clinical, practice, patient, and economic value, and is designed for real-world practice integration. There is an opportunity to redefine the care pathway in community settings and ultimately help patients remain cystectomy-free. These value pillars, as well as the market segmentation work we have done, are helping us to inform the ongoing work we are doing to characterize key accounts and ensure that we appropriately size our field force, which will grow over the next year as we plan for potential approval. We estimate that across our practice segments, there are approximately 11,000 urologists treating NMIBC.
While account mapping is ongoing, we believe we can build effective coverage for a market of this size with approximately 40 - 60 sales reps. In closing, our early commercial strategy is being informed by the key value pillars across which detalimogene can deliver. The combination of meaningful clinical efficacy, excellent tolerability, coupled with operational simplicity, workflow fit, and the potential for positive practice economics creates our clear differentiation. As we work towards initiating our BLA filing later this quarter, we are actively working to identify and characterize key accounts and refine our engagement strategy. We have MSLs actively deployed to increase scientific engagement and education. These ongoing strategies will help inform a precise and efficient field organization, which we will steadily scale as we approach a potential approval next year. With that, I will turn the call over to Ron for closing remarks.
All right. Good stuff, Amy. Thank you very much. In closing, we are very pleased with the progress we have made since our last data update in May. The high-risk NMIBC market is poised for significant growth in the coming years. We believe that detalimogene, as the first and only non-viral gene therapy option, with its balance of efficacy and excellent tolerability, and what we believe is unmatched ease of use, would offer a differentiated total product profile and meaningful value. We are already working on lifecycle management through the incorporation of a short surfactant bladder rinse prior to detalimogene administration. This may improve the efficacy and durability while reducing the dwell time to 30 minutes, potentially reducing burden on patients and improve throughput for urology practices.
We look forward to initiating our BLA submission for detalimogene before year-end and expect to provide initial clinical data from our surfactant cohort mid-next year and approval for detalimogene without surfactant next year. With that, I would like to open up the call for any questions.
We will now begin the question- and- answer session. Please limit yourself to one question and one follow-up. If you would like to ask a question, please press star one to raise your hand. To withdraw your question, press star one again. We ask that you pick up your handset when asking a question to allow for optimum sound quality. If you are muted locally, please remember to unmute your device. Please stand by while we compile the Q and A roster. Your first question comes from the line of Maury Raycroft with Jefferies. Maury, your line is open. Please go ahead.
Hi. Congrats on the data update, and thanks for taking my questions. Wondering what else you could say about the pre-BLA meeting interactions with FDA and whether there is a specific efficacy bar for approval and how FDA views the current dataset and whether the current dataset is sufficient as is.
Yeah. Thanks, Maury, for joining us, particularly on short notice. I think we had a really good pre-BLA meeting with the FDA. With the FDA, there are really three topics to discuss, administrative, clinical, and of course, people forget the manufacturing aspect of things. I am happy to report that from an administrative perspective, the FDA has confirmed to us that we are eligible for a submission, so we feel good about that. They obviously saw the clinical data, so there is no bar. That is a view that does not exist. The FDA looks at every filing on the basis of both efficacy and tolerability, so I would suggest that they see our clinical data as being sufficient for filing.
Then let's remember that the CMC module, which is often five times the size of the clinical module, I think this is an area where we've made tremendous progress and we're in very good shape with that. You put the whole package together, I think the FDA has given us pretty strong feedback that the package that we have is sufficient to initiate the filing.
Got it. That's really helpful, and it's nice to see the data improve. I guess, wondering if anything's changed in how you're calculating CRs or durability, or does the improvement solely reflect the better patient outcomes with longer follow-up?
Yeah, I think that as we said earlier this year when we put out interim data, that our data had not matured. All that you're seeing here is the exact same patients in the exact same study maturing. I think, as we've said, we've seen improvements in all efficacy measures. We're delighted with that.
Got it. Okay, thanks for taking my questions. I'll hop back in the queue.
Thanks, Maury.
Your next question comes from the line of Andres Maldonado with H.C. Wainwright. Andres, your line is open. Please go ahead.
Hi, guys. Congrats on the progress here. Really tipping the hat off to you. Couple questions from us. Maybe if I could get some color if Dr. Kamat is still on the line. In terms of how much of the durability improvement from responses comes from responses after induction, and are those responses as durable as those achieved initially? I would like to get his kind of commentary on what he sees in his practice for those patients that have to go re-induction, based upon any therapy of his choosing. And maybe one for a secondary question would be, since the maintenance requires a CR at 12 months, could we get a little bit more color on what that 100% durable responses statement tells us, kind of beyond that milestone, in terms of specifically the patient journey there and subsequent decisions that he might make afterward? Thank you.
I think Dr. Kamat, some comments around durability in general, I think, would be helpful.
Yeah, absolutely. No, I mean, that's a very good question, and I think what you're alluding to is exactly what patients ask, right? Patients are more interested in how likely they are to be free of disease at that one-year mark and beyond. To be honest with you, patients don't really care how they get there, right? When we first designed or recommended designing clinical trials in this space, re-induction was not allowed because back in the day, we didn't realize that it might be safe. We didn't know. As a community, we recommended, and of course, the trials were designed with no re-induction. Then subsequent to the first couple of approvals, a re-induction was allowed, as is allowed with standard BCG therapy.
In the re-induction phase, across most studies, and you'll see that, again, here data is maturing and evolving, and I might refer it back to Amy or Lauren to give you data that wasn't presented, if they can. But essentially, you would see that roughly about 20% of the time, a patient will require that repeat course. We call it re-induction, but it actually is just additional therapy. Patients, like I said, how they get to the 12-month mark, they don't really care. They want to get there. Whether it's continued maintenance or re-induction is a little bit of a semantic thing on the clinical side. Hope that answers your question.
Yeah, just to answer your second question, Andres, I think the way to think about the flow in this and the use of detalimogene, I think the first thing to think about is, if you are given detalimogene, 91% of the time, we'll know if you're a responder in the first three months. That is a good thing. On the downside, only 3% of patients progress. Then, what we're saying from the 100% of number is that if you finish the year, you finished a full course of therapy, and you go into the maintenance phase, which is half of the doses, 100% of those, right, have gone into being durable. I think that what this speaks to is, this is a product that very quickly, if it's going to work, if it does work, if it doesn't work, there's very little downside.
If you make it to the end of the first year, it's very likely that you're going to be durable, which makes it a really valuable agent for both patients and for urologists.
Great. Thank you very much.
Your next question comes from the line of Yanan Zhu with Wells Fargo. Yanan, your line is open. Please go ahead.
Great. Congrats on the data, and thanks for taking our questions. Wondering, maybe to follow up on an earlier question about the improved both DOR more than 12 months, as well as improved 12-month landmark CR rate. Does that improvement come from later enrolled patients who were still pending evaluation as of the interim readout last time? Does this speak to any potential variability in patient characteristics? Another question, just a quick one. Was wondering if Dr. Kamat can comment on utilization for detalimogene in the academic setting, because I think the company has made a very strong case for reasons why the drug should be a good option in a community setting. Wondering if Dr. Kamat, as an academic center expert, can comment on how you see this therapy of use in the academic setting. Thank you.
Yeah. Thanks, Yanan. Let me take the first one, and then Dr. Kamat can comment on the second one. When we look at the durability post 12 months, or the landmark number, all of those improvements are strictly a function of patients maturing. You may recall that we had 21 patients in the May update, 19 of which had a CR at that timeframe. All that's happened is those patients have been on therapy for a longer time and we're reporting that data. That's why we're delighted that, in fact, what you're seeing with an efficacy picture for detalimogene is all parameters have improved and have put us in range of the products that are approved and in the market. Dr. Kamat, maybe you can talk about utilization in the academic centers.
Sure. Absolutely. I recognize why you're asking that question is because we are, or at least it has been highlighted that this is an easy to store, easy to administer agent, and it does have certain advantages in the community. That doesn't mean the reverse, right? If I implied or somebody implied that we would not use it in academia, that was an error on my part. It is efficacious, which is what patients want to know. It is easy to administer, which is what the workflow likes to know, whether it's academia or in community. Of course, it's safe and patients tolerate it well, which everybody wants to know. So, there's no reason we wouldn't use it in academia, if that's what you were asking. Now, where it falls in the hierarchy or in the sequencing, it really is dependent on shared decision-making.
It's a lot of factors that we talk to the patient about. We talk about efficacy, we talk about frequency of patient visits, we talk about side effects, potential mechanism of action, and that's very individualized to patients. So, there's no reason we wouldn't consider it one of the options for patients in this space. I just think the advantages in smaller community practices are what were being highlighted.
Great. Thanks for all the color.
Your next question comes from the line of Sean McCutcheon with Raymond James. Sean, your line is open. Please go ahead.
Hi, guys. Thanks for the update, and thanks for taking our questions. A couple from us. Can you speak to any prospective or retrospective biomarker analyses you've looked at to define the responsive patient population and perhaps the durable patient population, and how this informs your view on the bladder wash cohort probability of success? Then second, understanding it's a relatively small cohort of patients with a mix of prior therapies, but can you speak a bit more to the outcomes in patients who received post-BCG therapies? Any insights from those patients, how they performed on the study, and how that informs the competitive profile and ability to sequence for detalimogene? Thanks.
Sean, you're getting ahead of us here. The data is still very fresh. I think, look, we haven't had a chance yet to look at biomarkers, but I think the key point is that detalimogene seems to work across a wide group of different types of patients. I think that the point that Dr. Kamat made is that, compared to studies done in a different era, that in fact the population that we see here is a real-life population, so it becomes even more about in a real-life perspective. To your comment about the surfactant bladder rinse, the one thing there is that we will be able to look at urine IL-12. That will be one important biomarker. So we'll have urine IL-12 data for detalimogene on its own, urine IL-12 data with the bladder rinse.
One note of caution, it won't be the same as within the mouse, right? Because within the mouse and NHPs, we're able to harvest those bladders, right? So the ratios won't be the exact same, but we hope to show a difference there. Then to your point about the subgroups, this is going to be a very rich database. The 125 patients we studied, it's one of the larger NMIBC databases. So, I'm pretty sure our new Chief Medical Officer with the medical affairs teams will be cutting that data multiple times and will be presenting it over time, so that's going to be very exciting to share.
Your next question comes from the line of David Dai with UBS. David, your line is open. Please go ahead.
Great. Thanks for taking my questions. I also want to congrats the wonderful data here. Two questions from me. One, just on BLA submission, what regulatory work remains before BLA can be initiated and completed? More particularly, which module will be submitted by year-end 2026, and what are the remaining key items before completing the rolling submission in 2027?
I think we're just gated on those last nine patients, right? Everything else is good to go.
Got it. Great, thanks. For the surfactant cohort data, which we should be expecting in 2027 or mid-2027, what's your development strategy for that cohort? Are you planning to run a pivotal trial based on the data, or could you actually add the surfactant rinse to the label without an additional trial?
I think our expectation is that the surfactant bladder rinse cohort will form an sBLA, and we're looking forward to that. We've selected a surfactant polidocanol, which is readily available in the marketplace. We've studied detalimogene without a surfactant in cohort one, pretty much the exact same patient population, so we feel pretty good about the development path for detalimogene plus a surfactant. We're also very excited about right now, detalimogene has improved across all efficacy measures and will be, we believe, the only non-viral medicine and a very useful tool in sequencing.
All right. Thank you so much.
Your next question comes from the line of Allison Bratzel with Piper Sandler. Allison, your line is open. Please go ahead.
Hi. Good afternoon, and thanks for taking the questions. Maybe just a clarification. Could you just clarify how you and FDA arrived at the agreed-upon efficacy-evaluable population? I think you said you're excluding five patients. Were those from the pre-protocol assessment cohort or post-protocol amendment? Then maybe just for Amy, you've done all this and presented all this market segmentation work and analysis. Could you just talk to the kind of pre-commercial activities you can undertake now to prepare the market and just any update on how you're thinking about pricing, given this updated data? Thank you.
Yeah. Thanks, Allison, for jumping in. I think, look, you have a protocol, you have an inclusion and exclusion criteria, but when you have multiple sites like we have, there are just some patients that leak through that do not fit the inclusion and exclusion criteria. In dialogue with the FDA, the five patients that have been excluded, [inaudible] from 125 to 120, as you can see, the main thing is these are patient who hasn't had an assessment, patient doesn't have CIS within the timeframe, patients who didn't have enough BCG. That's kind of fundamental to the patient population. As a result, they will be excluded. We're actually really pleased to have had a good dialogue with the FDA, constructive dialogue around the patient population. I think it provides us a lot of clarity for us going forward. Amy, do you want to take that next question?
Oh, yeah. Of course, on our pre-commercial activities, I think as we've outlined, we have our MSLs out in the field. We are really kind of advancing our scientific engagement, our education, as we've highlighted here, really understanding the patient journey and the key decision-making points within that journey. So, I would say just really building deep insights into the marketplace. On the pricing question, I'd just say, it's always too early to provide formal guidance. But we know that recently approved products have ranged between $500,000- $700,000 a year mark. As we've highlighted, our market research shows little payer sensitivity and pretty strong reimbursement for products with NCCN Category 2A guidelines and the inclusion in that. So, as I say, too early on the pricing, but obviously we think it's a growing market.
Your next question. If you would like to ask a question, please press star one to raise your hand. We ask that you pick up your handset when asking a question to allow for optimum sound quality. If you are muted locally, please remember to unmute your device. Your next question comes from the line of Silvan Turkcan with Citizens JMP. Silvan, your line is open. Please go ahead.
Yeah. Thanks, Ron, and congrats on this data update. I have two quick questions, maybe one for the doctor here. Could you just walk us through maybe the, as you said, there's a joint decision-making going forward. Walk us through the day of a patient if they would get the detalimogene versus maybe approved ANKTIVA or INLEXZO or potentially even the cretostimogene regimen. Maybe you focus on dwell time in the office, and prep time for the patient. Then, a follow-up on the new CMS rules for next year. Do you have any visibility in that detalimogene is impacted by this 340B hospital reimbursement that fell to ASP minus 28%? Is that already factored into your pricing, or could you just describe how that factor influences you? Thank you.
Dr. Kamat, maybe you take that first question.
Sure. I was hoping you would take the second question first, but I'll take the first one. I mean, do we have like an hour? Because that's how long it takes to talk to patients, right, about the different options. I wish I could give you an easy, succinct answer, and if I did, you shouldn't believe what I'm saying because essentially it was oversimplification. It's not one size fits all. It really isn't. We have patients that come from really far away, and they will ask about efficacy numbers, but they care more about the fact that, "Hey, can I go back to my community, and can I get this in my community?" Of course, it matters if a particular regimen is available locally. Can they start it at our place and get it locally? On the other hand, there's some patients that say, "You know what?
Throw the kitchen sink at me. I don't care if I have all the side effects in the world, but I want to hit that highest number that I read about." In that case, they might be okay with having something put in their bladder every day, every week, twice a week. There are some clinical trials with three times a week treatment, right? So, it's not really I mean, I wish I could make up some answer for you, but it's not a one size fits all. A patient who likely has had chemotherapy will be benefited by immunotherapy, so that's one thing I can say. If a patient had GemDoce, for example, we wouldn't put him on a chemotherapy regimen. On the other hand, someone who's just had a whole lot of immunotherapy probably wouldn't use an agent that's immunologically based.
That is something we may consider. I forget who asked earlier, but somebody asked earlier, and I am sure once and if we have biomarkers that suggest that a particular regimen is better for a particular patient, then of course it will make it simpler. But right now, it is a very, very complex decision. That is why I always say it is great to have more options because then we can present more options to our patients, and they can help us choose what works best for them.
Great. Thank you, Dr. Kamat.
Yeah, thanks for the question on 340B reimbursement. The one thing I would just say is we know that the reimbursement landscape will continue to change over time. We continue to monitor that and kind of understand that. I think the key, honestly, is going to be understanding our segments, understanding our accounts, and really going back to those four value pillars that we discussed, and really understanding this over time.
Yeah. I think, Silvan, I think what we are pretty excited about is, all our efficacy parameters have moved up, right? I think we are in the range of approved products. As Dr. Kamat has said, there is not one solution for these patients, right? The nice thing about it is that we are the only non-viral gene therapy, right? They are going to have choices of chemos, they are going to have bacteria choices, they are going to have gene therapy choices. But we are unique as a non-viral gene therapy that they can reach in the fridge or freezer to use. We think that confers some real advantages and should be able to work our way through reimbursement and work our way through establishing ourselves as an important cornerstone of therapy.
Great. Thank you.
There are no further questions at this time. This concludes today's call. Thank you for attending. You may now disconnect.