Stifel 2026 Healthcare Conference 2026

Nov 11, 2026

Summary

Early REVTORPYK launch indicators included broad reimbursement coverage and strategic-account ordering. Positive mutant and first-line breast cancer data support expansion plans, while a prostate phase II readout is expected in 2027.

All right, we're going to go ahead and get started. I'm Stephen Willey, one of the Senior Biotech Analysts here at Stifel, and glad to have with us as part of the next session, Chief Executive Officer of Celcuity, Brian Sullivan. Brian, thanks for being here this morning. Welcome. I know you guys have a lot going on. Maybe before we jump into Q&A, I'm not sure if there's any opening statements that you'd like to make. Well, I think it's helpful maybe just to look back on the past 18 months for us, because it's been a transformative period. We reported our first phase III data, middle of 2025 for PIK3CA wild-type patients in the second-line setting of breast cancer. It was very, very positive, unprecedented data. We've subsequently gotten an approval for that indication, so that's a big deal, and launched our first indication, and so we're in the process of that. We also announced data, positive data for a second phase III trial in patients who have PIK3CA mutations. Again, very positive. Certain components of that were unprecedented data. We've submitted an sNDA there. We did get a PDUFA date, so that'll be end of Q2. We're laying the groundwork. The launch itself is, and I'm sure we'll talk about that in detail, gotten off, in our view, to a good start. In that same period of time, we started two phase III studies in the first-line patient population. in two different populations. We're separately evaluating each of those patients in a single study. Market potential for that segment is very, very significant. 90,000 patients would potentially be indicated for gedatolisib if those trials read out positively. We have very favorable preliminary data in those populations, so we think the probability of success is high in those studies. We've also reported recently data in the metastatic castration-resistant prostate cancer population. Again, very encouraging, not surprising in our view, given the similar biology that underlies prostate cancer and HR-positive breast cancer. More to come. I mean, we're at an earlier stage, but that data was what we were hoping to see. Certainly then warrants further investigation. We'll be initiating additional work to further prosecute that. Okay. Maybe we can kind of start off working through the REVTORPYK launch. Sure. I know it's early. You're five weeks in, or you were five weeks in when you reported last week, but it does seem that you have made a meaningful amount of progress within a fairly short period of time. As you look at some of the early metrics that were provided on that call a week ago, which of those kind of gives you the most confidence that this drug, from the outset, is really kind of resonating with prescribers? Sure. Well, one of the challenges when you are launching a drug is that there are a lot of gatekeepers that you need to work with to lift the gate. For instance, insurers would be an example of that, right? If they do not incorporate our drug on their policies and patients cannot get reimbursed, that would be a huge barrier. So we were successful, and that just reflects the work done prior to launch, not just since launch, but prior to launch, to get incorporated in those policies, to get NCCN level 1 recommendation, so that now 90%-plus of patients have policies that will reimburse them for treatment with REVTORPYK. That is an important gate to get lifted. Another important gate is with what we label strategic accounts, okay? These are accounts that when you look at the total population that they collectively treat, represent 50% or treat 50% of breast cancer patients. Now, these accounts have multiple sites, so it is 36 accounts, but ultimately they represent well over 1,000 different treatment locations. Because of the way they are organized, think of them as they have a corporate approach to saying, "Okay, we want to have this drug on formulary. We want it to be incorporated in the pathway. We need to have order sets built. We need various committees within our organization to approve usage of this drug." That is a process. It is a lot of constituents, a lot of infrastructure that needs to get built. Until that is built or those approvals are received, that is a gate. If you have an account that has 1,000 or rather 400 treating sites, you need to get that gate lifted so that your reps can effectively engage with physicians who treat patients at those sites. We were successful there in getting roughly 80% of these key 36 strategic accounts to order, which essentially an order reflects that all of this pre-work- was successfully accomplished. Those were very much a focus of the initial launch period. Very, very pleased that a lot of these gates got lifted. Also pleased with the level of engagement that our reps have had at the account level, where we've prioritized accounts based on number of patients they treat, and they've been able to get into roughly 3,400 accounts that comprise or treat roughly 80-plus percent of breast cancer patients. So they're getting access to the doctors who are the accounts that really treat the great bulk of breast cancer patients. Then we've also been pleased with the interest in participating or attending peer-to-peer programs where they can listen to a regional KOL or a national KOL talk about the drug, present the data, get perspective from other physicians who are attending. That's very powerful because ultimately they'll listen to us, but they'll listen more to someone who's a fellow physician. We've been very focused on making sure those programs are scheduled and people show up, and so far so good. All of the kind of necessary groundwork that's required. I would say all of it. A lot of the necessary groundwork that's required has been accomplished as we had hoped. Okay. I know the distribution channel is a little bit unique here, right? You kind of are functionally the supply chain, right? Yep. You're kind of drop shipping vials directly to infusion centers. You don't really need the specialty pharmacies. I know that model limits some of the granularity of the data. Right that you can access. Right. Certainly investors usually try to track. Sure. Right? In the absence of having that, what are some of the key metrics that investors should expect to see from you guys on a go-forward basis? Well, they'll be fairly basic, right? Because we don't see, it's not a question of us sharing it. It's just not available to us because the orders come through the distributors and we just get site information. So certainly the revenue and number of vials will be the most important metric. We'll have a lot of internal metrics. We've got a laundry list of metrics that we'll be using primarily to assess momentum at accounts, compare different regions or different territories, assess how certain accounts are doing relative to what we think their potential is. So, a lot of performance metrics to gauge penetration at as granular a level as we can. It's not clear to us that initially, how we would translate that into stuff we would report. What we want to make sure is that when we do start reporting, A, we know we will be obligated to report it consistently going forward, but B, that it is interpretable. If we present information that results in 10 different interpretations, it is not very useful. I think as we do our analyses, we will be thoughtful about providing information that will be helpful to investors, but also not be counterproductive either. Okay. TBD. Okay. Beyond what I have just described. I know these infusion centers are generally, and we have talked about this, a bit averse to holding inventory. Maybe, and again, I know it's very early, but are you expecting that these infusion centers are going to be ordering single vials at a time, or are they going to be ordering three vials, i.e., a cycle of treatment at a time? As these infusion centers gain more experience on the reimbursement side, certainly get more comfortable with the temporary J-code you're going to have in place until April 1. Yep. Would you expect that pattern to change over time, or is it always going to be a single vial drop for each patient at each center? I think in this day and age, when you're able to provide inventory on a just-in-time basis, if we receive an order from our distributor for a site before 3:00 P.M., we're shipping that day, and they'll receive it the next day. We've received a kind of a I don't, I can't say the percentages, but some sites will order one vial, some sites will order two, some will order three. It just depends on their profile. That tells you they're just started a patient. Over time, they'll have more patients, and then it gets a little more complex for them to order. They may just say it's easier to order a cycle at a time. It's hard to predict. Because they don't have multiple steps of distribution the drug needs to go through, which creates uncertainty about timelines, that gives them the flexibility, and I think a lot of them will use it, to order on a just-in-time basis. We won't see stocking up really, and I think the maximum stock up would be related to ordering cycles of drug rather than weeks of drug. Mm-hmm. Okay. Yeah. I know your field force has been capturing some qualitative feedback. They obviously will on a go-forward basis. Again, it is still early, but what is that feedback telling you with respect to utilization as a function of either line of therapy i.e., second line or third line plus, and preference for doublet versus triplet? It is anecdotal, so we have to just caveat it. We do ask the reps to be diligent in trying to get this information, but there is a lot of steps along the way to get that information. What we are hearing is that for the most part, doctors are thinking of this drug and the patients they are going to treat, for those who are progressing, whether it is in the second line setting or potentially the third line setting. I think there has been this question around, you have warehousing or do you have a big bolus of salvage patients? Certainly there is, I would assume there is some referred, 1C, 2C discussions of that. I think the bulk of the volume, just based on what we are hearing from reps, is for patients who are presenting with progressive disease. Not just switching therapy from what they are currently receiving, which is what happens in a salvage setting. The second versus third line, again, I cannot assign a number to it, but I would say it sounds like a good chunk of the volume would be associated with second line patients. As far as the doublet versus the triplet, again, anecdotally, we are hearing that there is a preference for the triplet, which is what we have expected. That is what our research has indicated would be the case. We cannot assign a number to it, but I guess the most important information was that so far, we have not heard anything from the field force that is anomalous or varies from what our quantitative research did prior to the launch. Things are essentially so far consistent with what our expectations were. Okay. I know the second line treatment landscape for PIK3CA wild type is pretty fragmented. Yes. Right? When you look at all of those available therapies, which of those do you think will be the easiest to displace, and which of those, if any, do you think might take a little bit more work to displace? I think they all fall under the same category, as they're all kind of second-best solutions. I think the feedback we received is that the REVTORPYK regimen really is a first-best alternative. Now, you will always have doctors who are comfortable with what they're doing, and they may take time to switch. But if you have a doctor that's comfortable using ENHERTU in the second-line setting, which for these patients is probably a fairly, I won't say significant, but it's one of the more significant options that physicians select. Well, then it's a fairly straightforward discussion to explain to the doctor why going to endocrine therapy, because now there's a good endocrine therapy regimen available to them. That's very much consistent with the general consensus amongst breast cancer docs, which is, if there's a credible opportunity to induce a benefit with an endocrine backbone therapy, that will be preferred prior to inducing, or rather, prior to treating patients with chemotherapy or an ADC, which essentially is chemotherapy. Then, as in the case with ENHERTU as an example, and this is typical of chemotherapies, patients will get approximately the same benefit independent of what line of therapy they get them. So, there's no reason to move upline with, let's say, ENHERTU as an example. They'll get the same benefit as a third-line therapy, or the patients will. This is information that our reps have been gathering to understand the treatment paradigm they're currently using, and in turn, depending on what that is for a particular physician, help the physician understand how to maybe think through the process or to understand what the data is suggesting and why the REVTORPYK regimen would be a superior alternative for their patients. Okay. I know Lilly's Abema and imlunestrant just got approved in second line for ESR1 mutants. I believe Roche's giredestrant and everolimus has a PDUFA date that is coming up maybe before the end of the year. That is right. Yeah. How do you just think about REVTORPYK kind of competitive profiling against both of those regimens within that ESR1 mutant subset? Sure. As we segment the market or as we think about how to segment the market, we think there are three practical patient subgroups. There are the double mutant patients who are rather double wild-type patients that lack either a PIK3CA or an ESR1 mutation. That represents about 40% of patients. Then there are patients who have a PIK3CA mutation. That represents another 40% of patients. We think the REVTORPYK regimen has demonstrated superiority that is fairly unequivocal, which would lead to, we think, a high proportion of patients receiving that drug. Then the remaining 20% are patients who are ESR1 mutant, PIK3CA wild type. That is where we think that the Abema, Imlu doublet, and the giredestrant doublet with everolimus will be most competitive. Now, we think our data stacks up very well against those, and we've done meta-analysis, for instance, in the wild-type setting, that takes into account mutational status of ESR1, and shown with the study that Sertilani at Dana-Farber just presented at ESMO, that REVTORPYK regimens of triplet offers two times greater likelihood of a patient surviving without disease progression or death than Abema, Imlu. There's a very, very compelling efficacy story to be told for REVTORPYK regimen relative to these other regimens. We think that'll be a more competitive segment. But at the same time, to the extent that a physician, for whatever reason, decides that they want to use those regimens for that cohort of patients in the second-line setting, then we think it's not unreasonable to expect potentially the doctors to use a REVTORPYK regimen in the third-line setting. Okay. Yeah. I know there's been kind of some suggestion that maybe this incidence rate of ESR1 mutations might be higher than what is seen in the literature, right? The literature would suggest- Yeah right, as your kind of breakdown explained, is in the 30-ish percent range. 30%, 40%, I think. Right. But I think there's been some suggestion now where, if you were to give folks more sophisticated ctDNA testing Yeah exiting frontline with progression, that maybe that rate would trend up to 50%. Do you agree or disagree with that, or? I think that's speculative. I think people are just guessing. I don't think there's any I don't know the study that would say perfect testing yields that result. But I think you do have to take into account the practical real-world practice as far as what is the incidence of ESR1 testing. What are the methods used to detect an ESR1 mutation? When these clinical studies are done, there is 100% evaluation of those patients, and they yield about 40-plus percent. Yeah ESR1. Yeah, maybe. But I think it is unlikely that you would see much greater than that, because the clinical trial presents the best-case opportunity to detect an ESR1 mutation. They are using the best available technology to evaluate and identify those patients. Okay. You mentioned the sNDA submission for the mutant data from study two of VIKTORIA-1. I know the PDUFA date now is going to be in late June. You did show some additional subgroup analyses from these patients. Right. How does that data that you presented at ESMO further inform your perception regarding the value of the triplet? Sure and doublet within each of those patients? Well, I think the comparison is somewhat confounded by the fact that the triplet was a significant sample size with prescribed alpha assigned to that analysis, whereas the doublet was a third the size. You will have natural variation with a smaller population. But you did see in the intent-to-treat population a very similar PFS result. But when you dig into some of the additional data, I think you see pretty significant differentiation between the benefit of the triplet versus the doublet. We presented data for patients who lack liver mets, no liver mets. Represents about 70% of all patients. Only 30% have liver mets in the second-line setting, based on real-world data. Well, the triplet reported 21.5 months median PFS, whereas the doublet, 11.5 months. Very consistent with their intent-to-treat PFS. Well, that is a pretty significant difference. That is a 10-month difference, which provides a very compelling argument that the triplet would be a preferred option for that patient population. The overall survival data has been mature, but again, at this stage, you would look at the triplet as suggesting a more favorable OS trend. Then if you were to look at the objective response rate, 59% versus 43%. So clearly, there is more activity going on with the triplet than the doublet. It is not unusual when you have a small sample size that it may not represent the, quote, "true effect size," because you just have more potential opportunity for variance. That's all information that we think provides a pretty compelling rationale to use the triplet, similar to the rationale for using the triplet in the wild-type setting. Okay. How do you think the FDA is going to handle labeling? I know we've kind of talked about this a little bit before, but both regimens in that trial were clearly superior to control arm therapy, right? That's obviously an important observation. As you said, right, only the triplet regimen was allocated alpha in the analysis, but then the triplet didn't really establish the contribution of palbo relative to the doublet. I would imagine FDA is maybe a little bit hesitant to approve the triplet with No, no an additional That's not at all the case. Okay. The triplet was a primary analysis that demonstrated superiority. Yeah. So there's no question. The question the regulators are asking, will ask themselves, and again, this is obvious, is that when you don't have prescribed alpha and you're trying to use that analysis to support an approval, the agent tends to be very cautious because they're setting a precedent. And so there's kind of a laundry list of drugs that have attempted to use non-alpha supported, non-alpha allocated analyses to support an approval. And it's, as the agency calls it, complex. It's a review issue. So really, the only question is whether we get the triplet and doublet or just the triplet. Okay. Then just with respect to label expansion and the timeline itself, can you say if you requested priority review? So they give that to you one way or the other. We knew going in, we had two options. We could have gone in requesting only a triplet indication, and we think that would have been a very straightforward review, very clear data. We thought it was worth attempting to get a doublet indication. We think there is plausible rationale for that. But then that introduces what the agency refers to as a complex review. The guidance essentially says priority review is reserved for indications where there is no complexity involved in reviewing the data. As soon as you ask them to evaluate an indication that does not have an alpha, a pre-specified analysis, that is a complex decision. So, you always hope, but from a practical standpoint, we assumed we would just get a regular review because of that. That was our decision. We knew that the implication of asking for the doublet would lead most likely to a regular review. But we thought it was worth exploring with the agency. Okay. So I guess as that as kind of your base case assumption then, has that accelerated either the timelines or the urgency with which you have wanted to push that mutant data out into the public domain via a manuscript? Well, I mean, we had a timeline independent of that was we were in process of executing before we got the data. We kind of have certain steps to take and plans. We expect that manuscript to come out this quarter, and that manuscript would support a submission to NCCN for inclusion in the guidelines. Again, they will do their review and make their determination. But certainly the data would suggest that this would be a good alternative for the patients and appropriate to include in those guidelines. Okay. I know the KOL feedback that we have received would suggest there is certainly a high amount of enthusiasm to prescribe this regimen in mutants. There are obviously reimbursement mechanisms that are not necessarily tethered to NCCN if prescribers really want to kind of push the issue. Yeah. Mm-hmm. Do you get the sense that there is that level of enthusiasm here whereby those physicians are willing to jump through some of those hoops and fill out some of that additional paperwork in order to be able to write the script to a patient? Well, we obviously have to be very careful. Yeah. We can't promote that. To the extent doctors are taking those kind of steps, we don't want to be in the loop on that discussion, and we're not. There's a lot of enthusiasm for using the regimen in the patients with these mutations. I wouldn't be surprised if that was the case, but I don't really want to hear about it. The NCCN guidelines will create, assuming that they recommend the regimens, would create clarity because then it's clear insurance, for the most part, as long as you have a 2A or better recommendation, will reimburse patients for use of that drug. Okay. How do you think about the relevancy of some of the recent launch analogs that we've seen in the space? Every launch is different. I know the desire is to see if you could use this as a basis for assuming a trajectory or compare it. Depending on the setting, you can legitimately have a lot of salvage patients that are potentially receiving a similar type of drug, like an ADC that is launched as chemo. There is a rationale to say, "We will just swap out that chemo," right? You are essentially inserting a better chemo, and you may have a bolus of salvage patients, which means that the first quarter of revenue in that setting might not be representative of a true trend. In our case, I think the experience will be that patients who are presenting with progressive disease and need a new therapeutic option, that will be the population that we are primarily treating in the first quarter. But again, there is a variety of friction points that still have to be worked through the J-code. For instance, some sites will just decide they are not going to make it available until the J-code is available, right? That is just something you have to work through. It is going to limit the potential population you can treat. That is nothing specific to us, but it is part of just the environment that we are in. You have had other drugs launched in this space that are orals, tends to be a fairly significant load, so it is hard to discern exactly what patient demand is. We have tried to outline to folks how at least we think about projecting volume. Time to peak is an important metric when you are trying to determine the trajectory of a launch. We think the analogs that are most representative of us are from companies that have launched, or rather launching their first drug. If you look at those data points, you would see it takes about three years, 30-42 months. Pick 36. Then you look at the number of patients that you have available to treat, how long will they stay on treatment? We have provided those metrics. The price of the drug, obviously, it has been made available. The biggest variable that will essentially dictate kind of how big the drug, what the potential revenue is related to what your peak penetration assumption is. That is where you and analysts and investors will have to come to some determination. We obviously think that penetration potential is significant, but because of the size of the addressable market, when you use the metrics I have outlined, it is roughly $9 billion. It is a very large potential market for our drug, just given the pricing and the duration of treatment, that you can build a multi-billion dollar drug in this indication with a reasonable level of penetration, which I think just provides a sense of robustness to the opportunity that we have. Okay. I know the original launch date was delayed a little bit just in order to get visibility into the timing of a second supplier coming online. I think you are now guiding to that happening in the middle part of next year. Yeah. Is there anything you can say about just kind of what needs to happen at that site between now and then? Right. Two things. One, our current supplier will be capable of supplying us through 2027. Okay? We wanted to have visibility that a second supplier, who really serves two purposes. One, to provide redundancy, one, to provide additional capacity. We think that capacity will be required in 2028. The second component is that our plan is to get a third manufacturer going. We actually have another manufacturer in process in Europe, but that would be for European supply, eventually European supply. It is a case of building a group of manufacturers that, A, provide capacity, and then also flexibility in scheduling, and then additionally, so you have redundancy. We will have multiple things that will be happening in 2027 related to capacity. Okay. Maybe just lastly on prostate, we saw the updated data at ESMO. You are certainly seeing a dose-dependent improvement in depth of PSA reduction. You are pushing dose higher. up to 300 mgs. What do you need to see from this study to take the next step? Sure. Would you expect that next step to be a registrational effort, or would you want to size that out from a phase II proof of concept? We want to complete our phase II study, which will provide more definitive data, and also comply with Project Optimus requirements that the FDA has established. That we expect to happen in 2027. That would tell us two things. A, how to think about the population that we would seek to evaluate, and then secondly, the dose to use. One of the more interesting findings from the study so far is that the 180 dose induces a much different AE profile to the good in these men compared to women, i.e., substantially lower stomatitis, which we hadn't predicted. There was no basis to predict that. That's what motivated us to evaluate a higher dose. 240, we think, is the most likely dose that we would take forward beyond the 180 in this subsequent study. That gives flexibility in the dosing. With those two questions answered, i.e., which population and then dose, then we would expect, assuming the data is consistent with what we've already reported, we would expect to proceed with the registrational study. Okay. Anything we didn't talk about that you'd like to quickly highlight before we wrap it up? No, I think if you were to assess the addressable market that we're hoping to provide treatments for, it's as large as almost any other opportunity in solid tumors. The first-line setting is a very, very significant setting. This data that we've provided or that we presented on a preliminary basis, 48 months median PFS for first-line patients, which compares to 25 months that's currently available, that's been reported historically for these current standards of care. That provides a wide target zone for us for success. We think there's a very, we think, high probability of success for that study. The implications are pretty profound because of the number of patients, 60,000 patients would be eligible. That indication alone could be one of the more valuable indications in oncology. We're very excited about the opportunity to advance, get that study enrolled, and hopefully advance soon, not soon, but within the next few years to submission and hopefully an approval. Then I think as people reflect on the relevance of REVTORPYK, I think the data so far indicates that its relevance is a function of the importance of this pathway. The complexity of the pathway requires a drug like REVTORPYK, multi-target inhibitor. We're in a unique position to really prosecute these indications. Given the data today, we think that the probability of getting over the finish line is very high, and that has very significant implications for us as a business to the good. Mm-hmm. All right. Great. Thank you, Brian. Great. Thank you, everyone.