Syndax Pharmaceuticals, Inc. (SNDX) Earnings Call Transcript & Summary

Good day, everyone, and welcome to the Pivotal AUGMENT-101 top line Revumenib data conference call. Today's call is being recorded. At this time, I would like to turn the call over to Sharon Klahre, Head of Investor Relations at Syndax Pharmaceuticals.

Thank you, operator. Welcome, and thank you all for joining us today. I'm Sharon Klahre and with me this morning to provide a review of topline results from the Pivotal AUGMENT-101 trial of Revumenib in relapsed/refractory adult and pediatric patients with KMT2Ar, acute leukemia, are Michael Metzger, Chief Executive Officer; Dr. Neil Gallagher, President and Head of R&D. Also joining us on the call today for question-and-answer session are Dr. Peter Ordentlich, Chief Scientific Officer; Dr. Anjali Ganguli, Chief Business Officer; and Keith Goldan, Chief Financial Officer. This morning, we issued a press release and reported topline data for the Pivotal AUGMENT-101 trial of Revumenib in relapsed or refractory KMT2Ar acute leukemia, which can be found on the Investor page of Syndax's website. Please turn to our forward-looking statement disclosure on Slide 2. Before we begin, I'd like to remind you that any statements made during this call that are not historical are considered to be forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by these statements as a result of various important factors, including those assessed in the Risk Factors section in the company's most recent quarterly report on Form 10-Q as well as other reports filed with the SEC. Any forward-looking statements made on this call represent our views as of today, October 2, 2023 only. A replay of this call will be available on the company's website, www.syndax.com, following its completion. And with that, I am pleased to turn it over to Michael Metzger, Chief Executive Officer of Syndax.

Thank you, Sharon, and thank you all for joining us on the webcast. This morning, we are thrilled to announce topline data from the pooled analysis of Pivotal AUGMENT-101 trial of Revumenib, our highly selective Menin inhibitor in adult and pediatric patients with relapsed/refractory KMT2A rearranged AML and ALL. The Independent Data Monitoring Committee, or IDMC, reviewd the data and recommended the trial stop early for efficacy in accordance with a protocol defined interim analysis after achieving its primary endpoint with a CR/CRh rate of 23% in the evaluable efficacy population. Furthermore, the overall response rate was 63%, exceeding our expectations. We are excited to be one step closer to bringing Revumenib to patients with KMT2A acute leukemia, a population for whom there are no approved therapies and who could meaningfully benefit from Revumenib, if approved. The pivotal data we are presenting today continues to support Revumenib potential as a first and best-in-class agent. Slide 3 highlights the key aspects of Revumenib's clinical profile on which treating physicians are most focused. As mentioned, the majority of patients achieved a clinically significant response to treatment with high overall response rates in the pooled population as well as the AML population. Notably, a high proportion of these patients proceeded to potentially curative transplant much higher than the current benchmarks for patients with relapsed and refractory KMT2A rearrangements. And following transplant, many restarted Revumenib at its maintenance therapy. Several patients remain on therapy in the post-transplant setting, some for several months. The ability to offer relapsed and refractory KMT2A patients the opportunity to proceed to a potentially curative transplant signifies an important paradigm shift in how these patients can be treated. The historic rate of transplant for these patients is less than 5%, while Revumenib has consistently enabled approximately 40% or more of KMT2A patients to proceed to bone marrow transplant. Importantly, Revumenib continues to be well tolerated, which we believe supports its use not only as a monotherapy and maintenance treatment in the relapsed/refractory setting, but also its potential to be used in frontline standard of care combinations for which initial data is forthcoming later this year. Revumenib's potential advantage of having the only age and disease agnostic label in KMT2A acute leukemia sets the stage for first mover advantage to significant market opportunities. With this positive data now in hand, we expect to submit an NDA filing for Revumenib for the treatment of relapsed/refractory KMT2A acute leukemia by the end of 2023 and become a commercial stage company with potential approvals and launches of both Revumenib and Axatilimab in 2024. Just to remind you, Axatilimab is our anti-CSF1 antibody for which we presented positive top line data in July and we expect to complete a BLA filing with our partner Incyte, by year-end for the treatment of chronic graft versus host disease following 2 or more prior lines of therapy. Before I turn the call over to Neil, let me explain why we think Revumenib has the potential to change the treatment paradigm in patients with KMT2A and NPM1 acute leukemia. As described on Slide 4, there are currently no FDA-approved therapies targeting KMT2A or NPM1 acute leukemias, a population that together represents up to 40% of AML patients. With today's announcement, I'll focus on KMT2A acute leukemia. KMT2A acute leukemia represents approximately 10% of AML and ALL and this diagnosis is predictive of poor prognosis. Historically, a third-line patient with KMT2A acute leukemia is expected to have at best a 10% response to standard of care treatment. Fewer than 5% of these patients receive potentially life-saving transplants and life expectancy is less than 3 months. Today's data demonstrating a 63% overall response rate and significant numbers of patients receiving stem cell transplant followed by Revumenib in the post-transplant maintenance setting is why we believe Revumenib has the potential to change the treatment paradigm for patients and their physicians. I will now ask Neil to walk us through the Pivotal AUGMENT-101 topline trial results. Neil?

Thank you, Michael. I am very excited to be able to provide a topline overview of the results from AUGMENT-101 and to have an opportunity to be involved with a program that has the potential to make a significant difference in the lives of patients with acute leukemia. Revumenib is a first-in-class, highly selective Menin inhibitor. In the schematic on Slide 5, you see the binding of Menin into KMT2A, whether it retains its wild type C terminus as with mutant NPM1 AML or has acquired a novel C terminus as in KMT2A rearranged leukemia leads to transcriptional activation of leukemogenic genes such as HOX and MEIS1. Revumenib inhibits the interaction of Menin and KMT2A which suppresses the transcription of these leukemogenic genes, leading to differentiation and apoptosis of tumor cells and consequently, clinical remission. The Phase II portion of AUGMENT-101 was originally designed as 3 single-arm pivotal trials that independently enroll distinct patient populations, KMT2A rearranged ALL, KMT2A rearranged AML and NPM1 mutant AML. Up to 64 adult patients could be enrolled in each of the cohorts with the potential to enroll an additional 20 pediatric patients in each. Last fall, we show the results of the Revumenib Phase I trial in relapsed and refractory patients with KMT2A acute leukemia with the FDA and later at ASH. After reviewing the data, the agency agreed that the interaction of KMT2A fusion proteins with Menin is a critical driver of the leukemic transcription program in both KMT2A, ALL and AML and that the Phase I data demonstrated Revumenibs potential to provide substantial improvement over available therapies across both populations. Following the review, the agency granted breakthrough therapy designation for Revumenib, covering all KMT2A-rearranged leukemia patients, agnostic of age and leukemic presentation. The granting of BTD gave us the opportunity to work collaboratively with the FDA to update the trial in order to deliver Revumenib to patients as rapidly as possible. We reached an agreement that both KMT2A, AML and ALL cohorts would be pooled into a single analysis, which could support a comprehensive indication for the use of Revumenib for the treatment of adult and pediatric relapsed and refractory acute leukemia patients with a KMT2A rearrangement. The trial was designed to rule out the null-hypothesis that the median CR/CRh trait would not be expected to exceed 10%. Secondary end points included durability of CR/CRh, transfusion independence, overall survival and safety. Patients who undergo transplant in the trial have the opportunity to initiate maintenance therapy with Revumenib. This is an important option for patients, given that they are at high risk of relapse. Turning now to Slide 7. The data set we are reporting today is derived from the patients enrolled in both AML and ALL KMT2A cohorts. The updated protocol had prespecified the number of KMT2A patients that would define the efficacy-evaluable population for the pooled interim analysis. The protocol also specified that once those patients enrolled into the trial, they will be followed for 6 months before database lock. As we previously announced, the 57th evalue efficacy evaluable patient needed for the interim analysis was enrolled in the first quarter of this year. While those 57 patients were in the 6-month follow-up phase, the trial continued to accrue. Any additional patients treated in either of the pivotal KMT2A cohorts prior to the July database lock have been included in the safety population. The pool came into a safety population includes 94 patients and the pooled efficacy evaluable population includes 57 patients. Recently, the Independent Data Monitoring Committee met to review the data from the interim analysis and recommended stopping the trial for efficacy as prespecified in the protocol. Based on the IDMC recommendation, the company is closing the trial to further accrual in the KMT2A cohorts. The NPM1 mutant AML cohort is ongoing, and we expect completion of enrollment by year-end 2023, with data available in 2024. Slide 8 includes the baseline characteristics of both the efficacy and safety populations. The median age was approximately 35 years and primarily comprised AML patients. It is important to note that over half of the patients had relapsed, following treatment with at least 1 salvage regimen prior to enrollment, including nearly half, 46% and who had undergone prior stem cell transplant, reflecting the heavily pretreated stage of disease in this population. Patients had received a median of 2 prior lines of therapy. In this analysis, prior therapy was assessed according to the AML treatment guidelines for industry that the FDA published last October. The guidance defined for the first time how prior therapy should be assessed, including the recommendation that induction and consolidation therapy followed by transplant be considered a single line of therapy. Turning now to Slide 9. The Phase II data further established that Revumenib is an effective treatment for relapsed and refractory KMT2A acute leukemia. The trial met its primary endpoint with a CR/CRh rate of 23%, as you can see, the confidence infill of 12.7% to 35.8%, rules that the null-hypothesis of 10% with a p-value of 0.0036. More than 2/3 of patients who achieved a CR or CRh with a no measurable residual disease. The overall rate of response in this population was 63%. Patients were transplanted early in the course of treatment, and a notable proportion of patients receive maintenance therapy with Revumenib following transplant. The response rates observed in the KMT2A AML and stem population were consistent with those in the overall efficacy evaluable population. Overall, 65% of AML patients achieved a response and 24.5% achieved a CR/CRh. At the time of the data cutoff, the median duration of CR/CRh response was 6.4 months in both the efficacy evaluable and AML populations with 46% or 6 patients remaining in response. The data will continue to mature over time, and the duration of response may extend beyond 6.4 months with additional follow-up. As I mentioned a few moments ago, not only did we observe a high overall response rate of 63%. 39% of responding patients proceeded to transplant. Also of note on Slide 10, 8 patients were transplanted prior to achieving CR or CRh at the discretion of the attending physician. Clearly, the physicians had decided to wait a little longer prior to transplant, then more of these 8 patients could have achieved the best response of CR/CRh. I mean for example, if all 8 of these patients had achieved CR/CRh than the rate would have been 37%. Another important facet of the data summarized in the slide is the high proportion of patients 71% who either proceeded to post-transplant maintenance or who remained eligible for maintenance. As of the data cutoff, 7 of the 14 patients who have been transplanted received maintenance, including some who have commenced their 8th cycle. 3 additional patients remain within the post-transplant window and they choose to initiate maintenance. Similar to what we've previously shared at the European Hematology Association Congress, heavily pretreated acute leukemia patients in the compassionate use program, initiated Revumenib as post-transplant maintenance and stayed on therapy for extended periods of time, in some cases, almost a year. We look forward to being able to provide longer-term follow-up for these patients. Based on the clinical profile of Revumenib, we believe that it has the potential to change the current treatment paradigm by enabling physicians to aggressively treat KMT2A acute leukemia. Slide 11 contains a summary of the data from the safety population that I described earlier. The safety profile of Revumenib that was observed in the pivotal stage of AUGMENT-101 is generally consistent with what has been previously reported. Revumenib was well tolerated in this highly refractory population. And of note, only 6% of patients discontinued therapy as a result of treatment-related adverse events. The TR AEs of any grade in greater than 20% of patients included nausea, differentiation syndrome and QT prolongation. At 23% of QT prolongation -- I beg you pardon at 23% the rate of QT prolongation of any grade was less than half of what was previously reported in the Phase I data set. While the rate of grade 3 of 14% is consistent with previously reported data, as before, there are no grade 4 or 5 events and no patients discontinued as a result of a QT prolongation. The rate of differentiation syndrome observed was consistent with previous reports and only 1 case of Grade 4 Ds. All other cases were Grade 3 or less. No patients discontinued as a result of DS. In summary, we are extremely pleased with the data that we've released today. In this pivotal Phase II analysis, Revumenib has again demonstrated robust efficacy and a well-tolerated safety profile in a heavily pretreated acute leukemia population. With that, I would like to turn the call back to Michael.

Thank you, Neil. As you know, Syndax has designed impactful trials that position Revumenib for use in earlier settings with acute leukemia including combinations with approved therapies as outlined on Slide 12. We believe there is significant market opportunity in the frontline setting. And based on the data generated to date, we anticipate Revumenib will provide meaningful benefit across the acute leukemia landscape. Let me take a minute to remind everyone of a few of the other trials that we have ongoing. In the BEAT AML trial, Revumenib is being combined with venetoclax and azacitidine to treat newly diagnosed AML patients who are unfit for induction chemotherapy as part of our collaboration with leukemia & lymphoma society. Enrollment is ongoing, and we continue to expect data on safety at a potential RP2D from the trial by the year-end 2023. AUGMENT-102 trial is designed to assess the safety of Revumenib in combination with standard salvage chemotherapies for patients with relapsed or refractory acute leukemias. We expect to provide an update on the initial safety data and potential RP2D from the trial by year-end 2023. The SAVE trial is an interesting combination trial of an all-oral regimen, Revumenib with venetoclax and INQOVI, an oral hypomethylating agent in relapsed/refractory acute leukemia being led by Dr. Issa at the MD Anderson Cancer Center. We also plan to initiate a trial of Revumenib in combination with standard of care intensive chemotherapy known as 7+3, in newly diagnosed patients with acute leukemia by year-end 2023. This trial will include an option for maintenance with Revumenib monotherapy. We are highly encouraged by data being generated with clinically active doses of Revumenib in combination with standard of care agents and we look forward to sharing that data later this year. Turning now to Slide 13. The data we've shown today positions Revumenib with a best-in-class profile for relapsed/refractory KMT2A and further derisks NPM1 as the next indication. Revumenib is on track to own the largest addressable population in relapsed and refractory acute leukemia, which represents approximately 6,000 patients. These patients, together with their time on therapy are what build this to a significant market opportunity. As with other indications, moving into earlier lines of treatment opens additional avenues for Revumenib to benefit more patients and hopefully improve the overall course of their disease. We believe that Revumenib could become the backbone of treatment for acute leukemia patients with KMT2A rearranged and NPM1 mutant acute leukemias and realizing that potential could bring significant and additional value to the franchise. As you move into earlier lines, you not only access more patients, but they also have a greater chance of achieving a deeper and potentially more durable response to therapy. You can see from this chart that the opportunity for Revumenib could grow substantially as we look to take advantage of our first-to-market position and quickly add frontline indications to our initial relapsed/refractory approvals for KMT2A and NPM1. Turning to Slide 14. I'd like to briefly summarize why we believe Revumenib is positioned to be a blockbuster in acute leukemia? Through our conversations with leading investigators as well as our market research among treating physicians, we have come to understand that across the board Physicians are excited about the efficacy, tolerability and overall utility observed with Revumenib and KMT2A rearranged and mutant NPM1 acute leukemias. Approximately 250 patients have been dosed with Revumenib in AUGMENT-101. And Revumenib has shown an ability to provide deep durable responses for heavily pretreated patients as a monotherapy agent. It is both safe and well tolerated, and the overall experience across ongoing studies supports use in combination with standard of care treatments. Based on our discussions, physicians believe Revumenib enables their relapsed and refractory KMT2A patients to achieve remissions, go on to receive a stem cell transplant and follow-up with bone marrow maintenance with higher rates of success than experienced before in this patient population. Revumenib's oral route of administration allows for at-home administration and the availability of both a tablet and liquid formulation makes Revumenib broadly accessible across pediatric and adult patients. Turning to Slide 15. We have quite a few meaningful upcoming milestones for the Revumenib program on the horizon for the remainder of 2023. These include presenting the full AUGMENT-101 results at an upcoming medical meeting, filing an NDA submission for year-end 2023, initiating a combination trial with intensive chemotherapy or 7+3, presenting data from the additional ongoing Revumenib studies in combination with Ven and chemotherapy, completing enrollment of the NPM1 cohort and continuing to prepare ourselves for a potential commercial launch of KMT2A next year. On Slide 16, we would like to point out that we are making excellent progress executing on our milestones and corporate priorities. All of which has put us on a path to becoming a commercial stage biopharmaceutical company with potentially 2 best-in-class products. Syndax is in the enviable position of bringing 2 practice-changing novel agents through development to the verge of NDA FDA filings and we've been able to accomplish this in approximately 4 years from IND. This presentation marks the second positive pivotal data set we are reporting on in the last few months. The first was the AGAVE-201 data of Axatilimab in chronic graft versus host disease and now AUGMENT-101 data of Revumenib for relapse or refractory KMT2A acute leukemia and we look forward to presenting the full data set from both programs at the future medical meetings. Before we turn it over for questions, I would like to take a moment to extend our gratitude to the patients, their families, the investigators and the entire staff who participated in AUGMENT-101 as well as earlier and ongoing Revumenib trials. We would not be at this exciting juncture today without your valuable contributions and help to drive innovation forward. Additionally, I would like to thank the teams at Syndax for all of their hard work and incredible collaboration, which made this trial and the positive data readout possible. And with that, I would like to open the call for questions. Operator?

[Operator Instructions] Our first question comes from Anupam Rama from JPMorgan.

Congrats on the data. Quick one for me, the 50% of patients who received Revu post-transplant maintenance, what was the deciding factor in terms of starting a maintenance therapy versus not? And maybe should we be expecting a full system data update at maybe ASH?

Yes. Anupam, thank you for your question. So look, I think the deciding factor here for physicians, as we understand it from them, is that this KMT2A population is one where they're a little bit younger in their course of in their age, they're in the 30s and the treatment paradigm is that you want to take these patients as quickly as you can to transplant. So there's a drive to get them to transplant, the overall response rate that we reported today was in the mid-60s, which is an overwhelmingly good result and they're clearing their blasts. So they take them as quickly as they can to transplant, and we're seeing that, we saw that in Phase I, and we're seeing that in the Phase II, and it's going very well. And then taking patients in the 50%, taking them to post-transplant maintenance, we think that number could be as high as over 70% in this data set. I think that was a 50% was immediately put back on post-transplant. And then there are another 3 patients who are sort of in that window to potentially resume post-transplant maintenance. I think this is a new paradigm for physicians, and they're super excited to do that. So it's not going to be for everybody, but we do expect the majority of patients to go back on therapy after transplant. And then your -- I guess, yes, your second part of your question was about following up on data at a medical meeting. I think we're excited to get the NDA filed this year, and I think that's the #1 priority, but obviously, we'll follow up with additional data at a medical meeting, we generally don't comment on what that -- whether we -- until we have something accepted. So right now, we're anticipating having a presentation at an upcoming meeting for sure, where we present the full data set.

Our next question is from Brad Canino from Stifel.

Congrats from me on these results as well. And really thank you for all the data analysis provided. Two questions from me. First, I guess what are your expectations for the potential length of post-transplant maintenance? And I realize your data are still maturing here. But what have you learned from the experience of other AML targeted therapies and can you tell us if at least the initial Revumenib experience has matched that?

Let me ask Neil to make a comment on your question. Thanks for the question, Brad.

So the questions regarding the length of post-transplant maintenance. So as we've described in the data -- in the script, in fact, we've had patients commencing their 8th cycle of post-transplant maintenance in the current data set. And this is very consistent with what we've previously reported at EHA 2023 for instance, where we described patients receiving treatment for some patients for almost a year. So our expectation, as Michael alluded to, physicians are extremely encouraged by the ability of Revumenib to give them and their patients an opportunity to go to transplant. Many of these patients have actually failed prior transplant. And therefore, the opportunity to bring these patients to transplant is an extremely important one for patients and their physicians. Our anticipation is because these patients are at risk of relapse is that they will be kept on post-transplant maintenance as long as possible. And we have the prior experience to demonstrate that, that is the case. And the other point that I'd like to reiterate that kind of supports this is the point that Michael made is that either 70 -- over 70% of patients have either started post-transplant maintenance, 50% or could bring that number to over 70%. And I think that with these data coming out and when we present the data in more detail at a forthcoming medical meeting. That news is going to get out, right? The word will spread that this is a treatment option. And as more patients and their physicians have access to the drug and really understand the potential for the drug to provide benefit, we expect that patients will be kept on post-transplant maintenance for as long as possible.

Great. And if I can have a follow-up. I want to know how do today's results impact your view of the NPM1 probability of success? I think for the regulatory CR/CRh number next year. And do you anticipate any of the differences in use of transplant in the NPM1 trial population that could influence that CR rate?

Yes, Brad, thanks for the follow-up. So look, I think we're extremely encouraged by these results, the consistency that we've seen in the Phase I here. And what we reported in a Phase I for NPM1 was on the CR/CRh rate was the same as what we saw for KMT2A at 27%. And I think what's interesting and what we learned in this trial is these are different populations of patients, right? They're treated differently, meaning KMT2A versus NPM1. KMT2A, the patients are in their mid-30s in this trial, and that sort of lines up with demographics. So there are younger patients who are about 80% fit for transplant. So the goal of treatment for these physicians is to get them to transplant as quickly as possible and potentially put them back on drug. And that's a paradigm shift. Obviously, that we've reported on today, we're excited about. The NPM1 patients in contrast are older, right? So they're in the mid-60s generally. And the majority of those patients are not fit for transplant. And so what we would expect going forward, and we'll see what the results look like in the Phase II is that not only would the CR/CRh rate be similar to what we provided in the Phase I. But with the lower rates of transplant, we would expect that patients might be able to have full count recovery and higher numbers potentially before if they receive a transplant before getting there. There's just not that aggressive drive to move them to transplant as quickly. And so we think that could accrue potentially to a higher CR/CRh rate. So that's obviously data to come, but we're encouraged by and learning all the time from the data that we're generating.

The next question comes from the line of Eva Privitera from TD Cowen.

Congrats on the data. My question is on the durability. Do you expect the median duration response to continue to evolve and get longer? Even though there's less than half of 46% of patients still in response. And can you walk through how that calculation is done?

Sure. Thank you, Eva. Nice to speak with you, and thanks for your question. So just to point out a couple of things. First of all, 6.4 months includes about half of the patients still on therapy and in remission. So we do think it's a bit early days to -- even though there is a median, we calculate that based on the Kaplan-Meier curve. So there are several patients still continuing on and with -- we expect that to elongate over time. So that is an important thing to watch as we see. And we've seen that in the Phase I. Our Phase I experience is where they started and the duration lengthened over time. And so we're encouraged by watching these patients, obviously, with the impact of maintenance as well. Just to also point out, I think these are -- this is the first time we're reporting on duration of response on just KMT2A patients. Our Phase I experience included both NPM1 and KMT2A experience. So that is a little bit different than our Phase I, just pointing out the differences there. We do believe that the encouraging start to the duration calculation at 6.4 could certainly lengthen over time as we watch these patients.

Great. And a follow-up. Are you aware of studies looking at differences in transplant outcomes between patients who had CRI or CRP versus CR/CRh? Are there any differences in outcomes in waiting for the response to get to a CR/CRh or the outcomes are the same if the patient went into transplant with a CRI or CRP?

Yes. Let me ask Neil to comment on that question.

Yes. Thanks for the question. We are aware of a paper in press that does exactly the kind or at least under review that does exactly the kind of analysis that you're describing. We haven't seen the paper, but our understanding is that the analysis indicates that there is no difference in outcome, right? In other words, taking the right patient to transplant with -- in prior to achieving a CR/CRh, those patients have similar outcomes to patients who are transplanted with CR/CRh. Just a caveat, we haven't seen the paper, but we have -- we are aware that those data are right there.

The next question comes from Peter Lawson with Barclays.

Great. As regards to the patients that move on to transplant before reaching CR/CRh, any details you can provide around those, CRP or CRI, et cetera, and then the Phase I study where patients went on to transplant as well. But that's a similar scenario where some of them didn't achieve CRh?

Yes, Peter, thanks for the question. So look, I think the breakdown of patients, we had a very high percentage, relatively speaking, a high percentage of patients who were not fully recovered their counts. So I think we have that in the slides presented today. So CRP and MLFS roughly 40% of the patients were not completely recovered in their count. So a lot of patients who were, as I think we mentioned, 8 patients who were taken to transplant before being able to recover their counts. That's a significant number. And as you look at the CR/CRh rate, the pools analysis of 23% in the AML population was 24.5% and if you kind of do the mental math of if those patients have recovered their counts, I think as Neil pointed out in the script, you would have had upwards of the high 30s of response rate on the CR/CRh. Obviously, that's not the goal of treatment for these treating physicians. They drive them to transplant as quickly as possible, and they're not waiting for these count recoveries. So that's just how the practice works and the practice of medicine works here. But we are very encouraged, and we do know that some of these, call them, incomplete responders do quite well on transplant as Neil just pointed out, some of that data is unpublished but indicating that the paradigm is really important to follow up. And maybe Neil wants to make a comment.

Yes. Just one follow-up. I think you're asking about the breakdown of patients who went -- the 8 patients who went to transplant without achieving CR/CRh. We haven't provided that breakdown today. So we will be providing it at a forthcoming medical meeting.

Yes. That's true.

[indiscernible] sense that in the Phase I of the pivotal study that there were more patients getting to just achieving kind of an incomplete response before going on to transplant with physicians more comfortable with the drug to nurse the patients on the transplant?

Yes. Thanks for the question, Peter. Our fewer patients in the Phase I versus the Phase II had an incomplete response. So again, I think if you look at the patients who had gone on to transplant, in the Phase I, 8 patients had a CR/CRh before going to transplant versus 6 in this population in the Phase II. The non-CR/CRh going to transplant in the Phase I was 2 patients 20% versus in the Phase II was 8 patients. So again, 57% incomplete went to transplant in Phase II versus 20% in Phase I. It's a stark difference and it's based on what I've said today, I think those are -- it kind of lays it out pretty well.

The next question is from Michael Schmidt with Guggenheim.

Congrats on the results. Perhaps a couple of regulatory questions, with respect to the planned FDA submission, will this be exclusively based on the interim data percentage here? Or will you do another data cut before submitting to the FDA? And then the results in the BEATS, are they consistent with the overall analysis? And do you think you have enough data to potentially get a broad label, including BEAT as well. And lastly, any updates based on the results on your future studies or ongoing studies, any changes to some of the trials that you're conducting beyond the Relapsed and Refractory setting?

Yes, Michael, thanks for the questions. I'm going to ask Neil to comment on the regulatory FDA submission question first.

Thanks for the question. So as I mentioned in the script, we have BTD for KMT2A. So we've been in very close collaboration, many close discussions with the agency over the last year or so. The -- as I also alluded to in the script, the study design was modified in discussion with the agency to enable the interim analysis. Based on the 57 patients, so to exclude the null-hypothesis as we mentioned earlier. So the -- and as I also mentioned, the IDMC have recommended that the study be stopped for efficacy and the company is following that recommendation. So you can, obviously, we're discussing the filing with the agency at the moment. We don't get into detail of those discussions at the moment. But you can infer from what I've said that our expectation is that we would file based on the interim analysis since we've actually -- we followed the IDMC recommendation. In terms of the question with respect to pediatric patients, look, overall, the data are consistent across all of the patients that were included in the study. And then the third question was regarding any modifications of future studies. We're not really ready to discuss that at this time.

Yes. And I'll just follow on to Neil's comment about future trials. We are excited about some data that's coming towards the end of the year, as I mentioned in the script, about combinations and the ability to dose Revumenib combination, both with chemotherapy and Ven. And so we're excited to talk a little bit about that. And obviously, we're starting trials, frontline trials in addition to what we have going on. So it's a lot of momentum around new work that we're going to be doing with Revumenib and emerging results there as well. So stay tuned.

The next question is from Yigal Nochomovitz from Citigroup.