Kura Oncology at BofA Healthcare: Zifdomenib’s Promising Path Forward

On Tuesday, 13 May 2025, Kura Oncology (NASDAQ:KURA) presented at the BofA Securities 2025 Healthcare Conference, focusing on its clinical programs, particularly the development of Zifdomenib, a menin inhibitor. The company highlighted both opportunities and challenges, emphasizing the drug’s potential in cancer and diabetes treatment, while addressing strategic partnerships and financial stability.

Key Takeaways

• Kura Oncology is advancing Zifdomenib, targeting AML and other cancers, with a focus on safety and patient quality of life.
• The company has submitted an NDA for Zifdomenib, seeking priority review, and is exploring its use in combination therapies.
• A partnership with Kyowa Kirin supports Kura’s financial position and clinical program expansion.

Financial Results

• Kura expects to maintain a stable cash position through the end of the year, bolstered by its partnership with Kyowa Kirin.

Operational Updates

• NDA Submission: Submitted for Zifdomenib on March 31, with a request for priority review to expedite approval.
• FDA Interactions: Positive and collaborative, facilitated by the drug’s breakthrough therapy designation.
• Clinical Trial Enrollment: Progressing well across all cohorts, including COMET-7 and COMET-17, with strong interest from investigators.
• Partnership: The strategic alliance with Kyowa Kirin aims to aggressively advance Zifdomenib in various therapeutic lines.

Future Outlook

• ASCO Presentation: Upcoming data presentation on Zifdomenib’s efficacy in relapsed/refractory AML, focusing on response rates and safety.
• EHA Presentation: Planned release of data on Zifdomenib combined with intensive chemotherapy.
• Phase 3 Trials: Preparations are underway for Phase 3 trials in both intensive and non-intensive AML treatments.
• Clinical Updates: Expected throughout the year for GIST, diabetes, and FTI programs, with further updates on diabetes anticipated next year.

Q&A Highlights

• Safety and Tolerability: Zifdomenib boasts a favorable safety profile, with no significant drug interactions or toxicities, making it an attractive candidate for combination therapies.
• MRD Negativity: The company is pioneering a trial design leveraging MRD negativity for accelerated approval in AML.
• Combination Therapies: Evaluations include combinations with intensive chemotherapy, Venaza, and gilteritinib.

In conclusion, for a deeper understanding, readers are encouraged to review the full transcript below.

Full transcript - BofA Securities 2025 Healthcare Conference:

Jason, Host: Cura. Thank you so much for joining us. Thank you for the invitation and, for for hosting the conference in Vegas again. Absolutely.

Well, maybe just to start broadly for those less familiar with the story, can you provide a brief overview of your your two clinical programs just at a high level? I mean, you know, what is a menin inhibitor and and and how does it work? Yeah.

Troy Wilson, CEO, Cura: A menin inhibitor, Jason, is an epigenetic regulator of gene expression. It does different things in different contexts. And I think I was probably the first person to identify MEN inhibitors in the commercial space now ten years ago. At that time, we thought it was just this very small sliver of acute leukemia, KMT2A rearranged. As it turns out, it’s relevant to probably up to half of leukemia, again, working by a mechanism in that case that differentiates leukemic blasts.

It’s relevant to GIST. We now have a trial running of Zifdomenon in combination with imatinib. There, Menin is actually regulating the expression of KIT. So you’re attacking KIT, which is the key genetic driver of gastrointestinal stromal tumors from two different directions. In diabetes, menin regulates a number of things, including pancreatic beta cell growth and expansion.

And then in solid tumors, you might have seen some of the intriguing data from Scott Armstrong’s lab where and cat six combine to regulate some of the downstream targets behind the estrogen receptor. And so you’re going to see, I think, menin inhibitors play different roles, but there’s always this context that they are working in terms of regulating other genes. That’s the key. And I think this is the gift that keeps on giving, as we’ll talk about, I’m sure, over the next twenty five minutes or so.

Jason, Host: Yeah. Absolutely. Well, maybe just to dive right in, the upcoming ASCO meeting is shaping up to be critical for for CURA with the presentation of the, highly anticipated cifdomenon pitavutal data in MPM one relapsedrefractory AML. So beyond the CRCRH rates, what else do you plan to reveal and what do you think investors should be focused on?

Troy Wilson, CEO, Cura: Yeah, so there’s the Investors naturally focus on typically that which is closest to them. And that’s the dynamic between us and the competition, right? And I think there is a place in the market for multiple Menin inhibitors that’s actually good for patients. I think you’ll see that our data in the specific relapsedrefractory NPM1 mutant space is very competitive with the data that’s been published. A competitor just had a publication in blood just last week.

I think you’ll see we’re very competitive. I think we’re going to differentiate very favorably on safety and tolerability. And the reason I highlight that is that ultimately you’re going to see tomorrow, in fact, our abstract from the European Hematology Association Congress where we’re showing the expansion cohort of ZiftoMenib plus intensive chemotherapy. And their safety and tolerability is important because you can keep these patients on drug. Same thing in the non intensive setting, same thing as you go to other combinations.

So there’s the near term play, Jason. There’s the longer term play. I hope what you’ll take away from that ASCO data is this is a drug that’s active, it’s safe, it’s generally well tolerated. There’s really no surprises. And it looks to combine very well and probably be the market leader across different lines of therapy, particularly as we go into these different combinations.

Jason, Host: Interesting. So you mentioned safety tolerability as a key potential differentiator. Let’s dive a little bit deeper on this. I mean, beyond kind of the CRCRH rates, what would be encouraging here?

Troy Wilson, CEO, Cura: From a clinical activity or safety and tolerability? Safety tolerability. Safety and tolerability. So let’s kind of go down the list, right? These patients in general, particularly in the relapsedrefractory, the elderly setting, this is NPM1.

So these patients are typically 60 years and older. They’re on a lot of con meds and as a result put a premium on the absence of drug drug interactions, and in particular on the absence of dose limiting toxicities. ZIFTO has no clinically meaningful DDIs. It has, as you’ll see, I think no clinically meaningful toxicities to speak of. That makes it very easy to combine.

You don’t have to worry that the patient is going to take something that is going to shift the benefit risk in the wrong direction. The other is, and we’ve talked about this in the past, the absence of cardio tox. Unfortunately, AML has a number of drugs that interact with the cardiac tissue. Everything from the antifungals to the FLT3 inhibitors to intensive chemo. It’s less relevant, Jason, honestly, in the relapsedrefractory setting because you’re really trying to sustain life, to preserve life.

But when you’re talking about frontline, you want a patient on for twelve, eighteen, twenty four months, you want to make it as easy as possible. Something even as simple, just building on that, Zifto is once a day. It’s a once a day drug. We had an ad board meeting on Friday. We had 15 docs in.

We walked through the RevuMenib data, through the ZiftoMenib data. And one of the physicians described Zifto as a cruise medicine. And I said, you know, I thought to myself, what is a cruise medicine? And he said, these patients, these relapsedrefractory patients, keep it in perspective, ten percent of them will be alive at three years. That’s how grave this illness is.

He said a lot of times they want to maintain their quality of life. They want to take a cruise. And a drug that is given once a day, that has no toxicities, that doesn’t interact with anything else, that doesn’t require any kind of monitoring, that’s what I call a cruise medicine. And that’s ideal. I thought that was, you know, this is from someone who’s been doing this for a long time.

I think that’s kind of how you have to think about it. Let’s put the patients first. Yes, we need to be able to address their leukemia, but let’s not make them feel sick if we can. That really should be our goal. And I think ZIFTA will distinguish itself, you know, very well in that regard.

Jason, Host: Makes sense. So CURRA submitted its NDA March 31, where it had breakthrough designation. I guess the two question, two level question here is, how have your interactions with the agency been granted it’s still somewhat earlier? Then if you could speculate on timelines here, when do we get updates and kind of what does the pathway look for throughout the rest of the year?

Troy Wilson, CEO, Cura: Yeah. So we submitted our NDA on March 31. That was three and a half months from Datalog, which is remarkable. And just a shout out to my development team, my regulatory team, my clin ops team. We’ve really tried to close the gap on the competition.

When we submitted it, because we have breakthrough therapy designation, we requested priority review. Now that is at the discretion of FDA. But if they grant priority review, then you’re typically looking at a six month review period. And when we receive notice on whether the application has been filed and we get a PDUFA date, I would expect that we’ll press release that. I actually took your questions in opposite order, excuse me.

I have been, I think speaking for my team, very pleasantly surprised with how proactive, how collaborative, how engaged the agency has been. I cannot say enough good things about them. Again, this may be in part because we have breakthrough therapy designation, and so you get, that’s the real benefit. I think I said that at the time it was granted. When BTD matters is when you’re in regulatory review because the agency prioritizes those applications because of the unmet need.

But we’ve to date seen no interruption, no disruption. They have been just a pleasure to work with. It is early and we continue to monitor. We are doing everything we can to make it easy for them, understanding that they have a lot going on and a lot of applications probably to review.

Jason, Host: Got it. You’ve been doing this for a while. What do you think really kind of factors into the decision for, you know, yes or no via priority review? And I guess specifically, you know, with another menin inhibitor that’s already been approved, does that change the agency’s calculus?

Troy Wilson, CEO, Cura: I’ll say, look, it’s always at the agency’s discretion. I think the fact that there is another menin inhibitor approved but in a different indication, in that case KMT2A rearranged AML where that drug has breakthrough therapy designation, I think the agency’s goal is let’s get these therapies, let’s assess the benefit risk, let’s do a proper review, and then let’s get these to patients as quickly as we can. I don’t think we really expect that there is another product on the market or in review really to factor into the agency’s discussion. It’s likely reviewing them in parallel on different review teams but on parallel tracks. But that’s speculation on my part, as you say.

I’m older. Older.

Cameron, Analyst: So as you look to additional updates from the Frontline seven expansion cohort for the FIT seven plus three group, What should we be expecting in terms of updates here? And then maybe what would be a win in terms of MRD negativity, rates, and maybe duration of response?

Troy Wilson, CEO, Cura: Yeah, Cameron, good question. So you’re going to see the abstract that was done as of a data cut that was months ago. Expect new and updated data at EHA. In that context, maybe I think it’s important to sort of talk about what’s the problem you’re trying to solve. Because I hear when I get questions I hear some confusion.

Seven plus three in NPM1 mutant patients you would expect the complete response rate to be eighty to ninety percent. You don’t want to diminish that. You’re not looking I mean, it’d be great if you could drive 100% all the time. That’s likely not realistic. But you’re looking for high rates of response.

That’s not really where the problem is. The way that I think about it, there’s a reference, there’s a 2018 blood reference, think it is, where they go through the timing, the percentage of patients who go to relapse and the timing to relapse. And the way to think about it is of the patients who respond in the frontline setting, half of them will relapse within two years. And of those, sixty percent will relapse in the first year, eighty percent will relapse by the second year. And so what you’re trying to do with these triplet combinations is to deepen the response to delay the inevitable relapse.

Once they relapse, their life expectancy is three years or less. So to your question, there’s two parts to those, two dual primary endpoints. One is MRD negative CR. The other is event free survival. On the MRD negative CR, importantly, what we’re using is MRD negative CRS assessed in bone marrow.

And the reason for that is, in the literature, this is available, that’s about forty to forty five percent MRD negativity just with seven plus three. So you’re looking to drive a clinically meaningful benefit. As part of the impact consortium, of which Kura has been a member now for several years, that was an industry sponsored effort to work with FDA to evidence the connection between MRD negativity and survival. And the consortium did it on the basis of multiple meta analysis and whatnot, and we got the FDA there. That’s why the FDA was open to that accelerated endpoint.

What I would look for out of this dataset, Cameron, is high levels of response, good safety and tolerability, minimal to no dose interruptions, dose reductions. MRD is going to be an evolving story because it has to be assessed again in bone marrow and ideally centrally. But look at the timing of these patients on therapy and again think about, compare it to the relapse, to where they’re relapsing on seven plus three. What you’re going to see with the KMT2A patients, they almost all go to transplant and then they go on to ZIFTO post transplant. In the NPM1 setting, what is striking is the number of patients who remain on ZIFTOMENOM as monotherapy and continuation therapy for prolonged periods of time.

They don’t go to transplant. That is very interesting and the reason for that of course is if you’re an NPM1 mutant patient, you’re MRD negative CR, transplant is likely contraindicated. It’s not really clear what you’re trying to do. So the ability to offer these patients continuation therapy is really very different from anything we’ve seen before. That also plugs directly into the commercial case.

That’s what makes Menin inhibitors and ZIFTO different from other agents that have come before where, and it’s back to the question Jason was asking, why is safety and tolerability important? It’s important because you want these patients to take this drug once a day and essentially with minimal toxicity or minimal interactions. That’s how I’d think about it.

Cameron, Analyst: Great. And then you’ll receive initial insights into the dose expansion data, so removal of adverse risk criteria. How does that impact expectations or benchmarks for response rates and maybe how should we be thinking about the right comp to look at here?

Troy Wilson, CEO, Cura: Yeah, so in the dose escalation phase at the request of the agency, we escalated in patients who had AML, NPM1 mutant or KMT2A rearranged AML with adverse risk. KMT2A by definition are adverse risk. NPM1 you could get there with one of several different criteria. And at the time, Cameron, I said, probably the best comp is the Vyxeos control arm, intensive chemotherapy in an adverse risk population. That was about a thirty three percent, I think, thirty five percent CR rate.

Let’s be generous and say it’s fifty percent. Showed data at ASH where the NPM1 mutant was 100% CR, the KMT2A was eighty three. I said at the time, don’t get emotionally attached to one hundred percent. You should see robust rates of CR as you now move into the expansion. The expansion is all comers.

All comers means you have about a third of patients who are adverse risk and about two thirds who are not. So now the benchmark in NPM1 is again eighty plus percent. In KMT2A it’s probably lower, call it sixty five to seventy percent. You’re looking to do as good as that. You don’t want to antagonize.

You don’t want to bring the CR rate down. You’re looking to do that or better, and then again, deepen the response, drive a more durable response. We’re going to show you data from the six hundred milligram escalation and the six hundred milligram expansion. And the significance of that is, look at how many patients are still on therapy from the six hundred milligram escalation. And that goes to that question of, can you keep these patients in response without going to transplant for prolonged periods of time?

Save the transplant for later. Give them a higher quality of life. That’s how I would think about it. The MRD and all of that will continue to come into focus. But I can tell you, like all signs on both that one and the non intensive where we’ll show data hopefully in the second half of the year potentially at ASH, you know, at this point all the lights are green to start those phase 3s.

Cameron, Analyst: Perfect. And then just thinking about safety tolerability, what would be encouraging there? And then I know higher dropout rates and myelosuppressive related AEs are not uncommon, but maybe at what level would these be concerning?

Troy Wilson, CEO, Cura: So good, good, good, good question. Myelosuppression is potentially an issue in any combination. The risk there is, as companies present data, focus on the registrational endpoint. For example, in Venaza, ORR is not the registrational endpoint. CRC is not.

It’s CR. You need to get full count recovery. That is your registrational endpoint. So if you have an agent that has myelosuppressive activity, that can be problematic. It can cause dose adjustments, interruptions.

You’ve seen this in the monotherapy data. You’ve seen it in the initial data. Ziptoes are clean as a whistle. It doesn’t exhibit any myelosuppression. There’s no need to adjust the dosing of either chemo or Venaza.

You shouldn’t really see any safety and tolerability. You are going to see treatment emergent AEs, but you will see that those very much sit right on top of what you would expect from baseline disease. So look at it in that context. I don’t think you’re going to see any kind of additive toxicity. That’s certainly one of the key takeaways.

And that will be true in both contexts.

Cameron, Analyst: Maybe if you could speak a bit to the pace of trial enrollment and any investigator feedback you received, maybe based on qualitative feedback, what has interest been like in the trial?

Troy Wilson, CEO, Cura: Yeah. So all the cohorts, I think, are enrolling well. The more data we generate, the more excitement there is. Let’s talk about ’seven and then ’seventeen, which is the trials with the registrational intent. In ’seven, much to our surprise, you know, before we’d ever started, kind of the dogma was Venaza.

Venaza, Venaza, Venaza. And that’s true. There’s certainly a time and a place for Venaza. But the frontline KMT2A patients, they all go to seven plus They all want the prospect of a transplant. That’s their best chance for a cure.

They’re generally younger and healthier. Interestingly, if the older patients have to do seven days of intensive therapy but not go through transplant, they’re more willing to consider intensive chemo, particularly if they can take a menin inhibitor in a continuation setting. You’re seeing, to say that enrollment is going like gangbusters, I think would be the right adjective. And that’s across the board. That’s in both seven plus three and in Venaza.

Now going to the 17 protocol, which is two parallel phase threes, one in intensive, one in non intensive. The feedback that we’ve gotten from sites is this is super smart. It’s like one stop shopping. You can enroll any patient who’s eligible in the frontline with the exception of FLT3 patients who should take a FLT3 inhibitor in the frontline. Anyone else is eligible to come on.

And for sites that have the resources to only be able to do one global phase three, this is attractive because they don’t have to kind of mix and match protocols. This was Molly Leone’s idea. And it’s part of why we did the deal with Kyowa Kirin that we did because we’re now, I think, out in front of the competition trying to basically pick up all the best sites. And once sites in The US, Europe, Asia have experience with Zifto, they become fans, right? They become your advocates.

So that 17 protocol is allowing us, I think, to be very competitive in terms of getting the very best sites for the trial going forward.

Jason, Host: For your chemo combination, you’re pursuing a trial design that would leverage MRD negativity for accelerated approval, a first for AML.

Troy Wilson, CEO, Cura: What did you

Jason, Host: have to demonstrate to FDA to make them comfortable to allow that surrogate endpoint?

Troy Wilson, CEO, Cura: Yeah. So again, I give credit to my development colleagues for this. This is having a lot of forethought. So in ’20, I think it was 2018, I think J and J, for anyone else listening who was a founding member of Impact, I apologize. I think it was J and J that was the founding member.

Impact is M M P A C T, the Impact Consortium. And it has included Bristol, Celgene, I think Novartis at one time, Cronos. And it was an effort, Jason, between industry and the agency to do a meta analysis to help substantiate the link between MRD negativity, not mutant specific, but generally with survival. So there’s published data, there’s unpublished data. When we did the briefing documents for the phase threes and the question came up from the reviewers, well how are you going to handle this?

We were like, well we’re members of the Impact Consortium. They’re like, oh, okay. A lot of that work had been done. I give credit to the heavyweights who were there. I would not be surprised if one of those who is a Menin competitor chooses to take a very similar design because they have all the data we have.

FDA is data driven. And there was some consternation around the recent appointment of Doctor. Prasad at CBER and comments that were made in the context of multiple myeloma. I think there the issue was these patients are potentially living ten years. Are there safety concerns with accelerated approval?

In AML, we’ve talked about it. The unmet need is staring you in the face, right? And so the ability to get these therapies patients on an accelerated basis is very attractive to the agency. And you of course have the built in safeguard that you have EFS for the intensive and OS for the non intensive. And if you don’t hit on the survival based endpoints, you’re going to lose the accelerated approval.

And that should happen relatively quickly as these things go in development. That should provide the safeguard to patients.

Jason, Host: It’s a good segue for the next question. But turning to the second half of this year, you’ve guided to presenting data from COMET seven frontline non intensive AML. So this would be a ZIFTO plus Venase combo. Ideally, what do you think a menin inhibitor can provide on top of Venase? Is the former just eliminating an escape mechanism, or is there potential for kind of a synergistic improved CR?

Troy Wilson, CEO, Cura: There is potential for an improved CR. I would be hand waving if I told you I knew exactly what’s driving that. And I think we’ve commented on this publicly. We’ve seen evidence where menin inhibitors, zyftomenib in particular, appears to resensitize patients to venetoclax. I don’t know that I can tell you why that’s happening.

That seems to be happening. That’s what we’re hearing from the docs. So in that context, Jason, in contrast to the intensive chemotherapy, there your CR rate for NPM1 is about fifty to sixty percent. KMT2A, take it down another five to ten percent from that. You can make a clinically meaningful improvement in CR, which is why you don’t need MRD negativity in the non intensive setting.

And I would say I think the non intensive update will be a meaningful update. The only thing you and investors have seen to this point is really safety in the relapsed refractory setting in a dose escalation. We now have expansion cohorts in both the frontline and the relapsed refractory setting with Venaza and look forward to sharing that data later this year. I think you’ll see that it’s meaningful.

Jason, Host: Got it. Looking at COMET eight, so this is the phase one evaluating ZIFTO plus Phlegida, LDAC, and more importantly, I think, gilteritinib. Any updates you can provide there?

Troy Wilson, CEO, Cura: Just that the enrollment continues, dose escalations continue, there’s good interest. It isn’t really yet a focus this year on presenting data. You called out guilt, and I think that’s appropriate. It’s important to remember that in the relapsedrefractory setting, half of your NPM1 mutant patients will have a FLT3 co mutation likely. Part of the dynamic is not necessarily us versus our Menin competitor, it’s Menin inhibitors versus other out of class competitors.

So the option of publishing data that potentially combines gilteritinib and zifdomenib, I think, provides a really interesting option. Stay tuned. My team tells me regularly, Troy, everything can be a priority. It just can’t all be a priority at the same time. Fair.

You you know, you

Jason, Host: you bring up sort of an interesting point. I think if you talk to most docs, Veneta seems to be the combo partner that they point to in terms of opening up use in the frontline setting. Do you think that’s still the case?

Troy Wilson, CEO, Cura: I think it’s evolving. I think that people have been trying to put seven plus three out of business for forty years, thirty years, maybe. The thing is, it really works. And it’s seven days. And venetoclax is a remarkable therapy, but it is not a walk in the park.

It has some challenging toxicities. I think you want to give docs options. It’s interesting to see, this is, let’s go back to your very first question. These monotherapy studies and hopefully the approvals, the academic or the physician community views them as significant proof of concept of the mechanism and a license to combine as they see fit. They know all these agents.

They’ve worked with them. They all talk amongst one another. Imagine they’re on like some WhatsApp chat or something. And so giving them data and allowing them, you know, if you look at the practice patterns of MD Anderson versus Memorial, they look very different. You want to give physicians the options with what they feel most comfortable is the right thing for their patients.

And let’s let the data, let those regimens compete.

Jason, Host: Got it. Well, brief time that we have left, I did want to give an opportunity to discuss the partnership that you mentioned earlier with you. Kiran, in light of everything that’s happened, what made this partnership make sense to you strategically and financially?

Troy Wilson, CEO, Cura: Yeah, so particularly, it was, I think, met with some questions when we initially announced it. Now that every other day is some new headline risk in biotech, the ability to stay in a very strong financial position, to go toe to toe with an organization like Janssen, and to really move Zifdomenib aggressively into these various lines of therapy. Think our goal is maximize the value of Zifdomenib initially in AML for as many patients as possible. But Jason, it also allows us to make the investments in GIST, in diabetes, and in our FTI program. And you’re going to see clinical updates again throughout this year for the FTIs, potentially next year for GIST.

You’re going to see updates on diabetes. So you have a number of different value drivers. I have told my team, and I told investors in our one on ones today, said, Don’t plan on going back to the equity markets ever. I want them to be prepared, but we will end the year in a very similar cash position to where we started. And I think that puts us in good stead to be able to create value for patients and ultimately for investors.

Sounds good. Troy, been a privilege and

Jason, Host: a pleasure. Thank you so much for joining us.

Troy Wilson, CEO, Cura: Thank you both. My pleasure.

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