Q1 2025 Kura Oncology Inc Earnings Call
Please standby your program is about to begin if you need assistance during the conference today. Please press star zero.
Good day, everyone and welcome to today's first quarter 2025 care oncology financial results Conference call.
At this time all participants are in a listen only mode.
Later youll have the opportunity to ask questions. During the question and answer session. You May Register to ask a question at any time by pressing star one on your telephone keypad, you may withdraw yourself from the queue by pressing star two.
Please note this call maybe recorded and I will be see anybody if you should need any assistance.
Speaker Change: It is now my pleasure to turn the conference over to that poor virtually please go ahead.
Speaker Change: Thank you operator, good afternoon, and welcome to Cara Oncology first quarter 2025 conference call. Joining me on the call are Dr. Troy Wilson, President and Chief Executive Officer, Dr. Molly Leoni, Chief Medical Officer, Brian Powell, Chief Commercial Officer, and Tom Doyle Senior Vice.
Speaker Change: Didn't our finance and accounting before I turn the call over to Dr. Wilson I'd like to remind you that today's call will include forward looking statements based on current expectations such statements represent managements judgment as of today and may involve risks and uncertainties that could cause actual results to differ materially from expected results. Please refer.
Speaker Change: <unk> filings with the SEC, which are available from the B C or on the core oncology website for information concerning risk factors that could affect the company.
Troy Wilson: With that I'll turn the call over to Troy.
Troy Wilson: Thank you Paul Good afternoon, and thank you all for joining us over the past quarter, we've made substantial progress executing against our pipeline, we've achieved important clinical and regulatory milestones, which is clinically and financially de risked our preparations to commercialize just a minute and acute myeloid leukemia or <unk>.
Troy Wilson: Including the recent NDA submission for <unk> as a monotherapy in relapsed or refractory <unk> mutant AML.
Troy Wilson: The ZIP demented monotherapy phase II Registrational data were accepted for oral presentation at the 2025, <unk> annual meeting, which will be the first of multiple clinical data updates, we anticipate presenting at major oncology medical meetings throughout this year.
Troy Wilson: Earlier this week, we announced the first patients with advanced gastrointestinal stromal tumors or just after imatinib failure were dosed with the combination of just a minute but imatinib.
Troy Wilson: Our STI program continues to advance and we expect to share preliminary clinical data from multiple phase II cohorts. Later this year evidenced seeing the potential of F. T I S as combination therapies.
Troy Wilson: As we continue to build correct into a fully integrated organization. We're pleased to announce the appointment of Samir that Tom Pat him to our leadership team as senior Vice President Global program leadership Sameer.
Troy Wilson: Sameer joins <unk> with more than 20 years of experience in the biotech and pharmaceutical industry, including extensive leadership of global program teams, which has driven the development and commercialization of 16 novel medicines and indications across 13 diseases, primarily in oncology and hematology.
Troy Wilson: As adjusted for the $45 million milestone payment under our collaboration agreement with Kyowa Kirin upon receipt by FDA of the NDA submission for <unk> <unk> on a pro forma basis $703 $2 million in cash cash equivalents and short term investments as of March 31 2025.
Troy Wilson: Accounting for the $45 million milestone payment, we stand to receive an additional $375 million in near term anticipated milestones. This strong cash position provides sufficient capital to fund our ziff to men of AML program to commercialization in the frontline setting as well as advance our pipeline.
Troy Wilson: To multiple value inflection points with that overview, let's now dive in starting with just a minute.
Troy Wilson: On March 31, we submitted our new drug application or NDA for just a minute our once daily oral investigational <unk> inhibitor.
Troy Wilson: A reminder, <unk> is the first and only investigational therapy to be granted breakthrough therapy designation for treatment of relapsed or refractory NPM one mutant AML.
Troy Wilson: There are no FDA approved therapies targeting NPM, one mutant AML, which represents approximately 30% of new AML cases annually and has a disease of significant unmet need.
Troy Wilson: Priority review was requested which if granted would provide a target FDA review period of six months from FDA from NDA acceptance.
Troy Wilson: <unk> has been a collaborative and supportive partner and although it's early days in our review cycle, we've not experienced any disruptions or delays due to the changes underway at health and human services or F. D. A.
Troy Wilson: We look forward to continuing to work closely with the agency throughout the review process and are optimistic about the potential for ziff demanded to impact patients with relapsed refractory <unk> mutant AML with that introduction I'd like to turn it over to Molly to walk through updates to our development pipeline and programs Molly.
Molly Leoni: Thank you Troy.
Molly Leoni: First let's start with I was just a matter of development program earlier. This year, we announced common 001 or phase two registration directed trial of <unk> in patients with relapsed refractory MTN one mutant AML had achieved its primary Crs CRH endpoint and we're pleased the data will be shared in an oral presentation.
At the upcoming 2025, <unk> annual meeting in Chicago.
Molly Leoni: Benefit risk profile for <unk> in this patient population is highly encouraging and the safety profile remains consistent with data shared previously we believe the combination of favorable safety and Tolerability profile and clinical activity in this once daily oral medication support a competitive profile in the relapsed refractory setting.
Molly Leoni: As well as clinical development and the critical frontline indications our plan is to socialize the data among scientific and medical communities in the U S and Europe between the oral presentation at <unk> as well as an encore presentation of the data at the quality twenty-five Ehow Congress ahead of potential marketing a protocol.
Molly Leoni: Moving to the combinations, we continued to see robust enrollment in both the comment.
Molly Leoni: Kevin.
Molly Leoni: Zero eight studies, which are evaluating <unk> in combination with various standards of care. We're pleased.
Molly Leoni: To announce the preliminary clinical data from the phase one expansion cohorts evaluating the combination of stuff doesn't matter with intensive chemotherapy in the frontline setting has been accepted for an oral presentation at ehow.
Molly Leoni: And we anticipate presenting preliminary clinical data from that.
Molly Leoni: Oh, seven phase one expansion cohort evaluating <unk> in combination with another clock and they decided data in a frontline setting in the second half of the year.
Molly Leoni: These will be important updates as they will help inform the safety tolerability and potential clinical activity of the triplet combinations and directly inform the design and conduct of the phase three frontline trials under the comments zero 17 protocol.
Molly Leoni: In addition, we announced last quarter, we had reached alignment with FDA and EMA for the design and conduct of this trial, notably we also announced we had broken new ground to use I'm already negative CR, let's see our respectively as primary endpoints for accelerated approval in the United States and the intensive and not intense.
Molly Leoni: A chemotherapy frontline trials respectively.
Molly Leoni: As a reminder, the global protocol comprises of two independent randomized double blind placebo controlled phase III trials to evaluate <unk> in combination with both intensive and non intensive combination regimens in patients with newly diagnosed <unk> mutant <unk> empty to Avery arrange to AML.
Molly Leoni: We've received feedback from institutions investigators and their study teams.
Speaker Change: Your line of comment Oh, 17, with two independent phase III trials under a single protocol is very attractive to clinical sites because it simplifies trial startup conduct and provides a single option to nearly all of their eligible frontline patients.
Speaker Change: Zero 17 is now in study startup.
Speaker Change: On track to initiate the studies in the second half of this year.
Speaker Change: Turning our attention to solid tumors earlier this week, we announced the first patient dosed in a phase one comment zero 15 trial to evaluate the safety tolerability and preliminary ex tumor okay anti tumor activity and it's just a matter of in combination with imatinib in adults with just who are currently on our previously.
Speaker Change: Been treated with Imatinib therapy.
Speaker Change: Approximately four to 6000, new cases of jets are diagnosed each year in the U S advanced gist patients have limited treatment options.
Speaker Change: The frontline standard of care regimen in these advanced patients as Imatinib, which targets kit inhibition. The challenge is most patients eventually develop resistance to imatinib due to the development of secondary kit mutations Teekay is such as Sunitinib target in that neck resistant genotypes and her proven later lines.
Speaker Change: Sponsorships and long term outcomes are modest for new therapeutic options are needed.
Speaker Change: <unk> offers potential to shift the treatment paradigm in jest, although we could certainly pursue development and advanced gist patients who have failed imatinib treatment. This novel mechanism of action potentially permit system that up to prevent resistance from that and then in the frontline setting and approach which builds on the strengths of them that in of itself.
Speaker Change: And is not addressed with current therapies.
Speaker Change: The dosing of the first patients marks a significant milestone to develop a new combination treatment to protect us to potentially improve outcomes and address a meaningful unmet need for just to patients. We look forward to sharing clinical updates as it becomes appropriate.
Speaker Change: Moving from ours, if the amount of development programs for F. T. I development programs, we continue to make significant progress in the fifth 001 trial evaluating our next generation for natural transferase inhibitor Kao 20, 806, as both monotherapy and importantly in combination our approaches a revolutionary one.
Speaker Change: For F. T. I's are used in combination with targeted therapies to either blank or overcome resistance and potentially drive deeper and more durable responses reshaping the F. T I story.
Speaker Change: We expect to share combination data for 20, 806, plus cabozantinib and renal cell carcinoma in the second half of 2025.
Speaker Change: Also anticipate sharing data from the phase one monotherapy dose escalation of 20th or six in patients with Ras mutations in the second half of 2025.
Speaker Change: We are pleased to report the momentum continues in the fit Oh, one trial and we expect to initiate one or more expansion cohorts of 20, 806 and cabozantinib in patients with advanced renal cell carcinoma in the second half of 2025.
Speaker Change: We also continue to evaluate the combination of 28, six and <unk> in patients with K Ras G 12 C mutant solid tumors and finally, we are pleased to share that the current H N phase one trial that was evaluating kept a foreigner in combination without Palo said in patients with recurrent or metastatic pick for ECA dependent.
Troy Wilson: And next squamous cell carcinoma is now close to enrollment we anticipate sharing clinical data later this year and are evaluating the next steps for that combination and the program and with that I will turn it back over to Troy.
Troy Wilson: Thank you Molly regarding the.
Troy Wilson: The dynamic macro landscape in which we're now operating we continue to monitor developments and remain vigilant to the rapidly evolving situation and we're prepared to adapt as needed at this time, we believe the impact from tariffs would be negligible and Additionally, all of our intellectual property is domiciled in the United States.
Troy Wilson: Importantly in this challenging market, our strategic partnership with Kyowa Kirin provides us with greater development commercial and operational resources as well as increased financial stability, our partnership and cash resources enable our team to stay focused and execute on our pre commercial and launch preparation efforts as well as our efforts.
Troy Wilson: To create a clinically meaningful impact in other areas such as just renal cell carcinoma, and other solid tumors I'll now turn it over to Tom to provide the first quarter financial highlights. Thank you Troy and good afternoon, everyone collaboration revenue from our Kyowa current partnership for the first quarter of 2025 was <unk>.
Troy Wilson: $14 $1 million compared to no revenue in the first quarter of 2024.
Troy Wilson: Research and development expenses for the first quarter of 2025.
Troy Wilson: $6 million compared to $36 3 million for the first quarter of 2024.
Troy Wilson: General and administrative expenses for the first quarter of 2025 were $22 8 million compared to $18 2 million for the same period in 2024.
Troy Wilson: Net loss for the first quarter of 2025 was $57 $4 million compared to a net loss of $49 5 million for the first quarter of 2024.
Troy Wilson: This included noncash share based compensation expense of $7 $8 million compared to $8 5 million for the same period in 2024.
Troy Wilson: As of March 31, 2025, correct had cash cash equivalents and short term investments of $658 2 million compared.
Troy Wilson: Compared to $727 4 million as of December 31, 2024.
Troy Wilson: As adjusted for the $45 million NDA submission milestone payment under our collaboration agreement with Kyowa Kirin.
Troy Wilson: <unk> had on a pro forma basis, $703 2 million in cash cash equivalents and short term investments as of March 31 2025.
Troy Wilson: Based on our current operating plans, we believe that our cash cash equivalents and short term investments as of the end of the first quarter will be sufficient to fund our current operating expenses into 2027.
Troy Wilson: If we include anticipated collaboration funding and milestones under the <unk> agreement Curtis financial resources should support advancement of our system in an AML program through commercialization and the frontline combination study.
Troy Wilson: With that I will turn the call back over to Troy.
Speaker Change: Thank you Tom before we jump into the question and answer session. Let me lay out our anticipated upcoming milestones for <unk> and our Menin inhibitor programs. We look forward to presenting full data for comment 001 in Q2, 2025, and an oral presentation at <unk> in an encore presentation ADR present preliminary.
Speaker Change: Eliminate clinical data from the comment zero-zero, seven phase <unk> expansion cohort evaluating <unk> with intensive chemotherapy or seven plus three in the frontline setting at Ehealth.
Speaker Change: Presenting preliminary clinical data from the comment 007 phase <unk> expansion cohort evaluating <unk> with <unk> decitabine in the frontline setting at a medical meeting in the second half of 2025, initiating comment zero 17, two independent phase III registration, enabling trials in <unk>.
Speaker Change: Frontline intensive and non intensive AML in the second half of 2025 and nominating a development candidate for a next generation Menin inhibitor program in diabetes in mid 2025.
Speaker Change: For our Farnesol transfer Ace inhibitor programs, we expect the following milestones.
Speaker Change: Initiate one or more expansion cohorts of K O 2860, <unk> cabozantinib in patients with advanced renal cell carcinoma in second half 2025.
Speaker Change: Data from the fit 001 phase one trial evaluating 2008 O <unk> and Cabozantinib in patients with renal cell carcinoma in the second half of 2025.
Speaker Change: Present data from the fifth 001 phase one monotherapy dose escalation of $28 six in patients with Ras mutations in second half 2025, and finally to present data from the current HN trial evaluating <unk> in <unk> mutant head and neck squamous cell carcinoma in the second half of 2025.
Speaker Change: With that Jess we're now ready for questions.
Speaker Change: Thank you Sir at this time, if you would like to ask a question. Please press star one on your telephone keypad.
Speaker Change: You may remove yourself from the queue at any time by pressing star to.
Speaker Change: Once again that is star one to ask a question.
Speaker Change: We will move first <unk> with Cantor Fitzgerald.
Speaker Change: Hi team this is Dan on for Lee.
Speaker Change: Congrats on the progress and the milestone payment.
Speaker Change: Can you maybe put the expectation for the combo data coming later this year, especially the eight milligram cohort.
Speaker Change: What do you need to show up to be competitive.
Speaker Change: It looks like some of your competitors have kicked off.
Speaker Change: Three trial here.
Speaker Change: Just update on that as well what youre thinking about in terms of initiating the frontline trial.
Speaker Change: Thanks.
Speaker Change: Yes, thanks, Dan for the questions.
Speaker Change: So just just to recap so.
Speaker Change: What are we looking for from the Asia than cohort and then really.
Molly Leoni: Timing of initiation of that trial relative to the competition Molly would you like to take downs questions.
Speaker Change: Sure.
Speaker Change: I'll remind you we're always looking for in especially in these preliminary data presentations.
Speaker Change: Safety is our first second and third priorities. So they about ability to safely combine ours. After Amanda with does that is that in the frontline.
Speaker Change: <unk> is what we really want to establish them.
Speaker Change: And obviously now being able to finally see the data in these frontline patients will be able to see some some additional characteristics about time on treatment.
Speaker Change: Overall patients responses to therapy.
Speaker Change: So we look forward to being able to show you the combinability of lift in that with these agents that can be difficult to combine with it.
Speaker Change: The there was to be any added toxicity or any adverse events on combination.
Speaker Change: And then with respect to your question around timing, we remain on track to start the study in the second half of 2025.
Speaker Change: Okay. Thank you.
Speaker Change: Sure.
Speaker Change: We'll go to Jonathan Chang with Leerink partners.
Hi, guys. Thanks for taking my questions.
Speaker Change: First question can you discuss how the changes at the regulatory agency has been.
Speaker Change: Packet or not the potential approval processes and timelines for Cisco.
Speaker Change: Yes.
Speaker Change: Sure.
Jonathan: Yes go ahead, Jonathan go ahead, I'm sorry, the second part no no go ahead.
Speaker Change: The second question.
Speaker Change: Can you help set expectations for the upcoming <unk> presentation of the comments zero, one results and how should we be thinking about what will be in the abstract versus the full presentation.
Speaker Change: Yes.
Speaker Change: Yes, thanks, Jonathan Thanks for that so as for.
Speaker Change: Any any changes underway at FDA.
Speaker Change: As we said in the prepared remarks, we were not seeing any any impact at all.
Speaker Change: The agency has been responsive collaborative <unk>.
Speaker Change: <unk>.
Speaker Change: So we're not seeing any effects at all.
Speaker Change: We requested priority review will be able to give guidance, we would expect to receive notification from FDA on whether the application has been accepted for review in the Paducah date here in the second quarter and we can communicate that at that time, but at this point, it's business as usual I'll just add.
Speaker Change: Jonathan recall that we have breakthrough therapy designation in NPM, one mutant AML and as we said at the time. This is when that designation matters because.
Speaker Change: Agents that have breakthrough therapy designation generally get a higher priority and more resources within FDA because of course, they are directed at significant unmet needs.
Speaker Change: And needs for which there is no available therapy, and so whether or not that's playing into the fact that we're not seeing any any impact at all.
Speaker Change: I can't say, but I think we feel today pretty good about where we are with FDA with respect to the second part of your question about the monotherapy data.
Speaker Change: Again, we've answered this question I think many times the CR CRH rate will be between 20, and 30% will also look at time on treatment safety and Tolerability all the key considerations youll see much of that articulated in the abstract.
Speaker Change: <unk> presentation, we'll have a more fulsome dataset of course.
Speaker Change: Because you can go much beyond the abstract the timing of those two.
Speaker Change: B as of the same data cutoff, but of course, the oral presentation will have more data of course relative to the abstract.
Speaker Change: Got it thanks for taking my questions.
Speaker Change: Sure happy to.
Speaker Change: We will go next to Salim Sayed with Mizuho.
Speaker Change: Great. Thanks for the question and the commentary today, Troy and team I guess.
Speaker Change: One just for.
Speaker Change: And your upcoming expected launch and then the NPM one setting.
Speaker Change: Lot of people I think are thinking here that at least in terms of.
Speaker Change: CR rates here.
Troy Wilson: These two men and inhibitor molecules seem more or less the same just curious what your updated market share work is sort of telling you Troy how these are going to.
Troy Wilson: Sort of get used once if and when they are both on the mark in the relapsed refractory setting.
Speaker Change: Yes, let me Slim it's a good question, let me comment and then I would actually ask Brian to maybe.
Brian Powell: Add his thoughts.
Troy Wilson: Obviously.
Troy Wilson: Let's have that conversation I think I understand why youre asking the question.
Troy Wilson: We keep emphasizing safety tolerability clinical activity. It is the whole package, it's important as a mono therapy becomes increasingly important as one goes into combination. We have also have of course done internal demand studies I don't think we've ever made that public.
Troy Wilson: But we do believe that system and it will be very competitive in the relapsed refractory setting and.
Troy Wilson: We're obviously seeing very strong.
Troy Wilson: Enrollment in the combo settings.
Troy Wilson: Which I think again speaks to its ability to treat.
Brian Powell: More patients and ideally get them too to better outcomes overall, but Brian anything you'd add.
Brian Powell: Thank you you answered that thank you for I think you answered that pretty well I would just maybe add.
Speaker Change: As you said, we've been doing our demand study just trying to get a better understanding of where we see the potential profile differences between.
Speaker Change: It has to come forward and I think that what we're seeing is that this will be a competitive space as we expected but.
Speaker Change: Well, we have a very good.
Speaker Change: Confidence I think in the strength of our profile that we will be able to be competitive in that monotherapy launch.
Speaker Change: We're going to put the resources behind to support that as well.
Speaker Change: Yes.
Speaker Change: And to that point. This is one of the advantages of our partnership.
Speaker Change: We know we're going to be competing for patients right.
Speaker Change: This is this is it's an area of high unmet need it will we think be a competitive dynamic, but we are planning and we haven't will provide greater color on this as we get later in the year, but Brian and his team are working very thoughtfully with our colleagues at Kyowa Kirin and we expect to bring.
Speaker Change: The resources and the focus to bear on competing for every single patient so.
Speaker Change: And we're doing that ideally in the in the relapsed refractory setting but also.
Speaker Change: Ultimately, we do we are optimistic that you're going to see use of these men and inhibitors in earlier lines of therapy and in combination. So theres a lot of education to be done.
Speaker Change: Okay perfect. Thanks, so much.
Speaker Change: Of course.
Speaker Change: We'll move next to Jason Symanski with Bank of America.
Cameron: Hey, Tim This is Cameron on for Jason Congrats on the update and thanks, so much for taking our question.
Speaker Change: So connecting the dots from some of your earlier comments as you look to additional combination data from <unk>.
Starting with the <unk> population.
Speaker Change: In your view.
Speaker Change: From the seven plus three combo, specifically on response rates and came to two HAE and Amgen one.
Speaker Change: How does that change now that we should see data in non adverse risk patients and then as you start to get additional insights into MRV negativity, what would drive confidence here and a potential pathway to accelerated approval Francisco ultimately.
Speaker Change: Yeah. That's a really good question Cameron, let me ask Molly if she can if she can speak to us.
Speaker Change: Sure.
Speaker Change: As we've shown our frontline.
Speaker Change: David in the past week for obviously staying really high.
Speaker Change: High rates of response, even in the adverse risk posted it it would be hard to improve upon the 100% response rates, we were seeing them, nor do we expect to maintain that perfection.
Speaker Change: But throughout the literature, what we've seen is that in these patient populations you expect to see anywhere from a 60% to 90%.
Speaker Change: CRC rate, so you're maintaining and not interfering with the efficacy of the back Brian hopefully augmenting. It of course is the goal.
Speaker Change: How do you augment oh are right that's that impressive well you don't.
Speaker Change: Exactly target the LR right as you referenced we're looking at other science as well.
Speaker Change: Most of these patients will become relapsed refractory within a year youre going to see data on patients that are approaching a year's worth of therapy. So that you can get an idea of.
Speaker Change: Maybe the durability of these patients' responses to this combination of therapy.
Speaker Change: Obviously, we will be showing mardi data as well, however, I'm not sure if we will be able to show a centralized version at the time of the presentation. So it'll still be a mix of both.
Speaker Change: Both bone marrow and peripheral blood, which will make interpretation slightly more difficult but of course in bone marrow, you expect maybe 40% of patients to achieve <unk> negativity.
Speaker Change: So that should be.
Speaker Change: Anchoring point for looking at the data.
Speaker Change: Great. Thank you.
Kamran: Thanks Kamran.
Kamran: We'll go next to Charles Zhou with lifestyle capital.
Charles Zhou: Hey, everyone. Thanks for taking the question I'll start off by saying congratulations on us submitting your NDA and NPM, one AML interesting timeline relative to your competitors S. NDA, even though the hit topline once before you did but any way to answer the question.
Last one actually on for Nigel transfer Ace inhibitors regarding steel tornadoes six how do you envision a longer term in combination development with <unk> in RCC, just just given the movement in that space with Nextgen <unk>, plus checkpoint inhibitors and how do you see.
Charles Zhou: 20% to six fitting into that as it shapes out longer term. Thank you.
Charles Zhou: Yes, Charles Thanks for the question Molly do you want to.
Molly Leoni: Do you want to answer Charles question about how we see 28 of <unk> fitting into the evolving RCC landscape.
Molly Leoni: Of course, so I've been as you pointed out there are thankfully new therapies coming into this.
Molly Leoni: Area of high unmet need, but none of them are going to be curative.
Molly Leoni: And so until there's actually a cure there's always going to be the need for additional therapies, we see the combination of twentyish datasets with Cabo.
Molly Leoni: To be highly synergistic with the way physicians are currently comfortable trading.
Molly Leoni: So they will be able to make use of a drug they're comfortable with what they've seen high rates of response with and hopefully with the addition of 28 six they'll see deeper longer and higher rates of response for these patients. So we see a very big.
Molly Leoni: Potential for 28 or six to really augment the current treatment paradigms.
Got it great. Thank you.
Charles Zhou: Thanks Charles.
Molly Leoni: Yeah.
We'll go next to Alan Horn with TD Cowen.
Alan Horn: Great. Thank you for taking the question.
Speaker Change: Congrats on the NDA Super exciting.
Speaker Change: Couple of questions what is tier one growth strategy to capture the share of the MTM one market.
Speaker Change: It goes second to market position and what points of differentiation do you think will resonate most with our investors.
Speaker Change: Yeah.
Speaker Change: So maybe I can.
Speaker Change: Maybe I can take issue just with the thesis of the question, it's not obvious to us at the moment that will be second to market.
Speaker Change: We've disclosed that we submitted our NDA in March admittedly end of March let's call. It end of Q1, we requested priority review and we have breakthrough therapy designation.
Speaker Change: We're just as you are waiting to see kind of where the where our competitors are in terms of their submission timelines and regulatory review I think under any scenario. It is going to be very very close that's just our operating hypothesis. If you go back not that long ago I was hearing from people that we were a year.
Speaker Change: Behind her two years behind.
Speaker Change: <unk>.
Speaker Change: Kudos to our development and regulatory teams for closing that gap.
Speaker Change: Unfortunately, there isn't a large prevalent population of relapsed refractory <unk> mutant patients I wish we wish there were but that actually speaks to the significant unmet medical need these patients progress and unfortunately passed away within days or weeks, if you don't get them on therapy.
So what I think where youre going to end up is a competitive dynamic where physicians have choices.
Speaker Change: And.
Speaker Change: Choice is always good for patients and as Brian said in his prepared remarks too to the earlier question, we're going to Mount a significant we lead commercial.
Speaker Change: Commercial strategy, and we and killer Kiran and working together, we're going to be fighting for every patient and we're going to be looking both to promote within the relapse refractory setting and then to educate about the potential for Menin inhibitors in earlier lines of therapy, and <unk> and in combination that's why you see.
Speaker Change: Our comprehensive approach to us.
Speaker Change: Moving zipped amended to what we believe will be first in class and best in class throughout the treatment continuum. This is the first step is it's an important step and we're feeling pretty good about it.
Speaker Change: Yeah.
Speaker Change: Thank you Dan that's helpful.
Speaker Change: Yes pleasure.
Speaker Change: Sure.
Speaker Change: And once again it was star one if you would like to ask a question. We will move next to Peter Lawson with Barclays.
Okay. Thanks for taking my questions.
Speaker Change: Sure.
Speaker Change: <unk>.
Speaker Change: Thanks.
Speaker Change: What patient segments do you think you're going to be more responsive to the to the combination in particular subgroups you are going to be focused on weather is imatinib refractory or naive.
Brian Powell: So Peter that's actually one of the beauties of this program, but I'll, let Molly fill in the details. That's that's a great question and it actually I think you've put your finger on sort of what is potentially so transformational about this MLA.
Molly Leoni: Molly Yes, that's exactly that's exactly what I was thinking Troy is that that's what makes this approach so revolutionary and.
Molly Leoni: And potentially transformative is that it will be a mutation agnostic. So we do not have to worry about.
Molly Leoni: Taking the appropriate combination partner for each you know each line of therapy.
Molly Leoni: Using it with Imatinib.
Molly Leoni: Whether the patient has is currently on Imatinib or has already failed imatinib.
Molly Leoni: Previous additional previous lines of therapy, the combination should still work.
Molly Leoni: You know we effect was different than it does with the weak FX they kit transcription and the imatinib affects the kit stability. So overall these highly dependent cells are then deprived of what they need to continue being oncogenic.
Molly Leoni: Two different mechanisms and.
Molly Leoni: I said that should be independent of what the kit mutation is so we would of course starts to kiss show ourselves that it works in.
Molly Leoni: The second line plus setting, but we hope to move into the frontline setting as well cause.
Molly Leoni: We really think we could build a superb adjunct to imatinib therapy, which physicians are already so comfortable with on which patients tolerate so well.
How do you think thats kind of plays out does that play out.
Molly Leoni: Oh, <unk> PFS and kind of what are the goals I think.
Molly Leoni: The go forward decisions around those.
Molly Leoni: Yeah.
Molly Leoni: Yes.
Molly Leoni: Yeah, so as as we've looked at our preclinical data, we think it'll be a combination of both.
Molly Leoni: We think that we'll see it's very rare you see a complete response and adjust to patients.
Molly Leoni: So, we're hoping to see potentially deeper responses longer lasting responses and again, if we see a response in a patient where a madden. It has failed has now failed or is starting to fail.
Molly Leoni: Can save that response that that's also a very clear sign that this potential mechanism on trulia synergistic and that's what these patients need.
Molly Leoni: Perfect. Thank you so much.
Speaker Change: And Peter just I just wanted to pick up on something that Mollie said and this is in response to your question as well as Charles's question. What you see as you look across our portfolio as we're taking these targeted therapies and ideally layering them on to standard of care that is either generic or will be.
Speaker Change: Generic by the time, we get to market, so chemotherapy in AML potentially cabozantinib and RCC imatinib ingest that that if we can take.
Speaker Change: Opportunities, where the market is very well understood. Its physicians first choice and we can make a claim that we can actually do better for your patience.
Speaker Change: And you don't you have the ability to drive premium pricing as well because you're not layering on top of a novel another novel therapy, we see that as a really compelling opportunity across both liquid tumors and solid tumors. So we haven't talked much about the pipeline. There has just been so much focus on.
Speaker Change: On Ziff do but that is the theme that you are beginning to see come together and we will have much more to talk about this in the second half of the year and on into next year.
Speaker Change: Perfect. Thank you so much.
Speaker Change: Sure.
Speaker Change: We will go next to David <unk> with UBS.
David: Oh, great. Thanks for taking my questions and I also want to add my congratulations to a great quarter.
David: Just focusing on the phase III <unk> trial.
David: Some of the gating steps before you're able to initiate the second half.
David: And then just more specifically around that so how are the site activation scoring.
David: Wonder if some early reception from trial investigators on the use of <unk> profile that could really accelerate your initiation in those in that trial.
David: Yeah, David Thanks for the question I'll, maybe I'll take the first half and then I'll ask <unk> to speak to what we're hearing from from sites and investigators.
David: Said, we're in steady start up there's a lot that has to happen for a global phase III contracting budgeting as you say site initiation, we haven't been more granular on our guidance beyond saying its second half. The team is doing everything it can to pull those timelines in but as I like to say it's it's.
David: First patient in matters last patient in arguably matters more so.
David: So the team our team I think increasingly has shown an ability to execute at a very high level.
David: Development regulatory clinical operations, hopefully soon to be commercial.
Charles Zhou: We'll give you more color and around these trials as we get into the second half of the year in terms of what we're hearing about <unk> in the context of almost 17, let me ask Molly if she can speak to some of what she and her team are hearing from investigators.
Molly Leoni: Sure I mean really we have such a good preview of 17 from our Oh seven trial, but.
Molly Leoni: But it gives us a real good insight into how both the treaters and the patients are feeling and one of the things we hear most often is that it.
Molly Leoni: They don't realize they're taking another medication.
Molly Leoni: They feel you know.
Molly Leoni: The same or better than they did on the backbone therapies. So we don't really see that theres going to be much opposition to including.
Molly Leoni: With the addition of this amendment within these backbone therapies as well and there's a lot of excitement we get contacted directly from people that want to participate and I think as we share more data from the <unk> seven trial, the excitement will only build so it's all been very positive so far.
Molly Leoni: Yeah.
Molly Leoni: Okay. Thank you so much.
Molly Leoni: Thanks, David.
Molly Leoni: We'll move next to <unk> <unk> with BTG.
Speaker Change: Great. Thank you for taking the question and congrats again on the NDA submission.
Speaker Change: So my first question was we do hear anecdotal off label use of other currently approved <unk> inhibitors in relapsed refractory NPM one patient. So my question was what do you think is perhaps needed to pull clinicians who are perhaps familiar and comfortable with one agent over to sift out following potential approval. Thanks.
Brian Powell: Yes, Jay Thanks for the question, let me ask Brian if he can speak to it.
Speaker Change: Thanks, Steve.
Speaker Change: So.
Speaker Change: I've heard and we've seen reports around some of the competitor agents, having some off label usage of MTM, one I think to what choice point earlier is that there may we anticipate there may be some experimentation in use of.
Speaker Change: Other therapies in this setting, which I think demonstrates the very high unmet need in this space.
Speaker Change: To the point that Troy made earlier, we don't anticipate that there is a large pool of patients that are just waiting.
Speaker Change: For a therapy to be available so.
Speaker Change: If there are patients that are currently being prescribed another menin inhibitor.
Speaker Change: We think that there will still be patients as we get as we move forward to approval that will still be available for us and I think our objectives and goals will be able to appropriately communicate the risk benefit profile of our therapy relative to other competitors. So that we can understand and give physicians the information they need.
Speaker Change: What we understand and we think maybe a favorable profile relative to others. So I think on the one hand I am glad that.
Speaker Change: Physicians are excited about the introduction of <unk> inhibition in the treatment.
Speaker Change: The other I think that gives us a good position for us as we.
Speaker Change: <unk> for a launch to kind of.
Speaker Change: Be ready too.
Speaker Change: Go out there and get every patient possible to put on in just a minute.
Great. Thanks, maybe just a quick follow up with all that's going on in the FDI franchise between RCC.
Speaker Change: The RAF mutations and head and neck as well.
Speaker Change: Are you looking to advance all of these indications in parallel or what will it take to make you have a go no go decision.
Speaker Change: With all these into account.
Speaker Change: Yes.
Speaker Change: A very very good question and very forward looking thank you.
Speaker Change: Just so this goes back to a comment that I made I made earlier after Peter's question.
Speaker Change: It's important that biopharma companies of our size have a compelling sort of standalone proposition that one can finance and execute on independently.
Speaker Change: And then if you can add on to that and drive increasing.
Speaker Change: Uptake in revenue that's great what is there.
Speaker Change: That means.
Speaker Change: We think we'll be in a position if if the data supports moving forward into RCC and <unk>.
Speaker Change: Admittedly. It's early days, we haven't shown you any clinical data you've seen I think pretty compelling preclinical data clinical data coming in the second half of the year. If there is an opportunity we could arguably do that do that alone because by that by the time, we reached the market. The partner compound in this case cabozantinib would be.
Speaker Change: Likely generic.
Speaker Change: That's not true for <unk>.
Speaker Change: That may be true for for example, <unk> kinase alpha inhibitors, or <unk> inhibitors, there youre more likely going to need to partner with either a large company or a small company to find a path forward and I would I would put that in the bucket.
Speaker Change: G. A nice to have I don't think you want to have your companies.
Speaker Change: Strategy necessarily dependent on our partner.
Speaker Change: I think you want to have that as as optional and really high commitment from both parties, but what youre seeing again, we're in a great place with Kyowa Kirin in AML I think we'll look forward in the future to sharing data and just with you. We'll talk about FTE is we are trying to identify those.
Speaker Change: Products.
Speaker Change: Product opportunities Aida combinations, which Kirk can take forward.
Speaker Change: On its own or in the case of Ziff dough with Kyowa Kirin, that's how that's at a high level G. How we'll think about prioritizing. It below that then you ask what are the best agents, who are the partners. How do we think about working together, but it will be very much a <unk>.
Speaker Change: Starting with what is best for care for our patients and for our shareholders.
Speaker Change: Okay.
Speaker Change: Why don't we go to the next.
Speaker Change: George Farmer with Scotiabank.
George Farmer: Alright, thanks for taking my questions.
Speaker Change: So.
Speaker Change: Troy given that you do have BTG with.
Speaker Change: With Ziff, though in M. P M one disease.
Speaker Change: Since you have a good feel about whether you can get priority review by now.
Speaker Change: So do you think.
Speaker Change: You'll need.
Speaker Change: That FDA will want to convenient Kodak meeting and.
Speaker Change: Also regarding.
Speaker Change: The co primary endpoint in your phase III.
Speaker Change: <unk> negativity and CRC H, you need to hit both of those in order.
Speaker Change: For a win or can you just hit one of the other thanks.
George Farmer: Yes, George I'll take the first two questions and ill let Molly.
Speaker Change: The endpoint questions.
Speaker Change: We can't look we've said, we're feeling good there've been no disruptions the agency has been responsive.
Speaker Change: I think at this point, we've said as much as we can say next stop is hopefully the dossier is accepted for review and were assigned to <unk> date, we'll communicate that when it happens.
Speaker Change: Your second question was forgiven.
Speaker Change: Oh the O deck.
Speaker Change: I think it would be unusual I don't think were expecting a no doc here. Given this is a palliative setting given the agencies expressed a willingness for to grant full approval for agents in relapsed refractory AML and given George quite frankly that the safety and Tolerability profile tamales Paul.
Speaker Change: <unk> really.
Speaker Change: I think I think is very strong in the case of just a minute.
Speaker Change: Never say never but I don't think we're anticipating a no Doc Martin.
Molly Leoni: Molly do you want to.
Speaker Change: I don't know if you want to add anything modulator to my first two answers, but could you speak to Georges questions about the endpoints on the phase III.
Molly Leoni: Sure No I mean, I think your first two answers are spot on we will find out about priority review when they formally accept the dossier.
Molly Leoni: As for the co primaries, what I want to correct, if they're not they're not actually co primary endpoints their dual primary endpoints.
Molly Leoni: So even if you for some reason did not win on your accelerated endpoint. So you see our <unk> negative CR you could still win and the overall trial with the survival based on point, so that being the E. S. S. I think so.
Molly Leoni: So you can hit both or just the overall survival endpoint and still have a win.
Okay. Thank you.
George Farmer: Yeah. Thanks George.
Molly Leoni: Yeah.
Molly Leoni: We will go next to Roger song with Jefferies.
Speaker Change: Great. Thanks for the update and taking my question so the <unk>.
Speaker Change: First is.
Troy Wilson: Classification, maybe Troy.
Troy Wilson: I Misheard that you said you asked co abstract.
Troy Wilson: <unk> will be the same data. So just can you just come from that and then also you read at Schuh.
Troy Wilson: Or <unk> the first line combination pivotal.
Troy Wilson: How should we think about enrollment given you will assume half of that.
Troy Wilson: Approve the drug in the yard.
Troy Wilson: And then as soon as if demanded but won't be a proving that R&R steadying, how should we think about that the pivotal first nine year old man.
Troy Wilson: Yes.
Speaker Change: Yes, Roger Thank you for the questions. So.
Speaker Change: Yes, the abstract in the presentation, we will have the same data cut.
Speaker Change: And as I said the full presentation is a more expansive tour through the data as far as your second question Roger around the dynamic between a potentially approved ziff demanded product and the frontline trial. They are really there really shouldnt be well, let me put it this way there shouldnt be a drag.
Speaker Change: AG on enrollment.
Speaker Change: Enrollment in the frontline trials, because obviously, that's a patient population for which no menin inhibitor as approved and as Molly indicated in her answers to a couple of questions. We continue to see very robust enrollment in both the intensive and non intensive setting I think maybe the reciprocal question of <unk>.
Speaker Change: Will there be an impact of the frontline studies on the relapsed refractory setting.
Speaker Change: I don't think we know the answer to that we will be watching for that.
Speaker Change: But I think it's too early to say at this point.
Speaker Change: Whether they will have any impact of frontline development on.
Speaker Change: The availability of.
Speaker Change: Patients in the commercial setting in the relapsed refractory.
Speaker Change: Excuse me in the relapsed refractory setting.
Speaker Change: Yes, that's fair thank you.
Speaker Change: Thank you.
Speaker Change: It appears that we have no further questions at this time I will now turn the program back over to Troy Wilson for any additional or closing remarks.
Speaker Change: Thank you Jess and thank you all once again for joining our call today, we will be participating at several medical and investor conferences over the next couple of months, including <unk> and look forward to seeing many of you there.
Speaker Change: We also plan to host a virtual investor event following our oral presentation at <unk>. So look for more details in the coming weeks in the meantime, if you have any additional questions. Please feel free to reach out. Thank you and have a good afternoon and good evening everyone.
Speaker Change: Okay.
Speaker Change: Thank you Sir this does conclude today's program. We thank you for your participation you may disconnect at any time.
Speaker Change: Okay.
Speaker Change: Oh.
Speaker Change: Okay.
Speaker Change: Yes.
Speaker Change: [music].
Speaker Change: No.
Speaker Change: Yeah.
Speaker Change: Uh-huh.
Speaker Change: Uh huh.