Good day. My name is Chad, and I'll be your conference operator today. At this time, I would like to welcome everyone to Curio Farm Therapeutics Phase III seal study results conference call. There will be a question and answer session follow. Please be advised that this call is being recorded at the company's requests.
I would now like to turn the conference over to Mr. Ian Karp, Caryopharm's Senior Vice President, Investor And Public Relations. Please go ahead.
Thanks so much, Chad, and thank you all for joining us today's conference call to discuss the results from the Phase III deal study presented this morning at the Connected Tissue Oncology Society 2020 annual meeting. This is Ian Carpenter, but I'm joined today by Doctor. Michael Kaufman, Chief Executive Officer Doctor. Jatin Shah, Chief Medical Officer and Doctor. Sam Chawla, Director of the Sarcoma Oncology Center in Santa Monica, California.
On the call today, Doctor. Kauffman will provide some opening remarks about the phase 3 seal study and results. And then Doctor. Ciao will review the key findings from the study, which represented for the first time this morning at the CTAS 2020 virtual end meeting.
We will then open
up the call for Q And A, for which we also will be joined by Doctor. Rinnell Gaunder, Sarcoma Service, and developmental therapeutic service at the Memorial Sloan Cancer Center, at Memorial Sloan Cancer Center, who is also a lead investigator in the seal study. Please note that Doctor. Chaula and Doctor. Downer are non employees of Keriopharm and have been invited to participate on today's call as they were both investigators in the study and experts in the field of sarcoma.
Their comments and opinions shared today are completely their own and do not reflect official statements from Karyo Farm Boyer Institution. Earlier today, we issued a press release detailing the results from the Phase III study. This release as well as an accompanying slide presentation are available on our website at karyopharm.com. Before we begin our formal comments and for those following along on the slide presentation, you'll see on slide 3, I'll remind you the various remarks we'll make today constitute forward looking statements for the purposes of the Safe Harbor provisions under the Private Securities Litigation Act of 1995. These include statements about future expectations, clinical developments, regulatory matters, timelines, potential success of products or product candidates, including our expectations relating to the commercialization of Expedia.
Actual results may differ materially from those indicated by these forward looking statements as a result of various important factors, including those discussed in the Risk Factors section, of our most recent quarterly report, which is on file with the SEC and in other filings that we may make with the SEC in the future. Any forward looking statements represent our views of today. While we may elect to update these statements at some point in the future, we specifically disclaim any obligation to do so, even if our views change. Therefore, you should not rely on these forward looking statements, as representing our views as of any date subsequent to today. With that, I'll now turn the call over to Doctor.
Michael Kaufman, Chief Executive Officer.
Thank you, Ian, and good afternoon, everyone. We are delighted today to discuss the positive pivotal results from the phase 3 seal study, which met its primary endpoint with a significant increase in progression free survival in patients with unresectable de differentiated lymphocycoma. This is Karyopharm's first positive pivotal study in a solid tumor indication, and we believe represents a major advance to the development and commercial potential of Xpobio in oncology. We are tremendously excited about the results presented virtually this morning in an oral presentation at the CCOS annual meeting and believe they are particularly encouraging because advanced de differentiated liposarcoma remains a very difficult to treat cancer with no established standard of care and limited treatment options available to patients with heavily pretreated activity in a randomized trial in patients with previously treated liposarcoma. Before we discuss the CEL study in more detail, I'd like to take a moment to briefly provide some perspective on the foundational and unique approach we are taking toward innovating cancer drug therapy.
On Slide 4, We have highlighted 5 core pillars of cancer drug therapy, which are all foundational and used across tumor and patient types often in combination to give each patient the best chance at clinical improvement. Xpobio is the first approved anti cancer drug whose primary activity is the activation broadly of tumor suppressor proteins. There are about 20 of these proteins, including P53 BRCA-one and 2, retinoblastoma protein and the inhibitor event of Kappa B, called I Kappa B, which are a critical part of each cell's own natural defense tendism to prevent the generation of cancer cells and to direct a cell, which has become cancerous, to commit suicide. This broad anti cancer mechanism is potentially relevant to any malignancy. Our goal is to continue to demonstrate the impact that Expevio can have in combination with drugs from all of the other pillars in order to maximize the impact that Xplovio can have on improving outcomes for our large variety of patients.
Turning now to Slide 5. Our clinical strategy for Expobio continues to be to innovate with a purpose. Specifically, Our approach has been to demonstrate meaningful activity in different, in difficult to treat patient populations and then to expand dramatically. Into earlier lines of treatment and additional tumor types particularly in combination with other anticancer drugs. This strategy has been evident in our approach in both myeloma and DLBCL, where we began our successful storm and sales studies and has led to subsequent development in earlier line in combination settings.
Similarly, now that we have seen a positive Phase 3 seal data, we plan to follow the same path and continue and expand our clinical development approach in a number of important and much larger solid tumor indications, including endometrial cancer, with the ongoing Phase 3 SIENDO study, lung cancer, glioblastoma multiforme and non melanoma, which have been selected based on encouraging clinical data and commercial potential. If you'll please now turn to Slide 6. Here we have an overview of soft tissue sarcomas, which are a diverse group of rare malignant tumors that originate from the mesenchymal cells within the soft tissues, including fat, muscle, nerves and others. As outlined in the left pie chart, liposarcoma, which occurs in the body's fat cells, is the most common subtype and accounts for approximately 20% of all sarcomas. According to the National Cancer Institute, in 2020, About 13,000 new cases of soft tissue sarcoma will be diagnosed in the U.
S. And over 5000 Americans will die from this disease. Common first line treatments for sarcomas include radiation, surgery and chemotherapy. Advancing now to Slide 7, We have a deeper we'll have a deeper dive into the current treatment paradigm for de differentiated liposarcoma, which is a high grade type of liposarcoma that grows more aggressively than a low grade, well differentiated liposarcoma. D differentiated high grade liposarcoma is associated with a poor prognosis.
The median overall survival of these patients is unfortunately just 2.5 to 5 years. For patients whose disease is not fully resectable with surgery, First line treatment is typically chemotherapy regimens, including doxorubicin, a loaner in combination with thiophosphamide, but response rates are typically low in essentially all patients will need additional treatment options. In patients whose disease progresses, following frontline therapy, typical treatments include Trebectin, where previous studies have shown a 2.2 month median PFS and iribulin, which has been shown to have a 2.0 month median PFS in patients with BDLS. On Slide 8, we highlight that most sarcoma patients in the U. S.
Are treated as centers of excellence have particular expertise in treating patients with these types of tumors. There are about 90 sarcoma alliance affiliated centers in the U. S. And the seal study included patients at 26 of these sites throughout the country. In fact, the seal study was the largest trial ever in patients with de differentiated liposarcoma.
J. Espovio ultimately receive FDA marketing approval in DDLS. We believe we could effectively commercialize in this setting with a very small tailwind sales and marketing effort, primarily leveraging our existing infrastructure. I'd now like to ask you to move to Slide 9, where Doctor. Charla will detail the results from the CEL study.
Doctor. Charla?
Good day, everybody. I hope, this COVID crisis is soon over and we have a good better political climate, but we are very excited to hear the presentation for our patients who suffer from sarcoma. So a de differentiated liposarcoma. Sarcoma or all cancers are like criminals in the body and low grade tumors, which is like a well differentiated liposarcoma, is like a low grade criminal like, pick pocketing, home robbery and other things, while the differentiated part is like a gang not only it damages the organ where it is, but its metastasizes goes all over the body. So this drug is the first drug.
We did a very rigorous, randomized study in multicenter, largest number of patients, and a unique drug which is not chemotherapy, which we have only used. Just to, declare that I'm not employee of the company as stated before, I don't have any conflict, and I will devote 25 years of my life in taking care of the sarcoma patient. I'm participant to this study as well as I have played a pivotal role, in approval and a study of the previous drugs approved in liposarcomasets as trabectedin and arabulin. Now I'm on Slide 10. Celestino, as stated by Doctor.
Kaufman demonstrated anti tumor activity in the cell lines preclinical studies. And in phase 1 study, it showed impressive response of 40% as shown in the graph on your right side with the multiple patients. Now I'm moving to Slide 11. This was, the largest study in 2.85. Which is almost $300,000,000 to $1,000,000 randomization.
That means, almost 200 patients receive the actual drug and 97, which is almost 100 patients received the placebo. Patient had to show that the tumor was increasing by the radiological means, which was confirmed by the blinded radiologists. That means they didn't know they were not blinded. They were obviously blinded from the results. And then the scans were performed on progress and confirmed progress and then the label was opened and if the patients were on placebo were crossed over to actual drug.
The drug was oral, given 60 milligram twice a week. Now I'm moving to next slide, which is, slide 12. And inclusion criteria were standard that confirmed diagnosis, progressive disease within 6 months And patient must have received at least 2 prior treatments. The standard treatment are doxorubicin and other chemotherapy but maximum over 5 and standard lab criterias to be enrolled in this study. A patient were allowed even some mild renal insufficiency and creatinine clearance up to 30 ml per minute because this drug does not cause any nephrotoxicity.
Slide 13 shows the characteristics of both patients who are in Selects in our group and placebo group. And as you see, they are reasonably well matched for age facts, race, geographical area, performance status, and site of disease and majority, as you see, 80% were in retroperitonian or in abdomen. And as you see in both groups are about 70 percent head metastatic disease. And next slide, which is Slide 14. And again, continuing prestics of the patients, which were both missed, both in selexinor group, as well as in placebo group.
Coming to the Slide 15 shows the randomization, the 285 patients 187 to the, actual drug. And it the patients the drug was well tolerated in majority of the patients, but was discontinued in 57% in the digit because of digits progression, while in 69%, which is about 12% more because of the disease progressing in the placebo group. Rest of the things are similar as shown here. Currently, 18 patients are continuing on the active drug on the selexinab. Next.
This is the Kaplan Meier Curve, which shows the slight separation of the curve and showing there was a significant PFS in Celestinor 2.8 which we expected at 2.7 prior to the study start and it made the criteria while the placebo PFS was 2.07 with the 2 sided P value of 0.02. Hedged ratio of 0.7. That means 30% risk reduction in the relapse. This is significant, met the criteria, and it is similar PFS in the previously approved drug like Trabectodine and Eribule. Next on the Slide 17, it shows the overall survival, which is similar and these growth at multiple places.
All the numerically, selection or survival was 10 months and placebo was 12 months. And you, as we have said, they did cross those patients who went on placebo did get select signal. So it's not a appropriate comparison, but there's This is also not statistically significant. We are on the Slide 18 which shows overall survival, again, in the patients who were initially only received selection or whether to start with or on the placebo. And as you say, selection or group had a survival of about 10 months versus Placebo, which is 9 months.
Again, it is not statistically significant. Overall response is shown in the slide 19. And as you see, the RECIST criteria sure that a 2.7% of the patient in selection or achieved partial remission, which is a definition more than 30% reduction in the tumor. But if you took the overall group, which is more than 15% reduction in the tumor, was in 7.5%. While those 97 patients in placebo, there was no patient showed either more than 15% reduction or no no more or, no patients showed more than 30% reduction.
So there was no resist criteria response in placebo group. This slide of 20 shows the overall response, who did not crossover during open label. And finally, the 5.3% patients who achieved the response more than 50 let me say again. More than 15% reduction in the what's seen about 5.3% of the patient who the selection or after closing over and 3.5%, which is similar showed partial remission more than 30% reduction in the tumor side. Overall side effects were well tolerated substantially less than any of the chemotherapy.
There were no drug related deaths. And if you see the grade 34 in the right hand column, were in less than 10% and predominantly contained nausea and vomiting. Other mild side effects noted included 40 and dysguusia, which is the status change in the test. Next slide. Serious side effect like anemia thrombocytopenia was rare and anemia was in 8.2% which we do not, which is very mild as compared to any chemotherapy I'm sorry, in 18% in Sallaxinor group and 8.2% in the placebo group.
And it's mostly related to the disease rather than the drug, but of course, mild anemia and thrombocytopenia, neutropenia. Can occur with the drug in less than 10% to 20%. Next slide. So income In conclusion, this is the 1st novel drug, which is an Xpro-one inhibitor targeted drug which has shown, effective and de differentiated liposarcoma. We do not have any other drugs specifically approved for this disease, although for liposarcoma, we use Doxorubicin trajectory and and arabulin.
Overall activity of this drug was similar to them, and it is an oral drug. Future of the drug, this drug can be combined with other drugs and as stated earlier, this drug has a unique mechanism of action, so it can be combined with chemotherapy, immunotherapy, and is already approved in myeloma and a non Hodgkin's lymphoma. So if you have any questions, I'll be happy to answer and thank you for your, participation in this call.
Thank you, Doctor. Charla. And with the positive CL data now in hand, we look forward to submitting a new drug application the U. S. Food And Drug Administration during the first quarter of 2021, requesting approval of Xphobia to treat the patient population studied in seal.
If approved, Xpovio would represent the 1st oral non chemotherapy agent available for patients with de differentiated liposarcoma. Beyond liposarcoma, as we think about other solid tumor indications on Slide 25, We show areas that we plan to explore. As I mentioned earlier, we are already collecting data from our non small cell lung cancer We have less than a multiforme and colorectal cancer studies and have begun to explore additional settings where we'd like to initiate new studies.
We plan
to explore Expobio either alone or in combinations in a wide variety of lines of therapy and clinical settings in the future including in ovarian endometrial GBM, non small cell lung cancer, colorectal cancer and melanoma. Ultimately, We view Xpobio as a novel tumor suppressor activator and a potential pan cancer drug that could become an important option for patients across both across both blood cancers and solid tumors. We see Xpobia's greatest utility as a combination partner with other anticancer drugs and dose only once per week in these combination settings. With that, I'll now turn the call back over to the operator
Thank you so you. At this time, And the first question will come from Brian Abrahams with RBC.
Hi, this is David Ciesou on for Brian. Congrats again on these data and initial success in solid tumor I guess just to start, you mentioned, development in, several solid tumors planned, imminently perhaps next year. Guess I'm just looking for more color here and maybe could you provide some color on where and how you might prioritize across these solid tumors, as you think about factors such as mitigating, a trial performance in this current environment with increasing COVID lockdown or any regulatory considerations, as you engage the FDA with liposicoma data? Thanks.
I think for the COVID lockdowns, hopefully, the vaccine will get out relatively quickly and we'll be able to relieve some of the stress going on. But the fact is that these tumor cells keep dividing even in the pandemic and these patients are in dire need of additional therapy and pretty much all of the solid tumor lines that we, areas that we spoke about. The current ongoing studies are well supported by phase 1 data from the company and or investigator sponsored trials that have been going on and where we're privy to some of the early data from those studies. And that's what led to the initial trials that we're carrying out right now in non small cell lung cancer in combination with docetaxel. The combination with pembrolizumab and CRC and the ongoing studies that we have in glioblastoma multiforme now move from 2nd and third line into, 1st and second line.
For additional tumors, we have demonstrated in phase 2 studies, single agent activity in ovarian cancer, and we've also demonstrated single agent in melanoma and this melanoma activity was further strengthened by the investigator sponsored trial results presented this year at ESMO. From MD Anderson showing what appears to be at least additive activity of selinexor plus KEYTRUDA in frontline melanoma. So basically, we're looking at the clinical data that are available to us and additional data that are not public and we will be making decisions on going forward for the company sponsored trials, including on the size of these markets and the overall unmet medical need. I don't know of any particular regulatory specific hurdles that are in place at this time as essentially all of these tumors remain uncurable.
Next question.
Got it. Thank you.
And the next question will come from Maury Raycroft with Jefferies. Please go ahead.
Hi, everyone. Congrats on the updated data and thanks for taking my question. First one is just on dosing strategy. So in hemonc, I think part of the strategy there is to get patients to a response And then you can optimize dosing for the patient and manage tolerability. Do you think the dosing used in seal is in line with what will be used in the real world liposarcoma or is there some optionality possible in the real world setting?
And then for Doctor. Tawala, I was wondering if you can talk more about combo dosing strategy that might use in the real world setting and what data from CO can potentially help guide how to combine other drugs?
Okay, sure. Let's start with Doctor. Chuller because he may have things on that. Yes. If Doctor.
Chuller, are you still able to be on? I'm not sure.
This drug is an oral drug, long half life, given twice a week. So this is very easy to combine all other drugs. Since the side effects are minimal of this drug systemic toxicity of myelosuppression, or immune toxicities minimal, I think it will be a very well combination effort and can be easily combined And the real question will be that if you can see if this drug could be more efficacious and more synergistic, although our hope it will be because this is a unique mechanism of action.
Great. And briefly, Doctor. Shah on Jatin, maybe you can speak to our, the MD Anderson study. I think we're doing combination work with arabulin there.
Yes, exactly. So we're developing combination data. I mean, we're studying pre clinically, there's data in combination with various agents. In the clinical setting, there's ongoing kind of investigator sponsored studies both looking at selinexor whether that may have been GIST So sarcoma subtype, as well as with aribulin, ongoing ISTs, and we're exploring other options as well.
Got it. Thank you for taking my questions.
And the next question will come from Peter Lawson with Barclays. Please go ahead.
Great. Thanks for taking my questions. Just maybe for Michael initially, just your thoughts on the PFS that kind of dropped in phase 2 to phase 3. Is that anything we should be concerned about? Is that just kind of a larger end number or is there anything else we should be thinking through?
Yes. I think our assessment, a couple of points are very important. And the first one is, is that the PFS of Travected was reported at 2.2 months in an earlier population. The PFS of Aribulin in an earlier population was reported at 2.0 months, So here now with the 1st non chemotherapy, oral therapy, we're talking about a 2.8 month BFS, which doesn't sound like a lot perhaps, but when you have this devastating disease and you're in 3rd line with no options, this is pretty, interesting that you could have a PFS that's in the range or higher than what's been reported in earlier therapy. And I'll turn it over to Doctor.
Gauner in just one minute. I think, the move from phase 2 to 3 is just the numbers patients and the more robust data set that we have now. Doctor. Galler, do you want to add anything?
Yes, absolutely. I'd like to echo a lot of the comments you just made. If you just look at this is really heavily pretreated patients who mostly, they've all had docs Ruberson, they've had erobulin rejected and now coming in in the 3rd line, to the 5th line, to the 5th line setting. When we look at the retrospective data from many, many institutions, Sloan Kettering and De Anderson, Europe for just the de differentiated liposarcoma patients in the first line setting, in the first line setting with docs Ruberson, the PFS is 1.5 to 2 to 2.5 months. These are all like in that ballpark.
Even with combinations like, say, docs Rubicinplusiposimide, the PFS gets a little better, but the vast majority of the median PFS in the first line setting with docs service and based agents is somewhere between 2 to 4 months at most So in context of that, it's really surprising and very promising that in the 3rd line setting in patient who failed all these drugs, selinexor is performing just as well as Daphs Ruberson, which frankly is really came to me as a big surprise. And I'm very encouraging, to say at least.
I would like to add the comment to Doctor. Gounder's comment. When we talk about the improvement in the PFS, about a month or a little less, it doesn't mean that everybody gets that much benefit only. There's obviously big range and we have to decide some parameters as a PFS for improvement. If this was in the financial world, where everybody gets the, I mean, some people get rich, some people lose money, the, and that's how cannot be decided, but there are I treat the patients.
So there are patients who can get long term benefit for months. And some patients even on trabected and arabulin. And even this drug go for a year or 2 years. Of course, those are the minority of the patients. We just can't define at this stage, but that's what we are looking.
And so the average improvement in a for about 4 weeks does not mean everybody only gets improvement in 4 weeks.
Thank you.
Thank you.
Yes.
And if I just reiterate that, I think the other point, the point another way to look at it is that the tail of these curves is, is that we're approximately doubling the people with the long term benefits, which is quite important to those folks. And it's reminiscent frankly of other novel therapies where the medians are perhaps less affected than an important minority of patients who have a real, real benefit that otherwise would be left without therapy.
Great. And then a follow-up for the doctors on the line. Just whether you could potentially use this,
I
guess, either in earlier line or outside the differentiated liposarcoma just your thoughts there based on the strength of the data you've seen?
Yes, no, if I could jump on that question. I mean, yes, the study was designed in the 3rd line and beyond setting. But when you look at the totality, of the PFS benefit, it's in line similar to drugs in the second line and even the first line setting. So personally, when if I were to see a patient, there are many patients. I mean, you can see here that many of these patients were 65 years of age the median entry is 65% and 10% of patients were seventy five years of age.
These are patients who are have other comorbidities that could preclude drugs like docs Rubison. And we can only give 6 cycles of docs Rubison, then we have to stop for cardiotoxicity and then switch to something else. So I think in a case by case basis, one can make the, argument to use this in an off label setting for an individual patient and of course, have that conversation with patient. I personally think patients would preferred a drug like this, given that it had no neutropenic fever, had an oral dose inconvenience compared to a classic cytotoxic chemotherapy patients are always asking, are there oral tails for convenience or are there drugs that can, that are targeted? I mean, here, we don't know the exact, precise target, but I think a lot of that work will hopefully future work will show that even within this study itself.
So in summary, I do think that there, that we're not losing. We don't have to wait till the 3rd, 4th 5th line before we think about this drug. I don't think I will be thinking about it in the event that this gets approved by the FDA.
Perfect.
Thank you so much.
The next question will be from Jonathan Chang with SVB. Please go ahead.
Hi guys. Congrats on the data and thanks for taking my questions. First question for Doctor. Chala and Doctor. Grauer, can you help us contextualize the survival trend that wasn't stat sig in this gangster like disease setting?
Yes, I can take that. If you look at the overall survival, I think there is numerical differences, but the hazard ratio is solidly 1. And a lot of it is probably related to the crossover of about 60% of patients crossing over. So The, there is a survival trend, but, I'm not sure how much we can And we definitely see this hazard ratio, but I'm not sure how perfectly those arms are balanced or if they are randomized. I think it is what it is.
I'm not sure we can, like, say a whole lot about it. I don't know if Doctor. Chawla or Michael or someone else wants to add more, to that particular one.
Well, I can just give some context on the regulatory discussions. The point of the overall survival endpoint given the crossover because we all know that crossovers, especially given that these are rapid crossovers, typically less than 2 months, on the placebo arm crossover. Is that you muddy the waters downstream because you end up with a selinexor versus selinexor study. The point of the regulatory endpoint was to sure there was no inferiority here. So the fact that we came in with a hazard ratio of 1 or just slightly below 1 is great news.
And as Doctor. Gallagher said, when you actually look at the patients who did not cross open a selinexor, and you can do all sorts of sensitivity analyses, which have been done, but obviously couldn't be included in the short talk today. You end up with this at least trend that suggested getting selinexor at any time is better than not ever getting selinexor. The P value is about 0.12or0.14 and we'll have to wait over time as more events accrue. But at least it's trended in the right direction to suggest that getting some selinexor is beneficial.
Doctor. Charlie, did you want to comment?
No, I echo both comments of Doctor. Grounder and Doctor. Kaufman. Great.
Thank you. 3 doctors had agreed. That's pretty scary.
Just second question for me. Can you talk about how the adverse event profile in seal compares with the experiences in liquid tumors?
Yes, maybe I'll ask Doctor. Sean on our side to take that, Jason.
Yes, absolutely. Great question. So I mean, I think the first key point is that the side effects that we see with Selinex are all very clearly dosed and scheduled dependent. As well as the disease dependent. And so, what we see with, compared to the initial storm in myeloma, we have much lower doses with this.
And so obviously it's going to be better targeted, but also if we keep in mind that some of the we saw with storm, for example, was in 7th line therapy with multiple my plus of regimens, a very, very different patient population and dose, which is in that setting as we're starting to use these with lower doses, in earlier lines of therapy. It is better tolerated. We've seen improved silica profile qualitatively when we see lower grade lower incidences and lower intensity of side effects. Importantly, even when we compare to the same dose of 60 milligrams from the SABL experience with similar dosing schedules, we see a better side effect profile in these patients with sarcomas. Again, they've had less myosuppressive therapy less moment involvement from pain malignancies.
So, as we move into the solid tumor space, what we see a better side effect profile of less now is
a little bit far Yeah.
And then as the future goes, you know, obviously we'll continue to move forward to weekly in combination. It's an outlook to kind of change the static of profile that we see.
Got it. Thank you.
Next question?
Yes. The next question is from David Lebowitz with Morgan Stanley. Please go ahead. Thank you very much for taking my question. When you look at these results, I guess, how does it translate to the broader soft tissue sarcoma population, does liposarcoma results typically translate to these other indications and do physicians typically use them off label in these other indications?
Maybe Doctor. Gallagher and
then Doctor. Kachella?
No, I can take that question. I mean, I think the short answer is yes. I think there there are there is a value for selinexor in soft tissue sarcoma, and we haven't yet found exactly who those patients are. But We did have a very nice hint of activity in a phase 1 study that was published in the JCO, maybe about 3, 4 years ago, where we saw, you know, it was an all comers sarcoma study, and we clearly saw activity in D. Diff life liposarcoma, but we also saw activity in Lyomyosarcoma, MPNST, and a couple of other things.
And there's really, if you open up that paper and look at it, you'll see pretty impressive, swimmers plot. But the reason I think at that point, we didn't really sort of go broad is we really want to have a focused question and sort of a focused regulatory pathway. Some of the other you know, other sarcomas such as Laiya Mya sarcomas have had like specifically other drugs approved. So we felt that from a straightforward regulatory pathway and to keep the study really clean, we'll focus on Didip liposarcoma What was also interesting is in that study, we saw some signal in something called MPNST or malignant peripheral nerve sheet tumor, And this is not yet published. It's currently under review, but we're, surprisingly, I've seen in my own clinic several patients responding to selinexor, which we are using as a single patient, single patient use.
So I think as this drug becomes more and more gets into our hands, there will definitely be, people evaluating in different sarcomas. But a lot of that is going to be dependent on sort of like data, even if it is a small case series and things like that.
And the next question is from Arlinda Lee with Canaccord. Please go ahead.
Hi guys. Congratulations on the data. I had a couple of questions for Doctor. Gounder and Talwa. You guys have both worked on other drugs and other registrational trials.
I was curious, 1, what proportion of our conversations are de differentiated? And how do you like if a patient comes in, what kind of drives your decision on whether to give the patient what therapy to give them, and how might this fit into the treatment paradigm?
Doctor. Charla, you want to go first?
Yes. Overall,
and we have very limited therapy. Patients when they come with sarcomas, first thing is surgery if they are localized, if they are metastatic disease, standard chemotherapy and then other chemotherapies. And standard chemotherapy is doc service in our fosumide, which are substantially more toxic. And most patients will not respond. Then we move to the 2nd line, which are like, or clinical trials.
So there's a big unmet need in these diseases as far as the liposarcoma makes about 20% of all sarcoma and the differentiated liposarcoma makes another 20% of those. So it's a rare disease, but unmet need for, for any effective or promising drug. And, that's why we are excited about Celestino.
Yes, I can just piggyback off that comment. I think everybody in our field is, has a knee jerk reflex to use Docs Rubicon, and we've been using it now for 4 decades. But I think only but we've never really sort of dived into the specifics of how good is Doxorubicin in combination drugs. Specifically for DIF LIFO. And it doesn't look great.
I mean, and I think our field as a whole is knee jerk reflex to use Docsolution single agent or in combination in the upfront setting, we will need to think more carefully But so to answer your question, I don't think any of these single drugs improve overall survival. So I think a lot of this is you can one should really have a personalized approach to an individual patient and not just have this algorithmic knee jerk reflex of docs Rubicon and then this and then that. I think we really need to sort of. And I think our field, we are aware of it, but there is a certain sort of this condition response to Doxorubicin and other drugs. But I think that will that will change in the very near future.
Could I ask a follow-up? Since somebody mentioned off label use since it's already, selinexor has already approved in myeloma, has that been a possibility for Have you tried to use this off label for other patients before? Thanks.
So as I mentioned, we've tried to use it off label for MPN ST. It really came as a single patient to use program that carrier form has. To be honest with you, like, there are people who work in the solid tumor world they will not there's a, there's a huge barrier to start using drugs in the heme world. First, most people kind of don't really crisscross between the 2. So many people may not even be aware if you're just taking care of solid tumor patients, about drugs in myeloma lymphoma.
That's especially true like in the bigger institutions. So I think what if And when there is an indication in a solid tumor and there is safety in solid tumors, the the sort of the threshold to try it in an off label use really goes down a whole lot. But you know, a lot of these use of off label drugs is really sort of also dependent on how an individual insurance company also reacts to a request, like that. So even if a patient or physician is willing, the insurance companies may not always agree to that off label use.
I would like to comment about this off label, huge, although these are rare tumors and only certain big companies or companies like, this one has ventured to do a trial for rare diseases. And it takes long time. And we try to use the off label drugs, but we face a big challenge. These drugs are expensive. Most of the time, insurance company will deny.
They will write to have 2 review papers and those papers cannot be done without any clinical trials. Sometimes the drug companies are very kind enough and will give us the drug off label. But unlike 15 to 20 years ago, where we could use any drug as long as it was
Okay. Thank you very much.
Thank you all.
Ladies and gentlemen, this concludes our question and answer session. I would like turn the conference back over to CEO, Michael Kaufman for any closing remarks.
Thank you very much. I'd just like to thank everybody joining our call today.
The conference has now concluded. Thank you for attending today's presentation. You may now disconnect.