Good morning, ladies and gentlemen, welcome to the Karyopharm Therapeutics SOHO STORM Data Conference Call. At this time, all participants are in listen-only mode. Later, we will conduct a question-and-answer session, and instructions will follow at that time. If anyone should require assistance during the conference, please press star then 0 on your touch tone telephone. As a reminder, this conference call is being recorded. I would now like to turn the conference over to your host, Mr. Elhan Webb, Head of Investor Relations.
Thank you all for joining us on today's conference call to discuss Karyopharm's additional results from the Phase II-B STORM study. This is Elhan Webb, I'm joined today by Dr. Michael Kauffman, our Chief Executive Officer, Dr. Sundar Jagannath, Director of the Multiple Myeloma Program and Professor of Medicine at Tisch Cancer Institute at Mount Sinai School of Medicine and lead investigator of the STORM study, Dr. Dan Vogl, Assistant Professor of Medicine, Hospital of the University of Pennsylvania, Anand Varadan, our Chief Commercial Officer, Dr. Sharon Shacham, our Founder, President and Chief Scientific Officer, Mr. Michael Falvey, our Chief Financial Officer, finally, Dr. Jatin Shah, our Senior Vice President for Clinical Development. Following our prepared remarks today, we will then open up the call for your questions.
Yesterday evening, we issued a press release detailing the updated results of part 2 of the pivotal Phase II-B STORM study from the presentation at the Society of Hematologic Oncology, SOHO, 2018 Annual Meeting. The release is available on our website, as are the slides, at karyopharm.com. Before we begin formal comments, I'll remind you that various remarks we will make today constitute forward-looking statements for purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995. These include statements about future expectations, clinical developments, and regulatory matters and timelines, the potential success of clinical candidates, and our plans and prospects.
Actual results may differ materially from those indicated by these forward-looking statements as a result of such various important factors, including those discussed in the Risk Factors section of our quarterly report on Form 10-Q for the quarter ending June 30th, 2018, which was filed with the SEC on August 7th, any other filings we make with the SEC. Any forward-looking statements represent our views as of today and should not be relied upon as representing our views at any subsequent date. While we may elect to update these forward-looking statements at some point, we specifically disclaim any of the obligation to do so, even if our views change. Therefore, you should not rely on these forward-looking statements as representing our views of any date subsequent of today. With that, I'll now hand the call over to Dr. Michael Kauffman, our Chief Executive Officer.
Thank you, Elhan, and good morning, everyone. Thank you all for joining us. In April of this year, we announced positive top-line results from the pivotal phase II of our phase II-B STORM study evaluating oral selinexor plus low-dose dexamethasone in patients with penta-refractory multiple myeloma. Yesterday afternoon at the SOHO annual meeting, Dr. Sundar Jagannath presented updated and additional positive results from the study that continue to be encouraging and reinforce our belief in selinexor's potential to provide new hope for patients and families suffering from this difficult condition. Notably, based on independent review committee assessment, selinexor achieved 26.2% overall response rate or ORR, and a duration response or DOR of 4.4 months in patients with penta-refractory myeloma. In addition, Dr. Jagannath provided progression-free and overall survival data amongst various subsets.
On the call today, we are very pleased to be joined by the two renowned multiple myeloma experts, including Dr. Jagannath, the STORM Study Principal Investigator, who will provide an overview of the study results presented at SOHO, and Dr. Dan Vogl, a STORM Study Investigator who will expand upon the results in various subpopulations. Anand Varadan, our Chief Commercial Officer, will then provide an update on our commercialization efforts as we prepare to bring selinexor to the market pending the ongoing FDA review of our new drug application. As a reminder, Karyopharm completed the rolling submission of a new drug application with the FDA in August, seeking accelerated approval for oral selinexor plus low-dose dexamethasone for the treatment of patients with penta-refractory multiple myeloma. Selinexor has already achieved both orphan drug and fast track designations from the FDA for this indication.
I would now like to turn the call over to Sundar to discuss the STORM results. Sundar.
Thank you, Michael. Good morning to everyone. I'm delighted to join the team today to review the promising updated results from part two of the STORM study. Before diving into these results, I'd like to provide a short overview of the study design. The phase II-B STORM study is a single-arm, international, multi-center clinical trial evaluating selinexor in combination with low-dose dexamethasone. I would like to remind folks that part one was previously published in the Journal of Clinical Oncology by my colleague, Dr. Dan Vogl. You will hear from him. Part one of the study enrolled 79 patients with either quad-refractory or penta-refractory myeloma, so two different patient population, and investigated two dosing regimens. Based on those results, the trial was modified, and part two of the study enrolled a completely distinct and separate cohort of 122 heavily pretreated patients only with penta-refractory myeloma.
These patients had previously received two proteasome inhibitors, VELCADE and KYPROLIS, two immunomodulatory drugs, REVLIMID and POMALYST, and an anti-CD38 monoclonal antibody, DARZALEX, as well as alkylating agents. In addition, their disease must be refractory to glucocorticoids, at least one PI, at least one IMiD, DARZALEX, and to the most recent therapy. Patients started with 80 mg of oral selinexor twice weekly in combination with low-dose dexamethasone dosed at 20 mg twice weekly. Many of the patients entering the study had no other therapeutic options, including other clinical trials, and we would have normally recommended palliative care if the study was not made available to us. It is worth noting a few specific points regarding the STORM study population. First, there was a short washout period of only 2 weeks since last therapy, allowing patients whose disease is rapidly progressing to enter the study quickly.
Second, patients can enroll with moderate or even severe renal dysfunction, which is common in multiple myeloma, especially in late stages. In fact, one-third of the patients had moderate to severe renal dysfunction on the study. Third, the criteria for neutrophil, red blood cells, and platelet counts were also very liberal, such that heavily pretreated patients could participate in this trial. Finally, 2 recently published reports have documented that the average survival of patients with penta-refractory multiple myeloma is less than 4 months. Turning now to the study results, the average age of the patient was 65 years, and they had received a median of seven prior therapeutic regimens prior to entering the trial. Oral selinexor plus low-dose dexamethasone achieved an updated 26.2% overall response rate, the STORM study's primary objective. This included two stringent complete responses, six very good partial responses, and 24 partial responses.
The 2 stringent complete responses are particularly meaningful as they were negative for minimal residual disease, one at 10 to the minus 6 and the other at 10 to the minus 4 sensitivity. This level of response is particularly difficult to achieve in the setting of late-line refractory disease and provides further evidence of the potential potency of selinexor. 86 patients had previously received DARZALEX as a part of a combination therapy. We would expect these patients to have a particularly poor prognosis, yet we observed an overall response rate of 29% in this population. In addition to the rate of 26.2% overall response or partial response or better, 13.1% of the patients achieved a minimal response for a total clinical benefit rate of 39.3%. I will note that all responses were confirmed by an independent review committee.
For the secondary objective of the study, median progression-free survival, or PFS, was 3.7 months. The median duration of response, or DOR, was 4.4 months based on a range of less than one to 9.9 months, and the median overall survival across the entire population of 8.6 months. The median overall survival in nearly 40% of the patients with at least a minimal response to selinexor was statistically significant at 15.6 months, while patients whose disease progressed or were not evaluable had a median survival of only 1.7 months. These results are statistically significantly different, with a p-value of less than 0.0001. Across the relevant patient population, selinexor's adverse event profile was consistent with those reported previously from part one of this study and from other selinexor studies. The side effects were often managed with dose adjustments and supportive care.
The most common non-hematologic side effects were largely grade 1 or 2 and included fatigue in 70%, nausea in 69%, anorexia in 51%, and weight loss in 47%, all of which were anticipated. The most common grade 3 or 4 adverse events were cytopenias, thrombocytopenia in 54%, and anemia in 29%, and were generally not associated with clinical sequelae. I will also note that there were no major cardiac, pulmonary, hepatic, or renal organ toxicities which led to study discontinuation. These results are compelling and truly are exciting for the patients and families with penta-refractory disease. Selinexor, with its novel mechanism of action and convenient oral dosing, has the potential to be an important treatment option for patients with penta-refractory myeloma within the greater therapeutic paradigm. With that, I'll turn the call over to Dr. Dan Vogl for additional analysis of the results. Dan?
Thanks, Sundar, and good morning, everyone. I share Sundar's excitement about these results and wanted to specifically highlight the results in some of the subpopulations that we analyzed. Probably most notably, as Sundar already mentioned, about 70% of the patients in this study had disease that had progressed not just following treatment with DARZALEX, but specifically DARZALEX in a combination regimen before they enrolled on study and started treatment with selinexor and dexamethasone. The patients in this subgroup who had received a DARZALEX combination had an overall response rate of 29.1%. Because that combination is a standard way of treating relapsed myeloma these days, that response rate is particularly meaningful. We see that in previously published data, that the DARZALEX combination regimens, including with REVLIMID and VELCADE in earlier settings, result in very high overall response rates.
Prognosis is really poor for patients once their disease progresses following DARZALEX therapy, as they tend to have an overall survival in the range of 3 to 4 months, as Sundar mentioned. I think it's compelling that selinexor was able to demonstrate meaningful responses and an overall survival of 8.6 months across this entire population, and longer survival for patients with any kind of response. Finally, I'd also like to highlight the two patients from this study who had relapsed after receiving prior investigational CAR T-cell therapy. Both of those patients went on to achieve a partial response to oral selinexor treatment. While, of course, that's only two patients, as CAR T-cell therapy is still experimental and not widely available, these results further indicate the potential of selinexor in patients with heavily pretreated myeloma who have exhausted even other promising investigational therapies.
I'll turn the call over back now to the Karyopharm team and to Anand.
Thank you, Dr. Vogl, and good morning, everyone. The STORM data presented yesterday and discussed earlier on this call illustrate exactly why I'm so excited to have joined the Karyopharm team to build and lead our commercial efforts. Rarely do you have an opportunity to bring a completely new approach to treating patients with a life-threatening illness based on a revolutionary insight into fundamental biology. My team and I are acutely aware of the critical role that we play in helping patients benefit from the amazing work that has been done at Karyopharm over the last decade. Our growing commercial organization is focused on hiring the best team and building the necessary infrastructure to bring selinexor to the appropriate patients. Everything we will do is going to be done with the single-minded goal of maximizing the patient experience.
Our plans include working to provide unimpeded access when selinexor is indicated and ensuring patients have the support they need to fully benefit from treatment, based on all of our learnings from optimizing selinexor therapy. We're building plans to serve the needs of all key stakeholders, starting with the patients themselves and their families, and including myeloma experts, community-based clinicians, oncology nurses, key payer segments, and the supply chain. Of course, central to all of this is to have the right product with the right clinical data and an FDA-approved label. Market research that we've done recently with oncologists reinforces the importance of the clinical data generated from the STORM study. Our research indicates that physicians place significant importance in a completely new mechanism of action in treating patients with advanced myeloma, and that oral administration is welcomed.
Since there's no cure for multiple myeloma, clinicians are constantly seeking additional treatment options for the many patients who unfortunately will relapse or become refractory to therapy. Nearly 13,000 deaths from myeloma are expected this year in the U.S., underscoring the urgency for new treatments. Our research indicated that physicians value the clinical data from STORM based on the overall response rate, including the two MRD negative stringent complete responses in such a highly pretreated patient population. They also noted that the median time to response of one cycle could allow physicians to identify if patients are responding relatively quickly. Should the FDA grant our request for accelerated approval, selinexor could be available to patients in the first half of 2019.
Additionally, in the first quarter of 2019, we plan to submit an MAA to the EMA for conditional approval of selinexor in the European Union based on the data from the STORM study. The data presented yesterday is another positive step forward for these patients, and I look forward to providing future updates as we progress. I'll now turn the call back to Michael.
Thank you, Anand. To our knowledge, the data reported from STORM represent the first set of complete phase II data in this difficult-to-treat penta-refractory myeloma population. Our STORM data are an exciting milestone for our company, but more importantly, they represent an important advance for the greater myeloma community of patients, families, and treating physicians participating in this study. We want to recognize their support and express our sincere gratitude for their important contributions to the STORM study and the development of selinexor. In addition, we also wish to thank the Multiple Myeloma Research Foundation for being an invaluable partner for many years and for their extensive support and encouragement. We plan to continue to build our momentum and execute on the commercial initiatives outlined by Anand in order to introduce this treatment option to patients as rapidly as possible.
In parallel to our penta-refractory program, we continue to advance our development strategy aimed at moving selinexor to reach patients in earlier lines of treatment. Our pivotal randomized phase III BOSTON study is evaluating once-weekly oral sel-dex
In combination with once-weekly VELCADE compared to standard twice-weekly VELCADE dexamethasone alone in patients with myeloma who have had one to three prior lines of therapy. We expect to complete enrollment in BOSTON this year and report top-line data at the end of 2019. Our broad STOMP study continues to advance the evaluation of sel-dex in combination with several standard approved myeloma therapies, including REVLIMID, POMALYST, VELCADE, KYPROLIS, and DARZALEX in patients with relapsed or refractory myeloma, and in combination with REVLIMID in patients with newly diagnosed myeloma. We plan to provide periodic data updates from STOMP at upcoming medical meetings. Outside of myeloma, selinexor is also being developed for diffuse large B-cell lymphoma, or DLBCL, for liposarcoma, for endometrial cancer, and for other malignancies.
We remain on track to report top-line results from the phase II-B SADAL study evaluating single-agent selinexor in patients with relapsed or refractory DLBCL who are not candidates for stem cell transplantation by the end of 2018. Pending positive results of the SADAL study, we plan to submit an NDA requesting accelerated approval of selinexor in the first half of 2019 for this indication. We grow and advance our pipeline and organization, we remain motivated and committed to our mission of advancing the treatment of cancer through the discovery and development of novel oral therapies like selinexor. Our team is expeditiously working on behalf of our brave patient community to bring these therapeutics to market as quickly as possible, and today's data are another positive step towards meeting that goal. We appreciate everyone dialing in today, and we'll now turn the call over to the operator for questions. Operator?
Ladies and gentlemen, if you have a question at this time, please press star, then the number 1 on your touchtone telephone. If your question has been answered or you wish to remove yourself from the queue, please press the pound key. Your first question comes from Brian Abrahams from RBC Capital Markets. Your line is open.
Hello. Hi, everyone. This is Beau Miller on for Brian. We both congratulate you on the update. Just a few questions. I guess first, bigger picture, how have the discussions been going with the FDA, both pre-NDA and post your submission, and how might you characterize the frequency and nature of those interactions since you received the Fast Track designation? Can you help us get a sense of how the regulators might view the high median OS among responders and what their thinking is there?
We don't really comment on interactions. They've been very good. Obviously, FDA granted the Fast Track application, as you mentioned. They granted a rolling submission. We're in the usual discussions. It's been a great relationship. We hope to continue it, and that's all we're really prepared to say. On the overall survival, obviously, I think everybody's extremely excited about this. This population, as has recently been reported in two separate publications, has a dire prognosis. The fact that up to 40% of our patients who had at least a minimal response had such a significantly improved survival, I think bodes well for patients. Furthermore, even patients with stable disease on our study appear to achieve some benefit as we stop the progression of this refractory myeloma.
Great. Can you also contextualize the median PFS of 3.7 months and maybe among some of the subgroups you presented, how does that compare to other agents like daratumumab and Pom when looking at a similarly sick population?
I think when we look at median PFS of 3.7 months, that's really in line with what we saw with really carfilzomib, pomalidomide, and daratumumab. They're all around that three- to four-month range. I think similarly, I think the key point is that those patients who do respond with an MR or PR, you see a significant improvement in their PFS and OS. That really drives the clinical benefit. When you talk about median PFS in this late-line therapy, it's very much in line with what we'd expect to see and what we've seen with previous accelerated approvals. The key really is does that response rate drive to clinical benefit, which can be reflected in the PFS and OS.
My last question, and I'll hop back in the queue. On the safety profile, could you just provide a little more color on the persistence and manageability of some of the side effects you observed, like fatigue and nausea and some of the hematological AEs? To what extent do you feel that mirrors an increase in understanding by physicians to manage these side effects that is driving the better responses among responders?
These are pretty heavily pretreated patients who are generally sick, they are also losing weight because their cancer is not under control, and they have gone through all these different therapy. As I alluded to before, if this clinical trial was not available, we would have offered them a hospice care. Such being the case, here is an oral agent, which has the side effect of nausea, anorexia, and vomiting. Under those circumstances, this is an oral agent, so the patient can take the pills at home, but if you reach out to them and make sure they are able to eat and drink well, if not, just bring them and give them IV fluid hydration.
The very fact that you give the hydration alleviates the subsequent nausea sensation, and actually, they do better, rather than saying them to stay home and take more nausea medication. Those are the key element in managing these particular patients. Of course, the patients, advanced cancer patients, are usually fatigued. Keeping their motivation up, and especially the moment they show response, these patients get really motivated and then they stay on it. Monitoring them a little more closely, including with the laboratory parameters, because the responses are pretty fast, are seen within a month. We monitor the patient's response also fairly closely. This gave a feedback loop for the patient. These patients get more encouraged, and they do better.
Another reason for fatigue, the patients were allowed to come in the trial because they have been heavily pre-treated patients. They all come in with anemia. They come in with thrombocytopenia. There is an added component of fatigue from not only the disease burden and all the treatment they have, but actually they are quite anemic too. All of that adds to it. Supportive care plays an important role, and their tolerance gets better.
If I can ask Vogl if you can also comment, that would be great.
Great.
Your next question comes from Eric Joseph from JPMorgan. Your line is open.
Hey, guys. Thanks for taking the questions and congrats on the update. Just a couple questions from us. I guess first for either Dr. Jagannath or Dr. Vogl. I guess with dara combinations increasingly being early line standard of care, I am just wondering how we should think about patients achieving minimal response or stable disease, in that context, in a post-DARZALEX combination setting. How you think about achieving at least stable disease, how that would sort of factor in either decision to keep a patient on selinexor moving to a potential alternative or move over to another investigative agent, perhaps a clinical trial. Another question for the company. Maybe just you can help us kind of put the survival of 8.6 months into context. How does it contrast with sort of survival expectations in the post-dara refractory patient population? Thanks.
Going back to your first question, yes, the dara is being used more often in combination. Okay? That is true. The point is that this particular trial, that was the key component, that once the patient had failed penta-refractory, all the available agents, they were literally, they did not have any real approved treatment option in that setting. In those patients, even if they had failed daratumumab combination, this drug showed the same response rate, 29% overall response rate, showing that it didn't matter which class of drug the patient had been exposed to because this is the first-in-class new drug mechanism of action. What we are all impressed is that this is a true addition to the treatment armamentarium of myeloma, bringing in a new class of treatment.
Then, you know, there is already a BOSTON trial where they are moving it, comparing it with the VELCADE, dexamethasone, given twice weekly versus VELCADE, and the selinexor given once weekly regimen. I am very confident that will be very positive. For me, the first of all is currently getting an accelerated approval is this drug is very critical for patients who have failed penta-refractory and truly don't have an option. This drug really provides an important option and especially an oral agent. In the future, this drug could be properly primed in the right clinical setting. For the second question on the drug, yes.
Yeah. What we know, and it gets a little bit to the stable disease part is what we know is that the patients who progressed, which was a relatively small number of patients who had progressive disease or were not evaluable for response at the beginning, survived less than two months overall. Even patients with stable disease appeared to derive a benefit. That's because, as Sundar mentioned, they have penta-refractory disease. They're coming in with rapidly progressive myeloma. If the disease is not controlled immediately, these patients unfortunately will really run out of options and be in dire straits. The published data suggests that the responses, that the overall survival of this population is between three and four months without additional novel treatment, and our overall entire population survival was 8.6 months.
Just as a preview, we have also looked into this and tried to come up with additional independent data, which we hope to present in the future, suggesting this three to four-month range for survival in the real world is probably accurate. We're very excited about the entire population results.
Thanks. Thanks for taking the question.
Your next question comes from Maury Raycroft from Jefferies. Your line is open.
Hi, good morning, congrats on the update. First question is on the 3.5 median overall survival estimate referenced on slide four. Wondering if you can go into more details or background from the two publications you mentioned. Does that number come from multiple studies, or anything else you can provide on that would be helpful.
Could you repeat it again? Sorry.
The median overall survival estimate of 3.5 for penta-refractory patients, which is referenced on slide four. I think there were two publications that you guys mentioned too, which I haven't reviewed those publications, but I'm just wondering if you could talk more about the data that gets you to the 3.5 number.
The first publication is from Pickett et al. It's a study that was done in Israel including patients. They looked at patients that were treated with DARZALEX and followed them after. They included several populations. One of the populations that they investigated was patients that were treated with DARZALEX as a single agent that were heavily pre-treated. They looked at what happened with these patients post-progression on DARZALEX. They added additional drugs. They treated the patients with DARZALEX in combination, and after that, they went to other salvage therapy. The slide that was presented yesterday by Dr. Jagannath is from that paper, in which it shows the results of these 22 patients with heavily pre-treated myeloma that were treated with DARZALEX in combination and then moved into salvage therapy.
Okay. Then the other question I had was on the two CAR T patients. I'm wondering if you can provide more details on those patients, particularly treatment they received before or after the CAR T, and if they responded to the CAR T. Is there any read-through to your DLBCL program that we should consider?
I'll just comment, come back to your last question as well regarding the post-DARZALEX survival. There's, I think, a growing body of data now in that post-DARZALEX, and I think we'll see more and more of that coming. I'll refer you to a paper by Dr. Usmani as well, who looked at post-DARZALEX outcomes as well. I think you'll see consistently that the overall survival is quite short in these patients. There's another paper as well out from the Mayo Clinic by Dr. Lakshman, which is very similar as well. I think that there's a growing body of evidence now of this short overall survival across various single-center retrospective studies of this median overall survival that's going to be in that 3 to 4 range. I think that I just want to make sure I highlight that piece as well.
Coming back to your question about CAR T-cell, correct, and the two patients that responded post-CAR T-cell?
Yes.
I don't know the response to those prior CAR T-cells. We'll share that data at a future meeting as we go into that. I think the key is that even after a CAR T-cell therapy, these patients do respond to therapy. Your question is, does that apply from the large cell lymphoma data with CAR T-cells?
Yes.
Yeah. I think that it's difficult to tell. What I can clearly say is that when we look at across various subsets, if we look in patients who had prior daratumumab, prior daratumumab in combination, if they've had prior transplant, combination chemotherapy with DTPACE, or refractory, any of the novel therapies across any of those subsets, we see a really preserved response rate, even a little bit higher response rate in the post-DARZALEX patients. Regardless of their prior therapies that they got, again, going to the mechanism of action that this does not have any cross-resistance to any other prior therapies. Because of its novel mechanism, it doesn't really matter what the patients had or did not have before. We really have a preserved response rate. Is it novel therapies? Is it chemotherapies? Is it CAR T-cells?
These patients all benefit from selinexor.
Got it. Can you comment if you have any patients who have failed CAR Ts in the SADAL study?
We're not aware of any. These are transplant-ineligible and generally not the same population.
Right. Okay. Thank you very much, and congrats again on the update.
Your next question comes from Jonathan Chang from Leerink Partners. Your line is open.
Hello, thanks for taking my call. This is John Bae on for Jonathan Chang. I was just wondering if you could provide some context around the patients who switched from Very Good Partial Response to Partial Response.
Sorry, we're not aware of any patients that did that.
Usually in clinical trial, you'll give the best response to the patient, then later on you will see whether the patient progressed. You don't give data of a Very Good Partial Response patient going down to Partial Response, then going down to stable and then progressing or something like that. I'm not aware how to answer your question.
Understood. Then in terms of the market research you guys have been doing for the preparation for commercialization, you mentioned the doctors are very interested in the clinical data surrounding ORR and the complete responses. Has your market research focused on the doctors' opinions on the safety and tolerability of selinexor? What are some of the takeaways you've gleaned from those discussions?
Hi, this is Anand, John. Thanks for that question. Of course, in our market research, we addressed also the tolerability of the side effects that we're seeing in STORM as well as the efficacy data. Their response to that was generally, especially given our own experience within the study, that they would deploy the necessary supportive care for these patients, depending on the kind of side effects that they would face. Whether it was the cytopenias and the supportive care that would be necessary under those circumstances or the constitutional symptoms, they are accustomed to dealing with these kinds of side effects.
I think that the key learning for us is that they have a proactive posture towards this, that they're vigilant, and that they deploy that support for the patients so they can continue to benefit from the therapy.
Great. Thanks. That's all I had.
Just to go back here. I think we understand why you asked the question. I think between the abstract and then the final data. The abstract 6.5% VGPR rate included VGPR or CR. Nothing changed. The VGPR plus CR rate is 6.5%, including 4.9% VGPR and 1.6% CR.
I see. My apologies. That was my mistake then.
Again, ladies and gentlemen, if you have a question at this time, please press star then the number one on your touch-tone telephone. Your next question comes from Michael Ulz from Baird. Your line is open.
Hey, guys. Thanks for taking the question and congrats on the update as well. I have a question for Dr. Jagannath and Dr. Vogl. Maybe you can comment on what percentage of your current patients are penta-refractory, and is there any reason you wouldn't put them on selinexor?
That's a good question. At Mount Sinai, being in Manhattan, we primarily have been concentrating on relapse and refractory myeloma. We get a referral of patients who have been treated by community oncologists, and once they have failed all the options, the patients are referred to us. It is kind of a skewed population rather than, say, if you are the only freestanding cancer center for a long distance, you are only treating your own patient population. We see a lot of patients, and that's how we ended up being one of the high enrollers on this particular trial. Part of it was also driven by the fact that we have other clinical trials also open, including CAR T-cell therapy, et cetera.
We find that when the patients are being referred for CAR T-cell or the patient refer themselves, many of them are in such dire straits, the disease is progressing quickly. They need treatment right away that will be effective. Because as you know, in CAR T, needle to infusion time, as they call it, needle-to-needle time, you collect the T-cells, then they have to wait till the CAR T-cells are prepared, then you have to administer it, almost a month. Some of these patients cannot even wait that long. The second thing is, which is very important for this particular clinical trial, the entry criteria was a lot more liberal than any other clinical trial option. The platelet count is only 70,000. The hemoglobin could be 8.5, just above that. The creatinine, especially, you are allowed CKD stage 4.
That means eGFR at 20 milliliter per minute or greater. We don't have that luxury in any other clinical trial. There were many reasons why patients were able to enroll on this particular trial. That's why I feel like even when this oral agent gets approved, it would become very important for the myeloma community at this time.
Operator, I think we have time for just one last question.
Certainly. Your next question comes from the line of Konstantinos Aprilakis. Your line is open.
Hey, guys. Let me add my congrats on the update. I'm interested in getting some additional color from Dr. Jagannath on the two patients that achieved stringent complete responses. Could you perhaps share the disposition of these patients when they initially came on study and then the kinetics of their responses to seli? I got a quick follow-up.
Well, I'm only privy to one patient who happens to be my patient, who went into complete remission, stringent complete remission. That lady travels to Europe and comes back, and we had to make provisions for all that and actually have one of the IFM physicians do the CBC, et cetera, because she's still on clinical trial to get us the CBC and other information on a timely fashion so we are not violating the protocol. She is still being strong, I believe at least a year now. We are totally thrilled, and of course, that woman is thrilled because her life is back to herself and she is traveling and doing well. I can't answer about the other patient. It's not mine.
I think, this is Jatin Shah . I'll just comment on the second patient. A very similar experience that Dr. Jagannath has. A patient who's in a long-term remission, doing well and tolerating therapy, almost out to a year as well. Similar experience of a second patient as well with a durable CR.
You mentioned, Dr. Jagannath, your patient's still traveling, which is pretty remarkable, considering how late in treatment these patients are. Did you notice sort of an anecdotal difference in how that patient is tolerating the treatment versus the general patient population?
This patient, once she achieved complete remission. The other issue I always said is that the depth of response matters to the patient in many ways.
The side effects also, the asthenia, weakness, tiredness also gets somewhat mitigated once they have an excellent response. Physiologically, these are heavily pretreated patients whose disease is progressing. These patients, when they come to your clinic, they are sick. You literally see them, they are sick. They are not coming in, not like a first relapse patient or a biochemical relapse, so they are physiologically good. These are sick patients who has thrombocytopenic, have renal impairment, et cetera. Once they have a good response, there is improvement overall. The blood counts get better, so now their hemoglobin is better, their kidney function is better. Some of these other asthenia side effects we attribute to the medication actually gets mitigated. I think that's reason. You are absolutely right. There are some patients, just like you know VELCADE, you get peripheral neuropathy unpredictable.
We can't tell who's going to get neuropathy and who's not, and who's going to get a painful neuropathy and when it happens. Likewise, with this drug, there are patients who are able to take this drug and have no nausea or any side effects at all. There is also inter-patient variability, and that is something I think we need to, moving forward, have to look at it little more carefully. The only thing I would say is this drug is out there in lymphoma, in sarcoma, in GYN cancer, et cetera. There is a large body of safety information coming along. Along with that, I think we would also able to come up with a better dosing and scheduling and what therapeutic level is very critical and all those things. This is a new drug.
We are all sticking to the protocol-driven thing because it was phase I, now it is phase II. There is also a learning curve for all of us involved. I'm pretty sure this will become an important drug in the near future.
Perfect. Thanks very much. Congrats again.
I am showing no further questions at this time. I would now like to turn the conference back to Michael Kauffman.
Sure. Thanks so much. Thanks to all of our participants and everyone on the team here. In closing, I want to thank everybody for dialing in today and the questions which were excellent, we look forward to keeping you updated on our continued progress. Have a great day, everyone.
Ladies and gentlemen, this concludes today's conference. Thank you for your participation, and have a wonderful day. You may now disconnect.