Bellicum Pharmaceuticals, Inc.

Bellicum Pharmaceuticals, Inc.

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Bellicum Pharmaceuticals, Inc. (BLCM) Q2 2017 Earnings Call Transcript

Published at 2017-08-08 21:57:09
Executives
Alan Musso - Chief Financial Officer Rick Fair - President and Chief Executive Officer
Analysts
Srikripa Devarakonda - Citigroup Reni Benjamin - Raymond James Peter Lawson - SunTrust Robinson Humphrey Wangzhi Li - Ladenberg Thalmann Biren Amin - Jefferies
Operator
Greetings and welcome to the Bellicum Pharmaceuticals Incorporated Second Quarter 2017 Earnings Conference Call. At this time, all participants are in a listen-only mode. A question-and-answer session will follow the formal presentation. [Operator Instructions] As a reminder, this conference is being recorded. I would now like to turn the conference to your host Mr. Alan Musso, Chief Financial Officer. Thank you Mr. Musso, you may begin.
Alan Musso
Thank you, Tim. Good afternoon everyone and thank you for joining the call. With me today is Rick Fair, Bellicum's President and Chief Executive Officer. Earlier this afternoon, Bellicum released financial results for the second quarter ended June 30, 2017. If you have not received this release or if you would like to be added to the distribution list, you can do so on the Investor Relations page of the company's website. As a reminder, today's conference call will include forward-looking statements made under the Private Securities Litigation Reform Act of 1995, including statements regarding our research and development plans, clinical trials, plans regarding regulatory filings, and our financial outlook. These forward-looking statements involve a number of risks and uncertainties and reflect our opinions only as of the date of this call. We undertake no obligation to revise or publicly release the results of any revision to these forward-looking statements in light of new information or future events. Actual results may differ from those indicated by these forward-looking statements due to numerous factors, including those discussed in the Risk Factor section of our annual report on Form 10-K and the 10-Q for the quarter ended June 30, 2017 filed with the Securities and Exchange Commission. And now I'll turn the call over to Rick.
Rick Fair
Thanks Alan. Good afternoon, everyone. Thanks for joining us today. It’s been about six months since I joined Bellicum and during this time we’ve had the opportunity to do a thorough review of our strategy, our operations, and I have to say I’m more excited and optimistic than ever about the opportunities that we have. Today, what I’d like to do is summarize what we’ve learned and how we intend to move forward, starting with the headlines. First we’ve done a deep dive on the BPX-501 and have confirmed our belief that this represents a significant advance for patience and the meaningful commercial opportunity for our company. We’ve refined our plans a bit to seize this opportunity efficiently and I will tell you more about that shortly. Second, we remain excited about BPX-601 and BPX-701, our GoCAR-T and TCR product candidates. These are high risk, high reward programs using our novel technology platform against novel targets, and I’m proud that we’re taking on challenging diseases with such innovative therapies. As we move forward, we look to balance our portfolio risk both by de-risking these programs to the extent possible and by advancing lower risk candidates from our research pipeline. Third, as the self therapy feels evolves and advances, we remain convinced that our molecular switch platform to control the efficacy and safety of these therapies is increasingly irrelevant and the best-in-class. We will look for ways to maximize the impact we can have both through our internal pipeline and through external collaborations. Finally, with all the operate opportunities we have, we acknowledge the need to continue to build and strengthen our organization to deliver on them. We've recently identified strong leaders to assume the Chief Business Officer and General Manager of the Europe roles and are actively searching for a Chief Medical Officer and other experienced clinical and regulatory staff to bolster our existing team, and sure we can execute against a growing set of portfolio priorities. I’d like now to drill down a bit on each of these points starting with BPX-501. Over the last few months, we’ve conducted a thorough analysis, which included engagement with our investigators and extensive market research, both with treating physicians at major transplant centers and with payers in the US and in Europe. We’ve heard loud and clear that the data we’ve generated to date in children are compelling. We recently presented an update from our global BP-004 study at the European Hematology Association Congress in June. In a presentation selected as one of the meetings Top 6 to be included in the Presidential Symposium, data from 98 patients with both hematologic cancers and inherited blood disorders and at least six months of follow-up where reviewed. Investigators reported that the vast majority of patients in the study experience successful and rapid immune recovery and early hospital discharge. Incidence of GVHD was low and cases were manageable either with the administration of standard treatments or with rimiducid. Also at this meeting, a presentation of a subset of 47 malignant patients from this study showed similar favorable outcomes and lower rates of relapse, only 14% with a median follow-up of eight months. This compares favorably to historic controls in those patient population. In our market research, we heard from physicians that upon approval a product with these data will become a preferred option for patients receiving an allogeneic stem cell transplant who do not have an HLA matched donor. It would also be considered for some patients who currently receive a transplant from a matched unrelated donor, and for some who don't receive a transplant at all because they lack a match. We heard from payers that they see significant value in this profile in terms of across-the-board reductions in mortality, complications from GvHD and infections, relapse and hospitalization. Taking together this affirms our belief that BPX-501 represents not just an important option for these patients, but also a significant commercial opportunity for us. Based on these findings, we continue to progress toward the market. Our approach to registration for pediatric patients in Europe remains unchanged. Enrollment in the pivotal EU BP-004 trial remains on track for completion by the end of this year. Additionally, the observational trial in pediatric patients receiving transplants from matched unrelated donors or MUD without BPX-501 is initiating this quarter. Outcomes from these trials are expected to be the basis for filings of European Marketing Authorization Applications for both BPX-501 and rimiducid. Our regulatory threshold is to showed non-inferiority to a MUD transplant and will also test for superiority as well, MUD transplants to the standard-of-care for the approximately 75% of allogeneic transplant patients who don't have access to a match related donor. If BPX-501 can provide better outcomes in MUD transplants this could be transformational. With the six-month primary endpoint measure, we now expect that we will have top line results in the second half of 2018 with the EU filings planned for 2019. Beyond European pediatric patients, we have two opportunities to address, adult malignant patients, and US pediatric patients. We’ve prioritized our efforts in adult malignant patients as it represents approximately 80% of the global allogeneic transplant opportunity. We are currently finalizing the design for a randomized controlled trial in adults with acute myeloid leukemia comparing patients receiving a haplo transplant and post-transplantation cyclophosphamide. This we’ll call Baltimore regimen followed by BPX-501 versus the Baltimore regimen alone. Our objective for this study is to show superiority to the current standard of care and adult malignant patients who do not have a matched donor and to pursue registration in the US and Europe in this population. This study is being supported by $16.9 million grant from the cancer prevention and research Institute of Texas, and we greatly appreciate their support and collaboration. For US pediatric patients, we’ve revised our plans. We’re designing a registrational study in an ultra-orphan pediatric inherited blood disease where the need is most acute and a path to regulatory approval is most direct. This should allow us to efficiently secure a pediatric approval in the US and combined with the data we’re generating in the broader ongoing BP-004 study should get pediatric transplant specialist in the US, the data they need to guide their use of BPX-501. This will also allow us to conserve resources to invest in other portfolio priorities. We expect to initiate these two new studies in 2018. Turning to our CAR T and TCR clinical programs, the Phase 1 trials of the BPX-601 and BPX-701 were initiated earlier this year at centers in the US. Recruitment is ongoing and we anticipate reporting initial results for both in 2018. BPX-601 is our novel GoCAR-T, our first clinical candidate incorporating our proprietary iMC activation switch. This novel construct is designed to give physicians control over the level and persistence of stimulation and proliferation of these cancer fighting cells for improved efficacy and safety. BPX-601 target’s tumors expressing prostate stem cell antigen or PSCA and our Phase I trials being conducted in non-respectable pancreatic cancer. BPX-701 is a high affinity T cell receptor product candidate designed with our CaspaCIDe safety switch. It’s being tested in patients with relapsed or refractory AML and MDS, you test positive for PRAME or preferentially-expressed antigen in melanoma. I mentioned earlier that these are high risk, high reward programs. Moving forward, we will look to de-risk these programs, as well as to balance the overall risk in our portfolio. I will give two examples. First, we recognize the pancreatic cancer, the initial indication in our BPX-601 trial is particularly challenging for T cell therapies. As we safely move through dose escalation, we plan to amend this study to include other cancers that express PSCA like bladder cancer that have proven more responsive to T cell directed therapy. Second through academic collaborators we plan to evaluate our CaspaCIDe safety switch on CD19 CAR-T cells. Offsetting [ph] where CAR-T has a well established benefit risk profile this will help us better characterize the profile of CaspaCIDe for future CAR-T programs and if the result is just substantially differentiated, potentially give us an efficient start on a new product candidate. We expect our collaborators to initiate a clinical trial this year. These are only two examples of the representatives of the way they will evaluate manage risk in our portfolio decisions moving forward. Turning finally to the last few areas I emphasized in my opening, our platform and our organization. Bellicum's novel cellular control platform is one of the reasons I joined the company. Adopted cell therapies are coming of age and we eagerly await the first approval of the CAR-T therapy later this year. But I believe one of the major challenges preventing the cell therapy from addressing a much larger set of diseases is the lack of control of these treatments in vivo, the ability to dial up efficacy when needed, particularly in solid tumors and the ability to manage the inevitable toxicity that results. I believe we have an industry-leading platform that has the promise to do just this and we continue to invest to optimize it. During the quarter, we presented an exciting preclinical data on our novel dual switch technology at AACR. The potential advantage of a dual switch is the ability to both activate cells to enhance efficacy, and eliminating them to manage toxicity in a single product. We now have preclinical CAR-T and TCR candidates with the dual switch. We look forward to sharing more information with you as we select future candidates to move into the clinic either alone or through collaborations. We see many areas where we can apply our platform to create first and best-in-class therapeutics. We have a broad set of opportunities and our strengthening our organization to capitalize on them. Lastly, we announced the appointment of Greg Naeve, as our Chief Business Officer. In this role, he will lead our product and portfolio strategy and business development functions. Greg has an impressive background and strategy and business development and venture capital and research. We’re delighted to have him join the team. We’ve also identified the General Manager for our European operation who will build the team to launch BPX-501. I look forward to announcing him soon. With these additions and with additional recruiting we’re doing, particularly in our clinical organization, we’re building on the great team we have to scale to the exciting opportunities ahead. I’ll now turn the call back over to Alan to review our financial results.
Alan Musso
Thank you, Rick. Bellicum reported a net loss of $24.5 million for the second quarter of 2017 and $46.4 million for the six months ended June 30, 2017, compared to a net loss of $16.5 million and $31.6 million for the comparative periods of 2016. These results included non-cash share based compensation charges of $3.2 million and $6.6 million for the second quarter and six months ended June 30, 2017 and $3.1 million and $6.2 million for the comparable periods in 2016. As of June 30, 2017 cash, restricted cash, and investments totaled $139 million, and based on our current operating plans, we continue to expect to end 2017 with approximately $85 million to $95 million in cash and investments and our current cash resources will be sufficient to meet operating requirements through 2018. Research and development expenses were $18 million and $33.3 million for the three and six months ended June 30, 2017 respectively, compared to $12 million and $22.9 million during the comparable periods in 2016. The higher expenses in the 2017 period were primarily due to an increase in clinical development and manufacturing cost, due to increased patient enrolment in clinical trials, principally BPX-501, and increased personnel expenses, overhead charges, and manufacturing facilities start-up costs. Our general and administrative expenses were $5.5 million and $11.4 million for the three and six months ended June 30, 2017 respectively, compared to $4.2 million and $8.5 million during the comparable period in 2016. The higher expenses in the 2017 period were primarily due to our overall growth, including an increase in personnel related cost, principally due to hiring additional employees and severance costs, higher facilities cost, and increased legal, accounting, and travel expenses. And now I’ll hand the call back over to Rick.
Rick Fair
Thanks Alan. As you’ve heard today, we’re making progress across our pipeline and our operations, as well as setting our strategic priorities for the future. We’re excited about the potential role in advancing the field of cell therapy that Bellicum can play, and we look forward to keeping you updated on our progress. With that, I’ll now open the call to questions.
Operator
[Operator Instructions] Our first question comes from the line of Srikripa Devarakonda of Citigroup. Please proceed with your question.
Srikripa Devarakonda
Hi guys thank you so much for taking my question. I am on for Robin. We have five other calls today, so he apologizes for not being able to be on the call today. Congrats on the progress over the quarter, I just had a couple of questions. First one is, can you talk a little bit more about the changes you’ve made in the US pediatrics program that you were talking about that you will focus on ultra orphan, is this, how different is this from what you had talked about before? And also just a big picture question regarding post Novartis ad comm., what do you think were good takeaways and how do you think that this has - what sort of overarching implications does this have on the field in general? Thank you so much.
Rick Fair
Sure. Thanks for the questions. First question first. So, the program that we had been discussing in the US and pediatrics is really a broader based program for all pediatric patients eligible for a haplo transplant. These were patients with the he-malignancies and patients with non-malignant diseases in sort of a basket approach in the way that we've constructed the trial and reached agreement in Europe. And really what we concluded was that the investment to replicate that program to generate is essentially the same set of data in the US wasn't the best use of our resources, and so we’ve recast our plan in the U.S. to focus on a single ultra orphan disease where the unmet need is the greatest where the regulatory path is clear and straightforward, so that we can get an FDA approval and we believe with the FDA approval combined with the data set we’ve generated across the US and European programs that US treating physicians will have the information they need. So, it’s a much more streamlined and efficient program so that we can offer access to pediatric patients, but also preserve resources for other priorities. I think in the answer to your second question regarding the Novartis ODAC we are obviously enthusiastic about it. It was great news for ALL patients who will benefit from their CD19 product, also great news for this cell therapy community. I think the team at Novartis should be congratulated for their excellent work, the way they presented their package, the regulators, and I’m sure they are going to do it an equally excellent job in bringing their product to the market with the same level of expertise and professionalism. You know [indiscernible] cell therapy is coming to fruition and we have a supportive regulator in the US who buys into the benefit risk profile and is also cognizant of the issues that we will face in manufacturing and long-term safety follow-up that we were all vigilant about. But we think they’ve offered a very realistic path to market for cell therapy and I think that’s positive news for the whole community including us.
Srikripa Devarakonda
Okay great, thank you. So just one little clarification, have you specified, to my first question, which ultra orphan disease you are going to be focusing on in the US?
Rick Fair
We have not. We have put together a short list. We selected the shortlist based on [indiscernible] series of unmet need and those were regenerated positive data in the BP-004 trial, and we will be making a final decision on that based on clinical trial feasibility and regulator input and we will keep you posted.
Srikripa Devarakonda
Okay, great. Thank you so much.
Rick Fair
Thank you.
Operator
Our next question comes from the line of Reni Benjamin of Raymond James. Please proceed with your question.
Reni Benjamin
Hi good afternoon guys. Thanks for taking the questions. Maybe just a couple, can you talk a little bit more about your timing in terms of the top line results in the second half of 2018 and the filings in 2019, is it the observational study and how long that might take that may have pushed these timelines out, or is there something else? Anything else in terms of the color you can provide?
Rick Fair
Sure. Actually it is a couple of small things. I think the first is that when we laid out our current projections of enrolment of both BP-004 and the observational study and looked at final - last patient last visit, data collection and data cleaning, our database lock is now sitting on the second half of 2018. I think the other thing we did is really look through our very detailed operational review at our filing timelines and to be honest, I think our previous forecasts were too aggressive. We've updated that with a more industry-standard timeline from database lock to filing and that pushes us into 2019. So, no single descriptor, certainly not enrolment of BP-004, which continues to be healthy, but it’s really about laying up the timelines very clearly on enrolment data collection, database cleaning, data lock, and filing. I would also point out that it is a filing of two MAAs. Europe has taken the view that BPX-501 and rimiducid are two different products and we have two different MAAs to support so that we did to our thinking on the level of aggressiveness we could have on timelines there.
Reni Benjamin
Got it. And then just kind of thinking about the CAR T space, as we look at the way other companies are handling the side effects, it seems that over time we’re getting better at being able to handle those side effects from the toxicities, how do you I guess deal with kind of the current standard and what it is evolving to and where your platform may or may not be able to address the challenges let's say a couple of years from now?
Alan Musso
Well I think the sort of the philosophy behind our platform is that in order to have widespread use of CAR T’s you will need more active constructs and certainly we see ourselves and others innovating with a co-stimulatory demands and other features to dial up the level of activity of these constructs. As you do that we believe you will inevitably face toxicity. So the way we think about our platform isn’t simply about the safety switch and whether that applies to things like the CD19 CAR T's, but we think about it holistically as dialing up activity, as much as possible to try and treat difficult to treat cancers, particularly solid tumors. And when you inevitably over do it and face toxicity that you have a way to manage that. And to be honest we are - I think seeing a widespread level of acceptance of that principle many of the academic institutions that are innovating with more active constructs beyond second-generation cars are embedding safety switches on board and most often ours.
Reni Benjamin
Got it. Thanks for taking the questions.
Operator
Our next question comes from the line of Peter Lawson of SunTrust Robinson Humphrey. Please proceed with your question.
Peter Lawson
Hi, hope you can hear me. Just wanted to ask a couple of questions around timeline, so BPX -004 the data got pushed back to 2019, is that kind of a first half event or second half event, any comment around the brief help, it doesn't seem as if it was any one specific issue that drove that push back?
Rick Fair
Yes, I think I would try to clarify it was really a combination of re-forecasting of enrollment and data collection timelines along with, I think more realistic planning timelines around filing preparation. I don't think we're providing specific 2019 guidance on timelines yet, but we will have data in hand in the second half of 2018, we are confident in that and so you can make your own estimate from there.
Peter Lawson
Got you. And then refocus of the US trial? When do you think you have the data submitted there and what was the source of the push back, was it FPA, think it was too broader filing or any color around that would be really helpful.
Rick Fair
Sure, absolutely not. We don't have any pushback on the program that we have previously been devising in pediatrics in the US from the FDA. We - as I indicated it under a through strategic review of our entire portfolio, but particularly in BPX-501 recently concluded that and made the decision based on strategic priorities. As I mentioned, we felt like it wasn’t the best use of our resources to do a large basket like trial in pediatrics in the US that essentially replicated to BP-004 trial and the comparative MUD trial, which is the type of program that would have been required to get a comparable label. We see the largest opportunity in non-malignant setting and want to prioritize our resources that and on a future pipeline, but what we did see is the opportunity to provide access in the US by a streamlined program to get an FDA approval and to leverage the totality of the data that we will have generated in Europe and the US to guide clinicians. So it was a strategic choice not a regulatory driven decision. On your other question about timelines in the US, we’re not prepared to provide any guidance there. We will initiate the trials that I described both the adult malignant and the US ultra orphan pediatric trial in 2018, and as we finalized designs of those trials, particularly with FDA import we will provide more guidance on timeline.
Peter Lawson
And would you still be pushing for an approval in the non-malignant broad or you think you could kind of use the data from Europe to [indiscernible]?
Rick Fair
We will certainly eye that question, but our operating assumption is that the label reflect the trial that we’re doing in the US. So it will be a narrow label in the non-malignant pediatric population in the US.
Peter Lawson
Got you. Okay. And then just follow-me just around the route for CD19, is that kind of a partnership or something you are going to own yourself now?
Rick Fair
To date the clinical trial is being done through an academic collaboration and that trial will initiate this year. We did that because we thought it was an efficient way to do it, and we currently don't have a plan to take that forward to market, if we see surprisingly positive data then we may make a different decision, but at this point our primary objective is to evaluate CaspaCIDe in the context of the side effects associated with CD19 cars to inform future CAR T development, again if we see a really differentiated profile that we think is commercially viable then we will explore options to take that forward, but we’ve made no decisions on how we would do that.
Peter Lawson
Okay thank you so much. Thanks for taking the questions.
Rick Fair
Thank you, Peter.
Operator
Our next question comes from the line of Tony Butler of Guggenheim Partners. Please proceed with your question.
Unidentified Analyst
Hi, this is [indiscernible] on for Tony. Thanks for taking our questions. I just have two. First, if you can guys can discuss what the manufacturing process may look like and where you stand with that process if you have given a lot of thought to it? And the second question for 701, how - you've touched on it briefly, but maybe if you can talk more about specifically how the patient enrollment is going and what patients you have been recruiting specifically? Thank you.
Alan Musso
Sure. On your first question I don't think we have anything new to report on a manufacturing process. We have been - this year have been bringing on online our manufacturing facility here in Houston to supply clinical trial need here in the US, and we have partners to contract manufacturing organization partners in Europe who provide clinical supply and will support the initial launch in Europe. Our manufacturing process we continue to look for ways to automate that and have made quite a bit of progress in that regard. You know today our process and down it is about a two week process and we’re looking for ways to shorten that and continue to make it more efficient and cost-effective. On your second question, remind me.
Rick Fair
BPX-701.
Alan Musso
BPX-701. So enrolment goes one on BPX-701 trial. We have received quite a bit of interest from other sites besides the single centre trial, at the moment we’ve received quite a bit of interest given the unmet need in AML and MDS, the initial indications of that study for participation of that study. We may have to expand that once we clear the first dose cohort or two. No dose limiting toxicity is observed today and going well. We’ll keep you posted and look for provide initial safety information on that in 2018.
Unidentified Analyst
Yes.
Operator
[Operator Instructions] Our next question comes from the line of Wangzhi Li of Ladenberg Thalmann. Please proceed with your question.
Wangzhi Li
Hi good afternoon, can you hear me?
Alan Musso
Yes we can Wangzhi. Thank you.
Wangzhi Li
Hey thanks. A few questions, first of all for the adult image study in US, it’s not, I expect - this is not going to be a registration study right?
Alan Musso
Our intent will be to register it.
Wangzhi Li
Okay. And do you have any color on whether the control on and the point?
Alan Musso
Our current design is the Baltimore regimen plus or minus BPX-501. So it’s a standard-of-care treatment, randomized design.
Wangzhi Li
And looking for [indiscernible] PFS or?
Alan Musso
We’re still developing trial design and protocols. So, I will comment on employee. We certainly want to have that discussion with the FDA before we disclose that.
Wangzhi Li
Got you. And then for the pediatric and non-malignant these are often indication, is that going to be the different transplant protocol right, is that going to be the Alpha Beta depletion protocol?
Alan Musso
Yes.
Wangzhi Li
Okay good. Got you. Great. And then also question on the PBX-601, I think the first patient to do the [indiscernible], if I remember correctly the initial three patients they just have the sales without the rimiducid right? So, by now, I mean are those three patients now those that you expect to start aiding rimiducid to the patients?
Alan Musso
We have those three patients and we have seen the dose limiting toxicities. So, we will be advancing - we will be moving forward to the next step.
Wangzhi Li
Great. And any color or guidance on data readout or report?
Alan Musso
Just 2018, we haven’t [indiscernible]. It will really be based on what we see in the data and how enrolment proceeds from here. We will likely inform you our target meeting at our year-end call.
Wangzhi Li
Okay, got you. And maybe last question on your business development strategy, now you add a Chief Business Officer and you mention you identify a General Manager in Europe, so is your strategy still to - I mean you're still interested in partnering the European commercial rights or commercializing US on your own or is there different thinking now?
Alan Musso
We remain open and interested in value creating business development across the board. I think most of our proactive business development effort has been focused on our pipeline, not on BPX-501, and selection of a General Manager for Europe is our first step in preparing a commercial organization to launch that ourselves. That said we remain open if there were a value creating deal to do that with a partner we would deal with it.
Wangzhi Li
Okay, got you. Thank you very much for answering my questions.
Alan Musso
Thank you, Wangzhi.
Operator
Our next question comes from the line of Biren Amin of Jefferies. Please proceed with your question.
Biren Amin
Hi thanks for taking my questions, just on 601, I think you made some comments that you are looking at potentially evaluating that program beyond just pancreatic cancer, is there anything we can read through from your comments on that just from an efficacy standpoint in terms of patients that have been dealt so far and the fact that I think you mentioned pan cancer patients are tough to treat?
Rick Fair
Yes nothing to read through and results we wouldn't have expected at the [indiscernible] that we are apt to have seen activity and so we’re - we think we're not making any interpretation off of the small number of patients we have treated so far. It really comes back to the strategic review that we did looking for ways to de-risk our portfolio and certainly our belief is that we have a better chance of showing a positive signal and bladder cancer and more immunogenic tumor than we might in pancreatic cancer where the stronger component is problematic for T-cells. So it’s really more about biology than it is about reading data.
Biren Amin
On the 701, can you just talk about the enrolment challenges if any given, given patients are required to have both HLA-A two expression, as well as PRAME expression?
Rick Fair
Yes it is a higher screen fill [ph] rate, but we have seen ample patient volume. So there is a lot as you know AML patients are very high unmet need and so we’re screening quite a few patients. So, I don't think enrolment is a big challenge there.
Biren Amin
Got it. Thank you.
Operator
Our next question is a follow-up question from the line of Srikripa Devarakonda of Citigroup. Please proceed with your question.
Srikripa Devarakonda
Hi, thank you so much for taking my question again. Just a couple of small questions, first one is, so when can we get more clarity on the trial design in the US, the malignant setting? And also I know it’s a little too early, but is there anything you can tell us about your reimbursement plan either in the EU or the US, have you done any work on it and do you expect reimbursement to be captured under transplant or under treatment any color will be really helpful? Thank you.
Rick Fair
Sure. On your first question first, trial design in malignant patients as I indicated, our plan is to conduct a randomized trial. The standard of care for patients, adult malignant patients who are receiving a stem cell transplant who don't have a matched donor would be a haplo transplant using the Baltimore regimen. So our intent is to simply add to the standard of care, so it will be the Baltimore regimen plus or minus BPX-501 cells added back both side. So that’s the trial design that we’re working with, as far as providing more color about details about that endpoint sample size. We will do that after we had a chance to discuss the protocol with FDA and so when we come to you, we will have a trial design that we’re about to implement and that we’re comfortable that we will be registration or sending positive outcome.
Alan Musso
Transplant reimbursement, yes, so we’ve done quite a bit of market research there, our planning to execute of course is just getting built as we’re assembling a team, but I think what we’ve learned is that both in the US and in Europe the intent would be to reimburse BPX-501 as part of the capitated payments for a allogeneic transplant. And without getting into too much detail, I would say the European markets generally are receptive to additional payments given the value that they are seeing at our clinical profile I think I just described in my prepared comments that there is a good story for payers here in terms of reduced relapse, reduced hospitalization, reduced complications from GvHD and infections. And so they are enthusiastic about the data that we have so far and are open in Europe to providing add-on payments to the existing capitated payments to support reimbursement. In the US, we feel that the NCI centers and pediatric hospitals who have unique relationships with payers will have an opportunity to renegotiate rates based on valuable new therapy and so we will be working with them to ensure that that happens. I think our most challenging setting in the US quite frankly is probably Medicaid where stem cell transplant reimbursement rates are already very low and the opportunity for additional payments with the novel therapy are today non-existent, so we will - we have some time before we come to market and to do work on that from an advocacy and government affairs perspective, but that seems to be the one area that will present us with the most challenge. So for the bulk of the opportunity, I think we feel good that there is an opportunity to price a product and get fair reimbursement given the value of the profile.
Srikripa Devarakonda
Okay, that was very helpful. Thank you so much.
Rick Fair
Thanks.
Operator
There are no further questions at this time over the audio portion of the conference. I will now like to turn the conference back over to management for closing remarks.
Alan Musso
Thanks Tim. Thanks everyone for participating today. As always, I would like to thank our Bellicum team, our collaborators, and investigators for their efforts, and most importantly patients and families who participated in our clinical trials, who inspire our effort every day. Thanks and have a great evening.
Operator
This concludes today's conference. Thank you for your participation. You may disconnect your lines at this time and have a wonderful rest of your day.