Rick Fair - President & CEO Alan Musso - CFO Annemarie Moseley - COO & EVP, Clinical Development

Joshua Schimmer - Piper Jaffray Biren Amin - Jefferies Tony Butler - Guggenheim Securities Peter Lawson - SunTrust Robinson Humphrey Jim Birchenough - Wells Fargo Elemer Piros - Cantor Fitzgerald Reni Benjamin - Raymond James Wangzhi Li - Ladenberg Thalmann

Good afternoon ladies and gentlemen. Thank you for standing by. Welcome to the Bellicum Pharmaceuticals Fourth Quarter and Yearend 2016 Earnings Conference Call. At this time, all participants are in a listen-only mode. Later we will conduct a question-and-answer session and instructions will follow at that time. As a reminder, this conference will be recorded and available for replay. I would like to introduce your host today, Alan Musso, Chief Financial Officer. Please go ahead.

Thank you, Darren. Good afternoon, everyone and thank you for joining the call. With me today are Rick Fair, Bellicum's President and Chief Executive Officer; and Annemarie Moseley, our Chief Operating Officer and Executive Vice President of Clinical Development. Earlier this afternoon, Bellicum released financial results for the fourth quarter and yearend December 31, 2016. 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. 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, review and approval of 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 for the year ended December 31, 2016 filed with the Securities and Exchange Commission. And now I'll turn the call over to Rick.

Thanks Alan. Good afternoon, everyone. Thanks for joining us today. This is my first quarterly conference call as CEO of Bellicum having just joined six weeks ago, but before I launch into a review of our progress, I would like to give you a little color on what intrigued me about this opportunity in particular and why after I got to know the science and the pipeline and the team a bit better, I am convinced I made a great decision. What excites me about working in this industry is the ability to transform great science into life-saving treatments for patients. In cell therapies, I think we're on the eve of beginning to do just that, with products showing real promise in the clinic and advancing towards the market, but there's much left to do. On one hand, we've seem profound activity of T-cell therapies and liquid tumors, but associated toxicity that can at times be lethal. On the other hand, we've seen more limited activity in solid tumors where there remains so much unmet need. This is what really got me interested in Bellicum's technology platform. Novel molecular switches that enable us to control the cells, stimulating a targeted durable T cell response for greater efficacy when needed and modulating that response to manage toxicity. Now that I'm here Bellicum, I'm even more excited. We have a promising lead product in BPX-501 with highly encouraging data that we expect to launch in 2019. We've just brought our first ever controllable CAR-T and TCR products into their initial clinical trials and we have a really smart and committed team doing their best to make a difference for patients and their families. I really appreciate their dedication and the warm welcome I received from them. That's been great. With that, I'll turn now to our agenda for the afternoon. I'll start by giving you an overview of 2016 accomplishments and some recent updates. Then Alan will go through the financials and 2017 guidance. I'll wrap up before we take questions. As you read in our press release this morning, our team and our collaborators have made significant clinical and regulatory progress over the past year on BPX-501. As many as 70% of patients with hematologic malignancies or inherited blood disorders who require a potentially curative stem cell transplant don't have a match donor. These patients are sometimes offered a haploidentical or half-match transplant. Unfortunately, the outcomes with haplo transplants are much worse than matched, with higher rates of GvHD, a serious and sometimes fatal immune reaction and higher transplant-related mortality. In contrast, the data reported throughout the year from our ongoing BP-004 clinical trial demonstrated much more favorable outcomes, underscoring BPX-501's potential in this area of high unmet need. The most recent data presented at the BMT (Bone Marrow Transplant) Tandem Meeting last month, showed the cumulative incidence of transplant-related mortality remained very low with six-month and one-year survival rates of 98.4% and 97.2% respectively. No serious adverse events have been associated with the use of BPX-501 or rimiducid in the trial. The subset of 73 patients with six months of follow-up, experienced rapid immune reconstitution including full recovery and normalization of T cells, B cells and immunoglobulins improving their ability to fight infection of potentially life-threatening side effects of stem cell transplant. Rates of GvHD were low and all cases of acute GVHD resolved with administration of standard treatments or rimiducid to activate our CaspaCIDe safety switch. These data continue to be supportive of our approval path in Europe. We continue to enroll in the BP-004 trial toward the objective of treating approximately 120 valuable pediatric patients from European sites. We will also shortly initiate an observational trial in a comparable sample of approximately 120 retrospective and prospective patients receiving a matched unrelated donor or MUD transplant. The primary endpoint of these studies is event-free survival at six months, with events defined as transplant-related or non-relapse mortality, severe GvHD and serious infection. We expect both studies to be fully enrolled later this year and to submit applications to the EMA by mid-2018. In the U.S., we're pleased to report that we've made substantial progress in our ongoing discussions with the FDA on the design of U.S. registration trials. We expect to conduct two separate trials in pediatric patients receiving haplo transplants including a nonrandomized trial in patients with orphan inherited blood disorders and a controlled study in patients with blood cancers. We expect to finalize discussions with the FDA on both protocols in the second quarter of this year and begin enrollment of these trials during the second half. In addition, we plan to expand the evaluation of BPX-5012 adults with high intermediate risk AML, initiating a clinical trial later this year, funded in part by a $17 million award from the Cancer Prevention and Research Institute of Texas. The study will evaluate outcomes of patients who undergo a haplo transplant followed by a post-transplantation cyclophosphamide regimen with or without BPX 501. Post-transplantation cyclophosphamide or post-Cy is a widely used technique in adults for reducing some of the complications associated with haplo transplants. We believe that supplementing a post-Cy regimen with BPX-501 could improve outcomes for these patients. Turning to our CAR-T and TCR programs, we're now actively enrolling the Phase 1 trials of BPX-601 and BPX-701 at centers in the U.S. Starting with 601, our novel GoCAR-T product candidate, the first controllable CAR-T product to enter the clinic. It's differentiated by the inclusion of our proprietary IMC activation switch. We designed the product for enhanced proliferation and persistence, relative to traditional CARS, which we hope will translate to improved efficacy in the clinic. At the same time by including our switch technology, the level of stimulation and proliferation of these cells can be refined by administering rimiducid, providing a new level of control. BPX-601 targets solid tumors expressing prostate stem cell antigen or PSCA. Our first study as a nonresectable pancreatic cancer where there is great need for improved treatments and we intend to explore BPX-601 ask other tumors known to express PSCA as well. Turning to our other new clinical candidate, BPX-701 is our highest affinity T cell receptor product incorporating CaspaCID. Our initial study is in HLA-A two positive patients with refractory relapsed AML and MDS to test positive for PRAME or preferentially expressed antigen and melanoma. Our efforts to develop TCR therapies also included a research collaboration with Adaptimmune to evaluate our GoTCR technology with their affinity optimized T cells for potential co-development and co-commercialization. Our scientists are really excited about Adaptimmune's TCR optimization strategy and think while incorporating controllability, we have a great opportunity to develop best-in-class TCRs. Finally, our partnership with Leiden University Medical Center could also yield high affinity TCRs for development as we continue to expand our pipeline. Looking to the latter half of the year, we expect a Phase 1 trial with CaspaCIDe-enabled CD19 CAR to be initiated in patients with B-cell malignancies with initial data anticipated in 2018. The study is part of an expanded research collaboration to jointly develop novel controllable CARS and TCRs with our Italian partner, Ospedale Pediatrico Bambino Gesù (OPBG), one of the leading cell and gene therapy research centers and hospitals in Europe. The team at OPBG have been wonderful collaborators and we welcome this expanded relationship with them. We're very excited about the pipeline I just outlined and our collaborations to broaden it further, but we're not stopping here and our team continues to work to characterize and enhance our technology platform. We presented preclinical data on our GoCAR-T and GoTCR technologies at ASH in December, which continue to demonstrate advantages of our traditional CAR and TCR constructs. At AACR this April, we will present promising preclinical data on our novel dual switch technology, enabling control of engineer cells to both enhance efficacy and modulate toxicity when needed. We're excited about incorporating this technology in future products. With that update on our progress, I'll turn the call over now to Alan to review our financials and cash guidance for 2017.

Thanks Rick. Bellicum ended the year on December 31, 2016, with cash, restricted cash and investments, totaling $113.4 million compared to $150.4 million at December 31, 2015. In March 2017, Bellicum borrowed the final $10 million trench under its agreements with Hercules Capital. Based on our current operating plans, Bellicum expects the current cash resources will be sufficient to meet operating requirements through at least the first quarter of 2018. Projected cash outlays in 2017 include approximately $15 million for capital projects, primarily the completion of the build-out of in-house U.S. manufacturing facilities.' Our research and development expenses were $15.1 million and $51.3 million for the fourth quarter and year-end December 31, 2016 respectively, compared to $10.2 million and $33.6 million during the comparable periods in 2015. The higher expenses in the 2016 period were primarily due to an increase in BPX-501 clinical and manufacturing cost as a result of increased patient enrolment in clinical trials and an increasing cost related to BPX-601 and BPX-701, which entered the clinic in 2016 and an increase in general, research and development cost, including personnel cost and allocated overhead costs. Our license fees were $0.3 million and $0.6 million for the fourth quarter and year ended December 31, 2016 respectively compared to $3 million and $3.2 million for the comparable periods in 2015. 2015 license fees were primarily due to a new license agreement with Agensys, an affiliate of Astellas, as consideration for rights granted to Bellicum under an agreement related to BPX-601 product candidate. General and administrative expenses were $4.2 million and $16.9 million for the fourth quarter and year ended December 31, 2016 respectively, compared to $3.8 million and $12.7 million during the comparable periods in 2015. The increased G&A expenses in 2016 were primarily due to overall growth and public company related costs, including an increase in personnel, legal and accounting expenses and costs related to facilities, insurance and travel. Bellicum reported a net loss of $19.9 million for the fourth quarter of 2016 and $69.2 million for the year ended December 31, 2016, compared to a net loss of $16.8 million and $48.5 million for the comparable periods in 2015. The results included non-cash share-based compensation charges of $3.1 million and $12.3 million for the fourth quarter and year ended December 31, 2016 respectively and $2.5 million and $8.4 million for the comparable periods in 2015. At yearend, Bellicum had 27.2 million shares of common stock outstanding and I'll turn the call back over to Rick.

Thanks Alan. As you heard, we've made great progress in 2016. We expect 2017 to be another highly productive year for Bellicum and a pivotal one as we advance BPX-501 toward market and move our controllable CAR and TCR therapies into the clinic. With that, I'll open the call up to questions.

At this time, we'll be conducting a question-and-answer session. [Operator Instructions] Our first question comes from [indiscernible] of Citigroup. Please proceed with your question.

Hey guys, thank you so much for taking my questions. I'm wondering if you can give us some clarity on how we should think about EMA filing in terms of timelines and when can we get -- when can we expect to get an update on the entire dataset that you expect to file with the EMA and in the U.S., can you comment on the bars per data in both the malignant and non-malignant patients in the U.S. based on your talks with the FDA or is it too early to talk about that? Thank you very much.

Thanks [Freda]. So, to answer your first question, we would expect EMA filing in mid-2018 based on continuing to accrue our BP-004 and the comparative MUD trial that I mentioned. So, targeting a filing in mid-2018. As it relates to data, we will report data from BP-004 updates to the dataset both at EHA and ASH this year. So, you can see more current information on that. And I think it's pre-mature to comment on the expectations of the FDA. We've had good discussions. We expect to wrap up those discussions on protocols for U.S. approval in the second quarter of this year. We'll be able to update you mid-year.

Okay. Thank you so much.

Our next question comes from Josh Schimmer of Piper Jaffray. Please proceed with your questions.

Hi thanks for taking the questions. First is on the comparator a reminder for the European natural history study or for the FDA, I request in the malignant setting for a comparison, I guess MUD is the comparison in Europe. Can you talk about the choice of that as opposed to what might have been an easier borrow for evaluating in comparison to the haplo transplants approach and then for the FDA competitor arm, are you thinking it will be a much unrelated as well?

So, I’ll ask Annemarie to comment on the first question.

Hey Josh. So, with respect to the choice of the MUD compared to a haplo, the current haplo T-depleted program really has rather haplo in general with T-depletion has really had many different choices for T-depletion. The recent Alpha Beta TCR had a number of patients, but not widely used throughout either Europe and certainly not in the U.S. And currently many of the investigators really would not approach the Alpha Beta usage without the add back simply because of the reported issues within infection compared to those data from 501. When using the MUD competitor, one of the additional benefits in terms of gathering those data is both a comparability that is 501 comparability to the outcomes, but also a potential superiority, which we are also looking at of course based on the data and the use of those data for reimbursement as well. In terms of the FDA, while we are certainly in discussions with them on a strategy similar to that in Europe again, it's just a bit premature to talk about the final design and we'll be coming back to you with that.

Yes, Josh the only thing I would add to Annemarie’s comments is just that in addition to the reimbursement aspect, it's certainly attractive commercially to have demonstrated superiority to MUD. And I would say, based on our early read of the BP-004 dataset, we think there is some confidence in that.

Got it. And then as you think about the label, do you envision a [catch] label that will include [release] indications that you’ve studied as well as [one said] you haven’t studied right, the idea of a genetic disease type label or do you expect ultimately that label will reflect the patient populations that have been included in the clinical trial setting?

So, certainly based on our conversations to-date with the EMA, it would seem that it would for non-malignant and malignant label and especially considering the wide variety of in particular non-malignant patients who are and could be included on the trial and some of these are so rare that even waiting to get one on the trial would of course not be feasible. So, at this point, we are planning on a broader label. Of course, the finalization of that is going to be at the time of filing itself, but we have sort of had indications that it would be more board rather than limited at this point.

Thanks very much and congrats on the progress.

Thank you.

Our next question comes from the Biren Amin of Jefferies. Please proceed with your question.

Yes, thanks for taking my questions. Just on BPX-501 and I guess filing timelines in Europe for mid-2018, I guess that assumes patient enrollment by end of this year. Is that fair? Is that what’s really driving the MAA filing?

That’s right.

And then just on I guess the trial on AML What are your objectives for that study? What should we be looking for?

Yes, hi Biren. The real interest at this point in this particular setting in the post size setting and in the haplo is relapse. So, while these patients often have a fairly nice course throughout the transplant, the concern about the high relapse rate is one that certainly we hope to address with the 501 today. We’ve really focused a lot in the pediatric setting about -- on immune reconstitution, but certainly these cells have the ability for GVL effect and that’s really what we’re going to be focusing on in the adult setting where that has a great deal of value and in particular also in the post-size setting where relapse rate or relapse still remains a concern.

And then your discussions with U.S. FDA, do you expect that FDA would one -- some sort of controlled study in the malignant disorders.

Yeah, I think that's certainly the nature of the early discussions with them is that in the malignant setting, we would have control studies.

Great. Thank you.

Thank you.

Our next question comes from Tony Butler of Guggenheim. Please proceed with your question.

Thanks, very much. Annemarie, what is the relapse rate in the post side population, that’s question one. Number two is, can you speak to the enrollment of the MUDs EU? Where are you in that process? And number three if I may, if you, could you actually address how many centers currently you have open in Europe and would you care to actually share with us how many you think you might need to open in the U.S. under a protocol that the FDA would approve? Thanks very much.

So, in terms of order asked the current relapse rate in the post-side setting at one year I'll say that very generically exceeds 50%. That is relatively conservative number. For many centers, the numbers are higher than that. So, I think that there is a great deal of interest as I mentioned earlier and really beginning to -- begin to impact that unmet need and that's just a general number. Relatively to the enrolment of the competitor arm, we are currently at the committees for the retrospective and for the prospective arms of the pieces of that arm and that study will actually be at both the centers that are currently treating 004 patients as well as additional centers in terms of making up the retrospective data. So, the current centers that we have in Europe for the 004 study are GOSH in the U.K. New Castle in the U.K., which are the two referring centers for non-malignant diseases. The OPPG, which Rick mentioned earlier and we are opening up at the another [pending] in Madrid. So those are the studies that will both be highly contributing to both the patients and as well as the competitor arm. In the U.S. we currently today have about 16 centers who are participating on the existing 004 study. As we move forward into our later stakes trials both for malignant and non-malignant patients, we think that those centers are going to be adequate to enroll whatever we would need for that, although obviously, there are still some more interested studies. So, I think from that perspective, we are looking at -- it’s a pretty good load for the U.S. and then for Europe we are of course going to be enrolling additional countries and beginning of post market studies as we move on.

Thank you.

The only thing I would add just a point out for you and another, the retrospective and prospective nature of the MUD trial, so that the agreement with the EMA has to be able to gather data on MUD patients historically and then to enroll some additional prospective patients, which allows as we think to enroll this study in relatively short order and still make our filing timelines by mid next year. So that’s an important aspect I wanted to make sure you caught.

No. Thank you. I think I missed that question. Thanks.

Thank you, Rick.

Thanks Tony.

Our next question comes from Peter Lawson of SunTrust Robinson Humphrey. Please proceed with your question.

Annemarie, just thinking through the enrolment, how it's going, have you seen any slowdown or pickup? How is the pace being going and then as you think about a U.S. trial, do you think would proceed quicker or slower just thoughts around that would be great?

So, the enrolment at all centers is limited by their own transplant beds if you will. So, what we are seeing is a great deal of interest and I think right now, we are at our centers pretty much filling their beds up. So, the patients of course don’t have a matched donor. So, at this point in time, I think we’ve had a very as you know, a brisk enrolment and we expect that to continue not just to fulfil our trial needs, but actually because the physicians are excited about treating the patients who don’t have an option. In the U.S. I think that what we are seeing is that 2016 was a year of learning and growth for these centers and this year really, I think their approach has been sort of let’s treat the patients and get them going. So, at the moment, I think that we see really nice, very nice enrolment and recruitment. I would also say that we’re very excited that we’ve now opened up manufacturing here at our facilities in Huston and that really gets us a great deal of flexibility around as you can imagine in recruitment and enrollment from all of these various centers. So, there is really no limit and we really are seeing brisk interest from all the centers at this time.

Good. Thank you. And then the European data, would that be used in anyway by the FDA? Would that be included in the submission, help you with rolling submission.

Currently as you can imagine the data are very valuable from a planning perspective and from a safety perspective. Again, in our discussions today, we’re really seeing interest from FDA in these two planned trials and we’ll have a little bit more data on that probably by midyear.

Got you. Thank you. And then I may have missed this, but 701, 601 data is that kind of year end event?

Yeah, I think we might have little bit of safety data to report this year, but in particular on 601, given the really profound preclinical activity we’ve seen with IMC, we’re taking a relatively conservative approach to the dose escalation there and so we wouldn’t expect to have what we believe to be the optimally active dose in clinic until next year. So, I think may be an update late this year on safety of the early patients enrolled but more likely any efficacy measures would be a 2018 event.

But is that like a 40-ish day wait period between each patient.

Right now, there is in the first cohorts is a 30-day wait period and then for both 601 and 701, the first cohort and then in 601 in terms of -- there is another 30-day wait after the rimiducid again wanting to be sure that there is no late activity after the administration of the activator.

Perfect. Thanks so much. Thanks for taking my questions.

Thank you, Peter.

Our next question comes from Jim Birchenough of Wells Fargo. Please proceed with your question.

Hi guys. Thanks for taking the question and congrats on all the progress. I guess a few, the first one not to front run EHA, but since the update of ASH for the 004 study, has there been a need to use rimiducid in subsequent patients and is there anything to make you think you can't abort GvHD? And then on the post side study, do you have a similar question to the relapse rate data for rates of GvHD in that setting?

So, in answer to the first question, yes we have used rimiducid additionally and interestingly in a number of may be different clinical scenarios, we will be updating some of that at EHA and I would say that if anything we are, we continue to be surprised and excited about how well it works in a variety of different settings. In terms of the GvHD rate, in the post-side setting, as you can imagine, a little bit depending on the conditioning treatment that the patients receive and while the acute GvHD in many of the patients has been reduced from the early experience a number of years ago, the chronic GvHD if you just sort of look across the board, because there isn’t just a one post-side regimen, the chronic GvHD still does appear to be -- not insignificant. So, I think that, that is also something that in our design downstream, we will be working with the KOLs and the PIs on that, but in general those two features that of relapse and chronic GvHD have certainly been -- represent an unmet need within the post-side experience.

Great. Thanks for taking the questions.

Thank you.

Thanks Jim.

Our next question comes from Elemer Piros of Cantor. Please proceed with your question.

Yes, good afternoon Rick. Hello Annemarie. What I’d like to understand is in the European study did the size of the competitor arm increase from the previously anticipated 40 or you still spend at 40 patients roughly?

Yes, the 40-patients is the number we believe we need to enroll prospectively and so the 120 patients were clearing today reflects the total dataset, we aim for including the retrospective patients.

Okay. Thank you for clarifying that. Now if you think about it conceptually this European study which by the way its enrolling also in 16 U.S. centers with the competitor arm and with the comparison on the primary endpoint, in what way, shape or form would it be different then the control trial in cancer patients that the FDA would like you to conduct?

The end points that are being looked with respect to the --it’s a composite end point an agreement with the EMA. It in fact has a combination of both GvHD and immune reconstitution and of course survival. FDA in our discussion certainly has -- first of all, they view non-malignant outcomes and malignant outcomes differently. And in fact, from the standpoint, the overall outcome of the program is less of a composite and there are more specific primary endpoints. So, whereas in the EMA, the EU has spend that all patients regardless of disease benefit from the treatment of immune deficiency after post transplantation. So that way treatment of that immune deficiency reduces infection and therefore also enhances or reduces the TRM. FDA feels that because the overall outcome in malignant patients is not so much about the post-transplant period, but about disease treatment, they have a little bit of different view on this sort of paraphrasing. So, I would say that there is at the very highest level, a little bit of conceptual difference in terms of the product's view and its utility and all transplant versus its outcomes in the two different. So, I think that in Europe there is of course stratified analysis whereas prospectively designed at the FDA level. So, in principle there is not that much difference. It's just in this case really that just distinction between the disease outcomes in the malignant patients and there again in terms of all of the changes that are undergoing currently within the whole cancer field, there, I think interest in a controlled study versus looking at improvement of the transplant and enabling the transplant itself, which is sort of where at the EMA is coming to. So that’s where the data would be utilized in a safety manner and certainly stratified analysis could be used, but again it’s the overall primary design end point that is a little bit of a difference between the two agencies if that makes sense.

Yes, it does Annemarie. So, if you breakout the components of the composite, do you envision that the U.S. trials would look at different measures then survival, the severe inflections and/or severe GvHD?

Again, we will have more color on that for you in mid-year. but I think that it's safe to say that, that certainly survival in GvHD are probably more important endpoints with possibly infections and immune reconstitution representing more secondary endpoint, but again we’ll have a lot more color on that once we finalize our discussions.

Yes, and just one last point Annemarie, if you look at a non-malignant patient that is being treated in the European study in one of those 16 U.S. centers and if you look at the treatment in the upcoming U.S. trial of a non-malignant patient, would there be any difference between the treatment and/or devaluation of those apart from the composite nature of the endpoint and I’m focusing on non-malignant setting?

At this point, we do not see any difference in the overall design of the trials or the execution of the trials from where they have occurred to date.

Okay. Thank you so much.

Thank you.

Our next question comes from Reni Benjamin of Raymond James. Please proceed with your question.

Hi, good afternoon guys and thanks for taking the questions. Maybe for Annemarie, in the BMT tandem meeting, you guys announced some data in about 122 patients, but I didn’t get the breakdown between malignant and non-malignant cohorts and I guess in particular I’m looking for may be a GvHD and OS breakdown, which -- did they track fairly consistently or they, or as one really driving the overall benefit?

I believe that actually they’re almost recently equal that at the ASBMT that was a combination of both U.S. and European data and actually in terms of both GvHD and well, the overall survival was a tiny bit less in the malignant just obviously because there were a few patients I believe three or four who did relapse early. And in terms of the GvHD I believe they were approximately equal. So, I would say out of that 122, probably there is around 60 or 65 may be in the non-malignant and then 60 in the whatever it is, 62 and 60 in the malignant. So, it’s really very, very similar breakdown. Obviously, there is some center differences because certain centers are really primarily focused on their referral for their non-malignant patient, but if you take all of the patients together, we are not seeing a really big difference in terms of outcomes driven by disease in the early term points other than relapse, which of course occurs only in the malignant. So, from that perspective, the data are utilized for both as a whole and then there is also breakdown, which we also broke down the outcomes at ASBMT for the various diseases.

Reni, may be another thing I would add is that with the one year overall survival of over 97%, probably the breakdown of the mortality by disease areas are statistically meaningful at this stage.

Fair enough and then just thinking about the U.S. game plan, can you give us, should we be thinking about the two separate studies kind of at the same scope of the ongoing European studies or about 100 patients each and how long do you think one start this could take?

I don’t think we're ready to comment on that. I think it's important for us to wrap up the discussion with the regulator first and so I prefer to comment on that in the second quarter once we have a chance to nail that down with the FDA.

Okay. And then just maybe finally on the CAR T side, especially with 701 when you mentioned that you can be looking at patients testing positive for PRAME, can you give us some thoughts regarding what sort of expression you think might be needed and are there cut-offs that you are looking for?

So, we actually described this at the RAC that our collaborators in Leiden looked at a large number of AML samples. So, this is really initially specific to the AML in terms of copy number and some indications or sample copies that were greater than 100 to 200 copies, those were almost exclusively in the tumors and represented a positive signal. Obviously, some tumors went far, far higher than that and that is really our initially cut-off for expression. And so, from that perspective, we've actually looked at a number of samples and have been able to repeat that sort of cut-off and it's a pretty nice cut-off. So, whether or not there are ultimately differences in response based on very high expression or intermediate or low expression, again those are things that we would see coming out of the trials, but currently we have that as a sort of nicely cut-off between 100 and 200 copies.

And do you have a sense Annemarie in this patient population about how many patients would meet that criteria?

At this point, there is both expression of PRAME and expression of the A2. A2 is between 30% and 40% and then a PRAME has been sort of on the order of 40% to 60%, but again we are just continuing to look at the patient population because they have to first be refractory AML of course and then they have to get to the A2 expression first and then they have to get to the PRAME. So, I think those are the data that we're collecting and probably part of that package that Rick was talking about maybe later in the year, beginning of next year.

Yes. And I think because this is not an antigen commonly tested for, I think we'll get a relatively natural selection in the screening. So, I think we'll learn I think from the screening process about how prevalent both PRAME and A2 in this population.

Right. Thanks for taking the questions.

Thank you.

Thank you.

Our next question comes from the Wangzhi Li of Ladenberg. Please proceed with your question.

Hey, thanks for taking my questions and my congratulations to all the progress. So, I wanted to clarify about the BP-004, I think you mentioned about 140 patients needed for the MAA finding right. And out of the BMT tandem meeting, I think 122 patients are treated. So, I wonder like how many among those 122, how many are European patients versus a U.S. patients and then how many European patients are needed to finish -- to satisfy the 140-patient requirement? I guess the 140 patient means just for European patient right.

Yeah, why don't I take a stab at answering that and Annemarie can jump in if I miss something. Our target for both the 004 filing for Europe and the MUD competitor trial are 120 patients approximately in each of those studies and not 140 but 120. Currently the 122 patients, the large majority of those patients have been treated in European centers although the U.S. sites are enrolling now relatively rapidly. So, we’ll see that U.S. enrolment pickup, but I would say something in the order of 100, I’m looking at Annemarie our European patient so far. So, as you can hear from that, we're not far away from enrolment on 004 necessarily, but the MUD trial we're just initiating now as you know and have targeted 40 perspective patients there.

Right and just to clarify also just so that everyone doesn’t run around, run away and start doing the math, as you remember or might remember, this is an extension of the original 004 trial, which included dose escalation and included a number of different, sort of a variety of different patients. As we have reached agreements in the latter part of last year with the EMA, we did revise the protocol according to some of their suggestions, so while the number is indeed very close to 100, there are few patients such as the dose escalation and some of these other evaluable patients, non-evaluable patients who are not going to be included. So certainly, we will keep you posted on the progress with that as we get there, but I don’t think it's possible really to just say, to look at the numbers and say okay 120 minus 100 because it's not quite that simple.

Yes, the only thing I would add is yes, we were confident we can accrue the trial this year.

Absolutely.

Okay. Great. That's very helpful. And also, the question on the U.S. trial, you are finishing discussion with the FDA when you say the controller trial in bladder cancer patients, assuming that includes both the ALL and AML, pediatric patients?

Yes, that is correct.

Are there any kind of discussions in terms of distribution among these two indications disease or is random?

That’s still all under discussion and so Rick will be updating you in the middle of the year on that.

Okay. Got you. I think all the other questions are already asked. So that’s all my question. Thank you very much.

Thank you.

Thank you.

[Operator Instructions] There are no further questions at this time. I’d like to turn the call back over to Mr. Fair for closing remarks.

Thank you, Darren. Before concluding, I would like to acknowledge and thank all of the team here at Bellicum, our collaborators and investigators around the world for their continued hard work and dedication, our investors for their continued support and most importantly the patients and families participating in our clinical trials without whom none of this would be possible. It’s all of you we aim to serve. Thanks for participating today. We look forward to updating on our progress. Have a great evening.

This concludes today's conference. You may disconnect your lines at this time. Thank you for your participation.