Alan Musso - Chief Financial Officer Rick Fair - President and Chief Executive Officer William Grossman - Chief Medical Officer

Greg Harrison - Citi Biren Amin - Jefferies Wangzhi Li - Ladenberg Thalmann Peter Lawson - SunTrust Robinson Humphrey Reni Benjamin - Raymond James Elemer Piros - Cantor Fitzgerald

Good afternoon, ladies and gentlemen. Thank you for standing by. Welcome to the Bellicum Pharmaceuticals Fourth Quarter and Year End 2017 Earnings Conference Call. At this time, all participants are in a listen-only mode. [Operator Instructions] As a reminder, this conference will be recorded and available for replay. I would now like to introduce your host today, Alan Musso, Chief Financial Officer. Please go ahead.

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, and Bill Grossman, our new Chief Medical Officer. We will be using slides on today's which can be viewed on our live webcast in the investors and media section of bellicum.com. Earlier this afternoon, Bellicum released financial results for the fourth quarter and year-ended December 31, 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 Web site. 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, 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 Factors section of our annual report on Form 10-K for the year ended December 31, 2017 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. In addition to Alan, I am pleased to be joined today on the call by Dr. William Grossman, our Chief Medical Officer. Bill joined Bellicum in February from Genentech where he played a leadership role in their global clinical development group focused on cancer and immunotherapy. He also trained and spent his academic career in pediatrics, stem cell transplantation. So he has a terrific background for what we are doing at Bellicum. He has hit the ground running and today he will be reviewing some results from ongoing studies and be available during the Q&A session. Over the past year we made substantial progress towards our vision of delivering cures through controllable cell therapy. We moved three new CAR-T and TCR programs into clinical trials. We continued to validate and enhance our molecular switch platform for control, including initiating plans for the first clinical trials of dual switch CAR-T cells in 2019. We progressed the development of BPX-501 and are on track for our first European MAA filings in 2019. Lastly, we strengthened our organization with a number of key leadership hires and an expanding clinical team. Today, we will summarize the progress we made in each of our programs and will report on some promising new interim data on BPX-501 and our first case report on BPX-601. But first I would like to highlight how each of our projects fits into our strategy to be the leader in controllable cell therapies, to expand the therapeutic window for these treatments and extend the impact that they can make on cancer treatment. Each of our clinical programs is a potential product in its own right but also plays an important role in validating our platform BPX-501, BPX-701 and our CD19 CAR-T collaboration, all serve to evaluate how our CaspaCIDe safety switch can be used to manage the toxicities of T cell therapies. Our BPX-601 program will provide the first clinical evaluation of our inducible MyD88/CD40 activation switch, what we call iMC. Our ongoing study will allow us to optimize how we use iMC to driver T cell expansion and persistence and hopefully enhance efficacy. In our next generation of CAR-T projects we have designed both switch technologies into the same construct. To use an automotive metaphor, we have incorporated both the gas pedal and a brake to enhance the performance of these CAR-T cells. We are excited about the progress we have made in validating these technologies and look forward to generating the first clinical experience of dual switch CAR-T cells next year. With that as background, I would like to take you a little deeper into each of our programs, summarize our progress and preview what to expect in the coming year, starting with our most advanced candidate, BPX-501. Clinical results to date provide strong support for their potential of BPX-501 to improve treatment outcomes for patients undergoing a partial match haploidentical hematopoietic stem cell transplant. We recently completed enrollment in the treatment arm of the European BP-004 clinical trial in children with malignant and non-malignant diseases, and expect to have top line results at the end of 2018. Comparative data from the C-004 observational study in children receiving a matched unrelated donor or MUD transplant without BPX-501 will follow shortly thereafter. And we continue to expect filing marketing authorization applications for EU approvals of BPX-501 and rimiducid in 2019. Before I cover our plans for BPX-501 in the U.S., just a quick update on the FDA clinical hold. We submitted a full response to the agency last week. This response included our proposed changes to protocols with guidelines for comprehensive monitoring and management of neurologic adverse events, which are known risks in patients undergoing allogeneic stem cell transplant. We are optimistic that the changes will satisfy conditions for removal of the hold and look forward to hearing from the agency in the coming weeks. Earlier today we announced encouraging interim survival data from the BP-004 study in pediatric patients with AML and with primary immunodeficiencies or PIDs. Bill will share more detail and insight into these data later in the call. The takeaway is that the results from BP-004 continue to be impressive and confirm our belief that BPX-501 represents an important advance for patients and a significant commercial opportunity. Based on these strong clinical data. We are working with our investigators and the FDA to develop protocols for potential registration studies. We recently convened adult and pediatric advisory boards at the bone marrow transplant Tandem meetings in Salt Lake City, and gained valuable insight from opinion leaders as we finalized protocols for review with regulators in the coming months. As a reminder, we are planning a randomized study in adults with AML to support global filings and are evaluating pediatric studies in either AML or PIDs to serve as the basis for registration in the U.S. We will be able to provide more specific guidance at mid-year on trial designs and estimated timelines. Turning to our CAR-T program. You may have seen in this afternoon's press release that we reported the first case of the use of our iMC activation switch in humans. As observed in our preclinical research, we saw robust CAR-T cell expansion with the administration of rimiducid to activate iMC. While this is obviously very early data, it's highly encouraging sign that the novel technology is working as intended in a solid tumor patient. Bill will talk more about the case shortly. From here our sights are set on solidifying in vivo proof of concept as we continue to enroll and treat additional patients. We expect to report initial findings in medical meeting later this year. The study, as you may recall is in adults with nonresectable PSCA expressing pancreatic cancer, one of the more challenging cancers for T cell therapies or other treatments. We expect to expand the trial to other PSCA expressing cancers later this year. As for our Phase 1 study with BPX-701, our high affinity T cell receptor targeting PRAME incorporating CaspaCIDe, we continue to recruit patients with AML and MDS, and our team is working to add more clinical sites to increase the rate of enrollment. We expect to report preliminary Phase 1 findings for BPX-701 at a medical meeting later this year. While we have previously reported positive data on our CaspaCIDe safety switch from BPX-501 studies, we are just beginning to evaluate its potential impact in managing the acute toxicities of CAR-T and TCR therapies. The first patients have been treated in a Phase 1 trial of a CaspaCIDe enabled CD-19 CAR-T being conducted at OPDG, a leading European pediatric research center and hospital. We look forward to the results which may help us better characterize the profile of CaspaCIDe for future CAR-T programs. Now I will turn the call over to Bill to give you more color on the 501 and 601 data.

Thanks, Rick. First I would like to start off by saying just how excited I am to be here at Bellicum. My whole career has been focused in the areas of transplantation and immunotherapy and oncology. During my time in academics and over the past decade in the biopharmaceutical industry, I had focused on bringing transformative and hopefully curative therapies to patients with life threatening immune deficiencies and cancers. I believe Bellicum is at the cusp of driving the next generation of innovative controllable cellular therapies in both stem cell transplantation and the CAR-T, TCR space, and I am very thrilled to be here on the team. With that, I would like to now share with you some of the emerging data that Rick briefly mentioned in his review. First, you may have seen in our press release this morning, recent top line data from our interim analyses of clinical outcomes in a pediatric AML cohort of BP-004 study. The cutoff of these analyses was January 29 of this year. The two graphs shown here demonstrate the [indiscernible] estimate of both relapse free and overall survival in our cohort of 38 AML patients, which included nearly two-thirds of patients in CR2 for their second remission post-chemotherapy or transplant failure. As you can see, the relapse free survival estimate is 91.5% and the overall survival estimate is 97.3% with a median follow up of one year. These results appear favorable relative to one year rates reported in the literature for pediatric AML patients undergoing alternative donor transplants of approximately 60%-80%. We also present top line outcomes with our primary immunodeficiency cohort which had a wide range of PIDs including severe combined immunodeficiencies with [indiscernible] syndrome, chronic [indiscernible] disease, [indiscernible], and many other rare immunodeficiencies. As shown here, this cohort in 59 pediatric PID patients undergoing haploidentical identical transplants followed by infusion with BPX-501 T cells demonstrated disease free survival estimate of 88.1% and an overall survival estimate of 88.6% with a median follow up of one year. We believe these are strong results and also warrant further investigation. Having spend much of the latter part of my academic career developing the first statewide newborn screening program for SCIDs in Wisconsin, I am personally very excited to see these results in PID patients that don’t have a matched donor. Over the last ten years, 47 of the 50 states as well as Puerto Rico have adopted a new screening program for SCIDs, which is obviously a very critical first step to help identify these patients and infants earlier. However, only 50% of these infants will have a match related or unrelated donor. This realty makes us imperative to develop alternative donor approaches such as haplo-transplantation with BPX-501 cells, which could provide similar outcomes to those with matched related transplants, while minimizing transplant associated toxicities. Turning to BPX-601. We recently initiated rimiducid dosing in our BP-012 study of BPX-601 GoCAR-T cells. BP-012 is a Phase 1 study is in nonresectable pancreatic patients with failed standard therapies. This Slide presents an overview of our clinical trial design. As a reminder, we were first required to dose patients sequentially without rimiducid in the lead in phase or cohort 0 to evaluate the safety of the BPX-601 cells by themselves. We completed this cohort with no observed dose limiting toxicity. We are now I the safety evaluation phase combining escalating, or potentially be escalating cell doses of BPX-601 with a single dose of rimiducid on day 7 post cell infusion. The next Slide shows the peripheral vector copy number analyses for the presence of [MTU] [ph] BPX-601 cells. As demonstrated in the top row, our first three patients [indiscernible] cells alone had detectable low level of BPX-601 cells. The peak levels of 200 to 2000 vector copies around one week post-infusion. Cell infusions occurred in day zero and are noted by the blue arrow. Moving to our first patient dosed with rimiducid at the bottom of the Slide, you can again see the initial low level peripheral detection of BPX-601 cells after infusion. However, following a single dose of rimiducid on day seven as noted by the orange arrow, we observe nearly a 20-fold increase in vector copy numbers approximately one week after rimiducid dosing. This patient continued to be followed for safety and potential clinical activity. These results demonstrate an early proof of concept for the iMC activation switch with plans underway to expand the trial to other PSCA expressing cancer types later this year. So while these pharmacodynamic results are very preliminary, seeing such high levels of BPX-601 cell expansion for just a single dose of rimiducid is very encouraging and could be an important step to achieve CAR-T cell efficacy in solid tumors. And with that, I would like to hand back to you, Rick?

Thanks, Bill. Turning finally to the last two areas for discussion today, our platform and our organization. I have been with the company now for just over a year and I am more confident than ever that we are on the right path. I believe that the enormous promise of T cell therapy can be expanded by the ability to control therapeutic cells in vivo. To be able to administer cells, activate them to enhance efficacy when needed and then manage acute toxicities when they occur. I also believe we have an industry leading platform that has the potential to do exactly this and we continue to optimize it. We reported exciting preclinical data on our novel dual switch technology at several scientific conferences in 2017. We see dual switch as a combination of the current platform and have two dual switch product candidates in active preclinical development that we intend to move into the clinic in the first half of 2019. We see many areas where we can apply our powerful cellular control technologies to create first and best in class therapeutics. We have a broad set of opportunities and have strengthened our organization to capitalize on them. In addition to Bill Grossman, we have added key leadership talent to our clinical and quality teams in recent months and recently appointed seasoned industry executive Ed Harrigan to our board of directors. Alan Smith, who we promoted last year to Executive Vice President of Technical Operations has made a big impact and has led the completion of the build out of our cell and viral vector manufacturing facility in Houston. Finally, Greg Naeve, who joined last year as Chief Business Officer has contributed to the sharpening of our product strategies and has been hard at work renewing Bellicum's business development efforts. The CAR-T space has a lot of opportunity and with our platform and our integrated capabilities, I believe we are well positioned to forge collaborations to capitalize on the broad potential of our technology. I am proud of the progress we have made over the past 12 months and I am confident we have the team in place to execute our strategy for a highly productive year ahead. With that, I will turn it over Alan for the financial discussion.

Thanks, Rick. Bellicum reported a net loss of $22.2 million for the fourth quarter of 2017 and $91.8 million for the year ended December 31, 2017, compared to a net loss of $19.9 million and $69.2 million for the comparable period in 2016. The results included non-cash share-based compensation charges of $3.4 million and $13.6 million for the fourth quarter and year ended December 31, 2017 respectively, and $3.1 million and $12.3 million for the comparable period in 2016. Research and development expenses were $14.3 million and $65.6 million for the fourth quarter and year ended December 31, 2017 respectively compared to $15.1 million and $51.3 million during the comparable periods in 2016. Higher expenses in 2017 were primarily due to increased clinical trial costs, particularly for BPX-501, start-up costs related to our in-house manufacturing facility and our contract manufacturers in Europe, and increased personnel and consulting expenses. General and administrative expenses were $5.1 million and $21.1 million for the fourth quarter and year ended December 31, 2017, respectively, compared to $4.2 million and $16.9 million during the comparable periods in 2016. The increased G&A expenses in 2017 were primarily due to our overall growth, including an increase in personnel related costs, facility costs, and other administrative costs. On the balance sheet, as of December 31, 2017, cash, restricted cash and investments totaled $106.5 million. In the fourth quarter of 2017, we paid off our Hercules Capital debt facility with a $35 million loan from Oxford Finance. This new loan provided approximately $2.1 million in additional liquidity, interest-only payments until February 1, 2020 and a lower interest rate. Based on current operating plans, Bellicum expects that current cash resources will be sufficient to meet operating requirements through the first quarter of 2019. And I will turn the call back over to you, Rick?

Thanks, Alan. As you have heard today, we are making strong progress across the pipeline and operations and we have a strong team in place. In 2018, we expect four key data readouts across our programs, plan to initiate two new studies, expanding the 501 opportunity, and plan to expand our 601 trial to explore additional PSCA expressing cancers. In addition to these milestones, we will come back to you this summer with the targets and indications for the dual switch product candidates we intend to move into the clinic early next year. We are excited about where we are headed and look forward to keeping you updated on our progress. With that, I will now open the call for questions.

[Operator Instructions] Our first question comes from the line of Robyn Karnauskas of Citi. Please proceed with your question.

This is Greg Harrison on for Robyn. As far as potential partnerships that you could enter into, do you have any discussions with partners on what they are expecting from your activation switch technology and do they see activation of T cell expansion as being sufficient or is there expectation for persistence of the cells as well? And just looking forward to what sort of results you would be expecting in order to have that interest from potential partner.

Sure. Thanks, Greg. It's Rick. I would say that both the expansion and persistence are of interest both to us and to potential partners. I think CAR-T studies have demonstrated that more T cells for longer correlates with efficacy and so being able to create expansion of T cells and causing those T cells persist is both important features. And so we are excited to see this early data of expansion in a solid tumor patient.

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

Maybe if I could just start on 601 and the one patient, cohort 3, that experienced a 20-fold expansion after rimiducid. Can you just tell us a little bit about how the patient is doing or are there any adverse events that were seen after the cell expansion.

Hi, Biren, it's Rick. We are not going to comment today on the clinical status of the patient. Obviously, it's very early and I think our interest today was to share what we have seen in terms of biologic proof of concept to show T cell expansion in vivo since it's our first experience with that. We will present clinical results of this patient and the remaining patients in the cohort at a meeting later this year.

Got it. And so is that cohort continuing to enroll or have you gone on to the second cohort?

The cohort continues to enroll.

Got it. Okay. And then just on PRAME. Can you just tell us where you are at in terms patient enrollment and what type of data we could expect later this year?

Sure. BPX-701 enrollment is ongoing. It's a classic 3 by 3 dose escalation and de-escalation trial. I won't comment on exact patient numbers but I will say that enrollment has been a little slower than we hoped, keeping in mind that we are talking about relapsed and refractory AML and MDS patients at a single center. We are also talking about a patient population where there needs to be both PRAME positive and HLA-A two match. So we are in the process of engaging new sites to expand the number of sites that will be screening patients and look in the hopes of accelerating enrollment and we will record on the total patient experience, whatever that is, at a meeting late this year.

Our next question comes from the line of [Daniel Tavasoto] [ph] of Guggenheim Securities. Please proceed with your question.

Do you already have an idea which other cancer types you plan to enroll for BPX-601 that are PSCA positive? And the second question would be, since there is an increasing interest in allogeneic CARs, I was wondering if you have an interest in developing an allogeneic asset or moving some of your GoCAR technology into an allogeneic CAR-T cell. Thanks.

Sure. Answer to your first question, we are evaluating primarily GU and GI tumor types that I believe to express PSCA. And as we finalize that based on our understanding the expression there and interest from investigators, we will communicate more. But that’s the playing field. As to your second question about allogeneic CARs, we do have an interest in that area but no specific programs to disclose today.

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

Good afternoon. It's [Nick] [ph] in for Jim this afternoon. Congrats on the progress and all the data. Maybe just starting off on the interim data you presented from 401 on AML and PID. I understand that those are the diseases that you are interested in. Other data in [ALL] [ph] and hemoglobinopathies similar -- is the data you presented today representative of those other diseases?

Thanks, Nick. You know outcomes by patient types vary. So I don’t know what you mean by representative. I think what I would say is our overall data set is consistent with previous reports but I would say maybe in the case of the AML data that the overall survival and relapse-free survival data there are particularly compelling.

Okay. Because I think you have previously reported I think a relapse rate of around 15% in cancer patient. Is that correct?

That’s right.

Okay. And then you know with the FDA clinical hold, as you have discussed the data with your investigators. Has this changed the level of interest they have for sickle cell or beta thalassemia where perhaps the imperative for transplant is not as strong as in PID.

No, I don’t think we have heard specific concerns about the FDA clinical hold or neurologic adverse events from investigators. I think what we have heard from them is that these are adverse events that are commonly seen in allogeneic stem cell transplantations.

Okay. And then on 601, what is the duration of activity you would expect from a single infusion of rimiducid?

Bill, you want to tackle that?

No, we are still collecting that data so we are obviously very early on in collecting the data. We haven't enrolled any more patients yet that are scheduled to be infused soon with rimiducid. So right now we just don’t have any other data to comment on.

Okay. And then in terms of biomarker changes. Can you comment on the biomarker changes occurred in that patient that maybe indicative of CAR-T cell activity?

Yes. I think what, today we are going to stick to the chart that we showed T cell expansion. Again, that was our first trigger that iMC is working in vivo. We look forward to reporting additional biomarker data and clinical data at a meeting later this year.

Okay. Thanks. And then just last one, on that particular chart. I don’t if they are arrow bars or noise bars, is this from a single patient and the bars reflect multiple samples or is this from more than one patient? I am talking about the 20-fold expansion.

Yes. That’s a single patient and the arrow bars represent analyses multiple now seen on the same sample. So that’s the bar [indiscernible].

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

I would also start with BPX-601. So very encouraging to see the expansion. I wonder how you -- I mean in the figure you only showed a one detection of the expansion around day 14, I wonder how you also check the cells [data] [ph] after that, let's say day 20 or something like that.

Yes. Good question, Wangzhi. We did test for samples weekly at that time point. So you see what looks to be one really remarkable high figure. We evaluated samples in the first 24 hours after the administration of rimiducid and then weekly thereafter. So what you can infer from that is that we did see those cells disappear later in the time points on this graph. That is not necessarily particularly surprising. We will certainly monitor that in future patients, and bill, maybe you want to comment on what couple of our theories might be there.

So we are still collecting the data to understand the pharmacodynamic results that you have seen here. And so one of the potentials that we have seen here is the [expedited] [ph] cells are being [marginated] or potentially even though its [indiscernible] tumor micro environment. And so hopefully we can obtain tumor biopsies that will answer a lot of the questions. Alternatively, it could be that we need to re-dose rimiducid to continue to see the activation of these and expansion of these cells. And in fact we have pre-clinical data where we have done this and have shown the ability to re-dose rimiducid multiple times without parent T cells exhaustion. So we will be collecting all that information as we go along.

Okay. That’s is helpful. But just to clarify, so in the figure, like I said, the day, day 20 something, or on day 20, you are not showing any [indiscernible] there or is the cells all gone. I just wanted to clarify because you said you measured the results there but in the figure they are [masked] [ph] in there.

The cells are no longer detectable in the peripheral blood so as you heard from Bill, one theory is that they could be marginated or penetrate the tumor micro-environment. Another theory is they are gone in which case we may need to use rimiducid repeatedly to generate prolonged persistence.

Got it. And just maybe remind me what is the half life of rimiducid?

Yes. It's less than 12 hours.

Got. Okay. Great. That’s helpful. And then for PBX-501, the press release early this morning, I want to clarify one point. In the press release you said the relapse free survival and overall survival is a 91.5% and 97.3% for PBX-501 compared to 60% to 80% for the alternate donor transplant, right. What exactly is a alternative donor transplant?

It's a basket of papers we glean through the [search] [ph] that we refer to haploidentical core blood or matched unrelated donor transplants.

So it's a mixture of different types. Okay.

Correct. Correct. We really looked at the literature expansively beyond matched siblings transplant.

Got it. And is there any data there regarding like alpha, beta TCR lung transplant in pediatric AML. Was the relapse-free survival and overall survival one year and any historical data for that?

I don’t believe any data has been presented on AML with alpha beta T depleted haplo alone. None that we could find.

Okay. Got it. Last question is, the U.S. trial, you mentioned that this year you would initiate the pivotal U.S. trial in adult AML and then also pediatric AML or PID. When you say pivotal, that’s already kind of in front of the FDA or is the hope to be pivotal?

Our plan is that there will be pivotal and we will be engaging, in the case of the pediatric study, the FDA in discussion on that in the coming months. In the case of the adult trial, both the FDA and the EMA on the design to those trials to ensure they would view them as adequate for registration. That’s why we will report more specifically on designs that may be year after we have had a chance to talk to both health authorities.

Our next question comes from the line of Pete Lawson of SunTrust. Please proceed with your question.

Rick, just on 701, when could we expect that data? Kind of a midyear event or year-end event?

I would say later in the second half of the year events.

Got it. Thank you. And then CD19, I know it's not your trial but do you have any sense of when we could see an update around that or how long will this going and if there is any kind of interference from kind of approved CD19 out there.

Yes. The trial is being conducted in Italy so from what we have heard there hasn’t been any barrier to recruitment and they have already enrolled multiple patients. So we know that. I would say the earliest results we might expect since enrollment just started at the beginning of this year, would be late 2018.

Perfect. Thank you so much. And then just any further details around, I guess, the clinical hold to response you sent back to the FDA and just any color you could around that hold would be appreciated.

Sure. I think as we have previously communicated, the clinical hold, basically the terms of the clinical hold, the FDA really requested us to update our protocols to clarify for investigators monitoring and management of neurotoxicities or neurologic adverse events that occur in these patients. And so the teams -- we have three active BPX-501 protocols in the U.S. The team has updated these protocols. The team has updated our investigator brochure which discloses the potential risk of these types of adverse events. And it has updated our patient inform consents and has submitted all of that to FDA for their feedback. So we look forward to hearing back from them soon. We believe we have responded fully to everything that the FDA asked and so feel like we are optimistic that the hold will be lifted in short order but we need to wait to hear from the FDA.

Got you. Thank you. And then just finally, just from the data this morning for the AML, PID. Have you got any sense of what the FDA is going to want as regards to endpoints for AML, PID.

Yes. From previous regulatory discussions, I think in AML a relapse or survival type of an endpoint would be appropriate. And I think in the case of PIDs, more transplant related outcomes like, similar to the model that we have in Europe would be more acceptable. So shorter each [month] [ph] endpoint focused on the outcome of the transplant versus the cure of the disease, if you will.

And do you think there would be any change in the patients you would be enrolling for a pivotal?

Maybe I would look to Bill to see if he has any thoughts on that?

At this point we don’t have any other considerations that we believe that patient populations are likely the once we will be pursuing in that pivotal trial designed for adults and as well pediatrics. So those discussions we will need to have with the authorities to clarify patient population.

[Operator Instructions] Our next question comes from the line of Reni Benjamin of Raymond James. Please proceed with your question.

Congratulations on the progress. Maybe just starting off with 004. Can you remind us like how many patients have been recruited and can you give us a relative breakdown of the malignant versus non-malignant patients on the 004 study.

So the malignant, non-malignant breakdown as of the last reported presentation of the overall cohort was 40% malignant, 60% non-malignant and I believe the enrollment is maintained along those lines. At this point in Europe we have recruited a total of approximately 190 patients but I will point out that not all of those patients unfortunately survived to receive their transplant and BPX-501 cells. But that’s in terms of total enrollment, that where we are. And I would also point out that we have closed that trial to screening to we are no longer enrolling patients. We believe we have all we need to support our filing.

And can you give us a sense as to how many patients are sort of evaluable?

No.

Okay. And then just...

I think we previously communicated that we need a minimum of 100 evaluable patients based on our powering of the comparison to the MUD cohort and we feel confident we will hit that number.

Got it. That’s okay. And then just regarding the MUD cohort. Can you give us a sense, I know you mentioned that the data would come out certainly after the [probably] [ph] one data, but can you give us a sense as to how enrollment is going and what's the split between the retrospective and prospective groups might be.

Enrollment is going okay. So we have our sites opened and are actively recruiting patients. So our hope is that we will have the trial completely accrued when we provide a midyear update. And that would of course with a six month endpoint give us results around the end of the year, maybe early 2019. As far as the mix, we don’t have a pre-specified mix of prospective and retrospective patients but expect that the majority of them will be retrospective given the timeframe that we are accruing from late 2014 through the middle of '18.

Okay. And then just switching gears to the U.S., I just want to confirm there were no more cases of encephalopathy reported and in regards to the EU regulators, the information was submitted to both regulators at the same time, if I remember correctly from the previous call. You guys feel pretty confident that it’s been reviewed by Europe and has largely been -- it just will be a [draft] [ph] response as European regulators are concerned.

What I can say is we haven't received any additional reported cases of encephalopathy that are possibly related to BPX-501. And that the adverse events that we have received have been reported to all of the relevant regulators in the U.S. and Europe. I can't speculate on the European regulators view of this. Certainly we will engage them in discussions as they request. But so far our focus has been responding to the FDA where they have had the lion's share of the questions.

Got it. That sounds great. And then just one final one from me for 601. I guess I am trying to understand, so we see very nice expansion but can you give us a sense, is this the type of expansion that we would see of the CAR-T cells in the hematological [indiscernible]. Is it smaller, larger and kind of going back to a previous question regarding the cells? I know you have some good theories on where those cells could be, for example penetrating the tumor microenvironment. Am I thinking about it incorrectly in that I would think there would be some sort of base line of cells. Like maybe that was originally injected to so and so, that would still be somewhere in the periphery.

So let me offer one comment in response to that Ren and then maybe I will turn it over to Bill to speculate scientifically on some of the points that you raised. I think what I would say is that we are excited about these data because it shows that we can cause a really robust T cell expansion with rimiducid in combination with CAR-T cells recognizing PSCA. And that to my knowledge hasn’t been accomplished in a solid tumor CAR-T trial to date. So we feel like that’s a really important proof of concept of the technology and we ought to be able to apply that to drive expansion persistence and hopefully more robust efficacy in the future in the trials. As for the comparison across trial to hematologic malignancies, I will turn it over to Bill, but I imagine that might be speculative.

Yes. Exactly right. I think right now we are just at the very beginning of collecting this data and I think we will be able to give more insight into what we think the expansion results look like and potentially expect versus hemo versus solid. One other potential idea here is that eventually as we get more data to understand the co-relation of the expansion as it relates to the level of PSCA expression on the tumor cells and we will doing that as we collect more data from patients being enrolled.

The takeaway we hope you will have from today on this topic, again, very limited data, first patient etcetera. But the takeaway we hope you will have is, we have a technology platform now that we believe we will be able to induce expansion of T cells in vivo and we ought to be able to apply that through the application of that technology even if it requires repeat dosing etcetera, we ought to be able to apply that to achieve our goal in the solid tumor study. So it's a really good start.

Yes. I definitely agree. And maybe just one quick follow up. Was it one dose of rimiducid or did you have seven doses, seven daily doses of rimiducid?

No. This is a single dose of rimiducid on day 7. This was, again required too much of the initial cohort zero for cells only, so from a safety perspective. And so we will be evaluating this cohort of single dose rimiducid that our patients, if you go back to the slides and see that there is increasing of we are -- two more slides, sorry. The trial design that we showed earlier right here, you can see that we have increasing self doses but still it's a single dose of rimiducid administered on day 7?

Our next question comes from the line of Justin Kim of Cantor Fitzgerald. Please proceed with your question.

This is Elemer Piros for Justin Kim. And what I would like to ask is the data that you reported from a combined, if I did my math correctly, 97 patients. How much of this data would go into the analysis for the European submission.

You are raising a good question. Our current plan is to include European patients in the efficacy analysis for Europe and the total population which includes patients enrolled in the U.S. for safety. And off the top off my head I don’t recall the geographic mix of these patients to tell you the split of which of them are European versus the U.S. I know a majority of them are European based on the timing of the trial and the duration of the follow up but I don’t think we have that split today.

So it's a fairly good chunk of the 100 or so evaluable that you would like to include in the cohort. So the question is, have you looked at event-free survival rates at six months?

In this analysis we have not. We were more focused on this analysis on looking at disease free or relapse free survival and overall survival. Certainly as we get closer to the medical meeting where we present these data, we will look at that more carefully.

There are no further questions over the audio portion of the conference. I would now like to turn the conference back over to management for closing remarks.

Well, thanks, everyone. Appreciate your participation on today's call. As always, I would like to thank our entire Bellicum team and our collaborators and investigators for their effort. And most importantly, the patients and families who participate in our clinical trials who inspire our effort every day. Thanks, have a great evening.

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.