Bellicum Pharmaceuticals' (BLCM) CEO Rick Fair on Q2 2018 Results - Earnings Call Transcript

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About: Bellicum Pharmaceuticals (BLCM)
by: SA Transcripts

Bellicum Pharmaceuticals Inc. (NASDAQ:BLCM) Q2 2018 Earnings Conference Call August 7, 2018 5:00 PM ET

Executives

Alan Musso – Chief Financial Officer

Rick Fair – President and Chief Executive Officer

Bill Grossman – Chief Medical Officer

Analysts

Greg Harrison – Citi

Biren Amin – Jefferies

Jim Birchenough – Wells Fargo

Wangzhi Li – Ladenberg

Justin Kim – Cantor Fitzgerald

Pete Lawson – SunTrust Robinson Humphrey

Reni Benjamin – Raymond James

Operator

Good afternoon, ladies and gentlemen. Thank you for standing by. Welcome to the Bellicum Pharmaceuticals Second Quarter 2018 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 you to our host today, Alan Musso, Bellicum’s Chief Financial Officer. Please go ahead.

Alan Musso

Thank you, Roya. 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, Chief Medical Officer. Earlier this afternoon, Bellicum released financial results for the second quarter ended June 30, 2018. 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, review and approval of our product candidates, commercialization expectations 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 Factors section of our Annual Report on Form 10-K for the year ended June – our quarterly report on Form 10-Q for the quarter ended June 30, 2018 filed with the Securities and Exchange Commission.

And now I’ll turn the call over to Rick.

Rick Fair

Thanks, Alan. Good afternoon, everyone. Overall, we have made substantial progress with our lead programs this quarter. BPX-501, our allogeneic T-cell therapy and developments for patients with leukemias, lymphomas and genetic blood diseases, continues to advance on its path to market in Europe. BPX-601, the first controllable CAR-T cell therapy to enter clinical development is on track to report initial data later this year. We also made important advances with the balance of our pipeline. Including the development of two GoCAR-T candidates with our dual switch technology, which we expect to enter the clinic in 2019.

I’d like to give a little more color on our progress, starting with BPX-501. As you may have read in our press release today, we completed perspective enrollment in the BP-004 and C-004 studies in pediatric patients with hematologic cancers in orphan inherited blood disorders. We expect that these studies will serve as the basis for filing in Europe. C-004 is a multicenter, observational trial of pediatric patients receiving a matched unrelated donor or MUD transplant.

We are conducting this trial to service as comparative for our lead BP-004 study, the primary analysis will be a comparison of event free survival at six months, and patients receiving BPX-501 following a haploidentical transplant versus those receiving a MUD transplant. The goal of the study is to demonstrate non-inferiority. MUD transplants are currently the preferred option for the approximately 70% of patients, who do not have an HLA matched sibling donor.

If we can demonstrate the BPX-501 enables patients without a matched donor, to achieve outcomes at least as good as a MUD transplant, that would be very clinically meaningful and will bode well for commercial success. We’re looking forward to the American Society of Hematology Annual Meeting in December, as a form to present a comprehensive update on BPX-501. We’ve submitted abstracts including interim event free survival results from both BP-004 and C-004, disease outcomes from some patients subsets of interest, and clinical experience of the patients who received rimiducid to treat steroid refractory GvHD.

Final results from both the BP-004 and C-004 studies are expected to be available in early 2019, with the goal of filing marketing authorization applications for both BPX-501 and rimiducid in Europe in 2019. In addition to these pediatric studies, we’re working toward extending the potential clinical benefit of BPX-501 to adult malignant patients. As we reported at EHA earlier this year, relapse-free and overall survival in pediatric AML patients, who received BPX-501 following the transplant were 89% and 95% respectively, with the median follow-up of approximately 15 months.

We believe BPX-501 T cells, which are polyclonal and contain a broad and diverse repertoire of cancer fighting cells, may be highly effective at reducing or eliminating residual cancer cells following a transplant. We believe this may be why we’re seeing such durable responses when compared to available therapies. Based on the strong results in pediatric leukemias, we’ve designed a Phase 2, 3 study in adult AML and has submitted a protocol to FDA for review. We’re planning to initiate this trial by the end of the year.

We’ve also continue to explore potential avenues to secure an approval for BPX-501 in pediatric patients in the U.S. As previously communicated, we’ve been evaluating the feasibility of conducting a registrational trial in the U.S., in a single ultra orphan pediatric indication. Our current assessment of the cost, timeline and value of such a study, makes it less attractive than other opportunities to expand our portfolio. So we’re de-prioritizing a new BPX-501 pediatric study for now. We intend to review updated data from the BP-004 study with FDA, and if through this dialogue, we find an efficient and attractive path to pediatric registration in the U.S., we’ll revisit our prioritization and update you then.

Also was announced yesterday, we recently welcomed Dr. Thierry Darcis, as General Manager of Europe. Thierry has extensive experience launching orphan products and has built-in lag European operations to launch new products for the Zogenix, NPS Pharmaceuticals and ViroPharma.

He also held leadership roles previously with Novartis and GlaxoSmithKline. Thierry has hit the ground running and recruiting his team and initiating our pre-commercialization efforts. And I’m confident he’ll be instrumental in our plans to file for approval in Europe in 2019 and commercial launch in 2020.

With that, I’d like to turn to our CAR-T and TCR programs starting with BPX-601. As you know, BPX-601 is our first GoCAR-T product candidate and the first controllable CAR-T cell to enter human studies. We continue to be excited about it’s as well as the potential of the GoCAR-T platform. BPX-601 targets PSCA, and is equipped with our proprietary IMC activation switch. IMC is designed to drive T-cell proliferation and to modulate the tumor microenvironment to enhance immune activity.

As this is our first human experience with IMC, we’re learning a lot. And will leverage these learnings as we go to optimize its use. Particularly, the dose and schedule of the cell therapy in the small molecule activated rimiducid. We continue to enroll and treat patients in the Phase I study in pancreatic cancer, and expect to report preliminary findings from the cell dose escalation safety phase of the study at a medical meeting later this year. We recently published an amendment to the study to expand it to prostate and gastric cancers and to utilize more standard conditioning regimen.

We expect to begin enrolling patients under this amended protocol in the third quarter, and to expand the study to additional sites through the remainder of the year. Turning to BPX-701, our high affinity T-cell receptor targeting PRAME, we continue to recruit at a single center, patients with AML and MDS. Given the eligibility criteria, which require patients both to test positive for PRAME expression and to be HLA-A2 matched, as well as the rapid progression of these advanced AML patients. Enrollment in the study has been slow. We’re in the process of adding several additional clinical sites to the study, and evaluating if additional PRAME expression tumor type should be included. We expect to be enrolling patients in the new sites later this year, and intend to present initial clinical results from the study in 2019.

Looking beyond our clinical pipeline to the rest of the organization, our technical operations team continues to build out our capabilities. Having recently achieved full validation of our Houston cell therapy manufacturing facility. We currently produce BPX-501 for U.S. clinical trials sites there, and intend to transfer BPX-601 and BPX-701 production thereby year-end. I’m proud of what our team has been able to deliver. And we are well-positioned to ramp up production from more trials, new GoCAR-T projects and early commercial supply.

Our research team has also been busy advancing our next-generation GoCAR-T projects. These have been designed with both activation and safety switch technologies, essentially providing both the gas pedal and a break to potentially enhance efficacy and safety. We’re hard at work, preparing for IND and IMPD submissions, and expect to initiate clinical trials for two new dual switch GoCAR-T products in 2019.

On the personnel front, in addition to Thierry Darcis’s appointment as GM of Europe, we recently welcome on Shane Ward as General Counsel and Corporate Secretary to Bellicum. Shane has more than 20 years of pharmaceutical and biotech experience, overseeing legal, compliance and QA teams of both large and small public companies.

Finally, on behalf of all of our employees and our board, I’d like to take this opportunity to thank Alan Musso, our CFO, dedication and his many significant contributions to the company. Next month, Alan will move onto a new opportunity and while he will be miss by all of his [indiscernible] we wish him the very best. Rosie Williams, our VP of Finance & Controller will serve as our Interim Principal Accounting Officer, as we look for Alan’s replacement.

With that, I’ll hand the call back to Alan and he’ll cover second quarter financials.

Alan Musso

Thank you, Rick. Bellicum reported a net loss of $24.2 million for the second quarter of 2018 and $47 million for the six months ended June 30, 2018, respectively, compared to a net loss of $24.5 million and $46.4 million for the comparable periods of 2017. The results included non-cash, share-based compensation charges of $3.6 million and $7.2 million for the second quarter and six months ended June 30, 2018, and $3.2 million and $6.6 million for the comparable periods in 2017.

As of June 30, 2018, cash, restricted cash and investments totaled $135.3 million. Based on current operating plans, Bellicum continues to expect that current cash resources will be sufficient to meet operating requirements through 2019.

Research and development expenses were $18.4 million and $34.9 million, for the three and six months ended June 30, 2018, respectively, compared to $18 million and $33.3 million during the comparable periods in 2017. General and administrative expenses were $5.4 million and $11.1 million for the three and six months ended June 30, 2018, respectively, compared to $5.5 million and $11.4 million during the comparable periods in 2017.

And now Rick, I’ll hand the call back over to you.

Rick Fair

Thanks, Alan. As you’ve all heard, we’ve had a productive first half of the year with important milestones coming over the next six months for both BPX-501 and BPX-601. We’ll present a broad set of data from BPX-501 program in December with late interim analysis from both the BP-004 and comparative C-004 studies. We also expect to initiate our Phase II three trial in adult AML. Our Phase I trial with BPX-601 is expected to report out in the fourth quarter. We look forward to the work ahead, as we continue to strive to transform treatment of cancer through controllable cell therapies.

I’ll now open up the call to questions.

Question-and-Answer Session

Operator

Thank you. We’ll now be conducting a question-and-answer session. [Operator Instructions] Thank you. Our first question will come from the line of Robyn Karnauskas with Citi. Please proceed.

Greg Harrison

Hi guys, this is Greg Harrison on for Robyn. Congratulations on all the progress. Thank you for taking the question. Is there any update on the longer-term levels of BPX-601 cells? And have you gained any insight from dosing additional patients, or re-dosing the previous patients with you maybe said that the cells are able to expand again?

Rick Fair

Yes, thanks, Greg for the question. We haven’t disclosed any additional information about BPX-601, we’ll wait to report both clinical and biomarker results of the study to date, in the fourth quarter of this year. To be clear though, per your question, in the study protocol to date, we’re only dosing with a single dose of rimiducid. So keep in mind, this is the dose escalation safety study. We are evaluating the safety of the cells. The safety of the cells plus the single dose of rimiducid and if we advanced past that stage, certainly, we would consider a multiple doses of rimiducid, but at this stage, we’re dosing ones.

Greg Harrison

Okay, great. Just a quick follow-up. How will be amendment to expand to gastric and prostate cancer patients set the timeline of the trial?

Rick Fair

It’s a Phase 1 study with expansion possibilities into those different tumor types, based on the signal in the first phase. So of course, it depends on the results that we see. But we continue to enroll the study, we haven’t paused the study we continue general pancreatic cancer, so shouldn’t change the readout of the dose escalation portion.

Greg Harrison

Great. Thank you.

Operator

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

Biren Amin

Yes. Hi guys, thanks for taking my questions. Rick, I think you mentioned for BPX-601 amendment that you are utilizing a more standard for the depletion regiment. Can you talk about that a little bit?

Rick Fair

Sure, happy to. So you may recall that the time we were launching this BPX-601 study was around the time experienced several deaths in their JCAR015 trial. And at that time, they have hypothesized that those debts were associated with fludarabine. As a result, we had agreed with the FDA at that time to initiate the study using only cyclophosphamide conditioning. As that fludarabine hypothesis if you want to call it that, has been largely dispelled, we’re now amending the trial to – re-include fludarabine back into the conditioning regiment to enhancement for the depletion and create more room for our GoCAR-T cells to expand.

Biren Amin

And I guess, yes, once you have that, do you think that could potentially also influence cell persistence that you would potentially see in the trial?

Rick Fair

It could.

Biren Amin

Got it. And then on BPX-701, it seems you’re experiencing full enrollment. What’s been the screen failure rate in the trial?

Rick Fair

Yes, I don’t think we’re reporting exact screen failure rates but I would say that, our assessment of PRAME positivity has been less than 50%, something like 35% or 40%, in terms of PRAME expression. And then of course, they need to be HLA-A2 match. So that majority of the patients are screening out of the study. The only other piece that I commented on then I’ll reiterate, is that these are very advanced AML patients and they progress quickly. And so we certainly had a number of patients who qualified for the study but didn’t survive long enough to be treated.

Biren Amin

Got it. Thank you.

Operator

Thank you. Our next question comes from the line of Jim Birchenough with Wells Fargo. Please proceed. Jim Birchenough your line is now live at conference. So it has muted on your end sir.

Jim Birchenough

Hello, sorry.

Rick Fair

Hello. Hi, we hear you now.

Jim Birchenough

Although Bellicum, that’s right, Rick. Anyway, on Slide 1 with the data coming in, in 1Q, is that something rate limiting to the filing other than just analyzing and packaging it all up for the MAA?

Rick Fair

We’re also gathering some additional PK data on rimiducid. So that’s the other piece of data that we need to bring into the package. But those are the three main datasets and then it’s about doing the analysis and preparing the application.

Jim Birchenough

Okay. So that doesn’t sound too on those that the PK data…

Rick Fair

I think Bill will suggest that the filing of that magnitude of two MAAs is onerous, but…

Jim Birchenough

No. That part I agree, yes. And can you remind us the definition of event for your six months event free survival analysis?

Rick Fair

Sure. Bill you want to comment on that? Are you there?

Bill Grossman

Yes, I’m sorry. I was on the mute there. So be on the – U.S. disappear on the event free survival…

Jim Birchenough

Yes. What’s the definition of event?

Bill Grossman

It will be for six months, we’ll have look at event free survival as it relates to relapse free survival, disease free survival for malignant non-malignant patients as well as severe graft-versus-host-disease and severe infections.

Jim Birchenough

And if severe graft-versus-host-disease defined as Grade 3 and above?

Bill Grossman

Correct.

Jim Birchenough

Okay, great, thank you. And is there similar definition for severe infection?

Bill Grossman

That would be Grade 4.

Jim Birchenough

Grade 4, great, thank you. And then also for me and that is – and I know you no longer control the CD19 CAR-T, but do you know the expected collaborators in Italy to be presenting an update on that program later on this year as well.

Bill Grossman

This is – you’re referring to the clinical ablation or…

Jim Birchenough

OPBG…

Bill Grossman

OPBG – it was BPX-401 I guess one time.

Rick Fair

It’s a different CAR-T, right. So it’s a homegrown OPBG developed CD19 CAR with CaspaCIDe safety switch. It’s not an old construct from Bellicum.

Jim Birchenough

Okay. And do you expect them – go ahead…

Rick Fair

Bill, do you know if they’re presenting at ASH this year, I am not aware.

Bill Grossman

Yeah, I am not sure if they submit an abstract this year, we do know they have different six patients today but that’s – as they present EHA, I am not sure.

Jim Birchenough

Yeah, yeah, okay, great thank you very much and look forward to…

Bill Grossman

Thanks Nick.

Operator

Thank you. Our next question comes from the line of Wangzhi Li with Ladenberg. Please proceed.

Wangzhi Li

Thanks for taking my questions. Maybe just two questions, number one is for BPX-601. I guess the data readout will be like ESMO or in the first quarter?

Rick Fair

The BPX-601 readout will be at ESMO I-O in Geneva in December and that’s our current plan. Of course, we have to submit an abstract being get it approved, but that would be our intent.

Wangzhi Li

Okay, got it. And any color on the number of patients and also where we see the patient with old pancreatic cancer or maybe we see some new gastric and prostate cancer?

Rick Fair

Yeah, we’re not commenting on specific enrollment figures, but as we have talked about previously we will present the first cohort of patients who only received BPX-601 cells and then at least a couple of cohorts of patients who have received 601 cells followed by rimiducid.

Wangzhi Li

Okay, got it.

Rick Fair

And I would expect that the data would be largely or exclusively pancreatic cancer patients given the timing of the amendment adding others.

Wangzhi Li

Understand, okay, great. And then the BPX-501, the data ASH, you just mentioned the definition that event I just wonder are we going to see kind of more details about inspection data?

Rick Fair

Yeah, our intent would be to show the event-free survival of immune reconstitution graft-versus-host-disease all of the mortality – all of the aspects of that in both from our BP-004 study which is beyond treatment study if you will as well as the observational study in MUD. So that will be the first time we have an opportunity to present end points from both of those studies for a cross-trial comparison.

Wangzhi Li

Got it. And if I remember correctly the MUD arm is like 40 patients?

Rick Fair

No, the MUD arm to date has enrolled over 100 patients. We’re a little over accrued because we had a larger number of retrospective patients in that study than we expected. So as a reminder, it’s an observational study in MUD patients in the same centers we conducted BP-004 over the same time period. So in order to get the same time period, we had to collect some patient cases back to 2014 retrospectively, we also had a portion of that which was prospectively enrolled. So we would expect to be able to report on 100-ish patients at ASH.

Wangzhi Li

Yes, yes, but I mean for the prospects we enrolled patient member is about 40 all right, and then combined retrospective of patients to get it over 100.

Rick Fair

Yeah, we accrued a little bit less than 40 patients that’s correct in that neighborhood.

Wangzhi Li

Okay, got it. Great and maybe final question, any update on the BPX development front, given specially for PBX as you were is close to MM…

Rick Fair

Yeah, thanks for the question. Obviously, nothing I can comment on publicly. We remain interested in collaborations. We think we could do more with our platform and we would welcome the opportunity to do that with the right partner and discussions are active. And these are difficult things to predict, but we certainly remain interested in the collaborations with the right partner.

Wangzhi Li

Okay. I just go one more question coming to my mind. For the GMP manufacturing facility, you mentioned right now you are manufacturing PBX-501 in your own facility and you expect to transfer PBX-601 and the PBX-701 manufacturing in [indiscernible] next year, I just want to verify that if I hear it correctly.

Rick Fair

Later this year, so by the end of the year, we will be making 501, 601 and 701 all hear for the U.S. trials.

Wangzhi Li

Okay, great. All right thanks for taking my questions and congratulation on the results of the quarter.

Rick Fair

Thank you.

Operator

Thank you. The next question comes from the line of Justin Kim with Cantor Fitzgerald. Please proceed.

Justin Kim

Good evening. Just one from me. Based on the pace of enrollment on the study near completion. Could you contrast or qualify the level of our size of the data, we would observe at the end of the year, as compared with the full data in early 2019?

Rick Fair

I don’t think I can quantify that. But we expect the final dataset a couple of months after the ASH presentation, so we would expect the majority of the information from the patient's enrollment study will be part of the interim analysis at ASH.

Justin Kim

Okay, great. Thank you.

Operator

Thank you. Our next question comes from the line of Pete Lawson with SunTrust Robinson Humphrey. Please proceed.

Pete Lawson

Thanks for taking my questions. Just firstly, it’s always been a pleasure to be with you, Alan. And best wishes for the next move. And just, Rick, where are you at the hiring process and what additional senior levels hires should we be thinking about as you build out the team?

Rick Fair

What additional senior level hires in the entire organization; is that your question? Or the CFO hire?

Pete Lawson

Yes. Just kind of thinking about firstly, the CFO hire where you’re in that process, what additional senior levels we should kind of be thinking about over the next 12 months?

Rick Fair

Sure. So as it relates to CFO, recruiting and interviewing, so we'll keep you posted but certainly active – very active in the recruiting and interviewing part of the process. As it relates to other senior hires, I think our senior executive team is staffed beyond, once we fill the CFO role, I think we have the team in place that we need.

I would say, the additional hires on top of that will be Thierry building out his leadership team in Europe to commercialize BPX-501, he's made a lot of progress in identifying the right talent, he’s beginning to build that out in terms of Head of Market Access, Head of Medical Affairs, Head of Commercial and of course, General Managers for the major markets there. So that's probably the next 6 to 12 months of hiring will be focused on that.

Pete Lawson

Thank you. And then just on the expansion to the adult AML. What did you see in the pediatric leukemia that drove that expression? And at what point do we see that data that drove that expansion?

Rick Fair

Your question about why are we going into the adult AML. What do we see in the pediatric data?

Pete Lawson

Yes, exactly. What did – you kind of mentioned in the opening comments about the pediatric leukemia have been encouraging data and expansion.

Rick Fair

Yes, sure. So I think we've continued to get more confident in what we're seeing in terms of the leukemic subset from BP-004 as to follow as longer. When we had six months of follow-up, it's a little hard to say with confidence that you're preventing leukemia relapse. The data we've presented at EHA had 15.5 months of follow-up on average, which is certainly meaningful relapse-free survival of 89% and overall survival of 95%. In pediatric AML, where our read of literature suggests that sort of one year overall survival could be between 60% and 80%. So we seem to be having a meaningfully – meaningful impact on delaying relapse and extending survival in that setting.

And of course, I would also say that it's been well established for some time that donor T cells can play a valuable role in fighting leukemic relapse particularly in myeloid leukemias. The challenge was giving donor lymphocyte's fusion of course is, robust or terrible reference disease and we have an approach where we think we can make that manageable. So I think based on the great outcome so for in BP-004, and what's known in the literature about the importance of donor T cells are the potential impact of donor T cells in AML, we feel confident moving into the adult setting.

Pete Lawson

Rick, I apologize if I missed this. Pediatric and leukemia, when is the next readout we get for that?

Rick Fair

We've submitted an abstract to ASH, that includes both pediatric AML and ALL patients, so the whole pediatric leukemic subset. So you will see that in ASH if it’s accepted.

Pete Lawson

Perfect. Thank you so much. Thanks for taking questions.

Rick Fair

Yeah, thanks, Peter.

Operator

Thank you. Our next question comes from the line of Reni Benjamin with Raymond James. Please proceed.

Reni Benjamin

Hey, good afternoon, guys. Thanks for taking the questions. Rick, I jumped on the call a little late, so you have talked about this and if you have, I apologize. But can you talk a little bit about the prelaunch activities? And I assume that you've done kind of like a comparing contrast like as of the Zalmoxis launch and there’s probably a ton of learnings there. And while you might not be able to talk about everything, for competitive reasons, can you give us a sense as to how you see this unfolding?

Rick Fair

Sure. I'm not going to comment a lot on our commercial preparations because it's early days and we've got until 2020 to answer all those questions, when we lay that out. I would say that out primary take away from the disappointing Zalmoxis launch to date is that you need to prepare. I think unfortunately, they did a nice job of getting conditional approval with a very limited data set, but there was no preparation to successfully commercialize that product with the collaboration that they’ve established with down pay – I think the down pay team started to get a little bit of a late start more than year after the approval.

And now it seems, at least based on publicly report information there is to satisfaction that collaboration already. Our view is, successful commercial lunches happen with great products and great data where you actually are well prepared. Thierry Darcis and the team he’s assembling have successfully launched orphan products for several companies, they know how to do it. We have a couple of years to our approval comes in Europe and we’re beginning to prepare an earnest, we will be well prepared, we will have great health economics and reimbursement dossiers we will have engaged the thought leader community, we will have clinical experience amongst the thought leaders in Europe with our product and I’m confident, we’ll make it a success. We’ll certainly talk more about our preparations as they evolve and as we get closer to launch.

Reni Benjamin

Okay. And then just as a related question. When we talk with certain transplanters at certain facilities, it seems like there is different protocols, it tends to be center specific. Are we hearing things correctly? How do you decide, which transplant facilities to target? I assume that they have to all be conducting alpha-beta depletion type transplants, but maybe I’m wrong there. Can you give us just a little bit more color as to how you are thinking about that?

Rick Fair

So I think your comments about transplant are accurate, that there is a variety of approaches in the absence of real standardized labels and guidance that transponders adapt and tailor their approach based on what they try and was succeeds for them. That said, I think there are increasingly trends, for example the adoption in the adult setting post transportation cyclophosphamide, which was developed at Hopkins but has now been really adopted broadly by adult transplanters across the U.S. and Europe.

Transplanters can adapt when they see a good data, so I think in our market research what we hear from transplanters is, if you show me a product that can work as well as your target product profile, which is really based on our interim analyses. I would change my transplant approach in my facility to do that. Our view is that of course, there will be sort of phasing or sequencing is much easier to take a facility that’s already doing alpha-beta T-depleted haplo’s, and simply add 501 to it. That’s a pretty simple adoption step and those will probably be the early adopters. But certainly, we think there’s an opportunity to penetrate other facilities where they haven’t yet done T-cell depleted haplo transplant and our research supports that.

Reni Benjamin

And do you have a sense in the U.S. versus EU just what proportion of facilities are – could be those early adopters.

Rick Fair

Yes. So we’re profiling that in Europe as we speak that’s certainly a project for Thierry and his new team. I would say in the U.S. alpha-beta T-depletion has been only – it’s not approved approach and so it’s only reserved for using in clinical trials. So the experience here is certainly more limited. Certainly as we rollout adult AML, there will be more of an opportunity for more U.S. centers to gain clinical experience with that.

Reni Benjamin

Got it. And then just one final one for me. I’m always getting the number of patients, I don’t know the study is wrong. Can you just tell us, at the end of the year at ASH how many patients will we be looking at in the malignant cohort versus non-malignant. And I think you just mentioned to a previous question about 100 in the observational study in MUD.

Rick Fair

Bill, do you have a – some nail on number of patients in BP-004 and the rough split between malignant and non-malignant.

Bill Grossman

The split between malignant and non-malignant I have to look it up real quick that we have 38 patients that we’ve reported on AML and we’ll have roughly 54 patients in AML for leukemia portion. So that’s the fraction, not quite as what you would expect to see in our total set of patients.

Rick Fair

So the total will be in the range of 240 and I think at last analysis that I saw was about 60-40 split non-malignant, malignant, it’s sounds from Bill’s comment so that’s about what you’ll see.

Reni Benjamin

Perfect. Thanks very much guys.

Rick Fair

You bet.

Operator

Thank you. We’ve reached the end of our Q&A session. Allow me to hand the floor back over to Mr. Rick Fair for closing remarks.

Rick Fair

Thank you, Roya and thanks everyone for participating today. As always, I’d like to thank our Bellicum team, our collaborators and investigators for their efforts as well and importantly the patients and families who have participated in our clinical trials. They inspire our effort every day. Thanks have a great evening.

Operator

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