Bellicum Pharmaceuticals' (BLCM) CEO Tom Farrell on Q2 2016 Results - Earnings Call Transcript

Bellicum Pharmaceuticals (NASDAQ:BLCM)

Q2 2016 Earnings Conference Call

August 8, 2016 17:00 ET

Executives

Alan Musso - CFO

Tom Farrell - President & CEO

Annemarie Moseley - COO & EVP, Clinical Development

Analysts

Tony Butler - Guggenheim Securities

Daniel Wolf - Piper Jaffray

Biren Amin - Jefferies

Operator

Good day ladies and gentlemen, and welcome to the Second Quarter 2016 Bellicum Pharmaceuticals Incorporated 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 be given at that time. [Operator Instructions]

I would now like to turn the conference over to your host for today, Alan Musso, Chief Financial Officer. You may begin.

Alan Musso

Thank you, Sonia. Good afternoon everyone and thank you for joining the call. With me today are Tom Farrell, 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 second quarter ended June 30, 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 under the Private Securities Litigation Reform Act of 1995 including statements regarding our research and development plans and 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, 2015 and are reported on Form 10-Q for the quarter ended June 30, 2016 filed with the Securities and Exchange Commission.

And I'll now turn over the call to Tom.

Tom Farrell

Thank you, Alan and good afternoon everyone. Thank you for joining us on our mid-year corporate update call. Our goal today is to brief you on the progress of our pipeline, review the company second quarter financial results, and discuss our objectives for the second half of 2016 and into next year.

In the last six months, we've achieved important clinical and regulatory milestones across our stem cell transplant CAR-T and TCR controlled cell therapy programs. How these product candidates BPX-501 has activator reagent rimiducid received often drug status in both, the European Union and the U.S. and we have reached initial agreements with EMA around the pathway to filing for approvals in Europe based on the ongoing BP-004 clinical trial. In addition, we're pleased to report that the investigation on new drug applications for both, our BPX-701 TCR and BPX-601 GoCAR-T product candidates have been allowed by the FDA and we're preparing to begin their Phase 1 studies.

The level of the company-wide activity, energy and enthusiasm are high and we strongly believe that giving physicians the ability to control the potency and safety of infused activated T-cells in real-time is a critical differentiating factor for Bellicum in the cell therapy space.

As you know in the second quarter we reported encouraging interim data from BP-004 study with BPX-501 and rimiducid in both malignant and non-malignant diseases at the annual meeting of the European Society for Blood and Marrow Transplantation. Lead Investigator, Dr. Frank Locateli [ph] presented results demonstrating disease-free outcomes, reduced treatment related mortality, reduced infection rates, faster immune reconstitution and significant reductions in time to hospital discharge and re-hospitalizations compared to historic controls. The data also showed high BPX-501 cell viability, expansion and persistence and sustained improvement of immune reconstitution.

At EHA in June, data were presented on 25 high-risk leukemia patients with a median follow-up of eight months and only one relapse. Our high-risk compassionate use patients with relapse to refractory AML or metabolic disorders are also doing well.

The study continues to enroll at a brisk pace since EBMT we have enrolled 18 more patients in Europe and six in the U.S. and are continuing to see enrollment of children with my both, non-malignant and malignant diseases. For children with leukemia to lymphomas, we have completed escalation of the BPX-501 cells to 2 million and then 4 million cells per kilogram, and expect to continue with this higher dose for the duration of the study. For children with non-malignant disorders, including primary immune deficiencies, hemoglobinopath and anemia we are staying with a one million cells per kilogram dose which are investigated to believe strikes the right risk-reward balance for the goal of facilitating immune reconstitution.

We expect to provide a substantive update on ongoing results of the BP004 trial at ASH in December. Meanwhile, I can report that four patients to-date have developed uncontrolled GvHD triggering administration of rimiducid. In three cases of acute GvHD, the administration of rimiducid was completely effective in reducing BPX-501 cells and resolving GvHD confirming results from proof-of-concept trials at Baylor College of Medicine and first reported in The New England Journal of Medicine in 2011.

As reporters initially at EBMT, a fourth patient with leukemia developed severe chronic GvHD with activation of largely of the allograft T-cells. While administration of rimiducid in reduction of the BPX-501 T-cells and a partial resolution of the liver symptoms, rimiducid of course, does not eliminate allograft T-cells and the chronic GvHD was subsequently fatal. Since no BPX-501 cells were detectable in the patient at the time of GvHD progression, this event is not considered BPX-501 or rimiducid related.

In addition to the BP004 study, we're running multiple trials with this product candidate and recently initiated BP008, a Phase 1 study of BPX-501 to treat post-transplant relapse in adults and children with blood cancers. The safety trial which includes matched as well as haploid identical transplant recipients but also valuate the potential for a titrated dose of rimiducid to result uncontrolled GvHD while preserving a greater proportion of BPX-501 cells.

Moving on now to the approval pathway for BPX-501 and rimiducid; we announced last week that we met with EMA and intend to pursue European approvals under exceptional circumstances. While details are subject to further refinement in a formal protocol assistance process, we believe that data from the European arm of our BP004 trial with a six months follow-up time and expanded to enroll additional patients could form the basis of marketing authorization applications for BPX-501 and rimiducid for the pediatric patient population, including both malignant and non-malignant diseases.

The EMA's committee for medicinal products of human use has agreed that for giving approval under exceptional circumstances may be suitable recognizing that a randomized trial may not be feasible in the pediatric setting. In place of a randomized trial, we intend to collect data from a concurrent observational study of allogeneic HSCT outcomes in the pediatric setting and are working to initiate this trial as quickly as possible. We are pleased with the progress we have made towards defining an experienced pathway to the potential approval of BPX-501 and rimiducid for pediatric transplant patients in Europe. We're now initiating discussions with the FDA and expect to be able to provide additional guidance on the approval pathways in both markets during the fourth quarter.

Turning to our lead CAR-T and TCR product candidates, BPX-601 and BPX-701 both now have active INDs allowed by the FDA and our clinical team is completing final preparations to start Phase 1 trials. Our GoCAR-T product candidate BPX-601 will be evaluated initially in patients with non-receptible [ph] pancreatic cancer. The study will be conducted at Baylor Sammons Cancer Center in Dallas, and we expect to enroll upto 30 patients in a three plus three dose escalation deescalation design.

PBX-601 consists of CAR-T cells that are engineered to include our proprietary inducible MyD88/CD40 or IMC activation switch targeted solid tumors expressing prostate stem cell antigen or PSCA. Unlike standard CARs which are dependent on antigen for activation and proliferation, GoCAR-T places a coast symmetry signal on a rimiducid controlled switch designed to allow control of T-cells survival in the absence of antigen signal and full activation of proliferation in the presence of an antigen. In the event of serious side-effects, the level of activation of the GoCAR-T cells is designed to be attenuated by reducing the rimiducid administration schedule. Pre-clinical data has shown robust anti-tumor activity and enhanced T-cell proliferation of the system compared to traditional CAR constructs.

The Phase 1 trial of BPX-701, our first CaspaCID enabled high-affinity TCR will recruit patients with relapsed or refractory acute myeloid leukemia or AML and myelodysplastic syndromes or MDS with an additional study planned in metastatic uveal melanoma. BPX-701 is designed to target malignant cells expressing the preferentially expressed antigen in melanoma or PRAME, a cancer tested antigen. The study will be conducted at Oregon Health and Science University and Leiden University Medical Center and is expected to enroll upto 36 patients. Bellicum holds worldwide rights to BPX-701 and another TCRs targeting solid tumors expressing PRAME through our research collaboration with Leiden. We recently expanded that relationship securing exclusive rights to any high affinity TCRs discovered over its three-year term.

Lastly, we have decided not to advance our CD19 CAR product candidate BPX-401 into the clinic choosing instead to pursue clinical proof-of-concept for CaspaCID in the CD19 setting through European and U.S. academics collaborators. The three recently reported cases of fatal neurotoxicity associated with a standard second generation CD19 CAR together with the generally high rate of severe CRS and neurotoxicity reported across multiple constructs and trials reaffirm the opportunity for CaspaCID enabled CD19 CAR to both, increased patient safety and allow greater flexibility with respect to preconditioning regimens, dose levels and enrollment criteria.

However, the CD19 landscape is highly competitive and represents a relatively small market. And this is approach that I've outlined allows us to pursue proof-of-concept while redeploying resources to other programs. We look forward to providing additional information and guidance on these initiatives later this year.

With that I'll turn the call over to Alan to cover our second quarter results.

Alan Musso

Thank you, Tom. Bellicum reported a net loss of $16.5 million for the second quarter of 2016 and $31.6 million for the six months ended June 30, 2016, compared to a net loss of $10.5 million and $18.3 million for the comparable periods in 2015. The results included non-cash stock-based compensation charges of $3.1 million and $6.2 million for the second quarter and six months ended June 30, 2016 and $2.1 million and $3.6 million as a comparable period in 2015.

As of June 30, 2016 cash and investments totaled $136.6 million. Bellicum continues to expect to end 2016 with approximately $80 million to $90 million in cash and cash equivalents and investments, and that current cash resources will be sufficient to meet operating requirements through 2017. This guidance includes planned spending in the second half of 2016 of approximately $15 million for capital projects to enable in-house U.S. manufacturing. Our research and development expenses were $12.2 million in $23.2 million respectively for the three and six months ended June 30, 2016 compared to $8 million and $13.7 million during the comparable periods in 2015.

The higher expenses in the 2016 periods were primarily due to an increase in manufacturing and clinical expenses as a result of increased patient enrollment and our BPX-501 clinical trials, increased expenses for the IND enabling activities on our product candidate BPX-601, BPX-701 and increased personnel and infrastructure costs. Our general administrative expenses were $4.2 million and $8.5 million for the three and six months ended June 30, 2016 respectively, compared to $2.8 million and $5 million during the comparable periods in 2015. The higher expenses in 2016 period were primarily due to the growth of the organization including an increase in costs related to personnel, higher facilities costs, and increased legal accounting and travel-related expenses.

And now I'll turn to call back over to Tom.

Tom Farrell

Thanks, Alan. Operator, we'll now open the call upto questions.

Question-and-Answer Session

Operator

[Operator Instructions] And our first question comes from Robin [ph] from Citigroup. Your line is open.

Unidentified Analyst

Thanks for taking my question. This is Mohit for Robin and congratulations on the progress. So quick question on your EMA and then now you've had the priority for approvals. Just trying to figure out -- I mean, in terms of number of patients did you have any discussions with the EMA about how many patients worth of data they would require? And also any sense on timeline with the EMA requirements that when do you think you will have enough data to file for an approval? Thank you.

Tom Farrell

Great Mohit, so thanks for the question. So as I indicated, we're in some respects midway through this process, we had our initial scientific advice, now that we have open designation we're eligible to continue with the protocol assessment process. So there are a number of parameters to the path forward that remain under discussion and some of those will have an impact on numbered timelines comparisons etcetera. So with that stated, obviously we want to do what we can to inform you all about where we believe we stand today. So with that in mind, we do have an understanding that from a safety perspective that 120 patients would be expected to be sufficient. And indeed we have expanded -- if you look at clinicaltrials.gov we've already expanded the U.S. trial -- the European trial to 120 and the U.S. trial to 60 potential patients. How that plays out there in terms of timelines, I think I'm going to ask Annemarie to give a little more color on that.

Annemarie Moseley

Yes, thank you. So we -- during the assistance process, we will agree upon obviously the number and we think that we have enrolled approximately about half of those patients already and so to the degree that we will confirm with them the endpoints and also what they are going to require exactly for approval and we have some preliminary inside into our six months endpoint and so that we believe that certainly the 120; we hope will be sufficient. But again as Tom said, we will have some insight into that shortly once we finish this process.

Unidentified Analyst

Got it, very helpful. And if I can ask one more, regarding the BP008 trial, it seems like a safety study; could you please help us understand the objectives of this study and what we will learn from the results there?

Annemarie Moseley

Well, it's actually little bit more than a safety study, it's actually got a number of different facets to them and in addition to escalation of patient doses as a standard DLI would do, in this case we have the ability to look not only for tumor response that you would expect with DLI but we also have the ability to evaluate coming back to patients who might be at a higher risk for GvH and therefore, with the use of cells which will contain the suicide gene, we now have the ability to give those patients multiple doses, and with the use of the titrated rimiducid we have the ability to see potentially T's apart the GvH from the GVL affects.

So actually the endpoints are the objective of the study are to understand a multiple doses of the BPX-501 product given in the setting of relapse patients so to look at the effect on tumor response potentially, but also to look at the effect of GvH and its response to AP1903 and the effect of multiple doses in the setting of the GvH and also multiple doses of the AP1903 along with the titration. So actually this is a very exciting protocol for us, both at the scientific level, at the clinical level, and response level, and at the understanding of the titration and the ability to control the cells in the patients themselves. So from that standpoint where we think this is a really important one in terms of the treatment about malignancy.

Unidentified Analyst

Got you.

Annemarie Moseley

Right. And it's a big unmet need in so many patients -- many centers are very excited because right now for a number of these relapse post-transplant, they really have nothing to treat them with.

Unidentified Analyst

Got it, thank you.

Operator

Thank you. And our next question comes from Tony Butler from Guggenheim Securities. Your line is now open.

Tony Butler

Thanks very much. Annemarie that was helpful to the previous comments. But I wanted to ask about the four additional patients; three of which rimiducid worked well and the one that had leukemia which was not thought to be due to 501 protocol or anything related to the protocol 004, but in the latter patient was there or had -- was there a GVL effect before the patient got GvHD? And then in the other three patients, could you say anything about their immune reconstitution before they got rimiducid and after? Thank you.

Annemarie Moseley

Thanks, Tony. So with respect to the fourth patient, this we'll take in reverse order; this patient was actually a CR-2 patient and ALL CR-2 so -- and he had sort of an interesting course in general with his GvH and because as we've said from his allograft, in general, there seem to be reactive T-cells. He was about 246 days out when he developed the GvH or chronic GvH which is rather late actually and he had -- was really almost at a year at that time that he did succumb to the chronic GvHD. At that time he did not have any evidence of tumor relapse. Now again, this is very early still in this patient and we don't have any evidence one way or the other but certainly there was no evidence to say that he had a progressive disease. So in this particular case he certainly was responding to the overall treatment.

With respect to the other patients, many of these -- all of these patients responded developed the GvH within about 40 days and so they -- it was still very early in their course and they all had skin GvHD at which responded grade two that was progressing and responded to the AP1903 and have subsequently continued on with respect to their immune reconstitution. Again as Tom said, not unlike the process that was described in both, The New England Journal case, as well as the two blood papers reported from The Baylor Group in 2014. So overall, it seems that certainly across various diseases, especially and I would say the leukemic setting, those patients are much more susceptible to GvHD for a variety of reasons; we are really beginning to get a bit more information on the fact that when the T-cells, the BPX-501 T-cell result in a GvHD setting that in fact the immune reconstitution with respect to anti-viral T-cells and other immune responses are not affected or those cells continue to expand upon treatment with AP1903.

Tony Butler

Thanks, Annemarie, very helpful.

Operator

Thank you. And our next question comes from Daniel Wolf from Piper Jaffray. Your line is open.

Daniel Wolf

Hello guys, thanks for answering the question. A quick question regarding the safety profile as the dose goes up. How is the safety profile as you're changing the dose profile? And then one more question I have is, in terms of the target organs for the four GvHD cases, what was the target organ? And what cell dose were those?

Tom Farrell

I think I'll as Annemarie to respond to that.

Annemarie Moseley

Okay, so in terms of the safety profile, so far at all -- all the different doses that have been tried, all the way upto the 4 million, the safety profile has been excellent in terms of the infusion itself. There has been absolutely no symptoms of CRS or anything that would imply a safety concern. So certainly from a maximum tolerated dose that you would possibly think off, I don't think that we have reached it yet. However, in terms of the GvHD, one of those patients was seen at the highest dose of 4 million, the other two was seen at a 1 million, and the other was seen at a dose of 0.5 million. And in terms of the organs, all three had a grade 2 skin and one patient, the 1 million patient, had some nausea; it's unclear because often got biopsies are very inconclusive and because it was early, it's unclear what that is or isn't -- does or doesn't have a gut component to it. But basically, the center chose as soon as the patient developed nausea to go ahead and treat was AP1903 and in all three of those cases within 24 hours the symptoms were gone.

So these are sort of -- again, very similar to the settings that we have seen previously, and currently are not seeing any or certainly not aggressive grade -- higher grade GvHD at this time.

Alan Musso

So just to be clear, so these are case of GvHD that haven't responded to standard-of-care. So the four cases above that didn't respond to the standard-of-care that were given rimiducid, so we're obviously are treating with these atopical or I think in some cases, systemic steroids that performed it. So the overall GvHD profile is probably broader or more complex than just as score that received media center.

Annemarie Moseley

Exactly, but again from the clinicians perspective, both with respect to immune reconstitution, not just of T-cells but also of B-cells and the immunoglobulins and all the other features that go along with immune reconstitution in the non-malignant and the malignant cases. And then also in terms of preserving as much as possible the GVL effect if that's the potential case which we're still of course evaluating. From the clinicians perspective, they really are waiting to treat with the rimiducid until it's absolutely necessary from their clinical perspective, and that's a little bit different for each patient and each clinician.

Daniel Wolf

Thanks for answering the call. Do you guys -- can I ask one more question, if possible?

Annemarie Moseley

Sure.

Alan Musso

Sure, go ahead.

Daniel Wolf

Okay. So in terms of the pediatric allo-procedures in AU, that would be elected. What about plans for adult?

Alan Musso

So we actually have -- as I indicated, we got a number of trials that are ongoing currently, including in trials of the adult setting. We have focused on the pediatric setting for a number of reasons including of course the appeal of being able to cure children with non-malignant diseases and potentially children with leukemia and lymphoma as well, and that introduces an attractive -- as we are seeing now, early pathway towards approval. In the adult setting; first of all, I would point out that the Hopkins protocol or post-side [ph] defending on how you refer to it, it's being used in adults, it's not being used in children. So it's not of relevance comparators you like all the pediatrics across the development plans currently. It probably becomes more relevant in adult patients and particularly older adult patients. And with that I'm going to pass to Annemarie to give a little bit more color.

Annemarie Moseley

And so and we are continuing for the younger and more robust adult patients to push ahead the very actively with our alpha-beta [ph] regiments followed by the BPX-501 in a number of centers are participating on that, however we've also been approach by number of investigators, and we are now beginning in the development and it's still early and it's going to be as someone exploratory to understand what the PPX-501 product can do in the siting of pro-sign and obviously we don't know like we do when we have an autograph, the exact content of the bodies T-cells, but certainly in many of these regiments there is a suggestion that there is quite a deep kill of T-cells as evidenced by lack of immune recovery, and in some patients as well, of course, there is a certainly a lack of GVL. And so we will be examining as we have in the T-depleted, ex-vivo T-depleted therapy, we'll be looking also well with a number of centers at evaluating of the use of PBX-501 prophylactically for the treatment again of immune reconstitution and also of the evaluation of GVL in that post sight sittings, this is kind of an exciting and again as I said somewhat very exploratory area for us, but we feel that we want to be able to capture as much of the potential of the cells in the adult of setting as we can. So we are -- so again stay tuned for that as we progress along this pathway.

Daniel Wolf

Thanks for answering my question.

Operator

Thank you. And our next question comes from Peter Lawson from SunTrust. Your line is now open.

Unidentified Analyst

Hi, this is Saumitro [ph] from Peter Lawson from SunTrust. Thanks for taking the question. I just wanted to get a little clarity on the date for the registration filing in Europe. So for 50% of the target 120 patients been enrolled and -- If the primary readouts are six months or a year or should we think of the registration filing to the year in 2017 or the 2018. The second question be when you are expecting to meet with the FDA for discussing the U.S. filing. Thank you.

Alan Musso

So I am going to pass over those to Annemarie Moseley.

Annemarie Moseley

So as you can imagine that is actually the multimillion dollar question, so at this point I think -- I think we are a -- again we can't say anything until we finish as Thomas explained our final meetings with the protocol assistance when we will draw down exactly on the number of patients but I think that the time frame that you have concluded very quickly on my add, certainly is one that we are optimistically thinking about, you know assuming that everything and at the protocol systems soon is along our pathway, we are going to be meeting in the upcoming months with the FDA we had initially wanted because the majority of our patients today are in roles and in the European and jurisdictions, we wanted to get clarity from the EMA before we meet with FDA so that we can be fully aligned and maximizing leverage from a global perspective.

So again that also affects enrollments and timelines and filing. So again I think that we hope in the next few months to really have clarity for you and around the exact nature of the timing, but that I think you know sort of that pretty much I think as much as we can say at this point with clarity.

Alan Musso

So just to close the loop on that, I'll come back to -- something we're very excited out which is, you know, clearly -- EMA is looking for a broad data set, across all pediatric patients who don't have an available match donor, this is not a case of you know let's talk with Thalassemia patients, let's talk with will stop with the Immune deficiencies, moving up towards eventually a marketing authorization application that would be pretty broad in terms of capturing the children that we want to bring this treatment to. And as I think Annemarie touched on in her earlier answer, by the time, we'll expect to have some meaningful longer term follow-up. While six months is relevant for immune reconstitution, we also expect our longer term follow-up on many of the leukemia and lymphoma patients.

So we're pretty excited about sort of the Brex [ph] of the filing that we're anticipating and you know I think again just kind of real firm our position. We are going to be doing everything we can, subjects to things we can't control, to be in a position to file by late 2017. And I think that's puts us in a pretty exciting position. You know but in terms of the potential to get a product launched in Europe of the gene modified cells -- T-cell products, getting all of the infrastructure in place of for that including manufacturing, logistics, commercial, that we can leverage into everything we would do that's coming along behind. So I think that's kind of a unique features that Bellicum -- perhaps got little overlook to the extent at which -- where the physicians who established a leadership position in Europe, in addition to of course have a very compelling products for the U.S. markets.

Unidentified Analyst

I see, so you would say once you sort these out for the European filing, then you would think about meetings with FDA in 4Q you would say?

Alan Musso

No, I think the goal is that we're making that transition now, as I'm sure you know the end of phase 2 process has a clock that is associated with that. Now that we've completed everything in Europe we're kind of at the beginning of that cycle with the FDA, so certainly we would expect to have feedback from that process in the fourth quarter. So I think basically by the time we get to -- it's at ASH where we do expect a host and investor events. We would like to be able to provide not only sort of comprehensive updates on clinical data but also a nuance insight into the global Commercialization plan for BPX-501.

Unidentified Analyst

Thank you so much for the answers. Great.

Operator

Thank you. [Operator Instructions] And our next question comes from Norman Puro [ph]. Your line is now open.

Unidentified Analyst

Good afternoon, Tom and Annemarie. What I'd like to clarify if I may, is that at the MAA would be collectively both malignant and non-malignant patients that would be part A to my question. And then I have a question on the separate MAA for rimiducid as well.

Annemarie Moseley

Yes, so you are correct. It is both for malignant and non-malignant patients. And okay and on the rimiducid you say.

Unidentified Analyst

Yes so did you have it at least a preliminary discussion that from a safety perspective. And how many rimiducid treatment would they like to see at the minimum.

Annemarie Moseley

So currently the -- as you know in the United States the rimiducid and the BPX-501 are a combination products. So in Europe they are a separate products and so as a result of those two filings have been going side by side, but we are very own thankful and appreciative that at the EMA they have understood the linkage between the two products. And the fact that they -- in fact are inter related so we have been having discussions on the overall plan and again the details around the specifics for the rimiducid we believe are going to be very tightly linked to the BPX-502, and again the final clarity on that will come after protocol assistance. But they definitely do understand that rimiducid is a product that is not used with every patient.

So again getting that final clarity we expect that these will be somewhat similar in their overall -- again they've been reviewed together all say and they understand that this is used much as sort of an antidote of their words, and so I think that that we are very optimistic that the package that will be creating for the overall 501 filing including the rimiducid and the GVH responsive to rimiducid will be adequate but we will need to get clarity on that at these upcoming protocol assistance.

Unidentified Analyst

And then Annemarie maybe a follow or somewhat unrelated actually, in the pancreatic cancer trials would a single patient be eligible to be treated with multiple doses of rimiducid?

Annemarie Moseley

At this point in time the protocol calls for one dose, while we understand then the pharmacokinetics of the cells and the response. And so at this point should a single patient for some reason be eligible were being in need another goes through if we choose to do that we will go back, we list and certainly the conversation open with FDA to discuss with them to address this question, so at this point it is not planned and but again and that it could also they -- could very well happen with the given patient if that were the case.

Unidentified Analyst

But in the cohort testing multiple -- single doses of rimiducid if I understood correctly.

Annemarie Moseley

Correct, we are dose and we are trusting and each cohort actually is a single dose of rimiducid and the doses of cells increase. And so this is why we have not initially started with multiple doses of rimiducid because we first need to understand with the given dose of cells is that activity potentiated to the degree it has a safety effect or not, so this is sort of how the FDA has reviewed, this is how we design study in such a way to understand; first what is the relationship with the cell does and the rimiducid and then the pharmacokinetic side of the cell in the body in their activity, and then we will come back in a subsequent trial and of course and increase the rimiducid appropriately based on the tracking of the cells that we've done, because each of our construct [ph] has a marker so marker in it. So at this point we believe that the cells will have activity and that's that activity then will be potentiated with the rimiducid at the appropriate time that which we've been able to assess the safety of the cells themselves. So in many ways, this is an absolutely a dual component, we are looking for the safety of the cells and the safety of the rimiducid with the cells. And of course then, if any activity comes we would of course hope to see that as well.

Unidentified Analyst

Thank you very much.

Annemarie Moseley

Okay, thank you.

Operator

Thank you and our next question comes from Biren Amin from Jefferies. Your line is now open.

Biren Amin

Yes, thanks for taking my question maybe just a follow up on the last question on 601, have you - I guess or what instructed your choice on the rimiducid dose that you're using in the Phase 1? Thanks.

Annemarie Moseley

This is the dose we have the equivalent dose that we have used in all of our animal studies. It's the clinical dose that has been used and at this point the overall design has been such to evaluate the cells as I just said, and then give the dose that we know will act on the dose itself that is there. In the future, should the data warrant that, of course we have the ability to titrate and play with dose, if you will, but at this moment, we're using the dose that has been studied in the patient in our uses of rimiducid and so this is the dose that we're starting with now.

Biren Amin

Okay. And then for 501, the European authorities now have a new pathway, the Prime Pathway. Have you guys applied for that to potentially have accelerator approval for 501 once it's file?

Annemarie Moseley

Currently, we have already engaged with them, the scientific advice process, at the time that the prime process, if you will, was instituted. With our orphan status as well, and where we are with the rare diseases, and for exceptional circumstances, we believe that we are already, I would say, as about as accelerated as one can be for this. But we will certainly work with the Agency and ask if there's any additional benefits that we might have available to us through the Prime. But again, at this point, we've been very pleased with our actions with the European agencies and as I said, I can't say enough that we're so thankful that they understood the products and so worked with us to find a way to get a non-combination combination kind of review. So at this point, if there's anything else from an accelerated standpoint, we'll certainly explore that.

Biren Amin

Great, thanks.

Operator

Thank you. [Operator Instructions] And we do have a follow-up question from Pierre Lawson [ph] from SunTrust. Your line is now open.

Unidentified Analyst

Hey, Thomas, it's Peter Wilson. Just a follow-up just around what's been happening with some of your peers on the T cell side of things. Have you seen any extra scrutiny or extra inbound questions from the FDA or the AMA around the T cell programs, at least the 501?

Tom Farrell

No, our most recent interactions have been around BPX-601 and -701. I think our observation would be that they are being careful in their review. They appear to be applying some sort of consistent thinking. We see novel constructs coming through their office. But I don't think we've seen anything that we could say was explicitly tied to specific recent circumstances.

Unidentified Analyst

Okay, perfect. Thanks. And then, you filed -- or you're filing for the pediatric population. When do you start filing for an adult population for 501?

Tom Farrell

I don't think we're in a position to speculate on that at this point. We've got a number of avenues of exploration in the adult setting, including, as Annemarie described, looking to see what we can do to improve outcomes with the Hopkins Protocol post-side. so post-post side, if you like. And the BP008 Protocol, which is looking at control of relapse or treatment of relapse, in those settings, obviously the primary objective of giving the 501 cells is to capture the [indiscernible] leukemia benefit. And depending on the specific disease population, that could be a 6/12/24-month or longer follow-up in order to confirm whether we're seeing that GVL effect in the corresponding benefits in terms of overall survival or survival in some form.

So I think at this point, we'd have to say that that requires a little bit more data, a little bit more clinical experience, and sort of an idea which of those pathways might give us the most expedient pathway to an approval in the adult setting.

Unidentified Analyst

Great. Okay, thanks much.

Operator

Thank you. And we do have a follow-up question from Daniel Wolf from Piper Jaffray. Your line is now open.

Daniel Wolf

Thanks, guys. I have a follow-up regarding the exceptional circumstances. Can you just discuss the implications of what an exceptional circumstance means? And would full approval be needed? And how? Thank you very much.

Tom Farrell

So exceptional circumstances is provided for in EMA regulations. I believe it dates back to about 2005. And it's a provision that specifically acknowledges that there are certain orphan products and indications where it is not feasible to develop a complete dossier. So this is different from say, conditional approval, where the initial approval is based on a surrogate of some description but then there's a requirement to follow up with a randomized controlled trial to confirm the effects, and that then results in conversion from conditional to full approval. In the case of exceptional circumstances, as related to our specific circumstances, we believe that the EMA reviewers understand that, in this pediatric population, there really is not a feasible control arm for these no-option patients.

So this is a specifically -- an approval under circumstances that does have some follow-up requirements but there is no subsequent conversion of that approval to something else. So it is a, in our minds, a more attractive and complete outcome for an orphan product than the one that would be -- that you'd be looking at with a conditional approval. We think, too, that it's probably going to be -- we'll put it to the stronger position with respect to the dossier that would be required by payers to support reimbursement.

Daniel Wolf

And so, does the follow-up [ph] will not be needed?

Tom Farrell

No, that's correct. There would be no need for a follow-up in the pediatric setting. As I think we've indicated, the adult setting is another discussion because of the, kind of growing emergence of, or acceptance of, post-side of the Hopkins Protocol. It's really not standard of care, but it's becoming accepted as sort of a valid option for these patients. Of course this practice of medicine is not a regulated product, so it does not require any -- by itself -- any randomized controlled trials, and that indeed has resulted in a situation with a lot of uncertainty about the overall outcome from the procedure. Of course there's also a lot of heterogeneity in how the procedure is conducted. So as we think about that in potential opportunity, we're very interested in sort of lifting the curtain a little bit in terms of the longer-term outcomes from post-side, and how we can improve those and particularly relapse by the addition of BPX-501.

Daniel Wolf

Thank you very much.

Operator

Thank you. And this does conclude our question-and-answer session. I would now like to turn the call back over to Tom Farrell, Chief Executive Officer, for any further remarks.

Tom Farrell

Great, well, thank you again for participating on today's call. I hope it's been instructive and informative. As you've heard, 2016 is shaping up to be another highly productive year for us here at Bellicum. I'd like to acknowledge our leadership team and our terrific employees for their continued hard work and dedication, and of course, to thank our investors for their continued support. And with that, I wish you a wonderful evening. Thank you.

Operator

Ladies and gentlemen, thank you for participating in today's conference. This concludes today's program. You may all disconnect.

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