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Edited Transcript of BLCM earnings conference call or presentation 12-Mar-19 9:00pm GMT

Q4 2018 Bellicum Pharmaceuticals Inc Earnings Call

Houston Mar 15, 2019 (Thomson StreetEvents) -- Edited Transcript of Bellicum Pharmaceuticals Inc earnings conference call or presentation Tuesday, March 12, 2019 at 9:00:00pm GMT

TEXT version of Transcript

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Corporate Participants

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* Aaron E. Foster

Bellicum Pharmaceuticals, Inc. - Head of Research & Senior VP

* Atabak Mokari

Bellicum Pharmaceuticals, Inc. - CFO

* Richard A. Fair

Bellicum Pharmaceuticals, Inc. - President, CEO & Director

* William J. Grossman

Bellicum Pharmaceuticals, Inc. - Chief Medical Officer

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Conference Call Participants

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* Biren N. Amin

Jefferies LLC, Research Division - MD and Senior Equity Research Analyst

* James William Birchenough

Wells Fargo Securities, LLC, Research Division - MD and Senior Biotechnology Analyst

* Peter Richard Lawson

SunTrust Robinson Humphrey, Inc., Research Division - Director

* Reni John Benjamin

Raymond James & Associates, Inc., Research Division - Senior Biotechnology Analyst

* Wangzhi Li

Ladenburg Thalmann & Co. Inc., Research Division - MD of Equity Research of Biotechnology

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Presentation

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Operator [1]

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Ladies and gentlemen, greetings, and welcome to the Bellicum Pharmaceuticals Fourth Quarter and Full Year 2018 Financial Results. (Operator Instructions) As a reminder, this program is being recorded.

It is now my pleasure to introduce your host, Atabak Mokari, Chief Financial Officer. Thank you. You may begin.

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Atabak Mokari, Bellicum Pharmaceuticals, Inc. - CFO [2]

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Thank you. Good afternoon, everyone, and thank you for joining the call. With me today is Rick Fair, Bellicum's President and Chief Executive Officer; Bill Grossman, Chief Medical Officer; and Aaron Foster, Head of Research.

Earlier this afternoon, Bellicum released financial results for the fourth quarter and full year ended December 31, 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 Form 10-K for the year ended December 31, 2018, filed with the Securities and Exchange Commission.

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

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Richard A. Fair, Bellicum Pharmaceuticals, Inc. - President, CEO & Director [3]

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Thanks, Atabak. Good afternoon, everyone, and thanks for joining us. I'm excited to share with you today the progress we've made across our pipeline of controllable cell therapies as well as our key objectives for 2019.

On our call today, I'll provide an update and outline 2019 goals for our 2 strategic priorities, namely our GoCAR-T pipeline and rivo-cel. Before I do, I'll begin with a brief reflection on my time at Bellicum as context for our 2018 accomplishments and future prospects.

Over the last 2 years, we've made significant progress in strengthening our organization. We've recruited a new executive team with deep expertise. We've improved our capabilities across the company, in particular in clinical development. And we've instilled a patient-focused culture built on great science, performance and accountability.

While Bellicum has always had exciting science and technology, we've worked hard to match that with great execution, progressing product candidates efficiently to and through the clinic. The proof of these efforts is in the results. In 2018, we set a company record for achievement of our corporate goals and objectives, which included presentation of initial data from our BPX-601 study, progression of 2 preclinical dual-switch GoCAR-T programs with expected IND applications in 2019, completion of enrollment in our pediatric trials for rivo-cel and presentation of late interim analysis from these trials, initiation of launch preparation for rivo-cel in Europe for pediatric patients, initiation of THRIVE Phase II/III study for rivo-cel in adult and adolescent AML and MDS patients, and completion of the second phase of our Houston cell and viral vector manufacturing facility.

In addition to this goal attainment, we continue to recruit and develop top talent, as evidenced by our recent appointment of Atabak Mokari as CFO, promotion of Aaron Foster to Senior Vice President and Head of Research and addition of Judith Klimovsky to our Board of Directors. We also recently formed a new Scientific and Clinical Advisory Board comprised of leading academic clinicians and scientists to provide valuable external perspective on our programs. In short, I believe we've laid the foundation for even more progress in the coming years.

With that look back as context, let's turn now to our 2 strategic priorities, starting with our GoCAR-T pipeline. Our GoCAR-T platform incorporates our inducible MyD88 and CD40, or iMC, activation switch. As a next-generation CAR-T platform, we believe iMC may improve upon current generation products in several ways. First, co-stimulatory molecules, MyD88 and CD40, enhance T-cell proliferation and persistence. Second, our preclinical research suggests iMC may modulate the tumor micro environment by overriding common inhibitory pathways such as PD-1, PGE2 and TGF-beta. Third, we believe that iMC may enhance host immune activity to complement CAR-T efficacy by inducing pro-inflammatory cytokines and chemokines. Lastly, because iMC is driven by our switch technology, we can control the timing and frequency of CAR-T cell activation through the infusion of our small molecule rimiducid, which may enable clinicians to better manage the benefit-risk profile and treatment.

As you know, our first GoCAR-T product candidate is BPX-601, which targets prostate stem cell antigen, or PSCA. At the ESMO immuno-oncology meeting in December and ASCO GI in January, we presented data from Part 1 of our ongoing Phase I/II dose escalation study in patients with late-line metastatic pancreatic cancer expressing PSCA. In the study, a total of 12 patients with advanced disease were treated with escalating doses of BPX-601 cells in a 3 plus 3 design. In its early safety evaluation, the trial was designed conservatively. Patients received a reduced lymphodepletion conditioning regimen consisting of cyclophosphamide only. Three patients received a low dose of BPX-601 cells only, and the other 9 received a single dose of rimiducid to activate iMC once following BPX-601 treatment.

The interim results proved encouraging despite inadequate lymphodepletion, a single administration of rimiducid to activate iMC and the extreme challenge of treating second to sixth line pancreatic cancer. Initial data demonstrated that BPX-601 cells have a promising safety profile with no reported cytokine release syndrome or other high-grade treatment-related adverse events.

In addition, a single dose of rimiducid led to enhanced cell expansion and prolonged cell persistence in several patients, providing first proof of concept of iMC in humans. Furthermore, the data provided preliminary evidence of clinical activity and disease control with 4 of 6 efficacy evaluable patients achieving stable disease, 2 of whom experienced tumor shrinkage greater than 20%.

As this is our first human experience with iMC, we're still learning quite a bit. We've amended the trial to include standard cyclophosphamide/fludarabine, or Cy/Flu, conditioning and have expanded eligibility to gastric and prostate cancer patients expressing PSCA with the intent of strengthening the early efficacy signal we've seen. We expect to report initial results from this patient cohort midyear. Once we complete the safety evaluation of the Cy/Flu cohort, we plan to amend the BPX-601 trial to allow for scheduled repeat dosing of rimiducid in order to reactivate iMC over time.

As a reminder, the iMC switch was designed to be activated on a regular basis. Our preclinical experience suggests that regular rimiducid dosing can reactivate and expand GoCAR-T cells over time without creating T-cell exhaustion, maximizing the clinical efficacy potential. We expect initial results from this patient cohort by the end of this year.

Based on the totality of data to date, we're optimistic about BPX-601 as a product candidate and as a proof of concept for our GoCAR-T platform. We're excited to advance 2 controllable dual-switch GoCAR-T candidates into the clinic with IND submissions planned for 2019. Our dual-switch platform incorporates both the iMC activation switch and the CaspaCIDe safety switch. We're excited about this platform given the potential combination of efficacy benefits of iMC and safety benefits of CaspaCIDe, which may enable more aggressive treatment protocols that further enhance efficacy.

BPX-603 is Bellicum's first dual-switch product candidate and is designed to target solid tumors that express HER2. We selected HER2 as a target for BPX-603 because it is a thoroughly validated antigen for cancer therapy and academic CAR-T cell clinical studies have demonstrated antitumor activity. Previous HER2 CAR-T approaches have been limited by modest efficacy and off-tumor/on-target toxicity. We believe that our dual-switch technology may be uniquely suited to improve upon these earlier efforts by driving greater efficacy through iMC activation while enabling clinicians to manage any treatment-emergent toxicity with CaspaCIDe. We expect to file an IND application for BPX-603 and initiate a clinical trial in 2019.

Later in 2019, we expect to file an IND application for BPX-802, a dual-switch product candidate targeting an antigen expressed in hematologic malignancies. We will discuss the target and rationale later this year as we get closer to IND submission.

Now turning to our other strategic priority, rivo-cel. We presented late interim analysis of our pediatric study at the American Society of Hematology Meeting in December of 2018. The interim results on 249 patients support our confidence in having a positive trial at the final analysis as well as a commercially attractive profile.

The ASH presentations offered 3 important takeaways. First, the 6-month event-free survival, or EFS, in rivo-cel-treated patients is trending ahead of EFS in patients undergoing matched unrelated donor, or MUD, transplantation. As a reminder, noninferiority on EFS versus MUD transplant is our agreed-upon primary endpoint for European regulatory review. Second, the data provided further evidence of the durability of efficacy of rivo-cel with high rates of relapse free and overall survival with a median follow-up of almost 2 years. Lastly, the results demonstrated the efficacy of CaspaCIDe as 70% of patients that developed advanced or steroid-refractory GvHD responded when treated with rimiducid, the majority of which were complete responses.

We expect top line results from this study in the second quarter and intend to submit European Marketing Authorization Applications for rivo-cel and rimiducid in late 2019. Looking ahead, we recently initiated THRIVE, a randomized global Phase II/III clinical trial designed to potentially expand the registration of rivo-cel both to the U.S. and to adult patients. The study is enrolling adult and adolescent patients 12 years and older with intermediate and high-risk acute AML or MDS. In the Phase III portion of the trial, patients will be randomized to receive either the rivo-cel regimen or the Baltimore regimen, a T-replete haplo-transplant followed by post-transplantation cyclophosphamide, which is the current standard of care in the U.S. and Europe in adults without an HLA matched donor.

Lastly on rivo-cel, I'd like to turn to the commercial opportunity that we see for this product. Based on the preparation we've done to date and drawing upon my years of experience launching novel therapeutics in oncology and hematology globally, I'm excited about the commercial prospects for rivo-cel. My excitement and growing confidence are supported by a number of factors.

First, the addressable patient population is significant. Our initial addressable market opportunity in European pediatric patients is approximately 1,800 patients per year and is growing 4% annually. Our second launch in adult and adolescent AML and MDS patients would add almost 10,000 addressable patients per year in the U.S. and Europe to this opportunity.

Second, based on rivo-cel's compelling clinical value proposition and encouraging early feedback from KOLs and physician market research, we believe that rivo-cel has the opportunity to capture substantial market share. Specifically, we believe we have the opportunity to become the standard of care in haplo-transplantation and also to penetrate the segment of patients who might otherwise have received a MUD transplant.

Third, we're encouraged by our early payer market research and health economic analysis, which indicate payers will support favorable pricing and reimbursement consistent with currently approved cell therapies.

Fourth, our initial launch will require limited commercial investment as the pediatric transplant market in Europe is highly concentrated. We estimate the top 75 European centers represent approximately 80% of the pediatric market opportunity.

Lastly, we're excited about our recently recruited European commercial and medical leadership team, a talented, experienced group with a track record of successfully launching products. With them on board, we're making great progress toward our anticipated launch of rivo-cel in 2020.

With that, I'd like to hand the call over to Atabak to review our financials.

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Atabak Mokari, Bellicum Pharmaceuticals, Inc. - CFO [4]

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Thanks, Rick. Bellicum reported a net loss of $27.2 million for the fourth quarter of 2018 and $98.0 million for the year ended December 31, 2018, compared to a net loss of $21.9 million and $91.8 million for the comparable period in 2017. The results included noncash share-based compensation charges of $3.0 million and $13.8 million for the fourth quarter and year ended December 31, 2018, respectively, and $3.4 million and $13.6 million for the comparable period in 2017.

R&D expenses were $19.8 million and $71.2 million for the fourth quarter and year ended December 31, 2018, respectively, compared to $14.3 million and $65.7 million during the comparable periods in 2017. The higher expenses in the fourth quarter and full year 2018 compared to respective periods in 2017 were primarily due to an increase in costs related to our GoCAR-T product platform and general research and development expenses, partially offset by a decrease in expenditures related to rivo-cel.

General and administrative expenses were $7.0 million and $25.0 million for the fourth quarter and year ended December 31, 2018, respectively, compared to $5.1 million and $21.0 million during the comparable periods in 2017. The higher expenses in the fourth quarter and full year 2018 compared to respective periods in 2017 were primarily due to increased personnel-related costs due to hiring additional employees.

On the balance sheet front, as of December 31, cash, restricted cash and investments totaled $98.0 million. Based on current operating plans, Bellicum expects the current cash resources will be sufficient to meet operating requirements through the end of 2019.

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

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Richard A. Fair, Bellicum Pharmaceuticals, Inc. - President, CEO & Director [5]

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Thanks, Atabak. As you've all heard, we've had a productive 2018 and anticipate many important milestones throughout 2019 for both our GoCAR-T pipeline and rivo-cel. I'm proud of our team and all that we've accomplished together, and I'm as excited as ever in the promise of our controllable cell therapy platform.

I'll now open the call to questions.

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Questions and Answers

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Operator [1]

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(Operator Instructions) Our first question comes from the line of Brian (sic) [Biren] Amin from Jefferies.

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Biren N. Amin, Jefferies LLC, Research Division - MD and Senior Equity Research Analyst [2]

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On 501, given the MAA filing for [indiscernible] this year, how are you thinking about the launch? And can you just go through dynamics in Europe as it relates to Zalmoxis, which is on the market? And what have been the issues with that launch? And what are the key takeaways that you're going to learn from the Zalmoxis launch?

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Richard A. Fair, Bellicum Pharmaceuticals, Inc. - President, CEO & Director [3]

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Sure. Thanks for the question, Biren. Yes. so as mentioned, we've assembled a leadership team in Europe. That's begun launch preparation. We've already learned a lot from that. Of course, 2019 and 2020 will be good planning years in anticipation of the 2020 approval. I think the Zalmoxis launch has been disappointing, I'm sure. And as we look at that analog, I'd say, 2 things strike us. One is the product profiles are quite different. We have a stronger product profile. Kind of 3 things that we think about there. One is just the superior, much larger and higher-quality data package. Two is the superior safety switch. So the intent of this product is be able to deliver the benefits of T-cells while managing the toxicity. And our safety switch has been demonstrated to work more quickly and more comprehensively in patients. I think the third thing is the construct that they're using as immunogenic. And so we've shown great evidence with our construct that we have durable cells that stay around long enough to provide adaptive immunity in these patients for a long period of time, and we think that leads to a better efficacy profile to cells themselves. So I think those 3 ways are -- our product profile stands out as being superior, and that's a positive. I think the second piece of it is just launch preparation. It doesn't appear from the outside looking in that they prepared much in advance for that launch. And so they've been trying to do that post-approval, and that's difficult to do. We are well ahead of the game with the preparation underway 2 years prior to approval. We will be very ready to handle the pricing and reimbursement aspects, KOL development aspects and the commercial launch plan upon approval.

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Biren N. Amin, Jefferies LLC, Research Division - MD and Senior Equity Research Analyst [4]

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And then just a question on 601. Do you think that you would need a PD-1 inhibitor to optimize activity of 601 at some point? So I just want to get your thoughts on that.

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Richard A. Fair, Bellicum Pharmaceuticals, Inc. - President, CEO & Director [5]

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Certainly, it's possible. And certainly, it's something we're considering. I think we'd like to demonstrate some additional monotherapy activity in the study as currently constructed. So as you've heard, we're making modifications to that trial with the Cy/Flu lymphodepletion regimen and the repeat dosing of rimiducid to maximize the clinical activity in monotherapy, but certainly expect that a combination with a PD-1 or PD-L1 inhibitor could be the next step.

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Biren N. Amin, Jefferies LLC, Research Division - MD and Senior Equity Research Analyst [6]

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And so I guess when should we think about when you could enter into like a dose expansion phase where you would evaluate these sets of drivers?

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Richard A. Fair, Bellicum Pharmaceuticals, Inc. - President, CEO & Director [7]

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So our current plans are to have the Cy/Flu cohort enrolled and presented midyear to run the trial to allow for repeat dosing of rimiducid and begin enrolling patients such that we can present first patients from that cohort by the end of this year and move into full expansion in prostate, gastric and pancreatic cancer in early 2020.

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Operator [8]

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Our next question comes from the line of Jim Birchenough from Wells Fargo Securities.

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James William Birchenough, Wells Fargo Securities, LLC, Research Division - MD and Senior Biotechnology Analyst [9]

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Just a few. Just on rivo-cel to start with, when you think about the type of pricing discussions you're having and considering the approved product in Europe and pricing around that, could you maybe speak to the gross margins that we should be thinking about? Should we be thinking about CAR-T type gross margins or something better? And then I've got a few follow-ups.

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Richard A. Fair, Bellicum Pharmaceuticals, Inc. - President, CEO & Director [10]

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Sure. I don't think we can get specific on gross margins, but I do think the CAR-T pricing and cost of goods is a reasonable analog.

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James William Birchenough, Wells Fargo Securities, LLC, Research Division - MD and Senior Biotechnology Analyst [11]

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And then maybe just in terms of turning to 601 and expectations for data midyear, with Cy/Flu preconditioning, should we expect more robust tumor responses? Or is this really still focused on safety and cell expansion and will need maybe repeat dosing to expect durable responses? Just trying to get a sense of expectation for the midyear data.

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Richard A. Fair, Bellicum Pharmaceuticals, Inc. - President, CEO & Director [12]

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Sure. So we do think that, that Cy/Flu is important to lead to better engraftment and expansion of cells, which we just think is necessary for greater clinical activity. But we don't think our efficacy optimized regimen will occur until we are using iMC as planned with repeat dosing of rimiducid. So it's a step along the way. We'll present early results on both safety and activity. And if there's an impact, we'll show that, of course. But I would be looking more towards late this year to start seeing the impact of iMC and its efficacy-optimized schedule.

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James William Birchenough, Wells Fargo Securities, LLC, Research Division - MD and Senior Biotechnology Analyst [13]

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And maybe just one final one on 601. If you think about broader expression of PSCA, is there any thought of doing a basket trial? Or are you really capturing the main tumor types with prostate, gastric and pancreatic?

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Richard A. Fair, Bellicum Pharmaceuticals, Inc. - President, CEO & Director [14]

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No current plans to do a basket trial. Those 3 tumor types do capture the majority of PSCA expressing tumor patients as far as we know. The literature on this is a little skinny. So it's possible there's more PSCA expression out there than has been published on, but we do think we've covered the majority of patients with those tumors.

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James William Birchenough, Wells Fargo Securities, LLC, Research Division - MD and Senior Biotechnology Analyst [15]

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And maybe final somewhat related. But we've seen at least in relapse gastric cancer, several companies had [bait] and have to move earlier frontline because the patients progress so quickly. I think that's pretty typical of pancreatic late line as well. So how do you think about migrating earlier and when might that happen in your development?

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Richard A. Fair, Bellicum Pharmaceuticals, Inc. - President, CEO & Director [16]

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Well, I think this is -- and Bill, our CMO who's on the line, may want to comment. But just in anticipation of that, I think it's typical, of course, to look for safety and early activity in the later line population. In our study, we have moved up. So as I mentioned in the dose escalation phase, we had very generous guidelines. We had as late as sixth line pancreatic cancer patients, which, of course, are very difficult to treat. In our most recent modification, we've moved up to second line patients exclusively, partly for the reason that you cite. But certainly, we want to demonstrate some meaningful activity there before we started to compete with existing standards of care in frontline. Bill, do you have anything you want to add to that?

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William J. Grossman, Bellicum Pharmaceuticals, Inc. - Chief Medical Officer [17]

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Yes. I was just going to add to what you just cited, Rick, which was we have a much more homogeneous population of patients who are both second line (inaudible) and second line gastric (inaudible) pre-prostate. So we are cognizant of that runway for immunotherapy responses, and that's what we're trying to address with that new amendment.

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Operator [18]

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Our next question comes from the line of Wangzhi Li from Ladenburg.

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Wangzhi Li, Ladenburg Thalmann & Co. Inc., Research Division - MD of Equity Research of Biotechnology [19]

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Maybe starting with BPX-601. Just to clarify, will biopsy data be reported for the mid-'19 and late '19 data report?

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Richard A. Fair, Bellicum Pharmaceuticals, Inc. - President, CEO & Director [20]

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Sorry, Wangzhi, could you repeat that question?

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Wangzhi Li, Ladenburg Thalmann & Co. Inc., Research Division - MD of Equity Research of Biotechnology [21]

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So for BPX-601 data report initially at the mid-'19, right, and again later '19, we see any biopsy data to see if the cells getting to the tumor or the tissue?

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Richard A. Fair, Bellicum Pharmaceuticals, Inc. - President, CEO & Director [22]

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Yes, it's possible that we will. As you may know, when we amended this trial to incorporate Cy/Flu and to add prostate and gastric patient eligibility, we did put in a requirement where it's safe to do so to conduct on treatment biopsy. So it's possible that you'll begin to see the data this year.

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Wangzhi Li, Ladenburg Thalmann & Co. Inc., Research Division - MD of Equity Research of Biotechnology [23]

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Got it. Great. And then for the repeated dosing, any color on how frequent repeated dosing to think about or how many repeated dosing to think about? I mean, is there any insight on this at this moment or any color on that?

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Richard A. Fair, Bellicum Pharmaceuticals, Inc. - President, CEO & Director [24]

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Yes. So based on our evaluations preclinically, we think a weekly schedule will make some sense and dosing progression or dose-limiting toxicity might make some sense, but of course, we want to engage the FDA in that discussion. So we'll keep you posted as we modify that trial and where we land.

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Wangzhi Li, Ladenburg Thalmann & Co. Inc., Research Division - MD of Equity Research of Biotechnology [25]

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Got it. Okay. And then for the dual-switch CAR-T, could you share any information about the antibody [SGF] we are using for your HER2? Is it the same or better with CAR-T? Or is it proprietary different one?

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Richard A. Fair, Bellicum Pharmaceuticals, Inc. - President, CEO & Director [26]

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Maybe I'll turn that question over to Aaron Foster, our Head of Research, who's here with us. He can talk about the construct.

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Aaron E. Foster, Bellicum Pharmaceuticals, Inc. - Head of Research & Senior VP [27]

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Yes, sure. So the construct currently contains a modified version of trastuzumab antibody, [the clinical 45] that has mutations in it. That's lower the affinity to make it more selective for HER2 expressing tumor assist data that's published earlier at the (inaudible).

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Wangzhi Li, Ladenburg Thalmann & Co. Inc., Research Division - MD of Equity Research of Biotechnology [28]

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Good to have that -- or better differentiation in terms of recognized tumor HER2 versus normal tissue HER2 affinity or kind of titration?

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Richard A. Fair, Bellicum Pharmaceuticals, Inc. - President, CEO & Director [29]

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He's asking is it a recognized tumor HER2 better than healthy tissue?

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Aaron E. Foster, Bellicum Pharmaceuticals, Inc. - Head of Research & Senior VP [30]

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Right. So we've got a number of experiments obviously in our preclinical data set looking at the specificity and reactivity of this binder compared to others. As previously observed in the other studies, we noticed that there is a decreased reactivity against low-level antigen expression for HER2 with the modified binder. In addition, we've done a lot of efficacy studies comparing this binder to the previously published FRP5, which is the [nearing] binder that's been used in the Baylor study, and it has significantly higher activity against the number of different tumor models. So we think this is a good fit for the program. Of course, in this construct, we have 2 switches, one to control the activation state, of course, using iMC and again mitigating any tumor toxicities using caspase-9 if needed.

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Wangzhi Li, Ladenburg Thalmann & Co. Inc., Research Division - MD of Equity Research of Biotechnology [31]

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Got it. Maybe final question is so for this, are you targeting like Her2-positive breast cancer, gastric cancer or any color on what kind of patient you are going to treat for your Phase I trial initially?

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Richard A. Fair, Bellicum Pharmaceuticals, Inc. - President, CEO & Director [32]

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Sure, sure. So obviously, we want to have an IND approval and have FDA alignment on the design of the early trial, but it will certainly involve a dose escalation in the basket of HER2 expressing tumors. And certainly in future phases, we'll be looking at early signals of activity in a variety of HER2 expressing tumors. I don't think breast is our lead choice given the crowd there and given the many, many alternative therapies there. But certainly other tumor types like gastric, endometrial and others will be candidates for further development for this product.

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Operator [33]

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Our next question comes from the line of Peter Lawson from SunTrust Robinson Humphrey.

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Peter Richard Lawson, SunTrust Robinson Humphrey, Inc., Research Division - Director [34]

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When do you expect to see the 601 trial amended? Because I guess there's 2 amendments that need to be done.

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Richard A. Fair, Bellicum Pharmaceuticals, Inc. - President, CEO & Director [35]

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Well, the Cy/Flu amendment is done and implemented. The second amendment is the repeat rimiducid dosing. We expect to have that done by midyear.

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Peter Richard Lawson, SunTrust Robinson Humphrey, Inc., Research Division - Director [36]

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Perfect, okay. And then what further readout should we expect for rivo-cel ahead of the MAA submission?

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Richard A. Fair, Bellicum Pharmaceuticals, Inc. - President, CEO & Director [37]

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I think our current plan would be to present the final analysis this year from the BP-004 study. We expect the top line on that in the second quarter. Given that time line, it's unlikely we'd make EHA. So we're probably looking at an ASH presentation, but we haven't firmed that up. That will depend on once we have data on hand.

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Peter Richard Lawson, SunTrust Robinson Humphrey, Inc., Research Division - Director [38]

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Got you. And then just on the -- how should we think about that data? Or when should we think about the data for the Phase II/III THRIVE study for U.S. and adults?

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Richard A. Fair, Bellicum Pharmaceuticals, Inc. - President, CEO & Director [39]

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Yes. So you'll recall the design of THRIVE has a Phase II run in where we're evaluating a higher dose, 3 million cells per kilogram without GvHD prophylaxis in 10 patients to evaluate safety of that. We would expect to enroll all these patients and have long enough follow-up to draw conclusions on that this year. As to when and where we'll present those data, to be determined. But we'll have all of that in 2019.

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Operator [40]

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(Operator Instructions) Our next question comes from the line of Reni Benjamin from Raymond James.

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Reni John Benjamin, Raymond James & Associates, Inc., Research Division - Senior Biotechnology Analyst [41]

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Rick, you mentioned that the final top line data is in the second quarter of 2019. What's going to be materially different between what was presented at ASH versus now? About how many more patients? And should we really expect any sort of material change in terms of the efficacy or safety?

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Richard A. Fair, Bellicum Pharmaceuticals, Inc. - President, CEO & Director [42]

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Thanks, Reni, for the question. We'll have longer follow-up, all of the patients who have achieved the 6-month EFS threshold in the BP-004 data set, and we will have the remaining patients accrued in the comparative MUD study C/CP-004. But as you pointed out or implied in your question, these were late interim analyses that we presented at ASH. So they have most of the information that will be in the final analysis. So we wouldn't expect major changes in the outcomes, which is why we've expressed confidence that we have a file-able data set at the final analysis.

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Reni John Benjamin, Raymond James & Associates, Inc., Research Division - Senior Biotechnology Analyst [43]

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Right. Okay. And then just kind of switching gears to 601. You have a couple of patients that persist greater sort of than 3 weeks. You have a solid expansion that tends to have a decent bit of a variability between 3 and 20-fold. Can you talk a little bit about what you're learning in terms of how to bring those parameters maybe a little bit more online to have not as much variability? Do you need to? And just kind of thoughts on what might be causing these differences between patients.

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Richard A. Fair, Bellicum Pharmaceuticals, Inc. - President, CEO & Director [44]

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Yes. Maybe I'll start by asking Aaron Foster to comment and then Bill Grossman if he has anything he wants to add.

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Aaron E. Foster, Bellicum Pharmaceuticals, Inc. - Head of Research & Senior VP [45]

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Yes, sure. So I think one of the things that we've noticed, and of course, others in the field have with their CAR-T cell product is as they're coming from an autologous source, the patients' immune system are variable in that there is some potential to have variable performing products that come from these patients. I think that's by and large what we've seen as we looked at the product characterization profile in terms of transaction efficiencies and phenotypes and things like. And there's been a fairly uniform batch of cells that we've made. We started looking deeper into the functionalities, and we started to see some differences. I think one point that we're interested in exploring is can repeated doses of rimiducid to activate iMC switch overcome some of these expansion differences, and I think that's what we're hoping to see as we amend our clinical study.

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Richard A. Fair, Bellicum Pharmaceuticals, Inc. - President, CEO & Director [46]

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Bill, anything you care to add to that?

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William J. Grossman, Bellicum Pharmaceuticals, Inc. - Chief Medical Officer [47]

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One thing I'd expand on that answer would be that this was also done, as you know, under cytoxan lymphodepletion regimen, which was, as we reported, [as myelo], largely not lymphodepleting in majority of the patients. And so that, I think, also gave a lot of variability, as Aaron mentioned and as you pointed out. So I think once we get into the Flu/Cy regimen, we'll see hopefully more reproducibility around the proliferation and expansion that we didn't see with cytoxan across all patients.

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Richard A. Fair, Bellicum Pharmaceuticals, Inc. - President, CEO & Director [48]

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Maybe one last point is that we have extensive corollary studies that are taking place as we speak and continue the study. And one aspect, of course, that we're looking at is the expression level of PSCA and how that relates to CAR-T cell expansion. Other things like tumor burden and levels of lymphodepletion can all contribute to differential performance, both infusion.

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Operator [49]

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Thank you. Ladies and gentlemen, we have no further questions in queue at this time. I'd like to turn the floor back over to management for closing.

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Richard A. Fair, Bellicum Pharmaceuticals, Inc. - President, CEO & Director [50]

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Thanks, everyone, for participating today. As always, I would like to thank our passionate team of Bellicians, our collaborators and investigators for their efforts and most importantly the patients and families who participated in our clinical trials. They inspire our effort every day. Thanks, and have a great evening.

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Operator [51]

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Thank you. Ladies and gentlemen, this does conclude our teleconference for today. You may now disconnect your line at this time. Thank you for your participation, and have a wonderful day.

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