Alan Musso - Chief Financial Officer Tom Farrell - President and Chief Executive Officer Annemarie Moseley - Chief Operating Officer and Executive Vice President, Clinical Development

Tony Butler - Guggenheim Securities Biren Amin - Jefferies

Good day ladies and gentlemen, and welcome to the Bellicum Pharmaceuticals Inc. Fourth Quarter 2015 and Year End 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. [Operator Instructions] I would now like to introduce your host for today’s conference, Mr. Alan Musso, Chief Financial Officer. Sir, you may begin.

Thank you, Chemed. 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 quarter and full year ended December 31, 2015. 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 Relation's 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 plan 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 filed today with the Securities and Exchange Commission. And with that, now let me turn the call over to Tom Farrell, our CEO.

Thank you, Alan and good afternoon everyone. Thank you for joining us. Our goal this afternoon is to provide an overview of 2015, review financial results for the fourth quarter and full year, and outline the key objectives for the remainder of 2016. After that, we look forward to answering your questions. Overall I'm pleased to report that 2015 was a highly productive year for Bellicum and we made significant progress across all of our T cell immunotherapy programs. Our lead product candidate BPX-501, the next generation cell therapy for the hematopoietic stem cell transplant setting continues to demonstrate impressive results. As revealed at ASH 2015, pediatric patients with genetic blood disorders were alive and disease free after receiving a T depleted haploidentical transplant followed by an infusion of BPX-501 cells approximately two weeks following a transplant procedure to provide an immune reconstitution benefit. We are encouraged by these interim results and expect further updates in early April at the 42nd annual meeting of the European Society for Blood and Marrow Transplantation in both the malignant and nonmalignant settings. Investigators are reporting strong recruitment into the lead BPX-501 trial BP-004 with 63 patients in Europe and 12 patients in the U.S. enrolled to date. The combination of BPX-501 and rimiducid was recently granted orphan drug designation by the FDA as a T cell replacement therapy for the treatment of immunodeficiency and GVHD following allogeneic HSCT which we believe is an important milestone for the program. We look forward to meeting with regulators in Europe and the U.S. in the second quarter with the goal of defining the path to regulatory filing and approval initially in nonmalignant pediatric genetic diseases. Annemarie will provide additional details later in the call on the clinical results supporting our belief that BPX-501 could represent a vital treatment option for the many patients for whom a transplant is recognized as the preferred treatment, but who are not treated because they lack a perfect match donor. I’m also pleased to report that we continue to advance our TCR and CAR T product candidates all of which employ us our switch technologies throughout the clinic. These products are highly differentiated with unprecedented ability to control activation and elimination of the modified cells. We believe our cell therapies have disruptive potential with the unique safety and efficacy benefits. As you know, we recently met with the National Institutes of Health Recombinant DNA Advisory Committee which reviewed product candidates involving gene transfer about the BPX-501 and BPX-601 protocols. We believe the meetings went well and are moving forward with our plan to file IND with the FDA for these product candidates. As a reminder, BPX-701 is a high affinity T cell receptor or TCR product candidate incorporating our CaspaCID safety switch and is designed to target malignant cells expressing the preferentially-expressed antigen in melanoma or PRAME. We expect to begin enrollment and a Phase I clinical trial of BPX-701 in mid 2016 for initial planned indications of Refractory or Relapsed Acute Myeloid Leukemia and Myelodysplastic Syndromes. We also plan to initiate an additional Phase I clinical trial of metastatic uveal melanoma later in the year. Our GoCAR-T product candidate BPX-601 contains our proprietary iMC or inducible MyD88/CD40 activation switch that is designed to treat solid tumors expressing prostate stem cell antigen or PSCA. Unlike standard CARs, which is depended on antigen for activation and proliferation, GoCAR-T places the co-stimulatory signal on the rimiducid controlled switch which designed to allow control the T cell survival in the absence of antigen signaling and full activation and proliferation in the presence of both antigen and rimiducid. We will be studying BPX-601 in solid tumors over expressing PSCA, an attractive proprietary target in which there is significant unmet medical need. The initial planned indication is non-resectable pancreatic cancer and the initial Phase I clinical trial is also expected to begin enrollment in mid 2016. Preclinical data with BPX-601 presented at ASH 2015 showed strong antitumor activity and enhanced T cell proliferation, persistence and in vivo anti-tumor activity compared to traditional CAR T therapies. BPX-401 our CIDeCAR therapy constructed with Bellicum’s novel proprietary dual co-stimulatory domain MC again MyD88/CD40 and in this case the CaspaCIDe safety switch is on track to enter the clinic in the second half of 2016 for the treatment of CD19 expressing cancers. Bellicum presented a palliative preclinical data at ASH 2015 highlighting BPX-401’s potent anti-tumor effects showing that MC co-stimulation increased T cell proliferation at enhanced efficacy in lymphoma and solid tumor models in vivo compared to CAR-T cell that included the more commonly utilized co-stimulatory molecule CD28. The CD19 target at BPX-401 CAR-T therapy was also able to rapidly normalize a dose dependent elevation of cytokines, analogous to cytokine release syndrome upon administration of rimiducid without loss of tumor control. With that, I’ll turn the call over to Annemarie to elaborate on the BPX-501 program and plans going forward.

Thank you, Tom. This is an exciting time for Bellicum and even more so for the patients and physicians involved in the BP-004 study. As Tom mentioned, the interim results for BPX-501 are encouraging and demonstrate its potential to enable critical immune support and improve the outcomes in patients undergoing T-depleted haplo hematopoietic stem cell transplantation. Hematopoietic stem cell transplants are known to be curative and thus the standard of care for many cancers and genetic blood diseases are the challenge for about 70% of patients facing a transplant has always been finding the perfect match. For these patients, physicians will consider a haplo transplant that is a half match typically fulfilled by sibling, parent, child or first degree relative while haplo donors are much easier to identify the possibility of morbidity and mortality associated particularly with Graft versus Host Disease is a real concern. So haplo HSCT transplant is performed only after all other options are exhausted, a chance to mitigate the risk of Graft versus Host Disease typically involved either in vivo or ex vivo depletion of T cells from the Graft, which then leads to other serious challenges like non-engraftment, slow immune reconstitution, infections and relapse. BPX-501 with its CaspaCIDe safety net addresses physicians’ reluctance to perform these potentially risky haplo-transplants. In the BP-004 trial, mature Alpha Beta T cells are removed from the Graft, and in a separate collection of donor T cells, the CaspaCIDe molecular switch is introduced in these T cells and then the BPX-501 product is infused within 30 days after the transplant. Should severe GvHD occur the administration of rimiducid will activate the CaspaCIDe switch to ablate the gene modified donor T cells. In the clinical experience to date, the switches consistently worked is designed to quickly resolve the GvHD. In order to speed immune recovery and infection control and potentially control of relapse Bellicum uses full complement of T cells to be add back of its 501 product; the majority being Alpha Beta T cells. These are mature, powerful, infection fighting T cells, which physicians normally avoid because of their ability to drive GvHD, even though they are important to the therapy's overall success. The initial clinical trial results have shown faster immune recovery and significant reduction in time to hospital discharge among other benefits. As reported at ASH 2015, 21 children in the BTO for study was non-malignant genetic diseases, including Fanconi anemia, beta thalassemia, severe combined immune deficiency or bubble boy disease and Wiskott-Aldrich Syndrome demonstrated disease-free outcomes. Non-malignant patients in the study showed a mean improvement of 40 fewer days to reach a T cell count cells per micro leader and they were discharged from the hospital about 20 days sooner than historical controls and the experience reduced re-hospitalization, primarily due to reduced viral infection. In addition, none of the patients developed chronic GvHD or died of transplantation related complication. We will report the initial data on the patients in the study with blood cancers, which require longer term end points, as well as additional data on the non-malignant patients at the EBMT Congress in early April, as Tom has already mentioned and at the ASH Annual Meeting in December. We're very pleased with the progress of BP-004 study and are now preparing for regulatory meetings, which are planned in the second quarter with both the European medicines agency and the FDA to discuss the past toward regulatory filing and approval, initially for the non-malignant genetic diseases. And with that I’ll turn the call back over to Tom.

Thank you, Annemarie. Let me now speak briefly to a couple of corporate updates. In 2015, committed we build a in-house U.S. manufacturing capabilities, which we believe is strategically important. The company leased an additional 27,000 feet at its current location and we conceded the design phase of project. The facility is designed to support the efficient manufacturing of our novel cellular immunotherapies for clinical trials and early commercial requirements. We expect to complete construction on this facility by the end of 2016. 2015 we also made key highs to complement our senior leadership team and prepare for the progression and expansion of our pipeline of product candidates. In 2016, we expect to establish a European presence and initiate activities in anticipation of the potential commercialization of BPX-501 in future years. At this point, I’ll turn the call over to Alan to review our fourth quarter and full year financials.

Thanks Tom. Before I go through our financial results I'd like to provide an update on our recent debt financing agreement with Hercules Capital, which will be used to support to the build-out of our in-house U.S. manufacturing capability, as we mentioned in today's earnings press release. Under the loan term, we have the ability to borrow up to 30 million, with the final 10 million tranche depended on specified milestones and approval by Hercules Investment Committee. The loan is repayable over a 48 month term, with the initial 18 months or 24 months subject to interest only payment. There were no warrants associated with the financing. And now I will turn to the fourth quarter and full year financial results. Bellicum ended the year on December 31, 2015 with cash and investments totaling $150.4 million, compared to $191.6 million at December 31, 2014. Based on current operating plans, the company expects to end 2016 approximately $80 million to $90 million in cash, cash equivalents and investments and anticipates the current cash resources and capital available under our loan facility will be sufficient to meet operating requirements through 2017. Bellicum also expects to invest approximately $25 million to $30 million for capital projects in 2016 to enable in-house U.S. manufacturing and support the company's growth. Grant revenues were $34,000 and $282,000 for the fourth quarter and year ended December 31, 2015, respectively, and $14,000 and $1.8 million during the comparable period in 2014. The decrease in Grant revenues was primarily due to the June 2014 expiration of the Company’s Grant award from the Cancer Prevention and Research Institute of Texas. Research and development expenses were $10.2 million and $33.6 million for the fourth quarter and year-end December 31, 2015 respectively, compared to $4.2 million and $12.1 million during the comparable period in 2014. The higher expenses in the 2015 period were primarily due to an increase in BPX-501 clinical and manufacturing costs, as a result of increased patient enrolment in our clinical trial, an increase in cost related to our preclinical product candidate BPX-701, BPX-601, and BPX-401 IND enabling activities; and an increase in general, research and development cost, including personnel and allocated overhead. License fees were $3 million and $3.2 million for the fourth quarter and year ended December 31, 2015 respectively, compared to no license fees in 2014. The increase in fees in 2015 was primarily due to a new license agreement with the Agensys, an affiliate of Astellas, as consideration for rights granted to us under the agreement related to our BPX-601 product candidate, whereby we paid Agensys a non-refundable upfront fee of $3 million. General and administrative expenses were $3.8 million and $12.7 million for the fourth quarter and year ended December 31, 2015, respectively, compared to $2 million and $4.3 million during the comparable periods in 2014. The increased G&A expenses in 2015 were primarily due to our overall growth and public company related costs, including an increase in personnel, legal and accounting expenses and costs related to facilities, insurance and travel. Bellicum reported a net loss of $16.8 million for the fourth quarter of 2015 and $48.5 million for the year ended December 31, 2015, compared to a net loss of $74.3 million and $84 million for the comparable periods in 2014. The net loss amounts for the 2014 periods included a charge of $43.2 million incurred in the fourth quarter of 2014 in conjunction with the ARIAD license restructure transaction and a non-cash accounting charge of $24.4 million recorded for the change in fair value of warrants that were exercised in conjunction with Bellicum’s December 2014 initial public offering, of which $23.2 million was a fourth quarter expense. At year-end 2015, Bellicum had approximately 27 million shares of common stock outstanding. Now, I’ll turn the call back over to Tom.

Right, thank you, Alan. Let's open up the call for questions.

[Operator Instructions] Our first question comes from the line of Tony Butler with Guggenheim Securities. Your line is now open.

Yes, thanks very much. Tom, what’s the evidence that supports the notion of greater potency with MyD88/CD40 over the CD28 or 4-1BB, I mean if you have that or if there are some actual data that support that notion, would appreciate that? Thank you.

Sure. Hi, Tony, thanks for the question. If you listen to the rack webcast the other day and I believe it is now online that question was actually asked and Aaron Foster who is in our research group gave a fairly comprehensive answer to it. So, I would refer anyone that is interested in that to maybe look at that. There are also a number of positives and abstracts that are on the website that also, I’m assigned to have addressed that question. So with that said, we have done quite a lot of work now with MyD88 and CD40. It originally started out as ways you control the activation of dendritic cell were CD40 and MyD88 are very relevant because we started to explore a couple of years ago the control of T cell activation, proliferation assistance. We looked at a number of signaling pathways, including again MyD88 and CD40, which turned out to be a very interesting and certainly preclinical in vitro and in vivo studies when we put them head-to-head with CD28 or 4-1BB based constraints, we see a very nice enhancement in general of activity including for example durability of the cell. As you know, we are using it slightly differently in two settings. In one case where it is on a switch, on BPX-601 and in the other case where it is constitutively expressed in BTX-401, but in general some of the properties are consistent across those. So we are excited to take these constraints into human clinical trials to first of all understand safety, but also then [although] you can add to understand the potential to drive greater efficacy whether it’s in CD19 or if it is in solid achievements such as three targeting PSCA. So, I don't know if that’s a helpful answer, if we can expand a little more.

Sorry Tom. That was great. Thank you.

Okay. Welcome.

Appreciate it.

Thanks for the question.

Our next question comes from the line of Biren Amin with Jefferies. Your line is now open.

Yeah, thanks guys for taking my questions. Maybe just on the BP-004 trial, with BPX-501 are you planning on meeting with the Europeans after you’ve enrolled 60 patients, is there like a cut of on patient exposure before you go to the European medicines agency?

Hi Biren. Thanks for the question. The short answer is no. We are planning to meet with the European regulators with the data that we have at the time. We will allow the data to continue to mature in the coming months, but for the regulatory interactions we will go with the data that we have at the time. Annemarie, any further color to add to that?

No, I’ll just - well, I guess yes, the one thing I guess I would say is that these submissions are made to the various committees at very time various times, so in fact each time that you make a next submission the data are updated. So, even if they were cut off one would, you would file after that. So, we've already made a number of these submissions and continue to make updates to those submissions as Tom has said as we continue to enroll the patients.

And when does the company determine whether there is sufficient data to file on your own?

That is a key question for the regulators in the second quarter. That’s probably the number one question. It is how much data and how many patients, how much follow-up is required and are there any additional requirements in terms of the design of the trial that would then guide us as we plan for the next steps with BPX-501.

Got it. And then on BPX-601 you are going into pancreatic patients who are not eligible for surgical reflection, what can we expect or what are companies expectations from this trial are you looking at, I understand probably safety looks your main priority, but are you also looking at response rates and duration of response in these patients?

Thanks, Biren. So, with BPX-601 we have two components to the therapy. First of all we have the genetically modified cells, which contain the IMC switch and secondly we have rimiducid, which is given later to activate the cells and in the first trial we need to understand the safety of both components. So this initial trial and again it was more nuance discussion of this in the rack meeting, we will first give the cells and we will wait two weeks to understand the safety profile of the cells alone. At two weeks, we will then give a single dose of rimiducid to understand how the administration of rimiducid further changes if you like the safety profile. The dose escalation is around the dose of these cells. So for this initial trial we are really looking primarily to understand safety, but of course it is in - essentially a no option patient population with a very interesting target, PSCA where there is, some, if you like clinical principal with an anti-PSCA antibody that might lead us to expect to see some benefit in these patients. So that’s not the primary objective of the trial but again I think I’ll perhaps ask Annemarie if she would like to add any nuance to the question of potential efficacy signal.

So these patients as Tom just said are no option patients and as initially said they are non- resectable. So they would have both an initial math which can be followed but many also will be expected to have smaller metastatic lesions particularly in the liver. So we will be following all lesions of course in terms of signal and change over time potentially and as well as we’ll be looking at some biomarkers which also are being evaluated in the pancreatic setting as disease indicators. So as Tom said, the primary outcomes are safety, there will only be one dose of rimiducid given in the trial to evaluate both safety but also to evaluate the impact on the duration of the cells. After the study, we would then depending on the data we would expect to really then begin employing rimiducid on a continual basis as needed. So this study for us really we think will have multiple potential outcomes both in terms of the therapy itself, the safety of the combined products that is the cells and the rimiducid, and then some potentially track able outcomes in terms of looking at some of the metastatic signal there in these patients.

Okay, great. Thank you.

Thanks, Biren.

Our next question comes from the line of Robyn Karnauskas with Citi. Your line is now open.

Great, thanks. This is Mohit calling on behalf of Robyn. I have one question regarding the 501 data. So when we did some dark cost, they want to get comfortable around the relapse rates in the transplant setting and they worry that they might go up. Given that you think the relapse rates could go down with 501, how much follow-up do you think will be enough to make those doctors comfortable? Thank you.

Thanks, Mohit. Great question, I’m going to pass that one straight to Annemarie.

Hi, Mohit. Thanks, Tom. Currently we will be presenting some of the one year follow-up data at EBMT as we’ve already mentioned. It would be standard from an outcomes end point to look at least at two year follow-up data to look at the impact of certainly the initial impact on overall survival and disease-free survival and composite survival in terms of GvHD disease-free survival. Ultimately, some doctors are going to be very comfortable with that especially in a setting where these patients have no other options but to have a transplant like this a haplo. Other doctors would require a five year follow-up, but certainly that two year signal is an important point for us. And therefore also why we see the endpoints for the non-malignant and the malignant diseases being different is nothing else in terms of the duration to get those significant endpoints. So I would say initial comfort at two years and final definitive at the five years once from a post of market type setting.

Helpful. Thank you.

Thank you.

[Operator Instructions] At this time, I’m showing no further questions. I'd now like to turn the conference over to Tom Farrell, CEO, for further remarks.

Thank you very much. Thank you again for participating on today's call. Before we wrap up, I’d like to acknowledge our employees for their continued hard work and dedication and our shareholders for their interest and their support. We look forward to continuing the important work at discovering, developing novel cellular immunotherapies for cancers and orphan inherited blood disorders and improving patients life. Thank you very much and have a great evening.

Ladies and gentlemen, thank you for your participation in today's conference. This does conclude the program. You may now disconnect. Everyone have a great day.