Greetings, and welcome to the Bellicum Pharmaceuticals Fourth Quarter 2020 Financial Results and Corporate Update Conference Call. . I will now turn the conference over to our host, Robert Uhl of Westwicke ICR. Thank you. You may begin.

Thank you. Good afternoon, everyone, and thank you for joining the call. With me today on the call is Rick Fair, Bellicum's President and Chief Executive Officer. Earlier this afternoon, Bellicum released financial results for the fourth quarter and full year ended December 31, 2020. 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, 2020, and 10-Q for the quarter ended September 30, 2020, filed with the SEC and available on their website.

Thanks, Robert. Good afternoon, everyone, and thanks for joining us. On our call today, I'll provide an overview of our recent progress and update on our GoCAR platform and programs and a financial summary of the fourth quarter and full year of 2020. Our clinical progress in the fourth quarter was highlighted by the enrollment in apheresis of our first patient in our Phase I/II trial evaluating BPX-603 in patients with HER2-positive solid tumors, including breast, endometrial, ovarian, gastric and colorectal cancers. BPX-603 is our first dual-switch GoCAR-T product candidate to enter clinical development. In addition to our clinical progress in the fourth quarter, we refined our focus on our next-generation CAR-T cell therapies, which allowed us to take steps to reduce our cost structure. In October, we implemented a restructuring plan that included a reduction of our headcount, pause of our BCMA GoCAR-NK preclinical program, discontinuation of discovery research and sale of our research facility. We also strengthened our balance sheet by raising capital via an underwritten offering of common stock and warrants, and repaying our outstanding debt. While some of these changes were difficult, we expect they will result in significant cost savings going forward, enabling us to focus our resources on our GoCAR clinical programs. Before I go into the specifics of our programs, let me provide a brief reminder of our GoCAR platform, which is differentiated in 2 ways. First, we've engineered GoCAR in an effort to deliver more potent and durable efficacy. We intend to accomplish this primarily through our co-activation domain, MyD88/CD40, or MC. We believe MC signaling can boost effector cell proliferation and survival, enhance functional persistence by resisting exhaustion in the suppressive tumor microenvironment and stimulate the cancer patients' own immune system to eliminate cancer. Second, we've engineered GoCAR for higher performance, offering the potential for superior control via our molecular switch technology. Other CAR therapies behave unpredictably due to their autonomous activity. The GoCAR antitumor effects can be controlled by the scheduled administration of rimiducid. GoCAR activity can be dialed up or down by adjusting the interval between rimiducid doses or suspending rimiducid administration. Our dual switch product candidates are designed for further improved controllability by incorporating our CaspaCIDe safety switch, which can rapidly eliminate our cells when triggered to manage acute toxicities if they occur.

. Our first question comes from Wangzhi Li with Ladenburg.

Just a very quick one. So could you provide any further color on what kind of data should we expect when you report the data for 603 in the second half this year and the 601 in the first quarter next year?

Sure, Wangzhi. Thanks for the question. On BPX-603, I would expect the first dose level presented and perhaps a patient or two from the second dose level. I'll remind you that the design of that trial requires sequential enrollment through dose escalation. And so it does take a bit of time for us to accrue that study. So I'd expect safety data from the first dose cohort and potentially some patients from the second cohort. As it relates to the BPX-601 readout, obviously, we're at a higher dose level there already. We're treating patients with 5 million cells per kilogram, which is a reasonable cell dose. So certainly, in dose escalation, we still are evaluating safety, but we'd also expect a fulsome analysis of efficacy and biomarker endpoints to demonstrate what we're seeing looking for a signal in metastatic CRPC. I'd expect 2 to 3 cohorts of patients there, but of course, that will be dependent upon enrollment.

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This is Robert Uhl. I've had a couple of questions come in to me over e-mail from people on the webcast. So let me just fire away with those.

Sure, Robert.

Rick, yes, "Can you provide any additional color on the patients that died that resulted in the clinical hold? And what helped the FDA become comfortable so that they would let you restart the trial without any kind of change to the protocol?"

Sure. The patient who passed in the study was an elderly second line pancreatic cancer patient who received BPX-601 followed by 2 weekly doses of rimiducid. The patient developed a grade 4 cytokine release syndrome and was undergoing treatment with standard treatments. However, in parallel, the patient was diagnosed with aspiration pneumonia and sepsis syndrome, which was deemed unrelated to our treatments, of course. While these complications were ongoing, his family withdrew medical care and requested comfort care only, and the patient died subsequently later that day. The cause of death determined by both the investigator and Bellicum was aspiration pneumonia with sepsis syndrome, and that was supported by the review of the Safety Review Committee. That said, based on the temporal relationship of death just a couple of days after the second dose of rimiducid, the FDA put the trial on hold and asked for some additional data about the patient case and about the safety that had been observed in the trial to date. Once they reviewed the information we provided, they removed the clinical hold without any required modifications to the clinical protocol. So while they don't clarify how they interpreted the data that we submitted, I think it's reasonable to assume that their analysis was the same as ours that this was an unfortunate case of a very sick elderly pancreatic cancer patient who died of complications.

Okay. Thank you. And then one other one question that's come in is about the case report of the use of the CaspaCIDe technology. "You mentioned it's in a number of collaborations. So can you just tell us about those? And what kind of economics it represents for you? And does it report like this help bolster your BD effort to get that out there into more people's hands and into their programs?"

Sure. Thanks for the question, Robert. We remain very excited about the safety switch. We think it's a useful addition to really any cell therapy. And the performance observed, as I reported in both the previous experiences reported at ASH a couple of years back, in this case report suggests it's really a best-in-class solution for acute toxicities of cell therapies. We're open to partnering this technology. I would say, to date, most of the collaborations that we have are academic collaborations with investigators who are pursuing new cell therapy constructs or new targets that may have a particular safety liability and what the additional comfort of having the safety switch embedded. But of course, some of those then translate into more meaningful collaborations for us. For example, we previously announced our agreement with MD Anderson for their CaspaCIDe containing a CD19 CAR-NK candidate, which we share in the economics they receive for the project from their licensee, Takeda. That project continues to advance, and we remain open to pursuing deals of this nature opportunistically when they make financial sense and when they don't compete with our pipeline.

Okay. Terrific. Thank you.

Thank you. And there appears to be no more questions from our audience. I will now turn the call back to Mr. Fair for closing remarks. Thank you.

Thanks, everyone, for participating today. If you have additional questions, feel free to contact us any time. In closing, I'd like to thank our great team of Bellicians, our collaborators and our investigators for their efforts in these extraordinary times. And as always and most importantly, I'd like to thank the patients and the families who participate in our clinical trials. They inspire our effort each and every day. Thanks, and have a great evening.

Thank you. This concludes today's call. All parties may disconnect. Have a great evening.