Ladies and gentlemen, thank you for standing by, and welcome to the Atara Biotherapeutics conference call. At this time, all participant lines are in listen-only mode, so if you require operator assistance, please press star then zero. After the speaker's presentation, there will be a question-and-answer session. To ask a question during the session, you will need to press star then one. I'd now like to hand the conference over to your host today, Mr. Eric Hyllengren, Vice President, Investor Relations and Finance. Please go ahead, sir. Thank you, operator. Good morning, everyone, and welcome to the Atara Biotherapeutics conference call. On today's call, members from the Atara executive team will provide an update on our tabelecleucel regulatory progress. This morning, we issued a press release providing a regulatory update for tabelecleucel in EBV-positive PTLD. This press release is available in the investor and media section at atarabio.com. Joining me on today's call are Dr. Pascal Touchon, President and Chief Executive Officer, Dr. Jakob Dupont, Executive Vice President and Global Head of Research and Development, Dr. Manher Joshi, Chief Medical Officer, Joseph Newell, Chief Operations Officer, and Utpal Koppikar, Chief Financial Officer. We will begin with prepared comments from Pascal, then open up the call for your questions. We would like to remind listeners that during the call, the company's management will be making forward-looking statements. Actual results could differ materially from those stated or implied by our forward-looking statements due to risks and uncertainties associated with the company's business. These forward-looking statements are qualified in their entirety by the cautionary statements contained in today's press release and the company's SEC filings. These statements are made as of today's date. The company undertakes no obligation to update these statements. Now I'd like to turn the call over to Pascal. Pascal? Thank you, Eric, and thank you all for joining us this morning. We have made excellent progress during the fourth quarter to prepare for tab- cel regulatory submissions and potential approval and launch. The progress is on the heels of the interim analysis data we reported on the Q3 conference call for the tab- cel pivotal 302 study, or ALLELE. Recall, this interim analysis, which compiles subjects with six months duration of response follow-up at the time, achieved a 50% objective response rate by independent oncologic and radiographic review. This objective response rate and the safety profile for tab- cel in ALLELE are consistent with previously published investigator-assessed data from historical non-pivotal studies with no new safety signals. In addition, as part of a Breakthrough Therapy designation for tabelecleucel, we've had constructive and regular dialogue with the FDA over the last several months, and recall in particular that we reached agreement with FDA in October on several key aspects of the tab-cel regulatory package. Notably, that we can initiate a rolling BLA, and that we can complete the BLA submission with data from the ALLELE study currently in all patients with at least six months of follow-up for durability of response. The pivotal ALLELE study data will be the primary basis for approval. While the FDA confirmed that we can use efficacy and safety data from the historical non-pivotal studies as supportive data in the BLA filing. Based on these agreements, we expected to initiate the rolling BLA by the end of 2020 and finalize the BLA submission in Q3 2021 with the clinical module. In line with this expectation, we have been ready to initiate the BLA for tab- cel since early December, when we finalized the initial preclinical module for the BLA, for which the content had been agreed with the FDA. During our regular dialogue with the FDA, we received feedback that before the rolling BLA can be initiated, there is a procedural decision that the agency must take regarding how the clinical data from the historical non-pivotal studies are submitted in a BLA filing. Specifically, this procedural decision relates to whether we submit clinical data from the historical non-pivotal study as parallel analysis or as pooled analysis with the pivotal ALLELE study data. An FDA determination on how we provide the clinical data analysis from the tabelecleucel historical non-pivotal studies will be based on the assessment of the comparability of the drug product made at Memorial Sloan Kettering and used in the historical non-pivotal studies and the drug product manufactured by Atara and used in the ALLELE pivotal study. To assist the FDA in considering this specific question, we provided the agency with a comprehensive data package in August to demonstrate comparability between these drug products, and then complemented this with supplemental data in Q4 per their request. Based on our ongoing conversation with FDA, we had the understanding that this procedural decision will be made before the end of 2020, so that we could initiate the BLA by then. Unexpectedly, FDA decision on comparability between the MSKCC and the Atara-manufactured drug product has not yet been made. However, we continue to have constructive and ongoing dialogue with the FDA and expect such a procedural decision soon. We will then promptly initiate a rolling BLA since this decision has no impact on the initial preclinical module, which I noted is completed and ready to be submitted. Of utmost importance is that this pending FDA procedural decision has no impact on the critical path to completion for the BLA, and we are on track to do so in Q3 2021. Recall that this critical path is defined by data from six months' clinical duration of response on additional patients enrolled following the interim analysis. This data will mature in Q2 2021, will then be analyzed per the statistical analysis plan submitted to the FDA, and included as part of the final clinical module to complete the BLA submission in Q3 2021. We also expect to present this data in an appropriate congress in the later part of 2021. While this delay was unexpected, we are ready to initiate the rolling BLA submission after we receive clearance from the FDA. We remain on track to complete the submission in Q3 2021, and we are committed to progressing the regulatory package for tab- cel so that we can move closer to delivering this transformative therapy to those patients in need. I'll now turn back the call to the operator to begin the Q&A portion of the call. Operator? Our first question comes from the line of Salim Syed with Mizuho Securities. Great. Thanks so much for the question, guys, and Happy New Year. Couple from me, if I can. I'm just curious, just at the timing of this seems like something that the FDA could have made a decision on prior, and I'm just wondering why now they're bringing this up to you guys. Is there something specific regarding the data, or if there's a broader change at the FDA in the gene and cell therapy landscape that they're looking at? The second question here, if you do have to run a parallel analysis here in your submission, I know they've agreed that you can submit the data based on the pivotal, but is there any risk here at all of them changing their current stance and saying that you would actually require additional patients in the pivotal? Thank you. Thank you, Salim, and happy New Year to you as well. Jakob, do you want to start to answer the first question? Yes, certainly. Thank you, Syed, for the question. Regarding the topic of timing, sorry, Pascal, I lost the question. Can that be repeated, please? I think the question was about the timing. I think as we said, we have submitting all the package for comparability in August. We have had several interactions since then. They've asked some additional supplemental data we provided them with, and we were expecting till, I think, the last minute to have this particular decision being made by the FDA. We see that as mainly a timing issue there in terms of the decision. We believe that we provided them a comprehensive package to support their decision on the comparability. That's really where we are today there. That's why we say we have such a constructive dialogue with the FDA that we expect that decision to be taken, hopefully soon. On your second question, which was around, possibly linked with other FDA positions regarding cell and gene, here we believe that there is a very different situation. It has nothing to do with an assay or some kind of inconsistent data between some historical data and pivotal data in the IA. All this is consistent. It is really that they want to clarify whether this analysis of the data, which they very clearly say, these historical non-pivotal studies data will be considered as supportive data, whether or not they decide that the two direct products are comparable. They want to decide whether then this data should be submitted as a pooled analysis or a parallel analysis. Jakob or AJ, do you want to answer on whether we see any risk to the aspect of the approval, knowing that, as we say, the pivotal study, the ALLELE study, the FDA has been very clear that this is the primary study for the approval. This question on the aspect related to the analysis is, in fact, just a procedural question that they need to decide upon. Yeah, that's correct, Pascal, and maybe AJ can comment as well. We have received very clear feedback that the ALLELE study, the Study 302, will be the primary data source for the BLA and that the approval will be based upon the data from the 302 study. However, as you note as well, non-pivotal data from the historical trials will be reviewed by the FDA. We have a very clear agreement based upon that October interaction with the FDA in terms of the number of patients that are already enrolled in the ALLELE study, and we just need six months duration of follow-up for responders there. That data cut is coming up here in Q2 of this year. We feel very confident that we do not need to bring new additional patients into that data package from the ALLELE study. AJ, anything further to add? Nope. I think that's covered well. Thank you. Great. Thanks so much, guys. Our next question comes from Jonathan Miller with Evercore ISI. Your line is now open. Hey, guys. Happy New Year, and thanks for the update on this. My question is on the material itself. How different is the MSK clinical material and your internally produced materials? Is there anything that you've been able to see in that package you submitted to the FDA that would indicate that there's some meaningful difference between those two products? Thank you, Jon, for your question, and Happy New Year to you as well. We clearly believe that these two products have demonstrated comparability. Maybe, Joshi, you want to give a little bit more color to that about why do we believe that we have demonstrated comparability even though the process was slightly different, but the products, we believe, are comparable. Glad to, Pascal. We took a very comprehensive approach to our comparability study. For our protocol, we established prospective acceptance criteria that had to be met in order for us to conclude we had achieved comparability. We evaluated 15 product lots from each of the manufacturing process versions. Each of those lots were tested for nine different product attributes to assess comparability. Once testing was confirmed and results completed, we had passed each of the predefined acceptance criteria and a robust statistical analysis. Together, those two statements conclude that we strongly believe we've got a comparable product and strong process capability for the commercial product that we're making for inventory for commercial approval. From that prospective, Jon, I think we're in a pretty good position. Does it answer your question, Jon? Yeah. I suppose, given that your internal bars have been met, I suppose my real question is, what's the upshot if the FDA decides one way or the other? The FDA is not going to come out of left field here and say, "Actually, we think there is a meaningful difference," and that has an impact. I think your answer to Salim's question suggests that you think, regardless, you're going to be able to file on time, and it's not going to impact the submission process. Is that right? I think it's really a procedural question at the end of the day, Jon, here, because the FDA confirmed to us that whether they believe themselves or they estimate themselves that this is comparable or this is not comparable, will not change the fact that the data from historical non-pivotal studies will be considered as supportive data. They were very clear about that. It's just a type of analysis that they want to perform, they want us to perform in the final clinical module that is linked with that decision. If it's comparable, then you can pool the data together. If it's not comparable, we just use that as separate data. I had that experience in the past, and the FDA had that experience with other type of product there. It's not that surprising that they want to clarify those questions before confirming the type of analysis in the clinical module that they want us to file with. Okay. Makes perfect sense. Thanks, guys. As a reminder, ladies and gentlemen, that is star, then one, if you'd like to ask a question at this time. Our next question comes from Benjamin Burnett with Stifel. Your line is now open. Great. Thank you. Happy New Year to you guys as well. You touched on this during Salim's question. I just wanted to better understand sort of what the spectrum of outcomes are here. I guess in a scenario where the FDA could potentially conclude that this isn't comparable, I guess you would still use this in the data submission, but if I understood you correctly, you can't pool the data in the analysis. Does that change the hurdle or your ability to prove efficacy here at all? No. Thank you, Ben, for your question, and Happy New Year to you as well. No, it doesn't change the hurdle at all. Again, the pivotal study stands alone in itself as the primary analysis for the efficacy and safety of the product. It's just that there have been so much data and great data that have been presented in the past, both from memorial and also from the compassionate use, as well as the expanded access program that we performed and presented at ASH last year, that it makes sense for the FDA to want to have a look to this data as well. Type of data set that exists for this product. The question is really about this particular analysis. To me, it's very similar to when Novartis got KYMRIAH approved in its first indication. They had supportive data coming from UPenn, but these data were not pooled into the analysis. Each data set was analyzed separately that. We believe ourselves that here in that case, we have demonstrated comparability between the two direct products, but it's up, as usual, to the FDA to decide whether they agree with us. Again, whether they agree or don't agree with us on this particular aspect of comparability between the product made, manufactured at MSK, and the product made at Atara, it doesn't really matter in terms of the analysis of the pivotal study itself. Jakob or AJ, do you want to add anything to that on the analysis of the pivotal study? Pascal, I think that's absolutely right. I do think that we believe that we have a very strong data package in both scenarios, whether the data are presented in parallel or pooled, we believe that the data package supports the label of tab-cel for EBV-positive PTLD, who have received at least one prior therapy. We do believe that, however the agency decides upon this procedural step, that we're in a favorable position for the submission and the approval of tab-cel. Okay. That's great. I appreciate it. One more point of emphasis. AJ? I was just going to add one more point of emphasis. Sorry for interrupting there. We've talked a little bit about this notion that the pivotal study stands alone. This isn't just us stating that. This was reinforced by FDA during our ongoing discussions with them. At our last Type B clinical meeting, they reinforced very specifically that the [audio distortion] stands alone, period, and then that the historical non-pivotal data are supportive. When you think about or when you ask about is there a risk to the filing and a risk to the approval based on this comparability piece, 302 stands alone. We've talked about already the ORR that is achieved at the interim analysis. All of that put together gives us good confidence that there's really no impact on the overall view of the filing. It's really more just how the data will appear long-term in the label and other discussions. Okay. That's very helpful. Thank you. Our next question comes from Phil Nadeau with TD Cowen. Good morning. Happy New Year, and thanks for the update also. Just a couple procedural questions. Why not just cut the data both ways and leave it up to the FDA during the course of the review to decide whether the products are comparable and how the data should be represented on the label? Is that an option that you could follow should the FDA not make a decision anytime soon? Thank you. Happy New Year to you as well, Phil. Jakob, do you want to answer that question? Yes, absolutely. This is actually something that we also propose to the agency as they deliberate on this procedural issue of comparability. The agency really wants to provide clear feedback to us in terms of how the data should be presented in the filing. While we made that proposal, the agency really wants to see the data in one particular format. That's what we're waiting for with that procedural decision. Great. Then I guess the second question, if I heard you correctly in the prepared remarks, you noted that this decision doesn't have any impact on how the preclinical data are presented, and that module's ready to be submitted. Why not start the BLA submission with that module and then submit the clinical data, once this decision's made? Why does the initiation of the BLA have to await this decision if other parts of the other modules aren't affected? Thank you for your question. Jakob? Yeah, absolutely. The great news is that module is ready for submission, and that's been pre-reviewed in discussions with the FDA previously. From a procedural prospective, the agency really wants to give clear feedback to us and a resolution to all issues and procedural aspects before we initiate. This really is the FDA's preference to resolve this particular issue of the submission of the clinical data before we initiate the filing, and we're certainly adhering to that guidance from the agency. Perfect. Thanks for taking my questions. Thank you, Phil. Our next question comes from Maury Raycroft with Jefferies. Your line is now open. Hi, good morning, everyone, and Happy New Year. Thanks for taking my question. You said you've been in an ongoing dialogue with the FDA. Just wondering if you could provide more specifics on who you're speaking with at the FDA and who you're waiting to hear from. Are you interacting with the same people who you interacted with prior to the start of the phase IIIs when comparability was a key question, or key discussion point back in 2016 and 2017? Yeah, thank you. We are interacting with CBER, of course, there, and we have a clinical reviewer and a CMC reviewer and, of course, a team. Nothing very specific there. This is a team that has been following the tabelecleucel discussion for some time now. I don't know, Jakob, whether you recall whether back in, well, neither Jakob nor myself were there, but I believe that it's been a similar team or same team since the beginning there. Jakob? Any, or maybe AJ, any comment on that? Yeah. AJ, do you want to come in? Yes. As far as I'm aware, I've been with the company for a little over 4 years now, and this is the same group, at least in terms of a couple of leaders that Pascal mentioned. It's the same group, including on the CMC side, that's been with the program for years. Got it. Okay. Thanks for taking my question. The dialogue has been very constructive, and we are pleased with the way things are moving in terms of the discussion there. Again, the clarity that we had back in September and October is really what made us very confident together with the interim analysis showing this 50% objective response rate and no new safety signal. It's really what made us confident that we are moving forward, and we can really expect to complete that submission in Q3 2021 once we have the follow-up that is required for these additional patients. Got it. Thank you very much. Our next question comes from Anupam Rama with JP Morgan. Your line is now open. Hi, guys. Hi, this is Tessa on the call for Anupam this morning. Happy New Year from us. Just one quickly. In your view, what are the advantages to a pooled analysis versus separately kind of presenting a parallel analysis? Maybe you could just walk that through for us. Thanks so much. Yeah. No, thank you, Tessa, for your question. Clearly, as we said, we believe that these two products, these two drug products, have demonstrated comparability between the one manufactured by MSK used in this historical study and the one manufactured by Atara and used in the pivotal study, and by the way, that will then be used in the commercial phase there. We believe that they have demonstrated comparability, and we believe it may be advantageous to have all data be viewed as a pool analysis to have a more extensive dataset in the label there. It's, again, the studies, the pivotal studies stand alone, adding additional data, especially in terms of duration of response. As you know, we have had data with two years of overall survival in responders over 80%. These are data that have been published, they are out there already. We believe it could be good to have that, to have a more extensive data set. AJ or Jakob, anything to add? Yeah, go ahead, AJ. I think, Pascal Touchon, I think you covered it well. At the end of the day, it's like many other things. It's really just going to be a question of how the data appear in the label. To your question of does pooled versus parallel help you? You heard Pascal Touchon talk about how Novartis had essentially parallel data, right? You can have a very successful approval, launch, et cetera, with parallel. The pooled may give, the more data that they let you put together, in general, it makes it easier once you're out in the commercial setting, potentially. Really, these are small things. At the end of the day, the key win for us, and I just can't reinforce this more, is that FDA clearly stated that the historical non-pivotal data are supportive, which from our prospective, that means they're going to make their way into the label. It's just a question of what the overall format looks like. We don't believe that there's significant differences here, and certainly no impact on approvability or hurdles or bars for approval. Great. That's very helpful. Thanks for taking our question. Yeah, I think we believe we can be successful, really, with both. We just need the FDA to decide. Our next question comes from the line of Tony Butler with Roth Capital. Your line is now open. Yes, good morning. Happy New Year. Pascal, two questions, please. One is, I'm very respectful of the products made by MSK and by Atara. The question is, are any questions actually going back to MSK that they need to address? That's point one. Number two is, both you and I think AJ made reference to the KYMRIAH label. I don't recall, but was that UPenn data actually put in their label, even though it was separated from a pooled data set? Thank you. Thank you, and a happy new year to you as well, Tony. There is no need for MSK to do anything there in terms of answering questions. We have all the data we need to answer the questions. This comparability study that Joe was mentioning, very comprehensive, very robust analysis we did, has been done by Atara. We have all what we need, and we can answer questions from the FDA based on Atara available data there. That's one important aspect. Now, the second aspect of your question, what happened in this particular case of KYMRIAH and Novartis, that the data from UPenn were not part of the label, even though they were used as supportive data. You may remember they were part of the ODAC and the submission there. They certainly play a role there, but they were not part of the label. Perfect. Thank you so very much. Our next question comes from Yigal Nochomovitz with Citigroup. Your line is now open. Hi. Great. Thank you very much for taking the questions, and Happy New Year to everyone. Just one question, following up on the prior questions. Is there any advantage for the pooled versus the parallel analysis when it comes to commenting on the statistical power for the study? Or is that really not a relevant consideration? Thank you, Yigal. I'm giving over to you. No, it's not relevant. Jakob, AJ, do you want to give more flavor there? Again, the statistical analysis has been set up for the pivotal study, and we have the right number of patients there and the right duration of follow-up to come. AJ, Jakob, anything to add? Yeah, absolutely. I think that's absolutely right. As we've articulated, the ALLELE study really is the centerpiece of the BLA filing, and that's what the statistical analysis is based around. The great news is, as we achieved in October of this year, was the agreement with the agency that they will review the historical non-pivotal data as well, either in a pooled or in a parallel advantage. That data will contribute to the agency's overall assessment of efficacy and safety for tab-cel and EBV-positive PTLD. We think that we will get the benefit of that historical data, even if it's in the parallel analysis. Again, the statistical analysis plan really is focused upon the ALLELE study, the pivotal trial. Got it. Thanks. Then just one procedural question. Could you review how many modules are in the BLA and the cadence of when each of these modules is expected to be submitted in order to conclude the submission by 3Q 2021 as guided? Yeah. We're going to start with the preclinical module and end with the clinical module. In between, we'll have the CMC module. Jakob or AJ, do you want to give more details there? I can give a little detail, and AJ can follow. As mentioned, we'll start with that Module 4, which is the preclinical data. The expectation will be in the rolling BLA that we will next submit the Module 3, which will be the CMC module. The final piece will be the Module 5, the clinical data that will be submitted here in Q3 of 2021. Again, importantly, our guidance has not changed, that we're still on track to complete the BLA filing for tab-cel here by Q3, and that will be the final piece of the rolling BLA submission based upon this data cut of the ALLELE data that will occur here in Q2, which will give us then the six months follow-up on the additional patients that have already been enrolled on the study. AJ, anything further to add regarding the BLA? No, Jakob, I think that covers it. Thanks. Yeah. We have a planned schedule, Yigal, that is very clear, and that's why we are confident about our expectation to complete that BLA in Q3 2021 based on this additional follow-up that we're going to have on these patients that have been enrolled after the IA was done in Q3. Great. Thank you very much, Pascal and team. That concludes today's question and answer session. Ladies and gentlemen, this concludes today's conference call. Thank you for participating. You may now disconnect.