This conference call transcript was computer generated and almost certianly contains errors. This transcript is provided for information purposes only.EarningsCall, LLC makes no representation about the accuracy of the aforementioned transcript, and you are cautioned not to place undue reliance on the information provided by the transcript.
spk13: Thank you, Brandy, and good morning, everyone. Welcome to today's call, during which we will provide an update on the company and review our financial results for the full year of 2020. Earlier this morning, we issued a press release summarizing our financial results and progress across the company, which is available on our website at www.communicell.com. Here with me on our call today are Julian Adams, Chief Executive Officer, Ronit Simantov, Chief Medical Officer, and Shai Lankri, Chief Financial Officer. Michelle Corfin, our Chief Operating Officer and Chief Commercial Officer, and Tracy Lodi, our Chief Scientific Officer, are also on hand for the Q&A portion of the call following our prepared remarks. During this call, we may make forward-looking statements about our future expectations and plans, including clinical development and commercial objectives, the therapeutic potential of our product candidates, our operational plans and strategies, and projected operating expenses and cash runway. Our actual results may differ materially from what we project today due to a number of important factors, including the considerations described in the risk factor section of our Form 20F and in other filings that Gameta Cell makes with the SEC from time to time. These forward-looking statements represent our views only as of today, and we caution you that we may not update them in the future, whether as a result of new information, future events, or otherwise. Now I'd like to turn the call over to Julian.
spk07: Thank you, Josh, and thanks to everyone for joining us this morning. At GametaCell, we are at the forefront of developing and commercializing potentially curative medicines for patients by harnessing our proprietary NAM cell expansion technology. This platform provides us with an opportunity to create therapies that could redefine standards of care for patients with life-threatening diseases. 2020 was an important year for Gametacel as we made significant progress across our entire pipeline, including Omidubicel, which is poised to become the first FDA-approved cell therapy for bone marrow transplantation, and GDA201, and innate natural killer cell or NK cell therapy. Today, we'll review both programs and summarize our progress around plans to bring Omidubacill to patients in the commercial setting pending FDA review. Starting with our lead program, Omidubacill has completed a pivotal randomized phase three study. This global trial evaluated Omidubacill versus standard cord blood in patients with hematologic malignancies who needed a bone marrow transplant but did not have a suitable matched donor. The primary endpoint of the study was time to neutrophil engraftment, or the time it took for the white blood cells to recover following transplantation. In 2020, we reported the Omiduba cell phase three data, including primary and secondary endpoints, demonstrating its clinical benefit and made important advances to be launch ready upon FDA approval. Following our December Type B meeting with FDA, we now have a very clear understanding of the Omidubicel commercial manufacturing requirements. We are on track to fulfill these requirements to submit the full BLA by the end of 2021 and meet the commercial supply needs. As we continue to advance Omidubicel for a potential launch, and prepare to become a commercial organization, we are establishing key commercial capabilities, including the creation of Gameta Cell Assist, a program designed to support a positive patient and transplant center experience. With over 13,000 patients with hematologic malignancies eligible for transplant annually, and approximately 200 transplant centers in the U.S., we plan to focus on patient access and support of every individual and their caregivers at each step of the process. In addition, we have conducted extensive market research and have been encouraged by the feedback from physicians and payers indicating the opportunity for Omidubacil to improve safety and efficacy outcomes. Following FDA approval, the market research supports that Omidubacil will be an important therapy for all patients in need of an allogeneic stem cell transplant, who do not have access to an appropriate match-related donor. Omidubicel also has the opportunity to increase access for patients who are not currently able to find a match. Moving to the rest of our pipeline, we are also pleased to report significant progress for GDA201, our first NK cell-based product candidate. Natural killer cells are innate immune cells that hold tremendous promise for treating cancer, A challenge in the field includes the ability to expand NK cells in culture while preserving their functionality. Our NAM-based technology addresses this challenge and potentially improves their direct tumor-killing potential and antibody-dependent cellular cytotoxicity, known as ADCC. GDA201 has demonstrated impressive proof of concept in our Phase I study. which was designed to assess the safety of GDA201 in combination with a monoclonal antibody in non-Hodgkin lymphoma or multiple myeloma. The data from our ongoing phase one study has demonstrated striking early signs of efficacy with multiple durable complete responses while being very well tolerated in patients with advanced lymphoma. We have now developed a cryopreserved formulation in support of our vision to support a multicentered, off-the-shelf allogeneic cell therapy with durable, deep responses and a favorable safety profile. Furthermore, we are leveraging the NAM expansion platform to develop a pipeline of gene-edited NK cell product candidates with enhanced function for both hematologic malignancies and solid tumors. Based on the impressive clinical data generated by both Omidubacil and GDA201, we have significantly strengthened our financial position by raising approximately $220 million in the past year. These proceeds will support the development of both programs and provide sufficient capital through the potential Omidubicel product approval and launch by mid-2020. I want to conclude my introductory remarks by acknowledging the challenges of the past year as we navigated through the COVID-19 pandemic, and the resilience and dedication of the Gametacel team as we work to bring cures to patients. I'll now turn the call over to Ronit Simontov, our Chief Medical Officer, to provide further details on Omadubacil and GDA201. Ronit?
spk11: Thank you, Julian, and good morning, everyone. This morning, I'll review our clinical program, and highlight the data we presented throughout 2020 on both Omidubacil and GDA201. I'll start with the Phase III study of Omidubacil. In May of 2020, we were pleased to report that the study met its primary endpoint, time to neutrophil engraftment. In October, we reported that the Phase III study also met all three pre-specified secondary endpoints, platelet engraftment, infections, and hospitalization. These positive study outcomes, as well as additional exploratory endpoints, were presented in a peer-reviewed setting for the first time at the TCT meeting a few weeks ago. And there will be an encore presentation at the presidential session of the European bone marrow transplant annual meeting next week. The trial was a well-conducted and rigorously designed study, analyzed on an intent-to-treat basis. 125 patients were randomized, 52 to the omodubo cell arm and 63 to standard cord blood. Demographics and baseline disease characteristics were well-balanced and reflected a diverse population of patients with hematologic malignancies in need of allogeneic bone marrow transplants. Clinical outcomes supported the superior neutrophil engraftment demonstrated in the primary endpoint analysis. Specifically, patients randomized to omadubicel had fewer serious bacterial, fungal, and viral infections than the comparative group. Patients also spent significantly less time in the hospital in the first 100 days after transplant. Importantly, omodubacill was generally well tolerated, and in particular, the incidence of both acute and chronic graft-versus-host disease, which has been shown to be lower in patients transplanted with cord blood than in other modalities, was statistically similar in the two arms. While the study was not designed to detect a difference in relapse or mortality endpoints, We reported the results of these analyses, including non-relapse or transplant-related mortality, which was 11% for patients randomized to omodubacil and 24% for patients randomized to comparator, and overall survival, which was 73% in the omodubacil arm and 52% for control. The data we presented in 2020 continue to strengthen our confidence in the clinical potential of omodubacil. I want to thank the investigators, patients, and caregivers that participated in our study. We are grateful for their support as we move the field forward. With these data in hand, we anticipate submitting the BLA for omadubacil in the fourth quarter of this year. We believe that omadubacil also has potential to treat patients with diseases beyond hematologic malignancies and are currently investigating omadubacil in patients with severe aplastic anemia, a rare, life-threatening blood disorder. An investigator sponsored Phase 1-2 study being led by Dr. Richard Childs at the National Institutes of Health. At the recent American Society of Hematology Conference, we presented data from a total of eight patients, three who underwent stem cell transplant with omadubicel plus haploidentical stem cells, and five patients who received omadubicel as a standalone graft. The data showed that transplantation with omadubicel following reduced intensity conditioning was generally well tolerated and led to rapid engraftment. With a median follow-up of 10 months, seven of the eight patients had early and sustained engraftment and were no longer dependent on transfusion. Neutrophil and platelet recovery occurred at a median of 10 days and 31 days respectively. We are encouraged by these data in patients who are at high risk for graft failure with conventional cord blood transplants. The study is still ongoing. As Julian mentioned, in addition to Omadubacil, we are advancing our first MK cell therapy, GDA201, We have demonstrated impressive proof of concept in our phase one study, which is designed to assess the safety of GDA201 in combination with a monoclonal antibody in patients with non-Hodgkin lymphoma or multiple myeloma. The study is being conducted by Dr. Veronica Bacanova at the University of Minnesota. At ASH, we presented safety data from 35 patients with relapsed or refractory disease, 19 patients with non-Hodgkin lymphoma, and 16 with myeloma. GVA201 was generally well-tolerated with no dose-limiting toxicity, no GVHD, and importantly, no neurotoxicity observed. We were also very impressed with the promising clinical activity at all doses evaluated in patients with lymphoma. These patients were heavily pretreated with a meeting of three prior lines of chemotherapy. Of the 19 patients with lymphoma, 13 had complete responses and one had a partial response for an overall response rate of 74% and a complete response rate of 68%. Responses were observed in patients with both follicular and diffuse large B cell histologies. We're developing a cryopreserved formulation and will be submitting our IMD in the second half of this year. This will allow us to conduct a multicenter, multidose study to explore the potential of GDA201 as an off-the-shelf cell therapy in patients with lymphoma. I'm very proud of our team and their dedication to advancing our clinical studies during the global pandemic. I will now turn the call over to Shai to review our financial results.
spk06: Thank you, Renit, and good morning, everyone. Today, I will summarize our financial results for the full year of 2020. As of December 31st, 2020, we had total cash and cash equivalent of $127.2 million compared to $55.4 million as of December 31st, 2019. As Julian mentioned, during 2020, we were able to significantly enhance our capital position, with raising total of $144 million in gross proceeds from our May and December public offerings. In addition, in February 2021, we announced a $75 million financing with iBridge Capital Management. Those financing transactions extend our cash runway beyond the potential DLA approval of homidubicel expected by mid-2022. Research and development expenses for the year were $41.1 million compared to $31.5 million in 2019. The increase was mainly due to advancing the GDA201 clinical program and clinical activities related to concluding our Phase III trial, as well as additional headcount within the R&D organization. Commercial expenses in 2020 were $8.7 million compared to $4.7 million last year. The increase was mainly attributed to Omidubicel launch readiness activities, which includes addition to our commercial leadership team. General and administrative expenses were $12.2 million for 2020 compared to $12.1 million for 2019. The increase was mainly attributed to $1.3 million increase in professional services expenses including legal and insurance, offset by a decrease of $1.2 million in travel and non-cash compensation expenses. Finance expenses net were $10.4 million for the year compared to finance income net of $13.8 million in 2019. The increased expenses were primarily due to non-cash expenses resulting from revaluation of warrants owned by our shareholders and the revaluation of the Israel Innovation Authority royalty-bearing grant liability. Net loss for the year was $72.7 million, compared to a net loss of $34.4 million last year. We expect that our cash use for ongoing operating activities this year will range from $100 to $120 million. We anticipate that our current total cash position will support our ongoing operating activities into the second half of next year. This CASH one-way guidance is based on our current operational plan and excludes any additional funding that may be received or business development activities that may be undertaken. With that, I will turn the call back over to Julian.
spk07: Thanks, Shai. We are dedicated to finding cures for patients with hematologic malignancies and blood disorders, and we are excited about the opportunities ahead. With Omidubacill, we expect to submit the BLA in the fourth quarter of this year and are committed to being launch ready at the time of approval. With GDA201, we have very compelling data in lymphoma and are planning to initiate the GametaCell sponsored clinical study, which could transition into a registrational trial if the data are consistent with our phase one results. So as you can see, we expect 2021 to be a transformational year for GametaCell. and we look forward to updating you on our progress throughout the year. Now we will open the call for questions. Randy?
spk08: At this time, if you would like to ask a question, please press star then the number one on your telephone keypad. Again, it is star then the number one. Your first question comes from the line of Ted Tintoff with Piper Sandler.
spk09: Great. Thank you very much, and congrats on all the progress. Julian, maybe you can just walk us through sort of what's being done both in terms of preparing for the BLA submission, but also concurrently with respect to commercial buildup as you prepare for Omidubasil. Thank you.
spk07: Okay. Let me begin with the modules that are being prepared for the BLA submission. The non-clinical module is complete and ready. and the clinical module will be completed in the near term. And lastly, the CMC module will be completed by the fourth quarter, all of which will be necessary for BLA submission. Let me turn it over to Michelle to talk about our commercial preparations.
spk01: Excellent. Thank you, Julian, and good morning, Ted. In regards to commercial, there's really been three key aspects we're focused on. The first is leaders, and Shai alluded to this in his prepared remarks. We've brought in some outstanding leaders on the commercial team, Rocio Mangani leading market access with great experience in hematology and cell therapy. Linda Stamler was recently brought on to lead marketing and account management, and they join a couple of our other already established leaders. So we have a great leadership team in place. The next key area was to finalize the commercial strategy upon FDA approval. We've had great market insights that support the fact that upon FDA approval, Amadubacil will be a very important therapy option for patients who need an allogeneic stem cell transplant who do not have access to an appropriate matched related donor. It's a very exciting market opportunity for Amadubacil. And we are very clear in terms of what now needs to be done to execute on that strategy. And that's really the third part. So we've got our launch readiness plans underway, really that all-encompassing thought around making sure everything we're doing is leading to a positive experience for the patients and for the transplant centers. We're focused on four key aspects, making sure we educate the transplant centers, making sure that we educate the payers, The third is making sure we have that strong support system through Gameta Cell Assist. And finally, our commercial manufacturing readiness. Julian alluded to this in his prepared remarks. We're moving along very nicely with our new facility in Israel in Kirigat. We're also partnering with Lonza for their readiness for the BLA. So we're very much looking forward to the opportunity to introduce Amiduba Cell to patients upon FDA approval.
spk03: Great. Thank you very much.
spk01: So Julian, I'll turn it back to you. Thank you, Ted.
spk08: Your next question comes from the line of Jonathan Miller with Evercore ISI.
spk05: Hi, guys. Thanks for taking my question. I'll just follow up a little bit on that commercial ramp question. Obviously, with the BLA and 4Q, you've got some time to prep, and I understand you've got a strategy in place now. How do you think about spend ramping throughout the year as you hire folks, and how big a sales team will you need to achieve your commercial plan? Then, secondly... I'd love to ask about GDA201. One of the interesting factors about this product is naturalistic expansion of the NK cells with NAM. But now you're introducing additional, later in the pipeline, you're introducing additional engineering and gene editing to these products. How do you think about balancing adding that functionality while trying to maintain a healthy cell profile and maintain that biological profile that's kept that product distinct?
spk07: So, Michelle, please address the first question. And thank you, Jonathan. And I'll address the NK questions.
spk01: Yeah, absolutely. Thank you, Jonathan. Good morning. So in regards to the ramp up and personnel, obviously the personnel in the field will be key to our success. You know, the good news for us is this is a very efficient launch from the standpoint of personnel and also truly spending. So when you look at the target, so let's start with the transplant centers. There's roughly 200 FACT-accredited transplant centers in the U.S. We know many of these centers quite well. Ronit and her team had direct experience with at least 20 during the clinical trials, and then we certainly know more centers. And we also know that 70 centers make up about 80% of the transplants in the U.S. So we anticipate a very targeted and efficient launch in terms of field force In terms of actual numbers, so our field personnel will fall into three main categories with obviously supporting groups. The first will be on the commercial side, the account management team. If you look at benchmarks of other cell therapy launches in the U.S., you're probably looking at 20 to 25 personnel that would be needed to effectively launch the therapy and have that positive patient experience. We'll also have individuals focused on the payer space led by Rocio Mangani and That's also a relatively small footprint because the U.S. private payers are relatively consolidated, so probably about 10 personnel there. And then we'll have an outstanding group led by Ronit's medical affairs colleagues that are medical science liaisons who are supporting the educational initiatives with both the transplant centers and the payers. So we have a clear path going forward, and again, based on our experience in cell therapy, it will be a very efficient launch from a headcount and a spending standpoint. Julian, I'll turn it back to you.
spk07: Oh, John? Turning to the NK cells, so our first product candidate, GDA201, is a non-engineered expanded cell therapy in combination with frutoximab for lymphoma. And let me invite Tracy to talk about our plans for engineering the NK cells, particularly as we consider addressing solid tumors.
spk10: Sure. Thank you, Julian, and thanks for the question, Jonathan. And with regards to your first part, we're still, as Julian said, expanding up our NK cells with nicotinamide. And because of this, we have unique potential in our NK cells to maintain receptors that are on the cell surface, such as CD62L, which we believe uniquely causes our cells to home into heme malignancies and lymphoid organs, and as well as the very important CD16 receptor for ADCC. So additional editing efforts will maintain those properties and function of the cells that we see with NAM, but will further enhance what we think will allow our cells to survive in the suppressive tumor microenvironment by editing off some negative receptors and negative co-stimulatory receptors just to make the cells potentially more active and persistent in the tumor microenvironment as we expand upon this research effort in the lab this year to make these engineered products. So I'll turn it back to Julian.
spk07: Have we adequately answered your question, Jonathan?
spk05: Well, I look forward to seeing how that program develops and exactly how those folks behave. Yeah, for now I think that's it.
spk07: Yeah, we haven't named specific targets yet. We will do so at an appropriate time in the future. But right now the research is ongoing under Tracy's supervision, and we're very encouraged by what we're seeing.
spk08: And your next question comes from the line of Jason Butler, JMP Securities.
spk12: Hi, it's Roy on for Jason. Thanks for taking our questions. Sorry if I missed these in the prepared remarks, but anything at the EBMT meeting next week that we can expect that's different from the TCT meeting? And then for Omidubacil, just wondering if you guys can disclose how many patients have been treated in the expanded access? Then I'd have to follow up on 201. Thanks.
spk07: Ronit? This is to you.
spk11: Thanks. So with respect to the EBMT meeting, we're excited at the opportunity that the data will be presented in the European forum, and it's being highlighted in a high-profile session, the presidential session. There will also be a panel session in which the investigator, the European PI, Dr. Guillermo Sanz, who will be participating in a Q&A session live later on that same day. In terms of the data themselves, it's basically the same data set that were presented at the TCT meeting, but of course with some added nuance from Dr. Sands in his remarks and his panel discussions. In terms of expanded access, we haven't been updating on the number of patients or the progress at this point. The study is open at six sites across the US. It's open in a variety of sites, and there is enthusiasm from investigators to enroll patients. In the future, we do hope to provide some detail about the patients and their outcomes, but we haven't been giving those updates on an ongoing basis.
spk12: Fair enough. So, and then for 201, just what remains dating for the IND filing? And can you remind us if the cryopreserve formulation has been finalized? And is the FDA going to sign off on the formulation prior to the formal full IND filing, or how does that work? Thanks.
spk07: Tracy, would you address that, please?
spk10: Sure, sure. Thank you, Julian. In terms of what's left before we file the IND, it really is CMC section for GDA201 and finalizing their cryo formulation. The cryo formulation has been finalized in the lab. It's now just a matter of, and at scale, for clinical scale, it's now just a matter of manufacturing the enough of that for our clinical batches for stability at our GMP facility, which will be in Israel, before we're able to file the IND. And, yes, presumably we're in active discussions with the FDA. So both the protocol and the final formulation of the off-the-shelf aloe product that's cryopreserved will be discussed and agreed upon with them.
spk12: Great. Thank you.
spk08: Your next question comes from the line of Gregory Renza with RBC Capital Markets.
spk02: Hey, Jillian team. Congratulations on all the progress and thanks for taking my question. Jillian, just in regard to the modules, I just wanted to confirm, I believe you mentioned that the clinical module will be completely submitted shortly as well as the manufacturing by end of the year. Just curious if you if you can maybe characterize or quantify just the level of analytical and clinical comparability that is necessary from a Duba cell with extension, not disclosing the patients now, but just curious if there is a data set, a patient number that the FDA would like to see for their comfort there. And then just in terms of the preparation, it's great to hear all the callers from you and Michelle. I'm just curious, what has the extended timeline for rolling you know, four key 2020 Star BLA until end of this year, what has that afforded you as far as that preparation and getting that launch right? And maybe just the final question there, for Michelle, where are the gaps or the skeptical areas that are most likely to get in the way of realizing the Amadiba Cell vision ultimately? Thank you very much.
spk07: Yeah, so we, let me just start at a high level and turn it to Ronit to talk about the clinical module. and the CMC requirements. We had a very detailed conversation with FDA in December, and we have a very clear understanding of what they're looking for in terms of CMC, both analytical comparability as well as clinical comparability from our commercial manufacturing sites. Ronit, do you have anything more to add?
spk11: Yeah, I think we've shared data with FDA about our clinical results and we've told them or shared with them our plans in terms of the new commercial facilities. We believe that submitting high-level data on three to five patients will be sufficient for establishing that the clinical results are adequate to support production at those sites. So that's where we've landed in terms of the new production.
spk07: And Michelle, the second part of the question to you.
spk01: Yeah, absolutely. And thank you. And interestingly, there was a two-part question. They're actually both addressed in the same way. So what the extra time allowed us for commercial readiness and any concerns around potential gaps, what we were able to do with the additional few months was identify where were there potential challenges and how would we be able to overcome them. So now we have that ability to address them proactively. It really came down to three key areas. One was hiring. So as I mentioned, we have been able to take the opportunity to hire some great individuals with commercial experience both on the commercial side and then on the operation side too. The two specific areas that we've also been able to proactively focus on just addressing challenges would be around manufacturing for commercial readiness and our actual commercial launch. So manufacturing for commercial readiness, we're making very, very nice progress at our facility in Kirigat. you know, just not only in terms of the FDA requirements for the BLA, but also for overall commercial supply readiness upon FDA approval. So that is moving along nicely to plan. And then the second area around commercial readiness, this gave us some additional time to understand the needs of the transplant centers upon potential approval of Amadubasel, and that's in partnership with Ronit's medical affairs team. We also have the opportunity to also partner with Ronit's team to focus on the education of the commercial payers and really making sure that they understand the great unmet need for Amadubacil and understand the value proposition of Amadubacil, both in terms of the clinical data and the health economics side. So we're excited about the progress on both the manufacturing and commercial side for launch readiness. So Julian, I'll turn it back to you.
spk07: Yeah. I hope we've adequately answered your question. Any further follow-up on your end?
spk02: That's great. Thank you very much. I appreciate the color. Thank you.
spk08: Your next question comes from the line of Mark Bredenbach with Oppenheimer.
spk03: Hey, guys. Thanks for taking the questions. This is related to some of the others that have already come up. But, Julian, I was wondering if you could give us a little more color on the why the CMC module is the rate-limiting step in the BLA application for BLA filing. And have you reached an agreement with the FDA regarding sort of the criteria yet?
spk07: Yeah. I mean, it's not uncommon that CMC is usually the rate-limiting step for any product, but in particular in cell therapy. where there's a lot more attention and just different criteria to be adequate for supplying the commercial marketplace. So this is not unusual. Michelle, you can probably reflect on your experience at Kite when you were launching Yaskarta.
spk01: Thank you, Julie. And, you know, so we received very clear feedback from FDA at the December Type B meeting in terms of the requirements we needed to focus on for the CMC module. You know, just one point to recall is we manufactured our Phase III amaduba cell at a different Lonza facility. And we knew this wasn't a surprise for us. We knew we were going to be transitioning commercial supply to Lonza Netherlands facility in and also to cure GOTS. So what the FDA had discussed with us in December and what we're focused on now is the comparability requirements that are needed for the commercial facilities as compared to the phase three. So these are analytical comparability, the clinical data that we discussed, some of the methods validation work. All of that is mapped out and on track our Keurig God facility at this point in time, Lonza Netherlands had already begun some of the work on that. So the big takeaway is FDA was very clear in December and we're now moving along nicely in partnership with our research colleagues and also our clinical colleagues to assure not only readiness for the BLA, but this also gives us the opportunity to assure readiness for the commercial supply from the manufacturing standpoint too.
spk03: Okay, that's super helpful. And just a quick follow-up on GDA201, given that there are some key differences in the types of donors that you plan to use for your trial and the fact that you're using cryopreservation versus fresh product that we've seen in the academic sponsored study, I'm wondering if you have plans to present any preclinical, either in vitro cell killing assays or mouse data, demonstrating equivalence between these two versions of GDA201 later this year. Thanks.
spk07: Thanks for your question, Tracy. I'll turn that over to you as well.
spk10: Yeah, sure. Thank you. Yes, as you point out, for our IND filing, GDA201 will now be a completely allo off-the-shelf and cryopreserved product. It's still from the same source, from normal donor apheresis material, but just as a reminder, the clinical study that was done with collaboration with Minnesota was from a related donor, so the majority of those were haplodonors, whereas this will be completely allo. We do have plans to present some mechanistic work that we continue to do. understanding the further phenotype of our NAM-expanded NK cells over this new manufacturing protocol, which is cryopreserved, and with our sites presenting some of this work towards the end of this year at a conference, maybe likely ASH, but maybe another research conference. And at that time, we can present work both preclinically and the comparability between the fresh and the frozen That work is ongoing in the lab in Israel, and we have done quite extensive studies to show that we maintain both the function and the phenotype with the expanded NAM and K cells both at harvest and both after thaw of the cryopreserved formulation so that we will obviously present this data in the IND filing as well.
spk03: Got it. That would be super, super helpful to see. Thanks for taking the questions.
spk08: Your next question comes from the line of Gil Bloom with Needham and Company.
spk04: Hello, everyone, and thank you for taking our questions. So, just a quick one. We saw something of a trend with the overall survival in the pivotal study. Any thoughts on conducting an outcome study, maybe post-approval or Do you have any plans in that direction?
spk07: So, indeed, we saw a strong trend for overall survival. I would remind you that we were not powered for overall survival. And let me invite Ronit to further comment on follow-up of our patients.
spk11: Absolutely, Gil. So, thank you. So in terms of following additional information on patients, we will have additional follow-up for the patients in our Phase III study over time, as well as patients who enroll in our Expanded Access Study, which is basically a single-arm study of Omidubacill. Post-approval, we don't have any clear plans yet at this point on the type of follow-up that we'll do for patients in the real world, but all patients who are transplanted in the U.S. are followed in the registry by CIBMTR. So there may be an opportunity to leverage that in the post-marketing setting.
spk04: All right. Excellent. And maybe kind of an out-there question. So we see a lot of different benefits for the NAMP platform. Any thoughts about using this for gene therapy purposes? It allows the expansion of hematopoietic stem cells, and now you're looking at engineered hematopoietic stem cells, so the leap isn't really large there.
spk07: It's a very good point, and that is not lost on us, and we are exploring quite a number of avenues in the laboratory setting, and we have nothing to report at this time, But stay tuned because we think this platform will be the gift that keeps on giving as we entertain other expansion of different cell types. And again, we think this is a universal expansion platform and are doing a lot of preclinical work to understand with greater specificity, the mechanism of action, and how this can be applied across the board.
spk04: Excellent. Thank you for taking our questions, and congratulations on all the progress. Thank you.
spk08: There are no further questions at this time, and I would now like to turn the call back over to Julian for any closing remarks.
spk07: Thank you, everyone, for joining us on today's call. And we look forward to an exciting 2021 and future engagements with you all. Thank you.
Disclaimer