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spk06: Welcome to Gameda Sales conference call for the third quarter 2020 results. My name is Shannon, and I'll be your operator for today's call. Please be advised that this call is being recorded at Gameda Sales request. Now, I would like to introduce your host for today's conference, Mr. Josh Hammermesh, Chief Business Officer. Please go ahead.
spk12: Thank you, Shannon, 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 third quarter 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.gimitasel.com. Here with me on our call today is Julian Adams, Chief Executive Officer, Ronit Simintov, Chief Medical Officer, and Shai Lankrey, 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, and 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 GametaCell 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.
spk08: Thank you, Josh, and thanks to everyone for joining us this morning. At GametaCell, we are committed to developing cell therapies with the potential to provide cures for patients with blood cancers and rare serious hematologic diseases. We've continued to make very strong progress over the past few months with both of our programs, Omidubacill, which could be the first FDA-approved engineered graft source for bone marrow transplant, and GDA201, a natural killer or NK cell therapy, which has the potential in both hematologic malignancies and solid tumors. Today, we'll review both programs and summarize our progress around plans to potentially bring omadubacil to patients in the commercial setting. Starting with omadubacil, in October, we were thrilled to report our global randomized phase three study of omadubacil met all three secondary endpoints related to platelet engraftment, infections, and hospitalizations. These results strengthen our confidence in the clinical potential of Omidubacil and build upon the positive primary endpoint data we reported earlier this year. We expect to report additional details from the clinical study by the end of the year. We also anticipate initiating the BLA submission for Omidubacil on a rolling basis in the fourth quarter, which positions us for a potential launch in the second half of 2021. We're also making good progress with other key launch activities required to bring on MeduvaCell to patients following potential FDA approval. Our cell expansion platform also led to the generation of GDA201, our NK cell-based product candidate. NK cell therapies offer tremendous potential for transforming the care of hematologic malignancies. We are pleased to be pioneering a novel approach that harnesses the power of our cell expansion technology, which uniquely improves antibody-dependent cellular cytotoxicity, known as ADCC, and the in vivo homing potential of GDA201 to address the limitations of NK cells. With GDA201, our goal is to develop an off-the-shelf allogeneic cell therapy with response rates similar to the CAR-Ts in lymphoma, while potentially offering a more favorable safety profile. Data from our ongoing Phase I study have demonstrated striking early signs of efficacy with multiple complete responses in patients with advanced lymphoma. We will be presenting updated data next month at the American Society for Hematology, or ASH, annual meeting next month. Ronit will review our ASH abstracts in greater detail. We're continuing to plan for our next trial, which will be a Phase I-II multicentered study in patients with advanced lymphoma. We have developed a cryopreserved formulation of GDA201 and are working on GMP scale-up of this formulation to enable our IND submission in 2021. Our hope is, if the data are compelling, this study could serve as the basis for for a registrational trial through an accelerated approval pathway. As we advance our clinical programs and prepare for a potential launch next year, we're continuing to make key hires within the organization who bring new capabilities and further enhance our strong team. Today, we announced the appointment of Steve Jamison to the role of Senior Vice President, Information Technology. Steve brings 25 years of IT experience, primarily in the life sciences industry, including roles at Veristem, Infinity, Ariad, and OSI. Additionally, this week, Rocio Mangani joined Gamita Cell as a Senior Vice President, Market Access. She has nearly 20 years of experience in market access and patient support at commercial organizations, including Celgene and Roche. Of particular relevance, Rocio served in a leadership role at Kite Pharma and was instrumental in securing patient access for Yascarta. We are excited to welcome Steve and Rocio to the Gameta cell team and look forward to their contributions as we look toward a potential Omiduba cell launch next year. I want to conclude my introductory remarks by acknowledging the challenges of this year as we navigate through a global pandemic. We are committed to the health and safety of our employees and continue to advance our business while implementing work-from-home policies, shift work in our laboratories and manufacturing facilities, travel bans, and regular COVID testing for employees working onsite. We continue to watch our timelines very carefully. Today our anticipated milestones for both OMA-DUBA-Cell and GDA201 are unchanged. We continue to expect to initiate the BLA submission for OMA-DUBA-Cell on a rolling basis in the second half of 2021. Sorry, by the end of the year, which sets us up for a potential approval and launch in the second half of 2021. We are planning to submit the IND for GDA201 to enable our multicenter phase 1-2 study in lymphoma next year. I am very proud of the work we are doing in an effort to bring cures to patients, and I am impressed by the hard work and dedication of our employees. I'll now turn the call over to Ronit Simintov, our chief medical officer, to provide a further update on Omidubacill and GDA201. Ronit?
spk04: Thank you, Julian, and good morning, everyone. This morning, I'll review our clinical programs and highlight our recently published ASH abstracts describing data from the study of Omidubacill in patients with severe aplastic anemia and for GDA201 in patients with non-Hodgkin lymphoma. As Julia noted, this year we've reported that our global phase three study of Omidubacill in 125 patients with hematologic malignancies met the primary endpoint and all three secondary endpoints. We are proud of this rigorous, well-executed trial. and we truly appreciate the support of our collaborators in the transplant community who are working with us to help move the field forward, especially while working through COVID-19. I'll briefly highlight what we've reported so far. In May, we announced that the study met its primary endpoint, time to neutrophil engraftment, a key milestone in recovery from bone marrow transplants, signifying how quickly the stem cells patients received became established and began to make healthy new cells. The study showed that omodubicel was generally well-tolerated. In the intent-to-treat analysis, the median time to neutrophil engraftment was 12 days in patients randomized to omodubicel compared to 22 days for patients in the comparative group randomized to standard cord blood transplant. The p-value was less than 0.001. Last month, we announced that all three pre-specified secondary endpoints of the study demonstrated a statistically significant improvement among patients randomized to omodubicel versus the comparative group. Specifically, the secondary endpoints were platelet engraftment by day 42, incidence of grade 2 or grade 3 bacterial or invasive fungal infection, and the number of days alive and out of the hospital in the first 100 days following transplant. Rapid hematopoietic recovery, reducing infections, and shortening hospitalizations are all clinically meaningful outcomes valued by physicians, patients, and their families. Our data analyses are still ongoing, and we hope to present the data in a peer-reviewed forum at the end of the year. Overall, our phase three data, coupled with our recently presented observational study, which showed improved survival in patients with adult donors less than 30 years old, reinforce our belief that Omidubacill could be an important graft option for any patient who does not have a suitable matched donor. We believe Omidubacill has potential beyond hematologic malignancies and are evaluating Omidubacill in patients with severe aplastic anemia, a rare life-threatening blood disorder. This investigator-sponsored Phase I-II study is being led by Dr. Richard Childs at the National Institutes of Health. We previously reported data from an initial cohort of three patients who successfully underwent a reduced-intensity conditioning regimen and received a stem cell transplant consisting of Omidubacill plus a haploidentical stem cell graft. Dr. Childs then initiated a second cohort of patients to evaluate Omidubacill as a standalone graft. Initial data from this cohort will be presented at ASH next month. The abstract published last week included data from the three patients in cohort one and five patients in cohort two. The data showed that Omidubacill were generally well tolerated and led to rapid sustained engraftment. With a median follow-up of 10 months, seven of the eight patients had early and sustained engraftment with neutrophil and platelet recovery occurring at a median of 10 days and 31 days respectively. There was no evidence of acute or chronic GVHD, and these patients remained transfusion independent. The study, which is still ongoing, highlights the potential for omodubacil to be used in non-malignant bone marrow diseases and lends further support to evaluating omodubacil more broadly using reduced intensity conditioning, which could make omodubacil a more feasible treatment option for elderly or frail patients or others unable to withstand a myeloablative regimen. We look forward to the presentation of these data in a virtual poster session at ASH on Saturday, December 5th. In addition to Omijuba cells, we are advancing our NK program, GDA201. Natural killer cells are innate immune cells that have held tremendous promise for treating cancer. However, the field has faced several developmental challenges, including the ability to expand NK cells and culture while preserving their functionality. Our technology addresses this challenge. and our Phase I data provide impressive proof of concept. The clinical study is designed to assess the safety of three increasing doses of GDA201 in combination with a monoclonal antibody in relapsed or refractory patients with non-Hodgkin lymphoma or multiple myeloma, as well as to determine the recommended Phase II dose. The study is being conducted by Dr. Veronica Bakanova at the University of Minnesota. The abstract submitted to ASH, which was accepted for all presentations, includes data from 30 patients as of the cutoff in July 2020, 15 patients with non-Hodgkin lymphoma and 15 patients with multiple myeloma. First, it's important to note that GDA201 was generally well tolerated with no dose-limiting toxicities, no GVHD, and importantly, no neurotoxicity observed. I'll focus on the 15 patients with non-Hodgkin lymphoma. who were treated with GDA201 and rituximab. Histologies included eight patients with diffuse large B-cell lymphoma, six patients with follicular lymphoma, and one patient with mantle cell lymphoma. Patients had a median of three prior lines of therapy with a range of one to eight lines, and 87% had advanced stage disease. Given this patient population, we were thrilled to see clinical activity at all doses evaluated, The abstract reported that among these 15 patients, there were 10 complete responses and one partial response, for an overall response rate of 73%. Median duration of response was 8.7 months in this population, and we were encouraged to see responses out to 25 months. Also of note, four patients underwent retreatment with GDA201 without lymphodepleting chemotherapy, which likely contributed to deepening of responses in two of these patients. This finding supports our plan to evaluate multiple doses and to potentially exclude the requirement for lymphodepletion in our company-sponsored study, which is expected to begin next year. Next month in the oral presentation at ASH, we expect to include longer-term follow-ups to the patients already reported, as well as data from additional patients who were enrolled and evaluable since the time of abstract submission. I'm very proud of our team and their commitment to advancing our clinical studies during the global pandemic. And I look forward to sharing additional data from both programs at ASH in just a few weeks. With that, I will turn the call over to Shai to review our results.
spk07: Thank you, Renit. And good morning, everyone. Today, I will summarize our financial results for the third quarter of 2020. As of September 30th, 2020, we had total cash and cash equivalents of $73.3 million compared to $55.4 million as of December 31st, 2019. Research and development expenses for the quarter were $10.5 million compared to $7.5 million for the same period in 2019. The increase was mainly due to BLA readiness preparation, increase in clinical activities related to the advancements of QDA 201, and the initiation of the Omidubicel Expanded Access Study. Commercial expenses in the quarter were $1.9 million compared to $1.7 million for the same period last year. The increase was mainly attributed to Omidubicel commercial readiness activities. General and administrative expenses were $2.7 million for the third quarter of 2020 compared to $2.8 million for the same period in 2019. The decrease was mainly driven by reduced travel expenses. Finance income net was $0.3 million for the quarter compared to $1.7 million in the same period in 2019. The decrease was primarily due to non-cash income resulting from revaluation of warrants owned by the company shareholders. Net loss for the quarter was $14.8 million compared to a net loss of $10.1 million in the same period last year. We expect that our cash use for ongoing operating activities this year will range from $60 to $65 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.
spk08: Thanks, Shai. We're committed to finding cures for patients with hematologic malignancies and blood disorders, and we are excited about the opportunities ahead. With Omidubacill, we expect to present our Phase III data and initiate our rolling BLA submission by the end of the year. With GDA201, we have very compelling data in lymphoma and are working hard to initiate our own clinical study, which could transition to a registrational trial if the data are consistent with our ongoing Phase I study. We hope to finish the year strong and to carry our momentum into 2021, which we expect to be a transformational year for Gametacel as we prepare for a potential approval and launch of Omidubacel in the U.S. Now we will open the call for questions. Operator?
spk06: Thank you. Ladies and gentlemen, to ask a question, you will need to press star 1 on your telephone. To withdraw your question, press the pound key. Please stand by while we compile the Q&A roster. Our first question comes from Jonathan Miller with Evercore. Your line is open.
spk14: Thank you so much for taking the questions, guys, and congrats on all the progress. I'd love to start with Omiduva Cell. It clearly looks superior to standard cord blood and very intriguing. They said the transplant modalities as well, but the trial was against cord blood, obviously, and not those other sources. So I guess In your conversations with docs and payers, how have they characterized domidubazil's potential versus not just cord blood but other transplant sources that I know that you're targeting? Do you expect initial commercial update to mostly be replacing cord, or do you hope to start replacing other modalities in a meaningful way immediately as well? And then just on GDA201, You know, obviously the data in the ASH abstract looks very interesting, especially in follicular and probably in NHL. How are you planning on structuring the next set of trials? Is there any sign of activity in myeloma? Do you expect that to be potentially better with a different MAB combo agent? And how are you approaching the broader heme malignancy space there outside of NHL?
spk08: So let me ask Ronit to... cover the first part of your question, how do physicians view our data with omadubacil compared with other transplant modalities? And then, Michelle, you could comment on the potential launch activities and introduction of omadubacil to the marketplace.
spk04: Thanks, Julian. I will start with how physicians are reacting to omadubacil. So rapineutrafol engraftment is attractive no matter what the graft source. It's an opportunity for patients to have a superior outcome after transplant to get out of an isolation room, to have a lower incidence of infections and other complications, and generally to have improved outcomes. And so the physicians that we speak to are very impressed with the level of engraftment that we're seeing, supported by the other clinically relevant endpoints from the secondary endpoints. So we believe that Omidubacil is potentially an attractive graft source for patients who don't have a suitably matched donor from another source.
spk01: Excellent. Thank you, Ronit. Thank you for the question. So, you know, I'll reiterate what Ronit has said based on our discussions with physicians. If the patient does not have access to a match-related donor, there is the potential for them to be an amadubacil candidate. You know, physicians and payers have been very encouraged by the clinical profile to date for amadubacil across efficacy, safety, and the ability to provide the patient with amadubacil. In the clinical trial, our experience was four weeks from the time of identification of the cord for the manufacturing until return of amadubacil. So those three aspects, efficacy, safety, and the logistics have received very encouraging feedback from physicians. So there is an opportunity for amadubacil across all current treatment modalities from a graph source standpoint. Now, in regards to launch, you know, we're very excited, as Julian indicated, to begin to add exceptional personnel to our commercial team, including Rocio Mangani joining us this week. So we're building up the team and expectation of a potential launch in the second half of 2021. Our launch of Amaduba Cell is absolutely going to be focused on assuring a positive experience for the transplant center and the patient. So hence, we have four key tenants that we're working on to prepare for the launch. preparing to educate the transplant centers so they understand the Amaduba Cell Clinical Profile and the appropriate patients, educating the payers. We've been encouraged by the initial payer feedback, but we recognize that will be very important to make sure we continue on. Third is having that appropriate support group to help initiate the chain of identity to assure that that is established at the time of treatment decision, but also to provide any additional patient or reimbursement support And then the fourth key area is around manufacturing readiness. So we are excited by our progress in all four of those areas. So thank you for the question.
spk08: And related to GDA201, let me ask Ronit again to comment on the clinical experience so far, and then I'll ask Tracy to talk about the potential for a combination with other monoclonal antibodies.
spk04: Thanks. So in terms of GDA201, we're very impressed with the data that we've observed in lymphoma so far in both indolent histologies and aggressive histologies. And so what we foresee is that our next study will include the opportunity to evaluate GDA201 in different histologies, obviously with the appropriate methodology for evaluating them separately. And so that's what I can say about the design of our next study. We think that it will include both. In terms of myeloma, we just didn't see the level of responses that we observed in lymphoma. It potentially could be attributed to the antibody, and there may be opportunities to explore myeloma using either a different antibody or a different methodology. I'll turn it to Tracy.
spk05: Thank you, Ronit. And yes, and so I will reiterate that we're very excited about what we see in non-Hodgkin's lymphoma. In particular, to your question around multiple myeloma, we do believe, given research data, that we think the combo with the particular antibody, elutuzumab, is not optimal. And so we are working on a research-engineered program to genetically modify the NK cells, which will actually allow us to combine with different antibodies that we think will be more potent in multiple myeloma. But this will be not part of our trial next year. In addition to that, we continue to be excited about the activity of NK cells and the safety of NK cells with our NAM expanded platform. So we do have ongoing research programs looking at combinations with antibodies with solid tumors as well. And that will be in the future, and hopefully we will present that work at a conference sometime next year. So thank you for the question.
spk14: Yeah, thank you so much, guys.
spk06: Thank you. Our next question comes from Ted Tintoff with Piper Sandler. Your line is open.
spk11: Great. Thank you very much. Again, really exciting time for the company. We've got a lot going on. I wanted to get a sense that I think you guys have breakthrough designation and wanted to confirm that you would seek accelerated approval. We have been seeing just because of the pandemic. And do you anticipate this would require a ODOT adcom? Thanks.
spk08: So I'll take that, Ted. Thank you for your question. So just we do, to reiterate, we do have breakthrough therapy designation. But this is a pivotal phase three study, and we're seeking full approval, not accelerated approval. But we will be seeking priority review, which, as you know, is a six-month review. And we don't anticipate the need for an advisory committee because we've met all of our endpoints, both primary and secondary endpoints with statistical significance.
spk11: Great. And I appreciated the color on the commercial prep, and I'm looking forward to the presentations at ASH. Thanks so much. Thanks, Ted.
spk06: Our next question comes from Jason Butler with JMP Securities. Your line is open.
spk02: Hi. Thanks for taking the questions, and congrats on all the progress. First one on the DuvaCell question. Just in terms of the aplastic anemia market dynamics, can you just walk us through what the patient options are today and how Omiducacel can change that? And obviously it's a rare indication, but can you give us a sense of the size of the patient population in the U.S.? ? And then for 201, just any update on the manufacturing process here and, you know, to what extent you're through the work that needs to be done to submit the IND.
spk08: Ronit, could you discuss aplastic anemia?
spk04: Absolutely. So patients with aplastic anemia tend to be young. It's got a bimodal distribution, but most of the patients are young. and are treated with transfusions to support their red blood cells and platelets, and immunosuppressive therapy, because most of the time it's an immunologically mediated disease where the immune system attacks the bone marrow. Patients with severe aplastic anemia have very low counts that are very dangerous. They can be susceptible to infections, bleeding, and other complications. And the only cure for severe aplastic anemia is bone marrow transplant. For patients who don't have a match, there really is no cure. And, you know, they subsist on supportive therapy, but there's really no opportunity for a cure other than a bone marrow transplant. So what Omidubacill is offering is a potential for cure in patients who don't have a match. And particularly important are the results of the study that we are that were published in the abstract are particularly important because patients with aplastic anemia tend to have a higher rate of graft rejection. The transplants don't take easily in patients with severe aplastic anemia because of the way the bone marrow's got this immune system and there's just a difficulty in supporting engraftment. And so the fact that a single unit of Omiduba cells supported sustained and rapid engraftment is actually a very, very positive sign. So what Omidibofil can offer for these patients is a potential for cure that may just not exist for them currently. Severe plastic anemia is a rare disease and the numbers of patients in the U.S. are measured in the hundreds.
spk08: Thank you. Tracy? Can you talk about the GDA201 progress in our manufacturing?
spk05: Sure. And thank you, Jason, for the question. So I'll just remind you that the clinical results that Ronit just summarized were with a fresh product with our collaborator at Minnesota. So at Comedicel, we've made tremendous progress in making a cryopreserved formulation. So our intent next year is is to have an off-the-shelf cryopreserved product. We have that final formulation now, and it's just a matter of manufacturing this under GMP conditions, and we actually have the flexibility to manufacture this at Gevita Cell, and we're also looking at some external capabilities for manufacturing for the clinical trial for next year. But we remain on track to having that allo off-the-shelf product for the trial for next year.
spk02: Okay, great. Very helpful. Thanks.
spk06: Our next question comes from Gregory Renzo with RBC Capital Markets. Your line is now open.
spk13: Hey, good morning, Julian and team. Congrats on the progress and thanks for taking my questions. Just to build on some earlier questioning just around the progress of the BLA filing, what do you see as the gaining factors or critical path to this and perhaps for each module? Are there any components that you're still waiting for in terms of data or information and just trying to understand the expectation of timing once you start that, how long that will take for perhaps each module or in total in light of your goals for second half 2021 launch potential? Thank you.
spk08: Let me cover the cadence of filing. Our non-clinical module is ready. We anticipate beginning the filing with that particular module. That will be followed early next year by the clinical module. Lastly, the manufacturing module, Module 3, the CMC section, will complete within a six-month time frame the full filing of the BLA.
spk13: Gotcha. Very helpful. And then, Julian, we've spoken about this before, but I certainly get questions on the intellectual property of Amaduba, so I just wanted to see if you could to just take an opportunity to provide some color on your view of the protection you have there with respect to the product and the process. Thank you very much.
spk08: Greg, I'd be happy to. Our patent expiry, we have quite a number of patents, but our longest patent expiry is out to 2038. But importantly, because we have orphan drug status, We have 12 years exclusivity in the US for a BLA and 10 years in Europe. And finally, we have many trade secrets that are not in the public domain. And because Omidubacil is a personalized manufacturing where Omidubacil is consumed by the recipient patient, there's no inventory. So it's really not possible to make a biosimilar. And so we feel that the exclusivity period is very, very strong for us.
spk13: That's great. Thanks, Joe. I look forward to the data at the end of the year.
spk08: Thanks, Greg.
spk06: Our next question comes from Vernon Bernardino with HCWayrite. Your line is open.
spk09: Hi, everyone. Thanks for taking my question and congrats on the progress. and the very strong phase three results. A key announcement to me seems to be the expansion of the collaboration with the Be The Match therapies in October as far as supply of cord blood units. Can you remind us again as far as supply and any effects of the pandemic on that supply and I was just wondering also if you could comment on, I guess, any effects of the pandemic on donation of cord blood units and if there's any effect and what you have done as far as what would need to be done to provide a smooth process for the supply of the unit once the Omidubazil commercialization has begun, thank you.
spk08: So let me begin by discussing the pandemic and then I'll turn it over to Michelle to talk about commercial supply. As it would happen, it actually made it easier to ship cord blood units because there are no volunteers involved. So for a matched unrelated donor, one has to locate the donor and they still have to be willing to donate and come to an infusion center to donate their bone marrow. And that was dampened by the pandemic because obviously healthy adult donors were reticent to go to hospital settings. And that's certainly not the case with cord blood units since they're all cryopreserved and shipping was routine and we never experienced a delay based on the pandemic. And, Michelle, maybe you could comment on the sort of commercial supply agreement with Be The Match.
spk01: Absolutely, and thank you, Vernon, for the question. So we're very excited to continue our partnership with Be The Match. So we had an important partnership with them for the Phase III study, and we're excited to extend that partnership for commercialization upon potential FDA approval. Coming back to what I had said earlier, a very, very important part of our launch is assuring a positive experience for the transplant center and the patients. The transplant centers have an established relationship with Be The Match. So by us being able to continue our partnership, it provides for the continuation of the partnership that the transplant center already has. You know, they partner with Be The Match when they are looking for a graft source beyond a matched related donor or related donors. So we're excited to be able to continue that partnership. We do think that it will be very beneficial for the transplant centers in terms of streamlining the logistics associated with using Amadubasal upon potential FDA approval.
spk09: Yes, that's a very important relationship. Thanks for taking my question. Thanks, Julie, and thanks, Michelle. Good luck looking forward to the ASH data. Thank you, Vernon.
spk06: Our next question comes from Gil Bum with Needham & Company. Your line is open.
spk03: Hello, everyone. This is Gil on for Chad, and thank you for squeezing us in. My question is regarding the... clinical study of GDA-201 that's planned for next year. Could you kind of discuss a little bit what your expectations are, at least initially?
spk08: The key feature of next year's trial will be obviously with an off-the-shelf cryopreserved product, and it will be a multicentered study. It'll be designed as a Phase I-II study. And let me ask Rene to talk about how we're going to enroll different histologies, different cohorts of patients.
spk04: Thanks, Julian, and thanks, Gil. So I think, yeah, I think you pretty much captured it. So the main features are that it will be a multi-center study because the cryopreservation will allow us to ship the product to different sites, much as we did with Omidubacill. And that we will enroll patients of different... histology of lymphoma and analyze them in separate cohorts in order to best understand what the efficacy is in those different histologies. I think those are the broad strokes in terms of the expectations for that study.
spk03: All right. Furthermore, on GDA201, could you put into context the ability to use multiple doses of cellular therapy, especially considering standard of care?
spk08: Ronit?
spk04: Sure. So in the study, in the Phase I-II study that we've been conducting at Minnesota, there was interest in using a second dose of cells of GDA201 in patients for one of two reasons. First, we wanted to explore the possibility of administering the cells without lymphodepleting chemotherapy prior to administration. And second, we wanted to see if administering additional doses would deepen or consolidate responses in patients. And there were four patients who had that second dose with lymphoma. It was tolerated fine, and we were able to administer it without lymphodepleting chemotherapy. But more importantly, perhaps, we were able to see objective evidence of deepening of response in patients after that additional dose. And so that's, for us, we think that's an interesting opportunity to administer multiple doses, and we will explore that in further study.
spk08: One of the tenets of our approach is that we think that our NK cells in combination with rituximab in lymphoma are eliciting an adaptive immune response. There's certainly clinical evidence for that. and we think that that's what is sort of ideal. That's the biology of NK cells that we think is pertinent to the development of GDA201.
spk03: That makes sense. One last one on medubacill. You guys did have that press release about beta match. Going forward, and I'm thinking about the market as it expands, Do you think your relationship with Be The Match would be sufficient, or are you guys going to have to look into other sources of cord blood? Thank you.
spk08: Michelle?
spk01: Yeah, no, thank you, Gil, for the question. So, you know, as I mentioned prior, we're very excited about the partnership with Be The Match. You know, they are, you know, very, very much a partner not only now for us but also for the transplant centers. We don't foresee that there would be a challenge in terms of supply with utilizing Be The Match. They have a very extensive network amongst the core blood banks. So at this point in time, we don't see a concern in terms of the supply from Be The Match.
spk08: I might add to that exuberantly. You know, there are 4 million babies born every year in the United States, so we think that ultimately this is an inexhaustible supply as long as Omidubacill continues to serve patients.
spk03: Excellent. That's very helpful. Thank you for taking our questions, and we look forward for the presentations at ASH as well.
spk01: Thank you.
spk06: Thank you. Once again, ladies and gentlemen, if you wish to ask a question at this time, please press star then 1 on your touchtone telephone. Our next question is from Mark Breedenbach with Oppenheimer. Your line is open.
spk10: Hey, thanks, and so glad you could squeeze me in. Two questions on the DubaCell. With respect to the BLA filing, I'm just curious if you've reached alignment with the FDA regarding the contents of the CMC analytical package, especially given that this is a first of its kind product that the FDA would have to review.
spk08: So we're in regular discussions with the FDA. We've had multiple meetings this year, including one recently. We're not commenting on the details of those discussions, but I would say that they're very collaborative. We do enjoy the breakthrough therapy designation, and therefore the FDA and we speak often and very cooperatively.
spk10: Okay. And in your upcoming presentation of data from the Phase 3 trial, will you be reporting treatment-related mortality rate? And I'm just wondering if there's a defined TRM threshold that you think it's important to stay below relative to other graft varieties.
spk08: So, Ronit, would you care to comment?
spk04: Happy to comment on that. In our phase 1-2 study, rapid neutrophil engraftment was associated with lower treatment-related mortality in the 36 patients in the single-arm phase 1-2. Certainly, the issue of treatment-related mortality is very important to transplanters and to patients, and we will share those data when we present the rest of the secondary endpoints and the rest of the data of efficacy and safety data for the study.
spk10: Okay, but no specific thresholds that you think it's important to stay below?
spk04: I think if we observe data that are relatively consistent with what we've observed in our previous study, then that would give us confidence that rapid engraftment is associated with clinical benefit, much as some of the other clinical endpoints that we've seen.
spk10: Got it. Thanks for taking the questions.
spk06: Sure. Thank you. And I'm currently showing no further questions at this time. I'll turn the call back over to Julian Adams for closing remarks.
spk08: Thank you, Shannon. And thank you, everyone, for joining us on today's call. We are looking forward to the virtual ASH meeting and to sharing additional program updates in the coming weeks. Shannon?
spk06: That concludes today's call. You may now disconnect.
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