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spk00: Ladies and gentlemen, thank you for standing by. Welcome to GamidaCell's conference call for the first quarter 2022 financial results. My name is Carmen, and I'll be your operator for today's call. Please be advised that this call is being recorded at GamidaCell's request. Now I would like to introduce your host for today's conference, Heather DiBecchia, GamidaCell's chief of staff. Please go ahead.
spk03: Thank you, Carmen, 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 first quarter of 2022. 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.gamedacel.com. Please note that unfortunately, our Chief Executive Officer, Julian Adams, is unable to join us this morning due to contracting COVID-19. Company operations are unaffected, and Julian has advised us that his symptoms are improving and he expects to recover shortly. With that, here with me on our call today are Michelle Corfin, our Chief Operating Officer and Chief Commercial Officer, Ronit Simontov, our Chief Medical Officer and Chief Scientific Officer, and Shai Langree, our Chief Financial Officer. During this call, we may make forward-looking statements about our future expectations and plans, including in respect of the timing of initiation and progress of, and data reported from the clinical trials of our product candidates, anticipated regulatory filings, including the submission of the BLA for Amidubacil to the FDA, commercialization planning efforts, the potential life-saving or curative therapeutic and commercial potential of Gametacel's product candidates, including GDA201 and Amidubacil, and our expectations regarding our projected cash to be used for operating activities and cash runway. Our actual results may differ materially from what we project today due to a number of important factors, including the impact of COVID-19 pandemic on our operations, the scope, progress, and expansion of our clinical trials, and cost impact thereof, clinical, scientific, regulatory, and technical developments, and those inherent in the process of developing and commercializing product candidates that are safe and effective for use as human therapeutics. and in the endeavor of building a business around such product candidates, as well as those considerations described in the risk factor section of our most recent annual reports on Form 10-K and other filings that we make 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 Michelle.
spk01: Thank you, Heather, and thank you to everyone for joining us this morning. GametaCell had a productive quarter as we made important progress across all areas of our company. We continue to build momentum as we head into several inflection points expected this year, most near-term being the full BLA submission from AmadupaCell, which we are on track to complete in the second quarter of this year. Additionally, our GDA 201 program advanced, marked by the recent clearance by FDA of our IND and removal of the clinical hold for the cryopreserved formulation. We are excited to have reached this milestone and are now moving forward with our plans to initiate a company-sponsored Phase 1-2 study in patients with follicular and diffuse large B-cell lymphomas, which Roni will provide additional detail on. Beyond GDA201, which is our lead program in our expanding NAM-enabled NK cell pipeline, We are also looking forward to announcing a new product candidate from our genetically engineered NAM-enabled NK cell constructs later this year. We recently announced preclinical data at the ISCT conference supporting the development of GDA301 and GDA601. It's an exciting time in gametocel development, and we are focused on advancing our pipeline of potentially curative cell therapies. Our continued commitment is demonstrated by the new and recent data on Amaduba Cell that we presented this quarter, focused on Amaduba Cell's encouraging clinical data and our health economic efforts, which we will comment on later on in the call. With all we have accomplished only this past quarter, but also in the last few months, we are poised to be a leader in the development of NAM-enabled cell therapies with two promising and important clinical programs, and a robust pipeline of genetically modified NK cell product candidates in preclinical development. The progress we achieved this quarter continues to add to the important foundation that we have established. This strong foundation is due to our dedicated employees and their continued focus on patients and our vision of advancing therapies to help address unmet needs. With this, I will now turn the call over to Ronit.
spk05: Thanks, Michelle, and good morning, everyone. Thank you for joining us on the call this morning. Let me start with our lead program, Omidubacill, which has breakthrough therapy designation as well as orphan drug status and the potential to be the first FDA-approved cell therapy for stem cell transplant. Earlier this year, we were excited to initiate the rolling VLA submission for Omidubacill, starting with our non-clinical module, followed by the submission of our clinical module. I'm pleased that we are currently on track to complete the full BLA submission in the second quarter of this year, moving us closer to bringing this important potential therapy to patients. Our BLA for Omidubacil is supported by strong Phase III results, which met all primary and secondary endpoints. This past quarter, we announced the presentation of new data at the Transplantation Insulator Therapy, or TCT, meeting, as we've continued to add to the body of evidence demonstrating the potential of Omiduba Cell to address unmet needs in patients undergoing allogeneic stem cell transplantation. Patients often have serious infections after transplant due to the slow recovery of their immune system following treatment. In a presentation that received a TCT Best Abstract Award, Dr. Paul Zablosh presented data from our Phase III randomized trial of Omiduba Cell, showing rapid recovery of a broad repertoire of immune cells. which was associated with reduced infection rates in omadubicel-treated patients. In another oral presentation at TCT, Dr. Mitch Horwitz presented the final results of our Phase III study, including patient follow-up of over one year after transplant. The results showed that the early engraftment and lower infection rates previously observed in patients treated with omadubicel were accompanied by longer-term clinical benefits, including a reduction in transplant-related mortality and a trend towards an increase in overall survival, with no increase in relapsed or graft-versus-host disease compared to transplantation with standard umbilical cord blood. In addition, among our six posters at TCT, we presented 10-year follow-up data of patients treated in the Omiduba Cell Clinical Development Program, demonstrating long-term sustainable hematopoiesis and immune competence, with one case of secondary graft failure and one case of severe hematopoiesis. chronic GVHD among the 22 patients in the cohort. We also presented an analysis of resource utilization in the Phase III study, demonstrating that faster hematopoietic recovery and lower rates of infections in patients transplanted with omadupacil were associated with significantly shorter length of hospital stay, reduced admissions to intensive care unit settings, and lower healthcare resource utilization compared to control. Another poster outlined a quantitative analysis of well-established measures of patient health-related quality of life in a randomized Phase III study. The analysis demonstrated that omadubacil was associated with meaningfully greater preservation or improvement of quality of life, tying together clinical data in the Phase III study to important outcomes from a patient-focused perspective. Overall, we've continued to develop a robust set of clinical, scientific, translational, and quality-of-life data demonstrating the potential clinical benefit of transplantation with omodubicel. Now turning to our NK pipeline, I'll start with an update on GDA201. We're proceeding with our plans to initiate a study of GDA201 in patients with non-Hodgkin lymphoma. Although there have been recent advances for patients with lymphoma, we recognize that there is still an unmet need for patients with relapse and refractory disease. Given the responses observed in the investigator-led study at the University of Minnesota using the fresh formulation of GDA201, we've developed the choir-preserved formulation, which allows us to perform a multicenter study. We are very pleased to receive FDA clearance of our IMD for GDA201, removing the clinical hold and allowing us to move forward with the study. We are proceeding with the operational activities at several sites, and we anticipate conducting formal site initiation visits and opening sites for enrollment this quarter. Turning to our research and development activities, we're continuing to advance the preclinical data for our gene-edited NK cell pipeline aimed at hematologic malignancies and solid tumors. We recently presented two posters at the International Society for Cell and Gene Therapy, or ISCT, meeting in San Francisco. One poster presented new preclinical data on GDA301, our CISH knockout membrane-bound IL-15 expressing NK cell. Our poster showed cytotoxicity and potency of GDA301 in multiple tumor cell lines. The second poster detailed preclinical data demonstrating cytotoxicity of GDA601, our CD38 knockout anti-CD38 CAR expressing NAMNK cells against multiple myeloma cell lines. We look forward to presenting more data at scientific meetings this year. We plan to continue to conduct preclinical proof-of-concept studies for these genetically modified NK therapeutic candidates. And by the end of 2022, we plan to select a pipeline candidate for IND-enabling studies. With that, I'll turn the call over to Michelle to now talk more about omadubacil and commercial preparation. Michelle?
spk01: Thank you, Renita. I will now comment on our advancements with our CMC module and our launch readiness for amadubacil. Earlier this year, we initiated our rolling BLA submission for amadubacil, and we are nearing completion of the rolling BLA, which is on track for completion in the second quarter of this year. In the first quarter of this year, we reached an important milestone with the FDA's acknowledgement of the analytical comparability from our planned gametacel-owned commercial facility in Israel, as compared to the manufacturing facility used for the phase three study. This allowed us to move forward with completion of the remaining production runs in our facility in Israel to support the BLA. Our cross-functional team in Israel, including operations, quality, R&D, and regulatory, are progressing well on the CMC module in preparation for completing that module and the BLA submission in the second quarter. We are not only preparing for the BLA submission from an operations perspective, but also preparing for launch readiness. Our facility in Israel is modular, and upon FDA approval of Amadubasel, we will have the ability to add additional cores as demand grows for Amadubasel. We are confident we could support the launch demand requirements from our facility, not only from a production, but also a supply chain standpoint. The team is diligently working to ensure our manufacturing reliability, chain of identity, and chain of custody for our commercial process to ensure a positive transplant center and positive patient experience. Transitioning to launch readiness, the Gameta team recognizes the unmet need that Amadubacil may address for patients upon FDA approval. We are focused on our launch readiness to ensure, upon FDA approval, that patients could have access to Amadubacil. For patients with hematologic malignancies that are deemed eligible for an allogeneic stem cell transplant, the procedure may be their best chance for a potential cure. There are two key opportunities that Amadubacil may address for patients upon FDA approval, the first being potentially improving outcomes as compared to other donor sources based on transplant or feedback, and the second, potentially increasing access. In the United States, there are approximately 8,000 patients above the age of 12 with hematologic malignancies who undergo an allogeneic stem cell transplant each year. For potentially improving outcomes, we have conducted extensive market research over the last two years, and the insights are consistent and clear. Transplanters see important opportunities for amidoobacill to potentially improve outcomes based on their experience with other donor sources. This is due to the strength of our clinical data the ability to provide patients with predefined number of cells, and the ability to provide amidovacil within approximately one month of patient identification. The majority of the patients in the United States are still receiving their graft from an unrelated donor, and that can take on average at least two to three months to align the patient and the donor. This timing for unrelated donors unfortunately puts the patient at risk for relapse. Unfortunately, there are approximately 1,200 patients each year who are ages 12 and up with hematologic malignancies who are deemed eligible for an allogeneic stem cell transplant but cannot find an appropriate donor. In terms of potentially increasing access for patients, unfortunately, there is racial disparity in the U.S. in regards to access to allogeneic stem cell transplants. If you are non-Caucasian and do not have access to a family member donor, you have a very low likelihood of finding a match in the public database. For example, patients who are African-American may have less than a 20% chance of finding a match. Amadubacil has a less stringent matching criteria, and we demonstrated our ability to match racially and ethnically diverse patients in our Phase III study. Approximately 40% of the patients in our study were non-Caucasian. In a study recently highlighted in a poster presentation at TCT, we leveraged available registry data and population modeling to project the potential impact of amadubacil on racial and ethnic disparities. In the model population, increases in amadubacil use in eligible patients were associated with potentially higher proportions of patients undergoing transplant and overall potentially improved outcomes with improvements being greater among racial minorities. Taken together, we anticipate these two opportunities combined, pending FDA approval, both improving outcomes based on transplanter feedback and increasing access, may result in Amadubacil capturing approximately 20% to 25% of the addressable market once we reach peak market share. So upon FDA approval, this would equate to approximately 2,000 to 2,500 patients treated each year in the U.S. alone with Amadubacil. In regards to reaching transplant centers in the U.S., the transplant centers that perform allogeneic stem cell transplants are extremely concentrated. For reference in the U.S., there are approximately 200 transplant centers that perform allogeneic stem cell transplants. Seventy of those centers conduct approximately 80% of the transplants, allowing for an optimized approach to commercialization by initially targeting those centers. Another important aspect of our launch is engaging with payers to ensure that they understand the potential value of Amaduba Cell upon FDA approval. Payer conversations are underway with national and key regional payers, and we continue to hear consistent encouraging feedback on the overall value proposition of Amaduba Cell, including the strength of the clinical data and the health economic data we have published to date. We are also encouraged by their feedback on both the coverage and reimbursement approach they anticipate taking with Amadubacel upon FDA approval. We are excited to continue to hear positive feedback across all stakeholders and are extremely encouraged and driven by the potential of Amadubacel. We look forward to continuing to provide updates on our commercial preparations. I will now turn the call over to Shai to review our financial results. Shai?
spk10: Thank you, Michelle, and good morning, everyone. Today, I will summarize our financial results for the first quarter of 2022. As of March 31st, 2022, our total cash position was approximately $70 million compared to $96 million as of December 31st, 2021. Research and development expenses for the quarter were $11.3 million compared to $11.4 million in the first quarter of last year. The decrease was primarily due to a $1.1 million decrease in Omidubicil and GDA201 clinical studies activities, offset by an increase of $1 million in bordering our scientific capabilities and talent. Commercial expenses for the quarter were $3.9 million compared to $4.2 million in the first quarter of 2021. The decrease was mainly due to reducing our near-term commercial readiness expenses as we are assessing alternatives for the commercialization of Omidubicil including potential U.S. or global partnerships. General and administrative expenses were $4.1 million in the first quarter of 2022 compared to $3.5 million for the same period in 2021. The increase was mainly due to a $0.5 million increase in ad count and related expenses. Finance expenses net were $0.9 million for the first quarter of 2022 compared to $0.1 million for the same period last year. The increase was primarily due to a $0.6 million increase in interest expenses from our convertible nodes. Net loss for the first quarter of 2022 was $20.2 million compared to a net loss of $19.2 million in the first quarter of 2021. We expect cash use for ongoing operating activities for the entirety of this year to range from $65 to $70 million. We anticipate that our current total cash position will support our ongoing operating activities into mid-2023. This cash runway guidance is based on our current operational plans 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 Michelle.
spk01: Thank you, Shai. As we look ahead to the rest of the year, we are uniquely poised to deliver on our mission of developing potentially curative cell therapies for patients with blood cancers and other serious diseases. We look forward to our full BLA submission from AdubaCell, expected in the second quarter of this year, and the initiation of our Phase I-II multicenter Gameta-cell-sponsored study for the cryopreserved formulation of GDA201 in patients with follicular and diffuse large B-cell lymphomas this year. We are excited for the opportunity to continue to leverage our unique NAM-enabled platform across a broad range of cell therapy candidates, and we look forward to providing updates throughout the year. Now we will open the call for questions. Carmen?
spk00: Thank you. And to ask a question, simply press star 1 on your telephone. To withdraw the question, press the pound or hash key. Your first question comes from John Miller with Evercore. Please go ahead.
spk06: Hi. This is Eric Sanzo calling in for Jonathan Miller. Just had two quick questions on GDA 201. With trial sites opening in second quarter, when can we expect the first patient in? And what sort of dynamics do you see around enrollment? Like, do you expect COVID or summer travel to impact that? And then I have one follow-up.
spk01: Excellent. Thank you, Eric. Thanks for joining the call. I will turn to Ronit to address both of those. Thank you.
spk05: Thanks, Eric. So in terms of opening sites, so sites will open this quarter, and patients will need to be recruited and screened appropriately before the first patient is treated. So it usually takes several weeks for that to happen until a patient is actually treated with GDA-201. But, you know, there's a lot of interest among the investigators for this study. There is an unmet need. These are patients who are relapsed or refractory patients. and clearly need additional therapies and are engaged in enrolling in a clinical trial. And so with the high need for these patients and the fact that they are ill with very serious cancer, we believe that enrollment will be robust for these patients. I'll make one more point about this portion of the study. It is a phase one portion of the study. As this is a phase one where toxicities are being evaluated patient by patient, we will be evaluating toxicity in each patient before proceeding with enrollment to the next patient. So we can carefully gauge patient enrollment as we move forward in the next few months and evaluate dose-limiting toxicities. Once we evaluate that, then we can enroll patients more concurrently.
spk06: Got it. Very helpful. And just one more question. Do you expect any differences in enrollment timelines between the follicular and the DLDCL cohorts?
spk05: At this point, we don't anticipate a difference between those two cohorts, but that's something that we can gauge as we move along. We certainly have the ability to analyze them separately if need be. You know, if there is a discrepancy, we can manage that within the confines of the clinical trial design. But at this point, we don't anticipate that there will be a difference because our investigators have expressed that patients with both histologies are in need of new therapies.
spk06: Great. Thank you.
spk05: Thank you, Eric.
spk00: Your next question comes from Ted Tenhoff with Piper Sandler. Please go ahead.
spk08: Great. Thank you, and good morning, everyone. I'm curious what is sort of going on or what the latest is with respect to commercial prep for OB-GYN. I know you are considering potential strategic alternatives, but also really just trying to get a sense for, as you're finishing up the BLA program, what might be the most likely scenario for marketing?
spk01: Excellent. Good morning, Ted, and thank you for joining us. I'll go ahead and take that question. So, in regards to commercial preparation, we've accomplished some critical milestones to date. So, first off, was really honing the commercial insights to understand, first off, the overall opportunity, but second of all, what could Amadou Basel's potential be for that opportunity? So, We've, over the last couple of years and most recently, we've reiterated or re-looked at some of these market insights. We recognize the two key opportunities from a DubaCell. One is the ability to improve outcomes as compared to other donor sources, and this is based on transplanter feedback. And then the second is increasing access. In terms of improving outcomes, some of the key insights that transplanters share with us is the strength of the clinical data from our phase three study, both the efficacy and the safety that Ronit has presented. Also, the ability of having, you know, predefined number of cells. And then finally, the turnaround time. In the United States, the majority of patients currently are still getting their donor source from an unrelated donor And on average, we're hearing that takes at least two to three months to align the donor and the patient. And as you know, with acute leukemias and aggressive lymphomas, that puts the patient at risk for relapse. So with amadubacil being approximately one month from time of patient identification in the phase three study to return of amadubacil, that is a positive feature. And then switching to the increasing access or improving access, you know, unfortunately in the United States, we do see many patients who are deemed eligible for transplant that cannot find the donor. The latest data that we've assessed is approximately 1,200 patients each year, and that may end up growing. And unfortunately, it's also a situation of racial disparity. If you are non-Caucasian in the U.S. and don't have access to a family member, it's incredibly difficult to find a match. So to summarize, based on the encouragement of those market insights in regards to the opportunity for Amadou Bassel, we have hired our commercial leadership team, and we also have the operations and supply chain leadership team in place. So this way, that core group of leaders could continue to assess not only the opportunity, but most importantly, the launch readiness. And that's moving in parallel to our assessment of potential strategic alternatives. We feel very, very strongly that upon FDA approval, we do need to make sure patients have access to Amadubacil. So we've got our leadership team in place as we're also in parallel looking at potential strategic alternatives.
spk08: And would those alternatives include both the United States and overseas? Because this feels to me like a market that is appropriately sized for a biotech company to launch, especially since you guys have so much familiarity with the product with the treatment group. So I just want to get a little more color on that. Thanks.
spk01: Yeah, thank you, Ted. So we are assessing strategic opportunities, you know, globally, so including the U.S., but also ex-U.S., We have conducted initial commercial assessments in Europe and in Asia, specifically in Japan, and are very encouraged by the commercial opportunity for Amadubacil in those areas. So we are, as we discussed, strategic alternatives for potential launch of Amadubacil looking globally.
spk08: Great. Thank you very much.
spk00: Thank you, Ted. Your next question comes from Jill Blum with Meadham & Company. Please go ahead.
spk02: Good morning, everyone, and thanks for taking our question. Maybe a broader one. Considering recent results in donated NK cells in the space, I'm just curious how encouraged you are seeing that a company-sponsored study could lead to some very interesting early results, and how do you think that translates for GDA201? Thank you.
spk01: Excellent. Good morning, Gil, and thank you for joining us and for the question. Let me turn to Ronit to address Gil's question.
spk05: Sure. Thanks, Gil. So, you know, there have been some encouraging results that we've seen from other products, the NK cells type products recently. And overall, those are actually really encouraging because it sets the stage for NK cells. And In particular, some of the results we've seen are related to high doses delivered of NK cells with results seen with greater responses seen at the higher doses. And that actually sets us up quite well because we're able to deliver with GDA201 high doses of NK cells, high numbers of NK cells that are extremely functional and retain their killing capacity because the NAM technology that we've used to expand and maintain the stemness of stem cells actually expands and maintains the NK-ness or the killing potential of NK cells. So we actually think it sets us up quite nicely for our Phase 1-2 study. Our study is designed to evaluate the safety of the cryopreserved formulation at first and then move right into a formal efficacy evaluation. The entire study is unblinded, you know, open-label study, and we'll be able to see as we go what the potential for activity is with this formulation. So we're excited to start enrolling patients and seeing results.
spk02: Thank you, Olney. That was very helpful. Maybe also on a quick financial question. So on the SG&A spend, there was a line that mentioned that the there are some cost savings due to consideration of strategic options for commercialization. Just to help me understand, does that mean that the company is cutting back on commercial preparedness launch? Or maybe just help me understand that. Thank you.
spk01: Thank you, Gil. So I'll start with sort of the strategic aspect, and I'll not turn to Shai for any additions on the financial side. We were fortunate last year to hire some incredibly strong commercial leaders, Linda Stamler to lead marketing and account management, Rocio Mangani to lead market access. Those two individuals and their teams were able to conduct a lot of the critical market insights and beginning of launch preparation last year. So then that allowed us to use this past quarter more to evaluate the insights and less OPEX associated with having to gather additional insights, and also less hiring. We were able to hire our core group of commercial and operation leaders last year. So that's sort of the high-level strategic aspects of it. And let me turn to Shai if there's anything to add from the financial side.
spk10: Hi, Gail. Good morning. Thank you for the question. The way we see it, Gabida Cell is a company that continues to diligently manage our cash position, and as such, we announced back in January this year that we are assessing some strategic alternative for launching Omidubicel, as our vision is to bring Omidubicel to patients. Yes, we did prioritize the key activities, not only on the commercial side, but across all the business, to make sure we are not jeopardizing our launch activities. And as I mentioned, and Michelle mentioned before, making sure each patient will have access to Omidubisol upon the approval.
spk02: All right. Thank you both for taking our questions this morning.
spk00: Thank you, Gil. Your next question comes from Jason Butler with JMP Securities. Please go ahead.
spk07: Hi. Throw in for Jason. Thanks for taking our questions. I want to follow up on the partnering I guess, can you give a little more detail maybe on the level of interest that you're seeing? Do you think you could have a partnership in place before a potential approval maybe around year end? And can you remind us what structure you prefer for the U.S., maybe a co-promote? Thanks.
spk01: Thank you. Good morning, and thank you for joining us. I'll go ahead and take both of the questions. We won't comment on specifics in regards to our assessment at this point in time, but I do want to reiterate something very important that Shai had said earlier, and that's the fact that although we are assessing strategic alternatives for launch, we also do have the leadership team in place on both the commercial, the medical affairs, the quality, and the operations side to assure that patients have access to Amadu to sell upon FDA approval. So when you asked about sort of the latter part of the question in terms of timing. We do have the strategy in place, the plans in place to assure access to patients for Amidubacill upon FDA approval. So at this point in time, as we indicated earlier this year, we are assessing strategic alternatives, but also continuing to do our work internally around assessing the opportunity and assessing what would be needed for launch planning.
spk07: Okay, great. That's helpful. Thank you. Then for the the four NK candidates beyond GDA 201, are you going to take only one into the IND-enabling studies or potentially multiple candidates? And then what happens to the other candidates that don't go into IND-enabling studies this year? Do they go on hold? Are you going to continue to refine them preclinically? Thanks.
spk01: Excellent. Thank you very much. I'll turn to Ronit to address that question.
spk05: Thanks. And thanks, Jason. So one at a time. So we will take the lead candidate by the end of this year and do the appropriate IND-enabling studies and continue to develop, based on data, of course, the additional candidates and move those forward as we are able.
spk07: Thank you very much.
spk00: Thank you. Your next question comes from Mark Brater with Oppenheimer. Please go ahead.
spk04: Hi, this is Jacqueline from Mark, and thanks for taking our questions. So can you please remind us what dose levels will be tested in the dose escalation from the GDA201, and if there are any differences in lymphodepletion conditions to cytokine support or rituximab dosing?
spk01: Thank you for joining us, Jacqueline. I'll turn to Ronit to address both the dosing question and the question around lymphodepletion and rituximab in the protocol.
spk05: Absolutely. So the design of the study, the design of the administration or the treatment design is based on the Minnesota study using the fresh formulation. And so we will be testing doses that are similar to the ones used in the Minnesota study. And the administration of lymphodepleting chemotherapy, rituximab, and IL-2 are also very similar, if not identical, to what was done in the Minnesota study. So I don't think we've come out yet with the exact dose levels, but they are guided completely by the previous data in Minnesota.
spk04: And when do you expect to commence manufacturing runs for GDA201 for the Phase I trial?
spk01: Actually, I'll ask Ronita if you could also take that, please.
spk05: Of course. I'm happy to take that. So manufacturing, we are building, because in the Phase I trial, the GDA201 is produced ahead of time and not matched for patients, but basically readily available, we are building our inventory already and expect to have the product available for patients as soon as the first dosing is required.
spk04: Great. Thanks for taking our questions. Thank you.
spk00: Thank you. Your last question comes from Matthew Cross with Alliance Global Partners. Please go ahead.
spk09: Hi, all. Good morning. Best wishes to Julian for a speedy recovery. I had two quick questions related to the Phase 1-2 for GDA-201 and kind of following up on your comments there, Ronit, about the recovery. scheduling for lymphodepleting chemotherapy and antibody use. I guess considering this internal study will be using the cryopreserved formulation, not the fresh, just wanted to understand any of the kind of special considerations here, I guess, for the study compared to the prior one. It sounded like the schedule will be more or less the same or based off of the prior study, but given that you're not baking in time for NAM expansion with the cryopreserved. I just wanted to understand whether that scheduling would still look similar and maybe to kind of recap any modifications that would have to be made to the study that were needed to remedy the FDA's concerns around donor eligibility requirements and anything that may play into which sites are able to participate in the study given the combination of those donor eligibility requirement changes and the cryopreservation use here versus what was done previously. Thanks.
spk01: Excellent. Thank you, Matt. Thank you for joining us, and thank you for the well wishes. We will pass them along. Thank you. And I'll turn to Ronit to answer Matt's questions in regards to the Phase 1-2.
spk05: Sure. I'm happy to answer that, and I'll take the donor eligibility piece first. So the donor eligibility procedures and the assay qualifications, we needed to make sure that those are compliant with FDA requirements and so we replaced our donor testing laboratory with a CLIO certified lab in the US and we've now made sure that all appropriate tests are licensed and cleared and that eligibility requirement has been met. So there has been no change in the design of the study based on, the hold itself did not require any changes in the design of the study, the protocol, the sites, or anything that had to do with the clinical trial itself. It really was confined to the eligibility of the product. So that was that piece. Now in terms of cryopreserved formulation, it's great that you picked up on that. So in the fresh formulation study, patients had to have a donor available, that donor had to be recruited and the effervescence had to be done and then the product had to be produced over time. So that, because we have a readily available product now, it will be at the site when the patient is enrolled. And so that may save some time in terms of getting the patient up and treated because they'll have product there. They don't have to wait for the donor to get all ready, get, you know, the donation and and do all that. And then procedurally, there will be a thawing process. The product will be there. It will be frozen, and it will be thawed at the bedside to provide the patient with the product when they need it.
spk09: Perfect. Okay, super helpful. And I just had one quick follow-up, which was around the sizing. It looks like, per what you've drawn up on clinicaltrails.gov, that there's kind of a target enrollment of about 100 patients. So I guess I know you're not commenting at this point on the actual dose levels that you may step through, but just wanted to confirm whether that total sizing was based around kind of an expectation internally around the number of phase one cohorts that you may look at, if it will be ultimately still driven by the standard kind of safety evaluations as you step through doses, or maybe just kind of an expectation for the phase two population for that portion of the study. Just wanted to get a little bit of an understanding around that. the assumptions for that sizing.
spk05: Yeah, absolutely. So that number, which is put in clinicaltrials.gov and which is incorporated into the protocol itself, incorporates, you know, there is some flexibility there because you don't know for sure how many patients you're going to end up enrolling in phase one. It's a standard three-by-three design where if there is a dose-limiting toxicity observed, then the dose level changes. numbers are expanded to include more patients. And so this is the maximum size based on the possible expansion of cohorts in the phase one portion. And then it also includes the phase two portion where we're going to be evaluating separately the patients with follicular lymphoma from the patients with the aggressive lymphomas. And each of those sort of phase two portions has its own stats and analysis surrounding it. But, you know, they're relatively small cohorts to allow us to evaluate rather quickly efficacy results and then make decisions based on those early efficacy results that we see.
spk09: Understood. Okay. Thanks again for the clarity. Appreciate it.
spk05: Thank you. Thanks so much, Matthew.
spk00: Thank you. And this concludes our Q&A session. I will turn the call back to Michelle Corfin for her final remarks.
spk01: Thank you, Carmen. Thank you very much for joining us for the call today, and we look forward to keeping you all updated on our future milestones. Have a nice day. Thank you, Carmen. That will conclude our call.
spk00: Thank you, ladies and gentlemen, for participating, and you may now disconnect.
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