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spk02: Ladies and gentlemen, thank you for standing by. Welcome to Gameta Sales conference call for the third quarter 2022 financial 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 Gameta Sales requests. Now I would like to introduce your host for today's conference, Heather DeVecchia, Gameta Sales Director, Investor Relations and Corporate Communications. Please go ahead.
spk10: 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 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.gometafil.com. Here with me on our call today are Abby Jenkins, President and Chief Executive Officer, Ronit Simintop, Chief Medical Officer and Scientific Officer, Michelle Corfin, Chief Operating Officer and Chief Commercial Officer, and Shai Langree, Chief Financial Officer. During this call, we may make forward-looking statements about our future expectations and plans, including in respect to the timing of initiation and progress of, and data reported from, the preclinical and clinical trials of our product candidates, regulatory filings, including the review of the BLA for Amadubicel by the FDA, commercialization planning efforts, the potential life-saving or curative therapeutic and commercial potential of Gametacel's product candidates, including GDA-201 and Amadubacel, and our expectations regarding our projected cash, cash equivalents, and investments to be used for operating activities. 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 impacts to the cost thereof, clinical, scientific, regulatory, and technical developments, 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 quarterly report on Form 10Q, 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 or future events, except as required by applicable law. Now I'd like to turn the call over to our recently appointed President and CEO, Abby Jenkins.
spk03: Thank you, Heather, and thanks to everyone for joining us this morning. It is an exhilarating time for GametaCell as we mark another quarter of meaningful progress and prepare to shift from clinical to commercial stage with the potential approval of our transformative stem cell therapy candidate, Amidubucel. I would like to start off by saying how excited I am to speak with everyone today on my first earnings call as GametaCell's president and CEO, a role that I assumed in September. I was drawn to GametaCell for three reasons, the mission, the science, and the team. I've worked to bring innovative therapies with the promise of addressing diseases of high unmet need for over 25 years. While many of those products made an impact through treatment or disease prevention, none had the potential to be truly curative. Our mission of developing curative therapies for people with cancer or other serious diseases is incredibly inspiring and is made possible because of our remarkable science. Our NAMM technology both increases the number of stem cells and immune cells and enhances their functionality, enabling us to create potentially transformative cell therapies that go beyond what is possible with the existing approaches. NAM is the power behind AmidubaCell, our lead product candidate, which has breakthrough status and is currently under priority review with the FDA with a PDUFA date of January 30th, 2023. Amidubacil has the potential to transform the treatment landscape for patients in need of a stem cell transplant. Our Phase III clinical data demonstrate Amidubacil significantly reduced the time to neutrophil engraftment from 22 to 12 days versus standard cord blood transplant. Neutrophil engraftment is a key predictor of patient outcomes. Earlier this month, we further showed that in real-world data, Amidubicel produced the shortest median time for neutrophil engraftment of any hematopoietic stem cell transplant option, including that of a match-related donor source. Amidubicel achieved neutrophil engraftment in 10 days as compared with 15 to 20 days for other donor sources. We believe that Amidubicel offers a meaningful new transplant option as the data support that neutrophil engraftment occurs rapidly and is durable maintaining an effect for up to 10 years based on clinical studies. In addition, Omidubacill reduces total days hospitalized post-transplant, which improves outcomes while reducing costs to the healthcare system. If approved, Omidubacill will be the first FDA-approved allogeneic hematopoietic stem cell therapy to address an expanding unmet need for patients with blood cancers in need of a stem cell transplant. NAM is also the power behind our pipeline of allogeneic NK cell therapies, including our clinical stage therapy, GDA201. Ronit will be going into more details in this regard following my remarks. Speaking of, and bringing us to the third reason I was drawn to GametaCell, the team. The advancement of our NAM-enabled cell therapies is possible as a result of our talented team of experienced leaders like Ronit and Michelle, who have successfully developed and launched multiple life-enhancing treatments in hematology and oncology, including cell therapies. We have many experienced and talented team members across GametaCell discovering, developing, and preparing to deliver our advanced cell therapies to patients in need. Let's turn now to Q3 results. This was an important quarter marked by continued progress toward larger inflection points. Our BLA for amidubicel was accepted by the FDA and granted priority review. Beyond amidubicel, we progressed our Phase I-II clinical study, evaluating a cryopreserved, readily available formulation of GDA201, our lead NAM-enabled NK cell therapy, for the treatment of follicular and diffuse large B-cell lymphomas. GDA201 holds tremendous promise, and we look forward to its continued progression in the clinic. Beyond GDA201, we continue to be excited by our NK pipeline of cell therapy candidates, and we believe our NAM technology increases targeting, potency, and persistence of a broad range of innate and adaptive cell types, including NK cells. However, we are going to hold on selecting the next IND candidate from the NK pipeline at this time to both prioritize resources to the commercialization of Amidubicel and advancement of GDA201, as well as enable development activities on these canned visits to further progress. Next, we highlighted promising preclinical and clinical results across our pipeline at multiple medical and scientific meetings, including CoreBlood Connect, the Society of Hematologic Oncology, and the Society for Immunotherapies of Cancers. Additionally, we strengthened our financial position with a public offering in September 2022, raising $20 million as well as obtaining a commitment letter from Highbridge Capital for a $25 million senior secured convertible term loan. With this capital raise and with our FDA review of Amidu Bissell progressing on track, we are accelerating into launch mode. Michelle will provide more details on this very important initiative later on the call. I believe Gimita Cell is a company that is poised for long-term success. And I want to thank my predecessor, Julian Adams, for his vision and leadership in bringing the company to where it is today. Julian leaves us an incredible legacy of scientific innovation, which enabled us to develop assets like Ami Dubassell and GDA 201. We are grateful to continue to benefit from his guidance on our board of directors and wish him well in his retirement. I would also like to take an opportunity to commend my new colleagues. I am continually inspired by your teamwork and dedication with our important work every day, and that you're putting patients first in all we do as we advance on our mission. Additionally, I would like to sincerely thank all of the clinical trial sites and the patients and their families that have been such important partners as we advance our pipeline of NAM-enabled cell therapies. We believe we have a truly transformative therapy with Omidubacill, and we are motivated every day by the meaningful impact it can have on patients' lives. With that, I will now turn the call over to Roni to take us through key data supporting our NAM-enabled cell therapies. Roni?
spk12: Thank you, Abby, and good morning, everyone. Thank you for joining us on our call. This morning, I will review our clinical data on Omidubacill, provide an update on long-term follow-up, quality of life, and immune reconstitution data presented over this past quarter, and preview our upcoming real-world data presentation. I will then discuss our GDA201 study and the rest of our pipeline. As we eagerly approach our PDUFA date of January 30, 2023, we continue to add to the body of scientific and clinical evidence demonstrating the potential of omodubicel to address unmet needs in patients undergoing allogeneic stem cell transplant. Most relevant to our regulatory submission is our successful Phase III global randomized study, which met its primary and all secondary endpoints. The study was comprised of 125 patients, ages 12 to 65, with high-risk hematologic malignancies, who were in need of an allogeneic stem cell transplant, but had no readily available matched donor. The study demonstrated a median time to neutrophil engraftment of 12 days for patients randomized to Omidubacill, compared to 22 days for the comparative group transplanted with standard cord blood. These results were not only statistically significant, but also highly clinically significant. as neutrophil engraftment is a key milestone in recovery of patients undergoing stem cell transplant. This quarter, we presented long-term follow-up data from the Omidubacill Clinical Program at the Society of Hematologic Oncology, or SOHO, meeting, supporting the durable clinical benefit of Omidubacill in the years after transplant. These data included follow-up of 105 patients transplanted with Omidubacill in our Phase I, II, and III studies between 2006 and 2020. The data demonstrated three-year overall survival and disease-free survival of 63% and 56% respectively. Moreover, follow-up data of up to 10 years showed durable production of blood cells and immune cells, indicating sustained long-term recovery of the bone marrow in patients transplanted with omadubasal. Patient health-related quality of life data from our Phase III study were also presented at the SOHO meeting and recently published in the journal Transplantation and Cellular Therapy, the official publication of the American Society for Transplantation and Cellular Therapy. In an analysis of 108 patients who completed validated health-related quality of life surveys at screening and through the first year after transplant, measures of physical and functional well-being, as well as other health-related quality of life scores, were more favorable with Omidubacil than with control. These data suggest clinically meaningful and continual improvements in the physical, functional, and overall well-being of patients treated with Omidubacil. Prior to SOHO, we presented translational data at the Cord Blood Connect meeting in September, demonstrating recovery of immune cells in patients treated with Omidubacil. In an analysis of the T-cell recovery in a subset of 37 patients from the Phase III trial, Patients transplanted with Omidubacil had more rapid, robust, and diverse immune cell reconstitution, including higher numbers of recent thymic immigrants in the blood at one year post-transplant compared to patients transplanted with standard cord blood. These immune reconstitution data may provide mechanistic support for the observation that patients transplanted with Omidubacil have lower rates of bacterial and viral infections after transplant. While our phase three data compared omidubacil to standard cord blood, now, in collaboration with the Center for International Blood and Marrow Transplant Research, or CIBMTR, we have explored the effectiveness of omidubacil compared to other allogeneic transplant donor sources used in clinical practice. As recently announced, these data have been accepted as a podium presentation at the annual meeting of the American Society of Hematology, or ASH, meetings. We compared results from 52 patients who received Omidubacill in our Phase III clinical trial with results from 807 patients in the CIBMTR database with similar demographic and clinical characteristics who were transplanted with other donor sources, including matched unrelated, mismatched unrelated, and haploidentical donors. Our data show that Omidubacill was associated with significantly more rapid neutrophil recovery and, importantly, Other outcomes, including graft versus host disease, relapse, and overall survival, were comparable across donor sources. These real-world data are the first to demonstrate the comparative efficacy of omadubacil to donor sources beyond standard cord. The abstract has been posted on the ASH website, and the data will be shared at ASH in December. The new data presented this quarter continues to support the clinical benefit and safety of omadubacil. and give us confidence as we prepare to bring this potential therapy to patients following FDA approval. Before turning to Michelle, who will provide an update on our plans to launch Omidubacill in the U.S. market upon potential FDA approval, I would like to give you an update on GDA201, our lead product candidate in our NK cell therapy pipeline. GDA201 leverages our proprietary NAM technology in the expansion of NK cells to enhance functionality, tumor cell-killing properties, and antibody-dependent cellular cytotoxicity, or ADCC. Data from the investigator-led study at the University of Minnesota on the fresh formulation of GDA201 were reported at ASH in December of last year and demonstrated an overall response rate of 74% with durable responses and two-year survival of 78% in heavily pretreated patients with non-Hodgkin lymphoma. Despite the recent advances in the development of therapy for patients with non-Hodgkin lymphoma, we continue to hear from experts that there is a high unmet need among patients who have active disease after treatment. Our company-sponsored Phase I-II clinical study evaluating the prior preserved formulation of GDHU1 is progressing on track, and we are continuing to enroll patients in the Phase I dose escalation portion of the study. The study includes patients who have relapsed or refractory lymphoma after at least two prior treatments, which may include CAR T-cell therapy or stem cell transplant. Phase 1 includes patients with follicular, diffuse large B-cell, marginal zone, and mantle cell lymphoma histology. The Phase 1 portion of the study is designed to evaluate the safety of increasing doses of GDA201, with dosing similar to that in the previous investigator-led study. Up to four dose levels will be tested to determine the maximum tolerated dose and recommended Phase 2 dose. based on dose-limiting toxicities. The Phase 2 expansion portion of the study is designed to evaluate the safety and efficacy of GDA201 in two separate patient cohorts of approximately 30 patients each with follicular lymphoma and diffuse large B-cell lymphoma. The Phase 1 is currently ongoing, and we're looking forward to continuing to progress this important therapy candidate through the clinics. In our expanding cell therapy pipeline, we are also developing our genetically modified NAM-enabled NK cell therapies in hematologic malignancies and solid tumors. Our novel product candidates leverage CAR and CRISPR-mediated technologies to increase targeting, potency, and persistence, and are supported by robust preclinical data. We are evaluating multiple product candidates, including GDA301, GDA401, GDA501, and GDA601. This quarter, we announced new preclinical data supporting GDA501 at the CITSE meeting. In a poster presentation, GDA501, a HER2 CAR, NAM, and K-cell, displayed significantly enhanced in vitro cytotoxicity when cultured with HER2-positive cancer cells. Further, GDA501 showed increased potency based on biomarkers and elevated levels of pro-inflammatory cytokines compared with control cells. Importantly, increased cytotoxicity and potency were persistent. These preclinical data demonstrated potent anti-tumor activity of GDA501 and support its therapeutic potential as a novel cell therapy against HER2-positive cancers. With that, I will turn the call over to Michelle, who will talk more about Omidubacil and our advancements in manufacturing and our commercialization plans. Michelle?
spk05: Thank you, Roni, and good morning, everyone. I would like to reiterate how excited we are to be in this position as we are poised to bring our first NAM-enabled cell therapy, Amadubacel, to the U.S. market, which, if approved, will be the first and only FDA-approved allogeneic hematopoietic stem cell therapy for patients with blood cancers in need of a stem cell transplant. The potential clinical benefit of Amadubacil continues to be supported by a growing body of data that has been reported in blood for our Phase III data and in well-recognized medical meetings. Based on these data, we have heard consistent positive feedback from transplanters focused on our clinical outcomes, including rapid time to neutral engraftment, durability of response, and quality of life for their patients. As we approach the January 30th, 2023 PDUFA date for Amadubacil, we continue to work toward our goal of maximizing a positive patient and transplant center experience when using Amadubacil as the donor source of choice. As Ronit reviewed, the data that will be presented at ASH, in addition to extensive market insight feedback, transplanters can consider Amadubacil not just as an alternative to standard core blood, but as an alternative to all other donor sources. Upon approval, we are ready to deliver Amadubacil to transplant centers. We will work closely with transplant centers to make sure they have the necessary procedures and logistics in place to deliver a cell therapy like Amadubacil to patients in need. Importantly, we continue to hear positive feedback from payers, recognizing the meaningful impact of Amadubacil for patients potentially setting us up for well-defined paths for coverage and reimbursement. I am proud of all the work we have completed thus far to define the unmet need that Amaduba Cell could address and have a clear launch strategy and a well-defined launch plan. As a result of the successful financing this past quarter, we are now in a position to continue building out a specialized and integrated organization in preparation for a launch following potential FDA approval. This integrated team, including commercial, medical affairs, quality and operations, is focused on ensuring that we are ready to provide patients with access at the time of launch, and we have now moved to launch execution. During 2023, we will be primarily focused on ramping up transplant centers throughout the U.S. As we have discussed, this will be a targeted launch. As we know, 70 transplant centers make up approximately 80% of the transplants. This part of the launch execution for Amadubacil will be critical to ensure a positive patient experience and includes important programs such as education and training sessions and process and logistics review. Although Amadupacil has a less stringent matching criteria than other sources, there is still a matching requirement. So we need to work with centers to assure appropriate chain of identity and chain of custody. In addition, at time of potential FDA approval, we will have a patient support system in place to facilitate access. Now I would like to share with you some of our market research that drives our strategy and plans of action. As a result of extensive market insight studies, we have determined two critical differentiators that can position Amadubacil as a treatment of choice for transplanters and their patients. The first is driven by clinical studies and transplanter experience that leads hundreds of transplanters in market insight studies to indicate that they feel Amadubacil delivers better outcomes compared to other donor sources. And second, the benefit of Amadubacil increasing access so patients have broader access to transplant. Let me start with improving outcomes. Improved outcome results in capturing share from current donor sources. We have learned from our extensive insights that Amadubacil has the potential to capture share from all donor sources. From a donor source distribution, we know that matched unrelated donor shares approximately 45%, haploidentical approximately 22%, matched related donor approximately 22%, mismatched unrelated donor approximately 7%, and court blood about 5%. Insights support share capture across all donor sources. Starting with matched unrelated donors in the United States, The key unmet need that Amadubacil will address is time. It takes on average about two to three months to align an unrelated donor to the patient. That puts the patient at risk for relapse and then not being able to proceed to transplant. Amadubacil has consistently been delivered back to the transplant center in about 30 days. So this is a key aspect that would lead a transplanter to choose Amadubacil over an unrelated donor. For haploidentical donors, this is a family member that is a 50% match. The challenges that amaduvacil can address involve patient outcomes. Due to the partial match, haploidentical donor results in a delayed time to neutrophil engraftment, on average about 18 days, according to published literature, as well as an increased risk of infection and graft-versus-host disease, or GVHD. Although post-transplant cyclophosphamide is used to mitigate GVHD, that also introduces risk for the patient, especially due to cyclophosphamide's cardiotoxicity. Amadubacil's rapid time to neutrophil engraftment of 10 days and encouraging outcomes for infection rates and GVHD lead transplanters to indicate they see a benefit from Amadubacil over haploidentical, which would allow for greater access. Match-related donor challenges focus on donor age. The average age of diagnosis for an adult leukemia patient is about 60 years of age. Chances are your sibling, who could be your match-related donor, would also be in their 50s, 60s, or 70s. Published clinical data supports the older the donor, the worse the patient outcomes are. On the DubaCell, with our starting material being cord blood, prior to the expansion enhancement of our manufacturing, does not have an age concern since our starting material comes from a newborn. A mismatched unrelated donor poses two concerns for a transplanter. The partial match leads to suboptimal outcomes and the concern around the two to three months on average to align the patient and the donor. Mismatched unrelated donors have on average about 17 days to neutrophil engraftment And again, the Amadubacil clinical data and 30-day turnaround positions Amadubacil to take market share from mismatched unrelated donor sources. And finally, cord blood. Based on the statistically significant results from our pivotal Phase III study, transplanters anticipate a strong share capture for Amadubacil. Moving to increased access, it is estimated in the United States that there's approximately 1,200 patients 12 years of age and older with hematologic malignancies that are eligible for transplant but cannot find an appropriate donor. Unfortunately, there's health disparities. If you are non-Caucasian and you do not have access to family members for a donor source, it is incredibly difficult to find a match in the public database. For example, a patient who is black has less than a 20% chance of finding a donor source in a public database. Because of Amaduba Cell's less stringent matching criteria, we can match a diversity of patients quickly. Our phase three demographics supported that, with 40% of the patients being non-Caucasians. Most oncology clinical trials are probably under 10% of non-Caucasian patients. With the combination of improving outcomes, which means capturing share from other donor sources, and increasing access, Upon reaching peak market share, Amadu Basel has the potential to capture 20% to 25% of the addressable patient population, which would equate to 2,000 to 2,500 patients per year in the United States. In terms of launch readiness, the leadership of our commercial, medical affairs, and operations teams are working diligently to assure we are now in launch prep execution mode. The teams have had preliminary discussions with many of the top transplant centers to assess their needs for introducing a new cell therapy into their facilities. We have our full payer team in place for launch. Reimbursement mechanisms are also defined on both the commercial and Medicare sides, including the recent issuance of the CMS ICD-10 PCS code for Amaduba Cell, potentially allowing for payers to cover Amaduba Cell upon FDA approval without formulary review boards which has created reimbursement challenges for other novel cell therapies in the past. A very important aspect of a successful cell therapy launch is manufacturing. We have been successfully manufacturing clinical batches at the GametaCell-owned facility. The head of our manufacturing team, Vladimir Melnikov, brings 25 years of experience with manufacturing aseptic therapies, and his expertise with bringing therapies through regulatory approvals from the manufacturing perspective. The team under Vladimir's leadership has not only developed the required processes for commercial manufacturing, but we have also validated those processes. Upon FDA approval of Omidibacill, our manufacturing facility is ready. Knowing a transplant is the only potential curative option for patients with certain hematologic malignancies, January 30th, 2023 will be a very important day for patients, transplanters, and Gametacel. We have the opportunity upon potential FDA approval to provide access to Amadubacel and address the unmet needs that we consistently hear from transplanters. We have a clear understanding of the unmet needs, a well-thought-out launch strategy, and we are now executing on our launch plan. We have a great experienced launch leadership team in place that knows the importance of providing a life-enhancing therapy like Amadubacil to patients, realizing the sense of urgency and the important role that Amadubacil can play for patients with blood cancers, and how Amadubacil can change the way patients are treated in the future. I would now like to turn the call over to Shai to review our financial results.
spk00: Thank you, Michelle, and good morning, everyone. Today, I will summarize our financial results for the third quarter of 2022. As of September 30th, 2022, our total cash position, including the recent $20 million equity financing we closed on September 30th, was approximately $61.3 million compared to $96 million as of December 31st, 2021. Research and development expenses for the quarter were $9.9 million compared to $11.7 million in the same quarter last year. The decrease was mainly due to the $1.6 million decrease in clinical activities related to the conclusion of our Phase III clinical trial and a decrease of $0.2 million in the GDA201 clinical program. Commercial expenses for the quarter were $2.8 million compared to $5.8 million in the third quarter of 2021. The decrease was primarily due to a $2.5 million decrease in launch readiness activities and a $0.6 million decrease in ad count related expenses. General and administrative expenses were $4.4 million in the third quarter of 2022 compared to $5 million in the same period in 2021. The decrease was mainly driven by a $0.6 million decrease in professional services expenses and a $0.3 million increase in ad count related expenses. Finance expenses net were $0.7 million both in the third quarter of 22 and 21 with no material changes. Net loss for the third quarter of 2022 was $17.8 million compared to a net loss of $23.2 million in the third quarter of last year. We anticipate that our current total cash position will support our ongoing operating activities into mid-2023, excluding the cost of commercializing Omidubicel beyond the initial launch, which is now underway. Our 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 Abby.
spk03: Thank you, Shai. Before I turn the call over to the operator for questions, I would like to reiterate how excited we are to be in this position, on the verge of a turning point for the company as we rapidly approach our PDUFA date and target action date of January 30, 2023. We believe in the compelling value proposition of AmiDubiseau and the potential it has to transform patients' lives. We continue to advance our NAM-enabled NK cell therapy pipeline led by GDA201. With all that we've accomplished this quarter and this year overall, we will enter 2023 with strong momentum and poise now more than ever to deliver long-term growth for all of our stakeholders. Now, let's open the call for questions. Shannon?
spk02: Thank you. As a reminder, to ask a question, you will need to press star 11 on your telephone. Please stand by while we compile the Q&A roster. Our first question comes from the line of Edward Tintoff with Piper Sandler. Your line is now open.
spk06: Great. Thank you very much, and congrats on all the progress. Looking forward to the upcoming PDUCA date, obviously, and really appreciate all the detail with respect to the launch preparations. I'm trying to get a sense for what kind of sales force you need to field in order to cover those top 70 centers. And, you know, how many of those centers have been involved in the clinical trials and already have Omidubacill experience? I'm trying to get a sense for sort of what kind of cadence to expect from some of those top centers. Thanks so much.
spk03: Great. Michelle, do you want to take that?
spk05: Yeah, absolutely. Good morning, Ted. Ted, in regards to the first part of your question, so we know there's approximately 70 transplant centers that are responsible for about 80% of the transplants. And we already have relationships with many of those centers, including both their clinical experience with Amadubacil through clinical trials and our EAP program. We had previously discussed, and our approach has not changed, that we see up to 25 commercial account managers to address the footprint of the transplant centers in the United States. And in addition, Roni and her team will have medical science liaisons also. In regards to your second part of the question, let me turn to Ronit to discuss the number of centers in the United States that were part of our Phase III study.
spk12: Sure. Hi, Ted. So in our clinical trials for Omidubacill, which included both the Phase III trial and our previous trials in non-hematologic malignancies, a total of about 19 centers in the U.S. who've had some experience with Omidubacill over the past several years.
spk06: Great. That's really helpful. And just to kind of get a sense, so I'm trying to get a sense sort of how many transplants that maybe fit within this 20% to 25% or just how many transplants are performed on a monthly or quarterly basis. Just, again, sort of the cadence of how many transplants get performed in those sender stakes.
spk05: So, Ted, I'll talk about the 20% to 25%. So we talk about that as peak market share, where we would estimate the addressable transplant population to be approximately 10,000 to 11,000 patients each year.
spk06: That's helpful. I can work it out from there. Thanks so much.
spk05: Perfect. Thank you, Ted.
spk02: Thank you. Our next question comes from the line of Jonathan Miller with Evercore ISI. Your line is now open.
spk08: Hi, guys. Thanks for taking my question. And also I'll echo the congrats on the progress and the upcoming PDUFA. I'd love to dig in a little bit into what you mean when you say the runway, cash runway will cover initial launch. What are some of the things you're thinking about there in terms of timeline of launch, or maybe there are a particular set of goals that you think you can cover? Obviously, Michelle's been doing a lot of work commercially here already, but can you talk about what you still have to do that's covered by your current cash runway and what you would need additional financing to reach after the initial launch phase is over?
spk03: Maybe you start, and then, Michelle, you can chime in.
spk00: Hey, John, good morning. Thank you for your question. So, as we said, our cash runway is still mid-2023, and this excluding the commercialization of Omidubicel, of course, beyond the initial launch activity that's already underway. This cash runway guidance does not take into account, and it's a very important point, the iBridge commitment letter of $25 million, which I can say as a side note, both parties are working very closely to bring this to the finish line soon. Launch activities already being started, and we are bringing and we are focusing to bring Omidubicel to patients. We cannot comment today on current and future financing activities, but as you can imagine, as any biotech company, we are always evaluating our cash needs. and continue to assess all financing options and tools to support our corporate strategy. We are committed for this product and committed to ensuring there is a solid financial runway for this company. Michelle, do you want to elaborate?
spk05: Yeah, absolutely. And good morning, John. I'll start off with a very important point. We feel strongly at Comita that upon FDA approval, we want to make sure that patients can have access to Amaduba cells. One of the very, very important parts of that is manufacturing. So the positive news is our manufacturing facility is ready. We've been manufacturing clinical batches, and we have the production and the quality and supply chain individuals in place for time of launch. What we are focused on now, as Shai alluded to with the initial launch plan, is The initial hiring of personnel, predominantly in the United States, that will be interfacing with transplant centers. We have our respective commercial medical affairs leadership teams in place, but now the initial part of the launch planning is the hiring and also some operating expenses associated with the initial launch. We do anticipate in 2023 that we'll be ramping up transplant centers because we do know that transplant centers do need a period of time after FDA approval to develop their processes for cell therapies once they see final prescribing information. So what we also will then do is continue to ramp up our hiring as transplant centers ramp up. I'll clarify my answer to Ted's question because I mentioned up to 25 commercial account managers will ramp up the hiring of those commercial account managers over the next few quarters. John, let me turn back to you, John, to see if you have any follow-up questions.
spk08: Sure, thanks. That's very helpful. I guess then beyond OMI, maybe on GDA201, do you have any more color on progress there? Are you still expecting to move the Phase 2 portion next year? And do you have any plan to present Phase 1 data before that time and maybe in time for folks to get a look at it before any potential financing?
spk12: Ronit, over to you. Thanks, John. So the GDA201 study is proceeding in the phase one portion. The dose escalation is ongoing. Patients are being enrolled as prescribed by the protocol with the full evaluation of a dose-limiting toxicity period of 28 days between each patient. And so you can imagine that each patient is being evaluated over about a 28-day period, after which time the dose-limiting toxicities can be evaluated and then dose escalation can proceed. This will take us as we escalate through four potential dose levels through next year. And we don't have any particular plans to share the outcomes of phase one portion at this point, but we will continue to examine the results of that portion and see if there is an opportunity to share data before the end of 2023 for the phase As soon as our recommended Phase 2 dose is identified, we will then be able to start our Phase 2. We've already started to identify sites for Phase 2 and begin the operational activities to allow the Phase 2 to proceed as soon as the dose is identified.
spk08: Okay, thanks very much.
spk02: Thank you. Our next question comes from the line of Jason Butler with JMP Securities. Your line is now open.
spk01: Hi, it's Ryan for Jason. Thanks for taking our questions. I guess just to follow up on the last question, it sounds like the Phase 2 starts going to be a 2024 event. Is that a likely assumption?
spk12: We haven't pinpointed exactly when the Phase 2 will start. It really depends on the identification of the recommended Phase 2 dose. If the dose occurs at a lower dose level than the highest dose, then it could occur sooner. But it's driven by our progression through the dose escalation portion. In any case, most of next year will be spent in the phase one dose escalation portion, after which the phase two will begin.
spk01: Okay, got it. Thank you. Then, Omadou Bassel, how are you guys thinking about potentially partnering to supplement your own commercial efforts and then what's the uptake then in the expanded access program?
spk03: Thanks, Jason. What I would say on partnering is that we believe in commercializing AmiDubuCell in the U.S. and we're very focused on getting ready for that potential approval. We believe that there is an opportunity for AmiDubuCell access to expand beyond the U.S. into certain target ex-U.S. markets and we're actively engaging in exploratory conversations with potential partners to expand that access internationally.
spk01: And then expanded access? Renee?
spk12: Happy to take that one. So our expanded access program is open at six sites in the U.S., and it allows us to continue to treat patients until such time as the potential approval takes place. There's a continual interest in enrolling patients in that study amongst the investigators who are involved in the study. And there's a steady stream of patients, including patients from all those sites. And we will continue to treat those patients until we have commercialization potentially.
spk01: Okay, great. And then on the NK pipeline, I guess if the OMA-DUBA cell launch advances as you guys expect it to, when do you think you'll be in a position to nominate an NK pipeline candidate for an IND? Thanks.
spk03: What I would say is it will be a combination of information that we gather through a market landscape assessment and other things as well as the data that we're generating on that early pipeline. So at this point, we're not prepared to guide to a specific date, just that we're intending to move forward and identify a next IMD candidate at some point in the near future.
spk12: And what I will say is that the research is continuing and that we continue to be encouraged by all of the candidates that are currently in the pipeline with some very exciting and interesting research going on in our development organization.
spk02: Great. Thank you. Thank you. Our next question comes from the line of Gil Blum with Needham & Company. Your line is now open.
spk07: Good morning. Just a couple of questions from us. So on your ASH abstract, there's a clear advantage in the neutrophil engraftment over the other modalities. However, platelet engraftment seems more middling. How could this finding influence the potential length of hospitalization in these patients?
spk12: Thanks, Gil. So, yes, platelet engraftment is known to lag behind neutrophil engraftment. In general, platelet engraftment does lag behind, and it usually is not a factor in hospitalization because patients can receive platelet transfusions as outpatients. So generally what happens is that after neutrophil engraftment takes place, almost universally their platelets have not engrafted yet. And if they reach a certain low level while they're being followed closely as outpatients, then they get a platelet transfusion as an outpatient. I will say we've noticed that in our clinical trials and have not had any sort of clinical sequelae from a lag in platelet engraftment. It's actually expected in all types of donor transplants. Platelets take a little bit longer.
spk07: Okay. Thank you for that clarification. A separate question. The NAMM technology appears to improve metabolism of cells broadly. Have you guys given any thought on application outside of what you're currently pursuing in NK cells and AMI, particularly in a potential partnership?
spk12: There has been interest from a number of different academic and other organizations in using our NAMM technology. for other types of cells. And those are discussions that we have on a case-by-case basis and may pursue at some point. We're focused on the work that we're doing now for the NK cells, which we believe are very promising, and obviously omeduva cell. But there is certainly a scientific interest and research interest in looking at other cell types. Some of our own work in the past also indicates that other cell types are amenable to the NAMM So stay tuned. Yes, this is something of scientific interest, and we hope to be able to get to that when the time is right.
spk07: All right. Thank you for taking our questions.
spk02: Thank you. As a reminder, to ask a question at this time, please press star 1-1 on your touch-tone telephone. Our next question comes from the line of Mark Breedenbach with Oppenheimer. Your line is now open.
spk09: Hi, good morning. This is Jacqueline from Alpenheimer. Our first question is in the ASH abstract. It looks like there was a higher rate of acute GVHD for the cell versus the other graph sources. But at least most of it looks like it was a group two. Could you please comment on what factors might be driving the discrepancy? Thank you.
spk11: Bernie?
spk12: Absolutely. So we did notice that there was more grade 2 GVHD in the umbilical cord, in the omoduga cell group than in the other transplant groups. And interestingly, we believe that it's probably related to the earlier engraftment because GVHD is a process by which the donor cells recognize the host or the patient cell. And so that doesn't happen until the immune system comes back. The good thing about what we're seeing is that it's mild GVHD, it's grade two. When you look at grade three and four, those are the same throughout. So we don't believe that there is a concern here. We actually think it's a reflection of the recovery of the immune system, which with omadubacil is not only faster, but it also is actually really durable based on the results of our immune reconstitution data.
spk09: Great, thanks. Another question is on the EU regulatory path. Is there any updated thinking about an EU regulatory path for a meduva cell?
spk11: I'm sorry, Jacqueline. I didn't hear your exact question.
spk09: Yeah, sorry. So is there any updated thinking about a European regulatory path for meduva cells?
spk12: I think you asked if there's any update. Is that what you asked? Updated thinking. Updated thinking, yes. Thank you. Well, we're focused on working with FDA to bring this therapy to patients in the U.S., And we are excited and interested in opportunities internationally based on the data. And the fact is that we enrolled many patients from Europe in our clinical trials. And so we have international sites and investigators that are interested. So we will strongly work on that, but we don't have any updates at this point about the regulatory interactions with the EMA.
spk09: All right. Thank you. Thanks for taking our questions.
spk02: Thank you. I'm currently showing no further questions at this time. I'd like to hand the call back over to Abby Jenkins for closing remarks.
spk03: Our leadership team will be available after the call if there are any opportunities for follow-up discussions. We'll keep you current on all of our developments, and we thank you again for your interest and support in Gimme to Sell. Thank you, everyone, for joining us on today's call.
spk02: This concludes today's conference call. Thank you for your participation. You may now disconnect.
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