Gamida Cell Ltd (GMDA) 2020 Q3 法說會逐字稿

Welcome to Gamida Cell's 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 Gamida Cell's request. Now I would like to introduce your host for today's conference, Mr. Josh Hamermesh, Chief Business Officer. Please go ahead.

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.gamidacell.com. Here with me on our call today is Julian Adams, Chief Executive Officer; Ronit Simantov, Chief Medical Officer; and Shai Lankry, Chief Financial Officer. Michele Korfin, our Chief Operating Officer and Chief Commercial Officer; and Tracey Lodie, 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 Factors section of our Form 20-Fand in other filings that Gamida Cell makes with the SEC from time to time. These forward-looking statements represent our views only as of today and we caution you that we may not update them in the future, whether as a result of new information, future events or otherwise.

Now I'd like to turn the call over to Julian.

Thank you, Josh. And thanks to everyone for joining us this morning.

At Gamida Cell, we are committed to developing cell therapies with the potential to provide cures for patients with blood cancers and rare serious hematologic diseases. We have continued to make very strong progress over the past few months, with both of our programs, omidubicel, which could be the first FDA-approved engineered graft source for bone marrow transplant, and GDA-201, 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 omidubicel to patients in the commercial setting.

Starting with Oma Duba cell. In October, we were thrilled to report our global randomized Phase III study of omidubicel met all 3 secondary endpoints related to platelet engraftment, infections and hospitalizations. These results strengthen our confidence in the clinical potential for omidubicel 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 omidubicel on a rolling basis in the fourth quarter, which positions us for a potential launch in the second half of 2021. We are also making good progress with other key launch activities required to bring omidubicel to patients following potential FDA approval.

Our cell expansion platform also led to the generation of GDA-201, 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 GDA-201 to address the limitations of NK cells. With GDA-201, 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 are continuing to plan for our next trial, which will be a Phase I/II multi-centered study in patients with advanced lymphoma. We have developed a cryopreserved formulation of GDA-201 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 a registrational trial to 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 Jamieson to the role of Senior Vice President Information Technologies. Steve brings 25 years of IT experience, primarily in the life sciences industry, including roles at Verastem, Infinity, Ariad and OSI. Additionally, this week, Rocio Manghani joined Gamida 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 YESCARTA. We are excited to welcome Steve and Rocio to the Gamida Cell team and look forward to their contributions as we look toward a potential omidubicel 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 on site. We continue to watch our timelines very carefully. Today, our anticipated milestones for both omidubicel and GDA-201 are unchanged. We continue to expect to initiate the BLA submission for omidubicel 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 GDA-201 to enable our multicenter Phase I/II 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 Simantov, our Chief Medical Officer, to provide a further update on omidubicel and GDA-201. Ronit?

Thank you Julian, and good morning, everyone. This morning, I'll review our clinical programs and highlight our recently published ASH abstract describing data from the study of omidubicel in patients with severe aplastic anemia and for GDA-201 in patients with non-Hodgkins lymphoma.

As Julian noted, this year, we have reported that our global Phase III study of omidubicel in 125 patients with hematologic malignancies met the primary endpoint and all 3 secondary endpoints. We are proud of this rigorous well-executed trial. And we truly appreciate the support of our collaborators and 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 transplant, signifying how quickly the stem cell patients received became established and began to make healthy new cells. The study showed that omidubicel was generally well tolerated. In the intent-to-treat analysis, the median time to neutrophil engraftment was 12 days in patients randomized omidubicel 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 3 prespecified secondary endpoints of the study demonstrated a statistically significant improvement among patients randomized omidubicel versus the comparator group. Specifically, secondary the endpoints were platelet engraftment by day 42, incidents of grade 2 or grade 3 bacterial or invasive fungal infections, and the number of days alive and out of the hospital in the first 100 days following transplant. Rapid engraftment immune recovery, reducing infections and shortening hospitalizations are all clinically meaningful outcomes, valued by physicians, patients and their family. 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 III 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 omidubicel could be an important graft option for any patient who does not have a suitable match donor. We believe omidubicel has potential beyond hematologic malignancies, and are evaluating omidubicel 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 Institute of Health. We previously reported data from an initial cohort of 3 patients who successfully underwent a reduced-intensity conditioning regimen and received the stem cell transplant consisting of omidubicel plus haploidentical stem cell graft. Dr. Childs then initiated a second cohort of patients to evaluate omidubicel cell as a stand-alone graft. Initial data from this cohort will be presented at ASH next month.

The abstract published last week included data from the 3 patients in cohort 1 and 5 patients in cohort 2. The data showed that omidubicel was generally well tolerated and led to rapid sustained engraftment. With a median follow-up of 10 months, 7 of the 8 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 omidubicel to be used in non-malignant bone marrow diseases and lends further support to evaluate omidubicel more broadly using reduced-intensity conditioning, which could make omidubicel 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 post-presentation at ASH on Saturday, December 5.

In addition to omidubicel, we are advancing our NK program, GDA-201. 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 3 increasing doses of GDA-201 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. Veronika Bachanova at the University of Minnesota. The abstract submitted to ASH, which was accepted for oral presentation, 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 GDA-201 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 GDA-201 and rituximab. Histologies included 8 patients with diffuse large B-cell lymphoma, 6 patients with follicular lymphoma and 1 patient with mantle cell lymphoma. Patients had a median of 3 prior lines of therapy, with a range of 1 to 8 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 1 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, 4 patients underwent retreatment with GDA-201 without lymphodepleting chemotherapy, which likely contributed to deepening of responses in 2 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-up for 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 this 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.

Thank you, Ronit, and good morning, everyone.

Today, I will summarize our financial results for the third quarter of 2020.

As of September 30, 2020, we had total cash and cash equivalents of $73.3 million compared to $55.4 million as of December 31, 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 advances of GDA-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 noncash income resulting from revaluation of warrants owned by the company's 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 million 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 run rate 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 Julian.

Thanks, Shai.

We are committed to finding cures for patients with hematologic malignancies and blood disorders, and we are excited about the opportunities ahead. With omidubicel, we expect to present our Phase III data and initiate our rolling BLA submission by the end of the year. With GDA-201, 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 Gamida Cell as we prepare for a potential approval and launch of omidubicel in the U.S.

Now we will open the call for questions. Operator?

(Operator Instructions)

Our first question comes from Jonathan Miller with Evercore.

And congrats on all the progress. I'd love to start with omidubicel. It clearly looks superior to standard cord blood and very intriguing they sort of . Well, the trial was against -- cord blood obviously had [not] those other sources. So I guess in your conversations with docs and payors, how have they characterized omidubicel'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 GDA-201, obviously, the data in the ASH abstract looks very interesting, especially in follicular and broadly 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 hemo malignancy space there outside of NHL?

So let me ask Ronit to cover the first part of your question. How do physicians view our data with omidubicel compared with other transplant modalities? And then, Michele, you could comment on the potential launch activities and introduction of omidubicel to the marketplace.

Thanks, Julian, and I will start with how physicians are reacting to omidubicel. So rapid neutrophil 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 omidubicel is potentially in an attractive graft source for patients who don't have a suitably matched donor from another source.

Michele?

Excellent. Thank you. Thank you, Ronit. Thank you for the question. So I'll reiterate what Ronit has said based on our discussions with physician. If the patient does not have access to a match related donor, there is the potential for them to be an omidubicel candidate. Physicians and payors have been very encouraged by the clinical profile to date for omidubicel across efficacy, safety and the ability to provide the patient. With omidubicel, in the clinical trial, our experience was 4 weeks from the time of identification of the cord for the manufacturing until return of omidubicel. So those 3 aspects, efficacy, safety and the logistics, have received very encouraging feedback from physicians. So there is an opportunity for omidubicel all current treatment modalities from a graft source standpoint.

Now in regards to launch, we're very excited, as Julian indicated, to begin to add exceptional personnel to our commercial team, including Rocio Manghani joining us this week. So we're building up the team in expectation of a potential launch in the second half of 2021. Our launch of omidubicel is absolutely going to be focused on assuring a positive experience for the transplant center and the patient. So hence, we have 4 key tenants that we're working on to prepare for the launch. Preparing to educate the transplant centers, so they understand the omidubicel clinical profile in the appropriate patients. Educating the payors. We've been encouraged by the initial payor 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 4 of those areas. So thank you for the question.

And related to GDA-201, let me ask Ronit again to comment on the clinical experience so far. And then I'll ask Tracey to talk about the potential for a combination with other monoclonal antibodies.

Thanks. So in terms of GDA-201, 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 GDA-201 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, alternative tracing.

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, elotuzumab, 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.

Our next question comes from Ted Tenthoff with Piper Sandler.

Again, really exciting time for the company for 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 (inaudible) of the pandemic. And do you anticipate this would require a [o dot ad comm ?

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 III study, and we're seeking full approval, not accelerated approval. But we will be seeking a priority review which, as you know, is a 6-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 end points, with statistical significance.

Our next question comes from Jason Butler with JMP Securities.

Congrats on all the progress. First one, omidubicel. Just in terms of the aplastic anemia market dynamics, can you just walk us through what the patient options are today and how omidubicel 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 to what extent you're through the work that needs to be done to submit the IND?

Ronit, could you discuss aplastic anemia?

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 in 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 infection, 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 they subsist on supportive therapy but there's really no opportunity for here other than a bone marrow transplant. So what omidubicel 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 were published in the ASH sector. 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 marrows got this immune system and there's just a difficulty in supporting engraftment. And so the fact that a single unit of omidubicel support a sustained and rapid engraftment is actually a very, very positive sign. So what omidubicel can offer for these patients is a potential for cure that may just not exist for them currently. Severe aplastic anemia a rare disease, and the numbers of patients in the U.S. are measured in the 100s.

Thank you. Tracey, can you talk about the GDA-201 progress in our manufacturing?

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 in Minnesota. So at omidubicel, we've made tremendous progress in making a cryopreserved formulation. So our intent next year 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 Gamida 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.

Our next question comes from Gregory Renza with RBC Capital Markets.

Congrats on the progress. Just to build on some earlier questioning just around the progress of the BLA filing. What do you see as the gating factors or a critical path to this? And perhaps for each margin? Are there any components that you're still waiting for in terms of data or information? I'm 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?

Yes. So let me cover the sort of the cadence of filing. Our nonclinical module is ready, and that's -- we anticipate beginning the filing with that particular module. And that will be followed early next year by the clinical module. And lastly, the manufacturing module, Module 3, the CMC section will complete within a 6-month time frame, the full filing of the BLA.

Got you. Very helpful. And then, Julian, we've spoken about this before, but certainly get questions on the intellectual property of omidubicel. I just wanted to see if you could just take an opportunity to provide just some color on your view of the protection you have there with respect to the product and the process?

Greg, I'd be happy to. Our patent expiry, we have quite a number of patents, but our longest patent expiry's out to 2038. But importantly, because we have orphan drug status, we have 12 years exclusivity in the U.S. for a BLA and 10 years in Europe. And finally, we have many trade secrets that are not in the public domain. And because omidubicel is a personalized manufacturing where omidubicel 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.

Our next question comes from Vernon Bernardino with H.C. Wainwright.

Congrats on the progress and the very strong Phase III 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 the -- any effects of the pandemic on donation of our core blood units? And if there's any effect, then 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 omidubicel commercialization has begun?

So let me begin by discussing the pandemic, and then I'll turn it over to Michele 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 a -- in 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 are all cryopreserved and shipping with routine, and we never experienced a delay based on the pandemic. And Michele, maybe you could comment on the sort of commercial supply agreement with Be The Match.

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. They partnered 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 a omidubicel upon potential FDA approval.

Next question comes from Gil Blum with Needham & Company.

This is Gil on for Chad. 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?

The key feature of next year's trial will be, obviously, within off-the-shelf cryopreserved product, and it will be a multicenter study. It will be designed as a Phase I/II study. And let me ask Ronit to talk about how we're going to enroll different histologies, different cohorts of patients.

Thanks, Julian, and thanks. So I think -- yes, I think you pretty much captured this. So the main features are that it will be a multicenter study because the cryopreservation will allow us to ship the product to different sites, much as we did with omidubicel. 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 that's -- those are the broad strokes in terms of the expectations for that study.

All right. And furthermore, on GDA-201, could you put in the context the ability to use multiple doses of cellular therapy, especially considering standard of care?

Ronit?

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 GDA-201 in patients for 1 of 2 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 4 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, for us, we think that's an interesting opportunity to administer with multiple doses, and we will explore that in further studies.

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. That's -- 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're -- we think is pertinent to the development of GDA-201.

That makes sense. One last one on omidubicel. You guys did have a press release about beta match. Going, forward and I was thinking about the market as it expands, do you think your relationship with Be The Match will be sufficient? Or are you guys going to have to look at other sources of cord blood?

Michele?

Thank you, Gil, for the question. So as I mentioned prior, we're very excited about the partnership with. They are very, very much a partner, not only now for us, but also for the transplant centers. We 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.

I might add to that, exuberantly, 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 omidubicel continues to serve patients.

(Operator Instructions) Our next question is from Mark Breidenbach with Oppenheimer.

2 questions on omidubicel. With respect to the BLA filing, I'm just curious if you've reached an alignment with the FDA regarding the contents of the CMC analytical package, especially given that this is a first of its kind of product that the FDA would have to review?

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.

Okay. And in your upcoming presentation of data from the Phase III 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?

So, Ronit, would you care to comment?

Happy to comment on that. So in our Phase I/II study, rapid neutrophil engraftment was associated with lower treatment-related mortality in the 36 patients in the single-arm Phase I/II. 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, efficacy and safety data for the study.

Okay. But no specific thresholds that -- do you think it's important to stay below?

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.

And we're currently showing no further questions at this time. I'd like to turn the call back over to Julian Adams for closing remarks.

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?

That concludes today's call. You may now disconnect.