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spk02: Ladies and gentlemen, thank you for standing by. Welcome to GametaSales conference call for the fourth quarter and full year 2021 financial results. My name is Howard, and I'll be your operator for today's call. Please be advised that this call is being recorded at GametaSales request. Now I would like to introduce your host for today's conference, Heather DeVecchia, GametaSales Chief of Staff. Please go ahead.
spk01: Thank you, Howard, 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 fourth quarter and full year of 2021. 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. Here with me on our call today are Julian Adams, Chief Executive Officer, Ronit Simantoff, our Chief Medical Officer and Chief Scientific Officer, Michelle Corfin, our Chief Operating Officer and Chief Commercial Officer, and Shai Langrey, 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 Amidubicel to the FDA, commercialization planning efforts, the potentially life-saving or curative therapeutic and commercial potential of Amidubicel, 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 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 report on Form 20F and other filings that we make 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 Julian.
spk04: Thank you, Heather, and thanks to everyone for joining us this morning. Before I begin, I want to take a moment to acknowledge the major events in the Ukraine that are impacting the world globally over the past few weeks. While we don't have any operations in the Ukraine or Russia and expect minimal impact to our operations, as global citizens, our thoughts are with all those affected. At GametaCell, we are incredibly proud to be part of the significant advancements occurring in the field of cell therapy. as it continues to grow and progress towards cures for patients in need. 2021 was an important year for us as we progressed our two clinical stage NAM-enabled cell therapy programs, Omadubacil and GDA201, both of which hold significant potential to benefit patients suffering from hematologic malignancies and serious blood disorders. We further expanded our NAM-enabled platform with the addition of genetically modified NAM-enabled NK cell constructs, allowing us to leverage our expertise in NK cells to potentially treat various hematologic malignancies and solid tumors. I will start today's call by commenting on our lead program, Omadubacil, which has breakthrough therapy designation and the potential to be the first FDA-approved cell therapy for stem cell transplant. Throughout 2021, we announced several data presentations which contribute to the evidence for Omidubacil's potential efficacy, and also the positive health economic impact of Omidubacil, with data demonstrating a reduction in healthcare resource utilization. As Ronit will describe later on in this call, This reduction in healthcare resource utilization is very important for patients, hospitals, and payers. The robust data that we have generated in support of the potential for Omidubacill led us to our most recent and exciting milestone last month, namely the initiation of the rolling BLA for Omidubacill with the submission of our non-clinical module following the receipt the recent receipt of positive CMC-focused type B meeting correspondence from the FDA. In this correspondence, the FDA confirmed analytical comparability between our wholly-owned commercial manufacturing facility in Israel to the Omiduba cell batches that were produced at the clinical manufacturing sites for the Phase III study. This month, we also submitted our clinical module to the FDA We are on track to submit the remaining modules and complete the full BLA submission by the end of the second quarter. We are thrilled to have reached this inflection point and look forward to working with the FDA to bring Omidubacill to patients as soon as possible. Beyond Omidubacill, we are also developing our NAM-enabled NK cell pipeline, which is led by GDA201, GDA201 leverages our proprietary NAMM technology platform to expand natural killer cells to enhance their functionality, direct tumor cell killing properties, and antibody-dependent cellular cytotoxicity, or ADCC. We are highly encouraged by the potential of GDA201, which has produced truly remarkable results in a Phase I investigator-sponsored study, where we have seen very high and complete durable responses in both follicular lymphoma and diffuse large B-cell lymphoma. In September 2021, we submitted an IND application to the FDA for a phase 1-2 trial with a cryopreserved formulation of GDA201 in patients with diffuse large B-cell lymphoma and follicular lymphoma. Following this submission, we were placed on clinical hold in November of 21 prior to patients being dosed since the FDA had questions about donor eligibility procedures and assay qualification. We are actively working with the FDA to address their comments to enable an IND acceptance and study initiation. This year, we plan to initiate dosing in this Phase I-II study. Moving to our newest product candidates, our genetically modified NAMNK-enabled cell therapy programs. Last year, we were excited to establish a broad pipeline of NK product candidates that utilize CAR and CRISPR-mediated strategies to increase targeting potency and persistence against hematologic malignancies and solid tumors. Robust preclinical evidence gives us the confidence that these new NAM-enabled NK product candidates hold promise as potentially curative therapies for both hematologic cancers and solid tumors. Furthermore, we announced a research collaboration with the Dana-Farber Cancer Institute for GDA601, a CD38 CRISPR knockout combined with a CD38 CAR-NK cell construct that has demonstrated promising preclinical results against multiple myeloma cell lines. We are excited to leverage the expertise of researchers at Dana-Farber to study the in vitro NK cell killing activity of GD8601 in multiple myeloma. Throughout 2022, we plan to execute preclinical proof of concept studies for these genetically modified NK therapeutic targets. And by the end of 2022, select a pipeline candidate for IND enabling studies. With the progress we achieved in 2021, we are encouraged about what the future holds for Gametacel in 22. And of course, this wouldn't be possible without our employees whose dedication is focused on advancing therapies to help address unmet needs for patients. I am grateful for and proud of their continued determination and focus on patients, which has brought us to where we are today. Before turning the call over to Ronit, I would like to take a moment to congratulate her on her recent promotion, now assuming the role of not only Chief Medical Officer, but also Chief Scientific Officer. We at Gameta thank you all for your contributions thus far, and look forward to your continued work towards division with your expanded role. Ronit?
spk03: Thanks, Jillian, and good morning, everyone. Thank you for joining us on our call. In 2021, we had the opportunity to present a broad range of scientific and clinical data on Omidubasel, tying together preclinical and translational analyses with clinical data, patient experience, long-term outcomes, and value to the healthcare system. In preclinical presentations in 2021, We demonstrated that our nicotinamide or NAM platform generates a unique cellular phenotype through modulation of metabolic pathways, leading to highly functional and potent stem cells. In translational data, we showed that patients treated with Omiduba cell had robust functional reconstitution of T and B cell subsets, dendritic cells, and natural killer cells in the days and weeks following transplants. The translational data provided mechanistic support for the clinical results of our global phase three randomized trial, the results of which were published in October 2021, demonstrating rapid somatopoietic recovery, significantly decreased infections, and shorter duration of hospitalization for patients transplanted with omadubacil. Of note, the patients in our clinical trial were in critical need of allogeneic transplant for hematologic malignancies. with few or no alternatives available. Looking back at the totality of our experience with Omidubacil, we presented follow-up data from over 10 years of clinical studies demonstrating long-term sustainable hematopoiesis and immune competence in patients. We then went on to show that Omidubacil could decrease the cost to the healthcare system, as patients treated with Omidubacil had reduced healthcare resource utilization, including blood transfusions, outpatient procedures, and time in intensive care units. At next month's transplantation and cellular therapy meetings in Salt Lake City, we have eight abstracts accepted and plan to report new data focused on patient outcomes and health-related quality of life. Taken together, we have continued to add to the body of evidence supporting the mechanism of action, clinical activity, patient experience, and value of Omidubacil as a potential life-saving therapy. Moreover, we are confident in the scientific and clinical package we have assembled in our efforts to make Omidubacill available to patients in need of a Hamad-poietic stem cell transplant. We also made considerable progress in our NAM-based natural killer cell platform. We presented preclinical data characterizing the properties of GDA201. We showed that NAM mediates a set of cellular processes expanding NK cells while down-regulating differentiation cellular stress, and exhaustion pathways that are typically activated in culture. GDA201 cells are characterized by a rejuvenated NK phenotype similar to cytokine-induced memory-like NK cells and were shown to be highly cytotoxic in in vitro and in vivo assays. These findings were supported by the clinical data reported in the Phase I investigator-sponsored study at the University of Minnesota At ASH, we presented two-year follow-up data in patients with non-Hodgkin lymphoma, showing an overall response rate of 74%, including 13 out of 14 complete responses, with a median duration of response of 16 months, 78% overall survival and toxicities in line with those reported previously for GDA201. We've now developed a cryopreserved formulation and plan to initiate a multicenter company-sponsored study this year in patients with follicular lymphoma and diffuse large B-cell lymphoma. In addition, we reported that we have expanded upon our NAM platform to develop genetically modified NK cells, GDA301, GDA501, and GDA601, each of which is proceeding with preclinical proof-of-concept studies as we advance towards IND-enabling studies. Overall, 2021 was a tremendously productive year in research and development and clinical research as we look towards continuing to validate the therapeutic potential of our product candidates. I will now turn the call over to Michelle, who will talk more about our launch readiness for Amadou Basel. Michelle?
spk00: Thank you, Roni, and good morning, everyone. This year has started off strong already, as last month we initiated our rolling BLA submission for Amadou Basel. We continue to make excellent progress advancing our Gameta-owned manufacturing facility in Israel. The FDA acknowledgment of the analytical comparability from our planned Gameta commercial facility as compared to the facility used for the Phase III study was an important milestone as we work towards advancing Amadubacil. The commercial opportunity for Amadubacil is extremely encouraging based on our continued assessment of market insights, and we continue to focus on our commercial preparation. In parallel with our rolling BLA submission, we are additionally assessing strategic alternatives for the potential commercialization of Amadubacil, including potentially partnerships to expand the reach of Amadubacil. Amadubacil has the potential to be the first FDA-approved advanced cell therapy product for allogeneic stem cell transplant in patients with hematologic malignancies. For patients with hematologic malignancies that are deemed eligible for an allogeneic stem cell transplant, the procedure is their best chance for a potential cure. In the US, there are approximately 8,000 patients above the age of 12 with hematologic malignancies who undergo an allogeneic stem cell transplant each year. Unfortunately, though, there are approximately 1,200 patients each year who are also ages 12 and up with hematologic malignancies. who are deemed eligible for an allogeneic stem cell transplant but cannot find an appropriate donor. Unfortunately, we observe 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 only have about a 20% chance of finding a match. For patients in need of a stem cell transplant, this is often their only chance for a cure. We are diligently working to advance Amadubacel and to expand access to patients. Based on encouraging clinical data and the less stringent matching criteria, transplanter feedback indicates that Amadubacel has the potential to improve outcomes for allogeneic stem cell transplant patients compared to other donor sources and expand access for patients who cannot find a suitable donor. In the U.S., there are approximately 200 transplant centers that perform allogeneic stem cell transplants. 70 of those centers conduct about 80% of the transplants. And our medical team continues to engage with those top 70 centers through one-on-one meetings and at medical conferences. We also continue to assess market insights from transplanters in the U.S. Based on extensive market research, we see the potential for Amadubacil to treat approximately 2,000 to 2,500 patients per year upon reaching peak market share. This equates to approximately 20% to 25% market share of the addressable population. Our market access team is also actively engaging in meetings with national and regional commercial payers, and the feedback continues to be very encouraging. Payers recognize the overall value proposition, including the strength of the Amadubasol clinical data and the health economic data we have published to date. In summary, we are excited by the potential of amadubacil to be the first FDA-approved cell therapy for allogeneic stem cell transplant, and we are also encouraged by the clinical data and feedback from physicians and payers. I will now turn the call over to Shai to review our financial results.
spk06: Thank you, Michelle, and good morning, everyone. Today, I will summarize our financial results for the full year of 2021. As of December 31, 2021, our total cash position was approximately $96 million compared to $127.2 million as of December 31 of last year. Research and development expenses for the year were $50.2 million compared to $38.9 million for the same period in 2020. The increase was primarily due to a $5.4 million increase in omnidivisible commercial manufacturing readiness activities and the advancements of our NK programs, as well as an increase of $5.9 million in broadening our scientific capabilities and talent. Commercial expenses in 2021 were $20 million, compared to $8.9 million in 2020. The increase was mainly due to a $6.5 million increase in commercial readiness expenses and a $4.6 million increase in ad count within our commercial organization. Going forward, we anticipate reducing our near-term commercial readiness expenses as we are assessing alternatives for the commercialization of Tommy Doobie sales, including potential U.S. or global partnerships. General and administrative expenses were $17 million in 2021 compared to $13.2 million in 2020. The increase was mainly due to a $2.6 million increase in professional services expenses, and a $1.2 million increase in ad count and related expenses. Finance expenses net were $2.6 million in 2021 compared to $0.6 million in 2020. The increase was primarily due to a $4.4 million interest expenses from our convertible notes offset by a $2.1 million capitalization and other non-cash income and a $0.3 million increase in interest income from cash management. Net loss in 2021 was $89.8 million compared to a net loss of $61.6 million in 2020. We continue to expect cash use for ongoing operating activity this year to range from $60 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. I will now turn the call back over to Julian.
spk04: Thank you, Shai. As we look ahead into what we have set out to accomplish in this year, 2022, I believe we are well-positioned to deliver on our mission of developing potentially curative cell therapies for patients with blood cancers, solid tumors, and other serious blood disorders. We look towards our full BLA submission for omodubacil, expected in the second quarter of this year, and the planned initiation of our Phase I-II multicentered gametacel-sponsored study for GDA201 in non-Hodgkin lymphoma. We are excited for the opportunity to continue leveraging our unique NAM-enabled platform across a broad range of cell therapies, and I look forward to providing updates throughout the year. Now we will open the call for questions. Operator?
spk02: Ladies and gentlemen, if you have a question or comment at this time, please press star then 1 on your telephone keypad. If your question has been answered or you wish to remove yourself from the queue, simply press the pound key. Again, if you have a question or comment at this time, please press star, then 1 on your telephone keypad. Our first question or comment comes from the line of Jonathan Miller from Evercore ISI. Your line is open.
spk09: Hey, guys. Thanks for taking my questions. First, maybe on GDA201, it feels like the language here seems a little bit similar to last update, and I just wondered if you had any additional color on your recent interactions with the agency and what your current thoughts are on ability to get that IND cleared. And then maybe secondly, I'll have to do the runway guidance reinstated, but just wanted to get some clarity. How much commercial prep is included in the current runway guidance, and and maybe how much clinical work for GDA 201 is included in that clinical guidance.
spk04: So let me begin by addressing the first part of your question. We haven't had any direct communication with the FDA. The FDA provided absolute clarity on what issues we needed to resolve. Most of those issues are resolved, and we are getting ready to resubmit the amendment to the IMD and we'll be announcing when the IND is accepted. Regarding runway guidance, let me turn it over to Michelle and Shai to talk about launch readiness and use of cache for support of GDA201. Michelle?
spk00: Thank you, Julian, and good morning, Jonathan. So, Jonathan, I'll start, and then I'll turn to Shai for the additional financials. So in regards to runway guidance, so for 2021, we were able to complete a lot of the key milestones for commercial preparation that led to the increase that Shai discussed for OPEX. So some of those included, you know, the market insights such as quantitative assessments from transplanters and feedback from payers, In addition, a very, very important milestone for us in 2021 was the readiness of Gametacel Assist. Although Gametacel Assist sits within the commercial organization, it plays a critical role in starting the chain of identity and monitoring the chain of identity and chain of custody. Although Amadubacil has a less stringent requirement, it is still an individualized therapy, and hence we need to demonstrate chain of identity, chain of custody. That key work for Gametacel Assist including building the team, developing the processes, and establishing the IT infrastructure was all completed in 2021. So what that allowed us to do in 2022, as we are assessing our strategic alternatives or potential strategic alternatives for launch, it allowed us to decrease the spend in regards to some of the commercial preparation because of the fact that so much had been done in 21. So let me turn to Shai to give us the actual financial details, and then Jonathan, we'll see if you have any follow-up questions.
spk06: Thank you, Michelle. So Jonathan, for your question, I would divide the answer into two. As Michelle alluded for the commercial, we did an internal robust process of prioritization our expense in 2022 in order to meet the cash one-way guidance. And I can tell you that all the critical activities will continue to be advanced. In addition, as we mentioned in the previous comment, we did reduce some of the spending, which are less critical for the next few months. As for the R&D GDA201 in particular, I can assure you that we continue to advance the GDA201. As part of the prioritization, we didn't reduce any of the GDA201. We continue to advance this program. including the potential initiation of trials later this year and first patient in.
spk02: Thanks so much, guys. My pleasure.
spk04: Thank you, John.
spk02: Thank you. Our next question or comment comes from the line of Ted Pinhoff from Piper Sandler. Your line is open.
spk07: Great. Thank you very much, and good morning, everybody. I want to dig in a little bit more to the considerations with respect to when we do this on marketing. To me, it seems like the greatest value would be retained if this is a therapy that you take to market yourselves. But can you give us a little bit more just in terms of kind of how you're assessing this? You know, could this be overseas deal where you keep the U.S.? Could this be a global deal, you know, give us maybe a sense for how and what kinds of options you're considering. Thanks.
spk04: So, Ted, thank you for your question, and good morning. As was mentioned in the script, we're assessing our strategic alternatives for Omidubasel, and that's driven by our desire to see as many patients as possible gain access to Omidubacill. So we are discussing regional deals, potential regional deals, as well as potentially U.S. deals with partners that may have a unique interest in hematology oncology franchise and that pairs up with their portfolio and already has significant infrastructure to be able to deliver Omidubacill to as many patients as possible. Our involvement will always be very intimate because we are the manufacturer, and as you know in cell therapies, the manufacturing facilities is critical to supporting any kind of commercial product. So we haven't made any choices yet, and we're starting, and certainly once the BLA is fully filed and accepted, that will open up avenues to conversations with interested parties.
spk07: Yep. Very helpful, Julian. I appreciate that addition, Collin. My pleasure.
spk02: Thank you. Our next question or comment comes from the line of Jason Butler from JMP Securities. Your line is open.
spk08: Hi. Thanks for taking the question. Just another one. Can you Just talk about plans for additional data presentations this year, including from the open-label extension or expanded access program. Thank you.
spk04: So thank you, Jason, for your question. Ronit mentioned during the prepared remarks that we have eight abstract TCT, but maybe, Ronit, you could perhaps highlight other venues where we might present the maturing data set for Omidubazil.
spk03: Absolutely, and thank you, Jason, for your question. So with TCT, as I mentioned, we'll be focusing on long-term follow-up information as well as patient-reported outcomes, health-related quality of life, additional immune reconstitution data, and some preclinical data as well, and some information on the value and cost of care. In terms of the expanded access program, we are continuing to recruit patients for the expanded access program, have not made a definitive decision about what the appropriate time would be to present that growing cohort of patients. But when there is a, I think, critical amount of information that's worth presenting, we certainly will do that either by the end of the year at a hematologic meeting or maybe next year at the transplant meeting.
spk08: Great. Thank you.
spk02: Thank you. Our next question or comment comes from the line of Gil Bloom from Needham & Company. Your line is open.
spk10: Good morning, everyone, and thanks for taking our questions. So this one's for Michelle. From your discussions with physicians, what feedback have you been getting regarding their interest in an FDA-approved transplant versus what they've been doing to date? The focus here being whether they have an opinion on something that is FDA-approved and kind of the feedback that you've heard so far.
spk00: Thank you, Gil, and good morning. We've done a lot of work speaking to physicians, and what is encouraging for them about amadubacil, which falls into the category of an FDA-approved therapy, is the following. So, you know, first off, gametacil will take on, you know, the responsibility around chain of identity and chain of custody. from the time of the, you know, patient selection for amadubacil all the way through the return of the therapy to the center. So that's an important responsibility, and that's something that, you know, comes with being an FDA-approved therapy. The other aspect is around the amadubacil itself. You know, so the NAM technology allows us to not only expand but enhance the cells. And, you know, some of the challenges associated with using other donor sources is, do you have an adequate number of cells? Does the donor potentially have some comorbidities that could potentially be affecting the donor ability? So the fact with Amadubacil that we will be sort of a FDA regulated therapy with sort of a defined threshold around release criteria, that is viewed as a positive. I will say consistently the feedback we hear from U.S. transplanters is that when they see the Amadubacil clinical data they see Amaduba Cell opportunity for their patients falling into two key categories. The first, from the physician's perspective, is the ability to improve outcomes as compared to donor sources that they're currently using. And then the second, that very critical one, of those patients who are deemed eligible for transplant but can't find a match, they see a very important opportunity for amaduba cell, given the fact we have a less stringent matching criteria. We demonstrated in our clinical study, we had approximately 40% of patients that were non-Caucasian in our clinical study. So those two key opportunities really do resonate consistently when we speak to physicians.
spk04: And if I may add, Michelle, You know, there's no question that having a quality-assured product with specifications and an FDA label clearly gives us the opportunity to engage with physicians and educate. And over time, this is an exercise in behavioral economics, if you will. If physicians find patients that are suitable for omodubacillin, and have a good experience, that will go a long way to encouraging them to continue to prescribe homo adubo cell. So we think that an FDA label for the first ever sponsored randomized phase three study is a significant game changer for the field of hematopoietic stem cell transplant.
spk10: Thank you, Michelle, and thank you, Julian, for taking our questions.
spk04: Thank you, Gil. Thank you, Gil.
spk02: Thank you. Again, ladies and gentlemen, if you have a question or comment at this time, please press star then 1 on your telephone keypad. Our next question or comment comes from the line of Matthew Cross from Alliance Global. Your line is open.
spk05: Hi, all. Good morning, and thanks for taking a couple of questions from me, both on the NK cell side. One, I wanted to ask regarding the carb reservation status for GDA201. I know you'd previously tested Omidubacill as both a cryopreserved and non-cryopreserved product. Since you're finalizing these IND requirements with the FDA for 201, I was just hoping to get a recap on kind of the clinical supply chain for that asset in terms of cold chain and what's required for that product in particular, given continuing supply issues at a broad level.
spk04: So let me take that on. The team has worked closely very diligently through 2021 to perfect and optimize the cryopreservation. We now have product instability with many months of stability maintained at liquid nitrogen. So the goal of this is to have an off-the-shelf product to enable a multicenter Phase I-II study and be able to recruit patients with a a single treatment with GDA201. So all of those activities have been attended to, and we're just finalizing the amendment to the IND to resubmit to the FDA and begin the clinical study in 2022. Got it.
spk05: Okay. Thanks, Julian. I appreciate the confirmation there. And then you had stated that you're looking to select a candidate for IND-enabling studies this year out of the genetically modified NK-cell program. But I just wanted to confirm whether I'm taking that statement overly literally. Are you looking at advancing one of these four programs to pre-IND this year and kind of tabling the rest for a bit? Or are all of these kind of making progress and maybe just one will become a front-runner during the year? Thanks.
spk04: So, Matt, thank you for your very important question. So right now, we have three candidates that are progressing. It's all data-driven. And my comments about GDA601 further allowed us to expand our collaboration with Dana-Farber to look at multiple myeloma cell lines as well as fresh patient isolates, patients who have been heavily pretreated. So the idea is progress all three but select one candidate for IMD-enabling studies, and then subsequently continue to make progress on the other candidates. Again, it'll all be data-driven, and we'll select the candidate with the most compelling data going forward.
spk05: Great. Okay. Thanks for the clarification there as well. I appreciate all the answers.
spk02: Thank you. I'm sure no additional questions in the queue at this time. I'd like to turn the conference back over to Julian Adams for any closing remarks.
spk04: Thank you, Operator, and thank you, everyone, for joining us on today's call. We look forward to engaging with you, and have a good day, everyone, and see you next quarter.
spk02: Ladies and gentlemen, thank you for participating in today's conference. This concludes the program. You may now disconnect. Everyone have a wonderful day.
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